ACIP votes, Roche–89bio deal, Regeneron’s FOP win — This Week in Biotech #70
Early-child MMRV change, Hep B/COVID votes pushed for today, $3.5B 89bio deal, Opzelura label expands, Regeneron breakthrough, Mounjaro goes pediatrics and more (Sept 12-18, 2025).
Hi and welcome to This Week in Biotech by Biotech Blueprint, edition 70, covering biotech & pharma news from September 12th to 18th, 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 published yesterday (9/18), I teamed up again with Hartaj from the Biotech Capital Compass for part one of our mRNA therapeutics mini series. We covered the foundational science behind how RNA became medicine, the decades of bioengineering breakthroughs that made Covid vaccines possible, and what’s coming next in the infectious disease space. From Moderna’s ambitious pipeline targeting everything from flu-Covid combo shots to latent viruses like CMV and EBV, to Moderna’s and BioNTech’s custom cancer vaccines, we mapped out what’s actually coming in the next few years.
If you missed the associated deep dive article, you can find it here:
THIS WEEK IN BIOTECH VIDEO SUMMARY
THIS WEEK’S KEY TAKEAWAYS 🔑
ACIP’s early move + Friday cliffhanger: On Day 1, ACIP voted 7-3 (1 abstention) against using combo MMRV in kids <4, favoring separate MMR + varicella. The hepatitis B (timing of the birth dose for HBsAg-negative moms) and fall COVID recommendations will happen on Friday 9/19.
Deal of the week: Roche will acquire 89bio for up to $3.5B total, adding pegozafermin (FGF21) in phase 3 MASH. Strategically on-theme with Roche’s CVRM/obesity push and opens incretin-combo optionality if late stage efficacy holds. Closing targeted Q4 2025.
Lilly pushes tirzepatide into pediatrics: In SURPASS-PEDS, once-weekly Mounjaro cut A1C 2.2% vs 0% on placebo at 30 weeks. BMI ↓ 11% with adult-like GI tolerability. Lilly is filing for a peds T2D label.
Rare-disease standout: Regeneron’s garetosmab (anti-Activin A) hit in phase 3 FOP: 90–94% reduction in new heterotopic bone lesions and >99% drop in new-lesion volume at 56 weeks vs placebo. DMC recommended crossing placebo to drug. U.S. filing by end of 2025. First disease-modifier in FOP.
Derm win for kids: FDA cleared Incyte’s Opzelura (ruxolitinib) cream 1.5% for atopic dermatitis (AD) in ages 2-11. It is the first topical JAK inhibitor for pediatric AD in the U.S. that broadens Opzelura’s label and offers a steroid-free option.
Drug delivery chess move: Biogen is acquiring Alcyone Therapeutics to own ThecaFlex DRx, an implantable intrathecal port aimed at sparing repeated lumbar punctures for antisense oligonucleotide therapies (piloting with Spinraza).
BIOTECH/PHARMA NEWS 🧬
🔹 Heading into ACIP’s Sept. 18-19 session, AHIP said member plans will keep zero-copay coverage for all vaccines ACIP had recommended as of Sept. 1, 2025, including the updated COVID and flu shots, through end of 2026, effectively “grandfathering” access even if guidance shifts. The caveat is that UnitedHealth isn’t an AHIP member. This week HHS/CDC also added five members to the reconstituted ACIP (now 12 total after June’s purge), heightening attention on votes tied directly to coverage and pharmacy workflows. Vaccine makers moved to shore up data ahead of votes: Pfizer/BioNTech reported ≥4× neutralizing titers with its LP.8.1-matched shot in a phase 3 cohort, while Moderna said its LP.8.1 update drove >8× rises in an ongoing phase 4 study of older/high-risk adults.
