On February 13, 2025 CareDx, Inc. (Nasdaq: CDNA) – The Transplant Company – a leading precision medicine company focused on the discovery, development, and commercialization of clinically differentiated, high-value healthcare solutions for transplant patients and caregivers – reported new AlloHeme data presented at the 2025 Tandem Meetings, Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR, held February 12-15, 2025 in Honolulu, Hawaii (Press release, CareDx, FEB 13, 2025, View Source [SID1234650275]).

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In an oral presentation, a one-year interim analysis of the ACROBAT prospective, multi-center study showed that AlloHeme is an accurate and sensitive test for monitoring relapse after allogeneic stem cell transplantation in Acute Myeloid Leukemia (AML) and Myelodysplastic Syndromes (MDS). By measuring small changes in mixed chimerism, AlloHeme demonstrated excellent performance characteristics with a hazard ratio (HR) of 40.5 (p<0.001) for relapse, and a clinically meaningful median lead time to relapse of 36 days. The analysis included one-year interim follow-up data from a cohort of 229 patients with AML and MDS from 11 stem cell transplant centers across the U.S.

"We are extremely pleased with the results of the ACROBAT study which demonstrates that AlloHeme is highly accurate in predicting risk of relapse in patients who have undergone a hematopoietic cell transplant," said Dr. Monzr M. Al Malki, Associate Professor, and Director of Unrelated Donor Bone Marrow Transplant Program at City of Hope National Medical Center. "This study gets us one step closer to having a highly reliable molecular biomarker that enables us to assess the status of the stem cell engraftment and predict risk of relapse."

CareDx’s AlloHeme is an NGS-based test that has been shown to be more sensitive than the current standard of care methods for monitoring engraftment and relapse post-allogeneic hemopoietic stem cell transplantation for hematologic malignancies.

"The interim results of the ACROBAT study build upon our growth strategy to expand into hematology oncology. With AlloHeme, we can detect relapse after allogeneic stem cell transplantation prior to conventional methods, giving clinicians the significant lead time they need to intervene sooner," said Marica Grskovic, PhD, CareDx Chief Strategy Officer. "We are very pleased with these results demonstrating the high sensitivity of the AlloHeme assay and its selection for an oral presentation at the Tandem Meetings given the significant impact it may have on patient outcomes through earlier detection of malignancy recurrence."

On February 13, 2025 Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular metabolism and pyruvate kinase (PK) activation pioneering therapies for rare diseases, reported business highlights and financial results for the fourth quarter and year ended December 31, 2024 (Press release, Agios Pharmaceuticals, FEB 13, 2025, View Source [SID1234650239]).

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"Agios had a transformative year in 2024, continuing to successfully deliver on all priorities. Our PYRUKYND franchise is poised for multi-billion-dollar potential, driven by the key milestones we achieved last year, including filing for regulatory approval in thalassemia across four markets and completing enrollment in our Phase 3 RISE UP study for sickle cell disease," said Brian Goff, chief executive officer at Agios. "Backed by a strong balance sheet and a highly experienced team, Agios is focused on maximizing the potential PYRUKYND launches in thalassemia and sickle cell disease in 2025 and 2026, respectively, while advancing and diversifying our key pipeline programs and strategically deploying our capital to drive long-term growth. We are well positioned to bring significant value for shareholders, healthcare professionals and patients, as we build towards a breakout year in 2025."

Fourth Quarter 2024 and Recent Highlights

•PYRUKYND Revenues: Generated $10.7 million in net revenue for the fourth quarter of 2024, a 20 percent increase from the third quarter of 2024, primarily driven by year-end stocking and adjustments to certain revenue reserves. A total of 223 unique patients have completed prescription enrollment forms, representing an increase of 6 percent over the third quarter of 2024. A total of 130 patients are on PYRUKYND therapy, inclusive of new prescriptions and continued therapy.
•Thalassemia:
◦Presented positive results from the ENERGIZE-T Phase 3 randomized clinical trial evaluating mitapivat versus placebo in adults with transfusion-dependent alpha- or beta-thalassemia at the 66ᵗʰ American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition (ASH 2024) in December 2024.
◦Based on the favorable benefit-risk profile observed in both the ENERGIZE and ENERGIZE-T Phase 3 studies, filed regulatory applications for mitapivat (PYRUKYND) for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia with the U.S., European Union, Kingdom of Saudi Arabia and United Arab Emirates health authorities.
◦The U.S. Food and Drug Administration (FDA) accepted the company’s supplemental New Drug Application (sNDA) for PYRUKYND for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. The review classification for this application is Standard and the Prescription Drug User Fee Act (PDUFA) goal date is September 7, 2025.
•Sickle Cell Disease:
◦Completed enrollment of the Phase 3 RISE UP study evaluating mitapivat for the treatment of sickle cell disease patients who are 16 years of age or older. This Phase 3 study enrolled more than 200 patients worldwide.
◦European Commission adopted a positive decision for the designation of mitapivat as an orphan medicinal product for the treatment of sickle cell disease.
◦Presented Phase 1 clinical results on tebapivat (AG-946) in patients with sickle cell disease at ASH (Free ASH Whitepaper) 2024.
•Pediatric Pyruvate Kinase (PK) Deficiency:
◦Reported in a separate press release today, positive topline results from the ACTIVATE-Kids Phase 3 study of mitapivat in children aged 1 to <18 years with PK deficiency who are not regularly transfused.
•Lower-risk myelodysplastic syndromes (LR-MDS):
◦Initiated patient enrollment in the Phase 2b study of tebapivat in LR-MDS.
◦FDA granted orphan drug designation to tebapivat for the treatment of MDS.
•Medical Congresses: Presented 16 abstracts highlighting new data on mitapivat and tebapivat at ASH (Free ASH Whitepaper) 2024.
•Corporate: David Schenkein, M.D., has informed the company that he will step down from Agios’ Board of Directors, effective February 28, 2025, to devote more time to his other commitments. He will continue to serve as a strategic advisor to Agios’ Leadership Team, concentrating on advancing the company’s clinical development programs.

