On December 1, 2021 Zetagen Therapeutics, a private, clinical-stage, biopharmaceutical company dedicated to driving breakthrough innovation in the treatment of metastatic bone cancers and osteologic interventions, reported it has received Breakthrough Device designation from the Centers for Devices and Radiological Health (CDRH) of the U.S. Food and Drug Administration (FDA) for its ZetaMet technology (Press release, Zetagen Therapeutics, DEC 1, 2021, View Source [SID1234596376]). Previously known as ZetaFuse, ZetaMet is a synthetic, small-molecule, inductive biologic technology being developed to target and resolve metastatic bone lesions while inhibiting future tumor growth and regenerating bone.

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"We are pleased to receive this important designation from the Agency and look forward to partnering with them," said Joe C. Loy, CEO of Zetagen Therapeutics. "Our researchers have discovered an entirely new pathway for an established molecule which, if proven successful in human clinical trials, could create a new treatment paradigm for the hundreds of thousands of patients living with cancers that involve metastatic bone lesions."

ZetaMet works through a mechanism of action (MOA) which is a novel and patented molecular pathway. The small molecule, precisely-dosed, delivered to the affected area through a proprietary drug-eluting carrier, stimulates stem cells, activating cells to grow healthy bone known as "osteoblasts", and inhibits cells associated with bone degradation called "osteoclasts". The combination technology has, thus far, in preclinical studies, demonstrated its ability to resolve existing metastatic bone lesions, inhibit pain and stimulate targeted bone regeneration.

Bone metastases are common among cancer patients and occur when cells from the primary cancerous tumor relocate to the bone. When these cancers relocate, they can cause changes to the bone, damaging it in a process called osteolysis. Osteolysis can cause small holes within the bone, weakening it and increasing the risk of breakage. These holes are called "lytic lesions." Among cancers which metastasize to bone, Breast and Prostate are most prevalent, amounting to approximately 70-percent of cases.1

ZetaMet has successfully passed its preclinical trials and is being prepared for its first human clinical trial in early 2022.

On November 30, 2021 Athenex, Inc., (NASDAQ: ATNX), a global biopharmaceutical company dedicated to the discovery, development, and commercialization of novel therapies for the treatment of cancer and related conditions, reported that it will host a virtual key opinion leader (KOL) event on its CAR-NKT technology on Monday, December 13th, at 9:00 am ET (Press release, Athenex, NOV 30, 2021, View Source [SID1234596245]).

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The webinar will feature presentations by KOLs Leonid Metelitsa, M.D., and Carlos Ramos, M.D., both from Baylor College of Medicine, and Sattva Neelapu, M.D., from MD Anderson Cancer Center. Dr. Metelitsa will provide an overview of Athenex’s CAR-NKT cell-based approaches. Dr. Ramos will discuss the interim data from the ongoing ANCHOR study evaluating KUR-502 in relapsed or refractory lymphoma and leukemia. These data will also be featured in a poster presentation at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, being held from December 11th to December 14th. Dr. Neelapu will discuss the current treatment landscape and unmet medical needs in lymphoma/myeloma.

Presentations from Kurt Gunter, M.D., Chief Medical Officer for Cell Therapy at Athenex, and Daniel Lang, M.D. President, Athenex Cell Therapy, will follow. Dr. Gunter will discuss clinical milestones and company objectives.

A question-and-answer session will follow the formal presentations. To register for the webinar, please click here.

