Market Place
The pharma indices were back in the black in May, and the good streak continued through June with the Nasdaq Biotechnology Index (NBI) gaining 3 percent, the SPDR S&P Biotech ETF (XBI) index up over 3.1 percent and the S&P Biotechnology Select Industry Index (SPSIBI) rising 4.25 percent.The two summer months were similar in more ways than one. In May, Eli Lilly had announced an investment
of US$ 5.3 billion to boost the supply of Zepbound and Mounjaro. In June, Novo Nordisk followed suit,
investing US$ 4.1 billion to develop a new manufacturing facility to boost the supply of Ozempic and Wegovy.Similarly, the US Food and Drug Administration (FDA) continued to
grant vaccine approvals in June after okaying Moderna’s mRNA respiratory syncytial virus (RSV) vaccine in May. Last month, Merck’s next-generation pneumococcal
vaccine won an FDA nod, and the agency expanded the use of GSK’s respiratory syncytial virus (RSV) vaccine to include adults aged 50 to 59.However, June was a lackluster month for mergers and acquisitions.
The month had to be content with some tie-ups. For instance, Roche signed an up to US$ 1.8 billion deal with Boston-based
startup Ascidian Therapeutics to discover and
develop novel gene therapies for difficult-to-treat neurological diseases.
AbbVie inked a US$ 1.7 billion agreement with China’s FutureGen to bring the latter’s next-generation treatment (FG-M701) for inflammatory bowel disease to market. Takeda signed an option agreement with China’s Ascentage Pharma for an exclusive
license to a promising drug for chronic myeloid leukemia and other blood
cancers. And Day One Biopharmaceuticals struck a licensing deal with MabCare for a novel antibody drug conjugate (ADC) to treat multiple cancers. The deal’s potential value is US$ 1.2 billion.Access the Pipeline Prospector Dashboard for June 2024 Newsmakers (Free Excel)Merck’s pneumococcal jab wins FDA nod; Moderna’s combo vaccine scores trial winVaccines took centerstage in June. FDA approved Merck’s Capvaxive, a next-generation pneumococcal vaccine designed to protect adults from a broader range of
pneumococcus bacteria strains that cause serious illnesses and pneumonia. Capvaxive targets 21 bacterial variations responsible for about 85 percent of invasive pneumococcal disease cases in older adults. Similarly, GSK secured FDA approval for its RSV vaccine for
adults aged 50 to 59 years. This makes Arexvy the only RSV shot endorsed for that age group.In a late-stage trial, Moderna’s 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. The combo jab generated more antibodies than currently marketed flu vaccines and Moderna’s Spikevax. And the world’s first personalized mRNA cancer vaccine from Moderna has raised hopes for patients with skin cancer, as three-year data from a mid-stage trial showed some benefits on patients who took the vaccine in combination with Merck’s Keytruda.Despite these developments, Moderna’s stock dropped 19 percent in June after it said the efficacy of its RSV vaccine mRESVIA had vaned substantially to 50 percent after 18 months.Access the Pipeline Prospector Dashboard for June 2024 Newsmakers (Free Excel) Verona, Geron score maiden FDA approvals; BMS’ Augtyro bags tumor-agnostic nodTwo companies obtained their first FDA approvals last month. The
first one was Verona Pharma (stock up 15 percent) — it received its maiden FDA approval for Ohtuvayre for treating chronic
obstructive pulmonary disease (COPD). This is also the first inhaled COPD treatment with
a novel mechanism of action.The second company to get its maiden FDA approval was Geron, a commercial-stage
biopharmaceutical company. FDA signed off on Geron’s Rytelo for
treating transfusion-dependent anemia in patients with low- to
intermediate-risk myelodysplastic syndromes (MDS), a group of blood cancers.In June, FDA granted accelerated approvals to at least three
drugs. BMS’ Augtyro was granted FDA’s accelerated approval for treating 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.Ipsen received FDA’s accelerated approval for Iqirvo, a first-in-class,
once-daily oral medication for treating primary biliary cholangitis (PBC), a
progressive liver disease affecting mostly women.Sarepta Therapeutics’ (stock up 24 percent) Elevidys received accelerated approval for Duchenne muscular dystrophy (DMD) despite failing its primary endpoint in a late-stage trial.Amongst other approvals, Argenx’s Vyvgart Hytrulo was approved to treat chronic inflammatory
