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Cogent Biosciences Announces Breakthrough Therapy Designation for Bezuclastinib in Combination with Sunitinib for Patients with Gastrointestinal Stromal Tumors (GIST)

On January 26, 2026 Cogent Biosciences, Inc. (Nasdaq: COGT), a biotechnology company focused on developing precision therapies for genetically defined diseases, reported that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) for bezuclastinib in combination with sunitinib for patients with Gastrointestinal Stromal Tumors (GIST) who have received prior treatment with imatinib.

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"We are excited to announce this Breakthrough Therapy Designation which recognizes the potential for the bezuclastinib combination to substantially improve upon the currently available treatment options for patients with imatinib-resistant GIST," said Andrew Robbins, Cogent’s President and Chief Executive Officer. "We look forward to the continued collaboration with the FDA as we work to bring the first new treatment option in over twenty years to this patient population."

This Breakthrough Therapy Designation is based on results from the PEAK trial which demonstrated a substantial and highly statistically significant clinical benefit on the primary endpoint of progression free survival (PFS), reducing risk of disease progression or death compared to the current standard of care by 50% (hazard ratio of 0.50, 95% CI: 0.39 – 0.65). mPFS, as assessed by blinded independent central review, was 16.5 months for the bezuclastinib combination vs. 9.2 months for sunitinib monotherapy. The combination was well tolerated, and no new safety risks were observed when compared to the known safety profile of sunitinib. Breakthrough Therapy Designation is intended to expedite the review of medicines that treat a serious or life-threatening condition and have shown clinical evidence indicating the potential for substantial improvement over available therapies.

Earlier this month, the FDA agreed to accept Cogent’s NDA under the FDA’s Real-Time Oncology Review (RTOR) program which allows an applicant to pre-submit components of its NDA to allow the FDA to review clinical trial data before the complete filing is submitted and aims to provide a more efficient review process to ensure safe and effective treatments are available to patients as early as possible.

Cogent plans to present full results from the PEAK trial at a major medical meeting during the first half of 2026. Additionally, in mid-2026 Cogent expects to initiate a Phase 2 trial investigating the benefit of the bezuclastinib plus sunitinib combination for first-line GIST patients with exon 9 mutations who are naive to, or recently initiated treatment with, imatinib.

(Press release, Cogent Biosciences, JAN 26, 2026, View Source [SID1234662208])

BioMarin Announces Proposed Private Offering of Senior Notes and Syndication of New Senior Secured Term Loan Facility

On January 26, 2026 BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) ("BioMarin") reported that it intends to offer, subject to market and other conditions, $850 million of senior unsecured notes due 2034 (the "Notes").

BioMarin also announced that, in connection with the pending acquisition (the "Acquisition") of Amicus Therapeutics, Inc. ("Amicus"), it launched the syndication of a new $2 billion senior secured term loan "B" facility (the "Term Loan B Facility"), which Term Loan B Facility is in addition to a $800 million senior secured term loan "A" facility (the "Term Loan A Facility" and, together with the Term Loan B Facility, the "Term Facilities"), and a $600 million senior secured revolving credit facility into which BioMarin expects to enter in connection with the Acquisition (the "New Revolving Facility" and, together with the Term Facilities, the "New Senior Secured Credit Facilities").

BioMarin intends to use the net proceeds from the offering of the Notes, together with borrowings under the Term Facilities and cash on hand, to fund the consideration payable in connection with the Acquisition and related fees and expenses in connection with the Acquisition, the borrowings under the New Senior Secured Credit Facilities, and the issuance of the Notes. The company may also borrow up to $150 million under the New Revolving Facility to pay such fees and expenses.

Gross proceeds from the issuance of the Notes will be deposited into an escrow account at the closing of the Offering, pending consummation of the Acquisition. In the event that the Acquisition is not completed on or prior to December 19, 2026, or upon the occurrence of certain other events, BioMarin will be required to redeem all of the Notes at a redemption price equal to 100% of the initial issue price of the Notes plus accrued and unpaid interest from the date of issuance, or the most recent date to which interest has been paid or provided for, to but excluding the special mandatory redemption date.

The Notes will be jointly and severally guaranteed by certain of BioMarin’s subsidiaries that will guarantee the obligations under the New Senior Secured Credit Facilities, including, after the closing of the Acquisition, Amicus and certain of its subsidiaries that will guarantee the obligations under the New Senior Secured Credit Facilities.

The indenture governing the Notes is expected to contain customary covenants that, among other things, restrict, with certain exceptions, the ability of each of BioMarin and its subsidiaries to incur additional debt, pay dividends, make certain other restricted payments, incur debt secured by liens, dispose of assets, engage in consolidations and mergers or sell or transfer all or substantially all of its assets.