Key Upcoming Milestones & Priorities

Agios expects to achieve the following key milestones in 2025:
•Thalassemia: Receive FDA regulatory decision for PYRUKYND for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia (PDUFA goal date is September 7, 2025).
•Sickle Cell Disease: Announce topline results from the Phase 3 RISE UP study of mitapivat in sickle cell disease in late 2025, with a potential U.S. commercial launch in 2026. Additionally, begin patient enrollment for the Phase 2 study of tebapivat in sickle cell disease in mid-2025.
•LR-MDS: Complete patient enrollment in the Phase 2b study of tebapivat for LR-MDS in late 2025.
•Early-Stage Pipeline: File an Investigational New Drug Application for AG-236, a siRNA targeting TMPRSS6 intended for the treatment of polycythemia vera, in mid-2025.

Fourth Quarter 2024 Financial Results

Revenue: Net product revenue from sales of PYRUKYND for the fourth quarter of 2024 was $10.7 million, compared to $7.1 million for the fourth quarter of 2023, and $36.5 million for the year ended December 31, 2024, compared to $26.8 million for the year ended December 31, 2023.

Cost of Sales: Cost of sales for the fourth quarter of 2024 was $1.3 million and $4.2 million for the full year ended December 31, 2024.

Research and Development (R&D) Expenses: R&D expenses were $82.8 million for the fourth quarter of 2024, compared to $77.5 million for the fourth quarter of 2023, and $301.3 million for the full year ended December 31, 2024, compared to $295.5 million for the full year ended December 31, 2023.

Selling, General and Administrative (SG&A) Expenses: SG&A expenses were $51.7 million for the fourth quarter of 2024 compared to $35.3 million for the fourth quarter of 2023, and $156.8 million for the full year ended December 31, 2024, compared to $119.9 million for the full year ended December 31, 2023. The year-over-year increase was primarily attributable to an increase in commercial-related activities as the company prepares for the potential approval of PYRUKYND in thalassemia.

Net Income (Loss): Net loss was $96.5 million for the fourth quarter of 2024 compared to a net loss of $95.9 million for the fourth quarter of 2023 and net income was $673.7 million for the year ended December 31, 2024, compared to a net loss of $352.1 million for the year ended December 31, 2023.

Cash Position and Guidance: Cash, cash equivalents and marketable securities as of December 31, 2024, were $1.5 billion compared to $806.4 million as of December 31, 2023. This increase reflects payments from the royalty monetization for vorasidenib and a milestone payment from Servier for vorasidenib approval received in the third quarter of 2024. Agios expects that its cash, cash equivalents and marketable securities, together with anticipated product revenue and interest income, will provide the financial independence to prepare for potential PYRUKYND launches in thalassemia and sickle cell disease, advance existing programs, and to opportunistically expand its pipeline through both internally and externally discovered assets.

Conference Call Information

Agios will host a conference call and live webcast today at 8:00 a.m. ET to discuss the company’s fourth quarter 2024 financial results and recent business highlights. The live webcast will be accessible on the Investors section of the company’s website (www.agios.com) under the "Events & Presentations" tab. A replay of the webcast will be available on the company’s website approximately two hours after the event.

On February 13, 2025 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a late-stage pharmaceutical company with a broad portfolio of drug candidates targeting hard-to-treat tumors and viruses, reported it has entered into agreements with certain holders of its existing warrants for the immediate exercise of certain outstanding warrants to purchase up to an aggregate of 5,828,570 shares of common stock of the Company originally issued in December 2023 and August 2024 all at a reduced exercise price of $1.00 per share (Press release, Moleculin, FEB 13, 2025, View Source [SID1234650257]). The shares of common stock issuable upon exercise of the outstanding warrants are registered pursuant to effective registration statements on Form S-1 (File No. 333-280951) and on Form S-1 (File No. 333-276851). The aggregate gross proceeds from the exercise of the existing warrants is expected to total approximately $5.8 million, before deducting financial advisory fees.

