Ionis' Olezarsen Win, Novartis Licenses Arrowhead, CDC Vaccine Fight — This Week in Biotech #68
Ionis olezarsen’s Phase 3 in sHTG cuts triglycerides & pancreatitis. RFK Jr. reshapes ACIP again. Florida vaccine mandate rollback. United Therapeutics’ Tyvaso hits in IPF. (Aug 29–Sep 4, 2025).
Hi and welcome to This Week in Biotech by Biotech Blueprint, edition 68, covering biotech & pharma news from August 29th to September 4th, 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.
THIS WEEK IN BIOTECH VIDEO SUMMARY
THIS WEEK’S KEY TAKEAWAYS 🔑
Policy whiplash: Florida moves to end all vaccine mandates (including school shots), a first in modern times, while major medical groups warn of preventable outbreaks that will put health and lives at risk.
CDC turbulence: After firing the director, HHS installed Jim O’Neill as acting chief and may reshuffle the vaccine panel again before the Sept. 18-19 meeting.
Ionis shines: Olezarsen cut triglycerides by up to 72% and reduced acute pancreatitis events by 85% in severe hypertriglyceridemia. sNDA planned this year.
Real signal in idiopathic pulmonary fibrosis: United Therapeutics’ Tyvaso improved lung function with supportive secondaries and clean safety. Stock jumped ~40%.
Deals this week = platforms + delivery: Novartis pays $200M upfront for Arrowhead’s RNAi alpha-synuclein program (Parkinson’s), while Vertex teams with Enlaza on covalent biologics (>$2B milestones). The bet: differentiated delivery into tough tissues.
Gilead’s busy week: Lenacapavir PrEP to be supplied “at cost” abroad for up to 2M people over three years, while U.S. ADAP programs face proposed price hikes. Plus a new Foster City build as part of a $32B U.S. expansion.
Alzheimer’s monitoring tightens: FDA now recommends an extra MRI before Leqembi’s 3rd infusion, while a weekly subcutaneous maintenance autoinjector was approved, simplifying long-term dosing.
BIOTECH/PHARMA NEWS 🧬
🔹 The State Department, PEPFAR and the Global Fund will bring Gilead’s twice-yearly HIV prevention shot lenacapavir to market “at cost” in high burden countries, aiming to reach up to 2M people over three years, with Gilead supplying at no profit. In the U.S., the company is reportedly seeking high single-digit price increases on several HIV medicines used by state AIDS Drug Assistance Programs, which serve about 110,000 low income patients. Gilead also broke ground on a 180k sq ft development and manufacturing center at its Foster City HQ as part of a planned $32B U.S. build out through 2030.
🔹 Florida plans to end all vaccine mandates statewide, including long-standing school requirements for measles, polio, MMR, varicella and more, per Florida Surgeon General Joseph Ladapo, who framed mandates as “immoral” and likened them to “slavery.” The Health Department says it will drop non-statutory rules now and work with lawmakers on the rest, which would make Florida the first state in modern times to scrap school immunization requirements. Major medical groups, including the Florida chapter of the AAP and the AMA, warned the move risks outbreaks and reverses decades of progress. CDC analyses estimate routine childhood vaccination has averted ~508M illnesses, 32M hospitalizations and 1.13M deaths among U.S. children since 1994.
🔹 This week, Vertex teamed up with Enlaza Therapeutics to apply Enlaza’s covalent biologics platform to small-format drug conjugates and T cell engagers for certain autoimmune diseases and gentler conditioning in sickle cell disease and beta thalassemia (one time payment plus milestones that could exceed $2B). Novartis licensed Arrowhead’s preclinical RNAi program targeting alpha-synuclein for Parkinson’s disease, paying $200M upfront with up to $2B in milestones and royalties. The move marks a renewed bet on alpha-synuclein after Novartis’ prior UCB partnership fizzled, while Arrowhead’s TRiM platform pitches subcutaneous RNAi delivery to deep brain regions (key for CNS drugs). Arrowhead shares jumped ~16% on the announcement.
🔹 After the firing of CDC Director Susan Monarez, HHS installed Jim O’Neill as acting director. Reports say HHS may replace several members of the CDC’s Advisory Committee on Immunization Practices (ACIP) again just ahead of its September 18-19 meeting, after an earlier overhaul in June. On Capitol Hill, RFK Jr. defended the shake-ups and funding cuts to mRNA work amid bipartisan criticism. At the same time, Pfizer, Moderna and Novavax pushed back on claims that COVID-19 vaccine success isn’t well supported, with Pfizer noting >600 peer-reviewed publications and pledging fresh data on its updated strain in the coming days.
