On March 10, 2026 C-Further, an international consortium committed to creating new therapeutics for childhood cancers, reported the first early-stage therapeutic programmes for its pipeline, dedicated to paediatric oncology indications. Through its collaborative model, C-Further has partnered with investigators at UVA Comprehensive Cancer Center, Dana-Farber Cancer Institute and Mass General Brigham to advance CF-012 for the potential treatment of Ewing sarcoma. In parallel, the consortium has partnered with investigators at MiNK Therapeutics (Nasdaq: INKT) to advance CF-033, leveraging MiNK’s proprietary platform with translational support by investigators at the University of Southampton, for the potential treatment of multiple children’s cancers, including bone sarcoma, medulloblastoma and acute myeloid leukaemia.

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Enabled by C-Further’s core partners, Cancer Research Horizons, LifeArc and Great Ormond Street Hospital Charity (GOSH Charity), the consortium will support the progression of CF-012 and CF-033 up to preclinical candidate nomination, subject to completion of scientific milestones. Together, these projects form the foundation of C-Further’s expanding pipeline which is supported by an initial $40m (£30m) budget.

Lone Friis, PhD, C-Further Programme Co-lead said: "By combining the pioneering approaches of our scientific collaborators with industry-standard drug discovery capabilities, expertise of our core partners and reach into critical paediatric-focused networks, C-Further is advancing a pipeline of potential first-in-class therapies for childhood cancers. Guided by an indication-agnostic but child-first approach, we believe the initial CF-012 and CF-033 programmes have the potential to address a profound unmet need across multiple children’s cancers."

The selected research partners represent top academic and industry investigators from across the globe, underscoring the international reach and ambition of C-Further.

CF-012 targets ETV6, a newly identified and critical transcriptional dependency that is essential for tumour growth and metastasis. The aim is to develop a potential first-in-class inhibitor with a precise mechanism to disrupt tumour growth and metastasis in young patients.

John Bushweller, PhD, Professor at the University of Virginia School of Medicine and a member of UVA Comprehensive Cancer Center, investigator for CF-012, said: "We’re excited to partner with C-Further to progress CF-012. Children and young people have historically lacked effective, targeted cancer treatments and CF-012 has the potential to address an urgent unmet need in relapsed and metastatic Ewing sarcoma – a rare, aggressive bone tumour that most commonly occurs in young people. With C-Further’s collaborative, child-first drug discovery model, we believe we have the power to bring these medicines to market."

Other key investigators for the CF-012 therapeutic programme include Kimberly Stegmaier, MD, Chair of the Department of Pediatric Oncology at Dana-Farber Cancer Institute, and Miguel Rivera, MD, Assistant Molecular Pathologist at Massachusetts General Hospital.
CF-033 is a potential first-in-class allogeneic iNKT cell therapy engineered with a specific T-cell receptor targeting PRAME, which bridges innate and adaptive immunity and is designed to enable both direct tumour cell killing and broader immune activation within the tumour microenvironment. As an allogeneic, off-the-shelf approach without the need for toxic lymphodepletion, CF-033 has the potential to support more timely treatment delivery and improved tolerability, which are important considerations in paediatric oncology, where new therapeutic approaches are urgently needed.

Marco Purbhoo, PhD, Head of Translational Medicine at MiNK Therapeutics, investigator for CF-033, said: "CF-033 is a PRAME-targeted invariant NKT (iNKT) cell therapy developed to address the urgent needs of children with high-risk cancers, which are often marked by treatment resistance and a weakened immune response that cannot sustain tumour control. Unlike conventional CAR-T or TCR-T therapies, CF-033 harnesses the unique biology of iNKT cells — immune cells that can both directly kill cancer and help coordinate a broader, longer-lasting anti-tumour response. Importantly, this approach is designed to be delivered without the need for HLA matching or toxic lymphodepletion, which is particularly meaningful for this vulnerable paediatric population."

Ali Roghanian, PhD, Associate Professor at the University of Southampton, is the other key investigator leading the CF-033 therapeutic programme.
C‑Further welcomes expressions of interest from researchers, innovators and partners who share its mission to accelerate new tailored and well-tolerated treatments for children and young people with cancer. The deadline to be considered for the next round of submissions is 13 March 2026.

C-Further has also commenced preliminary research on a third, undisclosed project. Additional programmes are expected to be announced in 2026.

(Press release, C-Further, MAR 10, 2026, View Source [SID1234663427])

On March 10, 2026 Jacobio Pharma(HKEX: 1167) reported its annual results for the year ended Dec. 31, 2025, and provided updates on its key pipeline programs.

