Annexon Inc.’s stock (NASDAQ:ANNX) climbed 34% in early trading June 4 following news that a single infusion of C1q protein complex inhibitor ANX-005 at the 30-mg/kg dose delivered a statistically significant 2.4-fold improvement in Guillain-Barré syndrome (GBS) patients. Shares were trading at about $6.12 midday, up $1.54. The Brisbane, Calif.-based company measured the primary endpoint of the phase III trial on the GBS-disability scale at week eight. A higher dose of 70 mg/kg did not meet the primary endpoint. Nevertheless, Annexon intends to file a BLA with the U.S. FDA in the first half of 2025. ANX-005 has fast track and orphan drug designations in the U.S., as well as orphan drug designation in Europe. GBS is a neurological disease for which there are no approved treatments in the U.S.

Therapy component raises concerns at MDMA adcom

Therapist conduct is weighing on the minds of some of the members of the U.S. FDA’s Psychopharmacologic Drugs Advisory Committee today as they consider Lykos Therapeutics Inc.’s NDA for midomafetamine (MDMA), used in combination with psychotherapy, as a treatment for post-traumatic stress syndrome (PTSD). Panelist Walter Dunn, director of the interventional psychiatry service at the West Los Angeles Veterans Affairs Medical Center, brought up an incident in a phase II trial in which a therapist engaged in sex with a patient under the influence of MDMA. When Lykos learned of the “misconduct,” it immediately investigated and took steps to prevent similar incidents in the future. Bristling at the characterization of the incident as misconduct, the panel’s patient representative, Elizabeth Joniak-Grant, said it was more than misconduct; it was sexual misconduct. That’s more troubling given that many patients suffer from PTSD due to sexual attacks, she noted. Meanwhile, the FDA is pondering how to address the psychotherapy component in labeling if MDMA is approved. “We do not regulate therapy as a rule in terms of its specific content or details,” David Millis, an FDA clinical reviewer, said.

Bridgebio unveils phase II data with infigratinib in achondroplasia 

Bridgebio Pharma Inc. rolled out sustained positive results from Propel 2, a phase II trial with infigratinib in children with achondroplasia. The once-daily oral drug yielded what the Palo Alto, Calif.-based firm described as potential best-in-class efficacy and an encouraging safety profile. Infigratinib is an oral small molecule designed to inhibit FGFR3 signaling. Wall Street promptly began comparing data from Propel 2 with findings by San Rafael, Calif.-based Biomarin Pharmaceutical Inc. with Voxzogo (vosoritide), the C type natriuretic peptide analogue used to increase linear growth in pediatric patients with achondroplasia. Voxzogo, which consists of lyophilized powder for injection, was first cleared by the U.S. FDA in November 2021. Propel 3, the global phase III study with Bridgebio’s drug, continues to enroll on schedule, with completion estimated by end of this year. Shares of Bridgebio (NASDAQ:BBIO) were trading at $27.90, down $1.08, while Biomarin (NASDAQ:BMRN) was selling for $78.14, up $1.73. Achondroplasia is a genetic disorder that affects bone growth in the limbs and spine, causing dwarfism, of which it’s the most common type and strikes about one in 20,000 to 40,000 infants.

Summit expands rights, cash for Akeso’s China-approved NSCLC drug

Miami-headquartered Summit Therapeutics Inc. expanded rights to Akeso Inc.’s non-small-cell lung cancer drug, ivonescimab (SMT-112; AK-112), June 3 while raising $200 million to advance the therapy. Summit’s stronger hold on the drug comes after its Guangdong, China-based partner, Akeso, posted knockout results of ivonescimab against Merck & Co. Inc.’s Keytruda (pembrolizumab) in a head-to-head Harmoni-2 trial in patients with PD-L1-positive lung cancer – news that propelled Akeso’s stocks by 37.5% on May 31.

World Health Assembly extends deadline for pandemic agreement 

Although consensus was not reached on the World Health Organization’s pandemic agreement, the World Health Assembly (WHA) recognized the progress made by member states to develop a pandemic agreement and to strengthen International Health Regulations (IHR, 2005) during the 77th World Health Assembly meeting held May 27 to June 1 in Geneva. The WHA agreed on a package of amendments to the IHR, 2005, and extended the mandate of the WHO Intergovernmental Negotiating Body to finish its work to negotiate a pandemic agreement within a year.

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