Biotech Stocks Facing FDA Decision In September 2024

(RTTNews) - As September unfolds, let us review some of the regulatory news that made headlines last month and look ahead to what this month has in store.

Adaptimmune Therapeutics plc's (ADAP) Tecelra was granted accelerated approval by the FDA on Aug.1, 2024, for the treatment of adults with unresectable or metastatic synovial sarcoma, a rare form of cancer, becoming the first new treatment option for this disease in more than a decade. It also represents the first gene therapy for metastatic synovial sarcoma.

On August 7, the FDA approved Purdue Pharma's Zurnai, the first nalmefene hydrochloride auto-injector for the emergency treatment of known or suspected opioid overdose in adults and pediatric patients 12 years of age and older.

Another notable first on the regulatory front is the FDA approval of ARS Pharmaceuticals' (SPRY) neffy, the first nasal spray for the treatment of anaphylaxis, on Aug.9.

To enhance protection against the currently circulating variants and mitigate the serious consequences of COVID-19, the FDA approved updated mRNA COVID-19 vaccines manufactured by Moderna Inc. (MRNA) and Pfizer Inc (PFE) on Aug.22.

Another headline that captured global attention in August was the WHO declaring the Mpox outbreak as a public health emergency of international concern on Aug.14. Emergent BioSolutions Inc.'s (EBS) smallpox vaccine ACAM2000, on Aug.30, received FDA approval for expanded use in the prevention of Mpox disease in high-risk individuals.

Now, let's turn our attention to the biotech stocks that are anticipating FDA decisions in September.

Travere Therapeutics Inc. (TVTX)

The FDA is scheduled to determine whether or not to convert accelerated approval of Travere Therapeutics' Filspari to a traditional approval on September 5, 2024.

Filspari was granted accelerated approval in the U.S. to reduce proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression in February 2023. The drug generated net product sales of $27.1 million in the second quarter of 2024, compared to $3.46 million in the year-earlier quarter.

IgA nephropathy, also known as Berger's disease, is a rare progressive kidney disease. It occurs when a protein called immunoglobulin A (IgA), which normally helps fight infections, builds up in the kidneys. This buildup affects the normal filtering mechanisms in the kidney, leading to blood in the urine (hematuria), protein in the urine (proteinuria) and a progressive loss of kidney function over time.

Under a license agreement with Ligand Pharmaceuticals (LGND), Travere remains obligated to make payments to Ligand Pharma upon achievement of certain regulatory and sales milestones.

TVTX closed Friday's trading at $9.47, up 1.18%.

Avadel Pharmaceuticals plc (AVDL)

Avadel Pharma has sought FDA approval to expand the use of its lead drug Lumryz for the treatment of cataplexy or excessive daytime sleepiness (EDS) in the pediatric narcolepsy population. A decision is due on September 7, 2024.

Lumryz, indicated to be taken once at bedtime, received tentative approval from the FDA for the treatment of cataplexy or excessive daytime sleepiness in adults with narcolepsy in July 2022, and final approval last May. The drug was launched in the U.S. on June 5, 2023.

Net product revenue from Lumryz sales was $45.1 million in the second quarter of 2024, compared to $1.5 million in the second quarter of 2023.

AVDL closed Friday's trading at $15.17, up 1.88%.

Iterum Therapeutics plc (ITRM)

An FDA panel is scheduled to review Iterum Therapeutics' resubmitted New Drug Application for oral Sulopenem on September 9, 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 the investigational drug.

The U.S. regulatory agency had 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.

The final FDA decision is expected on October 25, 2024.

ITRM closed Friday's trading at $1.18, up 4.42%.

Roche Holding AG (RHHBY.OB)

The FDA decision on Roche Holding AG's (RHHBY) Ocrevus SC, proposed for the treatment of relapsing multiple sclerosis and primary progressive multiple sclerosis, is expected on September 13, 2024.

Ocrevus SC is a 10-minute injection, co-formulated with ENHANZE, Halozyme Therapeutics Inc.'s (HALO) proprietary recombinant human hyaluronidase enzyme, rHuPH20. It retains the twice-yearly dosing regimen of Ocrevus IV that was approved in the U.S in 2017 for both relapsing and primary progressive forms of multiple sclerosis.

If approved, Ocrevus SC has the potential to expand the usage of Ocrevus to treatment centers without IV infrastructure or with IV capacity limitations.

RHHBY.OB closed Friday's trading at $42.33, down 0.05%.

Vanda Pharmaceuticals Inc. (VNDA)

Vanda Pharma's lead drug candidate Tradipitant, proposed for the treatment of symptoms of gastroparesis, is under FDA review, with a decision due on September 18, 2024.

Gastroparesis is a serious medical condition characterized by delayed gastric emptying. This delay in gastric emptying can cause symptoms such as nausea, vomiting, bloating, fullness after meals and abdominal pain, as well as affect a person's social and work life. An estimated 6 million people in the U.S. are affected by gastroparesis, though many of these cases go undiagnosed.

If approved, Tradipitant will be the first novel drug to be approved by the FDA for the treatment of gastroparesis in over 40 years.

While the review is still in progress, the FDA recently issued a preliminary notice indicating that there are deficiencies that prevent discussion about labeling.

VNDA closed Friday's trading at $5.29, up 1.34%.

Zevra Therapeutics Inc. (ZVRA)

The FDA decision on Zevra Therapeutics Inc.'s (ZVRA) Arimoclomol, proposed for the treatment of Niemann-Pick disease type C, is expected on September 21, 2024.

