FDA Action Alert: Adaptimmune, Zevra, Lykos and scPharma

The FDA will welcome August with four major events over the next two weeks: three target dates for experimental treatments and an advisory committee meeting on an ultra-rare neurodegenerative disease.

Read below to learn more.

Adaptimmune seeks first approval of a genetically modified T cell therapy for solid tumors

By August 4, the FDA will publish its Verdict about Adaptimmune Therapeutics’ Biological License Application (BLA) for afami-cel, an experimental, genetically engineered T-cell therapy that the biotech company proposes to treat advanced synovial sarcoma.

The regulator has granted Afami-Cel priority review, shortening the review process from the usual ten months to six months.

Afami-cel works by targeting and binding to melanomaassociated antigen A4 (MAGE-A4), a protein that plays a central role in preventing cell cycle arrest and blocking cell death. MAGE-A4 is highly expressed in solid tumorsespecially synovial sarcoma.

Adaptimmune’s BLA, for which the company completed rolling submission in December 2023, is supported by data from the first cohort of the pivotal SPEARHEAD-1 trial. In March 2024, the biotech company published study results in The Lancet This resulted in an overall response rate of 39% in patients with synovial sarcoma.

Regarding safety, 37 cases of cytokine release syndrome were documented in SPEARHEAD-1, representing an incidence of 71%, although only one episode was Grade 3. There were no treatment-related deaths.

If approved, afami-cel could be the “first engineered T cell therapy for solid tumors and the first effective treatment option for synovial sarcoma in over a decade,” according to a press release from Adaptimmune announcing the FDA’s acceptance of the BLA.

scPharma wants to expand Furoscix injection

scPharmaceuticals proposes an expanded approval for its subcutaneous injection Furoscix (furosemide) to allow use in heart failure patients with New York Heart Association (NYHA) Class IV disease. The FDA Decision is due on August 10th.

Furoscix is ​​administered via a single-use infuser worn on the body and delivers an initial dose of 30 mg of furosemide in the first hour, followed by 12.5 mg of the drug every hour for the next four hours. label. Furoscix works by blocking sodium reabsorption in a specific area of ​​the kidney, which in turn promotes urine production. In patients with heart failure, this mechanism of action helps prevent fluid retention and congestion. Furoscix also helps with vasodilation.

Currently, Furoscix is ​​only indicated for patients with chronic heart failure of NYHA classes II and III. In August 2023, scPharma a meeting of type C completed with the FDA, where the company received “positive” feedback from the regulator regarding the potential expansion of Furoscix’s labeling to NYHA Class IV patients.

At the time, CEO John Tucker said the positive Type C meeting “paves the way for a potential expansion of the Furoscix indication” to NYHA Class IV patients. “We estimate that up to 10% of all heart failure patients are Class IV, and a significant percentage of those – up to 40% – could benefit from Furoscix,” he said.

Lykos awaits FDA ruling on MDMA therapy for PTSD

Lykos Therapeutics is developing an experimental MDMA-assisted therapy for post-traumatic stress disorder (PTSD) that uses oral MDMA tablets in conjunction with supportive psychiatric services. The FDA decision is expected on 11 August.

Lykos bases its application on data from the Phase III MAPP1 and MAPP2 trials, both of which met their primary efficacy endpoints. The MDMA-based regimen resulted in significant improvements in the Clinician-Administered PTSD Scale for DSM-5, a validated tool for diagnosing and assessing symptoms of PTSD.

The studies also met their secondary endpoints. MDMA treatment alleviated the functional impairments associated with PTSD. The trial regimen was also safe overall and was not associated with serious adverse events.

Despite these data, Lykos’ MDMA-assisted therapy has been the subject of controversy recently. In May 2024, the Institute for Clinical and Economic Review (ICER) published showed considerable uncertainties in the company’s clinical development programs, noting that the studies were “essentially unblinded” due to the psychedelic effects of MDMA.

ICER also announced that it had received reports that some study participants had been pressured to present the results of the Lykos trials in a “positive” manner.

A few weeks later, the FDA’s Psychopharmacologic Drugs Advisory Committee mentioned these problems in voted overwhelmingly against LykosThe panel of external experts concluded by a majority vote of 9 to 2 that the company had not provided sufficient data to support its MDMA-based candidate. On the question of the risk-benefit profile, the panel voted against Lykos by 10 to 1.

Zevra’s NPC drug must face newly established FDA adcome

On August 2, the FDA’s Genetic Metabolic Diseases Advisory Committee (GeMDAC). will meet to discuss the application for a new medicinal product for Zevra Therapeutics‘ oral drug candidate arimoclomol for the treatment of Niemann-Pick disease type C (NPC).

NPC is an extremely rare, progressive and neurodegenerative lysosomal storage disease characterized by severe physical and cognitive impairments as well as other neurological deficits manifesting as speech, motor and swallowing problems. NPC is irreversible and can lead to death within a few months.

NPC is caused by mutations in the NPC1 or NPC2 Genes that in turn affect the body’s ability to transport and break down cholesterol and other lipids within the cell. As a result, these molecules accumulate abnormally in various tissues and organs of the body, including the brain. There are currently no approved treatments for NPC.

Arimoclomol is designed to cross the blood-brain barrier. helps to make cells more robust and prevents their destruction even under stress. The drug candidate was already rejected by the FDA in June 2021, when it was still owned by Orphazyme.

In his renewed submission, which he submitted in December 2023Zevra addressed the regulator’s previous concerns by providing additional data to support arimoclomol’s mechanism of action. The biotechnology company also conducted nonclinical, natural history and real-world studies to better demonstrate arimoclomol’s efficacy and safety in NPC.