Presenting Key Opinion Leaders:

Dr. Carlos Ramos is currently an Associate Professor of Medicine in the Center for Cell and Gene Therapy at the Baylor College of Medicine, and an Attending Physician in the Blood and Marrow Transplantation Service at the Houston Methodist Hospital. As a faculty member, he has focused his research efforts on T-cell therapy for malignancies and viral infections. He has broad experience in cellular therapy for hematologic cancers, being the principal investigator in trials using chimeric antigen receptor (CAR)-modified T cells targeting CD19, CD30 and kappa light chain as therapy for non-Hodgkin and Hodgkin lymphoma and multiple myeloma. He is also implementing cellular therapy approaches for patients with Human Papilloma Virus (HPV)-associated malignancies, using HPV-specific T cells. Dr. Ramos earned his Medical Degree with Honors from the University of Lisbon, Portugal, and was a Resident in Internal Medicine at the Baylor College of Medicine, in Houston. He completed Fellowships in Hematology and Medical Oncology at the Memorial Sloan-Kettering Cancer Center, in New York, and in Blood and Marrow Transplantation at the University of Texas M. D. Anderson Cancer Center, in Houston.

Dr. Leonid Metelitsa is involved in research focused on understanding the role of Vα24-invariant natural killer T cells (iNKTs) in tumor immunity. Using clinical samples from neuroblastoma patients, his lab demonstrated for the first time that iNKTs localize to primary tumors and their presence at the tumor site is associated with good outcome. In the pursuit of the underlying mechanistic basis for the observed association between iNKTs and clinical outcome, Dr. Metelitsa’s group has published a series of high-impact papers that revealed the mechanisms of iNKT cell tumor localization and function in the tumor microenvironment. In one of these studies, they discovered the mechanism by which iNKTs attack tumor-associated macrophages (TAMs). That study has had a major impact in the fields of NKT cell and neuroblastoma research. First, it explained how iNKTs can mediate antitumor activity against CD1d-negative tumors, the majority of solid tumors in humans. Second, it showed for the first time that macrophages infiltrate primary tumors in a subset of neuroblastoma patients, their products directly support tumor growth, and their presence at the tumor site is associated with poor clinical outcome. This discovery has been validated in large international study validated using large independent cohorts of patients, concluding that the presence of M2-like macrophages is associated with a novel inflammatory signature in neuroblastoma that serves as an independent prognostic factor of extremely poor outcome. Most recently, Dr. Metelitsa has been focused on the development of therapeutic applications using iNKTs and their synthetic ligands.

Dr. Sattva Neelapu is professor and deputy chair of Lymphoma and Myeloma at The University of Texas MD Anderson Cancer Center in Houston, Texas. As a physician-scientist at MD Anderson, Dr. Neelapu is focused on clinical and translational development of novel immunotherapies for B-cell malignancies. His laboratory characterized some of the major immunosuppressive mechanisms in the tumor microenvironment of B-cell malignancies, identified TCL1 as a novel shared tumor-associated antigen for B-cell lymphomas, and investigated novel targets for CAR T-cell therapy in lymphoma and myeloma. His work on the pivotal trial of axicabtagene ciloleucel CD19 CAR T-cell therapy in aggressive B-cell lymphomas led to its FDA approval as the first CAR T therapy for lymphoma. Dr. Neelapu has authored or co-authored over 150 publications.

Presenting Athenex Cell Therapy Management:

Dr. Dan Lang has over 25 years of medicine, healthcare investment as well as leadership and business experience. Prior to joining Athenex, Dan was the Chief Investment Officer of the RS Value franchise focused on domestic equity strategies. Dan was on the executive team of RS Investment Management (RSIM) that led the sale of RSIM to Victory Capital in 2016. Prior to joining RSIM in 2009, he was an analyst at Farallon Capital Management covering biotech, medical device, pharma, and healthcare services globally. Previously, he was a senior associate at a venture capital firm, Brilleon Capital and the co-founder and CFO of Sapient Medical Group. Dan’s 25+ years of business and investment experience is preceded by a distinguished career in medicine. He was a post-doctoral research and clinical fellow in cardiology at the University of California, San Francisco and the Gladstone Cardiovascular Research Institute. He was board certified in internal medicine and a Chief Medical Resident at Mount Sinai Hospital in New York. Dan holds a BA in Chemistry from Cornell University and an MD from Cornell University Medical College.

