On January 12, 2026 Disc Medicine, Inc. (NASDAQ:IRON), a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of novel treatments for patients suffering from serious hematologic diseases, reported its recent pipeline progress and strategic priorities for 2026.

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"2025 was a transformative year for Disc, marked by strong execution across our portfolio and meaningful progress toward becoming a fully integrated clinical and commercial organization," said John Quisel, J.D., Ph.D., President and Chief Executive Officer of Disc. "Most notably, bitopertin received CNPV designation and we submitted our NDA under the accelerated approval pathway, positioning the NDA for rapid review by the FDA. We also reported positive initial Phase 2 data from the RALLY-MF study of DISC-0974 in anemia of myelofibrosis, demonstrating robust and broad hematologic activity, and advanced our third program DISC-3405 into proof-of-concept studies for polycythemia vera and sickle cell disease."

"As we look ahead to 2026, we are entering an exciting and pivotal period for Disc," continued Dr. Quisel. "We are preparing to execute a successful US launch of bitopertin for EPP while continuing enrollment in the global APOLLO confirmatory study. Across our pipeline, we expect multiple Phase 2 updates for DISC-0974 and DISC-3405, including important regulatory interactions and expansion into new indications. Together, we believe these milestones position Disc for sustained growth as we work to deliver meaningful new therapies to patients with serious hematologic diseases."

Summary of Key Achievements During 2025

Bitopertin NDA submitted and accepted under the accelerated approval pathway with priority review
Bitopertin awarded the Commissioner’s National Priority Voucher (CNPV), a pilot program designed to accelerate the NDA review period to 1-2 months
Transition to commercial-ready organization through build out of marketing, market access, medical science liaison, and sales teams and related infrastructure
Presentation of positive initial data from the Phase 2 RALLY-MF study of DISC-0974 for anemia of MF, demonstrating robust and broad hematologic efficacy across patient segments
Issuance of a Composition of Matter patent for DISC-0974, providing patent exclusivity until 2041, not including potential extensions
Initiated Phase 2 study of DISC-3405 in PV and Phase 1b study of DISC-3405 in SCD
Strengthened balance sheet through two equity offerings, bringing unaudited cash, cash equivalents, and marketable securities to $791 million as of December 31, 2025, which provides runway into 2029

Key Business Objectives and Milestones for 2026
Bitopertin: GlyT1 Inhibitor (Heme Synthesis)

Anticipate FDA approval decision regarding NDA for bitopertin for the treatment of EPP under the CNPV program
Successful launch and initial commercialization of bitopertin in the US, if approved
Continue enrollment of the global, confirmatory APOLLO study with topline data expected by early 2027

DISC-0974: Anti-hemojuvelin Antibody (Hepcidin Suppression)

Progress ongoing Phase 2 MF anemia trial with updated data expected H2 2026
Conduct End of Phase 2 meeting with the FDA about DISC-0974 in MF anemia in H2 2026
Initiate a Phase 2 study of DISC-0974 in patients with anemia and inflammatory bowel disease (IBD)

DISC-3405: Anti-TMPRSS6 Antibody (Hepcidin Induction)

Progress ongoing Phase 2 PV trial with updated data expected H2 2026
Progress ongoing Phase 1b SCD trial with updated data expected H2 2026

Bitopertin, DISC-0974, and DISC-3405 are investigational agents and are not approved for use as therapies in any jurisdiction worldwide.

(Press release, Disc Medicine, JAN 12, 2026, View Source [SID1234661946])

On January 12, 2026 ORIC Pharmaceuticals, Inc. (Nasdaq: ORIC), a clinical stage oncology company focused on developing treatments that address mechanisms of therapeutic resistance, reported operational highlights for 2025 and anticipated upcoming milestones.

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"2025 was a transformative and highly productive year for ORIC, marked by meaningful progress across our pipeline, including data that further strengthened our conviction in the potential best-in-class profiles of rinzimetostat in prostate cancer and enozertinib in lung cancer," said Jacob M. Chacko, M.D., president and chief executive officer. "We also bolstered our leadership team and substantially extended our cash runway into 2H 2028 in anticipation of these programs advancing towards registrational studies and, ultimately, commercialization."

