On January 9, 2024 Cyclacel pharmaceuticals presented its corporate presenation (Presentation, Cyclacel, JAN 8, 2024, View Source [SID1234639153]).

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On January 8, 2024 Cellectar Biosciences, Inc. (NASDAQ: CLRB), a late-stage clinical biopharmaceutical company focused on the discovery, development and commercialization of drugs for the treatment of cancer, reported data from its CLOVER WaM pivotal study, evaluating iopofosine I 131, a potential first-in-class, targeted radiotherapy candidate for the treatment of relapsed/refractory Waldenstrom’s macroglobulinemia (WM) patients that have received at least two prior lines of therapy, including Bruton tyrosine kinase inhibitors (BTKi) (Press release, Cellectar Biosciences, JAN 8, 2024, View Source [SID1234639149]). CLOVER WaM is the largest study to date in relapsed or refractory WM patients post-BTKi therapy and represents the most refractory population ever tested in clinical studies based upon a review of published literature.

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The CLOVER WaM study met its primary endpoint with a major response rate (MRR) of 61% (95% confidence interval [44.50%, 75.80%, two-sided p value < 0.0001]). The overall response rate (ORR) in evaluable patients was 75.6%, and 100% of patients experienced disease control. Responses were durable, with median duration of response not reached and 76% of patients remaining progression free at a median follow-up of eight months. These outcomes exceed real world data, which demonstrate a 4-12% MRR and a duration of response of approximately six months or less despite continuous treatment in a patient population that is less pretreated and less refractory to multiple classes of drugs. Notably, iopofosine monotherapy achieved an 8% stringent complete remission (sCR) in this highly refractory WM population.

"There is a critical need for new therapies with novel mechanisms of action to treat WM. There are no approved treatments for patients post BTKi therapy, where currently the expected response rate to salvage treatments is approximately 10%, and the expected duration of response in those patients is less than six months," said Sikander Ailawadhi, M.D., professor of medicine at Mayo Clinic, and lead investigator in the CLOVER WaM study. "The results from this pivotal study utilizing just four doses of iopofosine monotherapy in heavily pretreated patients are very compelling, demonstrating deep and durable remissions. The combination of the safety profile and deep durable responses with a high proportion of patients remaining treatment free is impressive."

CLOVER WaM is a single-arm registration study with a target enrollment of 50 patients. The study is fully enrolled and topline safety data is being reported on 45 patients meeting criteria for modified intent to treat (mITT) with a data cut-off date of January 3, 2024. Topline efficacy evaluable population (41) is defined as patients who have received a total administered dose of greater than 60 mCi and had follow up of at least 60 days post last dose. Among mITT patients, median age was 71 years, median IgM level prior to treatment with iopofosine was 2,185, 90% were refractory to either a BTKi (18/36 50%) or anti-CD20 therapy (18/41 40%), with 26.7% multiclass refractory, and 80% of patients were previously treated with a BTKi therapy.

Newton Guerin, International Waldenstrom’s Macroglobulinemia Foundation (IWMF) president and CEO, said, "These inspiring topline data represent important and exciting news for the entirety of the WM community battling this challenging disease. WM patients need new, clinically meaningful treatment modalities and currently, there are limited options for patients who have received prior BTKi therapy. Iopofosine’s product profile is notable because of its novel mechanism of action, fixed four-dose course of treatment completed within 75 days and the promise of an enhanced quality of life for patients, including a prolonged treatment-free interval."

Iopofosine I 131 was well tolerated and its toxicity profile was consistent with the Company’s previously reported safety data. There were no treatment-related adverse events (TRAEs) leading to discontinuation. The rates of Grade 3 or greater TRAEs observed in more than 10% of patients included thrombocytopenia (55%), neutropenia (37%), and anemia (26%). All patients recovered from cytopenias with no reported aplastic sequalae. Importantly, there were no clinically significant bleeding events, and the rate of febrile neutropenia was 2%. There were no treatment related deaths in the study.

"We are most grateful to the patients and their families, participating study sites, their staff and our dedicated employees for the successful completion of this study. Their respective contributions may provide a meaningful new treatment option for patients where there currently are no approved therapies," said James Caruso, president and CEO of Cellectar. "Iopofosine’s high major response rate and achievement of the study’s primary endpoint in highly refractory, Waldenstrom’s macroglobulinemia patients exhibits its potentially practice-changing clinical profile. We believe the currently impressive response rates and the duration of responses will continue to improve as the data matures. We plan to include these outcomes in our NDA submission and will be requesting an accelerated approval based upon our WM Fast Track Designation."

