Nanoscope Therapeutics Begins FDA Rolling Submission for MCO-010 in Retinitis Pigmentosa

Nanoscope Therapeutics has initiated a rolling submission of its Biologics License Application (BLA) to the FDA for MCO-010, a first-in-class, gene-agnostic optogenetic therapy targeting severe vision loss due to retinitis pigmentosa (RP). The therapy, delivered through a single, in-office intravitreal injection, is designed to restore vision in patients regardless of their underlying genetic mutation. Full submission of the BLA is expected by early 2026, and the application is eligible for priority review under the therapy’s fast-track designation.

“For the first time, patients who are considered to be on a path to permanent blindness may have a chance to regain sight,” said Sulagna Bhattacharya, CEO and Co-Founder of Nanoscope. “We are deeply thankful to the FDA for their guidance as we remain steadfast in our mission to restore vision and bring light back into the lives of those living in darkness.”

MCO-010, based on Nanoscope’s proprietary multi-characteristic opsin (MCO), reactivates surviving retinal cells by converting them into light-sensitive cells, bypassing dead photoreceptors. In the pivotal phase IIb RESTORE trial, MCO-010 met its primary endpoint, demonstrating visual acuity improvements of more than 0.3 LogMAR in both dose arms at 52 weeks. Notably, sustained vision gains have been observed through three years of follow-up, with no serious adverse events reported.

“Based on the preclinical science and evidence in clinical trials, MCO-010 represents a potential, important paradigm shift for patients and retina specialists,” noted Dr. Allen C. Ho, Director of Retina Research at Wills Eye Hospital. “This therapy provides hope for meaningful improvement in the quality of life for the neediest retina patients of all.”

If approved, MCO-010 could become the first gene-agnostic treatment for inherited retinal disease, offering a new standard of care for over 100,000 individuals in the U.S. affected by RP.

FDA Approves Bayer’s KERENDIA for Heart Failure With Preserved Ejection Fraction

Bayer announced that the FDA has approved KERENDIA (finerenone) for the treatment of adults with heart failure (HF) with left ventricular ejection fraction (LVEF) ≥40%, marking a significant expansion of its indication. This approval, granted following a Priority Review of the supplemental New Drug Application, positions KERENDIA as the only FDA-approved non-steroidal mineralocorticoid receptor antagonist (nsMRA) for this patient population.

“The FDA’s approval of finerenone expands treatment options for patients with heart failure with a left ventricular ejection fraction of ≥40% – a large and growing group of patients with a poor prognosis,” said Dr. Scott D. Solomon, Professor of Medicine at Harvard Medical School and Chair of the FINEARTS-HF study executive committee. “Based on the clinical efficacy we saw in the FINEARTS-HF study, finerenone can become a new pillar of comprehensive care.”

The approval is based on results from the Phase III FINEARTS-HF trial, which demonstrated a 16% relative risk reduction in the composite primary endpoint of cardiovascular (CV) death and total HF events compared to placebo. The effect was consistent across all subgroups, including those with and without SGLT2 inhibitor use. With an estimated 3.7 million U.S. adults affected by HF with LVEF ≥40%, the approval addresses a high-risk population not adequately served by current therapies.

“Even with standard treatment, many patients with HF with preserved ejection fraction still face high risks of hospitalization or CV death,” said Dr. Alanna Morris-Simon, Senior Medical Director, U.S. Medical Affairs at Bayer. “Now, as a core pillar of treatment, KERENDIA can help reduce those risks and support better long-term outcomes.”

Klotho Neurosciences Receives Orphan Drug Status for KLTO-202 in Amyotrophic Lateral Sclerosis

Klotho Neurosciences has announced that its lead gene therapy candidate, KLTO-202 (s-KL-AAV.myo), has received Orphan Drug Designation (ODD) from the FDA for the treatment of amyotrophic lateral sclerosis (ALS). KLTO-202 is a novel gene and delivery system designed to express secreted Klotho protein (s-KL) and target neuromuscular junctions in motor neuron diseases, including ALS.