Dr. Kurt Gunter has devoted his career to the development of cell and gene therapies and brings significant experience to Athenex from his previous positions at Kuur Therapeutics, Hospira, ViaCell, and Transkaryotic Therapies. As past President of the International Society for Cellular Therapy (ISCT), Dr. Gunter played a worldwide leadership role in promoting understanding of the clinical, regulatory, manufacturing, and marketing requirements for the successful development of cell and gene therapies. Prior to his biotech career, he worked at the United States Food and Drug Administration as a Medical Officer in the Center for Biologics and was appointed Acting Deputy Director of the Division of Cell and Gene Therapy within the Center for Biologics Evaluation and Research. He also served for 5 years on FDA’s Cellular, Tissue and Gene Therapies Advisory Committee. He earned his MD from the University of Kansas and also has a BS in biological sciences, with distinction, from Stanford University. His postdoctoral training included Johns Hopkins and the US National Institutes of Health.

ASH Poster Presentation:

Session Name: 704. Cellular Immunotherapies: Clinical: Poster II
Date: Sunday, December 12, 2021
Presentation Time: 6:00 PM – 8:00 PM
Location: Georgia World Congress Center, Hall B5

About the Phase I Study of KUR-502 (Allogeneic CD19 CAR-NKT Cells) in Patients with Relapsed or Refractory B-Cell Malignancies (ANCHOR)

The phase I study is an open-label, dose-escalation study. NKT cells were isolated from the leukapheresis product of one HLA-unmatched healthy individual, transduced with the CAR, expanded ex vivo for 14 days (99.8% NKT purity), and cryopreserved. Patients received 107 (DL 1) or 3×107 (DL 2) CAR-NKT cells per square meter of body surface area following lymphodepleting conditioning with cyclophosphamide/fludarabine. Adverse events were evaluated per NCI criteria. When accessible, patients underwent core biopsies of an involved site at 2-5 weeks post-infusion. Response to therapy was assessed at 4 weeks per Lugano Criteria (for NHL) or NCCN guidelines (for ALL).

For further information about the study, visit ClinicalTrials.gov, identifier: NCT03774654.

On November 30, 2021 Merck (NYSE: MRK), known as MSD outside the United States and Canada, reported that the Board of Directors has declared a quarterly dividend of $0.69 per share of the company’s common stock for the first quarter of 2022 (Press release, Merck & Co, NOV 30, 2021, View Source [SID1234596264]). Payment will be made on Jan. 7, 2022 to shareholders of record at the close of business on Dec. 15, 2021.

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On November 30, 2021 MANA Therapeutics, a clinical-stage company creating nonengineered, off-the-shelf allogeneic cell therapies that target multiple cancer antigens, reported the final closing of its Series A financing, bringing the total raised to $42 million (Press release, MANA Therapeutics, NOV 30, 2021, View Source [SID1234596284]). In addition, MANA closed a $7.5 million venture debt facility with Silicon Valley Bank with an initial tranche of $5 million available immediately.

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"This additional funding will enable us to accelerate the start of our clinical studies for our novel approach to off-the-shelf allogeneic cell therapy for patients with transplant-ineligible AML and select solid tumors," said Martin Silverstein, M.D., President and CEO of MANA Therapeutics. "It will also permit building out our pipeline of MANA-512 candidates, which will apply new sets of antigens to the EDIFY platform.

"In the meantime, MANA-312, an allogeneic donor-derived cell therapy candidate, has progressed steadily through the Phase 1 clinical trial that we initiated earlier this year. This is encouraging progress and the data we are collecting will inform the development of our off-the-shelf programs, including MANA-412, which is expected to advance into clinical trials in 2022," continued Dr. Silverstein.

