Daily Snap - 17. July 2025

Author

Joachim Eeckhout
July 17, 2025

Good morning! After watching her son stand from his wheelchair years into ALS, Mitze Klingenberg joined other patients urging the FDA to approve NurOwn—a stem cell therapy they say has prolonged lives, restored function, and offers hope for ALS patients. Despite results showing a 90% five-year survival rate, the FDA rejected the therapy twice. Patients are now collecting their own data in the hope that the drug will be approved.

Enjoy today’s read!

—Joachim E.

SNIPPETS

What’s happening in biotech today?

🤖 AI meets mRNA: Alphabet spinoff SandboxAQ has partnered with Florida-based biotech iOncologi to develop and commercialize a high-fidelity mRNA vaccine targeting glioblastoma. The collaboration combines SandboxAQ’s advanced AI and simulation tools with iOncologi’s immunotherapy and clinical expertise, aiming to bring the vaccine to clinical trials within 18 months. SandboxAQ recently secured over $450 million for AI-driven medical research, while iOncologi continues to expand its precision immunotherapy capabilities following its acquisition of Swiss-based TargImmune Therapeutics.

🤝Deal revival: Repare Therapeutics has secured a deal with Debiopharm to advance its paused PKMYT1 inhibitor, lunresertib, with Debiopharm acquiring the rights for $10 million upfront, plus up to $262 million in potential milestone payments and royalties. The agreement builds on an existing collaboration exploring lunresertib in combination with Debiopharm’s WEE1 inhibitor, Debio 0123, for treating difficult cancers. Debiopharm will now lead the ongoing study, citing promising early clinical data. Meanwhile, Repare is focusing on two phase 1 trials for its PLK4 inhibitor RP-1664 and Polθ ATPase inhibitor RP-3467, with clinical readouts expected in the second half of 2025.

🛑 FDA tensions: FDA vaccine chief Vinay Prasad has once again overridden internal reviewers by approving only limited use of Moderna’s COVID-19 vaccine Spikevax for children aged 6 months to 11 years who are at increased risk, citing insufficient evidence of benefit for healthy kids. This marks the third time since May that Prasad has narrowed COVID vaccine approvals against staff recommendations, reflecting his longstanding skepticism toward pediatric COVID vaccination and his broader push, alongside FDA Commissioner Martin Makary, for stricter regulatory standards.

❌ Rare disease flop: AstraZeneca’s experimental drug anselamimab failed to meet its primary goals in two Phase 3 trials for late-stage AL amyloidosis, a rare disorder marked by toxic protein deposits that damage organs. The trials showed no significant reduction in overall mortality or cardiovascular hospitalizations in the general patient population. However, the company reported a “highly clinically meaningful improvement” in a prespecified subgroup. The setback comes amid broader challenges in AL amyloidosis drug development and affects AstraZeneca’s rare disease strategy following its acquisition of Alexion Pharmaceuticals.

😌 Eye relief: Okyo Pharma’s urcosimod (OK-101) has shown promising results in a Phase 2 trial for neuropathic corneal pain (NCP), achieving a statistically significant average pain reduction of 5.5 points on a 10-point scale in the 0.05% dosage group. The placebo group saw a 2.75-point reduction, yielding a strong treatment effect size. With no current FDA-approved treatments for NCP, Okyo plans further development and FDA discussions.

SPEED READ

More news

  • The UK has approved Alnylam's Amvuttra for treating both wild-type and hereditary transthyretin amyloid cardiomyopathy by targeting the gene that drives disease progression

  • Ventus Therapeutics is laying off staff as part of a strategic shift to prioritize mid-stage trials, expand its AI-driven drug platform, and advance its immune-targeting pipeline.

  • VC firm Modi Ventures has closed an $88 million second fund to back AI, biology, and medtech startups

  • BriaCell Therapeutics raised $15 million through a public offering to fund operations and advance its cancer immunotherapy pipeline amid ongoing clinical development

  • FDA reviewers have raised doubts about GSK’s bid to reapprove Blenrep for multiple myeloma, citing unresolved efficacy concerns, unique eye toxicities, and insufficient dosing optimization.

  • Kezar Life Sciences has regained FDA clearance to resume zetomipzomib trials in autoimmune hepatitis and is considering reviving its lupus nephritis program despite prior safety concerns

TOUR OPERATOR

Upcoming events

Share the Snap!

Know someone who’d enjoy this? Hit forward and pass it along.