On May 16, 2024 Traws Pharma, Inc. ("Traws" or "Traws Pharma"), a clinical stage biopharmaceutical company developing oral small molecules for respiratory viral diseases and cancer, reported financial results for the first quarter of 2024, and provided a business update (Press release, Traws Pharma, MAY 16, 2024, View Source [SID1234643406]).

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"2024 has already been a transformative year for Traws Pharma to advance our portfolio of novel treatments for serious respiratory infections and oncology programs. We completed the acquisition of Trawsfynydd and concluded a concurrent $14 million private placement financing. In addition, we initiated first-in-human dosing for our COVID 19 product candidate, including completion of the first cohort dosing group. Furthermore, we completed the last dose escalation cohort for our CDK4+ inhibitor, narazaciclib." stated Werner Cautreels, Ph.D., Chief Executive Officer of Traws Pharma. "We believe that we are poised to make even more meaningful progress in the second half of 2024, as we advance our influenza treatment and ritonavir-free protease inhibitor for COVID 19 into expanded Phase 1 dose escalation studies and begin Phase 2 development."

"Based on the preclinical profile and early clinical data from our infectious disease candidates and narazaciclib, I am optimistic about the outlook for Traws’ portfolio and look forward to updating our investors with our progress through the year," concluded Dr. Cautreels.

Traws Proprietary Portfolio Highlights:

TRX100 (tivoxavir marboxil): a cap-dependent endonuclease inhibitor for influenza: Phase 1

· Targets the cap-dependent endonuclease of influenza and is a potent inhibitor of influenza virus replication including A and B strains

· First Phase 1 study demonstrated safety and tolerability in healthy volunteers with pharmacokinetics and pharmacodynamics (PK/PD) data to support the potential use of a single oral dose for treatment or prophylaxis

· We plan to initiate Phase 1 dose extension to evaluate one additional, higher dose prior to the initiation of Phase 2 studies in H2 2024. Topline data from the Phase 2 study are expected in H1 2025

TRX01 (ratutrelvir): a ritonavir-free Mpro protease inhibitor for COVID19: Phase 1

· Potent oral inhibitor of SARS-CoV-2 Mpro (3CL protease), effective against the original, delta, and omicron variants of SARS-CoV-2, that does not require co-administration with ritonavir, reducing the risk of drug-drug interactions. Preclinical data support once-daily dosing for 10days which could overcome viral rebound seen with other agents.

· We are in the process of conducting a Phase 1 first-in-human single ascending dose/multiple ascending dose (SAD/MAD) study in normal volunteers. The second dosing cohort is underway and topline data are expected H2 2024. A Phase 2 study is also planned to begin in H2 2024 in patients with moderate to severe COVID19. Topline data are expected H1 2025

Narazaciclib: CDK 4+ to treat solid tumors: Phase 1/2

· Available preclinical and clinical data suggest that narazaciclib is active in numerous tumor types, inhibiting CDK 2/4/6, CSF1R and ARK 5/NUAK1. Preclinical studies also showed reduced neutropenia, as compared to palbociclib, and inhibition of palbociclib resistant cancer cells.

· A dose escalation study to define the recommended Phase 2 dose (RP2D) recently enrolled the last cohort. A review of the clinical and PK/PD data is underway. We intend to utilize these data to define the clinical strategy, including selection of a lead indication and next steps in its development.

First Quarter 2024 Financial Results

Cash and cash equivalents as of March 31, 2024, were $16.4 million, compared with $20.8 million as of December 31, 2023.

In April 2024, the Company raised gross proceeds of $14 million from the sale of common and preferred stock to TPAV, LLC, an affiliate of Torrey Pines, and OrbiMed Private Investments VIII, LP, an affiliate of OrbiMed Advisors.

The Company believes that its cash and cash equivalents will be sufficient to fund ongoing clinical trials and business operations into the fourth quarter of 2024.

Revenue was fifty-six thousand dollars for the first quarter of 2024, consistent with the same period in 2023.

General and administrative (G&A) expenses were $3.4 million for the first quarter of 2024, compared with $2.1 million for the same period in 2023. The increase in G&A expenses was caused by higher legal and professional fees related to the Trawsfynydd acquisition on April 1, 2024, partially offset by lower bonus accrual as well as lower insurance, meeting, and public company expenses.

Research and development (R&D) expenses were $1.9 million for the first quarter of 2024, compared with $4.1 million for the same period in 2023. The decrease was primarily caused by lower costs related to narazaciclib drug substance and drug product manufacturing, a reduction in clinical development and consulting costs and lower personnel expenses due to lower bonus accrual.

