On January 12, 2026 Pierre Fabre Pharmaceuticals, Inc. reported a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) stating that the Agency is unable to approve the tabelecleucel Biologics License Application (BLA) in its present form.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

We are surprised and deeply disappointed by the FDA’s decision, particularly given the urgent and life-threatening unmet medical need faced by patients with Epstein-Barr virus–positive post-transplant lymphoproliferative disease (EBV+ PTLD) after failure of standard-of-care therapy. These patients have no FDA-approved treatment options and a life expectancy often measured in weeks to months.

The BLA was resubmitted following clear alignment with the FDA on the acceptability of the resubmission criteria and fulfillment of the conditions outlined in the January 15, 2025, CRL, which identified a single GMP-related deficiency and raised no concerns regarding safety, efficacy, or trial design. Upon acceptance of the resubmission in July 2025, the FDA granted tabelecleucel accelerated approval status.

In the new CRL, despite acknowledging that the GMP issue had been resolved and raising no safety concerns, the FDA stated that it no longer considers the previously accepted single-arm ALLELE study to be adequate to support accelerated approval and requested a new study. This represents a significant and unexpected change in position, and one that is contrary to extensive dialogue with the Agency over more than five years.

We are concerned that this decision may have far-reaching consequences for the development of rare disease treatments, effectively creating barriers for generating clinical evidence within a unique patient population with ultra-rare conditions thereby significantly delaying—or preventing altogether— patient access to urgently needed therapies.

We firmly believe that tabelecleucel represents an important treatment advance for patients with EBV+ PTLD and that the totality of data supports its efficacy and safety. We intend to engage with the FDA to urgently pursue a path forward, in collaboration with Atara Biotherapeutics (Nasdaq: ATRA) and our clinical and patient partners, to enable timely accelerated approval of tabelecleucel. We continue to be committed to making tabelecleucel available to patients through our Expanded Access Program.

Approval and real-world use of tabelecleucel over several years in multiple countries outside the United States further support its clinical value. We remain fully committed to securing approval of this critical treatment option for U.S. patients and the physicians who care for them.

(Press release, Pierre Fabre, JAN 12, 2026, View Source [SID1234661984])

On January 12, 2026 10x Genomics, Inc. (Nasdaq: TXG), a leader in single cell and spatial biology, reported a collaboration with Dana-Farber Cancer Institute to analyze tumor samples from patients with major solid cancer types to uncover biological features associated with treatment response, resistance and disease progression. The company also announced plans to establish a CLIA-certified laboratory. Together, these efforts mark the beginning of a multi-year research initiative to incorporate single cell and spatial tumor analysis into potential diagnostic workflows to support cancer patient care.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The study with Dana-Farber intends to examine samples from hundreds of patients to evaluate therapeutic targets and relevant tumor microenvironment features for the most promising emerging therapies in oncology, including antibody-drug conjugates (ADCs), radioligand therapies (RLTs), bispecific antibodies, immune checkpoint inhibitors and other precision approaches. These treatments are reshaping the standard of care, yet patients can experience dramatically different outcomes. By understanding why patients respond differently to the same treatments, this research initiative may eventually help clinicians optimize therapy accordingly and move beyond current assays to capture the cellular ecosystems, immune activity and spatial context that influence how tumors respond to treatment.

Using 10x’s Chromium Flex single cell assay and Xenium spatial platform, the investigators will generate molecularly detailed maps of tumors that integrate both cellular composition and spatial architecture. By pairing these profiles with known clinical outcomes, the collaboration aims to reveal the biological features that distinguish patients who benefit from treatment from those who do not. Additionally, 10x and Dana-Farber will collaborate in defining actionable biomarkers for future clinical reporting, exploring how spatial and single cell insights, such as target expression patterns, immune contexture or indicators of therapeutic sensitivity, could be summarized in a format designed to support oncologists as they navigate increasingly complex treatment decisions.

"Collaborations like this are important for advancing precision oncology," said Himisha Beltran, MD, Director of Translational Research in Medical Oncology at Dana-Farber. "Integrating high-resolution tumor profiling with clinical outcomes allows us to study treatment response and resistance across solid tumors, generating insights that can inform future clinical applications."

In parallel with this scientific collaboration, 10x’s planned CLIA laboratory build-out will provide the regulated infrastructure needed for assay implementation, analytical validation and clinical sample processing, and also create the critical infrastructure necessary to deploy innovative diagnostic services for future clinical use.

"Through partnerships announced in 2025, we’ve already seen how single cell and spatial profiling can impact translational research, and we believe this is only the beginning," said Serge Saxonov, Co-founder and CEO of 10x Genomics. "Our goal is to accelerate the arrival of a world where deep, multidimensional biology directly informs patient care. That means continuing to empower our customers in their research, while also engaging in clinical collaborations like the one with Dana-Farber that help us advance how these insights translate into improved outcomes for patients. Establishing a CLIA lab is a natural next step for 10x and will strengthen the support we provide to investigators in academia and biopharma as they design, validate and bring forward the next generation of precision medicines."