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During 2025, the company achieved several key advancements in KRAS-targeted therapy and innovative ADC platforms, including the approval and inclusion of the KRAS G12C inhibitor glecirasib in China into the National Reimbursement Drug List, a collaboration agreement with AstraZeneca for the pan-KRAS inhibitor JAB-23E73 worth a total of US$2.015 billion (including an upfront payment of US$100 million), and the entry of EGFR-KRAS G12Di tADC and HER2-STINGa iADC into the IND preparation stage.

Dr. Yinxiang Wang, Chairman and Co-Chief Executive Officer of Jacobio, said:
"2025 marked the tenth anniversary of Jacobio and a milestone year of value for the Company. The approval of glecirasib and its reimbursement inclusion in China marked Jacobio’s entry into commercialization. At the same time, JAB-23E73 achieved steady progress over the past year, including completion of the Phase I dose escalation phase of daily dosing in China, generation of preliminary promising clinical data, and the IND approval of its first-line pancreatic cancer combination study. Looking ahead, we will continue to focus on KRAS-targeted therapies and innovative functional-payload ADC platforms, including tADC and iADC, with the goal of bringing more treatment options to patients worldwide."

Pipeline Highlights

Glecirasib (KRAS G12C inhibitor)

In 2025, glecirasib was approved in China for the treatment of patients with KRAS G12C-mutated non-small cell lung cancer who had received at least one prior systemic therapy, and was subsequently included in the National Reimbursement Drug List. From June to December 2025, Jacobio recognized revenue of RMB 8.55 million.

A registrational Phase III trial evaluating glecirasib in combination with the SHP2 inhibitor sitneprotafib for the first-line treatment of non-small cell lung cancer is ongoing in China. Results from the Phase I/II study of this combination were published in The Lancet Respiratory Medicine, demonstrating a 71% objective response rate and a median progression-free survival of 12.2 months in the first-line setting.

Over the past year, multiple clinical studies of glecirasib were published in leading international journals, including Nature Medicine, The Lancet Respiratory Medicine, The Lancet Gastroenterology & Hepatology, and Cancer Communication.

pan-KRAS inhibitor JAB-23E73

JAB-23E73 is an oral small-molecule pan-KRAS inhibitor currently undergoing clinical development in China and the United States.

As of Jan. 15, 2026, a total of 42 patients had been enrolled in the Phase I study in China. Grade 3 treatment-related adverse events were reported in 11.9% of patients, and no Grade 4 or Grade 5 treatment-related adverse events were observed. Among 13 pancreatic cancer patients treated within the predicted efficacious dose range — including two second-line and eleven third-line or later patients — the objective response rate (ORR), including both confirmed and unconfirmed responses, was 38.5%, and the disease control rate (DCR) was 84.6%.

In February 2026, China’s Center for Drug Evaluation approved a Phase I/III clinical trial evaluating JAB-23E73 in combination with nab-paclitaxel and gemcitabine as a first-line treatment for KRAS-mutant pancreatic ductal adenocarcinoma.

ADC Programs

Jacobio continues to advance its xADC platform built around functional payloads.

JAB-BX600 (EGFR-KRAS G12D tADC) combines an EGFR-targeting antibody with a KRAS G12D inhibitor payload, enabling targeted delivery and a dual-mechanism approach that may overcome feedback resistance associated with KRAS inhibitor monotherapy. An IND submission is expected in the second half of 2026.

JAB-BX467 (HER2-STING iADC) uses a HER2-targeting antibody linked to a STING agonist payload designed to recruit lymphocytes in the tumor microenvironment and potentially convert "cold" tumors into "hot" tumors. An IND submission is expected in the second half of 2026.

Financial Update

As of the end of 2025, the Company had approximately RMB1.53 billion in cash, cash equivalents and available bank credit facilities. This balance is expected to exceed RMB2.0 billion in the first quarter of 2026, and the Company anticipates achieving profitability in 2026.

(Press release, Jacobio Pharmaceuticals, MAR 10, 2026, View Source [SID1234663426])

On March 10, 2026 Agenus Inc. (Nasdaq: AGEN), a leader in immuno-oncology innovation, reported that it has triggered the first $20 million contingent payment under its previously disclosed strategic collaboration with Zydus Lifesciences Ltd.

