Biotech Stocks Facing FDA Decision In September 2023

(RTTNews) - As August draws to a close, let's turn our attention to the list of companies eagerly anticipating the FDA's verdict in September.

1. BioLineRx Ltd. (BLRX)

The FDA's decision on BioLineRx's Aphexda for the proposed use in stem cell mobilization for autologous transplantation in multiple myeloma patients is due on September 9, 2023.

Autologous hematopoietic stem cell transplantation is the standard treatment for a number of blood cancers, including multiple myeloma. It involves using healthy hemopoietic stem cells, responsible for producing blood cells, derived from the individual's own body to substitute unhealthy bone marrow or marrow that has been damaged by cancer treatment.

Prior to the collection of blood stem cells, patients receive daily doses of growth factors like G-CSF (granulocyte-colony stimulating factor) to enhance the production of stem cells and facilitate their migration into the bloodstream, allowing for subsequent collection.

The potential overall stem cell mobilization market in the U.S. is estimated to be over $300 million.

BLRX closed Wednesday's (Aug.30, 2023) trading at $ 1.39, up 4.51%.

2. Alnylam Pharmaceuticals Inc. (ALNY)

Alnylam Pharma's lead drug Onpattro is scheduled to be reviewed by an FDA panel for the expanded use in treating cardiomyopathy of transthyretin-mediated (ATTR) amyloidosis on September 13, 2023.

Onpattro, an intravenously administered RNAi therapeutic, is already approved in the U.S. and Canada for the treatment of the polyneuropathy of hereditary ATTR amyloidosis in adults. The drug is also approved in the European Union, Switzerland and Brazil for the treatment of hATTR amyloidosis in adults with Stage 1 or Stage 2 polyneuropathy, and in Japan for the treatment of hATTR amyloidosis with polyneuropathy.

The regulatory agency's final decision on the expanded use of Onpattro is expected on Oct.8, 2023.

The global net product revenues for Onpattro in the second quarter of 2023 was $91 million, down 40% from the year-ago quarter.

ALNY closed Wednesday's trading at $197.16, up 1.39%.

3. Roche Holding AG (RHHBY.OB)

The FDA decision on Roche's subcutaneous formulation of its blockbuster drug Tecentriq in the treatment of non-small cell lung cancer patients is set for September 15, 2023.

Tecentriq, in intravenous formulation, is approved for non-small cell lung cancer, small cell lung cancer, hepatocellular carcinoma, melanoma, and alveolar soft part sarcoma. The drug generated annual sales of over US$4 billion in 2022.

According to the company, administered under the skin, the subcutaneous formulation reduces time spent receiving treatment to just minutes, compared with up to an hour for IV infusion.

The investigational subcutaneous formulation combines Tecentriq with Halozyme Therapeutics' Enhanze drug delivery technology.

RHHBY.OB closed Wednesday's trading at $37.30, up 0.32%.

4. GSK plc (GSK)

GSK's Momelotinib, an inhibitor of proteins JAK1, JAK2 and ACVR1, is under FDA review, with a decision anticipated on September 16, 2023.

Momelotinib is proposed for the treatment of patients with intermediate/high-risk myelofibrosis who have previously received a JAK inhibitor.

Myelofibrosis is a rare blood cancer that results from dysregulated JAK/STAT (signal transducer and activator of transcription) signaling and is characterized by constitutional symptoms, splenomegaly, and progressive anemia.

Inhibition of the JAK1 and JAK2 proteins could improve constitutional symptoms and splenomegaly. Moreover, direct inhibition of the ACVR1 protein has the potential to lower elevated hepcidin levels, which play a role in anemia associated with myelofibrosis.

Momelotinib was developed by Sierra Oncology Inc., and it came under GSK's fold when it acquired the former in July 2022.

GSK closed Wednesday's trading at $35.49, down 0.06%.

5. ARS Pharmaceuticals Inc. (SPRY)

The FDA decision on ARS Pharma's Neffy (epinephrine nasal spray) is due on September 19, 2023.

Neffy is proposed for the treatment of severe allergic reaction (Type 1), including anaphylaxis, for adults and children who weigh more than 30kg.

In May of this year, an FDA panel, which scrutinized Neffy, voted 16:6 recommending the product for adults, and 17:5 in favor for children. The regulatory agency usually follows the recommendations of its panels, although it is not required to do so.

Neffy (epinephrine nasal spray), if approved, could potentially become the first non-injectable medicine indicated to treat type I allergic reactions, including anaphylaxis, in the United States.

SPRY closed Wednesday's trading at $7.11, up 0.28%.

6. Brainstorm Cell Therapeutics Inc. (BCLI)

A panel of advisers to the FDA is expected to review Brainstorm Cell Therapeutics' investigational mesenchymal stem cell therapy NurOwn on September 27, 2023.

NurOwn is proposed for the treatment of amyotrophic lateral sclerosis (ALS), a rare disease that attacks and kills the nerve cells controlling voluntary muscles.

About 5,000 individuals in the United States are diagnosed with ALS annually, and about 20,000 Americans are currently living with this disease.

Amylyx Pharma's Relyvrio, approved in September 2022, is the latest ALS drug to make it to the market.

In a pivotal trial, NurOwn did not reach statistical significance on the primary or secondary endpoints in patients with more advanced disease but showed evidence of clinically meaningful effectiveness in ALS participants who have not progressed to advanced levels of disease progression, according to the company.

The FDA's final decision on NurOwn is expected by December 8, 2023.

BCLI closed Wednesday's trading at $1.68, down 4%.

7. Ocuphire Pharma Inc. (OCUP)

Ocuphire Pharma's Nyxol Eye Drops, proposed for the reversal of pharmacologically-induced mydriasis, (dilation of the pupil), is under FDA review, with a decision expected on September 28, 2023.

About 100 million eyes are dilated in the U.S. every year. This procedure is done to examine the back of the eye either for routine check-ups, disease monitoring or surgical procedures across all eye care practice groups.

Nyxol, a proprietary, preservative-free, stable, investigational eye drop formulation of phentolamine ophthalmic solution 0.75%, is designed to work by reducing pupil size through acting on the iris dilator muscle, allowing patients to return to their normal pupil size more rapidly.

If approved, Nyxol has the potential to be the first and only FDA-approved agent for the reversal of mydriasis uniquely modulating the iris dilator muscle.

The market for reversing mydriasis is projected to surpass $500 million.

OCUP closed Wednesday's trading at $3.99, up 0.76%.

8. Appili Therapeutics Inc. (APLI.TO, APLIF.OB)

The FDA decision on Appili's ATI-1501, a proprietary taste-masked liquid suspension formulation of metronidazole, is expected on September 23, 2023.

Metronidazole is a widely used front-line oral treatment for parasitic and anaerobic bacterial infections that predominantly affect elderly individuals.

Appili has commercialized the rights to ATI-1501 for Europe, Latin America and the United States markets to Saptalis Pharmaceuticals, a New York-based specialty pharmaceuticals company. Meanwhile, Appili still holds the worldwide rights to ATI-1501, excluding the United States.

APLIF.OB closed Wednesday's trading at $0.03. down 4.02%.