demyelinating polyneuropathy (CIDP). This is a novel treatment option for this
rare and debilitating neuromuscular disorder. And BMS’ Krazati got its second approval for treating colorectal
cancer with a specific KRAS mutation.Access the Pipeline Prospector Dashboard for June 2024 Newsmakers (Free Excel) GSK’s failed cancer drug posts trial win; Gilead’s HIV prophylaxis shows efficacyIn late-stage trials, GSK’s multiple myeloma therapy
Blenrep cut the risk of disease progression
or death by almost half compared to
standard-of-care treatments. The once-failed ADC was pulled from the
lucrative US market in 2022, but the results could signal a comeback for
Blenrep.Intra-Cellular’s antipsychotic drug Caplyta scored another remarkable late-stage win for treating major depressive disorder (MDD). In another late-stage trial, Gilead’s (stock up 6 percent)
long-acting injection proved to be more effective
in preventing HIV infection in women compared to the daily pill Truvada. 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, Sunlenca could be a game-changer in HIV prevention. Analysts estimate that PrEP-related sales could be over US$ 1.7
billion.In a blow to millions affected by long
Covid, Pfizer’s Paxlovid did not appear to improve the symptoms as was hoped. Pfizer’s stock fell 7 percent in June.Access the Pipeline Prospector Dashboard for June 2024 Newsmakers (Free Excel) Our view This was the second month when pharma indices showed an upward trend. It’s been a good first half for the US stock markets. But there are mixed
reports on how markets will perform in the second
half, given the uncertainties around interest rate cuts and the outcome of the presidential
elections in the US slated to be held in November. Though for those tracking
biopharma news, there are enough interesting developments to fret over the
markets.Access the Pipeline Prospector Dashboard for June 2024 Newsmakers (Free Excel)
Impressions: 2329
https://www.pharmacompass.com/pipeline-prospector-blog/pipeline-prospector-june-2024-fda-approves-merck-s-next-gen-pneumococcal-vaccine-verona-s-copd-therapy
#PipelineProspector by PharmaCompass
04 Jul 2024
Share
The New Year got off to a stable start, with some good news
trickling in from clinical trials and Big Pharma announcing a few acquisitions
at the annual JP Morgan healthcare meet.The benchmark index S&P 500 has been on a green streak, growing 15.33 percent since October. In comparison, pharma indices underperformed. The Nasdaq Biotechnology index gained 1 percent in January to 4385.06, mirroring the 1 percent it had gained in December 2023. The SPDR S&P Biotech ETF index lost 1 percent to end January at 87.43 after soaring 18 percent to 89.29 in December. And, the S&P Biotechnology Select Industry Index (SPSIBI) dropped 3 percent in January to 6813.77, after spiking 8 percent in December.During the month, Sanofi acquired rare disease
drugmaker Inhibrx for about US$ 2.2 billion and J&J acquired Ambrx for US$ 2 billion to enter the antibody-drug conjugates (ADC) space. ADCs are biopharmaceuticals designed as a targeted therapy for treating cancer.Cell therapies have been facing a rough period with narrower funding options that have forced many startups in the biotech sector to shut shop. The field received a further blow when the US Food and Drug Administration (FDA) mandated “black box” warnings — the highest safety warnings — on six major CAR-T cancer therapies over the risk of developing secondary cancer. However, the regulator is also helping out the sector and has finalized guidance for companies and academic researchers working on CAR-T cell therapies.Access the Pipeline Prospector Dashboard for January 2024 Newsmakers (Free Excel)Vertex’s non-opioid painkiller scores trial wins; Lilly’s therapy cures deafnessVertex Pharmaceuticals’ non-opioid painkiller VX-548 met its primary goals in two late-stage
trials, marking a major development in a decades-long
effort to have an alternative to opioids. The experimental non-opioid
painkiller reduced acute, post-surgical pain. It was found to be safe and did
the job without the risk of addiction. Vertex plans to file for FDA
approval by mid-2024. Analysts estimate VX-548 to bring in peak sales of US$ 5 billion, if approved.There was good news from clinical trials for those with deafness.
In an early-to-mid-stage trial, an 11-year-old boy on Eli Lilly’s investigational gene therapy
AK-OTOF, could hear within 30 days of a single administration.
