This Week in Biotech #40
Catch up on the latest biotech breakthroughs and upcoming trends (Feb 14-20).
Welcome to This Week in Biotech by Biotech Blueprint. I am excited to bring you this week’s edition, with a special highlight: don’t miss the newly revamped ON THE HORIZON section, featuring the latest biotech IPOs, mergers, and key FDA updates.
THIS WEEK’S KEY TAKEAWAYS 🔑
In January 2025, Moderna retained its position as the most shorted healthcare stock in the S&P 500, with short interest rising to 11.84%. This came amid a broader increase in short activity within the biotech sector. Goldman Sachs, however, remains bullish on biotech, advising investors to focus on stocks driven by company-specific factors rather than broader market trends. Moderna, with its pioneering work in mRNA technology, is seen as a key candidate for returns, even as volatility persists within the sector.
Meanwhile, biotech innovation is making strides. Dynavax Technologies’ HEPLISAV-B vaccine for hepatitis B captured 44% of the market in 2024, with further growth expected as it targets underserved populations. Zealand Pharma’s advancements in obesity treatments, such as its dual GLP-1/GLP-2 agonist dapiglutide, and Incyte’s partnership with Genesis Therapeutics to harness AI for drug discovery, underscore the sector’s continuing transformation.
The biotech landscape this week is also marked by regulatory approvals and market movements. Namely, Bavarian Nordic’s approval of the Vimkunya chikungunya vaccine by the FDA highlights the growing importance of tropical disease solutions. Solid Bio’s stock surged nearly 90% following the release of promising initial clinical data for SGT-003, a next-generation gene therapy for Duchenne muscular dystrophy.
As several promising treatments await FDA verdicts in March, including Vutrisiran for ATTR amyloidosis, GSK’s novel antibiotic Gepotidacin for UTIs and Milestone’s Etripamil nasal spray to treat paroxysmal supraventricular tachycardia, the biotechnology sector remains a dynamic and lucrative space for both innovation and investment.
🎙️ PODCAST
MARKET UPDATES
🔹 In January 2025, Moderna (MRNA) remained the most shorted healthcare stock in the S&P 500, with its short interest rising to 11.84% from 11.03% in December. The S&P 500 Health Care Index saw a slight increase in short interest, reaching 1.60% compared to 1.58% in December. The biotechnology sector continued to lead as the most shorted within the healthcare index, with short interest rising to 2.45%. The healthcare provider and services sector came second at 1.90%, while pharmaceuticals had the lowest short interest at 1.28%.
Stocks with the highest short interest in January were:
Moderna (11.84%)
DaVita (5.81%)
ResMed (5.65%)
On the other hand, the least shorted stocks included:
Boston Scientific (0.71%)
Danaher Corp and Johnson & Johnson (both at 0.78%).
In January, the Health Care Select Sector SPDR Fund (XLV) gained 4.6%, slightly underperforming the broader S&P 500, which rose by 4.9%.
🔹 Goldman Sachs recommends that investors aiming for alpha focus on stocks with high volatility and company-specific drivers, particularly in the biotech sector, alongside consumer discretionary and IT. They identify Moderna (MRNA) as one of the top biotech stocks with a high dispersion score, indicating that its performance is more influenced by company-specific factors rather than broader market trends. This focus on “micro-driven” volatility, where stock movements are due to individual company factors rather than macroeconomic forces, makes biotech, especially Moderna, a potential area for high returns as it continues to innovate and respond to market needs. The bottom line is that biotech industry, led by companies like Moderna, is expected to remain a fertile ground for stock-picking opportunities in 2025 due to ongoing technological advancements and product adoption.
