On January 10, 2025 Rein Therapeutics ("Rein") (NASDAQ: RNTX), formerly known as Aileron Therapeutics, Inc. ("Aileron") (NASDAQ: ALRN), a biopharmaceutical company advancing a novel pipeline of first-in-class medicines to address significant unmet medical needs in orphan pulmonary and fibrosis indications, reported that it has changed its name to Rein Therapeutics, Inc (Press release, Aileron Therapeutics, JAN 10, 2025, View Source [SID1234649585]). The new name, logo, website, and branding elements reflect the Company’s mission to develop first-in-class treatments to rein in diseases. The Company’s common stock will begin trading under the Nasdaq ticker symbol "RNTX" effective January 13, 2025. The CUSIP number for the Company’s common stock is not affected by the name change. The Company also highlighted its recent clinical and corporate achievements and provided its key strategic objectives for 2025.

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"Our rebrand to Rein Therapeutics reflects our unwavering commitment to address the critical needs of underserved patients with fibrotic diseases," said Brian Windsor, Ph.D., President and Chief Executive Officer. "This new chapter for the Company underscores our focus on reining in fibrosis and advancing our pipeline of novel candidates. We look forward to the initiation of a Phase 2 clinical trial for our lead candidate, LTI-03, in the first half of this year, aiming to bring hope to those affected by idiopathic pulmonary fibrosis."

Recent Clinical Achievements

LTI-03, a Caveolin-1 related peptide:
In November 2024, the Company announced positive topline data from Cohort 2 of the Phase 1b clinical trial evaluating the safety and tolerability of inhaled high dose LTI-03 (5 mg BID) and a set of exploratory biomarkers in patients with idiopathic pulmonary fibrosis (IPF). Four biomarkers showed statistical significance in the combined Cohort 1 and Cohort 2 dataset, and five demonstrated dose dependence with respect to low dose (2.5 mg BID) Cohort 1, indicative of active pharmacodynamics. High dose LTI-03 continued to exhibit a favorable safety profile.
2024 Corporate Highlights:
In March 2024, Brian Windsor, Ph.D., was appointed Chief Executive Officer of the Company, marking the Company’s sole focus on advancing a pipeline of first-in-class therapies for orphan pulmonary and fibrosis indications.

In May 2024, the Company completed an underwritten registered direct offering of its common stock and accompanying warrants raising net proceeds of approximately $17.7 million.
2025 Strategic Objectives and Anticipated Milestones

LTI-03: A Phase 2 trial of LTI-03 for the treatment of IPF is anticipated to be initiated in the first half of this year.

LTI-01: The Company’s Phase 2b-ready asset is a first-in-class therapy for the treatment of loculated pleural effusions (LPEs). It holds Orphan Drug Designation for the treatment of pleural empyema in the U.S and E.U. and Fast Track Designation in the U.S.

On January 09, 2025 Light Horse Therapeutics Inc., a developer of first-in-class small molecule therapeutics, reported a $62 million Series A financing (Press release, Light Horse Therapeutics, JAN 9, 2025, View Source [SID1234649637]). The financing was led by founding investor Versant Ventures and included Mubadala Capital as well as three strategic investors: Bristol-Myers Squibb Co, Taiho Ventures, and AbbVie. Light Horse is the latest company started by Versant’s Inception Discovery Engine, and its scientific co-founders are Brian Liau, PhD, Ben Cravatt, PhD, and Nathanael Gray, PhD.

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Light Horse has developed a proprietary platform that uncovers novel functional sites in disease-critical targets. The company integrated distinct approaches to build this precision genetic editing-based platform that interrogates proteins in their native environment, providing a faster discovery of clinical candidates.

The company’s technology turns conventional drug discovery on its head by first identifying novel functional domains and then screening for chemistry. This "function-first" approach is the converse of traditional "screening-first" methodologies.

Led by Light Horse’s CSO, Laure Escoubet, PhD, the Light Horse team has translated this methodology into first-in-class preclinical programs against high-value oncology targets and has enabled a discovery collaboration with Novartis that is described in a separate press release issued today. Light Horse internal programs are now progressing through preclinical testing, paving the way towards the development of disruptive therapies.

"We are very impressed with Light Horse’s chemical genomics capabilities to rapidly investigate protein complexes at scale and to uncover unprecedented functional sites in high-value targets," said Rami Hannoush, PhD, venture partner at Versant and a Light Horse board member. "We believe the talented team at Light Horse and its technology will enable a pipeline of first-in-class drug candidates."

"We have a unique capability to interrogate proteins and pathways, identifying functionally critical sites that can drive the development of groundbreaking first-in-class therapies," said Light Horse CEO Markus Renschler, MD. "Our initial focus addresses high-value, historically challenging oncology targets with the opportunity to apply the technology to other therapeutic areas in the future."

