This Week in Biotech #42
Catch up on the latest biotech breakthroughs and upcoming trends (Feb 28-Mar 6).
Welcome back to This Week in Biotech by Biotech Blueprint, edition 42.
THIS WEEK’S KEY TAKEAWAYS 🔑
After several weeks of challenges, Moderna is finally showing signs of positive momentum! Despite a 7% stock decline last week amid concerns over its $590M bird flu vaccine contract potentially being scrapped by the Trump administration, there’s good news ahead.
On Mar. 3, Moderna CEO Bancel purchased approximately $5M worth of company stock. Insider buying at this scale sends a message about leadership’s belief in the company’s future. Further, Moderna’s partnership with Merck on personalized cancer vaccines continues to make strides. Their lead candidate has completed enrollment for phase 3 trials in skin cancer, with a potential market entry in 2027. Moderna is expanding its reach beyond melanoma, planning to enter non-small cell lung cancer, renal cancer, and bladder cancer, creating exciting opportunities for revenue diversification beyond infectious disease treatments.
Here are some other interesting developments from this week:
Walgreens' $10B acquisition by Sycamore Partners marks the end of its 100-year run in the stock market.
Biotech stocks with negative enterprise values, such as Acelyrin, Sutro Biopharma, and Voyager Therapeutics, are still attracting strong buy ratings despite the sector’s underperformance.
Plus Therapeutics received FDA Orphan drug designation for its Rhenium treatment, leading to a remarkable 300% increase in its stock price.
A German court ruled that BioNTech and Pfizer violated Moderna’s COVID-19 vaccine patent, requiring them to pay damages.
Jazz Pharmaceuticals announced a $935M acquisition of Chimerix, adding a promising treatment for rare brain tumors to its portfolio.
AbbVie entered the obesity treatment market through a license agreement with Gubra to develop GUB014295, a long-acting amylin analog.
A growing measles outbreak in Texas (140+ cases) and the abrupt resignation of HHS Assistant Secretary Tom Corry after clashing with RFK Jr. over the response.
For more biotech and pharma insights, check out the full This Week in Biotech #42 or listen to our AI-generated podcast below.
Happy Friday!
🎙️ PODCAST
MARKET UPDATES
🔹 Walgreens has finalized a $10B deal to be acquired by Sycamore Partners, a private equity firm specializing in retail investments. Under the agreement, Walgreens shareholders will receive $11.45 per share in cash, with additional value from future monetization of the company’s debt and equity interest in VillageMD. The deal includes Walgreens’ various assets, such as its specialty pharmacy unit and pharmacy benefit manager (PBM). This marks the end of Walgreens’ 100-year presence in the stock market, following a series of struggles in the e-commerce-driven retail environment. The company reported revenue of $147B for fiscal year 2024 but also posted a loss per share of $10. This acquisition mirrors the trend of declining standalone retail pharmacy giants, with competitors like Rite Aid already transitioning to private ownership.
🔹 Gilead Sciences (GILD) was one of the top percentage gainers in the S&P 500 for Feb. 2025, with a notable increase of 19.6%. This growth came despite the overall market’s decline, influenced by various economic factors, including trade tensions and tariff actions by the Trump administration. Gilead’s performance was among the strongest in the index, which saw mixed results across sectors, with the healthcare and biotechnology sectors contributing to its positive momentum.
🔹 Despite biotech stocks facing underperformance in recent years, some companies with negative enterprise values are still attracting strong buy ratings from Wall Street. These companies, which include Acelyrin (SLRN), Sutro Biopharma (STRO), and Voyager Therapeutics (VYGR), are trading at values where their market capitalization is lower than their net assets. Investors could potentially buy these companies and end up with more cash than the cost of acquisition, given their negative enterprise values. Despite the general sector struggles, these stocks have garnered interest due to their potential, with a focus on those with market caps over $100M and positive ratings from analysts.
🔹 Over the past year, Aurinia Pharmaceuticals (AUPH) has experienced notable insider trading activity, with a total of 42 trades, including 20 open market buys and 22 sells. Insiders have traded over 5.2M shares, with 4.2M shares bought and 1.1M shares sold, resulting in a net positive activity of approximately 3.1M shares bought more than sold. On Mar. 5, Director Kevin Tang purchased 748,038 shares at $7.92, increasing his holdings to 10M shares. The company’s stock was up over 7% as a result.