"Next-generation cell therapies continue to be one of the most promising approaches in immuno-oncology, but face meaningful obstacles to be safely and economically available in an allogeneic format. MANA is utilizing its proprietary ManaMatch HLA matching protocol to build a bank of readily accessible nonengineered off-the-shelf therapies that could treat the majority of patients in targeted cancer indications and allow for repeat dosing," said Marc Cohen, Co-founder of Cobro Ventures and Co-founder and Executive Chairman of MANA Therapeutics.

The additional Series A funding comes from new investors J&W Partners, a South Korean private equity firm, and SK Securities, a leading securities firm in South Korea. As part of the agreement, J&W Partners will gain a seat on MANA’s Board of Directors.

About EDIFY

MANA Therapeutics’ EDIFY platform constitutes the next-generation cell therapy approach by leveraging natural immune system pathways to educate T-cells to target unique sets of antigens expressed by tumors, without the need for genetic modification. The EDIFY platform uses dendritic cells as antigen presenting cells. The dendritic cells are loaded with ManaMix antigens and stimulate and expand T-cells to generate product candidates. Product candidates developed from the EDIFY platform are designed to increase efficacy through multiple antigen targeting and to support a strong safety profile by utilizing a nonengineered approach that could permit for repeat dosing of patients. The EDIFY platform uses a simplified, high-yield manufacturing process.

On November 30, 2021 RedHill Biopharma Ltd. (Nasdaq: RDHL) ("RedHill" or the "Company"), a specialty biopharmaceutical company, reported its financial results and operational highlights for the third quarter ended September 30, 2021 (Press release, RedHill Biopharma, NOV 30, 2021, View Source [SID1234596301]).

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Dror Ben-Asher, RedHill’s Chief Executive Officer, said: "Our U.S. commercial business continues to drive growth, delivering a second consecutive quarterly net revenue record of $21.6 million despite the continuously challenging pandemic environment. Talicia generated another record quarter with 15% growth in new prescriptions, while Movantik continues to perform adding a 1.1% increase to new prescriptions. Both products are also continuing to make strides in gaining both commercial and government formulary coverage. In addition, gross margin increased from 51% in the second quarter to 57% in the third quarter. The Company has successfully attracted a strategic investment from South Korea’s Kukbo and continues to demonstrate responsible financial discipline across the entire business as we strive to achieve our long-term growth aims."

Mr. Ben-Asher added: "Given the recent emergence of the heavily mutated Omicron variant as well as likely emergence of other variants over time, the importance of drug candidates that act independently of the viral spike protein is growing. This makes both opaganib and RHB-107’s host-targeted mechanism of action, and expected maintenance of effect against new variants, increasingly more relevant in the battle against COVID-19. This quarter saw significant focus on our opaganib Phase 2/3 COVID-19 study. The initial top-line results demanded further investigation and our rigorous post-hoc analysis provided much greater clarity into the potential of novel, orally-administered opaganib in the underserved hospitalized moderately severe patient group. This is a group of patients for which no novel therapeutic pill has shown a benefit until opaganib, which demonstrated a 62% reduction in mortality, improved return to room air and earlier hospital discharge for opaganib-treated patients. The results of this analysis, in a group of more than half the total study population, were consistent with what we had seen in our Phase 2 study and compassionate use experience. Despite being a post-hoc analysis, the consistency across multiple endpoints and territories provides us with a high degree of confidence in the results showing opaganib’s effect in this patient population. This analysis also shed light on key issues of the COVID-19 disease severity classification, suggesting that FiO2 might be an improved method for classifying disease severity and predictor of treatment outcome. We have now provided regulators in various countries with all the necessary data to facilitate discussions on the next steps and we continue to provide the data to regulators in additional countries."

"In parallel, we continue to progress our Phase 2/3 study in the U.S. and South Africa with our other novel, once-daily, oral COVID-19 antiviral drug candidate, RHB-107, which has now completed enrollment for Part A of the study, with top-line results expected in the first quarter of 2022. Our Phase 3 study of RHB-204 in pulmonary nontuberculous mycobacteria (NTM) disease continues to enroll patients in the U.S. and progress with Phase 3-stage RHB-104 for Crohn’s disease is expected to speed up thanks to recent, much-awaited, potential progress in Mycobacterium avium subspecies paratuberculosis (MAP) detection research[2]."