“Receiving the Orphan Drug Designation for s-KL-AAV.myo underscores the importance of bringing new treatment options to patients suffering from this rare, universally fatal disease,” stated Dr. Joseph Sinkule, CEO of Klotho Neurosciences. “We aim to deliver the first gene replacement therapy addressing the neurologic insult and motor neuron degeneration associated with ALS.”

The designation provides Klotho with several regulatory incentives, including seven years of U.S. market exclusivity, tax credits for clinical trials, and a waiver of FDA application fees. KLTO-202 is being developed for both ALS and muscular dystrophies, leveraging a desmin promoter to drive s-KL protein expression and targeting delivery to the neuromuscular junction.

Klotho has completed preclinical proof-of-concept studies in two ALS animal models and is preparing for large-scale manufacturing and regulatory meetings with the FDA and EMA to finalize its development path. “After the FDA’s review of the data leading to the Orphan Drug Designation, we believe this validates both our science and our approach to treating ALS,” added Dr. Sinkule.

Trethera’s TRE-515 Granted FDA Fast Track Designation for Metastatic Prostate Cancer

Trethera Corporation has announced that the FDA has granted Fast Track designation to its investigational therapy TRE-515 in combination with radioligand therapy for the treatment of PSMA-positive metastatic castration-resistant prostate cancer (mCRPC). TRE-515, currently being evaluated in a phase I clinical trial for solid tumors, is designed to enhance precision medicine by pairing with approved radiopharmaceuticals like PLUVICTO (lutetium Lu 177 vipivotide tetraxetan).

“This designation marks a critical milestone in our mission to develop more effective, well-tolerated therapies for aggressive and treatment-resistant prostate cancers,” said Dr. Ken Schultz, Chairman and CEO of Trethera. “By combining TRE-515 with radioligand therapy, we aim to advance precision oncology and deliver long-term benefits to patients.”

TRE-515 is a novel first-in-class therapy targeting advanced prostate cancer that expresses prostate-specific membrane antigen (PSMA), a biomarker present in over 80% of prostate cancer cases. The FDA’s decision was backed by promising data from Trethera’s first-in-human phase I trial and encouraging preclinical results presented at the AACR Annual Meeting. The therapy has shown early antitumor activity and favorable tolerability, with no dose-limiting toxicities across 18-fold dose escalation.

“The chemical structure of TRE-515 was designed for specific, on-target binding to create an optimal drug profile,” said Dr. Michael Jung, UCLA Professor and cofounder of Trethera. “I believe TRE-515 has the potential to make a meaningful difference in the lives of prostate cancer patients.” Dr. Johannes Czernin, Trethera cofounder and Professor of Nuclear Medicine at UCLA, added, “The TRE-515 designation offers continued hope to the mCRPC community in need of new, precision-based treatment options.”

Adcentrx’s ADRX-0405 Granted FDA Orphan Drug Designation for Gastric Cancer

Adcentrx Therapeutics announced that the FDA has granted Orphan Drug Designation to ADRX-0405, a STEAP1-targeting antibody-drug conjugate (ADC), for the treatment of gastric cancer. ADRX-0405 is currently being evaluated in the phase Ia portion of an ongoing phase Ia/Ib clinical trial (NCT06710379) for advanced solid tumors, including metastatic castration-resistant prostate cancer, gastric cancer, and non-small cell lung cancer.

“Receiving orphan drug designation from the FDA is a notable milestone for Adcentrx and reinforces the potential for ADRX-0405 to improve the lives of patients with gastric cancer,” said Hui Li, Ph.D., Founder and CEO of Adcentrx. “We are encouraged by the progress of our phase Ia trial and look forward to further evaluating the safety, tolerability, and anti-tumor activity of ADRX-0405.”

Though STEAP1 is primarily associated with prostate cancer, its meaningful expression in gastric tumors opens the door for broader therapeutic potential. The FDA’s orphan designation supports treatments for rare diseases affecting fewer than 200,000 people in the U.S., offering benefits such as tax credits, user fee waivers, and up to seven years of market exclusivity post-approval.

Gastric cancer remains a challenging malignancy, often diagnosed at later stages. With over 30,000 new cases expected in the U.S. in 2025, ADRX-0405 offers a promising new approach for a population with significant unmet need.