This collaboration marks the beginning of a multi-year research effort through which 10x will collaborate with leading institutions to generate the scientific evidence needed to develop the clinical potential of single cell and spatial technologies. Additional studies and collaborations are planned, each contributing to the foundational work required to develop potential clinical tests in the future.

(Press release, 10x Genomics, JAN 12, 2026, View Source [SID1234661983])

On January 12, 2026 Abbisko Therapeutics Co., Ltd. ("Abbisko Therapeutics" hereafter, HKEX code: 02256.HK) reported that the New Drug Application (NDA) for its novel, orally administered, highly selective, and potent small-molecule colony-stimulating factor 1 receptor (CSF-1R) inhibitor, pimicotinib (ABSK021), for the systemic treatment of patients with tenosynovial giant cell tumor (TGCT), has been formally accepted by the U.S. Food and Drug Administration (FDA).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Pimicotinib was independently developed by Abbisko Therapeutics and has been licensed to Merck KGaA, Darmstadt, Germany, for worldwide commercialization. In December 2025, pimicotinib was approved by the China National Medical Products Administration (NMPA) for the treatment of adult patients with symptomatic TGCT for which surgical resection will potentially cause functional limitation or relatively severe morbidity. Additional applications are under review by regulatory bodies in other markets.

The FDA’s acceptance of the NDA for pimicotinib is supported by the robust efficacy and safety outcomes from the global, multicenter, randomized, double-blind, placebo-controlled Phase III MANEUVER trial. In the trial, TGCT patients who received once-daily oral pimicotinib achieved a statistically significant improvement in the primary endpoint of objective response rate (ORR) evaluated at Week 25 by blinded independent review committee (BIRC) based on RECIST v1.1.

The trial also demonstrated statistically significant and clinically meaningful improvements in all secondary endpoints related to key patient-reported outcomes in TGCT, including improvements in active range of motion and physical function and reductions in stiffness and pain.

Longer-term results with a median follow-up of 14.3 months further showed ORR continued to increase over time among patients treated with pimicotinib from the beginning of the study.

TGCT is a rare, locally aggressive tumor occurring in or around the joint leading to progressive swelling, stiffness and reduced mobility of the affected joint, significantly impacting daily activities and quality of life. If left untreated or in recurrent cases, TGCT may result in irreversible damage to the bone, joint and surrounding tissues. With regulatory submissions progressing across major markets worldwide, pimicotinib is expected to provide TGCT patients with a once-daily, oral, effective and well-tolerated therapeutic option, helping address unmet clinical needs in the management of TGCT.

About Pimicotinib

Pimicotinib is a novel, oral, highly selective, and potent small-molecule CSF-1R inhibitor independently developed by Abbisko Therapeutics. Positive results from the global Phase III MANEUVER study of pimicotinib for the treatment of Tenosynovial Giant Cell Tumor (TGCT) were announced in November 2024. Currently, pimicotinib has been approved by the National Medical Products Administration (NMPA) in China for the treatment of adult patients with symptomatic TGCT for which surgical resection will potentially cause functional limitation or relatively severe morbidity. In December 2023, Abbisko entered into an agreement with Merck KGaA, Darmstadt, Germany, pertaining to the commercial rights to pimicotinib, pursuant to which Merck KGaA, Darmstadt, Germany, is responsible for the commercialization of pimicotinib globally.

Outside of China, pimicotinib has been granted Breakthrough Therapy Designation by the US Food and Drug Administration (FDA) and PRIME Designation by the European Medicines Agency (EMA).

(Press release, Abbisko Therapeutics, JAN 12, 2026, View Source [SID1234661982])

On January 12, 2026 Oricell Therapeutics Holdings Limited ("Oricell" or "the Company"), a global leader in innovative cancer immunotherapy, reported the closing of a $70M Series C1 financing.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The round was co-led by Beijing Medical and Health Care Industry Investment Fund, Qiming Venture Partners and a leading global healthcare fund, with participation from a sovereign wealth fund, NGS Super (NGS), E-Town Capital, Elikon Venture, and Talon Capital. Proceeds will accelerate Oricell’s global expansion and clinical development, while strengthening its technological capabilities and paving ways to commercialization.

Oricell is advancing a differentiated pipeline of CAR-T cell therapies for solid tumors, underpinned by three proprietary platforms developed over the past decade:

OriAb: antibody discovery and engineering library
OriArmoring: enhancement of T-cell persistence and other crucial functions by TAs（Therapeutic Areas）
OnGo (Fast) CMC: rapid, scalable and effective manufacturing
Oricell has generated proof-of-concept (POC) clinical data across multiple pipeline CAR-T products. Its lead program, Ori-C101, is an autologous GPC3-targeted CAR-T therapy for advanced hepatocellular carcinoma (HCC). Both a Phase I Investigator-Initiated Trial (IIT) and a Phase I IND study of Ori-C101 are completed. With far-leading-industry efficacy and safety profile, Ori-C101’s clinical data have been featured at major academic conferences such as ASCO (Free ASCO Whitepaper) 2021 and ASCO (Free ASCO Whitepaper) 2025. Ori-C101 is entering the registrational pivotal trial and is well positioned to become the world’s first approved CAR-T therapy for HCC.

