Monarez Fired, Novo’s srRNA Deal, ImmunityBio Wows in GBM — This Week in Biotech #67
Public health politics escalate as COVID rules shift, Novo backs Replicate’s next-gen RNA, and ImmunityBio posts 100% disease control in glioblastoma pilot (August 22–28, 2025).
Hi and welcome to This Week in Biotech by Biotech Blueprint, edition 67, covering biotech & pharma news from August 22nd to 28th, 2025.
🎙️ Biotech Blueprint brings you weekly video updates on the latest biotech and pharma news, plus in-depth podcast interviews with industry leaders. You can find us on YouTube, Spotify, and Apple Podcasts.
In the latest episode, I teamed up with the Biotech Capital Compass to break down Sarepta Therapeutics’s recent safety crisis, and what the FDA’s partial green light means for Elevidys, investor confidence, and the broader AAV field. We dig into the science, regulatory dynamics, and what might come next.
THIS WEEK IN BIOTECH VIDEO SUMMARY
THIS WEEK’S KEY TAKEAWAYS 🔑
Novo Nordisk’s $550M deal with Replicate Bioscience adds validation to self-replicating RNA (srRNA) as a next-gen modality. The partnership is in early stages, but with obesity and cardiometabolic disease in scope, this could be the start of srRNA’s real commercial breakout. Watch this space.
CDC leadership was destabilized this week as newly appointed director Susan Monarez was fired after less than a month on the job. Her refusal to carry out politically motivated firings led to a wave of resignations. Beyond the headlines, this signals rising politicization of U.S. public health, just as Covid policies are being quietly reshaped.
Speaking of Covid. Updated vaccines from Moderna, Pfizer, and Novavax got FDA approval this week, but without broad eligibility. Only high risk groups are covered, while others may face barriers around insurance or cost. Critics warn that removing emergency use pathways could leave millions unprotected this winter.
Meanwhile, a very different Covid trade emerged as Invivyd surged 94% on speculation that mRNA vaccines may be pulled from the U.S. market. Investor appetite for monoclonal antibody prevention (VYD2311) is back, though it’s unclear whether this rally is grounded in policy shifts or political rumor.
ImmunityBio’s early glioblastoma trial showed 100% disease control using a chemo-free NK+IL-15 combo, a striking result in an aggressive cancer with poor long term survival rates. Also promising: BioXcel’s Igalmi (BXCL501) passed its phase 3 safety bar in at home treatment of agitation. An FDA nod could unlock the first at home option for crises related to bipolar disorder and schizophrenia.
On the regulatory front, Gilead’s lenacapavir (Yeytuo/Yeztugo) gained EU approval as the first HIV PrEP injection administered twice per year. A new 2M person access deal with The Global Fund underscores Gilead’s global ambitions and marks the start of a new era for long-acting HIV prevention.
Finally, Valneva’s chikungunya vaccine suffered a major blow after four serious adverse events prompted the FDA to suspend its U.S. license.
BIOTECH/PHARMA NEWS 🧬
🔹 Novo Nordisk has teamed up with Replicate Bioscience in a multiyear partnership worth up to $550M to develop next-gen treatments for obesity, type 2 diabetes, and other cardiometabolic diseases. The Danish pharma giant gains exclusive global rights to Replicate’s self-replicating RNA (srRNA) platform, a technology that enables cells to produce their own therapeutic proteins, potentially at lower doses and with longer-lasting effects than traditional mRNA. The deal adds momentum to Replicate’s growing srRNA pipeline, which includes a rabies vaccine that recently cleared phase 1 and preclinical candidates targeting cancer and autoimmune conditions.
🔹 Susan Monarez was removed as CDC Director less than a month into the role after reportedly resisting political directives from Health Secretary Robert F. Kennedy Jr. and President Trump’s administration. Her firing sparked a wave of senior resignations, including Drs. Debra Houry and Demetre Daskalakis, with concerns over political interference and public health integrity. The CDC now faces a leadership vacuum amid mass layoffs and rising mistrust in its scientific neutrality.
🔹 Amylyx Pharmaceuticals is discontinuing development of AMX0035 (sodium phenylbutyrate + taurursodiol) in progressive supranuclear palsy (PSP) after the drug failed to show any benefit in a phase 2b trial. Once approved for ALS before being pulled from the market, AMX0035 continues to be tested in Wolfram syndrome, but Amylyx’s strategic focus has clearly shifted to avexitide (its GLP-1 receptor antagonist now in a pivotal phase 3 trial for post-bariatric hypoglycemia).
