This Week in Biotech #37
Catch up on the latest biotech breakthroughs and upcoming trends (Jan 24-30).
Welcome to This Week in Biotech by Biotech Blueprint!
THIS WEEK’S KEY TAKEAWAYS 🔑
The FDA approval of Journavx (suzetrigine) by Vertex Pharmaceuticals is an exciting development this week, especially given the ongoing opioid crisis and the pressing need for safer alternatives in pain management. The fact that it’s the first new class of pain medication approved in over two decades highlights the importance of this milestone. It is fantastic to see an approval for a drug that targets pain by inhibiting the NaV1.8 sodium channel without the addictive side effects of opioids. This approval could be a game-changer for managing moderate-to-severe acute pain. The fact that it’s suitable for a broad range of acute pain types makes it even more promising.
Robert F. Kennedy Jr., president Trump’s nominee for Secretary of Health and Human Services (HHS), has faced intense scrutiny during his Senate confirmation hearings on Jan. 29 and 30, particularly over his controversial views on vaccines. While Kennedy attempted to downplay his anti-vaccine stance, stating that all of his children are vaccinated and that he believes vaccines play a critical role in healthcare, his past rhetoric and association with the Children’s Health Defense organization, known for spreading vaccine misinformation, have continued to raise concerns. During the hearings, Democrats, like Sen. Elizabeth Warren, strongly criticized him for promoting misinformation that could endanger public health, while Republicans were more supportive of his skepticism.
RFK Jr.’s answers during the hearings, particularly regarding the safety and effectiveness of vaccines, were often vague or evasive, raising doubts about his ability to lead the Department of Health and Human Services. Senators questioned his understanding of key health policies, including Medicare and Medicaid, and his past comments. As the hearings continue, vaccine makers such as GSK, Moderna, Pfizer, and others are under investor scrutiny, as the outcome of these proceedings could have significant implications for vaccine policy and the healthcare sector.
Given his controversial track record, RFK Jr.’s nomination could have major ramifications, not just for his potential confirmation but for U.S. health policy, particularly in areas like vaccine safety and public health. Investors will likely be watching closely, as the shifting political landscape could introduce volatility into vaccine stock prices.
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BIOTECH NEWS
🔹❗ On Jan. 30, Vertex Pharmaceuticals announced the FDA approval of JOURNAVX (suzetrigine), the first non-opioid oral pain signal inhibitor for the treatment of adults with moderate-to-severe acute pain. This approval marks a significant milestone, as Journavx is the first new class of pain medication approved in over 20 years. The drug works by selectively inhibiting NaV1.8, a sodium channel involved in transmitting pain signals, without affecting the brain and thus avoiding the addictive potential of opioids. Journavx is well-tolerated, effective, and suitable for all types of acute pain. The treatment is available as a twice-daily oral pill. Vertex has priced Journavx at $15.50 per 50mg pill and is also investigating its use for peripheral neuropathic pain. This approval provides an alternative to opioids, addressing the growing need for non-addictive pain relief.
🔹 On Jan. 27, the FDA approved the once-every-four-week maintenance dosing of Leqembi (lecanemab-irmb) for the treatment of early Alzheimer’s disease following an 18-month initiation phase with biweekly dosing. This maintenance regimen aims to make long-term treatment easier for patients and caregivers while continuing to slow disease progression. The approval is based on data from the phase 2 and Clarity AD studies, which showed that transitioning to the four-week dosing maintains clinical and biomarker benefits. Leqembi works by clearing amyloid plaque and toxic protofibrils in the brain. The treatment is associated with risks like amyloid-related imaging abnormalities (ARIA), especially in patients with the ApoE ε4 genetic variant.
🔹 On Jan. 27, Sage Therapeutics announced that its Board of Directors has initiated a review of strategic alternatives, including a potential sale, business combination, or transaction. The company rejected an unsolicited acquisition proposal from Biogen, which offered $7.22 per share for the remaining shares it doesn’t already own, deeming the offer significantly undervalued. Sage remains focused on establishing its drug Zurzuvae (co-marketed w/ Biogen) as the standard treatment for postpartum depression. Additionally, Sage is reportedly suing Biogen over a potential breach of a standstill agreement following the proposal.
