On March 17, 2025 Egle Therapeutics, a clinical-stage biotechnology company developing therapies targeting regulatory T cells (Tregs) for immuno-oncology and autoimmune diseases, reported that it has secured €9.3 million in state funding under the "Innovations in Biotherapies and Bioproduction" call for projects from the France 2030 plan, managed on behalf of the French government by Bpifrance (Press release, Egle Therapeutics, MAR 17, 2025, View Source [SID1234651202]).

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Founded in 2020 as a spin-off from Institut Curie, Egle Therapeutics is a clinical-stage biotechnology company specializing in immunomodulation, with the ambition to develop novel approaches to the modulation of Treg activity for the treatment of cancer and autoimmune diseases.

Egle Therapeutics has developed a proprietary translational platform to identify novel tumor-infiltrating Treg targets, with the aim of developing antibody-based candidate drugs to disable Treg function and restore an effective antitumor immune response.

In June 2020, Egle Therapeutics entered a strategic partnership with Takeda Pharmaceuticals, establishing a three-year research collaboration with an option agreement. Since its inception, the company has successfully raised nearly €56 million through equity financing and non-dilutive public funding, including the i-Lab Innovation Competition and DeepTech Development Grant.

During its Series A financing round in October 2021, the company raised €47 million, attracting renowned investors, including EQT Life Sciences, Bpifrance (INNOBIO 2 and Innovation 1), Fund Plus, Bioqube Factory Fund, T1D Fund, and Takeda Ventures.

In 2024, Egle Therapeutics achieved key milestones, including the launch of its Phase I/II clinical trial for EGL-001, the appointment of Michel Detheux as Chairman, and the appointment of Christophe Quéva as Chief Executive Officer.

"We are honored to receive this funding from the French government through France 2030. This recognition and immense support will help Egle Therapeutics advance its research and development programs in regulatory T cell modulation. The funding granted under the ‘Innovations in Biotherapies and Bioproduction’ call for projects will enable us to accelerate the development of EGL-001, our lead immuno-oncology therapeutic candidate. EGL-001 is currently being evaluated in a Phase I/II clinical trial in France and Spain."

On March 17, 2025 Nurix Therapeutics, Inc. (Nasdaq: NRIX), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of targeted protein degradation medicines, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to bexobrutideg (NX-5948) for the treatment of Waldenström macroglobulinemia (WM) (Press release, Nurix Therapeutics, MAR 17, 2025, View Source [SID1234651184]). Bexobrutideg is an orally bioavailable, brain penetrant degrader of BTK which is being evaluated in an ongoing Phase 1a/b clinical trial in adults with relapsed or refractory B-cell malignancies.

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The FDA’s Orphan Drug Designation program provides orphan status to therapies intended for the treatment, diagnosis, or prevention of rare diseases that affect fewer than 200,000 people in the United States. This designation provides certain benefits, including tax credits for qualified clinical testing, waiver or partial payment of FDA application fees and seven years of market exclusivity, if approved.

"The FDA’s Orphan Drug Designation for bexobrutideg, also known as NX-5948, represents an important milestone in our regulatory strategy and underscores the significant unmet medical need for improved treatments for Waldenström macroglobulinemia," said Arthur T. Sands, M.D., Ph.D., president and chief executive officer of Nurix. "Granting of the designation highlights bexobrutideg’s potential to provide patients with WM a promising new therapeutic option. We are also pleased to announce that our investigational therapy bexobrutideg has been assigned a nonproprietary name reflecting its novel mechanism of action, designated with the unique suffix "deg" for degrader."

In collaboration with the national naming authority, United States Adopted Name (USAN) Council, Nurix’s lead BTK degrader, NX-5948, was assigned the nonproprietary name "bexobrutideg." The U.S. and international drug naming convention is designed to select a single name of worldwide acceptability for each active substance that is intended to be marketed as a pharmaceutical. Most notable with bexobrutideg is the designation of a new suffix, "deg," which references bexobrutideg’s novel degradation mode of action. Targeted protein degraders are characterized by their bifunctional nature, binding to both a target protein and a ligase to drive ubiquitination and catalytic degradation of the target through the proteasome. The new deg suffix is an important recognition that the mechanism of action, pharmacokinetics and pharmacodynamics of targeted protein degraders are fundamentally different than inhibitors, which all use the "ib" suffix. The central stem of the name, "bruti," references the target, Bruton’s tyrosine kinase (as used in ibrutinib, zanubrutinib and acalabrutinib). The prefix "bexo" is the unique identifier of a specific agent in the class and is often used for ease of reference to the agent.

