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RedHill Biopharma Announces Full-Year 2024 Financial Results and Operational Highlights

On April 10, 2025 RedHill Biopharma Ltd. (NASDAQ: RDHL) ("RedHill" or the "Company"), a specialty biopharmaceutical company, reported its full-year 2024 financial results and operational highlights and associated filing of its annual report on Form 20-F for the year ended December 31, 2024 (Press release, RedHill Biopharma, APR 10, 2025, View Source [SID1234651876]).

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Dror Ben-Asher, RedHill’s Chief Executive Officer, said: "We have undergone an extensive overhaul reshaping ourselves financially, operationally and strategically. Major corporate and R&D moves, undertaken over the last 12 months, have resulted in new and clearer opportunities to deliver maximum value from both our commercial and R&D assets. The potential $60 million ex-North America global license of RHB-102 to Hyloris lays the groundwork for the ongoing development and commercialization in the large gastroenteritis, oncology support and IBS-D markets while enabling RedHill to maintain control of the key North American markets. The approximately $8 million plus legal costs New York Supreme Court summary judgment was a resounding win for RedHill and we are fully committed to pursuing the collection of this award. Last year’s recommendation by the latest ACG Clinical Guideline for Talicia as a first-line therapeutic option for H. pylori eradication, has, we expect further enhanced Talicia’s product profile as the leading branded U.S. gastroenterologist prescribed H. pylori therapy, winning additional formulary successes securing 25 million covered lives. After its successful launch in UAE, Talicia is also poised for a potential UK Marketing Authorisation Application (MAA) mid-year, presenting a promising pathway for Talicia’s entry into additional markets globally."

Mr. Ben-Asher continued: "Having successfully completed the Hyloris RHB-102 out-license deal, we are now advancing our next-generation candidate, RHB-204, in the $12 billion Crohn’s disease space, employing a novel, groundbreaking approach, supported by RHB-104’s positive Phase 3 clinical data. In parallel, opaganib continues to show its broad-acting potential and we are very excited to have initiated the innovative Bayer-supported Phase 2 study in combination with darolutamide, which may bring vital new hope to men with metastatic castrate resistant prostate cancer. Progress was also made with opaganib as a treatment for GI-ARS following positive results from new in vivo studies, undertaken as part of the U.S. government’s Radiation and Nuclear Countermeasures Program (RNCP) product pipeline development contract, further confirming opaganib’s radioprotective activity in models of GI-ARS. Discussions are ongoing regarding advanced development. Additionally, the ongoing U.S. Government-supported work in Ebola continues following our previously announced BARDA grant. We have started 2025 as we mean to go on – aggressively pursuing our business goals and aiming to deliver on our catalysts in a meaningful way."

Financial results for the 12 months ended December 31, 20242

Net Revenues for the year ended December 31, 2024 were $8.0 million, compared to $6.5 million for the year ended December 31, 2023. Talicia net revenues for the year ended December 31, 2024, increased to $9.0 million from $8.8 million for the year ended December 31, 2023, driven by approximately $1.0 million of revenues generated from the UAE partnership with Gaelan Medical. Net revenues for the years ended December 31, 2024 and December 31, 2023 included Movantik contra-revenues of $0.9 million and $2.6 million for Movantik, respectively, mainly due to product returns.

Cost of Revenues for the year ended December 31, 2024 was $3.2 million, compared to $3.5 million for the year ended December 31, 2023. The decrease was primarily due to lower inventory write-downs, which totaled $0.2 million in 2024 compared to $1.3 million in 2023.

Gross Profit for the year ended December 31, 2024 was $4.9 million, compared to $3.1 million for the year ended December 31, 2023, reflecting the increase in net revenues and the lower level of inventory write-downs in 2024.

Research and Development Expenses for the year ended December 31, 2024 were $1.6 million, as compared to $3.5 million for the year ended December 31, 2023. The decrease was attributable to the costs from closing the RHB-204 clinical trial, which were recognized in 2023, as well as ongoing cost-reduction measures.

Selling, Marketing, and General and Administrative Expenses for the year ended December 31, 2024 were $15.5 million, as compared to $31.0 million for the year ended December 31, 2023. The reduction was primarily attributable to ongoing cost-reduction measures and the divestment of Movantik in 2023, which led to workforce downsizing and other related expense reductions.