Similarly, an experimental gene therapy being developed by a Chinese drugmaker Refreshgene Therapeutics also restored hearing in five out of six children with congenital deafness. Both treatments worked on profound deafness caused by mutations of the OTOF (otoferlin) gene. Vera Therapeutics (stock up 138 percent), a clinical-stage biotechnology company, announced positive 72-week data from a phase 2b clinical trial of atacicept in participants with IgA nephropathy (IgAN), also known as Berger’s disease. IgAN is a serious and progressive autoimmune disease of the kidney, for which there remains a high unmet medical need. Vera holds an exclusive worldwide license from Merck KGaA for the development and
commercialization of atacicept in all indications.Meanwhile, Regenxbio touted positive interim data from a mid-stage trial for
its experimental treatment for wet age-related macular degeneration (wet AMD)
that uses a novel delivery, known as suprachoroidal delivery. Regenxbio has
developed the drug along with AbbVie.Access the Pipeline Prospector Dashboard for January 2024 Newsmakers (Free Excel) FDA finalizes CAR-T guidance;
hands Casgevy second nod for genetic blood disorderFDA has finalized guidance for companies and academic researchers working on
CAR-T cell therapies. The guidance enlists the clinical,
non-clinical and manufacturing expectations the agency has before a premarket
approval (PMA) application is made.Vertex Pharmaceuticals and CRISPR Therapeutics’ Casgevy has bagged a second FDA approval, this time for transfusion-dependent β-thalassemia, a rare genetic blood disorder that routinely requires blood transfusions.Across the pond, the European Commission granted marketing
authorization to Stada and Alvotech’s Uzpruvo, a
biosimilar to Johnson & Johnson’s Stelara (ustekinumab). Biosimilar competition in the €2.5 billion (US$ 2.7 billion) ustekinumab market in the
EU could significantly expand patient access to this life-changing biologic
therapy within gastroenterology, dermatology, and rheumatology, a press
statement said. Meanwhile,
Stelara sales grew by nearly 12 percent to about US$ 11 billion in 2023 compared to US$ 9.7 billion the year earlier.Access the Pipeline Prospector Dashboard for January 2024 Newsmakers (Free Excel) Sanofi buys Inhibrx for about US$ 2.2 bn; J&J enters ADC space with Ambrx dealSanofi has agreed to buy
California-based clinical-stage biopharmaceutical company Inhibrx for about US$ 2.2 billion. The acquisition is aimed at acquiring the biopharma’s mid-stage experimental treatment INBRX-101, which will bolster the French drugmaker’s rare genetic disease portfolio. Inhibrx’s other investigational drugs will be spun off into a different
company in which Sanofi will hold an 8 percent stake. INBRX-101 is a
potential treatment for alpha-1 antitrypsin deficiency, a rare disease that
causes the lung tissue to progressively deteriorate.Meanwhile, Johnson & Johnson announced the acquisition of
ADC drug developer Ambrx Biopharma for about US$ 2 billion. This makes J&J the latest drugmaker to bet on ADCs after Pfizer, AbbVie and Merck.Access the Pipeline Prospector Dashboard for January 2024 Newsmakers (Free Excel) Novo sales soar 36% due to
obesity drugs; Pfizer ekes out surprise Q4 2023 profit The month saw several drugmakers announce financial results for the full year 2023. Novo Nordisk saw sales rise 36 percent at
constant exchange rates (CER) to DKK 232.3 billion (US$ 34.36 billion) in 2023
compared to DKK 177 billion (US$ 26.18 billion) in 2022. Ozempic (semaglutide) clocked sales of DKK 95.7 billion (US$ 13.77 billion) compared to DKK 59.6 billion (US$ 8.57 billion), a 66 percent rise. Novo’s stock was up 13 percent in January.Pfizer’s 2023 revenue fell 42 percent year-on-year to US$ 58.5 billion (from the record US$ 100
billion revenue it had posted in 2022) due to a sharp drop in Comirnaty
and Paxlovid sales. Yet, Pfizer managed to eke out a profit of US$ 593 million in Q4,
when analysts were expecting a US$ 1.1 billion loss. Keytruda raked in US$ 25 billion in 2023, a 19 percent increase over the US$ 21 billion posted in 2022, helping Merck achieve sales of US$ 60.1 billion.Access the Pipeline Prospector Dashboard for January 2024 Newsmakers (Free Excel) Our viewThe pharma landscape is clearly changing. As financial results for
2023 pour in, we know that our list of top drugmakers is going to look a lot
different. We may see further shuffling in 2024, with companies shifting focus