On day one, ACIP voted 7-3 (one abstention) to not recommend the combined MMRV product for kids under 4, favoring separate MMR + varicella doses instead. But the committee pushed the hepatitis B and COVID-19 vaccine votes to Friday. The proposed Hep B language would delay the infant Hep B first dose until ≥1 month if the mother is HBsAg-negative, with “shared clinical decision-making” allowing earlier dosing. Note: those Hep B and COVID votes will land after this newsletter publishes Friday morning but we’ll follow up in the next issue and in Substack notes.
🔹 The FDA approved Incyte’s Opzelura (ruxolitinib) 1.5% cream for children ages 2–11 with mild to moderate atopic dermatitis, making it the first topical JAK inhibitor cleared in the U.S. for pediatric AD and expanding Opzelura’s label beyond its existing uses.
🔹 Biogen said it will acquire Massachusetts-based Alcyone Therapeutics for $85M upfront plus milestones, securing full rights to ThecaFlex DRx, an implantable subcutaneous port & catheter system designed to deliver antisense oligonucleotides (ASOs) into the cerebrospinal fluid and spare patients repeated lumbar punctures. Biogen will take over development, manufacturing and commercialization, initially evaluating ThecaFlex with its SMA therapy Spinraza (nusinersen) in the ongoing PIERRE/PIERRE-PK studies, and aims to introduce the new delivery option for Spinraza in early 2028 pending clinical success and regulatory approval. As part of the deal, Alcyone staff joining Biogen will bolster its drug–device capabilities, while Alcyone’s remaining assets will be spun into a new company, Neela Therapeutics, with convertible debt financing from existing investors including Biogen.
🔹 Roche is buying 89bio for $14.50 in cash per share plus up to $6.00 in CVRs (total up to $3.5B; ~$2.4B upfront), picking up pegozafermin, a phase 3 FGF21 analog for MASH across F2–F4 disease. The bid implies roughly an ~80% premium to 89bio’s last close, and the tender is slated to wrap after customary approvals, with closing targeted for Q4 2025. Strategically, it slots neatly into Roche’s CVRM/obesity-adjacent push (Zealand deal, Carmot buy) and sets up potential incretin combos if pegozafermin’s anti-fibrotic/anti-inflammatory profile holds in late stage data.
🔹 Biogen won European Commission approval for Zurzuvae (zuranolone), the first and only EU-approved treatment for postpartum depression, delivering a 14-day, once-daily oral option built on SKYLARK data showing rapid symptom relief by day 3, sustained through day 45 versus placebo. The EU label notes common AEs like somnolence, dizziness and sedation, and the green light expands a franchise already authorized in the U.S. (2023) and U.K. (Aug 2025).
🔹 On the eve of a potentially sweeping federal rewrite, the “West Coast Health Alliance” (California, Oregon, Washington, and Hawaii) issued their own seasonal vaccine playbook, reaffirming flu shots for everyone 6 months+ and recommending broad COVID and RSV coverage for young kids, pregnant people, seniors, and at-risk contacts, explicitly bucking HHS Secretary RFK Jr.’s narrower CDC posture. Less than 24h later, the Northeast is forming its own bloc, with New York and neighboring states rolling out parallel guidance while Gov. Hochul moves to preserve access (authorizing pharmacists and pressing insurers to keep covering recommended shots), creating a blue-state backstop even if ACIP pares back national recommendations this week.
🔹 In a tense Sept. 17 Senate hearing, ousted CDC director Susan Monarez said RFK Jr. pushed her to pre-approve whatever ACIP decided and to sack career scientists until the agency complied, and she lost her job for refusing. Former chief medical officer Deb Houry backed her up, describing “censored” science and a CDC hollowed out by churn, while Chair Bill Cassidy, an MD, anchored the stakes in data, defending the hepatitis B birth dose and warning that a flip in guidance could ripple straight into insurance coverage and newborn risk. The real hinge now is Sept. 18 ACIP meeting, recast with Kennedy’s picks, which will reveal whether U.S. vaccine policy still runs on transparent evidence or on political precommitments.