The Notes will not be registered under the Securities Act of 1933, as amended (the "Securities Act"), or any state or other securities laws and may not be offered or sold in the United States absent an effective registration statement or an applicable exemption from the registration requirements of or in a transaction not subject to the Securities Act and any state or other applicable securities laws. Accordingly, the offering of the Notes is available only to a limited number of persons who are either (1) reasonably believed to be "qualified institutional buyers" as defined in Rule 144A under the Securities Act or (2) non-U.S. persons outside the United States pursuant to Regulation S under the Securities Act. The Notes will be subject to restrictions on transferability and resale and may not be transferred or resold except in compliance with the registration requirements of the Securities Act or pursuant to an exemption therefrom and in compliance with any state or other applicable securities laws.

This press release is for information purposes only and is not intended to and does not constitute, or form part of, an offer, invitation or the solicitation of an offer or invitation to purchase, otherwise acquire, subscribe for, sell or otherwise dispose of any securities, or the solicitation of any vote or approval in any jurisdiction, pursuant to the proposed transaction or otherwise, nor shall there be any sale, issuance or transfer of securities in any jurisdiction in contravention of applicable law. This press release contains information about the pending offering of the Notes, and there can be no assurance that

(Press release, BioMarin, JAN 26, 2026, View Source [SID1234662204]) the offering will be completed. The offering of the Notes may be made only by means of an offering memorandum.

Akari Therapeutics Files Key Patent and Unveils Second ADC Program AKTX-102 Targeting CEACAM5 Expressing Solid Tumors

On January 26, 2026 Akari Therapeutics, Plc (Nasdaq: AKTX), an oncology biotechnology company pioneering next-generation antibody drug conjugates (ADCs) powered by novel RNA-splicing payloads, reported the filing of a new U.S. provisional patent application (No. 63/958,508) covering its second pipeline candidate, AKTX-102, an ADC directed against CEACAM5 (Carcinoembryonic Antigen-related Cell Adhesion Molecule-5), a well-validated but historically difficult-to-drug oncology target.

CEACAM5 is expressed in 80–90% of gastrointestinal cancers, including colorectal and pancreatic cancer, approximately 30% of bladder cancers, 25% of lung adenocarcinomas, and up to 50% of luminal A (HR+) breast cancers. Importantly, CEACAM5 expression has also been linked to aggressive genetic subtypes, including KRAS-mutated lung cancers, underscoring its relevance across multiple high-unmet-need solid tumor indications.

AKTX-102 is a first-in-class ADC that combines a novel CEACAM5-targeting antibody construct with Akari’s proprietary PH1 spliceosome-modulating payload, designed to deliver potent, differentiated tumor cell killing while simultaneously activating both the innate and adaptive immune responses to the tumor.

Abizer Gaslightwala, President and Chief Executive Officer of Akari Therapeutics, commented, "This patent filing marks another important step in expanding Akari’s differentiated ADC platform and rapidly growing pipeline. AKTX-102 builds on our deep and unique insights into CEACAM5 tumor biology and we believe demonstrates the versatility of our PH1 payload and our antibody expertise to unlock previously intractable targets. We believe PH1 can serve as the foundation for a pipeline of novel ADCs, and this program highlights our innovation on novel payloads for ADCs as well as with tumor antigen biology and antibody engineering to build best-in-class ADCs."

"With AKTX-102, we aim to improve cytotoxic efficacy while harnessing PH1’s unique properties, including innate and adaptive immune activation and activity against KRAS-mutated cancers. We look forward to sharing additional progress as we continue to advance this exciting program," added Mr. Gaslightwala.

Rapidly Expanding and Deepening Patent Estate Around Novel ADCs and Payload Innovation

This newly filed patent further accelerates Akari’s rapid build-out of a broad and defensible intellectual property portfolio spanning payload biology, ADC architecture, and combination strategies. While Akari’s 2025 patent filings (US63/882,631, US63/891,856, and US63/891,861) focused on novel mechanisms of action, payload-driven biology, and ADC combination approaches, this new filing extends protection to a previously undisclosed pipeline asset, AKTX-102, and introduces new composition-of-matter claims around novel antibody design and ADC constructs utilizing this antibody design.

These filings build upon Akari’s foundational PH1 payload patent family (PCT/US2018/051721) and its lead clinical program AKTX-101 (PCT/US2024/024997), collectively creating a layered and rapidly expanding patent moat around next-generation ADCs. Together, Akari expects this growing estate positions the Company to generate multiple first- and best-in-class ADC candidates across a wide range of validated cancer targets.

Cracking One of Oncology’s Toughest Targets

CEACAM5 has long been viewed as a high-value oncology target, but its unique and challenging biology—including extensive antigen shedding and the presence of both soluble and tumor-bound forms—has historically limited therapeutic success. Despite decades of effort, no CEACAM5-directed therapy has yet achieved regulatory approval, whether as a naked antibody, ADC, or T-cell engager.

Beyond its role in tumor growth and metastasis, CEACAM5 also functions as an immunosuppressive checkpoint, inhibiting T-cell and natural killer (NK) cell activity to promote immune evasion. Akari’s newly filed patent covers novel antibody constructs engineered to address these biological challenges, as well as ADCs that pair these antibodies with the Company’s PH1 payload, enabling a differentiated therapeutic approach utilizing PH1’s unique immuno-oncology and cytotoxic modes of action.

