On January 9, 2025 Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL), a commercial stage biotechnology company focused on hematologic disorders and cancer, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to R289 for the treatment of myelodysplastic syndromes (MDS) (Press release, Rigel, JAN 9, 2025, View Source [SID1234649549]). R2891, Rigel’s potent and selective dual inhibitor of IRAK1 and IRAK4, is being studied in an ongoing Phase 1b study evaluating the safety, tolerability, pharmacokinetics and preliminary activity in patients with LR-MDS who are relapsed or refractory to prior therapies.

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"Receiving Orphan Drug designation for R289 supports the development of this therapeutic candidate for the treatment of MDS and highlights the significant unmet medical need that exists for these patients," said Raul Rodriguez, Rigel’s president and CEO. "Orphan Drug and Fast Track designations, along with encouraging initial data from our ongoing Phase 1b study in patients with lower-risk MDS, represent significant milestones in the advancement of R289 as a potential new treatment option."

The FDA Office of Orphan Products Development grants orphan drug designation to support the development of medicines for rare disorders that affect fewer than 200,000 people in the U.S. Under the Orphan Drug Act, orphan drug designation qualifies a company for incentives including tax credits, exemptions from certain FDA fees for clinical trials and the potential for seven years of market exclusivity following drug approval.

R289 was previously granted Fast Track designation by the FDA for the treatment of patients with previously-treated transfusion dependent lower-risk MDS.

About R289
R289 is a prodrug of R835, an IRAK1/4 dual inhibitor, which has been shown in preclinical studies to block inflammatory cytokine production in response to toll-like receptor (TLR) and interleukin-1 receptor (IL-1R) family signaling. TLRs and IL-1Rs play a critical role in the innate immune response and dysregulation of these pathways can lead to various inflammatory conditions. Chronic stimulation of both these receptor systems is thought to cause the pro-inflammatory environment in the bone marrow responsible for persistent cytopenias in lower-risk MDS patients.

On January 9, 2025 Synaffix B.V. ("Synaffix"), a Lonza company (SWX:LONN) focused on commercializing its clinical-stage platform technology for the development of antibody-drug conjugates (ADCs) with best-in-class therapeutic index, reported that it has licensed its ADC technology to Mitsubishi Tanabe Pharma Corporation ("MTPC"), the pharma arm of Mitsubishi Chemical Group ("MCG") (Press release, Synaffix, JAN 9, 2025, View Source [SID1234649550]).

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Synaffix is responsible for the manufacturing of the components that are related to its proprietary technologies, and MTPC is responsible for the research, development, manufacturing and commercialization of the ADC.

Peter van de Sande, Head of Synaffix, said: "This latest licensing deal with MTPC highlights the appeal of our industry-leading ADC technology offering. MTPC is a key collaborator for us, expanding our presence in the APAC region, and we are excited to work alongside such a large and reputable corporation with the aim to make a tangible difference for patients in areas of high unmet medical need."

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 9, 2025 Boehringer Ingelheim and Synaffix B.V. ("Synaffix"), a Lonza company (SWX:LONN) focused on commercializing its clinical-stage platform technology for the development of best-in-class antibody drug conjugates (ADCs), reported that Boehringer has licensed Synaffix’s ADC technology (Press release, Synaffix, JAN 9, 2025, View Source [SID1234649552]). This new partnership significantly bolsters Boehringer’s ADC portfolio, driven by its subsidiary, NBE Therapeutics, to achieve the company’s aim of transforming the lives of people with cancer.

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ADCs are a cutting-edge class of cancer treatments that combine the targeting ability of antibodies with the potent cancer-killing power of drugs. This allows for the precise delivery of therapeutic agents directly to cancer cells, minimizing damage to healthy tissue and enhancing treatment efficacy.

Peter van de Sande, Head of Synaffix, said: "Boehringer Ingelheim is a leader in oncology treatment development, and the selection of our ADC technologies to further bolster their pipeline portfolio is a strong recognition of the potential of these technologies to maximize the therapeutic index of ADCs. This licensing agreement represents the culmination of a successful preclinical evaluation of our technology."

Lamine Mbow, Global Head of Discovery Research, Boehringer Ingelheim, said: "We are building a broad pipeline of ADCs addressing the novel tumor target space to develop next-generation cancer treatments. By combining our deep expertise in cancer treatment development with Synaffix’s clinical-stage platform technology, we aim to accelerate the delivery of first-in-class cancer treatments to improve cancer patient outcomes."

Synaffix has developed a clinically validated ADC platform technology that utilizes an enzymatic modification of native glycan anchor points on antibodies, enabling the development of best-in-class ADCs or bispecifics. With the access to Synaffix’s technology, NBE Therapeutics broadens its ADC portfolio. This will enable Boehringer Ingelheim to address novel tumor targets from its comprehensive portfolio to develop first-in-class cancer treatments that address high unmet medical needs in oncology.

Under the terms of the agreement, Synaffix will provide access to its proprietary ADC technologies for an agreed but undisclosed number of targets. The first target was nominated upon signature, and additional targets will subsequently be nominated within a predefined timeframe. In addition to the upfront payment, Synaffix is eligible to receive potential additional milestone payments of up to $1.3 billion, plus additional royalty payments on net sales of resulting products.

On January 9, 2024 Alessa Therapeutics, Inc., a privately held drug development company developing an innovative and proprietary localized drug delivery technology for the treatment of prostate disease, reported the enrollment of the first patient in the company’s Enolen clinical study at the National Cancer Institute (NCI), part of the National Institutes of Health (Press release, Alessa Therapeutics, JAN 9, 2025, View Source [SID1234649535]).

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The Phase I Study (View Source) is a first-in-man study evaluating the safety, tolerability, and preliminary efficacy of Enolen for localized sustained delivery of enzalutamide into the prostate. The Enolen study will treat up to 20 patients in the US. Enrollment for the NCI study is through the NIH Clinical Center in Bethesda, Maryland.

"We are excited to complete the first patient enrollment with our partners at the NCI. As a medical oncologist and Phase I drug developer, it has been a long-held dream to be able to administer an effective anti-cancer agent without its side effects. Our prostate selective therapy spares men from the disabling side effects of unnecessarily blocking testosterone in the rest of the body and the brain." said Dr. Pamela Munster, Professor of Medicine at UCSF and founder and CSO of Alessa Therapeutics.

Prostate cancer is the second most prevalent cancer among men in the United States. According to the American Cancer Society, there were about 299,010 men in the U.S. in 2024, and 3.3 million men live with prostate cancer. (View Source)

While some men with low-risk tumors choose to monitor their disease, most prostate cancer patients are treated by completely removing or radiating their prostate. Both surgery and radiation treatment have expected complications, including urinary incontinence and erectile dysfunction. Systemic anti-androgen and testosterone-lowering drugs are approved for high-risk localized or metastatic disease but associated with side effects, including muscle mass loss, cognitive issues, sexual dysfunction, metabolic syndrome and cardiovascular events. Alessa’s Enolen implant is designed to deliver an anti-androgen drug directly to the target tissue in the prostate, eliminating significant side effects and improving the quality of life for men living with prostate cancer while avoiding surgery or radiation therapy.

Dr. Peter Pinto, Head of the Prostate Cancer Section at the National Cancer Institute, and Principal Investigator on the study noted, "There is a clear unmet therapeutic need for effective treatment options for men with localized prostate cancer that avoids definitive surgery or radiation or androgen ablating systemic therapy."

To learn more about this study, please visit View Source