🔹 The CDC signaled an intent to sole-source a contract to Rensselaer Polytechnic Institute to probe vaccine–autism associations, citing RPI’s “unique ability” to link maternal-child cohorts for rapid statistical work, likely under biomedical engineer Juergen Hahn, reviving a question that large reviews have already answered with “no causal link.” The timing and structure matter as much as the science: it aligns with RFK Jr.’s broader push to re-litigate vaccine safety, while the SAM.gov notice leaves a 15-day window before CDC decides whether to compete the award, a procedural choice that will be read as policy direction.
CLINICAL TRIAL UPDATES 📊
🔹 Lilly: Mounjaro heads to pediatrics. After the SURPASS-PEDS phase 3 win in youths 10–18 with type 2 diabetes, Lilly said it will file to extend Mounjaro (tirzepatide) to pediatrics. At 30 weeks A1C fell ~2.2% vs ~0% on placebo, 86% on 10 mg hit ≤6.5% A1C, BMI dropped ~11%, and benefits persisted to 52 weeks. Safety was GI-weighted and consistent with adults, with no severe hypoglycemia.
🔹 Nanobiotix (NBTX) popped after unveiling fresh phase 1 data for JNJ-1900 (NBTXR3) in melanoma: in heavily pre-treated, anti-PD-1–resistant patients, the RT-activated nanoparticle plus checkpoint inhibitor showed a clean safety profile and early responses, leading investigators to call for randomized trials. Management is holding a briefing today. Shares spiked in early trading on the news.
🔹 Oruka’s ORKA-001, a long-acting IL-23p19 antibody for plaque psoriasis, posted interim phase 1 data showing an ~100-day half-life, over 3× longer than AbbVie’s risankizumab/Skyrizi, plus sustained downstream pathway inhibition and a clean safety read, a PK combo that plausibly enables once-yearly dosing if efficacy holds in patients. The EVERLAST-A phase 2a is enrolling now with first data expected in the second half of 2026, and the stock jumped on the news as coverage framed this as a potential category bend toward ultra-infrequent dosing.
🔹 Roivant/Priovant’s oral TYK2/JAK1 inhibitor brepocitinib hit in dermatomyositis: in the 241-patient, 52-week VALOR trial, the 30 mg dose lifted the mean Total Improvement Score (a composite of skin, muscle, and function) to 46.5 vs 31.2 on placebo (p=0.0006), separating as early as week 4 and winning all nine key secondary endpoints. Steroid use fell meaningfully, and two-thirds achieved at least a moderate response while nearly half hit a major response. Safety tracked prior experience and dose-response favored 30 mg. An NDA is planned for 1H26, putting a credible, once-daily targeted option on deck for a steroid-heavy disease.
🔹 Regeneron reported a decisive phase 3 win in fibrodysplasia ossificans progressiva (FOP), an ultra-rare genetic disease where muscle and connective tissue gradually turn to bone, with garetosmab cutting new heterotopic bone lesions by ~90–94% vs. placebo and shrinking total new-lesion volume by >99% over 56 weeks, leading the data-monitoring committee to recommend crossing placebo patients to drug. Safety showed more skin/soft-tissue infections with dose but no serious bleeding and no on-drug discontinuations. A U.S. filing is planned by end of 2025 and a pediatric study is next, positioning garetosmab as the first plausible disease-modifying therapy in FOP-tiny market, huge unmet need, and unusually clean efficacy signals.
🔹 aTyr’s phase 3 EFZO-FIT in pulmonary sarcoidosis missed its primary goal, lowering average daily oral corticosteroids at 48 weeks, yet the high dose still showed a pulse: more patients fully came off steroids and reported better KSQ-Lung quality of life scores, with lung function broadly maintained and safety clean. Because the analysis was hierarchical and the placebo group tapered more than expected, those wins are nominal, not confirmatory, hence the ~80% premarket selloff as investors marked down the odds of a straightforward filing.
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