Other Expenses for the year ended December 31, 2024 were $2.3 million, recognized as part of the Global Termination Agreement3, as compared to Other Income of $44.1 million for the year ended December 31, 2023. The Other Income in 2023 was comprised of (i) $35.5 million from the divestiture of Movantik, calculated as the difference between the fair value of the rights and the carrying amount of this asset and (ii) $8.6 million from transitional services provided to the buyer of Movantik.

Operating Loss for the year ended December 31, 2024 was $14.6 million, compared to Operating Income of $12.6 million for the year ended December 31, 2023. Both periods include items related to the Movantik divestiture, as described under Other Expenses – a $2.3 million loss in 2024 and $44.1 million income in 2023. Excluding these, the year-over-year change reflects improved operating performance driven by cost-cutting measures.

Financial Income, net for the year ended December 31, 2024 was $6.3 million, compared to Financial Income, net of $11.3 million for the year ended December 31, 2023. The income recognized for the year ended December 31, 2024, was primarily driven by the revaluation of financial instruments, partially offset by other financing expenses. The income recognized in the year ended December 31, 2023, was primarily attributable to a $20.6 million gain resulting from the extinguishment of the HCRM debt in exchange for the transfer of rights to Movantik, calculated as the difference between the carrying amount of the financial liability and the fair value of the rights transferred, partially offset by financial expenses related to the financial instruments and other financial expenses.

Net Loss for the year ended December 31, 2024 was $8.3 million, as compared to Net Income of $23.9 million for the year ended December 31, 2023. Both periods include impacts from the Movantik divestiture, as detailed under Other Expenses and Financial Income – a $2.3 million loss in 2024 and $64.7 million in income in 2023. Excluding these, the year-over-year change reflects improved performance driven by cost cutting measures.

Total Assets as of December 31, 2024 were $18.0 million, as compared to $23.0 million as of December 31, 2023. The decrease was primarily attributable to the decrease in cash balance, reduced inventory and a decline in prepaid expenses and other receivables, consistent with the Company’s scaled-down operations, as well as impact of balances settled as part of the Global Terminations Agreement, and a reduction in right-of-use assets following the termination of vehicle leases during 2024.

Total Liabilities as of December 31, 2024 were $22.7 million, as compared to $21.0 million as of December 31, 2023. The increase primarily reflects the impact of the Global Termination Agreement, under which the Company incurred liabilities related to Movantik that were allocatable to HCRM and its affiliates under their agreements with the Company, offset by payments made toward these liabilities during the period. Additionally, there was an increase in derivative financial instruments associated with warrant liabilities from offerings made during 2024. This was partially offset by a decrease in accounts payable and allowance from deductions from revenues, consistent with the Company’s scaled-down operations, as well as a reduction in lease liabilities due to the termination of car leases.

Net Cash Used in Operating Activities for the year ended December 31, 2024 was $9.4 million, compared to $35.8 million for the year ended December 31, 2023. The cash used in operating activities was primarily directed towards settling pre-closing liabilities related to Movantik and other operational activities. This was partially offset by proceeds received from the Global Termination Agreement, net of payments made to settle obligations arising from this agreement.

Net Cash Provided by Financing Activities for the year ended December 31, 2024 was $8.4 million, primarily generated through equity offerings. Net Cash Provided by Financing Activities for the year ended December 31, 2023, was $21.4 million, comprised primarily of proceeds from equity offerings and exercise of certain warrants in transactions consummated in each of April 2023, July 2023, September 2023 and November 2023, and from decrease in restricted cash, partially offset by repayment of payables in respect of intangible asset purchases.

Cash Balance as of December 31, 2024, was $4.8 million1.

Commercial and R&D Highlights:

Commercial – streamlined and revenue-generating:

With a significantly streamlined commercial operation, Talicia has generated net revenues of $9.0 million, supported by approximately $1.0 million of new revenues from the UAE partnership with Gaelan Medical, and remains the leading U.S. gastroenterologist-prescribed branded H. pylori therapy.

Significant effort has resulted in important accomplishments with Talicia, such as the inclusion as first-line option for treatment of H. pylori infection in the recently updated 2024 American College of Gastroenterology (ACG) Clinical Guideline, the securing of 25 million covered lives following the Medi-Cal renewal and Humana formulary win, a successful launch in the UAE and the potential for opening up new markets with the recently announced plan for a Talicia UK MAA. Talicia has now surpassed the 100,000 prescriptions milestone and our innovative warranty program, with minimal refunds claimed, reflects a positive patient experience.

