PARIS (dpa-AFX) - As July draws to a close, it's time to take a look at what's in store in the health and wellness space over the upcoming weeks.
August, the last month of summer, includes several health observances, such as National Immunization Awareness Month, Psoriasis Awareness Month, and Spinal Muscular Atrophy Awareness Month, to name a few.
Prevention is better than cure. The National Immunization Awareness Month (NIAM) is a campaign to highlight the importance of staying up to date on vaccinations in preventing serious diseases like measles, whooping cough, flu, and HPV-related cancers. According to the WHO, over the past 50 years, essential vaccines have saved at least 154 million lives and reduced infant deaths by 40%. In the U.S., the complete eradication of smallpox and the significant decline in cases of diphtheria, tetanus, pertussis, measles, mumps, and rubella can be attributed to the widespread use of vaccines.
Psoriasis is one of the most common immune-related diseases in the U.S., affecting the skin, nails, and joints. According to a report by Expert Market Research, more than 8 million Americans have psoriasis. The Psoriasis Awareness campaign aims to educate people about psoriasis, reduce stigma surrounding this condition, and support efforts to develop better treatments.
Spinal Muscular Atrophy (SMA) is a progressive neuromuscular disease that affects the functioning of nerve cells, affecting the patient's ability to walk, eat, or breathe. As part of raising awareness about this rare genetic disease and promoting research for developing advanced treatments, community events, social media campaigns, and fundraising initiatives will be undertaken.
Now, let's take a look at the biotech companies whose drug candidates are at the FDA altar, with decisions anticipated in August.
LENZ Therapeutics Inc. (LENZ)
LENZ Therapeutics' lead product candidate, LNZ100, proposed for the treatment of presbyopia, is under FDA review, with a decision expected on August 8, 2025.
Presbyopia is the inevitable loss of near vision associated with aging and impacts the daily lives of nearly all people over 45. In the U.S., Presbyopia affects an estimated 128 million people.
LNZ100 is a preservative-free, single-use, once-daily eye drop containing aceclidine.
In clinical trials, 71% of participants dosed with LNZ100 achieved three lines or greater improvement at 30 minutes, while 40% maintained that level of improvement at the 10-hour mark.
According to William Blair Analyst Tim Lugo, LNZ100, if approved, has the potential to achieve peak sales of $1 billion by 2030.
LENZ closed Tuesday's (Jul.29, 2025) trading at $31.67, up 0.51%.
PharmaTher Holdings Ltd. (PHRRF.OB)
The FDA decision on PharmaTher Holdings' resubmitted Abbreviated New Drug Application for racemic ketamine KETARX is due on August 9, 2025.
KETARX is proposed for the treatment of anesthesia, sedation, pain, mental health, and neurological indications.
This is KETARX's second go-around with the FDA. The U.S. regulatory agency had denied approval for it last April, citing deficiencies identified by Quality reviewers, and again in October 2024, requesting new and updated information and clarifications related to drug substance, drug product, manufacturing, and microbiology.
PHRRF.OB closed Tuesday's trading at $0.34, up 1.47%.
Insmed Inc. (INSM)
Insmed's New Drug Application seeking approval of Brensocatib for patients with bronchiectasis is under priority review by the FDA, with a decision due on August 12, 2025.
Bronchiectasis is a serious, chronic lung disease in which the bronchi become permanently dilated due to a cycle of infection, inflammation, and lung tissue damage. Symptoms include chronic cough, excessive sputum production, shortness of breath, and repeated respiratory infections, which can worsen the underlying condition. About 500,000 patients in the U.S., 600,000 patients in the EU5 (France, Germany, Italy, Spain, and the UK), and 150,000 patients in Japan suffer from bronchiectasis, and there are currently no approved therapies specifically targeting bronchiectasis in these regions.
Brensocatib is an oral, reversible inhibitor of dipeptidyl peptidase 1 (DPP1). The enzyme DPP1 is responsible for activating neutrophil serine proteases (NSPs), which are powerful proteins released by white blood cells (neutrophils). Excessively active NSPs can cause lung destruction and inflammation.
If approved, Brensocatib would be the first and only available treatment for bronchiectasis and the first DPP1 inhibitor.
According to GlobalData, Brensocatib has the potential to achieve sales of $1.2 billion by 2030.
INSM closed Tuesday's trading at $105, up 3.19%.
Tonix Pharmaceuticals Holding Corp. (TNXP)
The FDA decision on Tonix Pharma's TNX-102 SL, proposed for the management of fibromyalgia, is expected on August 15, 2025.
Fibromyalgia is a common chronic pain disorder that affects more than 10 million adults in the U.S., mostly women. Pfizer's Lyrica, Lilly's Cymbalta, and AbbVie's Savella are some of the FDA-approved drugs for fibromyalgia.
TNX-102 SL is a small, rapidly disintegrating, under-the-tongue (sublingual) product containing 2.8 mg of cyclobenzaprine HCl. It is a centrally acting analgesic that helps in relieving pain by improving sleep.