Net loss for the first quarter of 2024 was $5.0 million, or $0.24 per share on 20.8 million weighted average shares outstanding, compared with a net loss of $5.8 million, or $0.28 per share for the same period in 2023, based on 20.8 million weighted average shares outstanding.

On May 16, 2024 Flamingo Therapeutics ("Flamingo") reported that the first patient has been dosed in an Investigator-initiated Trial (IIT) evaluating the safety and efficacy of danvatirsen in patients with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) who have relapsed or are refractory to frontline therapy (Press release, Flamingo Therapeutics, MAY 16, 2024, View Source;utm_medium=rss&utm_campaign=flamingo-therapeutics-announces-first-patient-dosed-in-investigator-initiated-trial-iit-evaluating-danvatirsen-in-phase-1-for-aml-mds-cancers [SID1234643391]). The IIT is being conducted by leading researchers at Montifore Einstein Comprehensive Cancer Center (MECCC; New York, USA) and MD Anderson Cancer Center (MDACC; Houston, USA). In the Phase 1 trial, the STAT3 inhibiting oligonucleotide danvatirsen, discovered by Ionis, will be evaluated as a monotherapy and also in combination with venetoclax, an approved frontline treatment in relapsed/refractory MDS and AML.

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AML and MDS—two related blood diseases that disproportionally impact older adults—are notoriously difficult to treat and associated with high relapse rates. Although new therapies have improved survival, treatment options remain limited, and the prognosis for the 50% of people who experience disease relapse remains poor. STAT3 protein, which danvatirsen is designed to inhibit, has been associated with worse prognosis.

"We are excited to dose the first patient and initiate a potentially ground-breaking study to evaluate a novel therapeutic strategy targeting STAT3 with danvatirsen for patients suffering from AML and MDS. This clinical trial builds upon the work that my team has done in the research lab over the past decade, and is a good example of a bench to bedside partnership between academic medical centers and industry," said Aditi Shastri, M.D., Principal Investigator of the IIT, Member of MECCC’s Stem Cell & Cancer Biology Research Program and Blood Cancer Institute, and Associate Professor of Oncology, Medicine, and Developmental and Molecular Biology at Albert Einstein College of Medicine. "I look forward to working with the Flamingo team to continue generating promising results in the ongoing clinical trial."

The IIT is being funded by a multi-year grant awarded by the US FDA Office of Orphan Products Development (OOPD) to MECCC, with Dr. Aditi Shastri as the principal investigator, and was one of only 10 grants awarded by the FDA OOPD in 2023. MD Anderson Cancer Center (MDACC) is also participating in the trial with Dr.Naval Daver being the principal investigator at the MDACC site.

"Drs. Shastri and Daver are recognized among the world’s leading experts in AML and MDS and we couldn’t be more pleased to collaborate with them, MECCC, and MDACC," said Andrew Denker, MD, PhD, Chief Medical Officer of Flamingo. "We have tremendous momentum right now with danvatirsen, with ongoing clinical programs in head and neck cancer (PEMDA-HN) and in AML/MDS. The IIT announced today is very important to broaden Flamingo’s danvatirsen clinical development program, and advance our therapy towards patients who can benefit from novel treatments."

The IIT is a Phase I study (NCT05986240) investigating danvatirsen as both a monotherapy and in combination with venetoclax in patients with AML or intermediate/high/very high risk MDS who have relapsed or are refractory to frontline therapy. The sequential dose escalation design seeks to assess safety, efficacy, pharmacokinetics and pharmacodynamics, to inform a recommended phase 2 dose and further investigations.

More information on the IIT study can be found here.

On May 16, 2024 Erasca presented its corporate presentation (Presentation, Erasca, MAY 16, 2024, View Source [SID1234643408]).

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On May 15, 2024 Galapagos NV (Euronext & NASDAQ: GLPG) and Blood Centers of America (BCA) reported that they have entered into a strategic collaboration for the decentralized manufacturing of Galapagos’ CAR-T cell therapies through BCA’s network in the U.S (Press release, Galapagos, MAY 16, 2024, View Source [SID1234643392]).

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Under the terms of the collaboration agreement, BCA will provide access to its extensive network of over 50 community blood centers in 43 states for Good Manufacturing Practice compliant manufacturing for Galapagos’ CAR-T hemato-oncology clinical program. This agreement complements existing collaborations between Galapagos and Landmark Bio and Thermo Fisher Scientific.