R&D – focused on new opportunities:

Largely externally funded, with multiple U.S. Government and non-governmental collaborations, RedHill’s pipeline provides new and exciting opportunities in major indications: Crohn’s disease, prostate cancer, diabetes and obesity-related disorders, Ebola and other viral and pandemic preparedness indications as well as for gastrointestinal-acute radiation syndrome (GI-ARS) and other medical/chemical countermeasure uses:

Opaganib4:

A potentially broad acting, novel, oral, host-directed small molecule drug, with a demonstrated safety and efficacy profile, advancing in predominantly U.S. Government-supported, externally funded programs, directed at multiple underserved indications with sizeable multi-billion-dollar market opportunities and potentially advantageous pathways to approval.

Opaganib is in development for multiple oncology, viral, inflammatory and diabetes and obesity-related indications, including prostate cancer, COVID-19, Ebola, acute respiratory distress syndrome (ARDS) and radio/chemical protection, including GI-ARS:

A new approach in the $12 billion prostate cancer market:

Prostate cancer (PC) is the second most diagnosed cancer in the world, with around 1.5 million new cases per year, causing almost 400,000 deaths5. People with metastatic castrate-resistant prostate cancer (mCRPC) have few treatment options available to them.

In February 2025, the Company announced the initiation of a Bayer-supported Phase 2 study of opaganib in combination with Bayer’s darolutamide in mCRPC, evaluating the potentially enhancing effect of opaganib in patients with poor prognosis.

The study will utilize a companion lipid biomarker test (PCPro) to select mCRPC patients who have a poor prognosis due to standard of care (SoC) treatment and who may benefit from an opaganib + darolutamide combination treatment approach. The primary endpoint will be improved 12-month radiographic progression-free survival (rPFS).

Other opaganib programs/updates include:

U.S. Army and BARDA-grant funded program for Ebola. Opaganib is believed to be the first host-directed molecule to show activity in vivo in Ebola virus disease, delivering a statistically significant increase in survival and, separately, demonstrating a robust synergistic effect in vitro when combined with remdesivir (Veklury; Gilead Sciences, Inc.), improving viral inhibition while maintaining cell viability
U.S. Government- and non-government funded programs ongoing with the NIH / BARDA-funded nuclear and chemical medical countermeasure programs for GI-ARS and Phosgene inhalation injury. On December 10, 2024, the Company announced positive results from new in vivo studies of opaganib as a treatment for GI-ARS, undertaken as part of the U.S. Government’s Radiation and Nuclear Countermeasures Program (RNCP) product pipeline development contract. These results further confirm opaganib’s protective activity in models of GI-ARS and discussions are ongoing regarding advanced development.
Positive in vivo study results support potential of opaganib therapy in diabetes / obesity-related disorders – a market projected to be worth approximately $100 billion within the next decade. ‘Opaganib Promotes Weight Loss and Suppresses High-Fat Diet-Induced Obesity and Glucose Intolerance’ was recently published6 in the journal Diabetes, Metabolic Syndrome and Obesity
Orphan drug designation granted by FDA for neuroblastoma (opaganib has several such designations in multiple indications, with three in oncology)
ARDS, COVID-19 and Influenza programs continue to seek to address multi-hundreds of millions of dollars markets
RHB-204:

RHB-204 is a proprietary, fixed-dose oral capsule containing a combination of clarithromycin, rifabutin and clofazimine, at specific doses designed to safely and effectively treat Mycobacterium avium subspecies paratuberculosis-positive (MAP-positive)-related Crohn’s disease (CD). Unlike existing therapies that focus on symptom relief, RHB-204 is intended to target the possible root cause of Crohn’s disease, which is hypothesized to be caused by Mycobacterium avium subspecies paratuberculosis (MAP).

Patent protected until at least 2041, RHB-204 is a next-generation formulation of RHB-104, which successfully completed a Phase 3 study in Crohn’s disease, with an optimized formulation for the treatment of CD. It contains the same three antimicrobial agents with potent intracellular, anti-mycobacterial and anti-inflammatory properties, and with an optimized dosing profile, RHB-204 provides the potential for enhanced tolerability, safety and compliance with a 40% pill burden reduction. RHB-204 is supported by a strong foundation of clinical data from the positive safety and efficacy results achieved in the Phase 3 study of RHB-104 in CD, with its potential further demonstrated using mucosal healing imaging, considered to be the gold standard for efficacy evaluation in CD.