Niemann-Pick disease type C (NPC) is an ultra-rare, progressive, and neurodegenerative disorder caused by mutations in the NPC1 or NPC2 genes, which are responsible for making lysosomal proteins. Patients with NPC experience restrictions in both physical and cognitive abilities, characterized by significant neurological issues affecting speech, thinking, swallowing, walking, and precise movements.

This is the company's second attempt to secure FDA approval for Arimoclomol. The U.S. regulatory agency had declined to approve Arimoclomol in June 2021, requiring the company to submit additional data.

After addressing the concerns raised by the FDA, the company resubmitted the New Drug Application for Arimoclomol in December 2023, and now awaits the agency's final decision.

Meanwhile, on August 2, 2024, an FDA panel, which reviewed the Arimoclomol NDA voted 11-5 recommending its approval.

If approved, Arimoclomol would become the first authorized treatment specifically designed to slow the progression of Niemann-Pick disease type C.

Arimoclomol was initially developed by LadRx Corp. (LADX.OB), and in 2011, it sold the drug rights to Orphazyme A/S (now Zevra Therapeutics) in exchange for milestone payments and royalties. Later in 2023, XOMA Corp. (XOMA) acquired the royalty and milestone rights associated with Arimoclomol from LadRx.

ZVRA closed Friday's trading at $7.65, up 3.52%.

Heron Therapeutics Inc. (HRTX)

Heron Therapeutics' Prior Approval Supplement application for Zynrelef Vial Access Needle is under FDA review, with a decision anticipated on September 23, 2024.

Zynrelef is an extended-release, fixed-dose combination of the local anesthetic Bupivacaine and the nonsteroidal anti-inflammatory drug (NSAID) Meloxicam indicated for postoperative pain management. Currently, it is delivered via a single, needle-free application into the surgical site.

If approved, the introduction of the Vial Access Needle (VAN) will replace the current vented vial spike and has the potential to simplify aseptic preparation, while also significantly reducing Zynrelef's withdrawal time from up to three minutes down to between twenty and forty-five seconds. The user-friendly "container-like" design of the VAN may enhance the safe use of Zynrelef, increase adoption, and improve the preparation process, according to the company.

HRTX closed Friday's trading at $1.93.

Merck & Co., Inc. (MRK)

Merck has sought FDA approval to expand the use of its blockbuster cancer drug Keytruda to include the first-line treatment of patients with unresectable advanced or metastatic malignant pleural mesothelioma in combination with chemotherapy and a decision is expected on September 25, 2024.

Malignant mesothelioma is a cancer that originates in the linings of specific body areas, such as the chest, abdomen, heart, and testicles. Pleural mesothelioma, which develops in the lining of the lungs, is the most common form of malignant mesothelioma, accounting for about 75% of all cases. Malignant pleural mesothelioma often progresses rapidly, and the five-year survival rate is only 12%.

Keytruda is already approved in the U.S. for several indications, say, melanoma, metastatic, non-small cell lung cancer, Merkel cell carcinoma, renal cell carcinoma, esophageal carcinoma, head and neck cancer, Hodgkin's lymphoma, urothelial carcinoma, gastric cancer, cervical cancer, hepatocellular carcinoma, endometrial cancer, squamous cell carcinoma, hepatocellular carcinoma, breast cancer, and biliary tract tumor.

MRK closed Friday's trading at $118.45, up 0.84%.

Bristol-Myers Squibb Co. (BMY)

The FDA decision on KarXT, an antipsychotic, proposed for the treatment of schizophrenia in adults, is due on September 26, 2024.

KarXT came under Bristol-Myers Squibb's fold, following the acquisition of Karuna Therapeutics early this year for $14 billion. PureTech Health plc (PRTC) is a founder of Karuna and co-inventor of the KarXT program.

KarXT acts as a dual M1/M4 muscarinic acetylcholine receptor agonist in the central nervous system, which is thought to improve positive, negative, and cognitive symptoms of schizophrenia. Unlike existing treatments, KarXT does not directly block dopamine receptors, representing a potential new approach to treating schizophrenia.

If approved, KarXT will be the first new mechanism in over 50 years for patients living with schizophrenia.

BMY closed Friday's trading at $49.95, up 0.46%.

Regeneron Pharmaceuticals Inc. (REGN)

Regeneron Pharma and Sanofi (SNY) have sought FDA approval for the expanded use of Dupixent as an add-on maintenance treatment in certain adult patients with uncontrolled chronic obstructive pulmonary disease, and a decision is due on September 27, 2024.

Dupixent is being jointly developed by Regeneron and Sanofi under a global collaboration agreement.

The drug is already approved in the U.S. for the treatment of atopic dermatitis, asthma, chronic rhinosinusitis with nasal polyposis, eosinophilic esophagitis, and prurigo nodularis.

REGN closed Friday's trading at $1,184.69, up 0.49%.

Sanofi (SNY)

Sanofi's Sarclisa, in combination with a standard-of-care VRd regimen, proposed for the treatment of patients with transplant-ineligible newly diagnosed multiple myeloma is under priority review by the FDA, with a decision anticipated on September 27, 2024.

VRd is a three-drug regimen of bortezomib, lenalidomide, and dexamethasone and is the preferred first-line treatment option for patients with newly diagnosed multiple myeloma. (Source: NEJM).

The proposed indication, if approved, would be the third one for Sarclisa. The drug, in combination with pomalidomide and dexamethasone secured FDA approval for the treatment of adult patients with multiple myeloma who have received at least 2 prior therapies including lenalidomide and a proteasome inhibitor in March 2020. The following year, i.e., in March 2021, Sarclisa in combination with carfilzomib and dexamethasone was approved by the FDA for the treatment of adult patients with relapsed or refractory multiple myeloma who have received 1 to 3 prior lines of therapy.

SNY closed Friday's trading at $56.26, down 0.12%.