"With a steep reduction in quarterly operating and net loss and continued commercial business growth, leading to a potential commercial operational breakeven before the end of the year, coupled with advanced, exciting and timely R&D pipeline progress, I believe RedHill is well positioned for short, medium and long-term success."

Financial highlights for the quarter ended September 30, 2021[3]

Net Revenues were $21.6 million for the third quarter of 2021, as compared with $21.5 million for the second quarter of 2021. The increase is attributable to an increase in sales of Talicia and Movantik, partially offset by an increase in gross-to-net deductions, mainly commercial rebates and Medicare discounts.

Gross Profit was $12.4 million for the third quarter of 2021, compared to $10.9 million for the second quarter of 2021 – an increase of 14%. Gross margin increased from 51% in the second quarter of 2021 to 57% in the third quarter of 2021. The increase in gross profit was mainly attributable to a reversal of inventory write-off recognized in the third quarter of 2021 following the FDA approval of an extension to Talicia stock expiration date.

Research and Development Expenses were $5.8 million for the third quarter of 2021, a decrease of $4.5 million, a 44% reduction compared to the second quarter of 2021, mainly attributable to the completion of our global COVID-19 Phase 2/3 study with opaganib.

Selling, Marketing and General and Administrative Expenses were $24 million for the third quarter of 2021, a decrease of $1.5 million compared to the second quarter of 2021. The decrease was mainly attributable to expenses related to share-based compensation in the previous quarter.

Operating Loss and Net Loss were $17.4 million and $21.4 million, respectively, for the third quarter of 2021 compared to $24.9 million and $29.1 million, respectively, in the second quarter of 2021. The decrease was mainly attributable to the Talicia inventory expiration date extension, completion of our opaganib Phase 2/3 COVID-19 study and a decrease in expenses related to share-based compensation, as detailed above.

Net Cash Used in Operating Activities was $19 million for the third quarter of 2021, similar to the second quarter of 2021.

Net Cash Used in Financing Activities was $1 million for the third quarter of 2021, comprised primarily of payables with respect to the Movantik acquisition, partially offset by proceeds from utilization of ATM and from exercise of options.

Cash Balance1 as of September 30, 2021, was $51.5 million.

Additional Financial Highlights

In November 2021, the Company announced that it had entered into a strategic agreement with Kukbo Co. Ltd., a South Korean corporation, for the sale of RedHill’s American Depositary Shares (ADSs) in a private placement of up to $10 million, of which the first tranche of $5 million has been paid. As part of the agreement, the Company granted Kukbo a six month right of first offer for a license with respect to one or more of opaganib, RHB-107 (upamostat) and Talicia for South Korea and other Asian territories.

In addition, this month, the Company completed an underwritten public offering of approximately 4.7 million ADSs for gross proceeds of approximately $15.5 million, led by Cantor Fitzgerald.

Commercial Highlights

Movantik (naloxegol)[4]

The Company’s focus on driving Movantik performance and strengthening of market share continues unabated, resulting in another quarter of new prescription growth, increasing by 1.1% compared to the previous quarter.

The Company has achieved significant market access successes with U.S. major payors, continuing to increase the levels of payor coverage.

In July, one of America’s largest payors, serving many Blue Cross and Blue Shield Plans and more than 30 million members, had added Movantik as a preferred brand with no restrictions to its Commercial NetResults "A" series formularies and as a preferred brand on its other commercial formularies starting July 1, 2021. In April, Movantik was also included on the Part D formulary of another major payor with no restrictions. Almost 9 out of 10 U.S. commercial lives are now covered, and we continue to work toward additional formulary coverage for the remaining patients.