Paradigm shift in MAP-positive CD treatment approach

In March 2025, the Company announced its plans to advance its potentially groundbreaking late-stage RHB-204 Crohn’s disease program, building on statistically significant positive RHB-104 Phase 3 results. FDA guidance on pathway to approval is anticipated in the coming weeks. RedHill is actively pursuing funding opportunities and partnerships to advance this potential paradigm-shifting treatment.

The planned innovative Phase 2 study of RHB-204 is planned to be the first ever clinical study in CD patients who are all MAP-positive and will evaluate mucosal healing, a new gold standard in assessing efficacy in Crohn’s disease, and MAP eradication utilizing novel and decisive endpoints and imaging, allowing for a study design with a relatively small sample size.

RHB-204 builds upon RHB-104’s successful Phase 3 study, which successfully met its Phase 3 study primary and secondary endpoints demonstrating a statistically significant 64% improvement in efficacy versus standard of care. It also showed compelling mucosal healing data in CD patients who underwent colonoscopy. The inclusion of MAP-positive only patients in the planned study with RHB-204 is anticipated to demonstrate a more consistent benefit in the study population across all efficacy outcomes.

RHB-107 (upamostat) update:

On January 30, 2025, we were notified that funding from the U.S. Government Department of Defense’s Joint Program Executive Office for Chemical, Biological, Radiological and Nuclear Defense (JPEO-CBRND) for the ongoing 300-patient Phase 2 RHB-107 arm of the ACESO PROTECT platform trial for early COVID-19 outpatient treatment was subject to termination, requiring the study to cease enrollment on Feb 28, 2025. 93 patients have been enrolled out of a fully enrolled target patient population of 300. Due to the reduced number of patients enrolled in this study, the study result may not lead to conclusions regarding the efficacy of RHB-107 in this trial.

The U.S. Army-funded Ebola development program remains ongoing, with RHB-107 having demonstrated a robust synergistic effect in vitro when combined with remdesivir. Management of potential Ebola virus pandemic outbreaks represents a significant opportunity and is a key concern for global health agencies.

Annual Report:

A copy of the Company’s annual report on Form 20-F for the year ended December 31, 2024 has been filed with the U.S. Securities and Exchange Commission at View Source and posted on the Company’s investor relations website at:
View Source

Biocon Biologics Announces U.S. FDA Approval for Jobevne™, Biosimilar Bevacizumab, Expanding Its Oncology Portfolio

On April 10, 2025 Biocon Biologics Ltd (BBL), a subsidiary of Biocon Ltd (BSE code: 532523, NSE: BIOCON), reported that the U.S. Food and Drug Administration (U.S. FDA) has approved Jobevne (bevacizumab-nwgd), a biosimilar Bevacizumab for intravenous use (Press release, Biocon, APR 10, 2025, View Source [SID1234651875]). JOBEVNE, a recombinant humanized monoclonal antibody used to treat several different types of cancer, is a biosimilar to the reference product Avastin (bevacizumab). JOBEVNE is a vascular endothelial growth factor (VEGF) inhibitor that binds with VEGF and blocks the interaction with its receptors to prevent angiogenesis – combating cancer by restricting blood supply to the tumor.

The approval of JOBEVNE expands Biocon Biologics’ biosimilar oncology portfolio in the United States, which also includes OGIVRI (Trastuzumab-dkst) and FULPHILA (Pegfilgrastim-jmdb). The Company also markets Bevacizumab in Europe (approved February 2021) and Canada (approved November 2021) under the name ABEVMY.

The U.S. FDA has approved Jobevne (bevacizumab-nwgd) from Biocon Biologics to treat several different types of cancer.

Shreehas Tambe, CEO & Managing Director, Biocon Biologics Ltd., said: "The U.S. FDA approval of JOBEVNE (bevacizumab-nwgd) is a significant milestone—our seventh biosimilar approved in the U.S. and a strong addition to our robust oncology portfolio. It underscores the depth of our scientific expertise and commitment to expanding access to high-quality, affordable biologics. We look forward to working with all stakeholders to bring more treatment options to patients."

