RegeneRx Biopharmaceuticals Enrolled First Patient in the Phase 3 Neurotrophic Keratitis Clinical Trial with RGN-259 in the US

On April 12, 2023, RegeneRx Biopharmaceuticals, Inc. (OTCQB: RGRX) (“RegeneRx” or “Company”) announced that the first patient of Phase 3 clinical trial (SEER-2) of RGN-259, a novel treatment for neurotrophic keratitis (NK), has been enrolled and begun treatment.  The product candidate, RGN-259, is under development by ReGenTree, LLC, a U.S. joint venture between RegeneRx and HLB Therapeutics.

RGN-259 is a novel drug candidate that promotes cell migration, anti-inflammation, and wound healing and whose active ingredient is thymosin beta 4 (Tβ4). RGN-259 is formulated as a sterile, preservative-free eyedrop and has been shown to be safe and well-tolerated. As per the update, clinical results in patients with NK treated with RGN-259 have previously demonstrated efficacy in a small number of patients (18) in the first phase 3 clinical trial (SEER-1), and two clinical trials are underway simultaneously in the United States and Europe based on these results.

As per the updates, ReGenTree is aiming to significantly shorten the clinical development period by conducting the two phase 3 clinical trials (SEER-2 and SEER-3) at the same time rather than sequentially.  The double-masked, placebo-controlled trials will be conducted by administering RGN-259 or placebo eye drops for four weeks to approximately 70 NK patients in each trial, with a primary endpoint of “complete corneal healing. Furthermore, ReGenTree plans to recruit more than 30 clinical institutions in the U.S. to accelerate patient recruitment.

As per DelveInsight, the total Neurotrophic Keratitis prevalent cases in the 7MM were 137,000+ in 2021. The Neurotrophic Keratitis cases in the 7MM are expected to increase in the coming years. Among all the seven major markets, the United States accounted for the highest number of prevalent cases of NK. There were 65,500+ prevalent cases of NK reported in 2021 in the US. Among the EU5 countries, the highest diagnosed prevalence of Neurotrophic Keratitis was recorded in the UK, followed by Germany. In 2021, Japan had a total of 12,400+ prevalent cases of Neurotrophic Keratitis, out of which 8,300 cases were actually diagnosed with Neurotrophic Keratitis.

Currently, Oxervate, from Dompé Farmaceutici in Italy, is currently the only approved treatment for NK in the United States and is one of the most expensive pharmacy drugs in the U.S. To provide effective therapeutic options in the market, several major pharmaceutical and biotech companies are actively evaluating their lead candidate for Neurotrophic Keratitis. The approval and launch of emerging therapies such as RGN-259, among others, are anticipated to improve the Neurotrophic Keratitis treatment scenario. 

Aldeyra Therapeutics Completes Enrollment in Phase 3 INVIGORATE-2 Clinical Trial in Allergic Conjunctivitis

On April 13, 2023, Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) (Aldeyra) announced the completion of enrollment in Phase 3 INVIGORATE-2 clinical trial of topical ocular reproxalap for the treatment of allergic conjunctivitis. Reproxalap, an investigational new drug candidate, is a first-in-class small-molecule modulator of RASP (reactive aldehyde species), which is elevated in ocular and systemic inflammatory disease. 

The mechanism of action of reproxalap has been supported by the demonstration of statistically significant and clinically relevant activity in multiple physiologically distinct late-phase clinical indications. The therapy is being studied in more than 2,300 patients with no observed clinically significant safety concerns; mild and transient instillation site irritation is the most commonly reported adverse event in clinical trials.

“Today, millions of allergic conjunctivitis patients rely on therapies that may not provide sufficient relief or cannot be used chronically due to serious side effects. Reproxalap, which has demonstrated rapid and durable improvement in the symptoms and signs of allergic conjunctivitis across a number of clinical trials, could signify the first new therapeutic mechanism of action in decades for patients suffering from this persistently disturbing condition.”

Todd C. Brady, M.D., Ph.D., President and Chief Executive Officer of Aldeyra

The randomized, double-masked, crossover, vehicle-controlled Phase 3 clinical trial enrolled 131 seasonal allergic conjunctivitis patients who were evaluated for 3.5 hours in an allergen chamber designed to simulate real-world pollen exposure. Consistent with pivotal trials of approved allergic conjunctivitis products, the primary endpoint of INVIGORATE-2 is patient-reported ocular itching. Top-line results from the trial are expected in the first half of 2023. The protocol of INVIGORATE-2 is substantially identical to that of the Phase 3 INVIGORATE clinical trial and a Phase 2 clinical trial, both of which achieved the ocular itching endpoint (P<0.001).

