On January 9, 2024 Seres Therapeutics presented its corporate presentation (Presentation, Seres Therapeutics, JAN 9, 2024, View Source [SID1234639169]).

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On January 9, 2024 SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) ("SELLAS’’ or the "Company"), a late-stage clinical biopharmaceutical company focused on the development of novel therapies for a broad range of cancer indications, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to SLS009 (formerly GFH009), its novel and highly selective CDK9 inhibitor, for the treatment of relapsed/refractory (r/r) acute myeloid leukemia (AML) (Press release, Sellas Life Sciences, JAN 9, 2024, View Source [SID1234639168]). The Fast Track Designation is intended to facilitate the development and review of drugs to treat serious conditions and fill an unmet medical need.

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"Receiving Fast Track Designation for SLS009 for r/r AML, following the recent Orphan Drug Designation for the same indication, underscores the potential for SLS009 and highlights the critical unmet need for patients with AML who face a poor prognosis due to the progressive nature of the disease," said Angelos Stergiou, MD, ScD h.c., President and Chief Executive Officer of SELLAS. "The initial positive topline Phase 2a data at the 45 mg (safety) dose level demonstrate that SLS009 in combination with venetoclax and azacitidine (aza/ven) exhibits anti-leukemic effects with a favorable safety profile in AML patients resistant to venetoclax combination therapies. Importantly, as of the last follow-up, eight of the nine patients enrolled in the 45 mg cohort were alive. The first patient enrolled in the study achieved a complete response (CR) and continues on study in the seventh month with full peripheral blood recovery. The second enrolled patient is in the sixth month of treatment, further underscoring the potential benefit of adding CDK9 inhibition to the aza/ven regimen. We have now also enrolled several patients in the ongoing 60 mg dose cohort. Our team is committed to advancing the development of SLS009 with the goal of providing effective solutions to patients in need of viable treatment options."

Dr. Stergiou continued, "SLS009 continues to emerge as a promising treatment for hematologic malignancies, and we are pleased by the FDA’s recognition of its potential by the grant of Fast Track and Orphan Drug Designations for AML. These designations position us to accelerate SLS009 clinical development with the goal of delivering this potentially groundbreaking treatment to AML patients in need."

A total of nine patients have been enrolled at the 45 mg safety dose level. Eight patients (89%) remain alive (one patient succumbed to sepsis having previously contracted COVID 19) and six continue treatment. The first enrolled patient achieved a complete response and continues on the study in the seventh month with full blood recovery and the second enrolled patient is in the sixth month of treatment. The follow-up duration for the patients who are alive ranges from two to seven months and median OS has not been reached. Significant anti-leukemic effects (≥50% decrease in bone marrow blasts) were observed during treatment in seven out of eight (87.5%) assessable patients with no significant safety issues to date. No dose limiting toxicities were observed in any of the patients. In the Phase 1 study of SLS009 as monotherapy, a durable CR with no minimal residual disease (MRD) was observed in one patient with AML who had failed prior aza/ven therapy. The patient continues to be alive 16 months following commencement of treatment per last follow-up. Patients with AML that fail venetoclax-based therapies have limited treatment options and a poor prognosis with a median OS of approximately 2.5 months.

The Phase 2a clinical trial of SLS009 is an open label, single arm, multi-center study that is designed to evaluate safety, tolerability, and efficacy at two dose levels, 45 mg and 60 mg, in combination with aza/ven. In the 60 mg dose cohort, patients are being randomized to one of two groups, 60 mg flat (fixed) dose once per week and 30 mg fixed dose two times per week. Each group will enroll 5 – 10 patients. In addition to safety and tolerability of SLS009 in combination with aza/ven, the primary endpoints are composite complete response rate (CRc) and duration of response (DOR). Additional endpoints include event free survival (EFS), OS, and pharmacokinetic (PK) and pharmacodynamic (PD) assessments. Venetoclax combinations with hypomethylating agents are a commonly used regimen in this target population but despite high efficacy (up to ~67% complete response rate), approximately one-third to one-half of patients do not respond to venetoclax based regimens, and among those who respond almost all eventually relapse.

The Company expects to report additional data from the fully enrolled 45 mg (safety dose level) cohort and initial data from the 60 mg (recommended Phase 2 dose level) cohort in the first quarter of 2024 and expects the 60 mg cohort to be analyzed in the second quarter of 2024.

