Editor’s Briefing: September 10, 2026

In Short. The FDA locked permanent CDER and CBER directors after a year of turnover; Bayer’s Hyrnuo and Amgen’s Imdelltra each moved earlier in lung cancer; Tyra’s selective FGFR3 pill cleared a bar that Wall Street wanted higher; and menin biology left oncology for a Lilly-backed diabetes spinout while Encoded raised manufacturing money for a Dravet gene therapy.

By Vannevar Bush

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Continuity at the drug and biologics centers

HHS named Michael Davis permanent director of CDER and Karim Mikhail permanent director of CBER, converting the acting posts each had held. The move lands while Heidi Overton’s confirmation as commissioner remains unscheduled and Kyle Diamantas continues as acting FDA chief. RBC told investors the appointments look like continuity rather than another attempt to remake the agency. (BioPharma Dive; Fierce Biotech; BioSpace)

For readers who track how evidence moves through the system, the claim is institutional: after CDER saw five leaders in a year and CBER bounced through Vinay Prasad’s two tours, permanent center directors are the layer that still sets review practice while the commissioner’s chair waits.

HER2 lung, first line

The FDA expanded Bayer’s Hyrnuo (sevabertinib) into first-line locally advanced or metastatic non-squamous NSCLC with HER2 tyrosine kinase domain activating mutations. The accelerated decision rests on Soho-01: a 75 percent objective response rate in previously untreated patients (6 percent complete, 70 percent partial), with 73 percent of responses lasting more than six months. Confirmatory Soho-02 against standard of care is running. Boehringer’s Hernexeos already holds a first-line label in the same narrow 2–4 percent of NSCLC. (Fierce Pharma; FirstWord Pharma)

A mutation-defined lung niche is now a head-to-head oral TKI race timed by when clinics genotype, not by a broad chemo default.

DLL3 moves upstream in small-cell

Amgen said the phase 3 DeLLphi-305 trial of Imdelltra (tarlatamab), a DLL3×CD3 bispecific, scored an overall-survival win as first-line maintenance in extensive-stage SCLC after chemo-immunotherapy response. Only about 40 percent of SCLC patients reach today’s second-line setting where Imdelltra already holds a full approval; an earlier OS claim is the commercial and clinical point. Jazz/Roche’s Zepzelca–Tecentriq combo set one maintenance bar; Amgen is arguing DLL3 T-cell engagement can move the same earlier. (Fierce Pharma)

FGFR3 selectivity meets a Street CR bar

Tyra Biosciences reported initial phase 2 data for dabogratinib, an oral FGFR3-selective inhibitor, in low-grade intermediate-risk non-muscle-invasive bladder cancer. At three months, single-marker complete responses were 63 percent at 60 mg (n=8) and 50 percent pooled across doses (n=16); combined single- and multiple-marker CR was 57 percent at 60 mg. Shares fell about 38 percent as analysts had marked ~70 percent against J&J’s THOR-2 / Balversa benchmarks. Safety looked cleaner than pan-FGFR history: grade 3+ treatment-related events in 4.5 percent of a 44-patient safety cohort, with no dose reductions or treatment-related discontinuations at 60 mg. Tyra is completing 60 mg enrollment and adding a 70 mg cohort before a planned registrational start. (Fierce Biotech; BioPharma Dive)

The teachable object is selectivity versus bar-setting: FGFR3-over-FGFR1/2/4 design was meant to dodge Balversa toxicity; the market is still pricing three-month CR against pan-FGFR and gel chemotherapy precedents.

Menin leaves leukemia for the islet

Kura Oncology spun Caspian Therapeutics with $50 million, led by BVF and including Eli Lilly and the T1D Fund, to take KO-7246 — a menin inhibitor “purpose-designed for chronic metabolic diseases” — through preclinical work into first-in-human. Kura keeps roughly half of Caspian and the IP package; a second menin program for cardiometabolic disease rides along. Kura’s oncology menin inhibitor already has an AML label. Preclinical claims point at beta-cell regeneration and more insulin in Type 1 and Type 2 models, including human islet systems. (Fierce Biotech; BioPharma Dive)

Menin’s AML story was about blocking a KMT2A partnership that keeps blasts proliferating. Caspian is asking whether the same node, tuned for chronic dosing, can change islet mass — a different clinical claim that now has Lilly’s optionality sitting in the cap table.

Gene therapy cash for SCN1A and a plant

Encoded Therapeutics closed a $275 million series F, co-led by GV, to run a pivotal study of ETX101 in SCN1A-positive Dravet syndrome and to scale commercial manufacturing. ETX101 is an AAV9 therapy meant to raise SCN1A expression in inhibitory neurons; a phase 1/2 signal included a reported 78 percent seizure reduction, and phase 2 dosing began in May. Proceeds also fund a 2027 IND for ETX301 (AAV9 vectorized microRNA for post-amputation neuroma pain). Stoke/Biogen’s competing antisense approach still expects a phase 3 readout in the third quarter of 2027. (Fierce Biotech; FirstWord Pharma)

Growth plates and a biosimilar century

BioMarin detailed phase 3 CANOPY-HCH-3 results for Voxzogo in hypochondroplasia at ESPE and in NEJM: beyond the earlier 2.33 cm/year annualized growth-velocity win, standing height improved 2.35 cm and arm span 1.03 cm versus placebo at one year, with no treatment-related serious adverse events. An HCH filing is in, with a 2027 launch target if approved — while Ascendis’s rival now owes a U.S. royalty truce. Separately, Sandoz laid out Bio100: more than 100 biosimilars by 2040, LOE coverage aimed near 80 percent from 2035, and a $300 million Slovenia facility bet on vertical supply. (Fierce Pharma; Fierce Pharma; BioSpace)

Public-health counterpoint

Pennsylvania’s Lancaster County coroner confirmed two infant measles-related deaths from August: one from measles in a child with Amish lethal microcephaly, another where measles was a contributing condition beside a ruptured spleen. CDC’s site still withholds counting either death pending review, while the state outbreak exceeds 620 cases across 37 counties. (STAT News)

What to watch

pdufa.bio and today’s regulatory packet put Telix’s TLX101-Px decision tomorrow (September 11), Ultragenyx UX111 (ABO-102) for Sanfilippo A on September 19, Merck Winrevair (HYPERION) label work on September 21, Mirum/Incyte zilurgisertib on September 26, and a September 30 cluster with Novo Mim8 and Scholar Rock apitegromab. After Ultragenyx’s Aspire miss earlier this month, September 19 remains a commercial-path date, not a redo of Angelman.

Open mechanism questions from this tape: whether permanent CDER/CBER directors actually damp policy noise through Overton’s confirmation; whether Soho-02 and DeLLphi-305 turn mutation- and target-defined lung niches into durable first-line standards; whether FGFR3 selectivity can beat a 70 percent CR Street bar once 70 mg data arrive; and whether menin inhibition regenerates islets under chronic metabolic dosing the way it cleared AML blasts.