BIOTECH NEWS
🔹 Dynavax Technologies’ HEPLISAV-B vaccine, a leading hepatitis B vaccine in the U.S., saw strong growth in 2024, with net product revenue rising 26% year-over-year to $268M. The vaccine continues to capture a larger share of the market, reaching approximately 44% by the end of 2024, up from 42% in 2023. The expanding hepatitis B market in the U.S. is expected to reach over $900M annually by 2030, with Dynavax projecting HEPLISAV-B will secure at least 60% of the market share. This growth is fueled by the penetration of the unvaccinated adult population and the continued practice of revaccination. For 2025, Dynavax anticipates HEPLISAV-B revenue to increase to $305-$325M. This reflects the ongoing market expansion and the vaccine’s increasing adoption among adults aged 19-59, a group universally recommended for vaccination in the U.S. Beyond 2025, Dynavax expects HEPLISAV-B’s market potential to remain strong, benefiting from a broadening eligible population and consistent healthcare provider use, positioning the product for continued success beyond 2030.
🔹 Zealand Pharma announced its financial results for the full year of 2024, marking a transformative year with significant advancements in its obesity pipeline. Key highlights include positive phase 1b results for its long-acting amylin analog, petrelintide, and its GLP-1/GLP-2 receptor dual agonist, dapiglutide. Zealand is moving forward with large phase 2b trials for its obesity programs and has expanded its trials for key drug candidates. The company also made strides in rare diseases, particularly with dasiglucagon for congenital hyperinsulinism and glepaglutide for short bowel syndrome. Financially, Zealand Pharma’s cash position has significantly improved, allowing for increased investment in R&D in 2025, with a focus on next-generation peptide therapeutics.
🔹 UroGen Pharma has expanded its oncology pipeline by acquiring ICVB-1042, a next-generation oncolytic virus from IconOVir Bio. ICVB-1042 is designed to selectively destroy cancer cells while triggering a robust immune response, offering potential advantages over current treatments for bladder cancer. UroGen also announced multiple strategic research collaborations to explore the use of its RTGel technology to enhance the effectiveness of various immunotherapies. This acquisition and research push are part of UroGen’s long-term strategy to drive innovation in cancer treatment and strengthen its leadership in oncology.
🔹 Incyte and Genesis Therapeutics have entered a strategic collaboration to discover and develop novel small molecule therapeutics using Genesis’ AI platform, GEMS (Genesis Exploration of Molecular Space). The partnership will focus on Incyte-selected targets, leveraging AI technologies to optimize and identify breakthrough compounds. Incyte will have exclusive rights to develop and commercialize the resulting products. Genesis will receive a $30M upfront payment and could earn up to $295M in milestone payments per target, along with royalties on sales. This collaboration aims to accelerate the discovery of critical treatments for patients with severe diseases.
🔹 On Feb. 19, ImmunityBio has received FDA approval for an expanded access program to address the shortage of Tice BCG in the U.S. by providing urologists with an alternative source, recombinant Bacillus Calmette-Guérin (rBCG). Developed in collaboration with the Serum Institute of India, rBCG has demonstrated improved immunogenicity and safety in phase 2 clinical trials for non-muscle invasive bladder cancer (NMIBC). With shipments set to begin immediately, rBCG is expected to help alleviate the supply challenges for bladder cancer patients.
🔹 Alvotech and Teva Pharmaceuticals announced that the FDA has accepted their Biologics License Application for AVT06, a biosimilar to Regeneron’s Eylea (aflibercept), a treatment for eye disorders that can cause vision loss. This milestone brings the companies closer to offering a more accessible and cost-effective treatment option for patients. The regulatory process is expected to conclude by Q4 2025. Alvotech’s AVT06 and its other candidate, AVT29 (for Eylea HD), are part of their broader pipeline of biosimilars. AVT06 demonstrated therapeutic equivalence and safety in clinical trials. The development aims to provide alternatives to the widely-used Eylea product, which saw sales of $4.77B in 2024.