Conference Call & Webcast Details

Cellectar management will host a conference call for investors today, January 8, 2024, beginning at 8:00 am ET / 5:00 am PT. Dial-in: 1-888-886-7786. Webcast Link: Click HERE

On January 8, 2023 Biocytogen Pharmaceuticals (Beijing) Co., Ltd. ("Biocytogen", HKEX: 02315), a global biotechnology company focused on the development of novel antibody therapeutics, reported that it has entered into an exclusive option and license agreement with Radiance Biopharma Inc. ("Radiance"), a biotechnology company that specializes in the development of next-generation antibody-drug conjugates (Press release, Biocytogen, JAN 8, 2024, View Source [SID1234639130]).

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The agreement grants Radiance an option to license from Biocytogen a first-in-class, fully human anti-HER2/TROP2 bispecific antibody drug conjugate (BsADC) for the development, manufacture and commercialization of therapeutic products for all human indications worldwide. HER2 and TROP2 are two tumor-associated antigens (TAAs) that are commonly expressed and co-expressed by various tumor types, including breast, gastric, colorectal, bladder, pancreatic, and non-small cell lung cancers.

Under the terms of the agreement, upon exercise of the option, Biocytogen will be entitled to an option fee, a royalty, development and commercialization milestone payments, and single-digit royalties on net sales. In addition, Biocytogen has the right to collect any sublicensing fee between Radiance and a third party.

Dr. Yuelei Shen, President and CEO of Biocytogen, said , " We are pleased to collaborate with Radiance, a strong team with extensive drug development experience, to develop a leading proprietary, fully human bispecific antibody-drug conjugate. We are optimistic that the combination of our strength in BsADC discovery and the extensive experience of the Radiance team will help accelerate the commercialization of this dual BsADC."

Marc Lippman, MD, Chairman of the Board of Radiance, said , " We are pleased to enter into this exclusive option and license agreement with Biocytogen for a novel human bispecific antibody-drug conjugate targeting HER2 and Trop2. Preclinical data from in vitro and in vivo testing with this BsADC show promising high potency of anti-tumor activities in leading tumor indications. We look forward to working with Biocytogen to bring the product into practice for the benefit of patients."

On January 8, 2024 HighField Biopharmaceuticals, a clinical stage immuno-oncology company using lipid-based therapeutics to treat cancer, reported that it has completed its Phase 1a study of HF1K16 (Press release, HighField Biopharmaceuticals, JAN 8, 2024, View Source [SID1234639137]). Treating patients having multiple tumor types, the data demonstrated that the drug, administered as a single agent, is well-tolerated with only one dose-limiting toxicity (DLT) at the highest dose level. HF1K16 is a drug encapsulated immune modulating liposome containing all-trans retinoic acid.

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"We were especially encouraged by the outcome being correlated to the treatment duration, with the drug being tolerated for extended periods," said Dr. Yuhong Xu, CEO of HighField. "One glioma patient experienced complete remission after 10 months of treatment, remained on the treatment for two years and is cancer free. Another patient, with grade IV duodenal cancer, maintained stable disease for more than 5 months."

A total of 14 patients, suffering from a variety of refractory metastatic solid tumors such as gliomas and stomach, colorectal, liver, lung and ovarian cancers, were treated in China with HF1K16 at escalating doses beginning in 2022.

Overall, the disease control rate for the patients is about 35% with a median overall survival of 8.5 months. The maximum survival period has exceeded 24 months and five patients have survived more than 10 months.

"Given the disease state of the patients in the study, a monotherapy outcome of 35% disease control rate is impressive," said Dr. Xu. "Moreover, we found significant changes in the patients’ myeloid cell and T cell profiling after treatment. Because it shows a new mechanism and excellent safety profile, our next step is to explore HF1K16 in combination with chemotherapy and other immuno-oncology therapies."

HF1K16 is a unique liposome construct of ATRA, a small molecule metabolite of vitamin A. It is administered by infusion, travels through the blood stream and infiltrates the tumor microenvironment. ATRA is released and initiates the maturation of myeloid-derived suppressor cells (MDSCs).

MDSCs are immature myeloid cells which have not differentiated. ATRA promotes the maturation and differentiation of MDSCs into functional cells, such as dendritic cells, which then summon T cells to attack the cancer.

For more information on the Phase 1a open label trial see NCT05388487 at clinicaltrials.gov.

