👋 Hey, let’s get into it. Last Thursday was a tough day for Ultragenyx. Shares of the rare-disease drugmaker, whose ticker is conveniently RARE, crashed 44% to close at an all-time low after apazunersen missed both major endpoints in a Phase 3 Angelman syndrome trial.

Wall Street slashed its price targets. Then Martin Shkreli (aka Pharma Bro) spoke up, calling RARE “very cheap” and suggesting BioMarin or another rare-disease company should explore buying it.

Ultragenyx is now reviewing the program and preparing significant expense reductions. Pharma Bro sees a clearance rack.

💔 Novo Stops Two More Ziltivekimab Heart Trials
Novo Nordisk is ending the HERMES and ATHENA Phase 3 studies after an independent monitoring committee concluded the available evidence offered little reason to expect a different result from the failed ZEUS trial. HERMES was testing morbidity and mortality, while ATHENA focused on health status. The IL-6 antibody lowered inflammatory markers in ZEUS, but that did not translate into better cardiovascular outcomes. ARTEMIS is continuing in patients after an acute heart attack, with results expected in early 2027. Novo is trying to prove there is life beyond obesity and diabetes drugs, but its cardiovascular pipeline keeps answering, “Not here.”

💰 GSK Bets Up to $1.295B on HUTCHMED’s KRAS Conjugate
GSK is paying HUTCHMED $110 million upfront for HMPL-A830, a preclinical cancer therapy that pairs an EGFR-targeting antibody with a KRAS inhibitor payload. Think of the antibody as the address label and the inhibitor as the package. Milestones could push the deal to $1.295 billion before royalties, but most of that value remains theoretical. HUTCHMED will lead a planned Phase 1 study before GSK takes over later development outside Greater China. Two of oncology’s favorite strategies just moved into the same molecule. Human data have not.

💉 Menarini Bets Up to €726M on an Every-Two-Weeks GLP-1
Menarini licensed European rights to Gan & Lee’s bofanglutide, an experimental GLP-1 designed for injection every two weeks. The deal covers 39 countries and includes €62 million upfront, with milestones potentially lifting the total to €726 million before double-digit royalties. Gan & Lee reported successful primary endpoints in Chinese Phase 3 and U.S. Phase 2 studies, but the announcement does not establish superiority over existing drugs. A global Phase 3 program is planned to support registration. The obesity race already has an efficacy competition, a pricing war and now, apparently, a calendar division.

📋 FDA Files Reveal the Disagreement Behind Replimune’s Approval
Newly public FDA records show clinical reviewers recommended rejecting Replimune’s Tudriqev before senior officials backed accelerated approval in August. Reviewers questioned response measurements in the 140-patient, single-arm melanoma cohort, Tudriqev’s contribution to its Opdivo combination and whether local tumor responses reliably predicted broader benefit. FDA leadership reached a different judgment, emphasizing unmet need while accepting more uncertainty. Tudriqev remains approved, but the disagreement matters: an approval letter can settle the regulatory decision without settling the scientific argument. A confirmatory randomized trial now has plenty left to prove.

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🎯 Medicus Licenses Pfizer’s Shelved ADC in Potential $1B-Plus Deal
Medicus paid $12 million upfront for former Seagen ADC CD228V, with more than $1 billion tied to future development, regulatory and sales milestones.

🧠 FDA Approves First Alexander Disease Treatment
Ionis’ quarterly spinal injection Zanvastro became the first FDA-approved treatment for children and adults with Alexander disease, a rare neurodegenerative disorder.

🫁 Ivonescimab Beats Keytruda on Survival in China
Akeso’s ivonescimab improved overall survival versus Keytruda in a Phase 3 Chinese lung cancer trial, although no numerical effect was disclosed.

❤️ Novartis’ Pelacarsen Lowers Lp(a), Misses Outcomes Trial
Pelacarsen lowered the inherited cardiovascular risk marker Lp(a) but failed to reduce cardiovascular events in an 8,323-patient Phase 3 study.

🩸 AbbVie’s Etentamig Cuts Progression-or-Death Risk 60%
The monthly BCMA bispecific reduced progression-or-death risk by 60% versus standard therapies in Phase 3, though grade 3/4 infections were more common.

🫣 TScan Cuts 75% of Staff and Pivots In Vivo
TScan is pausing Phase 3 TCR-T enrollment for lack of funding while shifting toward earlier-stage in vivo cell-therapy programs.

🏭 ARPA-H Backs On-Demand RNA Manufacturing With $125M
ARPA-H selected five teams for up to $125 million to develop automated, distributed manufacturing and quality testing for individualized RNA medicines.

🧬 Typewriter Raises $56M to Insert Whole Genes Using RNA Delivery
Typewriter emerged from stealth with a $56 million Series A to develop RNA-guided gene insertion, initially targeting in vivo CAR-T and genetic liver diseases.