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The payment was triggered by contracted work orders for critical chemistry, manufacturing and controls (CMC) and production activities related to botensilimab (BOT) and balstilimab (BAL). These activities will allow Zydus to perform the initiation of its commercial supply of Agenus’ lead programs. They also include additional manufacturing work to satisfy regulatory requirements for BLA and MAA readiness, to build upon existing inventory in anticipation of increasing demand across clinical development programs, authorized early access pathways, and to support potential global commercialization.

This milestone marks the first operational activities between Agenus and Zylidac Bio LLC, the U.S.-based biologics manufacturing subsidiary of Zydus Life Sciences.

"This milestone reflects our commitment to progressing BOT and BAL to regulatory approval readiness, and to support our ongoing clinical development and paid compassionate access program needs," said Garo H. Armen, Ph.D., Chairman and Chief Executive Officer of Agenus. "As reimbursed access continues in France under the AAC framework and named patient programs expand in permitted countries and enrollment advances in the global BATTMAN Phase 3 trial, it is essential that we proactively align manufacturing capacity with anticipated demand. Our partnership with Zydus enables us to scale thoughtfully while maintaining capital discipline."

Agenus currently maintains sufficient cGMP clinical-grade BOT and BAL drug product inventory to support the ongoing BATTMAN Phase 3 trial, the ANSM-authorized French access program (AAC) program, paid named patient programs in select countries where permitted, and ongoing investigator-sponsored trials. The newly initiated manufacturing activities are designed to supplement existing supply and position the company to meet expanding demand across paid compassionate access, development and potential future commercial settings.

Under the collaboration agreement, up to $50 million in contingent payments may be triggered by BOT and BAL production orders. The $20 million payment announced today is contractually allocated specifically for production and CMC-related activities. This structure enables Agenus to execute critical manufacturing work in support of its development and access programs without additional capital expenditures impacting its cash position.

The strategic collaboration between Agenus and Zydus, originally announced in June 2025 and closed in January 2026, provides Agenus with long-term U.S.-based biologics manufacturing capacity to support BOT+BAL’s global development and potential commercialization.

(Press release, Agenus, MAR 10, 2026, View Source [SID1234663425])

On March 10, 2026 Aptevo Therapeutics Inc. (Nasdaq:APVO), a clinical-stage biotechnology company developing novel immune-oncology therapeutics based on its proprietary ADAPTIR and ADAPTIR-FLEX platform technologies, reported new interim data for mipletamig in combination with venetoclax and azacitidine in newly diagnosed acute myeloid leukemia (AML) patients who are either elderly or unfit for intensive chemotherapy. In data from two trials, the combination has demonstrated robust clinical activity, delivering an 86% clinical benefit rate (CR/CRi/PR*)with zero patients experiencing the common symptom of cytokine release syndrome (CRS). These data support an emerging efficacy profile coupled with differentiated patient safety and tolerability that is additive to the current AML standard-of-care therapy.

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"The emerging mipletamig data in frontline AML are highly encouraging and highlight the differentiated profile we believe is needed to advance treatment in frontline AML," said Dirk Huebner, M.D., Chief Medical Officer of Aptevo Therapeutics. "In this study we are observing strong remission rates in a growing number of evaluable patients together with a consistently favorable safety and tolerability profile, including the absence of cytokine release syndrome. Achieving meaningful clinical activity while maintaining this level of safety and tolerability is essential in the frontline AML setting, where therapies must be compatible with established regimens. These results reinforce our belief that mipletamig can be successfully combined with venetoclax and azacitidine, with the potential to enhance outcomes for older and/or unfit AML patients who continue to face poor prognosis and limited treatment options."

Huebner continued, "Importantly, four of the patients treated to date have proceeded to allogeneic stem cell transplant, which represents the best possible outcome in AML treatment and is rarely achieved in the older or unfit frontline patient population."

Data Highlights Include:

Among the evaluable frontline patient population treated to date (N=28), including 24 patients from the RAINIER trial and 4 patients from the completed dose expansion trial, mipletamig in combination with venetoclax and azacitidine has demonstrated:

100% of frontline patients have remained free of cytokine release syndrome (CRS)

86% clinical benefit rate

79% achieved CR or CRi

61% achieved CR

55% of patients who achieved CR/CRi had blast reductions that reached the important measurable residual disease-negative level, a result that is typically associated with stronger, more durable responses

35% of patients with remissions had the TP53 genetic mutation, a high-risk biomarker typically associated with poor prognosis in AML and for which most treatment options frequently fail

Collectively, these data demonstrate mipletamig’s potential to meaningfully enhance frontline AML treatment in older and/or unfit patients, by improving efficacy outcomes without materially increasing toxicity.

