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: 2331
Pharma indices had begun to recede in March. Their red streak accelerated in April with the Nasdaq Biotechnology Index (NBI) falling 6 percent from 4429.97 in March to 4162.33 last month and the SPDR S&P Biotech ETF (XBI) index tripping 10 percent from 94.34 to 84.62. Similarly, the S&P Biotechnology Select Industry Index (SPSIBI) was down 11 percent from 7402.50 to 6584.40 in April.This
muted performance was also reflected in the first quarter (Q1) results. Biogen, Biomarin, Roche, Gilead, Merck, Sanofi, BMS and Johnson & Johnson announced their Q1 results in April.
While Roche and Biogen reported a drop in revenues, BMS posted a loss in Q1, even though its revenues increased. And
Sanofi reported a 14.7 percent drop in operating income due to competition from generics and currency fluctuations.
BioMarin, Gilead and Merck reported a 9 percent, 5 percent and 9 percent increase, respectively, in their Q1 revenues over Q1 2023.There were a handful of deals announced last month, the biggest
being Vertex Pharmaceuticals’ acquisition of Alpine Immune Sciences for US$ 4.9 billion. The deal gives Vertex access to Alpine’s protein-based immunotherapies for autoimmune diseases.Japanese drugmaker Ono Pharmaceutical agreed to acquire Massachusetts-based Deciphera Pharmaceuticals for US$ 2.4 billion. The acquisition gives Ono
access to Deciphera's oncology pipeline. Denmark’s Genmab agreed to acquire
privately-owned ProfoundBio for US$ 1.8 billion. ProfoundBio has three next-generation antibody drug conjugate
(ADC) candidates in its pipeline that will now go to Genmab.Access the Pipeline Prospector Dashboard for April 2024 Newsmakers (Free Excel)FDA okays ImmunityBio’s Anktiva, Day One’s Ojemda for bladder, brain cancerLike March, April too saw several significant drug approvals,
especially in oncology. ImmunityBio (its stock was up 50 percent
in April) won its first US Food and Drug Administration (FDA) approval as
the agency greenlit Anktiva as part of a combination therapy to treat a type of bladder cancer. With this
approval, Anktiva will compete with Merck’s Keytruda.AstraZeneca (stock up 12 percent)
and Daiichi Sankyo’s Enhertu has been granted accelerated
approval in the US to treat adult patients with
HER2-positive solid tumors that have spread or cannot be surgically removed. These patients
have undergone prior treatment and have no satisfactory alternatives available to them. J&J and Legend’s Carvykti has become
the first and only BCMA-targeted
therapy approved by the FDA for patients with relapsed or refractory
multiple myeloma, who have received just one prior line of treatment. The approval is an important milestone in J&J’s plans to make Carvykti a US$ 5 billion-plus asset at peak yearly
sales.Day One Biopharmaceuticals’ Ojemda 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.Access the Pipeline Prospector Dashboard for April 2024 Newsmakers (Free Excel) Basilea’s antibiotic bags FDA nod; Vanda’s Fanapt approved for bipolar disorderThe agency also approved Vanda Pharmaceuticals’ (stock up 25 percent) Fanapt tablets for treating manic
or mixed episodes associated with bipolar-1 disorder in adults.
The atypical antipsychotic agent has been used for the treatment of
patients with schizophrenia since its FDA approval in 2009. This second
approval for bipolar-1 disorder patients (who experience manic episodes that
last at least seven days) could help revive Fanapt prescriptions.FDA has also approved Basilea Pharmaceutica’s Zevtera, an antibiotic for bacterial
infections including multidrug-resistant strains. The US agency has okayed it
for three conditions – treatment of adults with Staphylococcus aureus bacteremia, including those with right-sided infective endocarditis; adults with acute bacterial skin and skin structure infections; and adult and pediatric patients with community-acquired bacterial pneumonia.Access the Pipeline Prospector Dashboard for April 2024 Newsmakers (Free Excel) BMS’ schizo drug shows benefit sans weight gain; Imfinzi scores in lung cancer trialA common side effect associated with anti-psychotics is weight
gain. However, interim results from a late-stage trial
have shown that BMS’ schizophrenia drug KarXT continued to improve symptoms of the severe mental disease even at 52 weeks, and without weight gain.In another phase 3 trial, Imfinzi significantly improved overall survival and progression-free survival in
patients with limited-stage small cell lung cancer. This makes this blockbuster drug the first and only
immunotherapy to demonstrate survival benefit for this aggressive form of lung cancer. Imfinzi
had posted sales of US$ 4.24 billion in 2023.Intra-Cellular Therapies said a phase 3 study of its drug Caplyta in people with major
depressive disorder (MDD) met both its primary and key secondary endpoints.
Given once daily as an adjunctive therapy to antidepressants, Caplyta 42
mg significantly beat the
placebo at improving depression severity.Access the Pipeline Prospector Dashboard for April 2024 Newsmakers (Free Excel) Zepbound shows benefit in sleep disorder; Novartis’ Fabhalta reduces proteinuriaEli Lilly’s popular weight-loss
drug Zepbound cut irregular breathing episodes associated with a common sleep-related disorder – obstructive sleep apnea (OSA). Treatment with Zepbound reduced the frequency of irregular
breathing episodes by as much as 63 percent in adults with OSA and obesity across two late-stage trials. Lilly plans to submit the findings to FDA to expand Zepbound’s use for OSA. Roche’s Genentech said its drug Columvi,
in combination with
chemotherapy, demonstrated a statistically significant
improvement in overall survival for people with relapsed or
refractory diffuse large B-cell lymphoma (DLBCL). A late-stage study pitted the
Columvi-chemotherapy combination against a rituximab-chemotherapy combination in
patients who received at least one prior line of therapy and are not candidates
for autologous stem cell transplant. Patients in the Columvi group lived
longer.In another late-stage trial, Novartis' drug Fabhalta reduced proteinuria (protein in the urine) in patients with IgA nephropathy
by 38.3 percent. The Swiss drugmaker filed for FDA’s accelerated approval using the data, and the agency has granted it priority
review.Access the Pipeline Prospector Dashboard for April 2024 Newsmakers (Free Excel) Our viewAt
the start of 2024, the US market saw inflation falling and there were talks of
interest rate cuts by the Federal Reserve. But few months on, the outlook has darkened a bit with inflation refusing to let off steam and economic output slowing down in the US. This is getting reflected in the pharma indices and the Q1 results of several drugmakers. Though overall, we have faith in the pharma sector’s spirit of innovation and its zest for dealmaking. Despite the macroeconomic challenges, there is hope that the other three quarters of 2024 will look better.Access the Pipeline Prospector Dashboard for April 2024 Newsmakers (Free Excel)
Impressions: 1512