If approved by the U.S. regulatory agency, it would become the first member of a new class of non-opioid analgesic drugs for fibromyalgia and the first new drug for treating fibromyalgia in more than 15 years.
Analysts expect the drug to achieve peak sales of $500 million to $600 million in the indication of fibromyalgia, if approved.
TNXP closed Tuesday's trading at $42.63, down 9.87%.
Jazz Pharmaceuticals plc (JAZZ)
The FDA is scheduled to announce its decision on Dordaviprone, proposed for the treatment of H3 K27M-mutant diffuse glioma, on August 18, 2025.
H3 K27M-mutant diffuse glioma is a rare, aggressive brain tumor with low survival rates that most commonly affects children and young adults.
Developed by Chimerix, Dordaviprone became part of Jazz Pharma's pipeline after Jazz acquired Chimerix in March of this year.
Analysts at Truist Securities expect Dordaviprone, if approved, to achieve $800 million in peak sales by 2037.
JAZZ closed Tuesday's trading at $116.60, down 0.66%.
Regeneron Pharmaceuticals Inc. (REGN)
Regeneron Pharma has sought FDA approval to expand the label of EYLEA HD 8 mg to include the treatment of macular edema following retinal vein occlusion and to broaden the dosing schedule to include every 4 weeks (monthly) dosing across approved indications. The regulatory agency's decision is due on August 19, 2025.
Eylea HD is a higher dose formulation of Eylea, containing aflibercept 8 mg/0.07 mL. It is already approved in the U.S. for the treatment of Neovascular (Wet) Age-Related Macular Degeneration (wAMD), Diabetic Macular Edema (DME), and Diabetic Retinopathy (DR).
The current approved dosing for Eylea HD involves administration once every 8 to 12 weeks for DR or 8 to 16 weeks for wAMD and DME.
Eylea HD is being jointly developed by Regeneron and Bayer AG. The exclusive rights to EYLEA and EYLEA HD in the U.S. are held by Regeneron. Bayer has licensed the exclusive marketing rights outside of the U.S., where the companies share equally the profits from sales of EYLEA and EYLEA HD.
REGN closed Tuesday's trading at $558.15, down 0.09%.
PTC Therapeutics Inc. (PTCT)
The FDA decision on PTC Therapeutics Inc.'s (PTCT) Vatiquinone for the treatment of children and adults living with Friedreich's ataxia is anticipated on August 19, 2025.
Friedreich ataxia is a rare inherited condition that affects the central nervous system and the heart. It is caused by a mutation in FXN, a gene that provides instructions for making the mitochondrial protein frataxin. An estimated 4,000-5,000 individuals in the United States and between 18,000 and 20,000 people in the European Union have this genetic disease.
Vatiquinone works by inhibiting 15-Lipoxygenase (15-LO), an enzyme that is a key regulator of the energetic and oxidative stress pathways that are disrupted in Friedreich's ataxia.
Reata Pharmaceuticals' Skyclarys, approved in February 2023, is the first and only FDA-approved treatment for Friedreich's ataxia. Reata was acquired by Biogen in July 2023. The drug generated net revenue of $124 million for Biogen in the first quarter of 2025, up 59% year-over-year and 21% quarter-on-quarter.
PTCT closed Tuesday's trading at $49.29, up 11.16%.
Ionis Pharmaceuticals Inc. (IONS)
Ionis Pharma's Donidalorsen, an investigational RNA-targeted medicine, is at the FDA altar, with a decision expected on August 21, 2025.
Donidalorsen is proposed as a prophylactic treatment to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years of age and older.
Hereditary angioedema (HAE) is a rare and potentially life-threatening genetic condition that involves recurrent attacks of severe swelling (angioedema) in various parts of the body. HAE (Type 1 and Type 2) affects one in 50,000 people.
Donidalorsen is designed to reduce the production of prekallikrein (PKK), a protein that plays an important role in the activation of inflammatory mediators associated with acute attacks of HAE.
The drug was discovered and developed by Ionis Pharma. Theratechnologies has an exclusive license to commercialize Donidalorsen for use in Canada.
IONS closed Tuesday's trading at $41.48, up 0.75%.
Precigen Inc. (PGEN)
The U.S. regulatory agency is slated to announce its decision on Precigen's investigational gene therapy PRGN-2012 on August 27, 2025.
PRGN-2012 is proposed for the treatment of adults with Recurrent Respiratory Papillomatosis (RRP), a rare, non-cancerous viral tumor in the airways caused by the human papillomavirus (HPV) that can potentially be life-threatening. It is estimated that approximately 27,000 adult patients in the US and more than 125,000 patients outside of the US are affected by RRP.
There is no known cure for RRP, and the typical treatment involves frequent surgeries, which do not target the root cause of the disease and often lead to considerable complications.
PRGN-2012 is designed to elicit immune responses directed against cells infected with human papillomavirus (HPV) 6 or HPV 11. If approved, PRGN-2012 could become the first FDA-approved treatment for adults with RRP.
PGEN closed Tuesday's trading at $1.57, down 0.63%.
Telix Pharmaceuticals Limited (TLX)
Telix Pharma has sought FDA approval for its kidney cancer imaging PET drug product, Zircaix, and a decision is expected on August 27, 2025.