"We are excited that bexobrutideg has been recognized by the USAN Council as a unique entity and member of a new class of small molecule drugs, targeted protein degraders," said Gwenn Hansen, Ph.D., chief scientific officer of Nurix. "The catalytic mechanism of action and event driven pharmacology triggering ubiquitination and proteasomal degradation of a target protein is highly differentiated from inhibitors and allows degraders to eliminate the totality of a protein’s function. In our BTK degrader clinical program, we have also established that degraders can eliminate mutant oncoproteins that have proven to be resistant to inhibitor therapy."

About Bexobrutideg (NX-5948)
Bexobrutideg is an investigational, orally bioavailable, brain penetrant, small molecule degrader of BTK that is currently being evaluated in a Phase 1 clinical trial in patients with relapsed or refractory B cell malignancies. Nurix has previously reported encouraging safety and efficacy data in patients with WM treated in the ongoing Phase 1a/b clinical trial of bexobrutideg demonstrating early promise of clinical benefit with potential for durable outcomes. Nurix continues to enroll patients with WM in an ongoing Phase 1b expansion cohort and anticipates sharing additional clinical data in 2025. Additional information on the ongoing clinical trial can be accessed at clinicaltrials.gov (NCT05131022). Nurix is also developing bexobrutideg for the potential treatment of inflammatory diseases.

About Waldenström Macroglobulinemia (WM)
WM is a rare, slow growing type of non-Hodgkin’s lymphoma that is characterized by the replacement of normal bone marrow cells by malignant lymphocytic cells that produce monoclonal IgM. This replacement leads to anemia, bleeding, and impaired immune function, while the elevated IgM levels may cause neurologic symptoms. The incidence of Waldenström macroglobulinemia ranges from 0.361,2 to 0.573 per 100,000 people in the United States or approximately 1,200 to 1,900 annually. With a median disease duration approaching 10 years, 4 approximately 12,000 to 19,000 patients are living with Waldenstrom’s macroglobulinemia in the United States. Recommended first-line treatments including chemoimmunotherapy and BTK inhibitor (BTKi) therapy. There are no therapies approved to treat WM patients after a BTKi.

On March 17, 2025 CEL-SCI Corporation (NYSE American: CVM) reported it received comments from the U.S. Food and Drug Administration (FDA) on the confirmatory Registration Study’s Statistical Analysis Plan (SAP) submitted in December of 2024 for the study of Multikine* (Leukocyte Interleukin, Injection) as a neoadjuvant in the treatment of newly diagnosed previously untreated locally advanced head and neck cancer (Press release, Cel-Sci, MAR 17, 2025, https://www.businesswire.com/news/home/20250316883464/en/CEL-SCIs-Head-and-Neck-Cancer-Registration-Study-Protocol-Clears-FDA-Reviewin-Talks-with-Potential-Partners-Interested-in-Commercialization-of-Multikine [SID1234651203]). The FDA stated no response to their comments were required from CEL-SCI and that the agency presently has no comments on the confirmatory study protocol, which was submitted for FDA review contemporaneously with the SAP in December 2024.

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CEL-SCI’s 73,000 square foot Multikine manufacturing facility presently has the capacity to produce over 12,000 Multikine treatments per year. Over $200 million has been invested in the facility and the development and validation of its proprietary biologic manufacturing processes.

"Given Multikine’s excellent survival data, strong statistics and the recent focus on PD-L1 as a diagnostic biomarker for predicting the most effective treatment strategy for head and neck cancer, we are pursuing discussions with key parties to help CEL-SCI complete the last study needed in order to pursue marketing approval for Multikine," stated CEL-SCI CEO Geert Kersten.

"These discussions may lead to potential partnerships involving non-dilutive funding for the 212-patient confirmatory Registration Study designed to bring Multikine to market. We are seeing new interest from highly placed individuals and commercial entities that recognize that Multikine, based on its different mechanism of action, is uniquely positioned to treat about 70% of head and neck cancer patients who have low PD-L1 tumor expression, an area where commercially available PD-L1 inhibitors such as nivolumab and pembrolizumab cannot help. These data, combined with our dedicated Multikine manufacturing facility, position Multikine as a very attractive oncology asset," Kersten concluded.

