Market Place
This week saw a couple of developments in the lucrative US market for glucagon-like peptide-1 (GLP-1) drugs that treat diabetes and obesity. Novo Nordisk is investing US$ 4.1 billion to develop a new facility in North Carolina to boost the supply of Ozempic and Wegovy. And Israel’s Teva Pharmaceuticals has launched a generic version of Novo Nordisk’s Victoza to treat type 2 diabetes.In approvals, the US Food and Drug Administration (FDA) has approved Verona Pharma’s drug for chronic obstructive pulmonary disease (COPD), a chronic lung disease. The agency has expanded the use of Sarepta Therapeutics’ gene therapy for Duchenne muscular dystrophy (DMD) in patients aged four and above who cannot walk, despite the drug failing in its late-stage trial. Additionally, FDA granted an accelerated approval to BMS’s Krazati, to be used in combination with cetuximab, as a targeted treatment option for adult patients with KRASG12C-mutated locally advanced or metastatic colorectal cancer.In late-stage trials, Gilead’s twice yearly HIV prevention drug proved to be superior to its daily pill Truvada, which could make it a game changer in the pre-exposure prophylaxis (PrEP) space. AstraZeneca’s blockbuster drug Imfinzi meaningfully improved survival in patients with muscle-invasive bladder cancer. And Merck KGaA has had to scrap a phase 3 trial after its once promising candidate xevinapant failed to show significant survival benefits in patients with locally advanced head and neck cancer.Novo invests US$ 4.1 billion
in new facility in US to boost Ozempic, Wegovy supplyNovo Nordisk is investing US$ 4.1 billion to develop a new manufacturing facility in Clayton, North Carolina (US). The funds will be
used to create a 1.4 million square foot
facility that
fills Ozempic and Wegovy (both semaglutide) injector pens. The Danish
drugmaker already operates three facilities at the site. This new 56-acre
facility is expected to add 1,000 new jobs to the 2,500 already employed at the
other three facilities.Teva launches first GLP-1
generic in US: Teva Pharmaceuticals has launched a generic version of Novo Nordisk’s Victoza (liraglutide) to treat type 2 diabetes
patients, rendering it the first generic for a GLP-1 drug in the
United States.FDA okays Verona’s COPD therapy; AbbVie-Genmab’s Epkinly bags second approvalFDA
has approved a therapy for COPD — Ohtuvayre — from Verona Pharma. Ohtuvayre is a new, inhaled non-steroidal,
maintenance treatment for COPD in adult patients. The chronic condition causes
restricted airflow and breathing problems. According to the company, Ohtuvayre is the first inhaled product with a novel
mechanism of action available for the maintenance treatment of COPD in more
than 20 years.Epkinly okayed for follicular
lymphoma: After bagging an FDA approval last year for diffuse large B-cell
lymphoma (DLBCL), AbbVie and Genmab’s Epkinly (epcoritamab-bysp) has now bagged an accelerated approval in patients with relapsed or refractory follicular lymphoma (FL) who have received two or more prior treatments.First drug for excessive
underarm sweating okayed: FDA has approved the first drug to treat primary
axillary hyperhidrosis (excessive underarm sweating). To be sold as a gel, Botanix Pharmaceuticals’ Sofdra (sofpironium) is a new chemical entity that presents a safe and effective solution to
patients who suffer from this socially challenging medical condition, the
company said.Gilead’s twice yearly HIV prevention drug found to be superior to daily pill TruvadaIn a late-stage study,
Gilead’s long-acting injection has proven to be more effective in preventing HIV infection in women compared to the daily pill Truvada (emtricitabine/tenofovir). This is the first time
that an HIV pre-exposure prophylaxis (PrEP) has shown zero infections in a
phase 3 trial, the drugmaker said. Dosed just twice a year, lenacapavir could be a game-changer in HIV
prevention.Astra’s Imfinzi chalks up win in treating bladder cancer: AstraZeneca’s Imfinzi (durvalumab) meaningfully improved event-free survival and
overall survival in patients with muscle-invasive bladder cancer (MIBC) in a late-stage trial. In the study, Imfinzi was
used in combination with chemotherapy before surgery, and as a monotherapy
after, and was compared with pre-surgical chemotherapy alone.Merck KGaA scraps trial after
failure in head, neck cancer: Merck KGaA said its candidate xevinapant has failed in a
late-stage trial to show significant survival
benefits in
patients with locally advanced head and neck cancer. An
independent data monitoring committee said xevinapant is unlikely to meet the primary endpoint of the study and the German drugmaker is discontinuing it. Xevinapant was seen as a promising drug and Merck had paid €188 million (about US$ 226 million) in upfront cash to acquire its global rights from Debiopharm International.FDA expands use of Sarepta’s Duchenne gene therapy despite late-stage failureFDA
has granted accelerated approval for the use of Sarepta Therapeutics’ gene therapy
for Duchenne muscular dystrophy (DMD) in patients aged four and above who cannot walk. The agency
okayed Elevidys (delandistrogene
moxeparvovec) for this group despite it missing its
primary goals of a late-stage trial. FDA also granted Elevidys a traditional
approval for DMD in patients four years of age and above who can walk.Argenx’s Vyvgart Hytrulo okayed for autoimmune disease: FDA has
approved Argenx’s Vyvgart Hytrulo for chronic inflammatory demyelinating polyneuropathy (CIDP), making it a new treatment option for patients with this rare
and debilitating neuromuscular disorder. This marks the first drug with a
novel, precision mechanism of action in over 30 years for CIDP, Argenx said.BMS’ Krazati okayed for colorectal cancer: FDA has granted Krazati (adagrasib) an accelerated approval, when used in combination
with cetuximab as a targeted treatment
option for adult patients with KRASG12C-mutated locally advanced or metastatic
colorectal cancer. This is the second approval for Krazati, which works by
targeting a mutated form of the gene known as KRAS. It was previously approved
to treat KRASG12C-mutated locally advanced or metastatic non-small cell lung
cancer.Lilly, OpenAI join hands to
fight antimicrobial resistance; Glenmark recalls drugEli Lilly has partnered OpenAI to leverage its generative
AI to invent novel antimicrobials that can be used to treat
drug-resistant bacteria. Antimicrobial resistance is one of the top public
health and development threats across the world.Glenmark recalls 114 batches
of potassium drug: Glenmark has recalled 114 batches in the US of 750 mg potassium chloride extended-release capsules
because of failed dissolution. As a result, the capsules
may cause high potassium levels, also known as hyperkalemia, which can result
in irregular heartbeat that can lead to cardiac arrest, the company said. The recall is voluntary and Glenmark hasn’t received any reports of serious side effects yet.
Impressions: 631
https://www.pharmacompass.com/radio-compass-phisper/novo-invests-us-4-1-bn-to-boost-wegovy-ozempic-supply-verona-s-copd-therapy-bags-fda-nod
#Phispers by PharmaCompass
27 Jun 2024
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Merck wins FDA nod for pneumococcal vaccine; Roche partners Ascidian to develop novel gene therapies
This week saw the US Food and Drug Administration (FDA) approve Merck’s next-generation pneumococcal vaccine for adults. The agency also granted accelerated approval to Bristol-Myers Squibb’s Augtyro for adult and pediatric patients with solid tumors.In deals, Roche has promised up to US$ 1.8 billion to Boston-based startup Ascidian Therapeutics to discover and develop novel gene therapies for difficult-to-treat neurological diseases. The week also saw three deals involving Chinese companies, including AbbVie’s US$ 1.7 billion deal with China’s FutureGen Biopharmaceutical to develop and commercialize a promising treatment for inflammatory bowel disease (IBD).In trials, Intra-Cellular’s Caplyta scored another remarkable late-stage win for treating major depressive disorder (MDD). And a large study conducted by Stanford Medicine found the risk of CAR-T cell therapies causing secondary cancers to be low.There were some misses too. Takeda’s seizure drug failed to meet its endpoints in back-to-back late-stage trials. And AstraZeneca’s Truqap failed to prolong the life of patients with hard-to-treat breast cancer.FDA greenlights Merck’s next-generation pneumococcal vaccine for adultsFDA has approved
Merck’s next-generation vaccine designed to protect adults
from pneumococcus bacteria that causes serious illnesses and pneumonia. The
jab, known as Capvaxive, helped produce an immune response against all 21
variations (serotypes) of the bacteria that it targeted. These 21 strains
account for about 85 percent of invasive pneumococcal disease cases in adults
aged 65 and above. Pfizer’s Prevnar 20, the
current market leader, targets
strains that account for about 51 percent of cases in the said age group.
