4 Innovative Drugs are Expected to be Approved by the FDA in June

In June, the FDA is expected to make approval decisions on the following four drugs

Drug name: spesolimab

Company name: Boehringer Ingelheim

Indications: Generalized Pustular Psoriasis (GPP)

Boehringer Ingelheim's spesolimab, a humanized selective antibody that blocks activation of the interleukin-36 receptor (IL-36R), is seeking FDA approval for the treatment of generalized pustular psoriasis Onset of disease (GPP). Rapid clearing of pustules in adult patients with GPP was observed in a 12-week phase 2 clinical trial after only one week of treatment. The FDA accepted the Biologics License Application (BLA) and granted it Priority Review status in December 2021, with a PDUFA date set for June 15. The drug also received orphan drug designation and breakthrough therapy designation in the United States. If approved, spesolimab would be the first FDA-approved drug to treat this potentially life-threatening rare skin disease with an urgent unmet need.

Drug name: AMX0035 (taurine diol + sodium phenylbutyrate)

Company Name: Amylyx Pharmaceuticals

Indication: Amyotrophic Lateral Sclerosis (ALS)

Amylyx's AMX0035 is a proprietary oral fixed-dose combination for the treatment of amyotrophic lateral sclerosis (ALS). AMX0035 is a combination of two drugs, sodium phenylbutrate and taurursodiol. Sodium phenylbutyrate is a small molecule protein chaperone that reduces unfolded protein response (UPC) and prevents UPC-induced cell death. Taurine diol is a Bax protein inhibitor. They can improve the health of mitochondria and endoplasmic reticulum within cells, thereby delaying the death of nerve cells. The New Drug Application (NDA) submitted to the U.S. FDA is based on positive data from a Phase 2 clinical trial that enrolled 137 patients with ALS. The trial met its primary efficacy endpoint of significantly slower decline in motor function in ALS patients treated with AMX0035 at the end of the 6-month randomization period according to the revised ALS Functional Rating Scale. The NDA for this therapy was accepted by the FDA in December 2021 and granted Priority Review status with a PDUFA target date of June 29, 2022.

An analysis of recently published data further shows that early use of AMX0035 reduces the incidence of death, tracheostomy or permanent assisted ventilation in ALS patients. The company is conducting a Phase 3 clinical trial and initiated an Expanded Access Program for AMX0035 in the U.S. in March 2022 for ALS patients unable to participate in a Phase 3 clinical trial. If approved, AMX0035 would provide a new treatment option that could help delay medical interventions, such as mechanical ventilation, in ALS patients.

Drug name: AXS-05 (dextromethorphan + bupropion)

Company Name: Axsome Therapeutics

Indications: Major Depressive Disorder (MDD)

Axsome's AXS-05 is a proprietary combination of oral drugs for the treatment of major depressive disorder (MDD). It consists of dextromethorphan and bupropion, and uses Axsome's metabolic inhibition technology. Dextromethorphan is a noncompetitive NMDA receptor antagonist that is also a sigma-1 receptor agonist, nicotinic acetylcholine receptor antagonist, and serotonin and norepinephrine transporter inhibitor. Bupropion increases the bioavailability of dextromethorphan, is also a norepinephrine and dopamine reuptake inhibitor, and a nicotinic acetylcholine receptor antagonist. The company submitted an NDA for the therapy in 2021 and was granted Priority Review status by the FDA. This NDA is supported by two randomized, double-blind, controlled clinical trials in patients with moderate-to-severe MDD (Phase 3 GEMINI, Phase 2 ASCEND). Among them, in the GEMINI trial involving 327 adult patients with moderate to severe MDD, AXS-05 rapidly and significantly improved the patients' depressive symptoms compared with the placebo group, meeting the primary endpoint of the trial.

Following Axsome's recent agreement with the FDA on postmarketing requirements, the company expects the FDA to respond to the NDA in the second quarter of 2022. AXS-05 has also received breakthrough therapy designation for the treatment of agitation in Alzheimer's disease (AD). It is currently in Phase 3 clinical development for AD agitation and Phase 2 clinical development for smoking cessation. If approved, the drug combination will lead to new treatments for MDD and potentially other central nervous system disorders.

Drug Name: Peamprilumab

Company name: Kangfang Bio, Chia Tai Tianqing (China Biopharmaceuticals)

Indication: metastatic nasopharyngeal carcinoma

The humanized anti-PD-1 monoclonal antibody penpulimab, co-developed by Acumen Biopharmaceuticals and Chia Tai Tianqing, a subsidiary of Chinese biopharmaceuticals, is awaiting FDA approval for third-line treatment of metastatic nasopharyngeal carcinoma. The BLA was submitted to the FDA in May 2021 and was reviewed using the Real-Time Oncology Review (RTOR) pilot program. The program aims to speed up the drug approval process. In the U.S., based on promising clinical data, Piamprimab has been granted Breakthrough Therapy Designation and Fast Track Designation for third-line metastatic nasopharyngeal carcinoma, as well as Orphan Drug Designation for the treatment of nasopharyngeal carcinoma. Due to its unique PD-1 binding epitope, the drug has the potential to achieve better clinical efficacy.