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Roth Capital Partners is acting as the Company’s financial advisor for this transaction.

In consideration for the immediate exercise of the warrants for cash, the Company will issue new unregistered warrants to purchase shares of common stock. The new warrants will be exercisable for an aggregate of up to 11,657,140 shares of common stock, at an exercise price of $0.75 per share and will be immediately exercisable upon issuance and for a term of five years from the issuance date.

The transaction is expected to close on or about February 14, 2025, subject to satisfaction of customary closing conditions. The Company intends to use the net proceeds from the offering for working capital and general corporate purposes.

The new warrants described above were offered in a private placement pursuant to an applicable exemption from the registration requirements of the Securities Act of 1933, as amended (the "1933 Act") and, along with the shares of common stock issuable upon their exercise, have not been registered under the 1933 Act, and may not be offered or sold in the United States absent registration with the Securities and Exchange Commission ("SEC") or an applicable exemption from such registration requirements. The Company has agreed to file a registration statement with the SEC covering the resale of the shares of common stock issuable upon exercise of the new warrants.

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 offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or jurisdiction.

On February 12, 2025 AbbVie (NYSE: ABBV) and Xilio Therapeutics, Inc. (Nasdaq: XLO), a clinical-stage biotechnology company discovering and developing tumor-activated immuno-oncology therapies for people living with cancer, reported a collaboration and option-to-license agreement to develop novel tumor-activated, antibody-based immunotherapies, including masked T-cell engagers, leveraging Xilio’s proprietary technology (Press release, Xilio Therapeutics, FEB 12, 2025, View Source [SID1234650215]).

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Xilio has developed a proprietary, clinically-validated platform technology for tumor-activated biologics. The company is advancing a pipeline of novel, clinical and pre-clinical immunotherapies, including masked multispecific molecules, designed to achieve tumor-selective activation by leveraging masking and other unique components that are optimized for the specific target. This allows focused activity within the tumor microenvironment with the goal of minimizing systemic adverse events.

"AbbVie is committed to expanding our R&D efforts in oncology. This includes investigation of novel immunotherapy approaches that aim to generate improved next-generation cancer treatments for patients in need," said Theodora S. Ross, M.D., Ph.D., vice president, early oncology research and development, AbbVie. "This partnership with the Xilio team, further exemplifies our commitment."

"This collaboration with AbbVie, a global leader in developing and commercializing oncology therapies, allows us to accelerate the expansion of our technology to next-generation immunotherapies, including T-cell engagers," said Uli Bialucha, Ph.D., chief scientific officer of Xilio. "We look forward to working with the AbbVie team to apply our deep protein engineering expertise coupled with tumor-selective activation through our novel formats for masked T-cell engagers."

Under the terms of the agreement, Xilio will receive $52.0 million in total upfront payments, including a $10.0M equity investment, and will be eligible to receive up to approximately $2.1 billion in total contingent payments for option-related fees and milestones plus tiered royalties.

Xilio Investor Conference Call Information

Xilio will host a conference call and webcast today at 8:30 am EST. Viewers can access the webcast by using this link. Listeners who require dial-in access should register here to receive a unique PIN and information to join the call. Listeners are encouraged to join at least 15 minutes prior to the scheduled start time. The webcast will also be accessible under "Events & Presentations" in the Investors & Media section of the Xilio Therapeutics website at View Source A replay of the webcast will be archived on the website for 30 days following the presentation.

On February 12, 2025 Agenus Inc. (Nasdaq: AGEN), a leader in immuno-oncology, reported that BOT/BAL will be featured in two presentations at the upcoming American Association for Cancer Research (AACR) (Free AACR Whitepaper) IO Annual Meeting that will take place on February 23-26 in Los Angeles, California (Press release, Agenus, FEB 12, 2025, View Source [SID1234650218]). An oral presentation will highlight interim data from the ongoing Phase 2 study of botensilimab and balstilimab (BOT/BAL) in combination with MiNK Therapeutics’ iNKT cell therapy, AgenT-797, in patients with refractory (2L+) gastric cancer (NCT06251973). A Trial-in-Progress (TiP) poster will feature data from the ongoing Phase 1/2 study of BOT/BAL in first-line MSS colorectal cancer (NCT05627635).

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Presentation Details:

Abstract Title: First-line botensilimab and balstilimab optimization in microsatellite stable colorectal cancer (MSS-CRC) without liver metastasis (BBOpCo)

Session : Poster Session A

Session Date and Time: Monday, February 24th , 1:45-4:45 p.m. PST

Abstract Title: Biomarker analysis from phase 2 study of AgenT-797 (invariant natural killer T-cells), botensilimab (a Fc-enhanced CTLA-4 Inhibitor) with balstilimab (anti-PD-1) in PD-1 refractory gastroesophageal cancer (GEC)

Session : Proffered Papers, Session 2

Session Date and Time: Tuesday, February 25th , 1:00-1:45 p.m. PST