Galapagos will leverage BCA’s network to initiate technology transfer to multiple sites in parallel for the decentralized manufacturing of its CAR-T product candidates, close to cancer treatment centers, while also accessing apheresis capacity at BCA sites when required. In addition, BCA will play a crucial coordinating role by supporting site initiation and onboarding to accelerate Galapagos’ efforts and ensure consistent quality.

Galapagos’ innovative, decentralized manufacturing platform could address many of the limitations that currently available CAR-T production is facing. It has the potential to offer greater speed and scalability, with the delivery of fresh, fit cells with a vein-to-vein time of seven days and the possibility for greater physician control and improved patient experience.

"Our collaboration with Blood Centers of America marks a major milestone for Galapagos’ U.S. expansion by establishing centers for support of our pivotal studies, with the potential to be used for commercial introduction. Working with BCA’s extensive nationwide network allows us to efficiently scale up decentralized CAR-T therapy manufacturing across the U.S., while their established infrastructure helps us harmonize operations and access apheresis capacity," said Dr. Paul Stoffels1, CEO and Chairman of the Board of Directors of Galapagos. "This agreement complements the existing collaborations between Galapagos and Landmark Bio and Thermo Fisher Scientific and translates to a significant step forward in our aspiration to bring CAR-T therapies to more patients with a vein-to-vein time of just seven days."

"We are very pleased to partner with Galapagos in expanding their nationwide CAR-T manufacturing network," said Delisa English, BCA Board Chair. "Our decades of experience and advanced capabilities in regulatory compliant biologic processing across the U.S. will facilitate seamless technology transfer across multiple sites. We strongly believe that the combination of Galapagos’ decentralized manufacturing platform and our national cell therapy manufacturing footprint will ultimately benefit patients by providing convenient access to our local facilities for apheresis and local healthcare providers within their communities."

On May 16, 2024 Agenus Inc. ("Agenus") (Nasdaq: AGEN), a leader in discovering and developing novel immunological agents to treat various cancers, reported it will conduct a Type B End-of-Phase 2 (EOP2) meeting in July with the U.S. Food and Drug Administration (FDA) to discuss the botensilimab plus balstilimab (BOT/BAL) combination therapy studies in patients with relapsed/refractory metastatic colorectal cancer that is not MSI-high or dMMR (r/r MSS mCRC), as well as the critical elements of the program to support a future biologics license application (BLA) submission (Press release, Agenus, MAY 16, 2024, View Source [SID1234643410]).

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The meeting is scheduled as part of Agenus’ ongoing efforts to expedite the development of this promising therapeutic option in CRC, considered to be one of the most challenging types of cancer due to its high incidence and mortality rates. It is also one of the fastest-growing cancer types in the U.S., particularly noted for its increasing prevalence among younger adults. Agenus aims to collaborate closely with the FDA to outline the path forward, including the Phase 3 study design and other elements needed to support a BLA filing under the FDA’s accelerated approval pathway. The FDA granted BOT/BAL fast track designation in April 2023.

"Our upcoming End of Phase 2 meeting with the FDA represents a significant milestone in the ongoing development of BOT/BAL for patients diagnosed with metastatic MSS CRC who do not have active liver metastases," stated Steven O’Day, M.D., Chief Medical Officer of Agenus. "The results from our Phase 1 and Phase 2 studies contribute valuable insights into the potential of this therapy for managing a specific and challenging subgroup of colorectal cancer. We remain dedicated to further exploring innovative immunotherapeutic strategies."

The Phase 2 data will be submitted to a major medical conference later this year. In addition to advancing BOT/BAL in colorectal cancer, Agenus remains committed to exploring the potential of this combination therapy in other cancer indications and is preparing to present further data at upcoming medical conferences.

About Botensilimab

Botensilimab is a human Fc enhanced CTLA-4 blocking antibody designed to boost both innate and adaptive anti-tumor immune responses. Its novel design leverages mechanisms of action to extend immunotherapy benefits to "cold" tumors which generally respond poorly to standard of care or are refractory to conventional PD-1/CTLA-4 therapies and investigational therapies. Botensilimab augments immune responses across a wide range of tumor types by priming and activating T cells, downregulating intratumoral regulatory T cells, activating myeloid cells and inducing long-term memory responses.

Approximately 900 patients have been treated with botensilimab in phase 1 and phase 2 clinical trials. Botensilimab alone, or in combination with Agenus’ investigational PD-1 antibody, balstilimab, has shown clinical responses across nine metastatic, late-line cancers. For more information about botensilimab trials, visit www.clinicaltrials.gov with the identifiers NCT03860272, NCT05608044, NCT05630183, and NCT05529316.