SARM1

CINCINNATI--(BUSINESS WIRE)-- Asha Therapeutics (Asha) ( ), a life sciences company designing de novo disease modifying medicines for neurodegenerative diseases with high unmet medical need, announced today the nomination of a development candidate, ASHA-624 targeting SARM1 as a potential disease modifying therapy for Amyotrophic Lateral Sclerosis (ALS) with additional indications in Chemotherapy-Induced Peripheral Neuropathy (CIPN), Glaucoma, and traumatic brain and spinal cord injuries. This announcement follows the Company’s presentation of robust efficacy and safety data on its second lead neurology program, ASHA-091 in Alzheimer’s disease and Parkinson’s disease by Chief Scientific Officer & Scientific Co-founder Dr. Bradlee Heckmann earlier this month at the AD/PD 2024 International Conference in Lisbon, Portugal (see full release here). Dr. Michael Gold, MD, MS, a member of Asha’s Scientific Advisory Board noted, “SARM-1 is a well-validated therapeutic target that could yield novel therapies for patients suffering from a range of both central and peripheral nervous disorders. ASHA-624 prevents the activation of SARM-1 using a completely novel approach that has the potential to deliver a therapy with robust clinical efficacy and few, if any, off-target side effects. As a veteran CNS drug developer, I am excited to see this compound continue its progress towards clinical trials.” “Nomination of ASHA-624 as a development candidate for clinical translation in ALS, alongside Asha’s previously nominated ASHA-091 compound with indications in Alzheimer’s disease, Parkinson’s disease, and ALS, is a key juncture for Asha in supporting our mission of providing novel disease modifying therapies for conditions with current treatment options limited largely to symptomatic standard of care. We are committed through our work to the transformation of patient outcomes, and believe ASHA-624 is a significant step forward towards achieving that goal,” commented Dr. Heckmann, PhD. About ASHA-624: ASHA-624 is a first-in-class, novel intra-molecular glue compound that exploits the normal biology of SARM1, a key protein that promotes axonal degeneration and neurodegeneration by selectively “gluing” activated SARM1 into an inactive conformation, leading to robust neuroprotection through the prevention of axon and neuron loss. In ALS, the activation of SARM1 leads to axonal loss, neurodegeneration and ultimately motor dysfunction. ASHA-624 was designed to inhibit SARM1 with hyper selectivity, and in preclinical studies has demonstrated a robust safety pro a functional cure in models of ALS. ASHA-624 therapeutic intervention in preclinical models of ALS reversed motor impairment and dysfunction to similar levels as healthy controls while placebo treated groups exhibited continual and exacerbated decline in motor function. About ALS: Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig’s disease, is a progressive neurodegenerative disorder that affects nerve cells (neurons) in the spinal cord and brain. Loss of motor neurons in ALS patients leads to loss of muscle control, resulting in impaired motor function. ALS ultimately affects the regulation and control of muscles which are required for speaking, eating, and breathing. There is currently no available cure for ALS, and the disease is fatal. Asha anticipates the advancement of ASHA-624 and ASHA-091 into clinical trials by late-2024 for their respective disease indications. About Asha Therapeutics: Asha Therapeutics ( ) is a life sciences company at the forefront of a new era of precision drug design, leveraging the power of its proprietary PRISM™ technology to design de novo compounds to create disease-modifying and curative therapeutics for neurological and infectious diseases with high unmet medical need. Asha's lead therapeutic programs, ASHA-624 and ASHA-091 with indications in Amyotrophic Lateral Sclerosis, Parkinson's disease and Alzheimer’s disease, are anticipated to enter clinical trials by late-2024 and are disease modifying therapeutics. Forward-Looking Statements The matters discussed in this release that are not historical facts are “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934 and we intend that such forward-looking statements be subject to the safe harbor created thereby. Forward-looking statements include, but are not limited to, statements containing the words “believes,” “anticipates,” “intends,” “estimates,” “plans,” “expects,” “projects” and words of similar import. Readers are cautioned not to place undue reliance on these forward-looking statements, which are based on the information available to management at this time and which speak only as of the date of this release. The Company undertakes no obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise. These forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause actual results, performance or achievements of the Company or its industry to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. While the Company believes the information contained herein is reliable, the Company makes no representations or warranties regarding the accuracy or completeness of this information.