In September 2021 RedHill Biopharma Inc., AstraZeneca AB, AstraZeneca Pharmaceuticals LP and Nektar Therapeutics entered into a settlement and license agreement with Aurobindo Pharma USA, Inc. resolving their patent litigation in the U.S. in response to Aurobindo’s Abbreviated New Drug Application (ANDA) seeking approval by the FDA to market a generic version of Movantik. This follows the previously announced resolution of the Apotex litigation and brings to a close all presently pending Movantik patent litigation brought pursuant to The Drug Price Competition and Patent Term Restoration Act (the Hatch-Waxman Act). The earliest licensed entry date of any generic naloxegol in the U.S. is October 1, 2030.

Talicia (omeprazole magnesium, amoxicillin and rifabutin)[5]
Talicia achieved another record quarter, delivering a 15% increase in new prescriptions, compared to the previous quarter, reflecting 117% growth of Talicia as compared to Q3/2020.

In October, Medi-Cal – California’s Medicaid Health Care program covering two million beneficiaries – had added Talicia to its Contract Drug List (CDL) for H. pylori treatment, with no prior authorization required. This coverage is expected to expand to 14 million beneficiaries on January 1, 2022. During the same month, a new U.S. Patent covering Talicia was granted. This patent reinforces the protection for Talicia through 2034, and the Company has listed this patent in the FDA’s Approved Drug Products with Therapeutic Equivalence Evaluations, or Orange Book.

In July, the Company significantly expanded commercial coverage for Talicia, announcing that OptumRx, part of the UnitedHealth Group, a leader in healthcare coverage, partnered with more than 1.3 million healthcare professionals and 6,500 hospitals, had added Talicia to its Commercial Formulary as an unrestricted brand for H. pylori treatment, effective July 1, 2021. This agreement expanded access to Talicia to 26 million additional Americans and increased overall patient access to Talicia to greater than 8 out of 10 covered U.S. Commercial lives.

Aemcolo (rifamycin)[6]
The Company has maintained promotion of Aemcolo in the third quarter of 2021 supporting the initial momentum that Aemcolo was generating pre-COVID-19 travel restrictions. RedHill and Cosmo Pharmaceuticals N.V are currently in discussions with respect to the amendment of the Aemcolo License Agreement.

R&D Highlights

COVID-19 Program: Opaganib (ABC294640)[7]
In September 2021, the Company announced top-line results from the global 475-patient Phase 2/3 study in hospitalized patients with severe COVID-19 pneumonia (NCT04467840). Whilst results showed consistent trends in favor of the opaganib arm, the study endpoints did not achieve statistical significance.

A post-hoc analysis of data from 251 study participants requiring a Fraction of inspired Oxygen (FiO2) up to 60% at baseline (54% of the study participants), was subsequently reported in October 2021, demonstrating that treatment with oral opaganib resulted in a 62% reduction in mortality as well as improved outcomes in time to room air and median time to hospital discharge, in this large group of hospitalized, moderately severe COVID-19 patients.

The results provide a strong rationale for opaganib’s potential efficacy in hospitalized patients in need of oxygen supplementation up to 60% FiO2, a large proportion of hospitalized COVID-19 patients. The Phase 2/3 study results are also consistent with opaganib’s earlier U.S Phase 2 study results and the demonstrated potent antiviral inhibition of SARS-CoV-2 variants in human bronchial epithelial cells, providing further support for its potential in earlier stages of disease where viral load is higher.

Additional new preclinical results demonstrating opaganib’s efficacy in significantly decreasing renal fibrosis in a unilateral ureteral obstruction-induced renal interstitial fibrosis mode were also reported by the Company in September 2021.

The Company has submitted data packages for opaganib to the regulatory agencies in the U.S., EU, UK and other territories, ahead of planned regulatory advice, with the European Medicines Agency (EMA) having provided expedited evaluation process timelines. As previously stated, additional studies to support the potential of such applications and the use or marketing of opaganib are likely to be required. For example, the FDA has previously indicated that we will need to complete additional studies to support applications in the U.S. The strength of the safety and efficacy data generated from the opaganib studies will be key to regulatory applications.

