Biotech Stocks Facing FDA Decision In October 2024

(RTTNews) - September saw several notable "firsts" in the regulatory landscape alongside the approval of four novel drugs in the U.S.

Among these advancements, two new drugs to treat Niemann-Pick Disease, Type C, a rare genetic disease that results in progressive neurological symptoms and organ dysfunction, were approved by the FDA - IntraBio's Aqneursa and Zevra Therapeutics' Miplyffa.

Additionally, the first over-the-counter (OTC) hearing aid software device, Hearing Aid Feature, intended to be used with compatible versions of the Apple AirPods Pro headphones, received FDA authorization in the month.

Now, let's turn our attention to the upcoming drug approvals of October.

Biofrontera Inc. (BFRI)

The FDA decision on Biofrontera's request to increase the maximally approved dosage of Ameluz from one to three tubes per treatment is expected on October 4, 2024.

Ameluz, a topical gel, is used in combination with photodynamic therapy (PDT) using BF-RhodoLED lamp, a narrowband, red light illumination source, to treat a skin condition called actinic keratosis (warty overgrowths of skin) of mild-to-moderate severity on the face and scalp.

As per the current recommendation, no more than 2 grams of AMELUZ (one tube) should be applied in a single session and the application area should not exceed 20cm2.

Since many patients have actinic keratoses over large surface areas, the ability to treat these pre-cancerous lesions in one office visit will be more convenient for patients and more efficient for their dermatologists with simultaneous use of three tubes, according to the company.

BFRI closed Friday's (Sep.27, 2024) trading at $1.28, up 1.59%.

Zealand Pharma A/S (ZEAL.CO)

Zealand's investigational drug Dasiglucagon, proposed for the prevention and treatment of hypoglycemia in pediatric patients 7 days of age and older with congenital hyperinsulinism, is under FDA review.

The regulatory review is being conducted in two parts - Part 1 relates to dosing of Dasiglucagon up to three weeks, whereas Part 2 relates to the use beyond three weeks.

The FDA decision on Part I of the New Drug Application for Dasiglucagon is anticipated on October 8, 2024.

For Part 2 of the NDA, the FDA has requested additional analyses from existing continuous glucose monitoring (CGM) datasets, which the company expects to submit in the second half of 2024.

Bristol Myers Squibb (BMY)

Bristol Myers Squibb's applications seeking approval for neoadjuvant Opdivo with chemotherapy followed by surgery and adjuvant Opdivo for the perioperative treatment of resectable stage IIA to IIIB non-small cell lung cancer are under FDA review, with a decision due on October 8, 2024.

Non-small cell lung cancer is one of the most common types of lung cancer, representing up to 84% of diagnoses. Between 30% to 55% of non-small cell lung cancer patients who undergo surgery are said to experience disease recurrence.

If the proposed indication is approved, the company anticipates that Opdivo will provide a new Opdivo-based treatment option for patients who could benefit from it in the perioperative setting.

In the indication of NSCLC, Opdivo is already approved as a single and combination-based therapy for certain patients.

Melanoma, Metastatic, Renal Cell Carcinoma, Hodgkin's Lymphoma, Head and Neck Cancer, Urothelial Carcinoma, Colorectal Cancer, Hepatocellular Carcinoma, Esophageal Carcinoma, Malignant Pleural Mesothelioma, and Gastric Cancer are the other indications for which Opdivo has secured FDA approval.

The drug generated sales of $2.4 billion in the second quarter of 2024, compared to $2.1 billion in the year-ago quarter.

BMY closed Friday's trading at $50.91, up 1.58%.

Amgen (AMGN)

Amgen has sought FDA approval for its drug LUMAKRAS for, yet another indication and a decision is anticipated on October 17, 2024.

The proposed use involves LUMAKRAS in combination with Vectibix, compared to the investigator's choice of therapy, for treating KRAS G12C-mutated metastatic colorectal cancer. Vectibix, also from the stable of Amgen, is an approved drug for colorectal cancer.

LUMAKRAS was granted accelerated approval for the treatment of patients with KRAS G12C-mutated locally advanced or metastatic non-small cell lung cancer, following at least one prior systemic therapy, in May 2021.

The drug generated sales of $85 million in the second quarter of 2024, compared to $77 million in the year-earlier quarter.