In the U.S., sales of bevacizumab were approximately $2.0 billion in 2023.**

Biocon Biologics is a leading global player in biosimilars and insulin production and has achieved many "firsts" in the industry including the first to receive approval of a trastuzumab in the United States, OGIVRI (Trastuzumab-dkst), as well as FULPHILA (Pegfilgrastim-jmdb) and the first U.S. approval of an interchangeable biosimilar for SEMGLEE (insulin glargine). Serving over 5 million patients annually, Biocon Biologics has a comprehensive portfolio of in-market and in-development biosimilar products across multiple therapies, including seven approved biosimilars in the United States and six in Canada, with a robust development pipeline of 20 biosimilar assets, including insulins and monoclonal antibodies spanning multiple therapy areas.

About JOBEVNE:

The approval for JOBEVNE (bevacizumab-nwgd) was based on a comprehensive package of comparative pharmacokinetic, safety, efficacy, nonclinical, structural, analytical and functional data, which confirmed that JOBEVNE is highly similar to Avastin (bevacizumab).

The data demonstrated that there were no clinically meaningful differences between JOBEVNE and Avastin in terms of pharmacokinetics, safety, efficacy, and immunogenicity.

INDICATIONS AND USAGE:

JOBEVNE is a vascular endothelial growth factor inhibitor indicated for the treatment of:

Metastatic colorectal cancer, in combination with intravenous fluorouracil-based chemotherapy for first-or second-line treatment.*
Metastatic colorectal cancer, in combination with fluoropyrimidine-irinotecan-or fluoropyrimidine-oxaliplatin-based chemotherapy for second-line treatment in patients who have progressed on a first-line bevacizumab product-containing regimen.*
Unresectable, locally advanced, recurrent or metastatic non-squamous non-small cell lung cancer, in combination with carboplatin and paclitaxel for first-line treatment.
Recurrent glioblastoma in adults.
Metastatic renal cell carcinoma in combination with interferon alfa.
Persistent, recurrent, or metastatic advanced cervical cancer, in combination with paclitaxel and cisplatin, or paclitaxel and topotecan.
Epithelial ovarian, fallopian tube, or primary peritoneal cancer:
in combination with carboplatin and paclitaxel, followed by JOBEVNE as a single agent, for stage III or IV disease following initial surgical resection
in combination with paclitaxel, pegylated liposomal doxorubicin, or topotecan for platinum-resistant recurrent disease who received no more than 2 prior chemotherapy regimens
in combination with carboplatin and paclitaxel or carboplatin and gemcitabine, followed by JOBEVNE as a single agent, for platinum-sensitive recurrent disease
* Limitations of Use: JOBEVNE is not indicated for adjuvant treatment of colon cancer.

WARNINGS AND PRECAUTIONS:

Gastrointestinal Perforations and Fistula: Discontinue for gastrointestinal perforations, tracheoesophageal fistula, Grade 4 fistula, or fistula formation involving any organ
Surgery and Wound Healing Complications: In patients who experience wound healing complications during JOBEVNE treatment, withhold JOBEVNE until adequate wound healing. Withhold for at least 28 days prior to elective surgery. Do not administer JOBEVNE for at least 28 days following a major surgery, and until adequate wound healing. The safety of resumption of bevacizumab products after resolution of wound healing complication has not been established. Discontinue for wound healing complications of necrotizing fasciitis.
Hemorrhage: Severe or fatal hemorrhages have occurred. Do not administer for recent hemoptysis. Discontinue for Grade 3-4 hemorrhage.
Arterial Thromboembolic Events (ATE): Discontinue for severe ATE.
Venous Thromboembolic Events (VTE): Discontinue for Grade 4 VTE.
Hypertension: Monitor blood pressure and treat hypertension. Withhold until medically controlled; resume once controlled. Discontinue for hypertensive crisis or hypertensive encephalopathy.
Posterior Reversible Encephalopathy Syndrome (PRES): Discontinue.
Renal Injury and Proteinuria: Monitor urine protein. Discontinue for nephrotic syndrome. Withhold until less than 2 grams of protein in urine.
Infusion-Related Reactions: Decrease rate for infusion-related reactions. Discontinue for severe infusion-related reactions and administer medical therapy.
Embryo-Fetal Toxicity: May cause fetal harm. Advise females of potential risk to fetus and need for use of effective contraception.
Ovarian Failure: Advise females of the potential risk.
Congestive Heart Failure (CHF): Discontinue JOBEVNE in patients who develop CHF.
Please refer to full Prescribing Information for Jobevne (bevacizumab-nwgd) for more information. To report SUSPECTED ADVERSE REACTIONS, contact Biocon Biologics at 1-833-986-1468 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