Analysts see Capvaxive as a key growth driver for Merck.BMS’ Augtyro bags tumor-agnostic nod: FDA has granted accelerated approval
to Augtyro (repotrectinib) for adult and
pediatric patients (over 12 years) with solid tumors that have a neurotrophic
tyrosine receptor kinase (NTRK) gene fusion, regardless
of where they occur in the body. This makes BMS’ therapy the
only FDA-approved treatment option for such tumors.Roche inks potential US$ 1.8 bn deal with Ascidian to develop novel gene
therapiesRoche has promised up to US$ 1.8 billion to Boston-based
startup Ascidian Therapeutics to discover and develop novel gene therapies for difficult-to-treat neurological diseases. Ascidian gets an initial payment of US$ 42 million. The startup’s RNA exon editing platform is designed to advance the therapeutic possibilities of RNA medicine and treat diseases not addressed by today's gene editing technologies.Sanofi, Belharra in immunology deal: Sanofi is collaborating with Belharra Therapeutics in a deal worth up to US$ 700 million including US$ 40 million in upfront and near-term payments. The collaboration will leverage Belharra’s non-covalent chemoproteomics platform to advance the discovery of novel small molecule therapeutics for immunological diseases.AbbVie to pay up to US$ 1.7 bn to China’s FutureGen for next-gen IBD therapyAbbVie has agreed to pay China’s FutureGen Biopharmaceutical up to US$ 1.56 billion, including an upfront payment of US$ 150 million, to develop and commercialize a next generation treatment for inflammatory bowel disease (IBD) — FG-M701. Currently in preclinical development, FG-M701 targets TL1A antibody (a clinically validated target in IBD).Takeda to license Ascentage’s leukemia drug: Japanese drugmaker Takeda has signed an option agreement with China’s Ascentage Pharma for an exclusive
license to its promising candidate olverembatinib. This experimental
drug is being developed to treat chronic
myeloid leukemia (CML) and other hematological cancers. If exercised, the
option would allow Takeda to license global rights to develop and commercialize
olverembatinib in all territories outside of mainland China, Hong Kong, Macau,
Taiwan and Russia. Takeda has paid Ascentage US$ 100 million upfront,
and the Chinese drugmaker stands to receive an additional US$ 1.2 billion in milestone payments.Day One in biobucks deal with MabCare: Day One Biopharmaceuticals has entered into a licensing agreement with China-based MabCare Therapeutics for an experimental novel antibody drug conjugate — MTX-13. The biobucks deal for MTX-13 is worth up
to US$ 1.2 billion, if the drug meets all milestones.
The drug is being developed for multiple kinds of adult and pediatric cancers.Intra-Cellular scores late-stage win for depression drug CaplytaIntra-Cellular’s antipsychotic drug
Caplyta (lumateperone) has scored another remarkable late-stage win for treating MDD. After six weeks on the 42 mg daily dose, US participants on Caplyta as an adjunctive therapy experienced a 4.5-point reduction on the Montgomery-Asberg Depression Rating Scale (MADRS) compared to the placebo. The results were notably similar to a previous study conducted worldwide.The New York City-based biopharma said it is confident lumateperone will become the “drug of choice for patients suffering from MDD who are having an inadequate response to antidepressant therapy.” If approved, Caplyta will compete with AbbVie’s blockbuster
antipsychotic drug Vraylar (cariprazine). Like Vraylar,
Caplyta is approved for schizophrenia and bipolar disorder, but MDD is reportedly the most lucrative
indication. Analysts predict an additional US$ 1 billion in annual peak sales from MDD by 2033.Takeda’s seizure drug fails to meet main goals in back-to-back phase 3 trialsTakeda’s soticlestat being
investigated in two epileptic disorders has failed in phase 3 trials,
albeit “narrowly”. The drug was being tested in combination with the standard of care for adults and kids with Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS) – rare and severe types of epilepsy that cause frequent, long-lasting seizures that usually start in infancy or early childhood. Takeda paid US$ 196 million to Ovid Therapeutics for the global
rights to soticlestat. It has promised an additional US$ 660 million in
milestone payments.Astra’s Truqap fails trial for hard-to-treat breast cancer: AstraZeneca’s Truqap (capivasertib), in combination with the common
chemotherapy paclitaxel, failed to help patients with hard-to-treat breast cancer live longer. A late-stage study was testing
Truqap in volunteers with inoperable triple-negative breast cancer (TNBC) that
had spread. TNBC remains among the most challenging forms of disease to treat.Study finds risk of secondary cancers after CAR-T cell therapy to be lowA large
study conducted by Stanford Medicine has found the risk
of CAR-T cell therapies causing secondary cancers to be low. According to
results published in The New England Journal of Medicine, a 724-patient study saw about 6.5 percent of patients get secondary
cancers over a median follow-up period of three years.Zentalis’ cancer drug put on partial clinical hold: FDA has placed three studies of Zentalis Pharma’s cancer drug — azenosertib — on partial clinical hold. This action was taken after deaths of two participants in a mid-stage study. Azenosertib was being tested against solid tumors in an early-stage study and in patients with a type of ovarian and uterine cancer in two mid-stage studies.
Impressions: 817
https://www.pharmacompass.com/radio-compass-phisper/merck-wins-fda-nod-for-pneumococcal-vaccine-roche-partners-ascidian-to-develop-novel-gene-therapies
#Phispers by PharmaCompass
20 Jun 2024
The US Food and Drug Administration’s (FDA) advisory committee has voted unanimously in favor of the benefits of Eli Lilly’s Alzheimer’s drug donanemab outweighing its risks. FDA has also granted accelerated approval to Ipsen’s Iqirvo to treat a rare liver disease (primary biliary cholangitis) that has not seen a new therapy approved in nearly a decade. Moreover, FDA handed Geron Corporation its first approval for blood disorder drug Rytelo.In vaccine news, Moderna’s combination vaccine against influenza and Covid elicited a higher immune response compared to separate shots in people aged 50 and above. Also, adults aged 50 to 59 years now have an RSV vaccine after FDA expanded the use of GSK’s Arexvy to include this age-group.In a blow to millions who are affected by long Covid, Pfizer’s Paxlovid did not appear to improve its symptoms as was hoped, according to a study undertaken by Stanford University. In yet another blow to Pfizer, its experimental gene therapy — fordadistrogene movaparvovec — failed to improve motion function in patients with Duchenne muscular dystrophy (DMD) in a late-stage trial.In regulatory news, FDA has chided China’s Jiangsu Hengrui in a Form 483 after employees destroyed documents during an inspection. And Johnson & Johnson has reached a US$ 700 million settlement with 42 US states and Washington, DC, over allegations that it misled consumers into believing its talc products were safe.Lilly’s Alzheimer’s drug donanemab wins unanimous backing of FDA’s expert panelAn FDA advisory committee has voted unanimously in favor of the benefits of
Eli Lilly’s Alzheimer’s drug donanemab outweighing its risks. Alzheimer’s disease afflicts over six million people in the US and has no cure. However, donanemab modestly slowed cognitive decline in early-stage patients but came with noteworthy risks including swelling and bleeding in the brain. The Alzheimer’s Association has welcomed the unanimous voting by the
panel in favor of the drug.Ipsen’s Iqirvo bags FDA nod to treat rare liver disease; Geron gets first FDA approvalFDA has granted accelerated approval to Ipsen’s Iqirvo (elafibranor) to treat the rare liver disease known as primary biliary cholangitis (PBC). The decision renders Ipsen’s first-in-class peroxisome proliferator-activated receptor (PPAR) agonist the first new medicine approved in nearly a decade for the treatment of PBC. Iqirvo is taken orally once a day and is estimated to cost US$ 11,500 a month. PBC affects about 100,000 people in the US and can cause liver failure. It mostly affects women aged 30 to 60 years.Iqirvo was discovered and developed by Genfit to treat the fatty liver
disease, now known as metabolic dysfunction-associated steatohepatitis (MASH).