to new areas like obesity drugs, ADCs and rare diseases.The operating environment in 2024 faces “continued risk from geopolitical tension, domestic political uncertainty and heated campaign rhetoric, and increasing attention on regulatory enforcement around the world,” says a PwC report on the sector. Given this scenario, companies that are able to reinvent themself this year with new products and new strategies to fight the changing business and regulatory environment will emerge winners.Access the Pipeline Prospector Dashboard for January 2024 Newsmakers (Free Excel)
Impressions: 1984
https://www.pharmacompass.com/pipeline-prospector-blog/pipeline-prospector-jan-2024-vertex-s-non-opioid-painkiller-succeeds-in-trials-sanofi-buys-inhibrx-for-us-2-2-bn
#PipelineProspector by PharmaCompass
08 Feb 2024
After facing the worst year since 2008, biotech indices opened 2023 on a positive note. All the three indices posted gains in January — while the Nasdaq Biotechnology Index (NBI) was up 4.4 percent (at US$ 4,360), the S&P Biotechnology Select Industry Index (SPSIBI) rose 8.7 percent to US$ 6,896 and SPDR S&P Biotech ETF (XBI) increased 9 percent to US$ 88.90. In December 2022, the NBI was down 3.4 percent to US$ 4,213, while XBI had risen only 0.1 percent and SPSIBI had witnessed no change.Several major drugmakers have shared
their financial results for 2022 since last month. Pfizer posted an operational growth of 30 percent with 2022 revenues touching an all-time high of US$ 100.3 billion. Merck posted a 22 percent increase, with sales of Keytruda growing 22 percent to US$ 20.9
billion. GSK generated £29.3 billion (US$ 36.3 billion)
in revenue in 2022, an increase of 13 percent over 2021 figures, and Amgen posted a gain of 1 percent.Drugmakers like Eli
Lilly and Johnson
& Johnson saw a huge drop in sales of their
Covid-19 products. Lilly suffered a 9
percent drop and J&J saw a 4 percent decline in Q4 2022 revenue. More
companies are expected to release their 2022 results this month.Access the Pipeline Prospector Dashboard for January 2023 Newsmakers (Free Excel)Lower 2023 forecast, vaccine’s safety concern hammer Pfizer’s stockDespite the indices showing signs of recovery, stocks of several mega cap companies ended the month in the red. Pfizer’s stock suffered the biggest loss, dropping 14 percent in January, due to an interplay of various factors even as the drug behemoth reported best-ever sales in 2022, owing to its
Covid-19 vaccine Comirnaty and antiviral pill Paxlovid. First, the US Food and Drug
Administration (FDA) and Centers for Disease Control and Prevention (CDC) launched an investigation into the possibility of a stroke in older adults who had received its updated Covid-19 booster shot. Second, despite the stellar results, Pfizer’s forecast for 2023 was lower by 33 percent, with an estimated revenue of US$ 67 to 71 billion. With the pandemic receding, there has been a considerable drop in demand for Covid products. Pfizer’s Covid vaccine partner BioNTech also saw its stock go down 6 percent. The other big loser was AbbVie (drop of 9 percent) – its blockbuster immunology drug Humira (adalimumab) saw its exclusive run come
to an end in the US with Amgen launching a copycat — Amjevita. Seven other biosimilars are
due to be launched later this year.A federal appeals court in Philadelphia blocked J&J’s plan to use the Texas two-step bankruptcy strategy to resolve around 40,000 lawsuits alleging that its talc products cause cancer. In another setback, its Janssen unit halted a late-stage global trial of its HIV vaccine after the
drug was found to be ineffective at preventing infection. All these factors led
to a 8 percent drop in J&J stocks.AstraZeneca’s stock tumbled 6 percent in
January due to factors such as a second cancer lawsuit filed by Bristol Myers Squibb, wherein it claimed Astra’s cancer treatment Imjudo infringes on two patents
related to its blockbuster drug Yervoy. FDA also revoked the emergency use authorization (EUA) for AstraZeneca’s Covid-19 treatment Evusheld, expecting it to fail against the currently dominant Omicron sub-variant.January wasn’t a great month for Merck either, whose stock
plummeted 4 percent. The drugmaker halted a late-stage trial of its blockbuster immunotherapy drug Keytruda in prostate cancer after interim data showed that the drug failed to extend survival or help improve patients’ lives. Meanwhile, the drugmaker said it has identified the source of a potential cancer-causing agent – Nitroso-STG-19 – in its popular diabetes drugs Januvia and Janumet, and plans to