"Our frontline data show that mipletamig has the potential to play a meaningful role in the future frontline AML treatment," said Marvin White, President and Chief Executive Officer of Aptevo Therapeutics. "From the outset, our objective has been to develop an AML drug capable of integrating into the current standard-of-care and improving outcomes for patients who continue to face poor prognoses. The data reported reinforces our conviction that mipletamig may represent a differentiated approach with the potential to complement existing frontline therapies in a practical and impactful way. As enrollment continues, we remain focused on advancing our RAINIER trial and generating the data needed to support mipletamig’s long-term role in AML treatment."

*(Clinical Benefit Rate: CR = complete remission; CRi = complete remission with incomplete blood marker recovery; PR = partial remission.)

Consistent Safety and Tolerability Profile Maintained Across Patients Treated to Date

In frontline patients treated to date, no cytokine release syndrome (CRS) has been observed. Together with strong efficacy outcomes, this outcome underscores mipletamig’s safety and combinability, potentially offering a superior treatment in the future. This safety profile is particularly important in frontline AML, where tolerability and combinability are essential for treating older patients and/or those with comorbidities.

About the RAINIER Trial

RAINIER, a frontline AML study, is a Phase 1b/2 dose optimization, multi-center, multi-cohort, open label study. Subjects are adults aged 18 or older, newly diagnosed with AML who are not eligible for intensive induction chemotherapy. RAINIER will be conducted in two parts. First, a Phase 1b dose optimization study in frontline AML patients followed by a Phase 2 study. The Phase 1b trial consists of 28-day cycles of treatment across multiple, sequential cohorts.

About Mipletamig

Aptevo’s wholly owned lead proprietary drug candidate, mipletamig, being evaluated for the treatment of AML, is differentiated by design to redirect the immune system of the patient to destroy leukemic cells and leukemic stem cells expressing the target antigen CD123, which is a compelling target for AML due to its overexpression on leukemic stem cells and AML blasts. This antibody-like recombinant protein therapeutic is designed to engage both leukemic cells and T cells of the immune system and bring them closely together to trigger the destruction of leukemic cells. Mipletamig is purposefully designed to reduce the likelihood and severity of CRS by use of the CRIS-7-derived CD3 binding pathway, an approach that differentiates Aptevo from competitors. Mipletamig has received orphan drug designation ("orphan status") for AML according to the Orphan Drug Act. Orphan drug designation provides key advantages-including the opportunity to seek U.S. market exclusivity for a specific period of time upon approval, FDA fee reductions, and access to development and tax credits. Mipletamig has been evaluated in more than 120 patients over three trials to date.

(Press release, Aptevo Therapeutics, MAR 10, 2026, View Source [SID1234663424])

On March 10, 2026 Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage biotechnology company developing transformative therapies for the treatment of cancer and rare diseases, reported a business update and announced financial results for the fourth quarter and full year ended December 31, 2025.

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"Over the last few months, we reported meaningful clinical and regulatory progress across our portfolio of late-stage programs and are building on this momentum as we advance toward several important milestones in 2026," said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. "For TARA-002 in non-muscle invasive bladder cancer (NMIBC), the clinical dataset continues to support its potential as a differentiated intravesical therapy with compelling efficacy, a favorable safety profile and a streamlined approach to administration in the clinical setting for both Bacillus Calmette-Guérin (BCG)-Unresponsive and BCG-Naïve patients. We remain on track to complete enrollment in our BCG-Unresponsive registrational cohort in the ADVANCED-2 trial and to initiate the ADVANCED-3 registrational trial in BCG-Naïve patients in the second half of 2026."

Mr. Shefferman added, "For our rare disease programs, we reported positive interim results for TARA-002 in lymphatic malformations (LMs), followed by the receipt of Breakthrough Therapy and Fast Track designations for TARA-002 in LMs from the U.S. Food and Drug Administration (FDA). We are working with the FDA to bring this promising treatment to patients expeditiously and expect to provide a regulatory update in the first half of 2026. In addition, our THRIVE-3 registrational trial of intravenous (IV) Choline Chloride for patients on parenteral support remains on track, with interim results expected in the second half of 2026. With cash runway into 2028, we are well positioned to continue executing across our pipeline and advance our mission of delivering transformational therapies for patients with cancer and rare diseases."

