On January 7, 2025 Alloy Therapeutics Inc. ("Alloy"), a biotechnology ecosystem company dedicated to democratizing access to cutting edge drug discovery technologies, reported a target specific collaboration and license agreement for the use of their novel and proprietary AntiClastic Antisense Platform with Sanofi for a central nervous system (CNS) target (Press release, Alloy Therapeutics, JAN 7, 2025, View Source [SID1234649456]). In return, Sanofi will provide Alloy with upfront license fees and near-term preclinical milestone payments up to $27.5 million. Alloy will also be eligible to receive discovery, development, and commercial milestone payments of over $400 million, as well as tiered royalties on sales of any products resulting from the collaboration. This collaboration underscores a shared commitment to advancing innovative therapeutics in the CNS space.

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Alloy’s AntiClastic Antisense platform allows drug developers to realize the true potential of antisense therapeutics by reaching intracellular disease targets at the RNA level. The platform addresses limitations of current antisense chemistries relating to therapeutic index. This technology platform was launched by Alloy in 2023 after exclusively licensing its underlying intellectual property which implements a novel spatial conformation of the oligonucleotide developed by Sudhir Agrawal of Arnay Sciences.

"At Alloy Therapeutics, we’re transforming RNA therapeutics by bridging foundational insights with modern innovation," said Vinod Vathipadiekal, Chief Scientific Officer, Genetic Medicines at Alloy Therapeutics. "Our work on the AntiClastic Antisense platform and the data generated is pushing the technology beyond current standards and driving innovation that has the potential to redefine what’s possible in RNA therapeutics. With the capabilities we have built and validated, we are excited to work with Sanofi, and we look forward to continuing to deliver on Alloy’s commitment to open collaboration and accessible technologies to ensure these breakthroughs can drive the development of superior RNA-based therapies for patients."

Through the collaboration, Sanofi will leverage their neuroscience expertise and collaborate with Alloy to use the AntiClastic Antisense platform for delivery of therapeutics to the brain, aiming to develop a novel class of genetic medicine capable of crossing the blood-brain barrier.

"We’re excited to partner with Sanofi, a global leader in healthcare innovation, on this landmark licensing agreement for our AntiClastic Antisense platform," said Errik Anderson, Alloy Therapeutics CEO and Founder. "When we began working with Dr. Agrawal, a renowned leader in antisense therapeutics, to integrate his groundbreaking work into Alloy’s genetic medicines platform, we were confident in its potential to revolutionize antisense drug development and reshape the broader drug discovery landscape. This collaboration exemplifies Alloy’s adaptable, multi-modality approach, providing our partners with a comprehensive suite of discovery solutions and access to novel platforms to accelerate the development of the most effective therapies for patients in need."

Join the Alloy Therapeutics community by visiting alloytx.com and following Alloy on LinkedIn.

About the Alloy Therapeutics Genetic Medicines AntiClastic Antisense Platform
AntiClastic Antisense Platform, an offering under Alloy’s Genetic Medicines business unit, is a novel therapeutic format—exclusively available through Alloy Therapeutics collaborations—that is designed to overcome potency and therapeutic index challenges that have historically limited the promise of antisense drugs. Sudhir Agrawal invented the core technology, which combines improvements in the primary sequence with a novel spatial conformation of nucleic acid drugs to promote the delivery of antisense to target RNA, mitigate the inflammatory response, and improve a drug’s therapeutic index. The resultant drug candidates have shown a significant increase in potency compared to gapmer antisense formats. Partners can apply this format to existing antisense sequences or partner to discover new AntiClastic Antisense molecules against their intended target. Learn more at www.alloytx.com/genetic-medicines.