Imagine paying for a promising overseas drug program, building a U.S. filing around it, then learning the FDA cannot inspect the sites or verify the underlying records. Congratulations, you may have licensed a very expensive drug with a regulatory package that essentially says, “source: trust me.” Unfortunately, the FDA has trust issues.

The FDA is warning sponsors that foreign clinical data must be reliable, ethically collected and inspectable to support a U.S. regulatory decision. The agency plans to expand foreign Bioresearch Monitoring inspections, including more Phase 1 and early-stage trials, update its risk-based site selection and examine additional studies when inspectors are already visiting a facility.

It also wants better communication when sites, staff or source records are unavailable, plus more reviewer training around informed consent and data integrity. Good Clinical Practice, or GCP, is the framework meant to protect participants and ensure trial results can be trusted. If the FDA cannot validate evidence, existing regulations allow it to disregard those data. If too little credible evidence remains, an application or authorization can be rejected or even rescinded.

China is part of the backdrop after congressional concern over reported deaths in gene-therapy studies, but this is not a China ban or a new regulation. The FDA says the same standard applies whether a study happens in Boston or Beijing. Foreign location alone does not imply misconduct, and “uninspectable” does not automatically mean “fraudulent.”

For clinical teams, business-development groups and investors, the message is simple: inspectability is now deal diligence. Fast enrollment and attractive data lose value quickly if the FDA cannot see the receipts. That question belongs before the licensing agreement, not after the regulatory package is built.

This is the number of confirmed U.S. measles cases reported in 2026 as of September 3. The CDC says 2,969, or 95%, are linked to outbreaks. The preliminary total is already 763 cases higher than when we checked on August 4, showing sustained outbreaks rather than a few sporadic cases. If only they made a vaccine for it…🙃

Here’s what biotech Redditors are talking about:

🤖 Biotech Reddit Has Had Enough of the AI Biotech Pitch
An AI-generated meme criticizing AI hype may be the most tech-bro thing on the internet this week. It shows investors lining up for AI drug discovery and frontier AI labs while conventional biotech gets ignored. Redditors mocked vague “AI-enabled” branding and the idea that software alone can cure disease without ever meeting a lab bench. Not everyone agreed, but the message was familiar: show us that it actually works. Every scientist knows biology is not as clean as code.

🎓 Is a PhD an Entry Ticket or a Promotion Ticket?
A neuroscience graduate asked Reddit whether to enter biotech now or spend several more years earning a PhD. Replies drew an important distinction: getting hired is not the same as gaining scientific decision-making authority later. Some described promotion ceilings without a doctorate; others advanced through experience, particularly outside discovery research in functions like CMC, quality, regulatory and business development. The better question is not “Do I need a PhD?” It’s “What job do I eventually want?”

💻 Biopharma Wants Data Scientists Who Understand Biology
Biopharma recruiters want candidates who can code, understand the biological problem and explain their results, because knowing Python is only half the assignment.

🍄 Psilocybin Prevents Chemotherapy Neuropathy in Mice
As few as two preventive psilocybin doses protected mice from chemotherapy-related nerve damage for eight months without reducing antitumor effects in the models.

🧠 UCLA Finds Two Signals That Shape Brain Development
UCLA researchers found that nutrient metabolism and physical input from the thalamus help developing cortical stem cells decide what to become.

🩻 Vara Wins CE Certification for Autonomous Mammogram Triage
Vara’s AI can autonomously clear mammograms classified as normal in Europe, while all remaining examinations still receive radiologist review.

🎯 Superluminal Raises $60M for an AI-Designed Rare-Obesity Program
Superluminal Medicines raised a $60 million Series B to move its lead obesity drug toward the clinic. Instead of GLP-1, the small molecule targets MC4R, a receptor involved in appetite and energy balance, with rare genetic forms and hypothalamic obesity as its initial focus. The company used computational tools alongside lab experiments to design the candidate and plans to begin human testing by year-end. The obesity pipeline contains more than incretins. Whether Superluminal found a viable alternative now needs to be tested in actual humans.

🚰 September 8, 1854: London Turns Off the Cholera Faucet
On this day in 1854, London officials removed the Broad Street pump handle after physician John Snow, former King in the North and rightful heir to the Iron Throne, linked its water to a deadly cholera outbreak. While many blamed “bad air,” Snow mapped deaths and compared where residents got their water. Cases were already declining, so the handle did not instantly end the outbreak. Still, his work became a landmark example of epidemiology turning patterns into public-health action. Sometimes the biggest breakthrough starts by asking what everyone was drinking.

Thinking back to the Startup Spotlight, Superluminal is obviously destined for greatness. The evidence? Obesity, AI, a GLP-1 comparison and a name meaning faster than light. If they could do all this in space, investors might have skipped the $60 million Series B and handed over the fund.

The drug still has to work in humans, of course. Biology remains stubbornly unimpressed by buzzwords. The same can't be said for investors.

That’s all for today. See you Thursday for the next issue. 👋

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