This broad composition-of-matter protection provides Akari with ownership over a unique strategy to effectively target CEACAM5 as a best-in-class ADC therapeutic.

Execution, Momentum and Path to the Clinical Stage with Lead Program AKTX-101

As previously announced, Akari continues to execute on its strategy of advancing AKTX-101, its lead Trop2-targeted ADC, toward IND/CTA submission and first-in-human clinical evaluation, while simultaneously expanding a pipeline of next-generation ADCs enabled by its proprietary PH1 payload. With multiple validated targets, a growing IP estate, and a differentiated biological approach, the Company believes it is well positioned to deliver meaningful clinical impact and long-term value creation.

Key Catalysts and Milestones for AKTX-101 Development Program in 2026

Regulatory interactions with FDA in H1 2026 for feedback on our planned Phase 1 trial
Presentation of AKTX-101 data highlighting key areas of differentiation vs current Trop2 ADCs at a major scientific congress upcoming
Completion of CMC and non-clinical work including final GLP Toxicology for AKTX-101 to enable IND/CTA submissions at the end of 2026/ early 2027
Initiation of the Phase 1 clinical trial in late 2026 or early 2027, subject to regulatory clearance
Continued partnership discussions with pharmaceutical companies on our unique and differentiated PH1 payload/ADC approach and key catalysts forthcoming

(Press release, Akari Therapeutics, JAN 26, 2026, View Source [SID1234662202])

Entry into a material definitive agreement

On January 23, 2026, Lisata Therapeutics, Inc. (the "Company") and Qilu Pharmaceutical Co., Ltd. ("Qilu") reported to have entered into a Mutual Termination Agreement (the "Termination Agreement") relating to the Exclusive License and Collaboration Agreement between the Company (formerly Cend Therapeutics, Inc.) and Qilu, relating to the research, development and commercialization of certepetide (formerly known as CEND-1), dated February 11, 2021, as amended on April 26, 2021, and further amended by the Side Letter Agreement, dated November 10, 2023 (collectively the "License and Collaboration Agreement").

Previously, the Company and Qilu entered into the License and Collaboration Agreement, pursuant to which the Company granted Qilu a royalty-bearing exclusive license for the research, development and commercialization of certepetide in the Greater China territory (including Mainland China, Hong Kong, Macau, and Taiwan). Pursuant to the License and Collaboration Agreement, the Company was eligible to receive up to $200 million in development and commercial milestone payments and royalties ranging from 10% to 15% on licensed product sales.

Pursuant to the Termination Agreement, the License and Collaboration Agreement is terminated, effective as of January 23, 2026, and is no longer in effect, except that the termination does not relieve the parties from obligations under the License and Collaboration Agreement that accrued prior to the termination and certain other provisions expressly indicated to survive the termination.

(Filing, Lisata Therapeutics, JAN 23, 2026, View Source [SID1234662285])

Privo Technologies Successfully Completes Phase 1/2 Study of PRV211 Intraoperative Chemotherapy for Head and Neck Cancer Patients Novel Surgical Innovation Demonstrates Excellent Safety Profile with Potential to Reduce Tumor Recurrence

On January 23, 2026 Privo Technologies, Inc. reported the successful completion of its Phase 1/2 clinical evaluation of PRV211, a first-in-class intraoperative chemotherapy patch for head and neck cancer patients. The study demonstrated excellent safety outcomes across all eight patients, positioning PRV211 as a promising approach to preventing cancer recurrence at the surgical site.

Key Study Results:

Zero treatment-related serious adverse events (just one reported mild burning sensation while healing)
No systemic toxicities or dose-limiting toxicities
Normal wound healing with no surgical complications
Negligible systemic absorption confirming targeted delivery
Seamless integration into standard surgical procedures
Innovative Approach

PRV211 is applied directly to the tumor bed following surgical resection to address microscopic cancer cells that may remain in surgical margins. The nanoengineered patch delivers localized chemotherapy when tissue barriers are removed and access is optimal.

"This study represents an important step forward in our approach to cancer surgery," said Dr. Manijeh Goldberg, PhD, Founder and CEO of Privo Technologies. "PRV211 is customized medicine that enables surgeons to deliver targeted treatment precisely where recurrence risk is highest."

Addressing Clinical Need

Post-surgical recurrence remains a significant challenge in head and neck cancer. The study enrolled patients with invasive tumors (T1 – T4) requiring surgical excision, including advanced-stage cases requiring reconstructive surgery. PRV211’s targeted approach delivers chemotherapy directly to the surgical site with minimal systemic exposure.

Program Progress

PRV211 represents Arm 2 of Privo’s CLN-004 clinical program. Combined with Arm 1’s achievement of its primary efficacy endpoint and Arm 3’s dosing initiation, the program demonstrates continued progress in localized cancer therapy development.

Next Steps

Patients will be monitored for efficacy outcomes, including loco-regional recurrence at 12 months post-surgery. A future expansion study is planned to further evaluate PRV211’s potential to reduce cancer recurrence.

(Press release, Privo Technologies, JAN 23, 2026, View Source [SID1234662213])