🔹 Moderna CEO Stéphane Bancel bought approximately $5M worth of the company’s stock on Mar. 3, 2025. He acquired 102,821 shares at an average price of $31.04, totaling around $3.2M, and a second batch of 57,493 shares at $31.53, valued at about $1.8M. These purchases were made through Boston Biotech Ventures, a firm where Bancel holds the majority of equity. Following the purchases, Boston Biotech Ventures now owns 6.6M Moderna shares. Moderna’s stock rose about 4% in after-hours trading.
BIOTECH NEWS
🔹 Marty Makary, Johns Hopkins physician and Trump’s nominee for FDA commissioner, appeared before the Senate’s Health, Education, Labor, and Pensions Committee yesterday. While he did not make firm commitments on several issues, he did say he would convene a committee of vaccine advisers but refrained from promising to reschedule a canceled meeting on flu vaccine composition. Makary kept his stance in line with Trump administration policies on vaccines, abortion pills, and government staff cuts. Despite past controversial views, including opposing vaccine mandates, Makary stressed his belief in the benefits of vaccination. Makary is expected to be confirmed for the FDA role. The FDA, under scrutiny for job cuts and a measles outbreak, will face additional challenges, including staff reductions and controversies over vaccine safety and mifepristone.
🔹 Plus Therapeutics (PSTV) has been granted Orphan Drug Designation by the FDA for its investigational treatment, Rhenium (186Re) Obisbemeda, for leptomeningeal metastases (LM) in patients with lung cancer. This milestone follows the completion of the ReSPECT-LM phase 1 trial, which established the recommended phase 2 dose. The orphan designation provides benefits such as market exclusivity, tax credits, and fee exemptions. The company’s stock has risen by over 300% following the announcement. Additionally, Plus continues to advance its clinical trials for LM and recurrent glioblastoma, with ongoing support from the National Cancer Institute and the Cancer Prevention & Research Institute of Texas.
🔹 Novo Nordisk is exploring the potential of GLP-1 drugs, such as Ozempic, Wegovy, and Zepbound, to help patients struggling with addiction, according to Martin Holst Lange, the company’s head of development. These drugs, which are primarily used for diabetes management and weight loss, help control blood sugar levels and induce feelings of fullness. Novo Nordisk is now investigating whether GLP-1 drugs could also provide benefits in addiction treatment, in addition to their current medical uses.
🔹 A German court ruled on Mar. 5 that BioNTech and Pfizer violated a COVID-19 vaccine patent held by Moderna. The ruling came after Moderna asserted that BioNTech and Pfizer infringed on its patent rights during the development of their COVID-19 vaccine. The court found that while Moderna initially authorized the use of the patent, it revoked this permission on Mar. 7, 2022, not in May 2023 as claimed by BioNTech and Pfizer. As a result, BioNTech and Pfizer must pay damages for using the patent in the 14 months after the revocation. The exact amount of damages has yet to be determined, and the defendants still have the option to appeal the decision.
🔹 Moderna, in collaboration with Merck, plans to release a personalized cancer vaccine by 2027. The vaccine is currently in late-stage trials for skin cancer, with the phase 3 study fully enrolled as of Sept. 2024. Moderna’s president, Stephen Hoge, expressed optimism about the potential revenue from the vaccine, highlighting its market potential, especially in melanoma where significant improvements over existing treatments like Keytruda have been observed. Following the approval of the skin cancer vaccine, Moderna plans to expand its application to non-small cell lung cancer, renal cancer, and bladder cancer. The vaccine’s minimum efficacy target is set at 50%, with hopes of exceeding that in final trials.
🔹 Amgen has initiated two key late-stage trials for its experimental weight loss drug, MariTide, as it looks to enter the growing obesity treatment market. MariTide is a monthly injectable designed to compete with current weekly injectables from Novo Nordisk and Eli Lilly. One trial involves 3,500 people with obesity or those overweight without type 2 diabetes, while the second targets 999 individuals with both obesity and type 2 diabetes. The trials aim to measure weight loss over 72 weeks using different doses of the drug. In previous trials, MariTide helped patients lose up to 20% of their weight on average after a year, showing no plateau.
🔹 Merck’s new injected version of its cancer drug Keytruda may face a patent challenge from biotech company Halozyme Therapeutics. Halozyme claims that the injected version infringes on its patents related to drug delivery technology. A potential dispute could impact Merck’s efforts to drive growth with a more user-friendly version of Keytruda, especially as key patents on the original intravenous version expire. The injected version, which is still in clinical testing, is expected to launch by early 2026. Merck has partnered with Alteogen to develop the injectable form using hyaluronidase enzymes, but the two companies are facing competition from Halozyme, which uses similar technology. Merck has contested the validity of Halozyme’s patents and filed petitions with the U.S. patent office.
🔹 Jazz Pharmaceuticals has announced its acquisition of Chimerix for $935M, or $8.55 per share in cash. The deal, which has been approved by both companies, is expected to close in Q2 2025. This acquisition will add dordaviprone, a promising treatment for H3 K27M-mutant diffuse glioma (a rare brain tumor) with no FDA-approved therapies, to Jazz’s oncology portfolio. Dordaviprone is currently under FDA review for accelerated approval, with a target decision date of Aug. 18, 2025. This acquisition strengthens Jazz’s position in the rare oncology market and provides a near-term commercial opportunity if dordaviprone is approved.
🔹 Novo Nordisk has entered into a collaboration with the AI-driven protein design company Gensaic to develop tissue-targeted therapies for cardiometabolic diseases. The deal is valued at up to $354M, though financial details remain undisclosed. The partnership will leverage Gensaic's AI-guided ligand discovery platform, FORGE, which aims to improve precision drug delivery by identifying ligands that enable targeted treatment delivery to specific tissues, thus enhancing drug effectiveness and minimizing side effects. Gensaic’s platform aims to address the challenge of delivering therapies to tissues beyond the liver, a key issue in current drug development. Novo Nordisk sees significant potential in tissue targeting for treating various diseases. The partnership builds on Novo’s previous investments in advancing drug delivery technologies, such as a $600M deal with NanoVation in 2024 for RNA delivery targeting cells outside the liver.
🔹 On Mar. 4, the FDA placed a clinical hold on BioNTech’s early-to-mid stage trial for its experimental malaria RNA-based vaccine, BNT165e. The vaccine, designed to prevent malaria caused by the Plasmodium falciparum parasite, was being tested in healthy adults who have never had malaria. Following the announcement, BioNTech’s U.S.-listed shares dropped by 1.5%. The company has stated it is working to address the FDA’s requests and will collaborate with the regulator to determine the next steps.
🔹 PepGen has decided to pause its phase 2 CONNECT2-EDO51 study of PGN-EDO51 in Duchenne muscular dystrophy patients. This decision comes as the company focuses on reviewing data from the 10 mg/kg cohort, with results expected in Q3 2025. No new safety issues have been reported in the PGN-EDO51 program. PepGen aims to assess additional safety data, evaluate the impact of PGN-EDO51 on dystrophin levels, and possibly refine the CONNECT2 study design. PGN-EDO51, an exon-skipping oligonucleotide therapy, has received Orphan Drug and Rare Pediatric Disease Designations from the FDA.
🔹 On Mar. 4, the FDA approved BeiGene’s Tevimbra (tislelizumab-jsgr) for the first-line treatment of adults with unresectable or metastatic esophageal squamous cell carcinoma (ESCC) whose tumors express PD-L1 (≥1). This approval follows results from the global phase 3 RATIONALE-306 trial, which showed that Tevimbra in combination with chemotherapy significantly improved overall survival compared to chemotherapy alone in PD-L1 positive patients. The median overall survival for patients treated with Tevimbra was 16.8 months, a 34% reduction in the risk of death compared to 9.6 months with placebo. The treatment’s safety was assessed in the same clinical trial, with common side effects including anemia, fatigue, and nausea.
🔹 AbbVie and Gubra have entered a license agreement to develop GUB014295, a long-acting amylin analog for obesity treatment. This collaboration marks AbbVie’s entry into the obesity field, leveraging Gubra’s expertise in peptide-based drug discovery. GUB014295, currently in phase 1 trials, targets amylin and calcitonin receptors, aiming to suppress appetite and delay gastric emptying. Under the agreement, AbbVie will lead global development and commercialization, with Gubra receiving an upfront payment of $350M and the potential to earn up to $1.875B in milestone payments. The deal is subject to regulatory approval.