On January 7, 2025 GSK plc (LSE/NYSE: GSK) reported that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for GSK5764227 (GSK’227), its B7-H3-targeted antibody-drug conjugate (ADC) being evaluated for the treatment of adult patients with relapsed or refractory osteosarcoma (bone cancer) who have progressed on at least two prior lines of therapy (Press release, GlaxoSmithKline, JAN 7, 2025, View Source [SID1234649474]). The Breakthrough Therapy Designation aims to expedite the development and review of drugs with the potential to treat a serious condition and where preliminary clinical evidence may indicate substantial improvement over currently available therapy.1 This is the third regulatory designation for GSK’227, following the European Medicines Agency’s decision to grant Priority Medicines (PRIME) designation and the FDA’s decision to grant Breakthrough Therapy Designation for relapsed or refractory extensive-stage small-cell lung cancer in August 2024 and December 2024, respectively.2,3

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Hesham Abdullah, Senior Vice President, Global Head Oncology, R&D, GSK, said: "This latest regulatory designation for GSK’227 exemplifies the potential of our targeted ADC in patients with difficult to treat cancers. For patients with relapsed or refractory osteosarcoma, there is an urgent unmet medical need with no approved treatment options once the cancer returns a second time, and chemotherapy provides limited benefit in this setting."

The US FDA’s Breakthrough Therapy Designation is supported by data from the ARTEMIS-002 study. This is a phase II, open-label, randomised, multi-centre, clinical trial evaluating the efficacy and safety of GSK’227 in patients with relapsed or refractory osteosarcoma and other unresectable bone and soft tissue sarcomas, conducted by Hansoh Pharma. More than 60 patients were enrolled, including 42 patients with osteosarcoma. Results from ARTEMIS-002 were presented at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting.4 Last year, GSK acquired exclusive worldwide rights (excluding China’s mainland, Hong Kong, Macau, and Taiwan) from Hansoh Pharma to progress clinical development and commercialisation of GSK’227. GSK recently began a global phase I trial (NCT06551142) as a part of the development plan to support a registrational pathway for GSK’227.

Osteosarcoma mainly affects children and young adults and is the most common primary bone cancer, accounting for 20-40% of all bone cancers.5 It is a rare disease with an annual incidence of 3.3 patients per million in the US, representing less than 1% of all new cancer diagnoses.6,7 Approximately 20-30% of patients who present with localised (non-metastatic) osteosarcoma and 80% of those who present with metastatic osteosarcoma experience relapsed or refractory disease.8 Following first-line chemotherapy, treatment options for patients with relapsed or refractory osteosarcoma are severely limited, with no clear standard of care available.9 After patients progress on two prior lines of treatment, options become even more limited, with no approved therapies.

About GSK’227
GSK’227, also known as HS-20093, is a novel investigational B7-H3-targeted antibody-drug conjugate composed of a fully human anti-B7-H3 monoclonal antibody covalently linked to a topoisomerase inhibitor payload. HS-20093 is being developed by Hansoh Pharma for the treatment of lung cancer, sarcoma, head and neck cancers and other solid tumours in multiple phase I, II and III clinical trials in China. GSK’s global phase I trial for GSK’227 began in August 2024.

On January 7, 2025 BrainChild Bio, Inc., a clinical-stage biotechnology company developing CAR T-cell therapies to treat tumors in the central nervous system (CNS), reported the clinical development plan to advance BCB-276, its lead autologous CAR T-cell therapy targeting the immune checkpoint B7-H3, for diffuse intrinsic pontine glioma (DIPG), a type of incurable pediatric brain tumor (Press release, BrainChild Bio, JAN 7, 2025, View Source [SID1234649492]).

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The company plans to advance BCB-276 in a single pivotal registration trial designed to accelerate the path to submit a Biologics License Application for the treatment of children and young adults with DIPG. This clinical plan is based on alignment with the U.S. Food and Drug Administration (FDA) at a Type B meeting to proceed directly to a multi-center Phase 2 pivotal clinical trial. This potentially accelerated clinical path for BCB-276 is supported by the promising preliminary safety and efficacy data for SCRI-CARB7H3(s)1 – the research cell product that derived BCB-276 – from a Phase 1 clinical trial conducted by Seattle Children’s, published today in Nature Medicine in this paper.

"We are very pleased to have a solid path forward for our clinical development of BCB-276 in DIPG, enabling us to continue our progress for children and families struggling with this devastating brain cancer that currently has no approved treatments," stated Michael Jensen, MD, Founder and Chief Scientific Officer of BrainChild Bio. "We look forward to continuing to work with the FDA and to generate the additional data required to support a successful IND submission leading to the initiation of the BCB-276 pivotal trial by the end of 2025."