Zircaix is specifically proposed for the non-invasive diagnosis and characterization of clear cell renal cell carcinoma (ccRCC), the most common and aggressive form of kidney cancer.
In clinical trials involving adult patients with indeterminate renal masses (IRM), scheduled for partial or total nephrectomy (surgical removal of the kidney), Zircaix showed 86% sensitivity and 87% specificity and a 93% positive predictive value in detecting ccRCC.
If approved, Zircaix would be the first and only targeted PET agent specifically for kidney cancer to be commercially available in the U.S.
TLX closed Tuesday's trading at $13.49, down 3.37%.
Outlook Therapeutics Inc. (OTLK)
Come August 27, 2025, Outlook Therapeutics will learn whether or not its second attempt to secure FDA approval for ONS-5010, an investigational ophthalmic formulation of bevacizumab for the treatment of wet AMD, will pass regulatory muster.
Wet age-related macular degeneration (wet AMD) is a major cause of central visual impairment and blindness in people 65 years of age or older. It is estimated that 1.75 million people in the United States have wet AMD in 1 or both eyes.
ONS-5010 was denied FDA approval in August 2023 due to issues related to chemistry, manufacturing, and controls (CMC), as well as the need for additional confirmatory clinical evidence. Addressing the concerns mentioned in the Complete Response Letter that was issued at that time, the company resubmitted its BLA for ONS-5010 in February of this year and now awaits the decision.
ONS-5010, if approved, will be branded as LYTENAVA in the United States for the treatment of wet AMD and is expected to receive 12 years of regulatory exclusivity. LYTENAVA was approved in the EU for the treatment of wet AMD in May 2024.
OTLK closed Tuesday's trading at $1.97, down 1.99%.
Travere Therapeutics Inc. (TVTX)
Travere's supplemental New Drug Application requesting modification of liver monitoring and removal of embryo-fetal toxicity monitoring Risk Evaluation and Mitigation Strategy (REMS) for the kidney drug Filspari is at the FDA altar, with a decision scheduled for August 28, 2025.
Filspari was granted accelerated approval by the FDA for the reduction of proteinuria in IgA nephropathy in February 2023. The drug was granted full approval in the U.S. in September 2024 to slow the decline in kidney function in adults with primary IgAN who are at risk of disease progression.
Because of the risks of liver injury and birth defects, Filspari is available only through a restricted program called the Filspari REMS. Under the Filspari REMS, prescribers, patients, and pharmacies must enroll in the program.
In a bid to ease access for certain patients, the company has requested modification of liver monitoring and removal of pregnancy monitoring for Filspari.
Travere has a license agreement with Ligand Pharmaceuticals Inc. (LGND), signed in 2012, under which it obtained a worldwide sublicense to develop, manufacture, and commercialize Filspari. The company is obligated to pay Ligand an escalating royalty between 15% and 17% of net sales of Filspari, with payments due quarterly.
The drug generated U.S. net product sales of $55.9 million in the first quarter of 2025 compared to $19.8 million in the year-earlier quarter.
TVTX closed Tuesday's trading at $15.53, up 0.84%.
Sanofi (SNY)
Sanofi's investigational drug, Rilzabrutinib, being developed for the treatment of immune thrombocytopenia, awaits the FDA's decision on August 29, 2025.
Immune thrombocytopenia (ITP) is a rare, complex autoimmune disorder characterized by low platelet counts (less than 100,000/µL) resulting from both increased platelet destruction and decreased platelet production. People with ITP often experience bruising and bleeding, and in some cases, arterial or venous thrombosis. There are several approved drugs to treat this debilitating and life-threatening condition.
Rilzabrutinib is designed to block the activity of an enzyme called Bruton's agammaglobulinemia tyrosine kinase (Btk). This enzyme is involved in activating the immune system, leading to damage to platelets. By blocking Btk, the medicine is expected to reduce platelet damage caused by the immune system and improve symptoms of the condition (Source: European Medicines Agency).
SNY closed Tuesday's trading at $49.35, down 0.26%.
Eisai Co. Ltd. (ESALF.OB)
The FDA decision on Eisai's Biologics License Application for Leqembi subcutaneous autoinjector for weekly maintenance dosing is due on August 31, 2025.
Leqembi 100 mg/mL injection for intravenous use was approved in the U.S. for the treatment of Alzheimer's disease in 2023. In January of this year, the U.S regulatory agency expanded its approved use to include IV maintenance dosing to be administered once every four weeks.
Eisai and Biogen have a joint development and commercialization agreement for Leqembi. BioArctic has the right to commercialize Leqembi in the Nordic region.
The injection process with the proposed subcutaneous autoinjector is expected to take, on average, 15 seconds. The subcutaneous autoinjector is expected to be simple and easy for patients and their care partners to use and may reduce the need for hospital or infusion site visits and nursing care for IV administration. This could make ongoing maintenance treatment more convenient and further simplify the overall treatment process for Alzheimer's disease, according to the companies.
ESALF.OB closed Tuesday's trading at $25.59.
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