JOBEVNE is a trademark of Biosimilars Newco Limited, a Biocon Biologics Company.
OGIVRI, FULPHILA, SEMGLEE and ABEVMY are registered trademarks of Biosimilars Newco Limited, a Biocon Biologics Company.
BIOCON BIOLOGICS and the Biocon Biologics Logo are registered trademarks of Biocon Biologics Limited.
All other trademarks are the property of their respective owners.
**Sales projections are based on Biocon Biologics’ analysis of IQVIA 2023 data.

Omeros Announces the Omeros Oncology Clinical Steering Committee for AML to Help Guide Omeros’ Proprietary OncotoX-AML Clinical Program

On April 10, 2025 Omeros Corporation (Nasdaq: OMER) reported the establishment of the Omeros Oncology Clinical Steering Committee to advance Omeros’ OncotoX biologics program focused on acute myeloid leukemia (AML) (Press release, Omeros, APR 10, 2025, View Source [SID1234651874]). AML is the most fatal form of leukemia and accounts for approximately 80 percent of acute leukemias in adults and one-third of all cancers affecting the blood/bone marrow, representing a high unmet need. Omeros’ OncotoX program for AML consists of proprietary targeted, engineered molecules (about half the size of an antibody) that deliver a toxic payload within the cancer cells, thereby killing them.

Members of the Omeros Oncology Clinical Steering Committee include:

Naval Daver, M.D., Professor and Director of the Leukemia Research Alliance Program in the Department of Leukemia at MD Anderson Cancer Center (Chair of the Omeros Oncology Clinical Steering Committee)
Aref Al-Kali, M.D., Chair of the Acute Myeloid Group and Research Chair of the Acute Leukemia and Myeloid Neoplasms Disease Group at Mayo Clinic
Jessica K. Altman, M.D., Professor of Medicine in the Division of Hematology and Oncology and Director of the Leukemia Program of Robert H. Lurie Comprehensive Cancer Center at Northwestern University
Jayastu Senapati, M.B.B.S., M.D., D.M., Assistant Professor in the Department of Leukemia at MD Anderson Cancer Center
Mithun Shah, M.D., Ph.D., Assistant Professor of Medicine and Oncology, and Consultant in the Division of Hematology at Mayo Clinic
Anthony S. Stein, M.D., Co-director of the Gehr Family Center for Leukemia Research within the Hematologic Malignancies and Stem Cell Transplantation Institute at City of Hope
Eytan M. Stein, M.D., Chief of the Leukemia Service and Director of the Program for Drug Development in Leukemia in the Division of Hematologic Malignancies at Memorial Sloan Kettering Cancer Center
Roland B. Walter, M.D., Ph.D., M.S., Professor in the Translational Science and Therapeutics Division and José Carreras/E. Donnall Thomas Endowed Chair for Cancer Research at the Fred Hutchinson Cancer Center
The steering committee will, among other activities, assist Omeros with development of the OncotoX-AML program, clinical trial design and interactions with institutional review boards and will participate in OncotoX-AML clinical trials.

"AML is a devastating disease, and these patients have difficulty tolerating the side effects of chemotherapeutic agents and current antibody-drug conjugates, which generally have a narrow therapeutic index," stated Naval Daver, M.D., Professor and Director of the Leukemia Research Alliance Program in the Department of Leukemia at MD Anderson Cancer Center and Chair of the Omeros Oncology Clinical Steering Committee. "The OncotoX platform is designed to kill only dividing cancer cells while sparing normal cells. In animal models using cell lines derived from AML patients, the OncotoX-AML molecule has shown superior efficacy to current standard of care in AML treatment, and its potential utility extends broadly beyond AML to other types of leukemia. Together with my esteemed physician-scientist colleagues on the steering committee, I look forward to advancing the Omeros therapeutic for AML and bringing this novel treatment to patients as quickly as possible."