But after flopping in phase 3 trial, it joined the
graveyard of MASH failures. Ipsen then licensed it from Genfit for up to € 480 million (US$ 515 million) to treat PBC.Geron’s maiden approval: FDA has signed off on Geron’s Rytelo (imetelstat) for treating transfusion-dependent anemia in patients with low- to intermediate-risk myelodysplastic syndromes (MDS), a group of blood cancers. This is
the first FDA approval for Geron since its inception in 1990. The use of BMS’ Reblozyl (luspatercept) was expanded last year by
the FDA to treat the same disease indication. However, this is reportedly an underserved population.Moderna’s two-in-one Covid-flu vaccine superior to individual shots, says studyModerna said mRNA-1083,
an investigational combination vaccine against influenza and Covid,
elicited a higher immune
response compared to separate shots in people aged 50 and
over. A late-stage study saw the messenger RNA technology-based combination
generate more antibodies than currently marketed flu vaccines and Moderna's
Spikevax.FDA okays GSK’s RSV vaccine for adults aged 50 to 59: FDA has expanded GSK’s respiratory syncytial virus
(RSV) vaccine to be administered in adults between the ages of 50 and 59 years. This makes Arexvy the only
RSV shot endorsed for that age group and is expected to add 13 million
individuals to its eligibility pool. Arexvy has been dominating the US market
since its launch in 2023, outperforming sales of its rival Pfizer’s Abrysvo.Pfizer’s Paxlovid fails long Covid test, its DMD gene therapy flunks phase 3 studyIn a blow to millions affected by long Covid, Pfizer’s Paxlovid did not appear to improve the symptoms as was hoped. Pfizer’s antiviral treatment was shown to be safe in a 15-day study conducted by Stanford University. However, it didn’t lower select symptoms of the syndrome. Long Covid symptoms include fatigue, brain fog and the inability to exercise and scientists don’t yet know exactly what causes it.In yet another blow to Pfizer, its experimental gene therapy fordadistrogene movaparvovec failed to improve motion function in patients with DMD in a late-stage trial. The therapy did not show a significant improvement compared to a placebo with regard to the trial’s primary and secondary endpoints.China’s Jiangsu Hengrui issued scathing Form 483 after staff destroys documentsFDA has admonished China’s Jiangsu Hengrui
Pharmaceuticals after an employee “diverted” inspectors while colleagues tore up and discarded documents. The employee called ahead as soon as FDA sought access to the waste area and then led inspectors down a long route. On arriving, FDA observed “personnel in a rapid-like manner placing documents in the waste bin, along with approximately 2 to 3 other individuals watching,” a Form 483 said. FDA also “observed black mold like growth on the floor below and around the condenser, which are approximately a foot from released finished drug products.” Following the January inspection at the manufacturing site in the Chinese city of Lianvungang (Jiangsu province), FDA issued a Form 483 with eight observations.J&J to pay US$ 700 mn to US states over allegations of misleading talc customersJohnson & Johnson has reached a US$ 700 million settlement with 42 US
states and Washington, DC, over allegations that it misled consumers into believing its talc products were safe. This puts to bed an investigation into its marketing of baby powder and other talc-based products, which J&J had sold for over 100 years. The products in question were alleged to have caused cancer. J&J still faces tens of thousands of lawsuits related to its talc.
Impressions: 578
https://www.pharmacompass.com/radio-compass-phisper/fda-panel-backs-lilly-s-alzheimer-s-drug-agency-approves-ipsen-s-med-to-treat-rare-liver-disease
#Phispers by PharmaCompass
13 Jun 2024
This week saw the American Society of Clinical Oncology (ASCO) annual meeting in Chicago where cancer researchers presented detailed findings from hundreds of clinical trials. Central to AstraZeneca’s ambitious plan to clock US$ 80 billion in annual revenue by 2030 is its cancer drugs. Trial results for Astra’s top-selling cancer drug Tagrisso as a maintenance therapy for certain lung cancer patients and for Enhertu in breast cancer suggested they could become the new standard of care. Meanwhile, in the
treatment of the currently incurable multiple myeloma (MM), GSK's Blenrep cut
the risk of disease progression or death by almost half compared to
standard-of-care treatments. Sanofi boasted a first-in-class win wherein
Sarclisa as a combination therapy bested standard of care in newly diagnosed MM
patients.Among the data presented at ASCO, the world’s first personalized mRNA cancer vaccine for skin cancer has excited doctors after Moderna’s mRNA-4157 (V940) plus Merck’s Keytruda halved the risk of patients dying or the disease returning. Also, Moderna’s mRESVIA has become the first mRNA RSV vaccine approved by the US Food and Drug Administration (FDA).In what came as a disappointment for advocates of psychedelic drugs for medical use, an FDA advisory panel has voted against the first MDMA treatment for post-traumatic stress disorder (PTSD) amid concerns about flawed trials.ASCO: Astra’s late-stage Tagrisso data in lung cancer trial gets standing ovationData presented at ASCO showed that AstraZeneca’s Tagrisso (osimertinib) reduced the risk of disease progression or
death by a whopping 84 percent in patients whose non-small cell
lung cancer (NSCLC) had an EGFR mutation and couldn’t be surgically removed. Patients on Tagrisso saw an average of 39.1 months without disease progression
compared to 5.6 months in the placebo group. The late-stage results were met
with a standing ovation as experts said the treatment could become the standard of care for one of the leading causes of cancer death.Imfinzi boosts
lung cancer survival: The
British-Swedish drugmaker said its human monoclonal antibody Imfinzi (durvalumab) is the first and only immunotherapy to show survival
benefit in limited-stage small cell lung cancer (LS-SCLC), in a global
late-stage trial. Imfinzi reduced the risk of death by 27 percent versus the
placebo. At the three-year mark, 57 percent of Imfinzi treated patients were
alive.Breast cancer
drug Enhertu shows strong results: AstraZeneca and Daiichi Sankyo’s Enhertu delayed the progression of breast cancer by about five months in women with low levels of HER2 protein whose cancer
progressed following endocrine therapy. The findings could significantly
increase the range of breast cancer patients that may benefit from the
antibody-drug-conjugate (ADC).GSK’s blood cancer drug eyes US comeback after cutting risk of death by almost half in phase 3 studyGSK's MM therapy Blenrep (belantamab
mafodotin) has cut the risk of disease progression or death
by almost half compared to standard-of-care
treatments, data touted at ASCO showed. After a year, 71 percent of patients on Blenrep, in
combination with pomalidomide plus dexamethasone (PomDex), were alive and saw no
worsening of their disease compared to 51 percent of patients on Takeda’s Velcade (bortezomib) plus PomDex. The once-failed ADC was pulled from the lucrative
US market in 2022, but the results could signal a comeback for Blenrep.Sanofi says
Sarclisa combo beat standard of care in phase 3: Sanofi's Sarclisa, combined with standard
care, achieved a 40 percent reduction in the risk of
progression or death compared to standard care alone in newly diagnosed multiple myeloma patients ineligible for a stem cell transplant. Takeda’s Velcade (bortezomib), BMS’ Revlimid (lenalidomide) and the steroid dexamethasone (VRd) is the current standard of care. Sarclisa is the first anti-CD38 monoclonal antibody to significantly improve PFS in combination with VRd, the French drugmaker said.Moderna’s mRESVIA becomes first mRNA RSV vaccine approved by FDAFDA approved Moderna’s mRESVIA, an mRNA respiratory
syncytial virus (RSV) vaccine, to protect adults aged 60 years and older from lower
respiratory tract disease caused by RSV infection. This is the first messenger RNA-based (mRNA) vaccine not for Covid to be approved in the US and is seen as a much-needed second product for the Boston-based biotech.Moderna’s mRNA cancer vaccine plus Keytruda cuts risk of death by nearly half:
Moderna and Merck presented three-year data on their investigational vaccine for patients with the
most deadly form of skin cancer, in combination with the blockbuster Keytruda (pembrolizumab). A mid-stage trial showed that
after 2.5 years, 75 percent of melanoma patients on the combination had
recurrence-free survival, compared with 55.6 percent on Keytruda alone.