🔹 On Jan. 24, Moderna announced it has secured a tender to supply its mRNA COVID-19 vaccine to the European Union, Norway, and North Macedonia, which could last up to four years. This agreement will allow 17 participating countries to diversify their vaccine supply and access the vaccine in various formats, including pre-filled syringes.
CLINICAL TRIAL UPDATES
🔹 CARGO Therapeutics announced the discontinuation of its FIRCE-1 phase 2 study of firi-cel, an autologous CD22 CAR T-cell therapy, for relapsed/refractory large B-cell lymphoma. The decision was based on an ad hoc analysis revealing insufficient benefit-risk data due to safety concerns, including a high rate of immune effector cell-associated hemophagocytic lymphohistiocytosis-like syndrome (IEC-HS) in patients. Although firi-cel showed an overall response rate of 77% and a complete response rate of 43%, the durability of responses was low, with only 18% of patients maintaining a complete response after three months. As a result, the company will reduce its workforce by 50% to extend its cash runway, focusing on advancing other programs.
🔹 Alterity Therapeutics announced positive results from its phase 2 trial of ATH434 in patients with early-stage multiple system atrophy (MSA). The study showed that both doses of ATH434 significantly slowed clinical progression. Additionally, ATH434 reduced iron accumulation in key brain regions affected by MSA. The treatment also demonstrated trends in improved motor function and preserved brain volume. ATH434 was well-tolerated, with no serious adverse events reported. The stock surged nearly 110%.
🔹 Leap Therapeutics reported initial clinical data from its DeFianCe and DisTinGuish studies, and the company’s stock plunged 77% in response. In the DeFianCe study, combining sirexatamab (DKN-01) with bevacizumab and chemotherapy showed a 35% objective response rate in second-line colorectal cancer patients, outperforming the control arm’s 23%, with promising data suggesting further phase 3 trials. However, in the DisTinGuish study for gastric cancer, the combination therapy did not meet key progression-free survival endpoints and failed to generate a clear positive signal, leading to the decision to halt phase 3 plans for this indication. Despite the encouraging colorectal cancer data, the lack of success in gastric cancer, combined with uncertainties around the next steps, resulted in the significant drop in stock value.
🔹 Sarepta Therapeutics announced positive results from part 2 study for Elevidys, a gene therapy for Duchenne muscular dystrophy. Crossover patients showed significant improvements in functional tests like NSAA, Time to Rise, and 10MWR compared to an external control group. Patients treated in Part 1 continued to show sustained micro-dystrophin expression and minimal muscle pathology progression over two years. These benefits diverged further from natural disease progression, with no new safety concerns. ELEVIDYS is approved for DMD treatment in patients aged 4 and older in several countries.
🔹 On Jan. 27, Akero Therapeutics reported promising preliminary topline results from its phase 2b study, showing statistically significant reversal of compensated cirrhosis due to metabolic dysfunction-associated steatohepatitis (MASH) in patients treated with efruxifermin. The drug was generally well tolerated, with transient, mild gastrointestinal side effects in some patients. The company aims to further evaluate efruxifermin in its ongoing phase 3 study.
🔹 On Jan. 27, Allakos announced disappointing results from its phase 1 trial of AK006 for chronic urticaria. While the drug was well-tolerated, it did not show therapeutic efficacy, as preclinical results did not translate to clinical benefit. As a result, Allakos will discontinue AK006 development and reduce its workforce by 75%, retaining about 15 employees to explore strategic alternatives and manage administrative tasks. The company’s stock dropped over 75% after the announcement.
ON THE HORIZON
🔹 February FDA PDUFAs:
Feb. 1: Supernus Pharmaceuticals is expecting decision for the apomorphine infusion device (SPN-830) for the continuous treatment of motor fluctuations in Parkinson’s disease.
Feb. 8: Otsuka and Lundbeck expecting to hear from the FDA regarding the combination of brexpiprazole and sertraline for treating PTSD in adults.
Feb. 14: GSK’s 5-in-1 meningococcal ABCWY vaccine candidate.
Feb. 14: Bavarian Nordic is expecting the FDA’s decision for the CHIKV VLP vaccine.
Feb. 28: SpringWorks Therapeutics’ NDA mirdametinib for treating adults and children with NF1-PN.
Have a wonderful rest of your week and thanks for reading Biotech Blueprint!
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