Across extensive in vivo and ex vivo studies, the OncotoX-AML therapeutic has shown to be highly effective even at very low doses, providing a significant survival benefit over currently approved combination therapy (venetoclax and azacytidine). These studies, conducted with human cell lines in well-established animal models and considered predictive of clinical response, have targeted AML tumors with mutational backgrounds commonly found in nearly 90 percent of AML patients (TP53, NPM1, KMT2a, and FLT3) indicating that the OncotoX-AML therapeutic could be "mutation-agnostic," a currently large unmet need for oncologists and their patients. In studies using primary AML derived from patients, the OncotoX molecule preferentially and efficiently kills AML blasts (abnormal myeloid cells). OncotoX-AML also targets leukemia stem cells (LSC), which are often refractory to chemotherapy and represent another major challenge in the treatment of AML. The OncotoX-AML therapeutic is designed specifically to kill both AML blasts and LSCs that can lead to relapse. Preliminary in vivo tolerability studies demonstrate that the OncotoX therapeutic is well tolerated at doses substantially greater than one order of magnitude above efficacious doses without causing neutropenia or meaningful changes in blood chemistry values.

Omeros is initiating IND-enabling work for its OncotoX-AML therapeutic. Clinical efficacy in AML, considered one of the most aggressive leukemias, is thought to bode well for a therapeutic agent’s broad applicability across leukemias. In addition to OncotoX-AML, Omeros’ portfolio of oncology platforms include signaling and antigen-driven immunomodulators, immune memory-enhancing immunostimulators, and an adoptive T-cell technology that, unlike other cell therapy approaches, does not require cellular engineering.

"We are very pleased to be working with all the members of our oncology steering committee," said Gregory A. Demopulos, M.D., Omeros’ Chairman and Chief Executive Officer. "Each is an internationally recognized leader in the fields of leukemia and AML and has been integrally involved in the development of new treatments for their patients. Their willingness to invest time and effort in our OncotoX-AML program is a strong endorsement of our molecule’s potential and, with their help, we look forward to assessing this next-generation therapeutic in AML patients."

In the U.S. alone, AML is diagnosed in over 20,000 patients annually and is responsible each year for more than 11,000 deaths. In 2030, the global AML therapeutic market size is projected to be over $6 billion with the leukemia therapeutic market forecast at $29 billion.

Protara Therapeutics to Present Interim Analysis from the Phase 2 ADVANCED-2 Trial of TARA-002 in Patients with NMIBC at the American Urological Association Annual Meeting

On April 10, 2025 Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage company developing transformative therapies for the treatment of cancer and rare diseases, reported that two presentations and a panel discussion highlighting the ongoing Phase 2 open-label ADVANCED-2 trial of TARA-002 in patients with non-muscle invasive bladder cancer (NMIBC) will be featured at the upcoming American Urological Association (AUA) 2025 Annual Meeting taking place from April 26, 2025 to April 29, 2025 in Las Vegas (Press release, Protara Therapeutics, APR 10, 2025, View Source [SID1234651873]).

A copy of the abstract for the ADVANCED-2 interim analysis is now available on the AUA website. Updated safety and efficacy data, including data from patients who have reached the 12-month evaluation timepoint, will be featured during an interactive poster session on Saturday, April 26, 2025.

ADVANCED-2 (NCT05951179) is a Phase 2 open-label trial assessing intravesical TARA-002 in NMIBC patients with carcinoma in situ or CIS (± Ta/T1) who are Bacillus Calmette-Guérin (BCG)-unresponsive (n≈100) and BCG-Naïve (n=30). The BCG-Unresponsive cohort has been designed to be registrational in alignment with the U.S. Food and Drug Administration’s 2024 BCG-Unresponsive Non-muscle Invasive Bladder Cancer: Developing Drugs and Biological Products for Treatment, Draft Guidance for Industry.