🔹 CVS and Walgreens say availability of updated COVID-19 vaccines will vary by state as they follow new rules. Some states require prescriptions for certain ages or risk groups, and in a few states CVS says it cannot vaccinate at all due to local regulations. FDA authorized the new shots for all seniors and for younger people only with qualifying risk factors, which is shaping access. The American Academy of Pediatrics now recommends COVID vaccination for all children 6-23 months, creating a guidance mismatch with current CDC recommendations for healthy kids.
🔹 The FDA has updated safety guidance for Eisai and Biogen’s Leqembi (lecanemab), recommending an additional MRI before the 3rd infusion to catch early amyloid-related imaging abnormalities with edema (ARIA-E). The change moves monitoring up from the prior 5th/7th/14th-infusion schedule after regulators reviewed more than 100 serious cases and six early-treatment deaths. A day later, the agency approved Leqembi IQLIK, a once-weekly subcutaneous autoinjector for maintenance dosing after 18 months of IV therapy, with U.S. launch set for Oct 6, 2025.
CLINICAL TRIAL UPDATES 📊
🔹 AbbVie reported updated data from its phase 2 EPCORE NHL-6 trial showing that epcoritamab, a T cell-engaging bispecific antibody, can be safely given in the outpatient setting for patients with relapsed or refractory diffuse large B cell lymphoma (DLBCL). In the study, most patients received their first full dose outside of the hospital, with cytokine release syndrome (40%) and neurotoxicity (8%) mostly low grade and resolving within days. If outpatient dosing is validated, it could broaden access to epcoritamab, which is already approved in the U.S. after two or more prior lines of therapy.
🔹 United Therapeutics’ phase 3 TETON-2 trial of Tyvaso in idiopathic pulmonary fibrosis (IPF) improved lung function, with several secondary measures also positive and no new safety signals. The data sent United’s shares up more than 40% and lifted peers MannKind, Liquidia, and Insmed, underscoring expectations for a multi billion dollar IPF market. Tyvaso holds orphan status for IPF in the U.S. and EU, and expanded results will be presented at ERS Amsterdam on Sept 28.
🔹 Mineralys Therapeutics (MLYS) jumped ~85% after AstraZeneca unveiled full phase 3 BaxHTN results showing its aldosterone-synthase inhibitor baxdrostat cut systolic BP in hard-to-control hypertension. The read-through favors Mineralys’ own ASI lorundrostat, which delivered a similar placebo-adjusted reduction over 12 weeks in the pivotal Launch-HTN program, fueling talk of “best-in-class” potential.
🔹 Ionis Pharmaceuticals’ olezarsen hit its marks in the phase 3 CORE/CORE2 program for severe hypertriglyceridemia, delivering up to a 72% placebo-adjusted cut in fasting triglycerides and an 85% reduction in acute pancreatitis events across ~1,100 patients, with a favorable safety profile. Ionis plans to take these findings into an sNDA by the end of 2025. With ~3M U.S. sHTG patients (over 1M high risk), olezarsen targets a large, commercially significant market, and shares of IONS jumped nearly 35% on the news.
🔹 BeOne Medicines reported positive topline results from its phase 1/2 study of sonrotoclax, a next-generation BCL2 inhibitor, in mantle cell lymphoma (MCL). The trial, which enrolled heavily pretreated patients following BTK and anti-CD20 therapy, met its primary endpoint with meaningful overall responses and showed encouraging durability and safety. If approved, sonrotoclax would become the first BCL2 inhibitor available for R/R MCL, a rare and aggressive lymphoma with limited options and a five-year survival rate of ~50%. BeOne plans to submit the data to regulators worldwide, including the FDA and China’s NMPA, while a phase 3 confirmatory trial is already underway.
🔹 J&J has opted to discontinue development of the novel combination therapy pairing nipocalimab (Imaavy) with anti-TNFα treatment for refractory rheumatoid arthritis (RA), after its 12 week phase 2a DAISY proof-of-concept study failed to demonstrate meaningful added benefit over standard anti-TNFα therapy alone. The program had been watched as a potential rival to AbbVie’s Humira franchise, which has long dominated the RA market but is now losing ground to U.S. biosimilars. Despite the setback, J&J reiterated its commitment to nipocalimab’s broader clinical journey across rheumatic, rare autoantibody, and maternal-fetal indications, maintaining confidence in the antibody’s projected $5B+ peak sales potential.
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