Initial Phase 1 data in DIPG patients supporting BCB-276’s development

Seattle Children’s initiated BrainChild-03, a single-center, dose-escalation Phase 1 clinical trial of repetitive intracerebroventricular (ICV) dosing of its B7-H3 targeted CAR T therapy, SCRI-CARB7h3(s), in children with recurrent/refractory CNS tumors and DIPG. The clinical data from a subset of 21 DIPG patients from the Phase 1 study included twelve (12) patients who began their CAR T treatment after disease progression and nine (9) patients who began their CAR T treatment before disease progression.

Preliminary safety analyses demonstrated that repetitive ICV dosing of the B7-H3 targeted CAR T therapy up to 10×107 cells was well tolerated as an outpatient regimen and without lymphodepleting chemotherapy. Preliminary efficacy analyses demonstrate a median time from diagnosis to death for all 21 patients treated was 19.8 months. Three (3) patients, all beginning CAR T treatment prior to disease progression, remained alive at 44.6 months, 45.6 months, and 52.5 months from diagnosis. These data, while preliminary, suggest a meaningful improvement in overall survival for DIPG patients (for both the pre-‑progression and post-progession patient cohorts) as compared to current standard-of-care, which is limited to palliative focal radiation therapy and has a median time of survival from diagnosis of 8-11 months.

"This is a time of strong momentum for the CAR T discoveries and clinical trials in pediatric brain cancer that were spawned at Seattle Children’s and are now highlighted in Nature Medicine and serving as a driving force for the clinical progress with a CAR T therapy for DIPG," said Dr. Jeff Sperring, Chief Executive Officer of Seattle Children’s. "Our innovation model is built to accelerate technology to bring potential cures to kids faster, and it is gratifying to see that Seattle Children’s launch of BrainChild Bio is supporting the advancement of a CAR T therapy to reach children with an uncurable brain cancer."

About Diffuse Intrinsic Pontine Glioma (DIPG) and Application of CAR T-cell Therapies

Diffuse intrinsic pontine glioma (DIPG) is a primary high-grade brain tumor that arises in the pons and is uniformly fatal. DIPG affects 200-300 children per year in the U.S. with the majority of diagnoses made in children between 5 and 10 years of age. Current standard-of-care treatment remains limited to palliative focal radiation therapy which results in a median overall survival of only 8-11 months from diagnosis.2 Barriers to effective therapies for DIPG include the precarious location of the tumor in the brainstem, the infiltrative growth of the tumor throughout normal brainstem functional anatomy, and the blood brain barrier that remains relatively intact during tumor progression.

The barriers to effective therapies for DIPG can be effectively overcome by the locoregional delivery of appropriately targeted CAR T-cells directly into the cerebrospinal fluid via intracerebroventricular (ICV) dosing with an indwelling reservoir-catheter device. This enables the potential for extensive exposure of the pons to cerebrospinal fluid flow from the ventricular system, thus permitting infused CAR T-cells to directly access the tumor bed. This also allows for repetitive infusions of CAR T-cells to replenish the tumor bed, offering the potential for more durable and sustained efficacy. Additionally, with the blood brain barrier intact, this therapeutic approach can also minimize any on-target, off-tumor toxicities resulting from systemic exposure of CAR T cells.

On January 7, 2025 AnaptysBio, Inc. (Nasdaq: ANAB), a clinical-stage biotechnology company focused on delivering innovative immunology therapeutics, reported that Daniel Faga, president and chief executive officer of Anaptys, will present at the 43rd Annual J.P. Morgan Healthcare Conference on Tuesday, Jan. 14, 2025 at 4:30pm PT / 7:30pm ET (Press release, AnaptysBio, JAN 7, 2025, View Source [SID1234649458]).

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A live webcast of the presentation will be available on the investor section of the Anaptys website at View Source A replay of the webcast will be available for at least 30 days following the event.

On January 7, 2025 Biohaven Ltd. (NYSE: BHVN) reported that Vlad Coric, M.D., Chairman and Chief Executive Officer, will present at the 43rd Annual J.P. Morgan Healthcare Conference at The Westin St. Francis Hotel in San Francisco, California, on Monday, January 13, 2025, at 8:15 am (PT) (Press release, Biohaven Pharmaceutical, JAN 7, 2025, View Source [SID1234649476]).

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