This Week in Biotech #34
Catch up on the latest biotech breakthroughs and upcoming trends (Dec 20-Jan 9).
Welcome to This Week in Biotech by Biotech Blueprint! The first edition of 2025, let’s go!
THIS WEEK’S KEY TAKEAWAYS 🔑
The biotech sector kicked off 2025 with a wave of news spanning clinical trial results, regulatory updates, and M&A deals. Vir Biotechnology celebrated a win with its Covid prevention drug receiving breakthrough therapy designation, while Sana Biotechnology’s stock soared on promising results for its type 1 diabetes treatment. Concerns over bird flu sparked renewed interest in vaccine makers like Moderna and Novavax, highlighting the ongoing importance of infectious disease prevention.
On the flip side, Denali faced a setback with disappointing ALS trial results, and Acelyrin’s stock dropped despite positive data for its thyroid eye disease drug.
Aside from this week’s news, make sure to take a closer look at the key events on the horizon this month. Several companies are awaiting crucial FDA decisions in January, with potential approvals for novel treatments in areas ranging from acute pain to rare genetic disorders. I’m particularly interested in seeing how things unfold for Atara, Vertex, Stealth Bio, and Axsome. Be sure to check out the “On the Horizon” section for a more detailed overview of these upcoming PDUFAs.
Looking ahead, Evaluate Pharma’s 2025 top-selling drug forecast predicts a dynamic year for the industry and significant changes on the chart compared to previous years. Immunology and obesity treatments are expected to drive significant market growth, with established drugs like Stelara and Keytruda maintaining their dominance and newer therapies like tirzepatide and semaglutide poised to become major players. The forecast also anticipates a wave of new drug launches in areas like Alzheimer’s and cancer, which could reshape the treatment landscape and create new opportunities for innovation.
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MARKET UPDATES
🔹 Vir Biotechnology, a clinical-stage immunology company, has announced that the FDA has granted Breakthrough Therapy Designation to VIR-2482 for the prevention of COVID-19 in adults. This designation is based on positive results from a phase 2 study, which showed that a single dose of VIR-2482 provided significant protection against symptomatic COVID-19. The company is now working towards initiating Phase 3 trials. The company’s stock jumped over 50% after the announcement and the company was upgraded at Morgan Stanley to Overweight from Equal Weight.
🔹 Sana Biotechnology’s stock surged over 160% after the company announced positive clinical results from its first-in-human study of an experimental type 1 diabetes (T1D) treatment. The therapy involves transplanting islet cells engineered with Sana’s hypoimmune technology, allowing them to evade immune rejection without the need for immunosuppressive drugs. Early results show the transplanted cells survived, functioned, and produced insulin in a patient with T1D. This breakthrough could potentially lead to a functional cure for T1D, eliminating the need for insulin injections and immunosuppression.
🔹 Shares of vaccine makers like Moderna, Novavax, and CureVac jumped by double-digits after the first U.S. death from bird flu was reported. These companies are developing mRNA vaccines for the H5N1 virus, and the news has increased investor interest in their potential. While the CDC maintains that the risk to the general public is low, the fatality highlights the need for effective vaccines to combat the virus.
🔹 Vaccine manufacturers saw a surge in their stock prices following a report from the CDC highlighting a rise in respiratory illnesses, including flu, COVID-19, and RSV. CureVac experienced the most significant gains, with shares increasing by 16%. Other companies like Moderna, Emergent BioSolutions, BioNTech, and Novavax also saw increases in their stock values. This market activity reflects renewed investor interest in vaccine development and the potential for increased demand for vaccines in light of the rising infection rates.
🔹 Oppenheimer predicts that Viking Therapeutics, a company developing an obesity drug called VK2735, is a likely candidate for a buyout in the biotech sector. Analyst Matthew Biegler believes that increased M&A activity, particularly with a company like Viking, could boost the currently lackluster biotech market. He suggests that large pharmaceutical companies might be interested in acquiring Viking due to its leading position in the development of oral obesity treatments.
BIOTECH NEWS
🔹 IGM Biosciences is halting development of its autoimmune disease drugs imvotamab and IGM-2644 due to disappointing clinical trial results. The company is also implementing a significant restructuring plan, including a 73% reduction in workforce, to preserve cash. IGM Biosciences is now exploring strategic alternatives to maximize shareholder value.