Beyond Ori-C101, Oricell’s integrated platforms are also yielding multiple next-generation CAR-T products with multi-targets and multi-mechanism designs. The innovative secreting CAR-T, OriC902, has shown groundbreaking efficacy and durability in ultra-late-line and difficult-to-treat solid tumor patients. Additionally, the company has initiated an IIT study to evaluate its proprietary dual-targeted in vivo CAR-T.

"We are deeply grateful for the strong vote of confidence from our investors," said Dr. Helen Yang, Chairlady and CEO of Oricell Therapeutics. "Oricell will continue to push forward the global clinical advancement of our pipeline, as well as continuous R&D of in vivo products and new technologies. Leveraging our deep technical expertise, policy tailwind and expanding market opportunities, we are able to accelerate the development of our CAR-Ts in the clinic at full speed. Our mission is to bring efficacious and affordable cell therapies to cancer patients worldwide with the hope of cure. By doing that, we aim to become a leading global immunotherapy enterprise."

Mr. Peng Ren, Chairman and General Manager of Beijing Medical and Health Care Industry Investment Fund, noted: "We believe cell therapy is a pivotal frontier in the fight against solid tumors, and we are highly impressed by the clinical progress of Oricell’s GPC3 CAR-T in treating HCC. We strongly value the team’s R&D capabilities and commercial vision, and look forward to supporting the company in accelerating global clinical breakthroughs for its core products."

"As an early investor in Oricell, we’ve witnessed the Company’s evolution from a promising startup to a technology powerhouse—from advancing autologous CAR-Ts to secreting CAR-Ts, to pioneering a 3-day rapid manufacturing process, and now to exploring in vivo CAR-T in the clinic," said Mr. Xubo Hu, Managing Partner of Qiming Venture Partners. "Oricell’s relentless innovation efforts continue to impress us and we are delighted to back a team that is committed to building truly global and groundbreaking therapies."

(Press release, OriCell Therapeutics, JAN 12, 2026, View Source [SID1234661981])

On January 12, 2026 Merck, a leading science and technology company, reported that the U.S. Food and Drug Administration (FDA) has accepted the company’s new drug application (NDA) for pimicotinib as a systemic treatment for patients with tenosynovial giant cell tumor (TGCT). The application is based on the primary results and longer-term follow-up of the global Phase 3 MANEUVER study, which demonstrated deep and durable tumor responses and meaningful improvements in clinical outcomes with pimicotinib.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"With pimicotinib, we have an opportunity to significantly advance care for people living with TGCT, a painful and debilitating disease that has few effective and well-tolerated treatment options beyond surgery," said David Weinreich, Global Head of R&D and Chief Medical Officer for the Healthcare business of Merck. "Based on clinical trial results showing not only a reduction in tumor burden, but also the ability to help alleviate symptoms like pain and stiffness in the global Phase 3 MANEUVER study, we are confident in the important role pimicotinib can play for TGCT patients in the U.S. and worldwide."

The application is based on primary and longer-term results from the global Phase 3 MANEUVER study. In this trial, once-daily pimicotinib demonstrated a statistically significant improvement in the primary endpoint of objective response rate (ORR) assessed by blinded independent review committee (BIRC) by RECIST v1.1 compared with placebo at week 25. The study also demonstrated statistically significant and clinically meaningful improvements in all secondary endpoints related to key patient-reported outcomes in TGCT, including improvements in active range of motion and physical function and reductions in stiffness and pain. These findings were presented at the 2025 ASCO (Free ASCO Whitepaper) Annual Meeting. Longer-term results with median follow-up of 14.3 months presented at ESMO (Free ESMO Whitepaper) Congress 2025 showed ORR continued to increase over time among patients treated with pimicotinib from the beginning of the study.

TGCT is a rare, locally aggressive tumor occurring in or around the joint leading to progressive swelling, stiffness and reduced mobility of the affected joint, significantly impacting daily activities and quality of life in what is typically an otherwise healthy population. If left untreated or in recurrent cases, TGCT may result in irreversible damage to the bone, joint and surrounding tissues. A significant need remains for highly effective and well-tolerated treatments beyond surgery that can not only shrink tumors but also alleviate pain and restore function.

In December 2025, pimicotinib was approved by the China National Medical Products Administration (NMPA) for the treatment of adult patients with symptomatic TGCT for which surgical resection will potentially cause functional limitation or relatively severe morbidity. Additional applications are under review by regulatory bodies in other markets.

(Press release, Merck KGaA, JAN 12, 2026, View Source [SID1234661980])