2025 Key Accomplishments

Rinzimetostat: a potent and selective allosteric inhibitor of PRC2

Completed Phase 1b dose exploration in prostate cancer and selected provisional recommended Phase 2 doses (RP2Ds) of rinzimetostat to be tested in combination with the approved doses of darolutamide and apalutamide in dose optimization.
Reported potential best-in-class efficacy and safety dose exploration data in combination with darolutamide and apalutamide in patients with metastatic castration-resistant prostate cancer (mCRPC). Data demonstrated:
PSA responses and ctDNA reductions across all rinzimetostat dose levels and at comparable rates in combination with apalutamide or with darolutamide.
Broad and deep PSA responses that compare favorably to competitor PRC2 inhibitors, with 55% of patients (11/20) achieving a PSA50 response (confirmed in 40%), and 20% of patients (4/20) achieving a PSA90 response (all confirmed).
Rapid and deep ctDNA responses across a breadth of AR mutations and other gene alterations, with 76% (13/17) achieving > 50% ctDNA reduction, and 59% (10/17) achieving ctDNA clearance, which is greater than clearance rates observed in precedent trials with standard of care agents in comparable mCRPC patient populations.
Both combination regimens demonstrated a clearly differentiated safety profile compatible with long-term dosing, with the vast majority of treatment-related adverse events (TRAEs) Grade 1 or 2 in severity and consistent with PRC2 and AR inhibition.
Presented preclinical data at the EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) Symposium on Molecular Targets and Cancer Therapeutics demonstrating potential utility of rinzimetostat combined with AR inhibition in castration-sensitive prostate cancer and combined with KRAS inhibition in KRAS G12C-mutant NSCLC and colorectal cancer models.

Enozertinib: a brain-penetrant inhibitor that selectively targets EGFR exon 20 and EGFR PACC mutations

Reported potential best-in-class efficacy and safety data from a Phase 1b trial of enozertinib at the ESMO (Free ESMO Whitepaper) Asia Congress 2025 in NSCLC patients with EGFR exon 20 and EGFR PACC mutations. Data demonstrated:
Systemic activity in 2L EGFR exon 20 and pretreated EGFR PACC exceeding competitor benchmarks.
Highly competitive preliminary 1L systemic activity, with 67% ORR in EGFR exon 20 and 80% ORR in EGFR PACC.
Convincing 1L CNS activity, with 100% intracranial ORR in EGFR exon 20 and 100% intracranial ORR in EGFR PACC in patients with measurable CNS disease, including in patients with active brain metastases.
Competitive safety profile, with no significant off-target toxicity, resulting in low rate of treatment discontinuations.
Announced a clinical trial collaboration and supply agreement with Johnson & Johnson to evaluate enozertinib in combination with amivantamab and hyaluronidase-lpuj subcutaneous injection (SC amivantamab) for the 1L treatment of NSCLC patients with EGFR exon 20 mutations.
Announced publication in Cancer Research of preclinical data demonstrating enozertinib’s exquisite selectivity, strong potency, brain-penetrance, and anti-tumor activity across a broad range of EGFR atypical mutant models, including intracranial lung cancer xenografts.

Corporate Highlights:

Announced the appointment of Kevin Brodbeck, PhD, to the newly established role of Chief Technical Officer (CTO).
Strengthened cash position and extended runway with $244 million in gross proceeds raised from new and existing top-tier healthcare specialist funds via private placement in May 2025 and under the ATM (at-the-market) program throughout 2025.

Anticipated Program Milestones:

ORIC anticipates the following upcoming milestones:

Rinzimetostat in mCRPC:
1Q 2026: Combination dose optimization data with AR inhibitor
1H 2026: Initiate first global Phase 3 registrational trial in mCRPC
2H 2026: Program update
Enozertinib in NSCLC:
2H 2026: 1L EGFR exon 20 monotherapy data and combination data with SC amivantamab
2H 2026: 1L EGFR PACC monotherapy data

Financial Guidance
As of September 30, 2025, cash, cash equivalents and investments totaled $413.0 million, which the company expects will be sufficient to fund its operating plan into 2H 2028, beyond several potential value inflection points, including anticipated primary endpoint readout from first Phase 3 trial of rinzimetostat.

Presentation and Webcast
Jacob M. Chacko, M.D., president and chief executive officer, will present a company overview at the 44th Annual J.P. Morgan Healthcare Conference on Tuesday, January 13, 2025, at 9:45 a.m. PT. A live webcast will be available through the investor section of the company’s website at www.oricpharma.com. A replay of the webcast will be available for 90 days following the event.

(Press release, ORIC Pharmaceuticals, JAN 12, 2026, View Source [SID1234661964])

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.

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"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])

On January 12, 2026 Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage company developing transformative therapies for the treatment of cancer and rare diseases, reported recent updates and anticipated 2026 milestones.