🔹 Biogen and Stoke Therapeutics have entered a collaboration to develop and commercialize zorevunersen, a potential first-in-class treatment for Dravet syndrome, a rare genetic epilepsy disorder. Zorevunersen targets the SCN1A gene, the primary cause of Dravet syndrome. Under the agreement, Stoke will retain exclusive rights for zorevunersen in the U.S., Canada, and Mexico, while Biogen will have commercialization rights in other regions. Stoke will receive an upfront payment of $165M, shared development costs, and may earn up to $385M in milestone payments, along with royalties. Biogen’s global expertise in rare diseases and commercialization will help advance zorevunersen, which has shown promising results in reducing seizures and improving cognitive and behavioral outcomes in early studies. A phase 3 clinical trial, EMPEROR, is set to begin in Q2 2025, with results expected by late 2027. Dravet syndrome currently has no disease-modifying therapies, and this collaboration aims to meet a significant unmet need for better treatments. Zorevunersen has received FDA Breakthrough Therapy Designation based on its potential to improve on existing therapies.
🔹❗Bavarian Nordic announced that the FDA has approved Vimkunya, the first single-dose chikungunya vaccine for people aged 12 and older. This approval follows positive results from two phase 3 trials involving over 3,500 individuals. The vaccine showed up to 97.8% efficacy in inducing neutralizing antibodies within 21 days of vaccination. Vimkunya is a virus-like particle vaccine, which mimics the chikungunya virus without causing disease. The vaccine is expected to be commercially available in the U.S. by mid-2025. Bavarian Nordic also received a Tropical Disease Priority Review Voucher from the FDA. Vimkunya has also received positive opinions from the European Medicines Agency and is on track for launch in Europe in 2025. Chikungunya is a mosquito-borne illness, emerging across regions like Asia, Africa, and the Americas. The vaccine offers protection for travelers and those in affected regions. Bavarian Nordic aims to address this growing health challenge with the approval of Vimkunya.
CLINICAL TRIAL UPDATES
🔹 SELLAS Life Sciences announced positive results from a phase 2a trial of SLS009 (tambiciclib), a CDK9 inhibitor, in combination with zanubrutinib for relapsed/refractory Diffuse Large B-Cell Lymphoma (r/r DLBCL). The combination achieved a 67% overall response rate, significantly higher than zanubrutinib alone, and an 83% disease control rate in difficult-to-treat ABC DLBCL patients. Notably, one patient achieved complete response, and three had partial responses. After a median follow-up of 4.6 months, median overall survival was not reached, with six of the nine patients still alive. The results highlight the potential of SLS009 in improving outcomes for DLBCL patients, especially those with non-GCB subtypes.
🔹 Assembly Biosciences reported positive interim results from its ongoing phase 1a/b clinical trial of ABI-1179, an investigational long-acting helicase-primase inhibitor for recurrent genital herpes. ABI-1179 demonstrated a half-life of approximately four days, supporting the potential for once-weekly oral dosing. The drug was well-tolerated, with no serious adverse events observed. Based on these promising results, Assembly Bio will move ABI-1179 into the phase 1b portion of the study. Interim data are expected in fall 2025.
🔹 Precision BioSciences has announced promising initial results from its ongoing ELIMINATE-B phase 1 clinical trial, which is investigating PBGENE-HBV, a gene-editing therapy for chronic Hepatitis B. In the first cohort of three patients, the lowest dose was safe and well tolerated, with no serious adverse events reported. Additionally, two of the patients showed a substantial reduction in Hepatitis B surface antigen after just one administration. This early success demonstrates the potential of PBGENE-HBV to directly target and eliminate the root cause of Hepatitis B, namely the covalently closed circular DNA (cccDNA) and integrated HBV DNA. With additional doses planned and further patient recruitment underway, Precision BioSciences is optimistic about the potential of PBGENE-HBV to offer a functional cure for this widespread and debilitating disease.
🔹 Solid Biosciences’ stock surged nearly 90% following the release of promising initial clinical data for SGT-003, a next-generation gene therapy for Duchenne muscular dystrophy. The phase 1/2 INSPIRE DUCHENNE trial showed that SGT-003 achieved an average 110% microdystrophin expression in the first three participants, along with significant improvements in muscle health biomarkers and signs of cardiac benefit. The therapy was well-tolerated with no serious adverse events, and enrollment is ongoing with plans to dose 20 participants by Q4 2025.