On January 9, 2024 Sana Biotechnology presented its corporate presenation (Presentation, Sana Biotechnology, JAN 9, 2024, View Source [SID1234639167]).

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On January 9, 2024 Revolution Medicine presented its corporate presentation (Presentation, Revolution Medicines, JAN 9, 2024, View Source [SID1234639166]).

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On January 9, 2024 Precision BioSciences, Inc. (Nasdaq: DTIL), an advanced gene editing company utilizing its novel proprietary ARCUS platform to develop in vivo gene editing therapies for sophisticated gene edits, including gene elimination, insertion, and excision, reported completion of a strategic transaction with TG Therapeutics, Inc. (Nasdaq: TGTX) for an exclusive license to develop Azercabtagene Zapreleucel (azer-cel) for autoimmune diseases, and other indications outside of cancer (Press release, Precision Biosciences, JAN 9, 2024, View Source [SID1234639165]).

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"We are excited to extend the utility of our allogeneic CAR T assets into immunology by collaborating with TG Therapeutics as they advance novel treatments for B-cell diseases. Key factors in our decision to partner with the TG team include their recent development, regulatory and commercial successes in the multiple sclerosis space, which we believe are strong indicators of the commitment and expertise they will bring to the development of azer-cel in autoimmune diseases," said Michael Amoroso, President and Chief Executive Officer at Precision BioSciences. "As TG Therapeutics assumes development of azer-cel for immunology, Precision will remain focused on capitalizing on the utility of ARCUS for gene elimination and gene insertion, beginning with our wholly owned PBGENE-HBV program for chronic hepatitis B and PBGENE-PMM for primary mitochondrial myopathy."

"After an extensive review of the CAR T products available for development in immunology, we are excited to bring azer-cel into our portfolio as we look to expand our offerings for patients suffering from autoimmune diseases," said Michael S. Weiss, Chairman and Chief Executive Officer of TG Therapeutics. "We are pleased to partner with Precision BioSciences for azer-cel, and the equity investment we are making is indicative of our optimism in the near- and long-term opportunities for ARCUS for in vivo gene editing."

In exchange for global rights to azer-cel for autoimmune diseases and indications outside of cancer, Precision will receive upfront and potential near-term economics valued at $17.5 million. The upfront payment of $7.5 million will consist of cash and the purchase of 2,920,816 shares of Precision common stock by TG Therapeutics at a price of $0.77 per share, a 100% premium to the 30-day volume-weighted average price (VWAP) prior to purchase. Precision will also receive $2.5 million within 12 months, as an equity investment in Precision’s common stock at 100% premium to the then 30-day VWAP prior to purchase. Upon the achievement of certain near-term clinical milestones, Precision will receive an additional $7.5 million payment in cash and the purchase of Precision common stock by TG Therapeutics at a 100% premium to the then current 30-day VWAP. Precision is eligible to receive up to $288 million in additional milestone payments based on the achievement of certain clinical, regulatory, and commercial milestones, in addition to high-single-digit to low-double-digit royalties on net sales.

"With this deal and the Imugene oncology collaboration for azer-cel announced in August 2023, Precision has now completed two cell therapy collaborations to realize value from our allogeneic CAR T platform while enabling development of azer-cel for patients in diseases with high unmet need. These transactions are expected to extend our runway and will fund continued development of our wholly owned in vivo gene editing programs. As a result of these two accretive partnerships, Precision has received or is eligible to receive $47 million in upfront and potential near-term payments and has the potential to receive more than $900 million in development, regulatory and commercial milestone payments," added Mr. Amoroso.

Although it has not finalized its full financial results for the year ended December 31, 2023, Precision expects to report that it had approximately $116 million in cash and cash equivalents as of December 31, 2023. Upfront and potential near-term cash from azer-cel transactions, along with existing cash and cash equivalents, expected operational receipts, continued fiscal and operating discipline, availability of Precision’s at-the-market (ATM) facility, and available credit are expected to extend Precision’s cash runway into the first half of 2026 and through clinical phase 1 readouts for its wholly owned HBV and PMM programs.

Precision will continue to evaluate potential partners for other assets from its allogeneic CAR T platform that are no longer being developed internally, including PBCAR19B stealth cell and BCMA targeting CAR T assets for multiple myeloma.