It was a busy week for the biotech sector, with loads of data presentations at the 2023 American Society of Clinical Oncology (ASCO) Annual Meeting. Meanwhile, key regulatory and pipeline updates from bigwigs like GSK GSK and Biogen BIIB were in the spotlight too during the week.
Recap of the Week’s Most Important Stories:
Updates from Glaxo: GSK announced that it has obtained European Commission authorization for Arexvy (an adjuvanted respiratory syncytial virus vaccine) for active immunization for the prevention of lower respiratory tract disease (LRTD) caused by respiratory syncytial virus (RSV) in adults 60 years of age and older.
The authorization was based on positive AReSVi-006 phase III trial data in which the vaccine led to a statistically significant and clinically meaningful overall efficacy of 82.6%. Its efficacy was 94.6% in older adults with at least one underlying medical condition of interest, such as certain cardiorespiratory and endocrine-metabolic conditions. An approval was in the cards as the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) had given a positive opinion for the same in April 2023.
GSK also announced that the FDA has accepted its supplemental biologics license application (sBLA) seeking label expansion for Jemperli (dostarlimab). The sBLA seeks approval for Jemperli in combination with chemotherapy for the treatment of mismatch repair deficient (dMMR)/microsatellite instability-high (MSI-H) primary advanced or recurrent endometrial cancer. The FDA granted priority review to the sBLA with a decision expected on Sep 23.
GSK currently has a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
Pipeline and Regulatory Updates From Biogen: The company announced that it would discontinue one of the two studies of BIIB122, a small molecule inhibitor of leucine-rich repeat kinase 2 (LRRK2), which is being developed in collaboration with Denali Therapeutics Inc. for Parkinson’s disease.
The clinical development program on BIIB122 initially comprised two late-stage studies. The first study was a phase IIb LUMA study (initiated in May 2022) in participants with early-stage Parkinson’s disease, while the second study was a phase III LIGHTHOUSE study (initiated in September 2022) in patients with Parkinson’s disease associated with LRRK2 pathogenic mutations. LRRK2 mutations are one of the most common genetic drivers of Parkinson’s disease.
However, Biogen has now decided to discontinue the LIGHTHOUSE study as it believes the study is complicated and will be completed in 2031 only. Concurrently, the protocol of the LUMA study will be amended to now include only some eligible patients with a LRRK2 genetic mutation in addition to continuing to enroll eligible patients with early-stage idiopathic Parkinson’s disease.
Biogen’s partner Eisai has submitted a marketing authorization application for Alzheimer’s disease drug, Leqembi, to the Ministry of Food and Drug Safety (MFDS) in South Korea.
Additionally, the FDA issued briefing documents on Leqembi for the upcoming Peripheral and Central Nervous System Drugs Advisory Committee (“PCNSDAC”) meeting on Jun 9. The FDA briefing document acknowledges that treatment with Leqembi in the early stages of the disease reduced the rate of clinical decline in AD patients. The PCNSDAC is set to give a recommendation on whether the data from the phase III CLARITY AD study confirms the clinical benefit of Leqembi for treating AD.
Foghorn Study Hold Lifted: Foghorn Therapeutics Inc. FHTX announced that the FDA has lifted the clinical hold placed on the phase I monotherapy dose escalation study of FHD-286 in acute myelogenous leukemia (AML) and myelodysplastic syndrome (MDS).
The phase I study was put on hold in August 2022 due to suspected cases of fatal differentiation syndrome believed to be associated with the FHD-286 treatment. Differentiation syndrome is associated with AML/MDS therapeutics that induce differentiation, causing undifferentiated cancer cells to mature, and is an effect that has been seen with, and is believed to be on-target for, the proposed mechanism of action for FHD-286. The hold was lifted as of Jun 1, 2023.
Consequently, Foghorn amended the protocol and plans to commence a phase I study of FHD-286 in combination with decitabine or low-dose cytarabine in relapsed and/or refractory AML patients. Data demonstrating FHD-286’s effect as a broad-based differentiation agent, its safety profile, as well as supportive pre-clinical combination data, including robust efficacy data in multiple CDX and PDX models formed the basis for the advancement to this phase I study.
Day One Gains on Positive Data: Shares of clinical-stage biopharmaceutical company Day One Biopharmaceuticals DAWN gained on Jun 5 after the company announced new data from the registrational phase II FIREFLY-1 study evaluating the investigational agent tovorafenib (DAY101) at the 2023 ASCO Annual Meeting. The open-label study treated 77 patients and evaluated tovorafenib as a once-weekly monotherapy in patients aged 6 months to 25 years with relapsed or progressive pediatric low-grade glioma (pLGG).
The data showed an overall response rate (ORR) of 67% and a clinical benefit rate (CBR) of 93% in 69 heavily pretreated Response Assessment for Neuro-Oncology High-Grade Glioma (RANO-HGG) evaluable patients. Among a total of 77 treated patients, the median duration of tovorafenib treatment was 10.8 months, with 74% of patients on treatment at the time of data cut-off as of Dec 22, 2022. Safety data, based on 136 patients treated in both Arm 1 and Arm 2 of FIREFLY-1, indicated monotherapy tovorafenib to be generally well-tolerated.
Additionally, the company announced that it initiated a rolling new drug application (NDA) to the FDA for tovorafenib as a monotherapy in relapsed or progressive pLGG in May 2023. The rolling submission allows Day One to submit portions of the regulatory application, which the FDA can review on an ongoing basis. Day One anticipates the rolling NDA submission of tovorafenib to be completed in October 2023, following the submission of an amended clinical study report (CSR) that will include safety and efficacy data from a planned June 2023 data cutoff.
The Nasdaq Biotechnology Index has gained 1,90% in the past five trading sessions. Among the biotech giants, GSK has gained 2.26% during the period. Over the past six months, shares of MRNA have declined 29.8%. (See the last biotech stock roundup here: Biotech Stock Roundup: MRTX Falls on Study Failure, LXRX Drug Wins Approval & More)
Image Source: Zacks Investment Research
What's Next in Biotech?
Stay tuned for more pipeline updates.
Just Released: Free Report Reveals Little-Known Strategies to Help Profit from the $30 Trillion Metaverse Boom
It's undeniable. The metaverse is gaining steam every day. Just follow the money. Google. Microsoft. Adobe. Nike. Facebook even rebranded itself as Meta because Mark Zuckerberg believes the metaverse is the next iteration of the internet. The inevitable result? Many investors will get rich as the metaverse evolves. What do they know that you don't? They’re aware of the companies best poised to grow as the metaverse does. And in a new FREE report, Zacks is revealing those stocks to you. This week, you can download, The Metaverse - What is it? And How to Profit with These 5 Pioneering Stocks. It reveals specific stocks set to skyrocket as this emerging technology develops and expands. Don't miss your chance to access it for free with no obligation.>>Show me how I could profit from the metaverse!
The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.