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"On the heels of a remarkable year marked by meaningful progress across the entirety of our pipeline, we are entering 2026 with unwavering resolve to continue to execute on our mission to deliver transformative therapies to patients with cancer and rare diseases," said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. "For our non-muscle invasive bladder cancer (NMIBC) program, our growing body of clinical data continue to support that TARA-002 could serve as a differentiated, easy to administer treatment option across the NMIBC treatment landscape. Following our recent ADVANCED-2 update in which TARA-002 demonstrated meaningful and durable activity in BCG-Naïve NMIBC patients, we look forward to providing an interim update on the registrational BCG-Unresponsive trial later this quarter."

Mr. Shefferman added, "We have also made important progress in our rare disease programs. Following positive results from the ongoing Phase 2 STARBORN-1 trial of TARA-002 in lymphatic malformations (LMs), we were pleased that the FDA granted this program both Breakthrough Therapy and Fast Track designations and we expect to provide a regulatory update defining the path to registration in the first half of this year. Additionally, we were pleased to announce that the first patient has been dosed in our registrational THRIVE-3 trial of IV Choline Chloride in patients on long-term parenteral support (PS) and expect to provide an interim analysis in the second half of 2026. We believe we are well positioned for continued success with several key milestones anticipated in the year ahead."

Recent Company Updates and Planned 2026 Milestones

TARA-002 in NMIBC

Protara remains on track to report in the first quarter of 2026 interim results in approximately 25 six-month evaluable patients from its ongoing Phase 2 open-label ADVANCED-2 trial in NMIBC patients with carcinoma in situ or CIS (± Ta/T1) who are Bacillus Calmette-Guérin (BCG)-Unresponsive.

In December 2025, the Company reported positive interim results from its ongoing Phase 2 open-label ADVANCED-2 trial in NMIBC patients with CIS (± Ta/T1) who are BCG-Naïve at the 26th Annual Meeting of the Society of Urologic Oncology (SUO) in which TARA-002 demonstrated meaningful response rates at six and 12 months and a favorable safety and tolerability profile. Based on feedback from the U.S. Food and Drug Administration (FDA), the Company plans to commence a registrational trial of TARA-002 compared to intravesical chemotherapy in BCG-naïve patients in the second half of 2026.

Protara continues to evaluate subcutaneous dosing through priming and maintenance combined with intravesical dosing, as well as exploring combination treatments with TARA-002 in NMIBC patients with CIS.

TARA-002 in LMs

Protara recently announced that the FDA granted TARA-002 both Fast Track and Breakthrough Therapy designations for the treatment of pediatric patients with macrocystic and mixed cystic LMs. TARA-002 previously was granted Rare Pediatric Disease designation for the treatment of LMs.

The Company plans to share a regulatory update on the path forward for registration for TARA-002 in LMs in the first half of 2026.

TARA-002 Manufacturing Update

Protara recently announced that TARA-002 has been selected to participate in this year’s FDA Chemistry, Manufacturing, and Controls (CMC) Development and Readiness Pilot (CDRP) Program. The FDA created the CDRP Program to facilitate CMC development for therapies with compressed clinical development timeframes based on the anticipated clinical benefits of earlier patient access to the therapy. The initiative is designed to promote earlier and more structured engagement between sponsors and FDA on CMC development strategies, and since its inception, has led to increased collaboration with the FDA so sponsors can confidently scale up manufacturing capacity while clinical development is ongoing.

IV Choline Chloride for Patients on PS

The Company recently announced that the first patient has been dosed in THRIVE-3 (NCT06910943), a seamless Phase 2b/3 trial designed to assess the efficacy and safety of low and high dose IV Choline Chloride in adolescent and adult patients receiving long-term PS when oral or enteral nutrition is not possible, insufficient, or contraindicated. Following an 8-week Phase 2b open-label, dose-confirmation trial in 24 patients, approximately 105 additional patients will be enrolled in a 24-week Phase 3 double-blinded, randomized, placebo-controlled trial. The primary endpoint of the trial is the change in plasma choline concentration from baseline compared to placebo. The Company expects to report interim results in the second half of 2026.

Corporate Update

In December 2025, the Company announced that it closed an underwritten public equity offering of approximately $86 million before deducting underwriting discounts and commissions and offering expenses payable by Protara. The proceeds from the offering are expected to extend the Company’s cash runway into 2028.

(Press release, Protara Therapeutics, JAN 12, 2026, View Source [SID1234661965])

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.

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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])