🔹 Roche has successfully acquired Poseida Therapeutics for $9.00 per share in cash ($1.5B), plus potential additional payments. Poseida, a clinical-stage biopharmaceutical company focused on cell therapies and gene editing technologies, will now become a wholly owned subsidiary of Roche.
🔹 On Jan. 7, Annovis Bio announced that the FDA has accepted a streamlined protocol for its pivotal phase 3 Alzheimer’s disease study of buntanetap. Instead of two separate trials, the new protocol combines the symptomatic and disease-modifying assessments into a single 18-month study. This change is expected to accelerate the drug’s development timeline, with potential for an earlier NDA filing based on the 6-month symptomatic data.
🔹 Orna Therapeutics and Vertex Pharmaceuticals have partnered to develop new gene editing therapies for sickle cell disease and beta thalassemia. The collaboration will combine Orna’s expertise in lipid nanoparticle delivery with Vertex’s gene editing technology. Orna will receive $65M upfront and is eligible for over $1B in milestone payments and royalties. This partnership aims to create more effective treatments for these inherited blood disorders.
🔹 On Jan. 3, Regeneron Pharmaceuticals acquired Oxular Limited, a UK-based biotech company specializing in treatments for retinal diseases. Oxular’s portfolio includes OXU-001, a promising drug candidate for diabetic macular edema and other retinal conditions, and Oxulumis, a proprietary drug delivery device. This acquisition expands Regeneron’s ophthalmology portfolio and provides them with innovative technology for treating eye diseases.
🔹 On Dec. 27, BioNTech announced it has reached settlements with the U.S. National Institutes of Health (NIH) and the University of Pennsylvania regarding royalty disputes over its COVID-19 vaccine. BioNTech will pay $791.5M to the NIH and $467 million to UPenn, who claimed underpayment for the use of their mRNA technology. Pfizer, BioNTech’s partner, will reimburse a portion of these payments. The settlements include amended licensing agreements and frameworks for future collaborations, but BioNTech does not admit liability in either case.
🔹 On Dec. 27, the FDA approved BeiGene’s Tevimbra (tislelizumab-jsgr) in combination with chemotherapy for the first-line treatment of advanced gastric or gastroesophageal junction (G/GEJ) cancer in adults. This approval is based on positive results from a phase 3 trial showing improved overall survival in patients treated with Tevimbra plus chemotherapy compared to chemotherapy alone. This marks the second FDA approval for Tevimbra in 2024.
🔹 On Dec. 23, AVITA Medical received FDA approval for RECELL GO mini, a new version of their RECELL GO system designed to treat smaller wounds. This smaller disposable cartridge will make the technology more accessible to clinicians, particularly in trauma and burn centers where smaller wounds are prevalent. RECELL GO mini uses the same technology as the original RECELL system. It’s a spray-on skin cell technology that harnesses the patient’s own skin cells to create a regenerative solution.
🔹 Dec. 20, 2024, was a busy day for FDA decisions:
As I mentioned in last week’s deep dive linked below, the FDA approved Eli Lilly’s Zepbound (tirzepatide) for the treatment of moderate-to-severe obstructive sleep apnea (OSA) in adults with obesity. This marks the first drug specifically approved for OSA and builds on Zepbound’s existing approval for weight loss. In clinical trials, Zepbound significantly reduced breathing interruptions during sleep and helped many participants achieve remission from OSA symptoms. The drug also promoted substantial weight loss, with average reductions of 18-20%.
The FDA approved Rhythm Pharmaceuticals’ Imcivree (setmelanotide) for children as young as 2 with rare genetic obesity disorders caused by Bardet-Biedl syndrome and deficiencies in POMC, PCSK1, or LEPR genes. These conditions lead to insatiable hunger and early-onset obesity. Imcivree works by targeting the MC4R pathway in the brain, which is impaired in these disorders. Clinical trials showed the drug significantly reduces weight and hunger in these patients. This approval makes Imcivree the first precision medicine for these rare diseases in both the U.S. and Europe, offering a much-needed targeted treatment option. However, potential side effects include skin hyperpigmentation, nausea, and headaches.
The FDA also approved Vertex Pharmaceuticals’ Alyftrek, a once-daily cystic fibrosis treatment for patients 6 years+ with at least one responsive mutation in the CFTR gene. This approval marks Vertex’s fifth CFTR modulator and expands treatment options to those with 31 additional mutations not covered by previous therapies. In clinical trials, Alyftrek showed comparable efficacy to Vertex’s existing drug Trikafta while further reducing sweat chloride, a key indicator of cystic fibrosis severity.