If approved for the expanded indication, LUMAKRAS will need to compete with Bristol Myers Squibb's Krazati, which received accelerated approval for colorectal cancer in June of this year.

AMGN closed Friday's trading at $322.67, up 1.12%.

Camurus (CAMRF.OB)

The FDA will decide whether or not to approve Camurus' Oclaiz, proposed for the treatment of acromegaly, on October 21, 2024.

Acromegaly is a rare, slowly progressive disease, typically caused by a pituitary gland tumor that produces excess growth hormone, leading to elevated levels of insulin-like growth factor 1 (IGF-1). This causes abnormal growth of bones and tissues, resulting in enlarged hands, feet, facial features, and internal organs. Patients may also experience symptoms such as fatigue, joint pain, headaches, visual disturbances, excessive sweating, and tingling sensations.

Oclaiz is a once-monthly, subcutaneous (SC) long-acting depot formulation of the approved somatostatin analogue Octreotide. It is designed to enable easy self-administration, including the option of a pre-filled pen. In clinical trials, Oclaiz demonstrated an approximate five-fold higher bioavailability compared to the currently approved, long-acting, intramuscular Octreotide, according to the company.

CAMRF.OB closed Friday's trading at $61.90, up 6.32%.

Iterum Therapeutics plc (ITRM)

The FDA decision on Iterum Therapeutics' resubmitted New Drug Application for Oral Sulopenem is anticipated on October 25, 2024.

Oral Sulopenem is proposed for the treatment of uncomplicated urinary tract infections (uUTI) in adult women. This is the company's second attempt to secure FDA approval for oral Sulopenem in the proposed indication.

The U.S. regulatory agency declined to approve Oral Sulopenem in July 2021, requiring the company to conduct at least one additional adequate and well-controlled clinical trial. The company addressed the concerns raised by the FDA and resubmitted the New Drug Application for Oral Sulopenem in April of this year, which is now under FDA review.

On September 9, 2024, the FDA's Antimicrobial Drugs Advisory Committee (AMDAC) met to evaluate the overall benefits and risks of Oral Sulopenem for treating uncomplicated urinary tract infections (uUTI) caused by specific susceptible microorganisms in adult women over 18 and discussed the considerations that would be important for medical providers to know to ensure its appropriate use.

The FDA Committee recognized oral Sulopenem as an important treatment option for certain patients with uUTI.

ITRM closed Friday's trading at $1.05, up 0.96%.

PharmaTher Holdings Ltd. (PHRRF.OB)

PharmaTher Holdings' New Drug Application for Ketamine, which is under FDA review, is assigned a goal date of October 29, 2024.

Ketamine is an essential medicine used for anesthesia and analgesia (pain relief) and has been on the FDA's drug shortage list since February 2018.

Upon approval, the company is expected to launch Ketamine in the U.S., under the brand name Ketarx, in the first quarter of 2025.

PHRRF.OB closed Friday's trading at $0.20, up 5.79%.

Lexicon Pharmaceuticals Inc. (LXRX)

An FDA panel is slated to discuss Lexicon Pharma's drug candidate Zynquista as an adjunct to insulin therapy for glycemic control in adults with type 1 diabetes and chronic kidney disease on October 31, 2024.

Zynquista is an oral dual inhibitor of two proteins responsible for glucose regulation known as sodium-dependent glucose co-transporter types 1 and 2 (SGLT1 and SGLT2). SGLT1 is responsible for glucose absorption in the gastrointestinal tract, and SGLT2 is responsible for glucose reabsorption by the kidney.

This is Zynquista's second go-around with the U.S. regulatory agency.

The FDA declined to approve Zynquista in March 2019. The company resubmitted its New Drug Application for Zynquista in June of this year and awaits the FDA's final decision, which is due on December 20, 2024.

Zynquista was granted marketing authorization in the European Union in April 2019 as an adjunct to insulin therapy to improve glycemic control in adults with type 1 diabetes mellitus. However, the European Commission withdrew the drug from the European Union in March 2022 at the request of Guidehouse Germany GmbH, the holder of Zynquista's marketing rights in Europe, which opted not to sell the product for commercial reasons.

LXRX closed Friday's trading at $1.60, up 3.90%.