Furthermore, at 34.9 months, mRNA-4157 (V940) in combination with Keytruda
reduced the risk of recurrence or death by 49 percent compared to Keytruda alone in these patientsSummit, Akeso's
antibody bests Keytruda in head-to-head phase 3 trial for NSCLC:
Ivonescimab met its primary endpoint of significantly improving
PFS over the world’s bestselling drug Keytruda. Ivonescimab
received marketing authorization in China in May and is licensed to Summit Therapeutics in the US, Canada, Europe,
and Japan. Keytruda is a PD-1 inhibitor, while ivonescimab is a bispecific
antibody targeting both PD-1 and vascular endothelial growth factor (VEGF)
proteins.FDA panel votes against first MDMA
treatment for PTSD amid concerns about flawed trialsAn FDA advisory panel has voted 10 against one, saying the benefits of using the party drug MDMA to treat PTSD do not
outweigh the risks. Data submitted by Lykos Therapeutics was reportedly marred by inconsistencies, poor study design and allegations of misconduct. While FDA is not required to follow the panel’s advice, a final decision is expected by August
11. There hasn't been a new treatment for PTSD in over two decades
and this marked the first time FDA advisers considered a Schedule I psychedelic for medical use.FDA's Operation Warp Speed for rare
disease treatments gets first candidatesSelected companies have joined the FDA's Operation Warp Speed for
rare disease treatments, known as the Support for Clinical Trials Advancing
Rare disease Therapeutics (START) Pilot Program. This initiative facilitates
closer collaboration between early-stage participants and FDA staff. Grace Science's GS-100 for NGLY1 deficiency, Denali's DNL126 for MPS IIIA (Sanfilippo syndrome type A), Larimar's nomlabofusp for Friedreich’s ataxia, and Neurogene's NGN-401 for Rett syndrome were among the chosen therapies.
Impressions: 651
https://www.pharmacompass.com/radio-compass-phisper/astra-s-cancer-drugs-impress-at-asco-moderna-s-mresvia-becomes-first-mrna-rsv-vaccine-approved-by-fda
#Phispers by PharmaCompass
06 Jun 2024
This week’s Phispers comes packed with news of investments, acquisitions, deals and trials. Eli Lilly has more than doubled its investment at its Lebanon (Indiana) site in the US by investing an additional US$ 5.3 billion. This investment will enhance Lilly’s capacity to manufacture the active pharmaceutical ingredient (API) used in its obesity and type 2 diabetes drugs — Zepbound and Mounjaro.In deals, Merck announced it is buying clinical-stage ophthalmology biotech EyeBio for US$ 1.3 billion. Johnson & Johnson has scooped up the rights to an eczema antibody for US$ 1.25 billion. Japanese conglomerate Asahi Kasei said it is buying Sweden’s Calliditas for US$ 1.1 billion. And, Takeda has inked a potential US$ 1.2 billion deal with Shanghai-based Degron Therapeutics to discover and develop a new class of drugs, known as molecular glue degraders (MGDs).The US Food and Drug Administration (FDA) has approved a
once-daily treatment to treat attention deficit hyperactivity disorder (ADHD) in patients who are six years of age or older. And, an FDA advisory panel has voted against the benefits of Novo Nordisk’s once-weekly Awiqli (insulin icodec) outweighing its risks.At the European Renal Association (ERA) congress, Novo Nordisk presented detailed results that showed Ozempic reduced the risk of death due to kidney disease in people with diabetes. The ERA congress also saw Novartis tout two promising late-stage readouts for rare kidney disease treatments. And, preliminary results from a confirmatory trial showed NS Pharma’s Viltepso failed to reach its primary endpoint after it was given FDA’s accelerated approval to treat Duchenne muscular dystrophy.Lilly to invest additional
US$ 5.3 bn in US site to boost Zepbound, Mounjaro supplyThe battle to gain a larger share of the
obesity-diabetes pie just got fiercer. Eli Lilly has more than doubled its investment at
its Lebanon (Indiana, US) manufacturing site with a new US$ 5.3 billion commitment. This raises Lilly’s investment at this site to US$ 9 billion, up from US$ 3.7 billion. This expansion will enhance Lilly’s capacity to manufacture the API — tirzepatide — for its Zepbound and Mounjaro injections so that more adults with chronic diseases like obesity and type 2 diabetes may benefit from these important treatments, the company said.Merck set to buy
clinical-stage ophthalmology biotech EyeBio for US$ 1.3 bnMerck has announced it is buying ophthalmology
biotechnology firm Eyebiotech Limited (EyeBio) for US$ 1.3 billion. The
deal also includes US$ 1.7 billion in additional milestone payments. London-headquartered EyeBio’s lead candidate Restoret has finished early to mid-stage
testing for diabetic macular edema (DME)
and neovascular age-related macular degeneration (NVAMD).J&J scoops up rights to eczema antibody for US$ 1.25 bn: Johnson & Johnson has agreed to buy the rights to an experimental skin disorder treatment from Numab Therapeutics for around US$ 1.25 billion. The therapy — NM26 — is phase 2-ready and targets two clinically proven pathways in atopic dermatitis (AD), commonly known as eczema. Last week, J&J had acquired Proteologix for US$ 850 million, which was also to
help it address key unmet needs for AD patients.Takeda inks potential US$
1.2bn deal with Degron; Asahi Kasei to buy CalliditasJapan’s Takeda has inked a deal with Shanghai-headquartered
Degron Therapeutics to discover and develop novel molecular glue degraders
(MGDs) for multiple targets in oncology, neuroscience, and inflammation.
The deal could be valued at around US$ 1.2 billion. MGDs are an emerging class
of potentially therapeutic compounds touted as exciting opportunities for novel drug discovery.Meanwhile, Japanese conglomerate — Asahi Kasei — said it has offered to buy out Swedish drugmaker
Calliditas Therapeutics for SEK 11.1 billion (US$ 1.1 billion) as the former seeks to become a global
player in the pharma industry. Calliditas focuses on novel treatments for
orphan indications and patients with unmet needs.FDA okays Tris Pharma’s once-daily, non-stimulant pediatric ADHD drugFDA has okayed Onyda XR (clonidine hydrochloride), a once-a-day treatment for attention deficit
hyperactivity disorder (ADHD) for patients six years and older. This makes it the first
liquid non-stimulant ADHD drug approved in the US. It is expected to be
available in pharmacies in the second half of 2024. FDA panel votes against Novo’s weekly insulin for type 1 diabetesFDA’s advisory committee on endocrinologic and metabolic drugs has voted seven to four against the benefits of Novo Nordisk’s once-weekly Awiqli (insulin
icodec). The panel said the benefits of Awiqli do not outweigh its risks. The Danish drugmaker was
aiming to bring to market the first weekly insulin product, which would lower
the treatment burden for diabetes patients. An increased risk of low blood sugar was
flagged by the advisors in patients with type 1 diabetes.Ozempic impresses in
late-stage trial for kidney disease: Detailed results presented at the ERA congress and published in the New England Journal of Medicine showed Ozempic (semaglutide) lowered the risk of death in type 2 diabetes patients
with chronic kidney disease. A late-stage trial with 3,533 people showed weekly
semaglutide injections reduced kidney failure and death due to kidney
complication by 24 percent.Novartis tees up two rare
kidney disease therapy approvals after late-stage winsThe ERA congress also saw Novartis tout two promising late-stage readouts for rare kidney disease treatments as it tees up two potential approvals in
the renal therapy space. Interim results showed the Swiss drugmaker’s experimental drug atrasentan, along with supportive care, reduced proteinuria (elevated protein in the urine) by 36.1 percent in patients with IgAN. Novartis plans on applying for FDA’s approval in the first half of this year.The
other drug is Fabhalta (iptacopan), which was being trialed in patients with the ultra-rare kidney
disease C3 glomerulopathy (C3G). In combination with supportive care, Fabhalta
lowered proteinuria by 35.1 percent at six months. C3G currently has no
approved therapies.NS Pharma’s DMD drug fails confirmatory trial: Preliminary results from a confirmatory trial show NS Pharma’s Viltepso (viltolarsen) has failed to reach its primary endpoint after it was given FDA’s accelerated approval in August 2020 to treat Duchenne muscular dystrophy (DMD). However, NS Pharma remains confident in viltolarsen and said it is “conducting further detailed data analyses”.Four deaths mar J&J’s prostate cancer trial: Johnson & Johnson’s experimental prostate cancer drug that uses a rare
radioactive particle called actinium-225 has helped three patients by reducing their tumors
significantly. However, those results were marred by the death of four patients in the early-stage study. The first-in-human study concluded that “JNJ-6420 elicited profound and durable biochemical and radiographic responses.” However, 61 percent of the participants experienced grade 3 or higher treatment-emergent adverse events.