The Company also continues its discussions with U.S. and other government agencies and non-governmental organizations around potential funding to support the ongoing development of opaganib. Discussions are also ongoing with potential partners who are interested in the rights to opaganib in various territories.

COVID-19 Program: RHB-107 (upamostat)[8]
RedHill continues to advance the Phase 2/3 study of novel, once-daily, orally-administered, antiviral drug candidate, RHB-107, in the treatment of non-hospitalized patients with symptomatic COVID-19 in the early course of the disease who do not require supplemental oxygen – the vast majority of COVID-19 patients. The study plans to test for the Omicron variant.

Further to announcing in September 2021 that South Africa had joined the U.S. in approving the Phase 2/3 study, along with the expansion to additional U.S. sites, the Company announced this month that the last patient had been enrolled in Part A of the Phase 2/3 study. The study is a 2-part trial designed to evaluate time to sustained recovery from illness as the primary endpoint and for dose selection. A total of 61 patients have been enrolled in Part A and based on safety and tolerability results of part A, a dose for part B will be selected. Top-line results from Part A of the study are expected in the first quarter of 2022, with Part B of the study expected to follow subsequent to discussions with regulators.

RHB-204 – Pulmonary Nontuberculous Mycobacteria (NTM) Disease
A U.S. Phase 3 study is ongoing to evaluate the efficacy and safety of RHB-204 in adults with pulmonary NTM disease caused by Mycobacterium avium Complex (MAC) infection. The Company is also assessing potential expansion of the RHB-204 Phase 3 study to additional territories.

The Company previously announced that the FDA granted Fast Track designation for RHB-204, providing early and frequent communications and a rolling review of any New Drug Application (NDA). RHB-204 is also eligible for NDA Priority Review and Accelerated Approval.

RHB-204 was granted FDA Orphan Drug designation and Qualified Infectious Disease Product designation, extending its U.S. market exclusivity to a potential total of 12 years upon potential FDA approval.

RHB-104 – Crohn’s Disease
Based on recent published research, potential progress in Mycobacterium avium subspecies paratuberculosis (MAP) diagnostic technology may enable us to advance the program towards a confirmatory study in approximately 150 MAP positive moderate-severe Crohn’s patients, subject to required regulatory input.

Opaganib – Prostate Cancer and Cholangiocarcinoma
In August 2021, we announced that, based on a preliminary review of partial and unaudited data, the ongoing Phase 2 study for prostate cancer had met its primary endpoint of at least six subjects demonstrating disease control (defined as stable disease or better after 16 weeks on treatment) among at least 27 evaluable subjects. Upon further review and analysis of the unaudited data, the Company reported that the study did not meet its primary endpoint in the study arm evaluating opaganib in combination with enzalutamide. Patient enrolment continues for the study’s other arm, evaluating a combination of opaganib and abiraterone. Accrual and data entry are ongoing and results for the study remain subject to further review and analysis.

The Phase 2a study evaluating the activity of opaganib in advanced cholangiocarcinoma (bile duct cancer) is ongoing at Mayo Clinics in Arizona and Minnesota, Emory University and the Huntsman Cancer Institute at the University of Utah. Enrollment has been completed for the first cohort of 39 patients, evaluating the activity of orally-administered opaganib as a stand-alone treatment. Preliminary data from this cohort indicated a signal of activity in a number of subjects with advanced cholangiocarcinoma. Enrollment is ongoing for a second cohort, evaluating opaganib in combination with hydroxychloroquine, an anti-autophagy agent.

Conference Call and Webcast Information:

The Company will host a webcast today, Tuesday, November 30, 2021, at 8:30 a.m. EST, during which it will present key highlights for the third quarter of 2021.

The webcast including slides will be broadcast live on the Company’s website, View Source, and will be available for replay for 30 days.