Allergic conjunctivitis is the most common inflammatory disease that affects the front of the eye and is characterized by itching, redness, and tearing. The signs and symptoms of allergic conjunctivitis are persistently disturbing, affecting the quality of life and leading to loss of work, which can create a substantial economic burden for patients and their families. At present, there are mainly eight therapeutic classes of drugs contributing towards the market size of Allergic conjunctivitis. The approved (and their generic versions), as well as off-label &/or OTC (over-the-counter) products, are being used to treat Allergic Conjunctivitis, belonging to different therapeutic classes. The ongoing clinical trial activities and commercial development in the therapeutics domain raise high hope for better treatment options in the Allergic conjunctivitis market in the upcoming years. 

Oncolyze Announces FDA Orphan Drug Designation for OM-301 for the Treatment of Multiple Myeloma

The FDA has granted an orphan drug designation to OM-301 for the treatment of patients with multiple myeloma, according to an announcement from Oncolyze.

OM-301 is an investigative fusion peptide that selectively induces cell death by binding to HDM2 on the surface of cancer cells and creating pores in them. The HDM2-binding element in OM-301 is taken from the HDM2-binding component of the p53 protein. Once one end of OM-301 attaches to HDM2 on the cell surface, the other end is designed to penetrate the cell membrane.

OM-301 has shown efficacy against multiple myeloma in preclinical research. It was able to target eight different multiple myeloma cell lines, including those with p53 mutations and null cell lines. Furthermore, in an in-vivo proof-of-concept study, OM-301 prolonged survival.

In addition, a preclinical study revealed that even though OM-301 was initially intended for p53-selective cells, it could potentially interact with BCL-2 to trigger mitochondrial dysfunction and cell death, regardless of TP53 status. These findings indicated that OM-301 could be a new and promising therapeutic alternative for individuals with multiple myeloma.

OM-301 was previously granted an orphan drug designation by the regulatory agency for the treatment of acute myeloid leukemia (AML).

OM-301 was observed to be effective in human AML that had been transplanted into mice. At 4, 8, and 12 weeks, mice that received treatment with OM-301 showed a significant decrease in the percentage of CD45-positive cells in the peripheral blood compared to mice that received the vehicle treatment.

The administration of OM-301 resulted in an almost twofold increase in survival in mice with human AML transplants (P < .0001). By day 100, all secondary transplant mice that were treated with OM-301 were alive, whereas only approximately 10% of those that received the vehicle treatment survived. None of the mice that were given the vehicle treatment survived beyond 110 days, whereas 50% of the OM-301 treated mice survived past 150 days.

Oncolyze is making progress with OM-301 by conducting Good Manufacturing Practice (GMP)-compliant toxicology studies. The company anticipates submitting an Investigational New Drug (IND) application to the FDA and initiating the first phase 1/2 clinical trial for OM-301 in 2023.

Soligenix Provides Regulatory Update on HyBryte™

Soligenix has announced that it held a Type A Meeting with the United States Food and Drug Administration (FDA). The purpose of the meeting was to discuss the contents of a refusal to file letter that the FDA had issued earlier concerning the company’s New Drug Application (NDA) for HyBryte™ (synthetic hypericin sodium). The drug was intended for the treatment of early-stage cutaneous T-cell lymphoma (CTCL), a rare cancer for which it demonstrated statistically significant results in a Phase 3 clinical trial (also known as the FLASH study or Study HPN-CTCL-01).

The FDA has specified that in order to approve the New Drug Application (NDA) for HyBryte™, positive results from a second clinical study are required, in addition to the Phase 3 randomized, double-blind, placebo-controlled FLASH study that was previously conducted for this orphan indication. The FDA has expressed willingness to discuss the protocol for the second clinical study. In response to this feedback, the company has decided to engage in discussions with the FDA to jointly define the protocol and assess the feasibility of conducting the additional clinical trial.

Soligenix’s President and CEO, Christopher J. Schaber, Ph.D., expressed disappointment regarding the delay caused by the refusal to file letter from the FDA, but emphasized the company’s continued commitment to collaborate with the FDA to bring HyBryte™ to the CTCL patient population. The Phase 3 FLASH study was the largest double-blind, randomized, placebo-controlled clinical trial ever conducted for CTCL, and despite the unexpected decision by the FDA, the company understands that the regulatory landscape has evolved. Soligenix is prepared to conduct another Phase 3 study to support the NDA for HyBryte™, and during the Type A meeting, they discussed the elements of the additional confirmatory study’s protocol design with the FDA. The company aims to work closely with the FDA to advance these discussions and design a study that meets the FDA’s requirements as quickly as possible.