🔹 Moderna reported a clinical hold on its Norovirus vaccine (mRNA-1403) following a single adverse event of Guillain-Barré syndrome. The incident is currently under investigation. Despite this hold, Moderna clarified that the ongoing phase 3 study is not expected to experience delays in efficacy readouts, as enrollment for the Northern Hemisphere has already been completed. The timing for phase 3 results will depend on case accruals, with the company continuing to work through the investigation with the FDA. This is part of a broader set of updates Moderna provided in its Q4 2024 financial report, including financial losses and expectations for 2025.
🔹 On Feb. 14, Corbus Pharmaceuticals presented promising phase 1 data for its next-generation antibody-drug conjugate targeting cancers that express Nectin-4, CRB-701, at the 2025 ASCO Genitourinary Cancers Symposium. The data, from a study conducted in the U.S. and U.K., showed good safety with no dose-limiting toxicities and minimal adverse effects, such as low rates of peripheral neuropathy and skin toxicity. Additionally, encouraging clinical responses were observed in multiple tumor types, including head and neck squamous cell carcinoma, cervical cancer, and urothelial cancer. Despite a 10% stock drop today, following a 60% decline in September 2024 after concerns about its obesity drug CRB-913, the data strengthens the outlook for CRB-701, which is being developed as a targeted treatment for cancers expressing Nectin-4.
PUBLIC HEALTH SPOTLIGHT
🔹 The CDC’s Advisory Committee on Immunization Practices (ACIP) meeting, originally scheduled for February 26-28, 2025, has been postponed. This meeting would have been the first since Robert F. Kennedy Jr. became the U.S. Secretary of Health and Human Services. HHS cited the need for public comment before the meeting, but did not specify when it would be rescheduled. The postponement has raised concerns, particularly from vaccine experts and pharmaceutical companies, as the meeting included critical discussions on vaccine recommendations for meningococcal, chikungunya, and influenza vaccines. This delay could potentially impact insurance coverage for these vaccines. Additionally, it could fuel tensions between Kennedy and Senator Bill Cassidy, who had previously received assurances about changes to vaccine programs under Kennedy’s leadership.
🔹 The Trump administration has launched the “Make America Healthy Again Commission,” chaired by RFK, with a focus on assessing and potentially altering the use of psychiatric medications for children. This initiative, established through an executive order, seeks to evaluate the prevalence of and concerns around prescribing drugs like SSRIs, antipsychotics, stimulants, and mood stabilizers. The order calls for reports on these practices within 100 days and proposes recommendations within 180 days. While some medical professionals agree with addressing overprescribing, concerns have been raised about how such a federal initiative might disrupt the patient-physician relationship and limit access to vital medications for children, particularly in light of the growing youth mental health crisis. The commission's actions could significantly impact treatments for conditions like ADHD, depression, and anxiety, which millions of children currently rely on.
ON THE HORIZON
🔹 Upcoming biotech IPOs:
Feb. 21: Apimeds Pharmaceuticals US, Inc. (APUS) is a clinical-stage biopharmaceutical company focused on developing Apitox, a bee venom-based toxin administered intradermally with potential therapeutic benefits. Currently marketed in South Korea as Apitoxin, the drug has been approved by the Korean Ministry of Food and Drug Safety to treat osteoarthritis pain and mobility issues. Apimeds is now advancing its development in the U.S., targeting knee pain in osteoarthritis patients who have not responded to conventional treatments. The company is offering 4.5M shares in its upcoming IPO, priced between $4.00 and $5.00 per share.
Feb. 21: Danam Health, Inc. will go public with an IPO on Feb. 21 on the NASDAQ exchange under the ticker WGRX. The company plans to price shares between $4.50 and $5.50, with a 180-day insider lock-up period ending on Aug. 20, 2025. Danam Health is a holding company with a portfolio focused on pharmaceuticals and healthcare services, including a pharmacy, wholesale operations, and a technology division offering a novel platform for hub and clinical services.
🔹 Upcoming and recent biotech mergers (transaction dates):
Feb. 7: TuHURA Biosciences completed a merger with Kineta, Inc., following the definitive agreement made on Dec. 12, 2024. The merger integrates Kineta’s novel cancer immunotherapy, KVA12123, into TuHURA’s pipeline. KVA12123, a VISTA-blocking antibody, is being developed for cancers like AML and has shown strong preclinical results.
Feb. 18: Cara Therapeutics and Tvardi Therapeutics announced a merger agreement to create a clinical-stage biopharmaceutical company focused on fibrosis-driven diseases. Under the agreement, Cara’s stockholders will hold 17% of the combined company, while Tvardi’s investors will hold 83%. Tvardi’s lead product, TTI-101, targets STAT3 and is being tested in phase 2 trials for idiopathic pulmonary fibrosis and hepatocellular carcinoma.
🔹 February 2025 FDA PDUFAs:
Feb. 14: GSK’s 5-in-1 meningococcal ABCWY vaccine candidate. ✅ APPROVED
Feb. 14: Bavarian Nordic is expecting the FDA’s decision for the CHIKV VLP vaccine. ✅ APPROVED
Feb. 28: SpringWorks Therapeutics’ NDA mirdametinib for treating adults and children with NF1-PN. ✅ APPROVED
🔹 March 2025 FDA PDUFAs:
Mar. 18: NT-501, developed by Neurotech Pharmaceuticals, is a treatment designed for Macular Telangiectasia Type 2 (MacTel), a rare neurodegenerative eye disease that causes progressive central vision loss. NT-501 uses a cell-based delivery system, known as Encapsulated Cell Therapy, to provide sustained release of ciliary neurotrophic factor, a neuroprotective protein that supports photoreceptor survival and slows retinal degeneration.
Mar. 23: Vutrisiran is an investigational RNAi therapeutic being developed by Alnylam for the treatment of ATTR amyloidosis with cardiomyopathy (ATTR-CM), a serious and progressive disease characterized by misfolded transthyretin proteins that accumulate in tissues such as the heart. This therapy works by reducing both mutant and wild-type transthyretin (TTR) proteins, addressing the underlying cause of ATTR amyloidosis. If approved, vutrisiran would be the first therapy available to treat both the polyneuropathy and cardiomyopathy manifestations of this disease.
Mar. 26: Gepotidacin is a novel, oral antibiotic developed by GSK that is currently under investigation for the treatment of uncomplicated urinary tract infections (UTIs) and gonorrhea.
Mar. 27: Milestone’s Etripamil nasal spray, with a PDUFA date of Mar. 27, is designed to be self-administered as a nasal spray to rapidly treat episodes of paroxysmal supraventricular tachycardia (PSVT), a type of abnormal heart rhythm, allowing patients to manage their symptoms at home without immediate medical intervention; it works by blocking calcium channels in the heart to slow down the rapid heart rate.
In one of my January 2025 deep dives where I discussed some of the most exciting drugs that could potentially receive FDA approval this year, I covered both gepotidacin and etripamil:
Mar. 28: Sanofi’s Fitusiran is an investigational therapy for hemophilia A or B, used to prevent bleeding in patients, including those with inhibitors. It works by lowering antithrombin to enhance blood clotting, and is administered subcutaneously, potentially reducing treatment frequency to just six doses a year.
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This is exactly the kind of deep dive the biotech industry needs! Moderna’s short interest spike is a fascinating trend especially when paired with Goldman’s bullish outlook. The biotech sector thrives on innovation, but volatility makes it a playground for risk tolerant investors.
And AI driven drug discovery like incyte and genesis therapeutics seems to be gaining serious traction. Any thoughts on which biotech firms are leading the AI charge in 2025?