Recent Progress and Highlights

TARA-002 in NMIBC

At the ASCO (Free ASCO Whitepaper) Genitourinary Cancers Symposium in February 2026, the Company announced updated interim results from the ADVANCED-2 trial in evaluable NMIBC patients with carcinoma in situ or CIS (± Ta/T1) who are BCG-Unresponsive and BCG-Naïve. As of the January 28, 2026 data cutoff:
In the BCG-Unresponsive cohort of patients, TARA-002 demonstrated a complete response (CR) rate at any time of 65.7% (23/35) with CR rates of 68.2% (15/22) at six months and 33.3% (5/12) at 12 months. Among responders, the Kaplan-Meier (KM) estimated probability of maintaining a CR for six months was 71.1% (95% CI: 46.7, 95.5). Additionally, among responders, 100% (5 of 5) maintained their CR from nine to 12 months. Re-induction therapy was successful with 61.5% (8 of 13) of re-induced patients converting to a CR at six months.
In the BCG-Naïve cohort of patients, TARA-002 demonstrated a CR rate at any time of 72.4% (21/29) with CR rates of 66.7% (18 of 27) at six months and 57.9% (11 of 19) at 12 months. Among responders, the KM estimated probability of maintaining a CR for six months was 73.1% (95% CI: 52.9, 93.4). In addition, among responders, 100% (11 of 11) maintained their CR from nine to 12 months. Re-induction therapy was successful with 66.7% (4 of 6) of re-induced patients converting to a CR at six months.
The majority of treatment-related adverse events (TRAEs) were Grade 1 and transient with no Grade 3 or greater TRAEs and no related serious adverse events as assessed by study investigators. No patients discontinued treatment due to TRAEs. The most common TRAEs were dysuria, bladder spasm, fatigue and micturition urgency. Most bladder irritations resolved shortly after administration or within a few hours to a few days.
The Company has completed enrollment of the BCG-Naïve cohort and expects to complete enrollment of the BCG-Unresponsive cohort in the second half of 2026.
The Company remains on track to commence ADVANCED-3, a registrational trial of TARA-002 compared to intravesical chemotherapy in BCG-Naïve patients (who have never been exposed and those who have not received BCG within the last 24 months and are ineligible to receive BCG, have a contraindication to BCG, cannot tolerate BCG, do not have access to BCG or refuse BCG) in the second half of 2026.
TARA-002 in LMs

In January 2026, the Company announced that the FDA granted TARA-002 Fast Track and Breakthrough Therapy designations for the treatment of pediatric patients with macrocystic and mixed cystic LMs. TARA-002 was previously granted Rare Pediatric Disease designation for the treatment of LMs.
The Company remains on track to share a regulatory update on the registrational path for TARA-002 in LMs in the first half of 2026.
IV Choline Chloride for Patients on Parenteral Support (PS)

THRIVE-3, the Company’s registrational Phase 3 clinical trial, is ongoing, and the Company expects to report interim results in the second half of 2026.
Corporate Update

Fourth Quarter and Full Year 2025 Financial Results

As of December 31, 2025, unrestricted cash and cash equivalents and marketable debt securities totaled $197.9 million, including proceeds from the Company’s $86.3 million public offering in December 2025. The Company expects its cash and cash equivalents and marketable debt securities will be sufficient to fund its planned operations and milestones into 2028.
Research and development expenses for the fourth quarter of 2025 increased to $13.1 million from $9.5 million for the prior year period, and for the full year increased to $42.6 million compared to $31.7 million for 2024. The fourth quarter and full year increases were primarily due to the start-up and advancement of clinical trials across our portfolio.
General and administrative expenses for the fourth quarter of 2025 increased to $6.0 million from $4.8 million for the prior year period, and for the full year increased to $21.9 million compared to $17.5 million for 2024. The fourth quarter and full year increases were primarily due to increases in personnel-related expenses and other general and administrative expenses primarily related to professional and consulting services.
For the fourth quarter of 2025, Protara incurred a net loss of $17.3 million, or $0.37 per share, compared with a net loss of $12.8 million, or $0.48 per share, for the same period in 2024. Net loss for the year ended December 31, 2025 was $57.4 million, or $1.34 per share, compared with a net loss of $44.6 million, or $2.17 per share, for the year ended December 31, 2024. Net loss for the fourth quarter of 2025 included approximately $1.0 million in stock-based compensation expenses. Net loss for the year ended December 31, 2025 included approximately $3.8 million in stock-based compensation expenses.
About ADVANCED-2