Impressions: 1094
https://www.pharmacompass.com/radio-compass-phisper/lilly-to-invest-additional-us-5-3-bn-to-boost-obesity-drug-supply-fda-panel-votes-against-novo-s-weekly-insulin
#Phispers by PharmaCompass
30 May 2024
In this week’s news, AstraZeneca has laid out an ambitious plan to achieve US$ 80 billion in revenue by 2030. The drugmaker also announced an investment of US$ 1.5 billion to set up an antibody drug conjugates (ADCs) plant in Singapore.In news from clinical trials, Eli Lilly’s once-a-week insulin shot — efsitora — scored two late-stage wins. GSK’s long-acting experimental asthma drug depemokimab reduced asthma attacks in two late-stage trials. And Bayer’s non-hormonal menopause drug significantly reduced hot flashes in two phase 3 studies.In approvals, the US Food and Drug Administration (FDA) has granted accelerated approval to Amgen’s targeted immunotherapy Imdelltra for adults in the advanced stages of small cell lung cancer that has proved hard to treat or has worsened despite chemotherapy. FDA also okayed the first two biosimilars for Regeneron’s blockbuster eye drug Eylea.A group of cancer victims have sued Johnson & Johnson accusing it of fraud in its plans to settle the talc lawsuits.In deals, Biogen is acquiring Human Immunology Biosciences (HI-Bio) for up to US$ 1.8 billion, Johnson & Johnson is buying California-based Proteologix for US$ 850 million and Eli Lilly has inked a potential US$ 1.1 billion collaboration with Aktis Oncology.Astra sets 2030 revenue
target at US$ 80 bn, invests US$ 1.5 bn to build ADC facilityAstraZeneca has laid out an ambitious plan to deliver US$ 80 billion in total revenue by 2030. That will be a 75 percent leap from the US$ 45.8 billion in revenue it posted in 2023. Astra expects to launch 20 new medicines by 2030. Astra’s targets are driven by its cancer portfolio, as also its biopharmaceuticals and rare disease portfolios. The drugmaker’s oncology portfolio had brought in US$ 17.1 billion last year.The update comes on the heels of Astra’s investment of US$ 1.5 billion in building a manufacturing facility in Singapore for ADCs to
enhance the global supply of its ADC portfolio.Meanwhile, Pfizer has launched a new multi-year program to cut costs by around US$ 1.5
billion by 2027-end. Last year, it had announced a US$ 4 billion cost cutting
plan.Lilly’s weekly insulin proves to be as effective as daily doses in two late-stage trialsEli Lilly’s once-weekly insulin jab, efsitora, showed blood sugar reduction consistent with the daily
insulins widely used today by patients with type
2 diabetes. Efsitora met its primary endpoints in two phase 3 trials. Lilly is
in a race with Novo Nordisk to bring to market weekly
injections with long-acting insulins, which would lower the treatment burden
for diabetes patients.There was disappointing news from its rival Novo on its long-acting weekly insulin — Awiqli (insulin icodec). FDA said the use of this med came with an increased risk
of low blood sugar in patients with type 1 diabetes, who are more
insulin-dependent. An FDA panel will meet on May 24 to review Novo’s application seeking approval for this once-weekly insulin.GSK sells remaining stake in
Haleon; scores trial wins for asthma drug: GSK has raised £ 1.25 billion (US$ 1.52 billion) by
dropping the remainder of its stake (4.2 percent) in Haleon. It also touted two late-stage wins for its experimental asthma drug
depemokimab. The med reduced asthma attacks over a year at a rate that
was clinically significant.Bayer’s menopause drug reduces hot flashes in trials: Bayer said its non-hormonal drug, elinzanetant, significantly reduced debilitating bursts of body heat (hot flashes) associated with menopause. This tees it up to become
only the second non-hormonal drug to do so after Astellas’ Veozah (fezolinetant).Amgen’s med grabs FDA’s accelerated nod for small cell lung cancerFDA has granted accelerated approval to Amgen’s Imdelltra (tarlatamab-dlle)
for adults in the advanced stages of small cell lung cancer
(SCLC) that has proven to be hard to treat or has worsened despite platinum-based chemotherapy. The bi-specific antibody is
a targeted immunotherapy that shrank tumors in 40 percent of patients in a
mid-stage trial. SCLC is usually more aggressive and deadlier than non-small
cell lung cancer.First biosimilars for Regeneron’s Eylea okayed: FDA has approved the first interchangeable biosimilars to Eylea (aflibercept) to treat macular
degeneration and other eye conditions. The US agency greenlit Biocon Biologics’ Yesafili (aflibercept-jbvf) and Samsung Bioepis and Biogen’s Opuviz (aflibercept-yszy) to be used as a substitute for Regeneron’s blockbuster eye drug.Cancer victims sue J&J over fraudulent bankruptciesA group of cancer victims have sued Johnson & Johnson accusing it of fraud for the repeated efforts to use a shell company’s bankruptcy to resolve tens of thousands of lawsuits alleging its talc products contained asbestos and caused cancer.Biogen to buy HI-Bio for up to US$ 1.8 bn; Merck KGaA to buy Mirus BioBiogen has agreed to acquire Human Immunology
Biosciences (HI-Bio) for up to US$ 1.8 billion, including an upfront
payment of US$ 1.15 billion. The deal gives Biogen a promising candidate — felzartamab — that may be a cure
for a range of immune-mediated diseases.Meanwhile, Merck KGaA has agreed to buy Wisconsin-based Mirus Bio for US$ 600 million. Mirus Bio is a specialist
in the development and commercialization of transfection reagents that are used
to help introduce genetic material into cells. These reagents play a key role
in the production of viral vectors for cell and gene therapies.J&J to buy Proteologix for US$ 850 mn: Johnson & Johnson is acquiring
Proteologix for US$ 850 million in cash to advance its dermatology portfolio
with the opportunity to address significant unmet need in atopic dermatitis.Aktis in radiopharma deal with Lilly: Aktis Oncology is collaborating with Eli Lilly to discover and develop novel anti-cancer radiopharmaceuticals. Aktis will receive an upfront payment of US$ 60 million, and will be eligible for
milestone payments of US$ 1.1 billion.
Impressions: 462
https://www.pharmacompass.com/radio-compass-phisper/astra-targets-us-80-bn-revenue-by-2030-invests-us-1-5-bn-in-adc-plant-lilly-s-weekly-insulin-scores-late-stage-wins
#Phispers by PharmaCompass
23 May 2024
In this week’s news, France attracted investments from pharma majors such as Sanofi, Pfizer, AstraZeneca and GSK during the ‘Choose France’ summit.In approvals, the US Food and Drug Administration (FDA) expanded the use of Bristol Myers Squibb’s cancer cell therapy Breyanzi for the treatment of adults with follicular lymphoma. The agency also okayed Roche’s HPV self-testing kit for cervical cancer.In deals, beleaguered Novavax signed a US$ 1.2 billion deal with
Sanofi to license and commercialize its existing Covid vaccine and develop a
combo jab to protect against Covid and influenza.
AbbVie and Gilgamesh signed a deal worth over US$ 2 billion to develop a new
class of psychedelic compounds to treat psychiatric conditions.Takeda grabbed the opportunity to license Alzheimer’s therapies from AC Immune, including an Alzheimer’s vaccine. Novo Nordisk tied up with Metaphore to develop next-generation obesity drugs.And deaths from drug overdose reduced for the first time
in six years in the US, giving hope that the ongoing opioid crisis in the
country might be receding.France draws US$ 2.25 bn
investment from Sanofi, Pfizer, Astra, GSKDuring the ‘Choose France’ summit held this week, Sanofi said it is investing over € 1 billion (US$ 1.1 billion) to create new bioproduction capacity at its sites in Vitry-sur-Seine, Le Trait, and Lyon Gerland.