resolve the issue by the end of the year.Access the Pipeline Prospector Dashboard for January 2023 Newsmakers (Free Excel) Bayer gains on sales forecast for Kerendia, Nubeqa;
lawsuit settlements benefit TevaThree large cap companies — Bayer AG (14 percent), Teva (10 percent) and Vertex Pharmaceuticals (12 percent) – posted double digit gains on the bourses last month. Bayer hiked sales targets for two of its meds – heart drug Kerendia and prostate cancer
treatment Nubeqa – to US$ 3.2 billion each.Israeli drugmaker Teva said
it has either already settled with or confirmed participation of 48 US states to move forward with its US$ 4.25 billion proposed national settlement agreement to resolve thousands of lawsuits over its alleged role in the US opioid crisis.Vertex Pharmaceuticals said it plans to file for the US approval of its CRISPR gene-editing therapy exa-cel as a treatment for beta-thalassemia and sickle cell disease in the first quarter. If approved, the drug is expected to generate peak annual sales of over US$ 2 billion. Vertex has already applied for the drug’s authorization in the EU and UK. Vertex is also
testing a non-opioid drug, VX-548, for acute pain in a late-stage trial.Among small cap companies,
Massachusetts-based Theseus Pharmaceuticals posted a 180 percent surge in its stock price in January. The drugmaker
said it plans to release clinical data from a phase 1/2 trial of its lead candidate – THE-630 – for advanced gastrointestinal stromal tumor patients, later this year. Analysts expect THE-630 to compete against Pfizer’s Sutent (sunitinib), with projected sales of over US$ 1
billion by 2035. Theseus is also developing two other early-stage candidates.Access the Pipeline Prospector Dashboard for January 2023 Newsmakers (Free Excel) Accelerated nods bring gains for Biogen, Seagen; Lilly loses on Alzheimer’s setback Biogen’s second Alzheimer’s drug – Leqembi (lecanemab) — received FDA’s accelerated approval to treat patients
who are in the earliest stages of the neurodegenerative disease. However, Eli Lilly’s bid for an accelerated approval pathway for its experimental Alzheimer’s disease drug donanemab suffered a setback. FDA rejected the drug as Lilly did not provide enough data from patients who were treated for at least a year. However, days later Lilly’s cancer drug Jaypirca (pirtobrutinib) won accelerated approval as a treatment for
mantle cell lymphoma, a rare form of blood cancer.
FDA also accepted Lilly-Boehringer Ingelheim’s application for cancer drug Jardiance (empagliflozin) as a treatment for chronic kidney disease (CKD) in adult patients. Overall, while Biogen’s stock was up 5 percent in January, Lilly’s was down 7 percent.In another cancer drug
update, FDA granted accelerated approval to Seagen’s Tukysa (tucatinib) to treat HER2-positive unresectable or metastatic colorectal cancer. Seagen’s stock rose 8 percent last month. And BeiGene’s cancer drug Brukinsa
(zanubrutinib) received an approval in the US as a
treatment for adult patients with chronic lymphocytic leukemia (CLL) or small
lymphocytic lymphoma (SLL), pushing its stock up 11 percent. The drug has also received authorization in the UK.Access the Pipeline Prospector Dashboard for January 2023 Newsmakers (Free Excel) Our viewAs always, the new year began with the JP Morgan Healthcare Conference (San Francisco, January 9 to 12). For the last three years, the conference has been lackluster insofar as M&As were concerned. But this year, there were a number of buyouts – AstraZeneca said it will acquire US-based drug
developer CinCor Pharma for up to US$ 1.8 billion. Italy’s privately-held Chiesi Farmaceutici announced it is
buying Ireland-based rare diseases drugmaker Amryt Pharma for up to US$ 1.48 billion. And Ipsen picked up Albireo for US$ 952 million. Stocks of
CinCor, Amryt and Albireo shot up by over 100 percent following the takeover
news. While we do hope these buyouts signal an uptick
in M&A activity, we also know that drugmakers who made billions from the pandemic
are now facing a steep Covid cliff. Among these are Pfizer, BioNTech, Moderna, Gilead Sciences, AstraZeneca and Merck. The 2022 results announced so far confirm this trend. Insofar as biotech indices are concerned, we hope January has set the tone for the coming months.Access the Pipeline Prospector Dashboard for January 2023 Newsmakers (Free Excel)
Impressions: 1768
https://www.pharmacompass.com/pipeline-prospector-blog/pipeline-prospector-jan-2023-mega-cap-drugmakers-face-rout-at-bourses-even-as-biotech-indices-inch-upwards
#PipelineProspector by PharmaCompass
09 Feb 2023