On January 8, 2024 Schrödinger, Inc. (Nasdaq: SDGR), Schrödinger (Nasdaq: SDGR), whose physics-based computational platform is transforming the way therapeutics and materials are discovered, reported an update on its progress across the business and announced its development and operational goals for 2024 (Press release, Schrodinger, JAN 8, 2024, View Source [SID1234639136]).

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Today Schrödinger announced an expanded, three-year, software agreement with Eli Lilly and Company. The three-year agreement builds on the collaboration established in 2022. The agreement provides Lilly with large-scale access to Schrödinger’s full suite of technologies to enable and accelerate all stages of drug discovery, from target enablement and assessment of target druggability to hit discovery and lead optimization activities through development candidate identification. Schrödinger will provide advanced support to ensure full integration and optimization of the platform across Lilly’s research sites.

"We made important progress across the business in 2023, working with Lilly and other companies to enable more discovery programs at scale, expanding the capabilities of our platform, and increasing our focus on our proprietary pipeline,1 which now includes two clinical-stage development candidates," stated Ramy Farid, Ph.D., chief executive officer at Schrödinger. "We see continued opportunities this year to grow our software business, progress a third development candidate into the clinic, and advance our ongoing clinical studies to enable data readouts from our first two patient trials in late 2024 or 2025."

"Our conviction about our own programs is increasing. We continue to be selective about entering new collaborations, and we will allocate more of our drug discovery resources to our proprietary pipeline this year," stated Geoff Porges, MBBS, chief financial officer of Schrödinger. "We expect our drug discovery revenue in 2024 to be similar to or lower than 2022,2 reflecting these changes in our strategic priorities and the strategic decisions by certain partners to return programs in 2023. We believe our increased focus on our proprietary pipeline creates multiple paths for generating meaningful value in the coming years."

2023 Achievements

Today Schrödinger highlighted several 2023 achievements:

Proprietary Pipeline

Presented initial results from the healthy volunteer study of SGR-1505, its MALT1 inhibitor
Advanced the Phase 1 study of SGR-1505 in patients with advanced B-cell malignancies, including expanding the study at additional sites in the U.S. and Europe
Received FDA orphan drug designation for SGR-1505 in mantle cell lymphoma
Received IND clearance for SGR-2921, its CDC7 inhibitor, and initiated a Phase 1 study in patients with AML and MDS
Advanced SGR-3515, its Wee1/Myt1 inhibitor, to development candidate status and initiated IND-enabling activities
Presented preclinical data for SGR-1505 and SGR-2921 at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and for the NLRP3 program at the Inflammasome Therapeutics Summit
Progressed newly disclosed discovery programs, including EGFRC797S, PRMT5-MTA and NLRP3 discovery programs
Platform

Released new and major improvements to existing technologies, including enabling the optimization of certain key ADMET properties, the first full release of technology to predict antibody affinity as a function of pH, software to more accurately predict small molecule pKa values, and a computational approach to identify brain penetrant molecules
Published 21 peer-reviewed articles in life sciences and materials science journals
Business

Appointed Margaret Dugan, M.D., as chief medical officer to lead clinical development and regulatory strategy for Schrödinger’s proprietary pipeline
Strengthened balance sheet with $147.3M in cash distributions from Nimbus’s sale of its TYK2 inhibitor to Takeda
Renewed and expanded battery research agreement with Gates Ventures for second three-year term
Published inaugural corporate sustainability report
2024 Development and Operational Goals

Today Schrödinger outlined the following development and operational goals for 2024:

Development Goals

Advance the Phase 1 study of SGR-1505 in advanced B cell malignancies to enable initial data release in late 2024 or 2025
Advance the Phase 1 study of SGR-2921 in patients with AML/MDS to enable initial data release in late 2024 or 2025
Submit the IND application for SGR-3515 in the first half of 2024 and initiate a Phase 1 study in 2024
Advance the discovery-stage proprietary pipeline to enable an additional IND submission in 2025
Present additional data from proprietary programs in one or more peer-reviewed forums
Progress platform capabilities associated with biologics
Publish research from the Gates battery research project building on existing publications
Operational Goals

Drive continued scale up and adoption of Schrödinger’s software platform across life sciences and materials science industries
Generate positive returns from strategic investments of Schrödinger’s technology, expertise and capital
Schrödinger will report its fourth quarter and full-year financial results and provide 2024 financial guidance on Wednesday, February 28, 2024, after the financial markets close. The company will host a conference call and webcast at 4:30 p.m. ET.