Attention Biotech Investors: Mark Your Calendar For These June PDUFA Dates

Zinger Key Points
  • PDUFA dates are key make-or-break catalysts for biotech stocks
  • A PDUFA goal date refers to the deadline by which the FDA has to announce its verdict regarding the approvability of a drug
  • The PDUFA calendar for June is fairly loaded, with Regeneron-Sanofi, Bristol-Myers Squibb among companies knocking at the FDA altar

May turned out to be a month of mixed fortunes as far as regulatory reviews are concerned. New molecular entity approvals, an indicator of innovation in drug research, totaled three during the month, bringing the total for the year to 15. This compares to the 24 NMEs the Food and Drug Administration approved by May 2021.

Among the companies that received the FDA's greenlight were Phathom Pharmaceuticals, Inc. PHAT, Bristol-Myers Squibb Company BMY and Novartis AG NVS.

On the other hand, Verrica Pharmaceuticals Inc.'s VRCA viral skin infection treatment candidate was rejected by the regulator for the third time. Amid the adverse ruling, the stock lost about three-quarters of its value. The FDA extended the review periods of Pfizer, Inc. PFE-Myovant Sciences, Inc. MYOV combo's application to expand the label of Myfembree and Amicus Therapeutics, Inc.'s FOLD Pome disease application.

Here are the key regulatory decisions scheduled for June:

Acer-Relief Await Nod For Urea Cycle Disorder Treatment

Company: Acer Therapeutics, Inc. ACER & Relief Therapeutics Holding AG RLFTF
Type of Application: new drug application (NDA)
Candidate: ACER-001 (sodium phenylbutyrate)
Indication: urea cycle disorders
Date: June 5

The two companies are seeking approval for ACER-001 under the 505(b)(2) pathway, which allows manufacturers to apply for approval without having to repeat all the drug development work done for an innovator drug.

UCDs are a group of disorders caused by genetic mutations that result in deficiency of one of the six enzymes that catalyze the urea cycle. This can lead to an excess accumulation of ammonia in the bloodstream. Acute hyperammonemia can cause lethargy, somnolence, coma, and multi-organ failure, while chronic hyperammonemia can lead to headaches, confusion, lethargy, failure to thrive, behavioral changes, and learning and cognitive deficits.

Current medical treatments for patients with UCDs include Horizon Therapeutics plc.'s HZNP Ravicti and Buphenyl. Acer noted that there have been reports of non-compliance associated with available medications due to unpleasant taste, frequency with which medication must be taken, required number of pills, and the high cost of the medication.

Can Regeneron-Sanofi's Dupixent Snag Another Nod

Company: Regeneron Pharmaceuticals, Inc. REGN & Sanofi SNY
Type of Application: supplemental biologic license application (sBLA)
Candidate: Dupixent
Indication: atopic dermatitis
Date: June 8

The FDA accepted the sBLA for priority review on Feb. 10, with the application seeking approval of the combo's inflammatory disease drug as an add-on maintenance treatment for children, aged 6 months to 5 years, with moderate-to-severe atopic dermatitis.

Atopic dermatitis is a chronic type 2 inflammatory skin disease, and 85%-90% of patients develop symptoms before 5 years of age, which can often continue through adulthood. Current treatment options in this age group are primarily topical steroids, which can be associated with safety risks and may impair growth when used long-term.

Related Link: As COVID Subsidies, Here Are 8 Infectious Diseases That Could Emerge As Potent Threats

Rhythm Strives to Score Another Approval For Obesity Drug

Company: Rhythm Pharmaceuticals, Inc. RYTM
Type of Application: supplemental NDA
Candidate: Imcivree (setmelanotide)
Indication: Bardet-Biedl syndrome or Alström syndrome
Date: June 16

Rhythm, which develops treatments for rare genetic diseases of obesity, has an FDA decision date of June 16 for its Imcivree, which is being evaluated as a treatment option for obesity and the control of hunger in adult and pediatric patients 6 years of age and older with BBS or Alström syndrome.

The original PDUFA date of March 16 was extended by three months, as the FDA needed additional time to review clinical efficacy data submitted by the company upon the agency's request to conduct additional subgroup analyses.

Can Bristol-Myers Squibb's Blood Cancer Therapy Get Label Expansion?

