Jennifer Boggs

Following the recent recommendation of its advisory committee, the U.S. FDA gave the nod to Zevra Therapeutics Inc.’s arimoclomol, a day ahead of its Sept. 21 PDUFA date, as the first treatment for Niemann-Pick disease type C (NPC), a rare genetic disease linked to progressive neurological symptoms that is almost always fatal. Branded Miplyffa, it is indicated for use in combination with enzyme inhibitor miglustat in adults and children, ages 2 and older.

Speculation about potentially wider meaning among developers of psychedelic drugs was reignited after the complete response letter (CRL) from the U.S. FDA to Lykos Therapeutics Inc. for midomafetaminecapsules, which the company wants to use in combination with psychological intervention to treat post-traumatic stress disorder (PTSD). CEO Amy Emerson called the FDA move “deeply disappointing,” adding that San Jose, Calif.-based Lykos plans to request a meeting with the FDA to ask for reconsideration.

First psychedelic-assisted PTSD therapy delayed by FDA CRL to Lykos

The disappointments continue for Fibrogen Inc., which is terminating work on its once-promising anti-CTGF monoclonal antibody, pamrevlumab, after reporting missed endpoints in two late-stage pancreatic cancer studies, and cutting its workforce by about 75%.

Cognition Therapeutics Inc. said it is moving to the next stage of clinical testing with oral, small-molecule candidate CT-1812, despite phase II efficacy results falling short of statistical significance in patients with mild to moderate Alzheimer’s disease and sending shares of the Purchase, N.Y.-based company (NASDAQ:CGTX) falling 44%, or $1.04, to close July 29 at $1.33.

In one of the largest venture rounds for biopharma in 2024, Cardurion Pharmaceuticals Inc. closed a $260 million series B financing, with funds slated to advance and expand its pipeline for potentially first-in-class drugs targeting cardiovascular disease, including two programs in phase II development.

Two days before the PDUFA date, the U.S. FDA handed down a complete response letter (CRL) for Rocket Pharmaceuticals Inc.’s Kresladi (marnetegragene autotemcel), delaying potential approval of the lentiviral-based gene therapy as the first therapeutic option for leukocyte adhesion deficiency type I, a rare, inherited immune disorder. But the Cranbury, N.J.-based company has suggested that delay won’t be long, as the CRL requests only “limited” chemistry manufacturing and controls (CMC) informati…

Shares of Avidity Biosciences Inc. hit a 52-week high on reports of promising data from the first efficacy cohort of its phase I/II Fortitude study testing delpacibart braxlosiran (del-brax) in facioscapulohumeral muscular dystrophy (FSHD), including impressive biomarker results that could indicate a path for potential accelerated approval.

Pfizer Inc.’s Beqvez (fidanacogene elaparvovec) won FDA approval for use in adults with hemophilia B, making it the second adeno-associated viral (AAV) vector-based gene therapy available for patients in the U.S., following the late 2022 approval of CSL Behring’s Hemgenix (etranacogene dezaparvovec).

Eli Lilly and Co. is planning to file for U.S. FDA approval later this year after reporting that tirzepatide met all primary and key secondary endpoints in two phase III trials in obstructive sleep apnea (OSA). Assuming approval, tirzepatide could become the first drug approved specifically for OSA, while providing potential entry access for Medicare Part D coverage, which is denied for the GLP-1 class of drugs approved as obesity medications.

Investors might not have been overly excited, but Genmab A/S executives enthused about the “complementarity” of its proposed acquisition of antibody-drug conjugate (ADC) specialist Profoundbio Inc. for $1.8 billion in cash. The deal, expected to close in the first half of 2024, marks the biggest by far for the Copenhagen, Denmark-based biopharma and the latest transaction for the red hot ADC space.

BioWorld Content on ‘rinatabart sesutecan’

In what co-founder and CEO Claudia Ulbrich called the “right decision with right partner at the right point in time,” Cardior Pharmaceuticals GmbH agreed to an acquisition by Novo Nordisk A/S in a deal worth up to €1.025 billion (US$1.1 billion) that puts a potentially disease-modifying heart failure candidate in the hands of a big pharma player in the midst of establishing its presence in cardiovascular disease.

The EMA’s Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending approval of Pfizer Inc.’s Emblaveo (aztreonam-avibactam), an antibiotic combination that would offer a new option to patients with serious bacterial infections caused by multidrug-resistant gram-negative bacteria. If approved, Emblaveo would be among the first beta-lactam/beta-lactamase inhibitor combos cleared for use in Europe.

A first-quarter 2024 launch for Alzheimer’s drug donanemab appears to be off the table as Eli Lilly and Co. disclosed a last-minute decision by the U.S. FDA to convene an advisory committee to review data from the phase III Trailblazer-ALZ 2 trial.

The good news is that the U.S. FDA’s complete response letter (CRL) for Venatorx Pharmaceuticals Inc.’s intravenous antibiotic combination, cefepime-taniborbactam, in complicated urinary tract infections (cUTIs) requested no additional clinical testing. The bad news is the inevitable delay for a drug Venatorx and partner Melinta Therapeutics Inc. are aiming to position as a much-needed option for the fight against drug-resistant gram-negative infections.

When founders of Latigo Biotherapeutics Inc. first set out a few years ago to establish a biopharma firm focused in the area of pain, the plan had been to get a head start by in-licensing promising assets in the space. But that proved easier said than done. “With the exception of very early chemical matter” from the Lieber Institute for Brain Development, “we really couldn’t find anything else of quality to bring in, which I think is a testament to how little pain research and investment was ong…

Shares of Corbus Pharmaceuticals Inc. (NASDAQ:CRBP) doubled on opening Jan. 26 and ended the day up a whopping 249% as investors got a look at data from a first-in-human study testing next-generation Nectin-4-targeted antibody-drug conjugate (ADC) CRB-701. Licensed from CSPC Pharmaceutical Group for $7.5 million up front in February 2023, CRB-701 has IND clearance from the U.S. FDA, and Corbus plans to start its own clinical testing in the first quarter of 2024.

The zeal for antibody-drug conjugates (ADCs), a prominent focus for dealmaking in 2023, shows no signs of waning, as Johnson & Johnson greeted attendees of the J.P. Morgan Healthcare Conference (JPM) with news of its $2 billion buyout of Ambrx Biopharma Inc., picking up rights to an ADC platform along with a promising candidate targeting advanced prostate cancer. Under the terms, J&J agreed to acquire all outstanding shares of San Diego-based Ambrx for $28 apiece, marketing a 105% premium to the firm’s Jan. 5 closing price. Unsurprisingly, Ambrx’s stock (NASDAQ:AMAM) gained 101.5% to close Jan. 8 at $27.47.

With its launch barely three years in the rearview mirror, Remix Therapeutics Inc. has inked its second potential $1 billion pharma collaboration, drawing Roche Holding AG to the table with the promise of its RNA processing platform and the potential to develop small-molecule therapeutics targeting the underlying drivers of disease. At the same time, the Cambridge, Mass.-based firm added $60 million in a venture round, bringing its total financing to date to $201 million.