- NB-4746 is being developed as a neuroprotective therapy for neurological diseases - Milestone marks Nura Bio’s transition to a clinical-stage company SOUTH SAN FRANCISCO, CA, USA I August 25, 2023 I Nura Bio, Inc. (Nura Bio), a biopharmaceutical company developing neuroprotective, small molecule therapies for the treatment of debilitating neurological diseases, today announced the initiation of the Phase 1 clinical trial of its SARM1 inhibitor, NB-4746. NB-4746 is believed to be a first-in-class, oral, brain-penetrant, neuroprotective SARM1 inhibitor with broad therapeutic potential across diseases of the central, peripheral, and ocular nervous systems. NB-4746 confers significant structural and functional protection in several preclinical disease models including Amyotrophic Lateral Sclerosis (ALS), Multiple Sclerosis (MS), Traumatic Brain Injury (TBI), and Chemotherapy-Induced Peripheral Neuropathy (CIPN). In preclinical models, NB-4746 significantly reduced and in some cases prevented, an increase in levels of the axonal injury-dependent, disease-associated biomarker neurofilament light (NfL). “We are excited about the potential of NB-4746,” said David Lau, PhD, Senior Vice President, Development Sciences, Nura Bio. “We expect to enter clinical trials in a patient population in 2024, to evaluate the impact of NB-4746 on axonal injury and NfL levels.” The Phase 1 clinical trial is a randomized, double-blind, placebo-controlled, single ascending dose and multiple ascending dose study to assess the safety, tolerability, and pharmacokinetics of NB-4746 in healthy volunteers. “The initiation of the Phase 1 trial of NB-4746 marks Nura Bio’s transition to a clinical-stage company,” said Shilpa Sambashivan, PhD, Chief Scientific Officer, Nura Bio. “With our strong scientific foundation and dedicated team, we are rapidly progressing NB-4746 through early development. We also continue advancing additional molecules through our pre-clinical pipeline, with the goal of delivering novel and potentially life-changing neuroprotective medicines.” About NB-4746 NB-4746 is the lead asset in Nura Bio’s small molecule pipeline. NB-4746 targets SARM1, a neuronally enriched NAD hydrolase that has emerged as an important axon-intrinsic metabolic sensor and central driver of axon degeneration. Axon degeneration is an early hallmark of several neurological diseases. Halting axon degeneration early can confer significant structural and functional neuroprotection and has tremendous potential in the treatment of several neurological diseases. About Nura Bio Nura Bio, Inc. (Nura Bio) is a clinical-stage biopharmaceutical company developing neuroprotective therapies for the treatment of a broad range of neurological diseases. Nura Bio’s research and early development small molecule pipeline spans discovery through Phase 1. Nura Bio’s efforts are focused on developing therapies that halt axon degeneration and/or modulate microglial responses to degeneration and injury, with the goal of conferring neuroprotection, across diseases of the central, peripheral, and ocular nervous systems. For more information, visit www.nurabio.com . SOURCE: Nura Bio

LONDON--(BUSINESS WIRE)-- Ellipses Pharma Limited (“Ellipses”), a global drug development company focused on accelerating the development of new oncology treatments, will present a “Trial in Progress” poster detailing the design of a Phase 1/2 trial of vosilasarm (EP0062) in advanced breast cancer at the San Antonio Breast Cancer Symposium (SABCS) in San Antonio, Texas on Tuesday, 6 December from 5:00pm to 6:15pm CST. Vosilasarm is a selective androgen receptor modulator (SARM) under development for the potential treatment of advanced breast cancer. This study is designed to further extend the evaluation of vosilasarm as a potential therapy for AR+/HER2–/ER+ advanced breast cancer, with the primary aim of identifying a recommended phase 2 dose (RP2D). Recruitment has commenced and the study will recruit up to 130 patients globally. Presentation details Title A phase 1/2 study to evaluate the safety and efficacy of EP0062, an oral Selective Androgen Receptor Modulator (SARM), for the treatment of AR+/HER2-/ER+ advanced breast cancer Presenter Professor Elgene Lim, Institute of Medical Research, University of New South Wales, Sydney, Australia; Abstract number OT1-02-02 Date and time Tuesday December 6, 2022; 5:00 PM - 6:15 PM Session name Trial in Progress Session Location Henry B. González Convention, San Antonio, Texas Professor Hendrik-Tobias Arkenau, Global Head of Drug Development and Chief Medical Officer at Ellipses, said: “Developing promising assets at speed is fundamental to the work of Ellipses, and we are excited to be presenting this ongoing study at such an important scientific conference. We look forward to presenting further details on this trial, including results, in the future.” Dr Rajan Jethwa, CEO of Ellipses, said: “The initiation of this trial for vosilasarm is another key milestone towards our goal of accelerating the development of promising cancer drugs. I am excited by the potential across our pipeline to make available much-needed drugs for patients with cancer.” About vosilasarm / EP0062 Vosilasarm is an oral, non-steroidal, SARM currently being developed for the treatment of AR+/HER2-/ER+ advanced breast cancer. The efficacy and safety of vosilasarm has previously been investigated in a small Phase 1 clinical trial of AR+/HER2–/ER+ advanced breast cancer, and was demonstrated to have acceptable tolerability with preliminary evidence of clinical efficacy (LoRusso et al. Clinical Breast Cancer 2022 22;1 67-77). About AR+/HER2–/ER+ advanced breast cancer Despite recent progress, advanced ER+/HER2- breast cancer remains an area of high unmet medical need. It is estimated that 75-90% of advanced ER+ breast cancers are androgen receptor (AR) positive. It has been established that the AR acts as a tumour suppressor in multiple contexts of ER+ breast cancer, including where resistance to current endocrine-based regimens develops (Hickey et al. Nature Medicine 2021 27; 310-320). This provides the rationale for evaluating vosilasarm, an AR agonist, as a potential treatment strategy. About Ellipses Pharma Limited Ellipses Pharma is a global drug development company based in London, focused on accelerating the development of cancer medicines and treatments through an innovative drug development model that combines unbiased vetting to de-risk initial asset selection with an uninterrupted funding flow to minimize the time it takes to advance lead products through clinical trials and reach patients. For more information, please visit ellipses.life View source version on businesswire.com: Contacts Ellipses Pharma Ltd Suzanne Wood media@ellipses.life Source: Ellipses Pharma Limited View this news release online at: