On January 12, 2026 Novita Pharmaceuticals, Inc. ("Novita"), a privately held, clinical-stage pharmaceutical company dedicated to developing novel cancer drugs through its proprietary fascin inhibitor technology, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its investigational small-molecule fascin inhibitor, NP-G2-044, for the treatment of pancreatic cancer. Pancreatic cancer is among the deadliest malignancies, with a five-year survival rate of approximately 12% and limited effective treatment options, particularly in advanced disease.

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!

"FDA Orphan Drug Designation for our fascin inhibitor represents an important regulatory milestone for Novita and validates the Company’s scientific and clinical approach in the fight against pancreatic cancer, as it remains one of the most lethal solid tumors with limited therapeutic progress over decades," said Stewart Campbell, Chief Executive Officer of Novita Pharmaceuticals. "Fascin inhibition offers a novel approach with the potential to enhance anti-tumor immune activity and improve outcomes for patients with this devastating disease, and we look forward to continuing advancement of NP-G2-044 to address the high unmet need."

Orphan Drug Designation is granted to investigational therapies intended to treat rare diseases affecting fewer than 200,000 patients in the United States. The designation provides development incentives, including eligibility for tax credits on qualified clinical trial costs, exemption from certain FDA user fees, and the potential for seven years of market exclusivity upon regulatory approval.

The FDA’s Orphan Drug Designation was granted by the FDA’s Office of Orphan Products Development (OOPD) and includes treatment of pancreatic cancer in the setting of immune checkpoint inhibitor (ICI) therapy, which falls within the scope of the approved orphan indication. As noted by the FDA, orphan designation applies to the active moiety of the drug rather than a specific formulation, and eligibility for orphan drug exclusivity will ultimately depend on the approved indication and demonstration of clinical benefit relative to any approved therapies in the same indication.

About Novita’s Pioneering Research in Fascin Inhibition
Cancer metastasis is the primary cause of over 90% of cancer-related deaths, yet there is currently no drug on the market specifically targeting metastasis. Furthermore, while Immuno-Oncology (IO) therapies, particularly immune checkpoint inhibitors, have made significant strides in cancer treatment, a large proportion of patients do not respond to existing IO treatments. Novita aims to address both of these critical medical needs by developing fascin inhibitors, which target a key protein involved in tumor cell motility and highly expressed in tumor cells and antigen-presenting cells within tumor tissues. The Company’s lead asset, NP-G2-044, is a small-molecule fascin inhibitor that has demonstrated the ability to block metastasis in both preclinical and clinical studies. Additionally, when combined with immune checkpoint inhibitors, NP-G2-044 has shown potential to reinvigorate anti-tumor immune responses. Novita’s multicenter Phase 2 clinical trial, titled "NP-G2-044 as Monotherapy and Combination Therapy in Patients with Advanced or Metastatic Solid Tumor Malignancies," is currently ongoing.

(Press release, Novita Pharmaceuticals, JAN 12, 2026, View Source [SID1234661986])

On January 12, 2026 CARsgen Therapeutics Holdings Limited (Stock Code: 2171.HK), a company focused on developing innovative CAR T-cell therapies, and Dispatch Bio, a biotech company engineering a universal treatment across solid tumors leveraging its first-in-class Flare platform, reported a clinical collaboration to conduct a Phase 1 trial in China, planned to begin in 2026.

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 trial will evaluate DISP-11, an investigational therapy leveraging Dispatch’s first-in-class Flare platform – including DV-10, the company’s novel tumor-specific virus – and CARsgen’s zevorcabtagene autoleucel (zevor-cel, R&D code: CT053), an autologous B-cell maturation antigen (BCMA)-targeting CAR T-cell therapy, in patients suffering from solid tumors. Zevor-cel is approved by the National Medical Products Administration (NMPA) in China for the treatment of multiple myeloma.

"CARsgen is working to transform outcomes for patients with solid tumors by advancing the potential of CAR T technologies," said Zonghai Li, M.D., Ph.D., Founder, Chairman of the Board, Chief Executive Officer and Chief Scientific Officer of CARsgen. "Dispatch’s Flare platform offers a differentiated and highly complementary approach to expanding where and how CAR T can be applied, particularly for solid tumors lacking specific targets. We are excited to explore this combination as part of our broader efforts to unlock meaningful benefit for patients."

In the planned study, patients with solid tumors of epithelial origin, which account for 90% of all solid tumors, will first receive DV-10, followed by zevor-cel.

"We are working to expand the impact of the Flare platform, and this collaboration represents a step forward in a region with infrastructure for efficient and impactful oncology development and significant unmet medical need," said Sabah Oney, Ph.D., Chief Executive Officer of Dispatch. "CARsgen’s scientific rigor and track record of rapid clinical advancement make them an ideal partner. As our second collaboration with a company that has successfully commercialized a CAR T therapy, this partnership reinforces the growing confidence in Flare’s potential across a variety of immunotherapies and will help shape our global development strategy."

About Zevor-cel

Zevor-cel is a fully human, autologous BCMA CAR T-cell product for the treatment of multiple myeloma (MM). Zevor-cel was approved by the NMPA on February 23, 2024, for the treatment of adult patients with relapsed or refractory MM who have progressed after at least three prior lines of therapy (including a proteasome inhibitor and an immunomodulatory agent). Zevor-cel received Regenerative Medicine Advanced Therapy (RMAT) and Orphan Drug designations from the U.S. FDA in 2019.

(Press release, Carsgen Therapeutics, JAN 12, 2026, View Source [SID1234661985])

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])