Lexicon Pharmaceuticals received a rejection from the FDA for its New Drug Application for Zynquista, a type 1 diabetes treatment. This was anticipated, as Lexicon had already decided to stop pursuing Zynquista’s launch and focus on other drug candidates. Despite this setback, Lexicon remains committed to its clinical development pipeline, particularly LX9211 for diabetic neuropathic pain, with results expected soon.
CLINICAL TRIAL UPDATES
🔹 Viking Therapeutics has started a phase 2 clinical trial to test an oral tablet formulation of VK2735, its drug for obesity. VK2735 works by activating GLP-1 and GIP receptors, which are involved in regulating appetite and blood sugar. This trial will evaluate the safety and effectiveness of the oral tablet in people who are obese or overweight.
🔹 On Jan. 7, Immuneering released positive data from its phase 2a trial of IMM-1-104, a potential new treatment for pancreatic cancer. The drug showed promising response rates when combined with modified gemcitabine/nab-paclitaxel in first-line patients, with an overall response rate of 43% and disease control rate of 86%. IMM-1-104 demonstrated activity as a monotherapy in second-line pancreatic cancer patients, supporting its potential use in combination therapies. Notably, IMM-1-104 continues to show a strong safety profile, differentiating it from other MEK inhibitors. Based on these positive results, Immuneering plans to expand the phase 2a trial with additional combination arms in melanoma and non-small cell lung cancer.
🔹 Arcturus Therapeutics has begun phase 2 clinical trials for two mRNA therapies: ARCT-032 for cystic fibrosis and ARCT-810 for ornithine transcarbamylase (OTC) deficiency. ARCT-032 aims to deliver functional CFTR protein to the lungs of patients who don’t benefit from existing CF treatments. ARCT-810 is designed to enable the liver to produce the OTC enzyme, addressing the root cause of OTC deficiency. Interim data from both trials is expected in the first half of 2025. Read more about what we can expect in the field of mRNA:
🔹 On Jan. 6, Denali Therapeutics announced that its phase 2/3 trial of DNL343 for the treatment of ALS did not meet its primary endpoint of slowing disease progression. While the drug was found to be safe and well-tolerated, it did not significantly improve overall function, muscle strength, or respiratory function in ALS patients compared to a placebo. Further analyses of the data, including biomarker and subgroup analyses, are expected later in 2025.
🔹 On Jan. 6, Acelyrin announced positive phase 2 data for lonigutamab, a potential new treatment for thyroid eye disease (TED). The drug showed promising efficacy in reducing eye bulging and other TED symptoms, with a potentially better safety profile than current treatments. Based on these results, Acelyrin has finalized its phase 3 trial program, which is expected to begin soon. The company believes lonigutamab could become a preferred treatment option for TED, offering improved efficacy and safety with a more convenient subcutaneous administration. Acelyrin’s stock dropped by over 30% after the update.
🔹 On Jan 2., Neumora Therapeutics announced disappointing results from its phase 3 KOASTAL-1 study of navacaprant, a potential new treatment for major depressive disorder (MDD). The study did not meet its primary endpoint of significantly reducing depressive symptoms compared to a placebo. However, the company noted a potential efficacy signal in female participants and plans to further analyze the data. Navacaprant was generally well-tolerated with a safety profile comparable to the placebo. Despite this setback, Neumora remains committed to developing navacaprant and will share further updates at the J.P. Morgan Healthcare Conference.
🔹 On Dec. 20, Novo Nordisk announced results from their REDEFINE 1 trial for CagriSema, a new weight loss drug combining cagrilintide and semaglutide. While the trial showed CagriSema led to superior weight loss compared to either drug alone or a placebo, the results fell short of expectations. Participants taking CagriSema achieved an average weight loss of 20.4%, with some achieving a reduction of 25% or more. However, this fell short of the anticipated 25% average weight loss, causing Novo Nordisk’s stock to drop significantly. Despite this, the drug was found to be safe and well-tolerated, with mostly mild to moderate side effects. Novo Nordisk plans to conduct a new trial with optimized dosing to further explore CagriSema’s potential for weight loss.