Leadership team and scientific founders

Light Horse is led by experienced executives who possess expertise in oncology drug discovery and development. They are complemented by scientific founders who are world leaders in chemoproteomics and chemical biology.

Thomas Daniel, MD, Chairman
Dr. Daniel has more than 25 years of experience leading and building research and development organizations and leading investments in new companies at the forefront of innovative application of science to healthcare. He served as President of Global Research and Early Development at Celgene and previously held senior leadership roles at AmbRx, Amgen and Immunex.
Markus Renschler, MD, CEO
Dr. Renschler is a proven biotechnology executive with more than 25 years’ experience in building successful companies. Prior to Light Horse, he was President and CEO of Cyteir Therapeutics, Inc. leading it from a private discovery stage startup to a publicly traded clinical-stage company. Prior to Cyteir, he held leading roles in clinical R&D, business development and medical affairs at Celgene, Pharmion and Pharmacyclics, where he helped to develop and/or launched some of the most successful cancer drugs approved to date.
Laure Escoubet, PhD, CSO
Dr. Escoubet has almost 20 years of experience in building successful drug discovery teams and driving drug discovery programs into the clinic. Prior experiences include Vice President, Biology, at Zentalis, Head of Epigenetic Drug Discovery at Bristol-Myers Squibb/Celgene and other leading roles in early drug discovery in oncology.
Brian Liau, PhD, scientific co-founder
Dr. Liau is an Associate Professor of Chemistry and Chemical Biology at Harvard University. His research group develops chemical genomic approaches to interrogate the mechanisms of gene regulatory complexes and their promise as therapeutic targets.
Ben Cravatt, PhD, scientific co-founder
Dr. Cravatt is the Gilula Chair of Chemical Biology and Professor in the Department of Chemistry at Scripps Research. His research group develops and applies chemical proteomic technologies for protein and drug discovery on a global scale and has particular interest in studying biochemical pathways in the nervous system and cancer. Dr. Cravatt is a co-founder of multiple successful biotech companies.
Nathanael Gray, PhD, scientific co-founder
Dr. Gray is the Krishnan-Shah Family Professor of Chemical and Systems Biology at Stanford, Co-Director of Cancer Drug Discovery Co-Leader of the Cancer Therapeutics Research Program, Member of Chem-H, and Program Leader for Small Molecule Drug Discovery for the Innovative Medicines Accelerator. His research uses the tools of synthetic chemistry, protein biochemistry, and cancer biology to discover and validate new strategies for the inhibition of cancer targets.
"Light Horse has deep scientific talent, a very experienced management team, and a strong syndicate of strategic and institutional investors to enhance its delivery of exceptional drug candidates addressing unmet medical need" said Dr. Daniel.

On January 09, 2025 Light Horse Therapeutics Inc., a developer of first-in-class small molecule therapeutics, announced a multi-target collaboration with Novartis to identify and develop potentially first-in-class therapeutics using the biotech’s platform (Press release, Light Horse Therapeutics, JAN 9, 2025, View Source [SID1234649563]). Under the terms of the agreement, Light Horse will receive a $25 million upfront payment. The company is also eligible for $1 billion in further research, development and sales milestones, in addition to royalties on licensed therapeutics. Further details are undisclosed.

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"Light Horse’s high-caliber team has built a cutting-edge discovery platform that rapidly enables the exploration of core biological areas of interest, the identification of novel targets and the functionalization of high-value targets," said John Tallarico, Head of Discovery Sciences at Novartis. "This collaboration has the potential to create first-in-class therapeutics capable of driving meaningful benefit for patients."

"The Novartis collaboration presents an extraordinary opportunity to leverage our unbiased genetic screening platform and proprietary chemical libraries to address novel, high-value targets previously considered hard to drug," said Light Horse CEO Markus Renschler, MD. "We are excited to work with the Novartis team to potentially accelerate the development of innovative cancer treatments over the coming years."

On January 9, 2025 A2 Biotherapeutics, Inc. (A2 Bio), a clinical-stage cell therapy company developing first-in-class logic-gated cell therapies to selectively target tumor cells and protect normal cells, reported the closing of its $80 million Series C financing round, supported by a syndicate of investors that includes The Column Group and Samsara BioCapital (Press release, A2 Biotherapeutics, JAN 9, 2025, View Source [SID1234649562]). Proceeds will fund the company’s three clinical development programs and advancement of its pipeline of CAR-T cell therapies based on its proprietary Tmod platform technology.

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"We are excited by the initial clinical data from our lead programs, which we believe validates our proprietary logic-gate technology approach to solid tumor cancers," said Jim Robinson, chief executive officer of A2 Bio. "This funding will enable us to continue ongoing clinical development of our CAR-T cell therapies as well as fund the potential next phase of development."