This new investment will create over 500 jobs.Meanwhile, Pfizer pledged another € 500 million (US$ 539 million) toward its five-year investment plan in France, bringing the
total to over € 1.5 billion (US$ 1.63 billion). AstraZeneca announced an investment of € 360 million (US$ 388 million) at its production facility in Dunkirk. Others to announce investments in France were GSK with € 140 million (US$ 151 million), AbbVie with € 36 million (US$ 39 million), and Novartis with € 30 million (US$ 32 million).Sanofi bails out Novavax, pays US$ 1.2 bn
to commercialize, develop Covid jabsNovavax has received a timely shot in the arm, as Sanofi will pay the former up to US$ 1.2 billion in exchange for a license to commercialize its existing Covid vaccine and develop a combo jab to protect
against Covid and influenza. The struggling Maryland
biotech’s management, or lack thereof, has come under fire from investors.
Sanofi can also use Novavax’s Matrix-M adjuvant technology to develop new vaccines. Novavax will receive another US$ 200 million for this. Sanofi also gets a minority 4.9 percent stake in Novavax.AbbVie, Gilgamesh ink deal to
develop psychedelic drugs for psychiatric conditionsAbbVie and Gilgamesh Pharmaceuticals have inked a deal potentially worth over US$ 2 billion to develop a new class of psychedelic compounds to treat psychiatric conditions. The collaboration marries AbbVie’s expertise in psychiatry and Gilgamesh’s innovative research platform to discover novel neuroplastogens (a novel class of compounds inducing rapid and enduring neuroplasticity).US drug overdose deaths drop for first time in six years: In a rare ray of hope in the ongoing US opioid crisis, overdose deaths in the
country fell for the first time in six years, according to preliminary data released by the Centers for
Disease Control and Prevention (CDC). However, drug overdose deaths in 2023
still went over the 100,000 mark. Overall, drug overdoses
claimed 107,543 lives in the US last year compared to 111,029 in 2022, a 3
percent drop.FDA expands use of BMS’ Breyanzi to treat follicular lymphomaFDA has expanded the use of Bristol Myers Squibb’s cancer cell therapy Breyanzi for the treatment of adults with a type of blood cancer known as follicular lymphoma that has returned or has not responded to prior treatments. This marks the fourth approval for Breyanzi.FDA okays Roche’s HPV self-testing kit for cervical cancer: Roche said FDA has approved its cobas human
papillomavirus (HPV) self-collection solution, one of the first available in the US. The
move is expected to significantly improve access to testing by providing women
the option to privately collect their own sample.WHO clears Takeda’s dengue vaccine: WHO
has granted prequalification to Takeda’s dengue vaccine TAK-003,
thereby okaying it to be procured by agencies like UNICEF and PAHO. This is only the second dengue vaccine to receive WHO prequalification. Sanofi’s Dengvaxia received it in 2020 for people aged between six and 45 years.Takeda seizes option to license Alzheimer’s therapies from AC ImmuneTakeda is paying Swiss biotech AC Immune US$ 100 million upfront and betting a further US$ 2.1 billion for an exclusive option to license global rights to an Alzheimer’s vaccine and “active immunotherapies targeting toxic forms of amyloid beta.”Novo ties up with Metaphore
to develop obesity drugs: In its quest to develop novel treatment approaches for cardiometabolic and rare diseases, Novo Nordisk has penned a strategic partnership with Flagship Pioneering company Metaphore Biotechnologies. The Danish Big Pharma will
pay up to US$ 600 million in upfront, development, and commercial milestone
payments to develop up to two next-generation therapeutics for obesity
management.Five die in MacroGenics’ prostate cancer trial; Keytruda fails phase 3 trialMacroGenics reported five deaths as well as an over 50 percent rate of grade 3 or worse
adverse events in a phase 2 trial. Its investigational ADC
(vobramitamab duocarmazine) was being trialed in the treatment of metastatic
castration-resistant prostate cancer. Two of the fatalities were deemed to be
unrelated to the study. The other three deaths are still being investigated.Keytruda-chemo combo fails in
late-stage trial: Merck’s Keytruda, in combination with chemotherapy as adjuvant treatment with or without
radiotherapy, did not meet its primary endpoint of disease-free survival
(DFS) for the treatment of patients with newly diagnosed, high-risk endometrial
cancer after surgery.Novo’s hemophilia drug, Cytokinetics’ heart drug score late-stage winsNovo Nordisk’s Mim8 has hit its primary endpoint in a late-stage trial to treat people with hemophilia A. This paves the way for filing for approval later this year and competing with Roche’s Hemlibra (emicizumab-kxwh) that raked in CHF 4.1
billion (US$ 4.5 billion) last year. The once-weekly and once-monthly Mim8
demonstrated superior reductions of 97 percent and 99 percent in treated
bleeds, respectively, compared to those who received no prophylaxis treatment.Late-stage win for Cytokinetics’ heart drug: Primary data from Cytokinetics’ highly anticipated phase 3 trial showed that
its investigational cardiac myosin blocker aficamten significantly improved
exercise capacity in patients with obstructive hypertrophic cardiomyopathy. Approval will pit it against Bristol Myers Squibb’s oral drug Camzyos (mavacamten), which was the main candidate in BMS’ US$ 13.1 billion acquisition of MyoKardia.
Impressions: 805
https://www.pharmacompass.com/radio-compass-phisper/france-draws-us-2-25-bn-investment-from-big-pharma-sanofi-strikes-us-1-2-bn-deal-with-novavax
#Phispers by PharmaCompass
16 May 2024
Last year, the US Food and Drug Administration (FDA) had published a new draft guidance for researchers investigating the use of psychedelic drugs for potential treatment of psychiatric disorders. This week, the agency took a major step in that direction by announcing that its panel of independent advisers will deliberate on June 4 on whether it should recommend approving the first midomafetamine-assisted therapy for post-traumatic stress disorder (PTSD). Midomafetamine or MDMA is a psychedelic, commonly known as ecstasy.In deals, Novartis has acquired Massachusetts-based
radioligand therapy developer Mariana Oncology for an upfront payment of US$ 1
billion plus US$ 750 million in additional milestone payments.After much delay, FDA has announced that its panel of independent advisers will discuss Eli Lilly’s experimental Alzheimer’s disease drug donanemab on June 10.In trials, Regeneron’s investigational gene therapy (DB-OTO) dramatically improved hearing to normal levels in a child with profound genetic deafness within 24 weeks. Pfizer’s mid-stage trial of its experimental gene therapy to treat Duchenne muscular dystrophy (DMD) took a tragic twist when a young patient died. And AstraZeneca clinched a late-stage win for Calquence in untreated mantle cell lymphoma. Meanwhile, Pfizer has agreed to settle over 10,000 lawsuits pertaining to cancer risks concerning the now discontinued heartburn drug Zantac.After over three billion doses, the Oxford-AstraZeneca
Covid vaccine is being withdrawn worldwide. And in compliance news, Indian
drugmaker Zydus Lifesciences got issued a second Form 483 in one month.Novartis dishes out US$ 1.75
bn for Mariana to strengthen radioligand med pipelineNovartis has agreed to buy Massachusetts-based Mariana Oncology for an upfront payment of
US$ 1 billion plus US$ 750 million in additional milestone
payments. Preclinical-stage biotech Mariana focuses on developing novel
radioligand therapies (RLTs) to treat cancers with high unmet need. The
acquisition encompasses a robust portfolio of RLT programs spanning lead
optimization to early development across a range of solid tumor indications
such as breast, prostate and lung cancer. RLTs are a form of precision medicine
that combines a tumor-targeting molecule (ligand) with a therapeutic
radioisotope (a radioactive particle). This targeted approach enables the
delivery of radiation to the tumor, while limiting damage to the surrounding
cells.In a first, FDA panel to
consider psychedelic-assisted therapy for PTSDFDA’s panel of independent advisers will deliberate on June 4 to discuss whether it should
recommend approving the first MDMA-assisted therapy for PTSD, said Lykos Therapeutics, a drugmaker working in the mental healthcare
space. MDMA (or midomafetamine) is a psychedelic, commonly
known as ecstasy. This will be the first FDA advisory committee
meeting to review a potential new PTSD treatment in 25 years.PTSD is a mental health condition triggered by a terrifying event.
For long, mental health researchers have been captivated by psychoactive
ingredients found in cannabis and LSD. A number of companies are testing such
drugs to find cures for a range of mental health disorders.Meanwhile, a London-based biotech — Compass Pathways — has announced top-line results from a phase 2 study
evaluating the safety and tolerability of an investigational psilocybin
treatment (COMP360) in 22 patients with PTSD. The study met its primary safety
endpoint and available secondary efficacy endpoints. Psilocybin is a naturally
occurring psychedelic compound.Panel to meet on June 10 to discuss Lilly’s donanemab: On June 10, FDA’s panel of independent advisers will discuss Eli Lilly’s experimental Alzheimer's drug donanemab. The drug was
declined accelerated approval in January 2023. If approved, it will compete with Eisai and Biogen’s Leqembi (lecanemab-irmb), which saw FDA approval last year.AstraZeneca to withdraw Covid
vaccines globally, cites surplus of newer vaccinesAstraZeneca has said it has begun a worldwide withdrawal of its Covid vaccine — Vaxzevria, also sold as Covishield — due to the surplus availability of other updated vaccines. Astra denied the decision had anything to do with the claim that the vaccine caused unusual blood clots, which were sometimes fatal. This easy to store vaccine was once heralded as “a vaccine for the world.” “As multiple, variant Covid-19 vaccines have since been developed there is a surplus of available updated vaccines,” the company said.Pfizer reports patient death in phase 2 study on DMD: A young patient died from cardiac arrest after
receiving Pfizer’s experimental gene therapy as part of a mid-stage trial for the muscle-wasting
disorder Duchenne muscular dystrophy (DMD), the company said. The patient
received the investigational gene therapy, fordadistrogene movaparvovec, in
early 2023. Pfizer is in the process of reviewing the data to understand the
potential cause.Meanwhile, the drug behemoth has agreed to settle over 10,000
lawsuits pertaining to cancer risks concerning the now discontinued heartburn drug
Zantac, a Bloomberg News report said.Astra clinches late-stage win
for Calquence in untreated mantle cell lymphomaIn a phase 3 study, AstraZeneca’s Calquence (acalabrutinib) in combination with standard-of-care
chemoimmunotherapy, bendamustine and rituximab, demonstrated a statistically significant and clinically meaningful
improvement in progression-free survival (PFS) as compared to standard of care
in previously untreated adult patients with mantle
cell lymphoma (MCL). While this came from interim analysis, it renders
Calquence the first BTK inhibitor to show a favorable trend in overall survival
versus standard-of-care chemoimmunotherapy in previously untreated MCL.Regeneron’s gene therapy ‘dramatically’ improves hearing in kidsRegeneron Pharmaceuticals’ investigational gene therapy DB-OTO dramatically improved hearing to normal levels in a child with profound genetic deafness within 24 weeks in phase 1/2 trial. The
child, the youngest in the world to receive a gene therapy for genetic
deafness, was dosed at 11 months of age. In a second child, dosed at four years
of age, initial hearing improvements were observed at six
weeks.Zydus’ Gujarat plant hit with Form 483 with 10 observationsZydus Lifesciences has received its second Form 483 within a span of one month. Following an inspection at the company’s site in the Indian state of Gujarat between April 15 and April 23, FDA issued a Form 483 with 10 observations. The agency found that Zydus skirted and skipped over procedures that prevent microbiological contamination of sterile drug products. After cross-contamination was found and batches rejected, other lots made with the same equipment were shipped to the US without thoroughly being investigated. Furthermore, an employee was reported to be collecting samples but had not actually done so. The task was instead delegated to an unqualified staffer.
Impressions: 566
https://www.pharmacompass.com/radio-compass-phisper/fda-to-consider-psychedelic-assisted-drug-for-treating-ptsd-novartis-buys-mariana-oncology-for-up-to-us-1-75-bn
#Phispers by PharmaCompass
09 May 2024
The price of drugs is the highest in the US, and the White House has been working on bringing down drug prices for several years now. This week, the US Federal Trade Commission (FTC) intensified its efforts to bring down the price of drugs by disputing the accuracy and relevance of over 300 patent listings in US Food and Drug Administration’s (FDA’s) Orange Book. It also shot off warning letters to 10 drugmakers in the matter.The White House got another shot in the arm when a federal judge tossed out Johnson & Johnson and Bristol-Myers Squibb’s legal challenges to the constitutionality of price negotiations under the Inflation Reduction Act (IRA).Meanwhile, FDA approved the first gene therapy Pfizer that
treats hemophilia B, a rare bleeding disorder. The agency also handed X4
Pharmaceuticals its maiden approval as it greenlit Xolremdi capsules to treat
patients 12 years of age and older with WHIM syndrome, a rare immunodeficiency
disease. In acquisitions and deals, Japan’s Ono Pharmaceutical has agreed to acquire Massachusetts-based Deciphera Pharmaceuticals for US$ 2.4 billion in cash in a bid to expand its oncology portfolio and presence in the West. Novartis expanded its peptide discovery collaboration with Japan-based PeptiDream in a deal worth over US$ 2.71 billion in milestone payments. And, Morphosys has confirmed that its takeover by Novartis is advancing steadily.FTC challenges over 300 patent listings across 20 brands in FDA’s Orange BookFTC has shot off warning letters to 10 drugmakers and notified the FDA that it disputes the accuracy or relevance of
over 300 patent listings in its Orange Book across 20 different brand name
drugs. The list includes Novo Nordisk’s blockbuster diabetes drug
Ozempic (semaglutide).The consumer protection agency contends that drugmakers are needlessly
listing dozens of extra patents for branded medications to keep their drug
prices high and stall entry of generic competitors into the US market.Among other drugmakers that received warning letters for “bogus patent listings” were AstraZeneca, Boehringer Ingelheim, GSK, Novartis, and Teva for their diabetes,
weight-loss, asthma, and chronic obstructive pulmonary disease (COPD) drugs.FDA approves first gene
therapy from Pfizer to treat rare bleeding disorderFDA has approved Pfizer’s one-time gene therapy — Beqvez (fidanacogene
elaparvovec-dzkt) — for adults with hemophilia B, a rare genetic bleeding disorder. This marks the first gene therapy by the American pharma giant
to win clearance in the US. Beqvez was listed at US $3.5 million, the same
price as its rival gene therapy Hemgenix from Australia’s CSL.X4 lands maiden FDA approval: Boston-based
X4 Pharmaceuticals has landed its maiden FDA approval after the agency okayed Xolremdi (mavorixafor) capsules in patients 12
years of age and older with WHIM syndrome
(warts, hypogammaglobulinemia, infections and myelokathexis). Xolremdi is the first therapy approved in the US for this
rare genetic disease.Judge tosses out lawsuits
contesting constitutionality of IRA price negotiationsA federal judge in New Jersey rejected legal challenges by J&J and BMS to the Biden administration’s Medicare drug-price negotiations, ruling that the program is constitutional. The judge contended that the price negotiations
with Medicare and Medicaid are voluntary and dismissed the notion that they violated the drugmakers’ Fifth Amendment rights, which cover illegal taking
over of property. This was the fourth federal judge to rule in favor of the
White House as it chalks up yet another win in a bitter legal battle with
several pharma companies over the constitutionality of the IRA price negotiations.J&J proposes US$ 6.5 bn settlement to end all talc lawsuits: J&J has offered a US$ 6.48 billion
settlement plan to resolve nearly all of its pending talc
lawsuits, to be paid over 25 years. An overwhelming 99.75
percent of the lawsuits to be resolved claim that the talc caused ovarian cancer. J&J will allow ovarian cancer claimants to vote on the offer and should it reach a 75 percent support threshold, the company can end litigation and prevent future lawsuits via a bankruptcy filing of its subsidiary LTL Management.Ono to buy Deciphera for US$
2.4 bn to expand oncology portfolio, US presenceJapanese drugmaker Ono Pharmaceutical has agreed to acquire Massachusetts-based Deciphera Pharmaceuticals for US$ 2.4 billion in cash. The acquisition
gives Ono access to Qinlock (ripretinib), a kinase inhibitor approved in over 40 countries including in the US, Europe, and China, as a fourth-line treatment for gastrointestinal stromal tumor (a type of cancer that begins in the digestive system). Ono will also get another key investigational drug — vimseltinib — that has shown efficacy in phase 3 trials in patients with tenosynovial giant cell tumor (a benign lesion).Novartis, PeptiDream ink radiopharma deal: Novartis has expanded
its peptide discovery collaboration with Japan-based PeptiDream in a deal worth over US$ 2.71 billion in milestone payments, plus
an upfront payment of US$ 180 million. Under the multi-program
agreement, PeptiDream will use its proprietary Peptide Discovery Platform
System technology to identify and optimize novel macrocyclic peptides against
targets selected by Novartis. Peptide-drug conjugates (PDCs) are the next generation
of targeted therapeutic drugs after antibody-drug conjugates (ADCs) and
Novartis is the only big pharma with FDA-approved radioligand PDCs thus far.BMS, Repertoire tie-up for
vaccines: BMS and Repertoire Immune Medicines have entered into a potential US$ 1.8 billion, multi-year collaboration to create tolerizing vaccines for up to three autoimmune diseases. Repertoire will utilize its T cell receptor (TCR)-epitope
discovery platform, Decode, and its proprietary lipid nanoparticle delivery
technology to discover and develop treatments for
patients suffering from autoimmune diseases by resetting the immune system.Buyout by Novartis on course, says Morphosys: Morphosys has confirmed that its takeover by Novartis is advancing steadily and is
anticipated to close in the first half of 2024.