Presentation Details:

Title: Preliminary Anti-Tumor Activity and Safety Results from ADVANCED-2: A Phase 2 Open-Label Study of Intravesical TARA-002 in Adults with High-Grade Non-Muscle Invasive Bladder Cancer
Session: Bladder Cancer: Non-Invasive I
Presenter: Gautam Jayram, M.D., Director, Advanced Therapeutics Center, Urology Associates P.C., Nashville, TN
Session Date and Time: Saturday, April 26, 2025, 7:00 a.m. – 9:00 a.m. PT
Location: Marco Polo 701
Title: ADVANCED-2: A Phase 2 Open-Label Study to Evaluate the Safety and Efficacy of Intravesical Instillation of TARA-002 in Adults with High-Grade Non-Muscle Invasive Bladder Cancer
Session: Clinical Trials in Progress: Bladder Cancer
Presenter: Brian Mazzarella, M.D., Vice President of Research at Urology America, Austin, TX
Session Date and Time: Monday, April 28, 2025, 9:16 a.m. – 9:24 a.m. PT
Location: Hall C, The Square, Learning Lab
AUA Learning Lab Featured Trials Panel Discussion Details:

Title: ADVANCED-2: A Phase 2 Open-Label Study to Evaluate the Safety and Efficacy of Intravesical Instillation of TARA-002 in Adults with High-Grade Non-Muscle Invasive Bladder Cancer
Moderator: Jacqueline Zummo, Ph.D., Co-Founder, Senior Vice President, Chief Scientific Operations Officer, Protara Therapeutics
Speakers:
Timothy D. Lyon, M.D., Associate Professor of Urology and Urology Residency Program Director at Mayo Clinic, Jacksonville, FL
Brian Mazzarella, M.D., Vice President of Research at Urology America, Austin, TX
Alex Sankin, M.D., MS, Director of Clinical Trials Program, Associate Program Director of Urology Residency, Associate Professor and Attending Physician at Montefiore Medical Center, Bronx, NY
Date and Time: Monday, April 28, 2025, 11:00 a.m. – 11:30 a.m. PT
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 Designation by the U.S. Food and Drug Administration. 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 and also approved in Taiwan. Protara has successfully shown manufacturing comparability between TARA-002 and OK-432.

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 release of cytokines such as tumor necrosis factor (TNF)-alpha, interferon (IFN)-gamma IL-6, IL-10, 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.

About Non-Muscle Invasive Bladder Cancer (NMIBC)

Bladder cancer is the 6th most common cancer in the United States, with NMIBC representing approximately 80% of bladder cancer diagnoses. Approximately 65,000 patients are diagnosed with NMIBC in the United States 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.

Pasithea Therapeutics Announces Positive Safety Review Committee (SRC) Recommendation from its Ongoing Phase 1 Clinical Trial of PAS-004 in Advanced Cancer

On April 10, 2025 Pasithea Therapeutics Corp. (NASDAQ: KTTA) ("Pasithea" or the "Company"), a clinical-stage biotechnology company developing PAS-004, a next-generation macrocyclic MEK inhibitor, for the treatment of neurofibromatosis type 1 (NF1) and other cancer indications, reported that the external Safety Review Committee recommended that the Company’s Phase 1 clinical trial of PAS-004 in advanced cancer should proceed to Cohort 6, 30mg capsule, without modification (Press release, Pasithea Therapeutics, APR 10, 2025, View Source [SID1234651872]). This recommendation was based on the review of the safety data from three patients from Cohort 5 and the absence of any dose limiting toxicities (DLT’s). In addition, no rash has been observed to date during the DLT period in any of the first 19 patients in either capsule (15 patients) or tablet (four patients) formulation of PAS-004. Rash is a common adverse event (AE) that is observed at low doses with competitor MEK inhibitors and may lead to the discontinuation rate in real world practice.

"We are seeing substantial enrollment demand and have already identified Cohort 6 patients. In addition, we continue to observe substantial exposure levels of PAS-004, and remain excited about the possibility of delivering relevant pERK inhibition below the no observed adverse effect levels (NOAEL) as we modeled and observed during our previously conducted nine-month chronic toxicity studies. The on label rash rate for both approved MEKi for NF1 exceeds 80% which leads to patients discontinuing who otherwise should remain on treatment for longer periods of time", stated Dr. Tiago Reis Marques, Chief Executive Officer of Pasithea. "We will provide additional safety, pharmacokinetic (PK) and pharmacodynamic (PD) data over the next several weeks."

The ongoing Phase 1 clinical trial is a multi-center, open-label, dose escalation 3+3 study design to evaluate the safety, tolerability, pharmacokinetic (PK), pharmacodynamic (PD), and preliminary efficacy of PAS-004 in patients with MAPK pathway driven advanced solid tumors with a documented RAS, NF1 or RAF mutation or patients who have failed BRAF/MEK inhibition (NCT06299839).