ADVANCED-2 (NCT05951179) is a Phase 2 open-label trial assessing intravesical TARA-002 in non-muscle invasive bladder cancer (NMIBC) patients with carcinoma in situ or CIS (± Ta/T1) who are Bacillus Calmette-Guérin (BCG)-Unresponsive (Cohort B N=75-100) or BCG-Naïve (Cohort A N=31). Trial subjects received an induction course, with or without a reinduction, of six weekly intravesical instillations of TARA-002, followed by a maintenance course of three weekly instillations every three months. The BCG-Unresponsive cohort is designed to be registrational based on the FDA’s August 2024 Draft Guidance for Industry on BCG-Unresponsive Nonmuscle Invasive Bladder Cancer: Developing Drugs and Biological Products for Treatment.

About TARA-002

TARA-002 is an investigational cell therapy in development for the treatment of NMIBC and of LMs, for which it has been granted Rare Pediatric Disease, Breakthrough and Fast Track designations by the FDA. TARA-002 is a first-in-class TLR2/NOD2 agonist and novel immunopotentiator derived from inactivated Streptococcus pyogenes with a mechanism of action that includes the activation of innate and adaptive immune pathways within the bladder wall. When TARA-002 is administered, it is hypothesized that innate and adaptive immune cells within the cyst or tumor are activated and produce a pro-inflammatory response with the release of cytokines such as tumor necrosis factor (TNF)-alpha, interferon (IFN)-gamma, IL-6, IL-10 and IL-12. TARA-002 also directly kills tumor cells and triggers a host immune response by inducing immunogenic cell death, which further enhances the antitumor immune response.

TARA-002 was developed from the same master cell bank of genetically distinct group A Streptococcus pyogenes as OK-432, a broad immunopotentiator marketed as Picibanil in Japan by Chugai Pharmaceutical Co., Ltd. Protara has successfully shown manufacturing comparability between TARA-002 and OK-432.

About Non-Muscle Invasive Bladder Cancer

Bladder cancer is the sixth most common cancer in the United States, with non-muscle invasive bladder cancer (NMIBC) representing approximately 80% of bladder cancer diagnoses, or approximately 65,000 patients in the U.S. each year. NMIBC is cancer found in the tissue that lines the inner surface of the bladder that has not spread into the bladder muscle.

About Lymphatic Malformations

Lymphatic Malformations (LMs) are rare, congenital malformations of lymphatic vessels resulting in the failure of these structures to connect or drain into the venous system. Protara’s focus is on macrocystic and mixed cystic LMs, for which there are no currently approved therapies. They are most frequently present in the head and neck region and are diagnosed in early childhood during the period of active lymphatic growth, with more than 50% detected at birth and 90% diagnosed before the age of three years. The most common morbidities and serious manifestations of the disease include compression of the upper aerodigestive tract, including airway obstruction requiring intubation and possible tracheostomy dependence; intralesional bleeding; impingement on critical structures, including nerves, vessels and lymphatics; recurrent infection; and cosmetic and other functional disabilities. TARA-002 has been granted Rare Pediatric Disease, Breakthrough Therapy and Fast Track designations by the FDA for the treatment of LMs.

About IV Choline Chloride for Patients on Parenteral Support

IV Choline Chloride is an investigational, intravenous phospholipid substrate replacement therapy in development for patients receiving parenteral support (PS). Choline is a known important substrate for phospholipids that are critical for healthy liver function and play an important role in modulating gene expression, cell membrane signaling, brain development and neurotransmission, muscle function and bone health. PS patients are unable to synthesize choline from enteral nutrition sources, and there are currently no available PS formulations containing choline. Approximately 78% of patients dependent on PS are choline-deficient and of those approximately 63% have some degree of liver dysfunction, which can lead to hepatic failure. Every year in the U.S. there are approximately 90,000 people who require PS at home and of those approximately 30,000 are on long-term PS. IV Choline Chloride has the potential to become the first FDA approved IV choline formulation for PS patients. It has been granted Orphan Drug designation by the FDA for the prevention and/or treatment of choline deficiency in patients on long-term parenteral nutrition and has been granted Fast Track designation as a source of choline when oral or enteral nutrition is not possible, insufficient or contraindicated. The U.S. Patent and Trademark Office has issued Protara a U.S. patent claiming a choline composition and a U.S. patent claiming a method for treating choline deficiency with a choline composition, each with a term expiring in 2041.

(Press release, Protara Therapeutics, MAR 10, 2026, View Source [SID1234663422])