The news comes after STAT News
published a report stating the lead candidate
of the € 2.7 billion (US$ 2.89 billion) deal — pelabresib — had run into safety problems in a phase 3 trial. The
promising investigational drug is being developed to treat myelofibrosis, a
rare kind of blood cancer that affects the bone marrow.Metagenomi, Moderna part ways: Metagenomi and Moderna have mutually agreed to walk out on their collaboration to develop gene editing therapies.
The deal was reportedly worth up to US$ 3 billion in biobucks.
Impressions: 789
https://www.pharmacompass.com/radio-compass-phisper/ftc-disputes-relevance-of-over-300-patent-listings-japan-s-ono-to-buy-deciphera-for-us-2-4-bin
#Phispers by PharmaCompass
02 May 2024
This week’s Phispers comes packed with news about drug approvals by the US Food and Drug Administration (FDA). The agency approved three drugs for cancer and one for Crohn’s disease. Amongst these was ImmunityBio that bagged its maiden FDA approval for its bladder cancer drug Anktiva. The agency also granted accelerated approval to Day One’s Ojemda to treat the most common form of childhood brain tumor. Roche’s Alecensa secured FDA approval to become the first adjuvant treatment for early-stage lung cancer after surgery, and Takeda’s self-administered version of Entyvio got FDA’s nod for Crohn’s disease.The week also saw two deals in the RNA space. Ipsen and
Skyhawk Therapeutics inked a potential US$ 1.8 billion deal to discover and
develop RNA-targeting small molecules for rare neurological diseases.
Boehringer Ingelheim and Ochre Bio also signed a deal to develop regenerative
therapies for liver diseases. Besides these, Incyte agreed to buy Escient
Pharmaceuticals for US$ 750 million, including two candidates that have shown
potential in treating inflammatory skin conditions.In trials, Sanofi’s rilzabrutinib saw a late-stage win in treating a blood disorder known as immune thrombocytopenia, and Neurocrine’s Takeda-partnered experimental drug NBI-1065845 showed promise in a depression study.ImmunityBio bags maiden FDA
nod for bladder cancer med AnktivaIn what marks the first approval for ImmunityBio, FDA has greenlit Anktiva
(nogapendekin alfa inbakicept-pmln) as part of a combination therapy to treat a type of bladder cancer. Anktiva was okayed alongside the Bacillus Calmette-Guérin (BCG) vaccine to treat patients with BCG-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS). Anktiva is a first-in-class IL-15 agonist, a next-generation immunotherapy beyond checkpoint inhibitors. It creates long-term immunity by activating the so-called natural killer (NK) cells and T-cells. It will compete with Merck’s Keytruda. The approval is a shot in
the arm for ImmunityBio, given that it had reiterated doubts about its ability to stay in
business only last month.Day One’s Ojemda bags accelerated approval: Day One Biopharmaceuticals’ Ojemda (tovorafenib) has been granted FDA’s accelerated approval to treat certain types of
pediatric brain cancer. The pan-RAF kinase inhibitor has been okayed for patients six months of age and older with relapsed or
refractory pediatric low-grade glioma (LGG) harboring a BRAF fusion or
rearrangement, or BRAF V600 mutation. This is the first FDA approval of a
systemic therapy for treating what is the most common form of childhood brain
tumor, including fusions.Genentech’s Alecensa okayed for early-stage lung cancer: FDA has approved Genentech’s Alecensa (alectinib) as an adjuvant treatment
following tumor removal in patients with anaplastic lymphoma kinase
(ALK)-positive non-small cell lung cancer (NSCLC). The approval helps address
an urgent unmet need, with about half of the
people living with early-stage NSCLC experiencing disease recurrence following
surgery, despite adjuvant chemotherapy. The decision comes over a month ahead of Alecensa’s PDUFA date of May 22.Takeda’s Entyvio approved for Crohn’s disease: FDA has approved Entyvio (vedolizumab) subcutaneous administration
as a maintenance therapy in adults with moderately to severely active Crohn’s disease (CD) after induction therapy with intravenous Entyvio. A phase 3 trial showed a significant finding — 48 percent of patients who received subcutaneous
Entyvio achieved long-term clinical remission.Ipsen, Skyhawk ink potential
US$ 1.8 bn deal for rare neurological diseasesSkyhawk Therapeutics has swooped in on a deal to discover and develop novel small molecules that
modulate RNA for rare neurological diseases. Skyhawk’s unique platform accelerates building RNA-targeting small molecules across several therapeutic areas, including rare neurological diseases. The promising technology allows for the exploration of previously unmet needs. Ipsen has committed up to US$ 1.8 billion but didn’t go into details.Boehringer, Ochre Bio sign potential US$
1 bn deal to develop liver therapiesBoehringer Ingelheim and Ochre Bio have struck a deal potentially worth over US$ 1 billion to collaborate on therapies for liver diseases, such as metabolic dysfunction-associated steatohepatitis
(MASH) cirrhosis. Liver is the only organ that can regenerate itself. Ochre Bio will seek to develop drugs that aim to harness the organ’s capacity to regenerate. Ochre Bio will utilize its proprietary discovery
platform combining machine learning with human big data, including
advanced imaging and deep genomic phenotyping, as well as in-house RNA
chemistry.Incyte to buy Escient Pharma: Incyte has agreed to buy Escient Pharmaceuticals for US$ 750 million, which gives the former
ownership over a couple of candidates that have shown potential in treating inflammatory skin conditions and itching related to kidney and liver diseases.Lilly buys Nexus’ Wisconsin plant: Eli Lilly has bought a manufacturing
facility in Wisconsin that produces injectables from Nexus Pharmaceuticals for an undisclosed sum. A surge in demand for glucagon-like peptide-1
(GLP-1) agonists, a class of medications highly effective in treating both
diabetes and obesity, has resulted in supply constraints for companies like
Lilly and Novo Nordisk. Lilly expects to begin
production at the site at the end of 2025.Sanofi’s rilzabrutinib triumphs in late-stage trial for blood disorderData from a late-stage study of Sanofi’s BTK inhibitor rilzabrutinib has shown that it led to durable platelet
response in treating immune thrombocytopenia (ITP). Having met the primary
endpoint in a phase 3 study, Sanofi plans on filing regulatory submissions in
the US and Europe this year. Sanofi had acquired rilzabrutinib through its US$ 3.68 billion acquisition of
Principia Biopharma. ITP is a serious, acquired autoimmune blood disorder
characterized by autoantibody-mediated platelet destruction and impaired
platelet production, leading to thrombocytopenia and an increased risk of
life-threatening bleeding episodes.Neurocrine’s experimental drug shows promise in depression: Neurocrine Biosciences said its experimental drug NBI-1065845 significantly reduced depression severity in adults with major depressive disorder in a phase 2
study that had enrolled 183 patients (who had previously shown inadequate
response to antidepressants). Neurocrine is developing NBI-1065845 in
partnership with Takeda. If approved, the drug could
become the first of its kind.Leqembi sales treble: Despite reports of physician skepticism, Biogen’s Leqembi (lecanemab) reported nearly three-fold increase in sales of the Alzheimer’s drug — at US$ 19 million in the first quarter of
2024 as compared to the previous quarter sales of US$ 7 million.
Impressions: 585
https://www.pharmacompass.com/radio-compass-phisper/immunitybio-bags-maiden-fda-nod-for-bladder-cancer-ipsen-skyhawk-partner-to-develop-neurological-therapies
#Phispers by PharmaCompass
25 Apr 2024