A2 Bio is currently conducting two seamless phase 1/2 clinical studies: EVEREST-1 studying A2B530 for pancreatic, lung and colorectal cancers by targeting carcinoembryonic antigen (CEA); and EVEREST-2 studying A2B694 for pancreatic, ovarian, lung, colorectal and mesothelioma cancers by targeting mesothelin (MSLN). Patients are enrolled in EVEREST-1 and EVEREST-2 through the BASECAMP-1 prescreening study. BASECAMP-1 utilizes artificial intelligence (AI)-enabled precision diagnostics, as a cost-effective, high-yield approach to identify eligible patients for all A2 Bio clinical studies.1

A2 Biotherapeutics, established in 2018, is a fully integrated discovery, development and manufacturing company based in Agoura Hills, Calif. A2 Bio plans to direct the Series C funds to support the company’s three clinical studies, as well as to advance preclinical programs using its Tmod platform for potential clinical targets representing significant unmet needs in cancer and beyond.

About the Tmod Platform

A2 Bio has pioneered a precision-targeting cellular system – the Tmod platform – that incorporates two receptors, an activator and a blocker, to aim the powerful armaments of immune cells directly at tumors to unequivocally differentiate tumors from normal tissues. The activator recognizes antigens on tumor cells that trigger their destruction, while the blocker recognizes antigens on normal cells that protect them. This novel blocker technology enables precise, personalized and effective T cell targeting. The blocker component equips Tmod cells with the capacity to identify tumors as distinct from normal cells.

On January 9, 2025 Nimbus Therapeutics, LLC ("Nimbus Therapeutics" or "Nimbus"), a biotechnology company that designs and develops breakthrough medicines for patients through its powerful computational drug discovery engine, reported the advancement of its clinical oncology therapeutic pipeline, and the appointment of Peter J. Tummino, Ph.D., to President of Research and Development (Press release, Nimbus Therapeutics, JAN 9, 2025, View Source [SID1234649561]). Dr. Tummino will be responsible for advancing the company’s product portfolio through all phases of discovery and clinical development.

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The company announced the completion of an investigational new drug (IND) application submission for NDI-219216, a novel Werner syndrome helicase (WRN) inhibitor in development for the treatment of microsatellite instability high (MSI-H) tumors. Nimbus plans to initiate the first clinical trial of NDI-219216 in the first half of 2025 under the leadership of Anita Scheuber, M.D., Ph.D., Senior Vice President, Therapeutic Area Head, Oncology.

"We are encouraged by the building momentum in our oncology portfolio including important advances in our non-covalent WRN inhibitor program, with data indicating NDI-219216 has the potential to be a best-in-class agent," said Dr. Tummino.

"NDI-219216 has demonstrated compelling preclinical data, including significant tumor regression and complete responses at low oral doses across tumor types. The preclinical safety studies suggest a promising benefit-risk profile as we prepare to evaluate this compound in patients. We are excited to advance NDI-219216 into a first-in-human clinical trial later this year," said Dr. Scheuber.

The company recently completed its Phase 1/2 clinical study of NDI-101150, a highly selective and potent hematopoietic progenitor kinase 1 (HPK1) inhibitor, for the treatment of advanced solid tumors. Clinical data presented in November 2024 at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 39th Annual Meeting demonstrated monotherapy efficacy with a favorable safety profile that supports potential combination therapy approaches. The company is actively evaluating opportunities to maximize the therapeutic potential of this program.

Nimbus continues to progress drug discovery efforts across metabolic and autoimmune targets as well, including the development of novel therapies that activate AMP-activated protein kinase (AMPK) to treat metabolic disorders as part of an ongoing research collaboration and exclusive worldwide license agreement with Eli Lilly and Company. Nimbus has also expanded its pipeline with the addition of new undisclosed therapeutic targets leveraging its novel computational and structure-based drug design approach.

"Nimbus made significant progress in 2024 in our discovery and development programs across oncology, immunology, and metabolism, bringing us one step closer to our ultimate goal of delivering transformative medicines to patients," said Jeb Keiper, M.S., M.B.A., Chief Executive Officer of Nimbus. "Peter’s appointment to President of R&D reflects his exceptional leadership and deep expertise in drug discovery and development, and his expanded role will be instrumental in advancing our innovative pipeline. We continue to broaden our drug discovery engine to unlock new difficult-to-drug targets with compelling biology through deep computational expertise, a breadth of internal drug discovery expertise, and key partnerships. We look forward to multiple key milestones in the coming year."

Mr. Keiper will provide an overview of the company’s progress, pipeline, and anticipated milestones for 2025 and beyond at the 43rd Annual J.P. Morgan Healthcare Conference on Monday, January 13, 2025 at 7:30 a.m. PT.