🔹 On Feb. 28, Moderna announced that the U.K. Medicines and Healthcare Products Regulatory Agency (MHRA) granted marketing authorization for its RSV vaccine, mRESVIA (mRNA-1345), to prevent lower respiratory tract disease caused by respiratory syncytial virus (RSV) in adults aged 60+. This authorization is based on positive results from the ConquerRSV phase 3 trial, which involved 37k adults across 22 countries and showed no serious safety concerns. Moderna plans to produce mRESVIA at its new innovation and technology centre in Oxfordshire, opening later in 2025. The vaccine, Moderna’s second approved product in the U.K. after its COVID-19 vaccine, has also been authorized in multiple countries, including the U.S., EU, Canada, Qatar, UAE, and Taiwan.
🔹 Regeneron Pharmaceuticals announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended conditional marketing authorization for linvoseltamab, a treatment for adults with relapsed/refractory multiple myeloma (MM). The recommendation applies to patients who have received at least 3 prior therapies and have shown disease progression. Linvoseltamab is still under investigation, and the European Commission is expected to make a final decision soon. Additionally, the FDA is reviewing linvoseltamab, with a target decision date of Jul. 10, 2025. Linvoseltamab is a bispecific antibody designed to activate T cells to kill MM cells. It is being evaluated in the LINKER-MM1 trial, and the ongoing phase 2 portion of this trial focuses on safety and anti-tumor activity in patients who have previously undergone multiple treatments.
CLINICAL TRIAL UPDATES
🔹 On Mar. 3, Voyager Therapeutics announced positive topline data from its single ascending dose (SAD) trial of VY7523, an anti-tau antibody aimed at inhibiting the spread of pathological tau in Alzheimer’s disease. The trial, conducted in healthy volunteers, demonstrated VY7523’s safety, tolerability, and dose-proportional pharmacokinetics, supporting its potential as a best-in-class treatment. Following the SAD trial, Voyager has initiated a multiple ascending dose (MAD) study in patients with early Alzheimer’s disease, with initial tau PET imaging data expected in late 2026. VY7523 targets pathological tau and has shown a 70% reduction in tau spread in preclinical studies. The MAD trial will assess the antibody’s safety, tolerability, and its ability to prevent tau spread, with secondary endpoints including pharmacokinetics and immunogenicity. Voyager is optimistic about the future of tau-targeting therapies, having also advanced a tau silencing gene therapy approach.
🔹 Protagonist Therapeutics and Takeda announced positive topline results from the phase 3 VERIFY study of rusfertide, a first-in-class hepcidin mimetic peptide, for the treatment of polycythemia vera (PV), a type of blood cancer. The study met its primary endpoint, showing significantly more clinical responders in the rusfertide group (77%) compared to placebo (33%) during weeks 20-32. All four key secondary endpoints were also met, including reductions in phlebotomies and improvements in patient-reported outcomes. Rusfertide was generally well tolerated, with no new safety concerns. Protagonist will receive a $25M milestone payment from Takeda for the positive results. The companies plan to submit additional findings for regulatory review and future presentations.
PUBLIC HEALTH SPOTLIGHT
🔹 Tom Corry, the assistant secretary of public affairs at the U.S. Department of Health and Human Services (HHS), abruptly resigned on Mar. 3, just two weeks after starting the role. His resignation comes amid a growing measles outbreak in Texas, which has seen over 140 cases and one death. Reports suggest that Corry clashed with HHS Secretary Robert F. Kennedy Jr. and his aides over the handling of the outbreak. Corry was reportedly uncomfortable with Kennedy’s “muted response” to the crisis. Kennedy, known for his vaccine skepticism, initially downplayed the seriousness of the outbreak but later acknowledged its impact and emphasized the importance of making vaccines accessible. Despite this, Kennedy did not directly call for mandatory vaccinations, leaving room for controversy. Corry’s resignation follows a broader tension over the administration’s approach to public health, particularly during this measles epidemic.
ON THE HORIZON
🔹 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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