SAB Biotherapeutics Granted Fast Track Designation from FDA for SAB-176 Influenza Immunotherapy with High Cross-Reactivity to Multiple Strains of Influenza

SAB Biotherapeutics, a clinical-stage biopharmaceutical company with a novel immunotherapy platform that generates specifically targeted, high-potency, fully human, multi-epitope binding immunoglobulin (hIgG) antibodies without the use of human donors, announced that the FDA had granted Fast Track designation for SAB-176, an investigational therapeutic for Type A influenza and Type B influenza illness in high-risk patients, including those who have h The Fast Track designation is intended to assist the accelerated development and expedited assessment of medications that treat urgent illnesses and meet an unmet medical need, with the objective of having promising treatments approved and available to patients as soon as feasible. SAB also received FDA guidance and regulatory alignment on moving SAB-176 into the next phase of development by initiating a Phase IIb dose-range finding effectiveness and safety trial in high-risk patient populations. 

“We are delighted to have received FDA Fast Track designation for SAB-176. “With an abnormally high number of hospitalisations and deaths each year, influenza remains one of the world’s most serious public health challenges. We are excited about SAB-176’s potential role in combating a highly mutagenic pathogen like influenza.”

Eddie Sullivan, PhD, co-founder, President & CEO of SAB Biotherapeutics

“SAB-176 has the potential to be a game-changer in the fight against influenza. Its multi-pronged mechanism of action, long half-life, and low risk of resistance emergence could make it a superior therapeutic for achieving and maintaining efficacy against this ever-evolving virus.” This Fast Track designation validates the promise of our revolutionary DiversitAb technology, which is designed to respond quickly to changing infectious illnesses like influenza.”

Alexandra Kropotova, MD, SAB Biotherapeutics’ Chief Medical Officer

SAB-176 has been subjected to a number of clinical and pre-clinical trials, including a Phase I trial in healthy volunteers and a Phase IIa challenge study that was completed last year. According to the findings, SAB-176 provides broad antibody protection against several strains of this rapidly evolving virus. SAB-176 demonstrated extensive cross protection in the Phase IIa research, including strains that were not particularly targeted in the therapeutic’s production.

Aviceda Announces FDA Clearance of the Investigational New Drug (IND) Application for AVD-104, a Novel Glyco-Mimetic Nanoparticle, Enabling Initiation of Phase 2 Clinical Trials for the Treatment of Geographic Atrophy from Macular Degeneration

Aviceda Therapeutics, a private clinical-stage biotech company focused on developing next-generation immunomodulators by harnessing the power of glycobiology to modulate the innate immune system and alleviate chronic, non-resolving inflammation, today announced that the FDA has cleared the IND for AVD-104, allowing the company to move forward with Phase II clinical trials of its lead intravitreal ocular asset for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The Phase II SIGLEC study is set to commence in the second quarter of 2023.

“The FDA’s acceptance of our IND application to initiate the clinical evaluation of AVD-104 brings us one step closer to introducing a potentially paradigm-shifting treatment for people living with geographic atrophy and macular degeneration. The AVD-104 IND clearance represents an important milestone for Aviceda as we continue our track record of execution as the glycome field leader.” We are excited to progress the development of AVD-104 in the United States and are working hard to enrol patients in the Phase II section of the research as soon as possible. We want to release additional statistics later this year.”

Mohamed Genead, M.D., Aviceda’s Co-Founder, President, and Chief Executive Officer

I am very excited to bring the power of our glycobiology-based technology platform to address the significant unmet medical needs in macular degeneration patients.” As a practising vitreoretinal surgeon, I witnessed firsthand the devastation caused by this disease on my patients. “This is the first step in bringing a novel and differentiated therapy to these patients.

Aviceda Chief Medical Officer David Callanan, M.D.

AVD-104 is an intravitreal nanoparticle molecule with a novel dual mechanism of action for the treatment of GA that involves the inhibition of retinal macrophage activity and the repolarization of activated macrophages to their resolution state, as well as the inhibition of complement cascade amplification. Age-related macular degeneration (AMD) is a leading cause of moderate to severe vision loss in adults over the age of 60 globally, impacting roughly 11 million people in the United States. As per DelveInsight analysis on the latest published “Age-related Macular Degeneration (AMD) Market”, report, the total prevalent cases of AMD were approximately 52 million in the 7MM, which might increase at a CAGR of 1.6% by 2032.