CEL-SCI completed a randomized controlled Phase 3 study conducted in 928 locally advanced, resectable head and neck cancer stage 3 and 4a patients in 23 countries on 3 continents. The Phase 3 study demonstrated strong statistical results and improved survival with Multikine neoadjuvant treatment over control. The 5-year survival rate of the target patient population that will be treated in the confirmatory Registration Study increased to 73% when patients were treated with Multikine before standard of care vs 45% for control patients who received only the standard of care treatments. The survival advantage over control was accentuated in patients who had low to zero expression of PD-L1 on their tumors and had lower disease burden by having N0 (no nodal involvement).

On March 17, 2025 Orca Bio, a late-stage biotechnology company committed to transforming the lives of patients through high-precision cell therapy, reported positive results from the pivotal Phase 3 Precision-T study of Orca-T, its lead investigational allogeneic T-cell immunotherapy, in patients with acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), high-risk myelodysplastic syndrome (MDS) and mixed-phenotype acute leukemia (MPAL) (Press release, Orca Bio, MAR 17, 2025, https://orcabio.com/orca-bio-announces-positive-results-from-the-pivotal-phase-3-study-of-investigational-orca-t-compared-to-allogeneic-stem-cell-transplant-for-the-treatment-of-hematologic-malignancies/?utm_source=rss&utm_medium=rss&utm_campaign=orca-bio-announces-positive-results-from-the-pivotal-phase-3-study-of-investigational-orca-t-compared-to-allogeneic-stem-cell-transplant-for-the-treatment-of-hematologic-malignancies [SID1234651185]). Orca-T is manufactured using highly purified regulatory T-cells, hematopoietic stem cells and conventional T-cells derived from peripheral blood from either related or unrelated matched donors.

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In the randomized Precision-T study, Orca-T met the primary endpoint of a statistically significant improvement in survival free of moderate-to-severe chronic graft versus host disease (cGvHD) with Orca-T. At one year, the rate for patients who received Orca-T was 78% compared to 38% for patients who received a conventional allogeneic hematopoietic stem cell transplant (alloHSCT). Patients in the Orca-T group achieved an estimated overall survival (OS) of 94% compared to 83% in the alloHSCT arm at one year.

"Today, treating patients with serious blood cancers using allogeneic stem cell transplants requires a difficult risk-benefit trade-off as clinicians aim to cure the disease while avoiding potentially deadly treatment-related toxicities, like GvHD," said presenting author Everett Meyer, M.D., Ph.D., hematologist and associate professor of medicine in Blood and Marrow Transplantation and Cellular Therapy at Stanford Health Care. "The Precision-T study showed double the rate of survival free from GvHD with Orca-T versus a conventional transplant, a relapse-free survival rate of 76% and no new safety concerns. These findings are highly encouraging and provide compelling new evidence as we work to solve for the critical factors contributing to the needs of this patient population."

Precision-T Study Results

In the study, all patients (n=187) with a median age of 43.5 years (range 19-65 years) were randomized 1:1 to Orca-T plus single-agent tacrolimus (TAC) or alloHSCT plus TAC methotrexate (TAC/MTX). Patients across both groups received myeloablative conditioning (MAC) and used a related or unrelated matched donor. Patients had a median follow-up time of 11.4 months (range 0.2-24.3 months) across both arms. Key results from the Precision-T study at one year are summarized below:

The primary endpoint of survival free of cGvHD was 78% (95% CI: 65%, 87%) in the Orca-T arm (n=93) and 38% (95% CI: 26%, 51%) in the alloHSCT arm (n=94) (HR 0.26; p<0.00001).
An interim analysis of the secondary endpoint of OS was 94% (95% CI: 86%, 97%) in the Orca-T arm and 83% (95% CI: 73%, 90%) in the alloHSCT arm (HR 0.49; p=0.11823).
An additional secondary endpoint of cumulative incidence of moderate-to-severe cGvHD was 13% (95% CI: 5%, 23%) and 44% (95% CI: 31%, 56%) in the Orca-T and alloHSCT arms, respectively (HR 0.19; p<0.00002).