Company: Bristol-Myers Squibb
Type of Application: sBLA
Candidate: Breyanzi (lisocabtagene maraleucel)
Indication: large B-cell lymphoma
Date: June 24

Breyanzi is a CAR T cell therapy that has already been approved for treating patients with relapsed or refractory large B-cell lymphoma, a type of blood cancer, after two or more lines of systemic therapy. Bristol-Myers Squibb's application for label expansion seeks approval of the cell therapy for large B-cell lymphoma patients following a failed first-line therapy.

The standard-of-care currently is salvage chemotherapy followed by a high-dose chemotherapy, plus an autologous stem cell transplant. The company now thinks Breyanzi has the potential to be a new standard-of-care.

Bristol-Myers Squibb-Merk Expect Smooth Sailing For Reblozyl Following Extended Review Period

Company: Bristol-Myers Squibb & Merck & Company, Inc. MRK
Type of Application: sBLA
Candidate: Reblozyl (luspatercept)
Indication: anemia in adults with non-transfusion dependent beta thalassemia
Date: June 27

Reblozyl is an erythroid maturation agent that promotes late-stage red blood cell maturation in animal models. It's being jointly developed by Bristol-Myers Squibb and Merck in the U.S. Merck received rights to the treatment through its acquisition of Acceleron. The FDA had extended the original review period of March 27 by three months.

Related Link: The Week Ahead In Biotech (May 30-June 4): FDA Decision For Eton's Partnered Epilepsy Drug, ASCO Conference Gets Underway And More

Spero's Lead Drug May Not Receive Approval Due to Data Insufficiency

Company: Spero Therapeutics, Inc. SPRO
Type of Application: NDA
Candidate: tebipenem HBr oral tablets
Indication: complicated urinary tract infections (cUTI)
Date: June 27

Tebipenem HBr is Spero's lead product candidate that is being developed as the first oral carbapenem antibiotic for use in cUTI, including pyelonephritis.

Spero said in early May that its discussions with the FDA suggested that the data package may be insufficient to support approval during this review cycle. Following the development, the company announced a reduction in its workforce by approximately 75% and a restructuring of its operations to reduce operating costs and reallocate resources towards the clinical development programs of SPR720 and SPR206.

Is Amylyx In For Disappointment With Its ALS Drug?

Company: Amylyx Pharmaceuticals, Inc. AMLX
Type of Application: NDA
Candidate: AMX0035
Indication: amyotrophic lateral sclerosis (ALS)
Date: June 29

FDA's Peripheral and Central Nervous System Drugs Advisory Committee, which met in late March to discuss the NDA, voted that the submitted Phase 2 data do not support the efficacy of the drug in treating ALS.

Adcom Calendar

FDA's Vaccines and Related Biological Products Advisory Committee (VRBPAC) is scheduled to meet on June 7 to discuss Novavax, Inc.'s NVAX emergency use authorization (EUA) request for its vaccine to prevent COVID-19 in individuals 18 years of age and older.

The Cellular, Tissue, and Gene Therapies Advisory Committee will deliberate on bluebird bio, Inc.'s BLUE two BLAs for gene therapy candidates elivaldogene autotemcel and betibeglogene autotemcel. The former is being evaluated for the treatment of patients less than 18 years of age with early cerebral adrenoleukodystrophy and the latter for treating patients with β-thalassemia who require regular red blood cell transfusions.

The Adcom is scheduled to meet for two days – on June 9, between 10 am ET and 6 pm ET, and on June 10, between 10 am ET and 4 pm ET.

The VRBPAC will meet again on June 14 and 15 to discuss expanding the EUA accorded to the COVID-19 vaccines of Moderna, Inc. MRNA and Pfizer, Inc. PFE-BioNTech SE BNTX. On June 14, the committee will discuss authorization of the primary series of Moderna's vaccine for children and adolescents, six years through 17 years.
The very next day, the committee will review the application for expanding the authorizations of the vaccines of both Moderna and Pfizer-BioNTech for infants and children, six months through four years of age.

On June 17, the Psychopharmacologic Drugs Advisory Committee will discuss sNDAs for Nuplazid tablets, submitted by Acadia Pharmaceuticals Inc. ACAD, for the proposed treatment of hallucinations and delusions associated with Alzheimer's disease psychosis. Incidentally, Nuplazid was rejected by the FDA for the same indication in April 2021.

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