🔹 The FDA approved updated COVID-19 vaccines from Moderna, Pfizer, and Novavax, but limited eligibility to those at higher risk, mainly adults 65+, people with serious health conditions, and immunocompromised individuals. While others can request a shot via doctor consultation, access may be restricted by cost, insurance, or state laws on off-label use. Emergency authorizations were revoked, and vaccine makers must now run placebo-controlled trials. The CDC will decide whether to recommend the new vaccines, which would influence insurance coverage. Critics, including major medical groups, warn that the changes could leave millions, including pregnant women and children, vulnerable ahead of a possible winter surge.
🔹 Invivyd surged 94% on renewed COVID therapy interest. Shares of Invivyd (IVVD) soared after reports that the Trump administration may withdraw mRNA COVID vaccines from the U.S. market. The rally follows recent FDA alignment on an expedited approval path for VYD2311, Invivyd’s monoclonal antibody therapy built on the same construct as Pemgarda, currently authorized for use in immunocompromised patients. VYD2311 showed promise in early trials as a long-acting COVID prevention option, fueling investor enthusiasm.
🔹 Gilead’s lenacapavir injection (Yeytuo) received accelerated marketing authorization in the EU as the first and only twice-yearly HIV PrEP option. Backed by strong phase 3 data showing near-perfect prevention rates, approval follows prior U.S. and WHO endorsements. Gilead aims for global access, backed by a supply agreement with The Global Fund to reach 2M people in low- and middle-income countries.
🔹 AbbVie announced a deal to buy bretisilocin (GM-2505), Gilgamesh Pharmaceuticals’ lead compound for major depressive disorder, currently in phase 2. The novel, short-acting 5-HT2A receptor agonist showed a significant -21.6 MADRS score reduction vs. -12.1 for comparator, with rapid onset and no serious adverse events. The acquisition includes upfront and milestone payments, and Gilgamesh will spin out its other programs into a new entity, Gilgamesh Pharma Inc., including its oral NMDA antagonist blixeprodil and a cardio-safe ibogaine analog.
🔹 The FDA has suspended Valneva’s chikungunya vaccine Ixchiq license following four new serious adverse events resembling chikungunya illness, mostly in elderly individuals. The decision reverses an earlier August move to lift restrictions on vaccinating those 60+, and halts all U.S. sales and distribution. Valneva says the serious adverse events align with known risks outlined in the vaccine’s labeling and occurred outside the U.S. The company is reviewing the FDA’s decision but maintains its commitment to global rollout, especially in endemic low- and middle-income countries. Ixchiq generated €7.5M in H1 2025 sales.
CLINICAL TRIAL UPDATES 📊
🔹 Vor Bio and its partner RemeGen announced that telitacicept met the primary endpoint in a phase 3 trial for IgA nephropathy (IgAN), reducing proteinuria by 55% at week 39 (p<0.0001) with a favorable safety profile. The randomized, placebo-controlled study enrolled 318 patients in China and supports telitacicept’s disease-modifying potential across autoimmune conditions. A BLA submission in China is planned, which could mark its fifth approved indication there. Telitacicept, a dual BLyS/APRIL inhibitor, is already approved in China for lupus, RA, and myasthenia gravis, and is being studied globally in gMG and other autoimmune diseases.
🔹 BioXcel reported positive phase 3 results for Igalmi (BXCL501) in at-home treatment of acute agitation tied to bipolar disorder and schizophrenia. The 120 mcg sublingual film met its primary safety endpoint with no serious side effects across 2,400+ episodes, and patients were able to self-administer the treatment effectively. Mild side effects were consistent with prior studies. An sNDA is planned for Q1 2026. If approved, Igalmi could become the first FDA approved at home option for agitation, expanding its market potential beyond institutional use.
🔹 Chinese biotech Akeso reported that its PD-1/VEGF bispecific antibody ivonescimab met the overall survival endpoint in the final analysis of the phase 3 HARMONi-A trial for NSCLC, marking a major milestone for potential global approvals. The drug outperformed tislelizumab + chemo in another head-to-head phase 3 trial in squamous NSCLC and is being studied in 13 registrational trials across lung cancer and other solid tumors.
🔹 ImmunityBio reports 100% disease control in early glioblastoma trial. In a five-patient pilot study for recurrent glioblastoma, all participants achieved disease control using a chemo-free combo of Anktiva (IL-15 agonist), NK cell therapy, and the Optune Gio tumor treating device. Two patients showed near complete response, while the others maintained stable disease. The regimen also restored lymphocyte counts suppressed by prior chemo-radiation. Encouraged by these early results, ImmunityBio plans a randomized phase 2 trial in second-line GBM, a disease with a dismal <10% five-year survival rate in adults over 45.
🔹 Regeneron’s cemdisiran met phase 3 goals in generalized myasthenia gravis, improving symptoms and inhibiting 74% of complement activity. The siRNA therapy outperformed placebo and was well tolerated. A U.S. filing is expected in Q1 2026.
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DISCLOSURE: I have no business relationships with any company mentioned in this article.