PUBLIC HEALTH SPOTLIGHT
🔹 Despite alarming headlines and social media posts about a surge in human metapneumovirus (HMPV) cases in China, public health experts say there is no cause for panic. HMPV is a common respiratory virus that typically causes mild cold-like symptoms, and the current situation in China appears to be within the normal range of seasonal infections. The heightened anxiety surrounding HMPV reflects lingering fears from the COVID-19 pandemic, with any reports of illness in China now attracting outsized attention. Experts emphasize that HMPV is not a new or particularly dangerous virus, and the best prevention measures remain the same as for other respiratory illnesses: handwashing, covering coughs and sneezes, and avoiding contact with sick individuals.
🔹 On Jan. 6, Louisiana health officials reported the first human death in the U.S. related to the H5N1 bird flu. The individual, who was over 65 and had underlying health conditions, contracted the virus after exposure to both backyard poultry and wild birds. While officials stressed that the risk to the general public remains low, they advised those working with birds to take precautions. This case highlights the importance of avoiding contact with sick or dead birds and practicing safe handling of poultry products.
🔹 The CDC recently reported mutations in the H5N1 bird flu virus, identified in a critically ill patient in Louisiana, which may enhance the virus’s ability to bind to human upper airway cells. This mutation was also found in a case from Canada. These changes likely developed during the patients’ infections rather than being present in the initial virus from infected animals, suggesting they arose through viral replication in humans. While these mutations raise concerns, experts caution that the current public health risk remains low, as the virus still requires additional changes to enable person-to-person transmission.
As of Dec. 2024, the U.S. has reported 66 confirmed human H5N1 cases, primarily linked to exposure through poultry or dairy cattle, with no evidence of sustained human-to-human transmission. The virus is widespread in wild birds, with sporadic outbreaks in poultry and dairy herds, but the CDC continues to monitor the situation closely. Public health officials emphasize precautions, particularly for those in close contact with infected animals, though no travel restrictions are currently advised. While genetic mutations in H5N1 are being closely watched, the risk of a pandemic remains low at this time.
ON THE HORIZON
🔹 January 2025 is packed with FDA PDUFAs. I’m especially keeping a close eye on Atara, Vertex, Stealth and Axsome:
Jan. 2: ✅ APPROVED: As mentioned above, on Dec. 20, the FDA accepted Vertex Pharmaceuticals’ New Drug Application for Alyftrek, a new cystic fibrosis treatment (even though the PDUFA was set for Jan. 2). This triple combination therapy is designed to improve the function of the CFTR protein, which is defective in people with CF.
Jan. 15: Atara Bio’s tab-cel, a potential treatment for a rare complication affecting transplant patients, is awaiting FDA approval by January 15th. If approved, it would be the first therapy in the U.S. for this condition. The drug has shown promising results in clinical trials and has already been approved in Europe. Read more about tab-cel in my most recent deep dive:
Jan. 17: The FDA has delayed its decision on Amgen’s application for sotorasib plus panitumumab in patients with KRAS G12C-mutated metastatic colorectal cancer. The target action date moved from Dec. 24, 2024 to Jan. 17, 2025. This delay allows the FDA more time to review recently submitted data. The application is based on the CodeBreaK 300 trial, which showed the combination improved progression-free survival compared to standard therapies.
Jan. 29: Stealth BioTherapeutics is awaiting decision on its New Drug Application for elamipretide, a potential treatment for Barth syndrome. If approved, elamipretide would be the first therapy available for this rare genetic condition, which causes severe heart and muscle problems.
Jan. 29: AstraZeneca and Daiichi Sankyo have submitted a new application for accelerated approval of datopotamab deruxtecan in the U.S. for patients with previously treated advanced EGFR-mutated non-small cell lung cancer. This new application is based on results from the TROPION-Lung05 phase 2 trial and replaces a previous application that has been withdrawn. The companies made this decision based on feedback from the FDA, suggesting a more targeted approach for this specific lung cancer subtype.
Jan. 30: Vertex also awaits an FDA decision on suzetrigine, a potential non-opioid pain medication, by Jan. 30. If approved, suzetrigine would be the first new pain drug in over two decades, offering a non-addictive option for acute pain.
Jan. 31: Axsome Therapeutics expects an FDA decision by Jan. 31 on its resubmitted application for AXS-07, a new oral treatment for acute migraine. The drug offers a potential non-opioid option for migraine relief by inhibiting CGRP release and providing sustained pain freedom. If approved, AXS-07 would be a valuable addition to Axsome’s growing portfolio of neurological treatments.
Have a wonderful rest of your week and thanks for reading Biotech Blueprint!
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Very informative and great insights about certain diseases