Exploratory endpoints at one year include the rate of relapse-free survival which was 76% and 74% in the Orca-T and alloHSCT arms, respectively (HR 0.80, p=0.49). The cumulative incidence of non-relapse mortality was 3% in the Orca-T arm and 13% in the alloHSCT arm. Additionally, the cumulative incidence of Grade 3 or 4 acute GvHD was 6% and 17% in the Orca-T and alloHSCT arms, respectively.

No new safety issues were identified with Orca-T. Grade ≥ 4 infections per CTCAE scoring were noted in 6% and 10% of patients in the Orca-T and alloHSCT arms, respectively.

Orca-T was manufactured in Orca Bio’s centralized GMP facility and delivered to patients at 19 treatment centers across the U.S., with all infusions occurring within a vein-to-vein time of 72 hours or less.

"Approximately 46,000 people are diagnosed with AML, ALL and MDS in the U.S. each year, but only a fraction of them receive an allogeneic stem cell transplant within the current paradigm," said Rawan Faramand, M.D., Blood and Marrow Transplant and Cellular Immunotherapy, Moffitt Cancer Center. "Additional treatment options are needed, and the introduction of a cell therapy like Orca-T that leverages a precision-based approach could pave the way for a new standard of care for patients with various hematologic malignancies."

"These exciting results underscore Orca Bio’s vision of transforming the treatment landscape for patients living with serious blood cancers, potentially standardizing curative treatment for diseases like AML, ALL and MDS," said Ivan Dimov, Ph.D., co-founder and chief executive officer at Orca Bio. "We are working closely with the FDA and expect to submit a Biologics License Application this year. These results support the validity of our high-precision platform as we continue to advance our robust pipeline of allogeneic cell therapies for the treatment of hematologic malignancies, autoimmune diseases and beyond."

Orca Bio is grateful to the patients and families, donors and trial site investigators who participated in the Precision-T study.

The complete results will be presented on April 2, 2025, at the 51st Annual Meeting of The EBMT in Florence, Italy.

About Precision-T
Precision-T (NCT05316701) is a randomized, open-label multi-center study that evaluated the safety, efficacy and tolerability of Orca Bio’s lead investigational allogeneic T-cell immunotherapy, Orca-T, compared to conventional allogeneic hematopoietic stem cell transplant (alloHSCT). Orca Bio received guidance from the U.S. Food and Drug Administration on the design of Precision-T, which evaluated Orca-T in patients with acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), high-risk myelodysplastic syndrome (MDS) and mixed-phenotype acute leukemia (MPAL). There are 19 leading treatment centers participating in the trial, which enrolled 187 patients across the U.S.

On March 17, 2025 CEL-SCI Corporation ("CEL-SCI" or the "Company") (NYSE American: CVM), a Phase 3 cancer immunotherapy company, reported the pricing of a best-efforts offering of 16,000,000 shares of its common stock (or pre-funded warrants ("Pre-Funded Warrants") in lieu thereof) (Press release, Cel-Sci, MAR 17, 2025, View Source [SID1234651204]). Total gross proceeds from the offering, before deducting the placement agent’s fees and other offering expenses, are expected to be approximately $2,560,000. The offering is expected to close on March 18, 2025, subject to satisfaction of customary closing conditions.

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The Company intends to use the net proceeds from the offering to fund the continued development of Multikine, general corporate purposes, and working capital.

ThinkEquity is acting as sole placement agent for the offering.

The securities will be offered and sold pursuant to a shelf registration statement on Form S-3 (File No. 333-265995), including a base prospectus, filed with the U.S. Securities and Exchange Commission (the "SEC") on July 1, 2022, and declared effective on July 15, 2022. The offering will be made only by means of a written prospectus. A final prospectus supplement and accompanying prospectus describing the terms of the offering will be filed with the SEC on its website at www.sec.gov. Copies of the prospectus supplement and the accompanying prospectus relating to the offering may also be obtained, when available, from the offices of ThinkEquity, 17 State Street, 41st Floor, New York, New York 10004.

This press release shall not constitute an offer to sell or a solicitation of an offer to buy, nor shall there be any sale of these securities in any state or jurisdiction in which such an offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.