On March 18, 2021 Applied Therapeutics, Inc. (Nasdaq: APLT), a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need, reported financial results for the fourth quarter and full year ended December 31, 2020 (Press release, Applied Therapeutics, MAR 18, 2021, View Source [SID1234576862]).

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"The fourth quarter was a productive period of internal planning and execution," said Shoshana Shendelman, PhD, Founder, CEO and Chair of the Board of Applied Therapeutics. "We are excited for what lies ahead in 2021, where our focus will be on advancing our late stage assets toward commercialization while initiating clinical development of our two new rare disease programs."

Recent Highlights

Announced Restart of Pediatric Galactosemia Study. In February 2021, the Company announced that the FDA lifted the clinical hold on the AT-007 ACTION-Galactosemia Kids pediatric clinical study, and the study resumed immediately. Applied Therapeutics worked closely with FDA to modify the trial, with the shared goal of ensuring that all patients have the opportunity to receive clinical benefit, and remains on target to submit an NDA no later than Q3 2021.

Announced the Launch of a Galactosemia Awareness and Education Initiative. Galactosemia Together is the first and only industry-led Galactosemia awareness and education campaign. Developed in partnership with the Galactosemia community, this initiative aims to address gaps in education by providing updated, reliable and credible resources to help connect, educate and support those families impacted by this disease.

Announced Magnetic Resonance Spectroscopy (MRS) Data from ACTION-Galactosemia Study. In December 2020, the Company shared MRS data on reduction of galactitol levels in the brain of Galactosemia patients treated with AT-007 in the ACTION-Galactosemia adult study. Overall, plasma reduction in galactitol correlated with brain reduction in galactitol. At the two doses which demonstrated statistically significant reduction in plasma galactitol, 20 and 40mg/kg, 3 out of 4 patients displayed substantial galactitol reduction ranging from 61.94% to 69.80% reduction from baseline.

Closed Public Offering of 3,000,000 Shares of Common Stock at a Price to the Public of $23.00 Per Share. In February 2021, the Company completed an underwritten public offering of 3,450,000 shares of common stock, including the exercise in full of the underwriters’ option to purchase 450,000 additional shares of common stock at a price of $23.00 per share, resulting in aggregate net proceeds of approximately $74.3 million.
Financial Results

Cash and cash equivalents and short-term investments totaled $96.8 million as of December 31, 2020, compared with $38.9 million at December 31, 2019. This does not include approximately $74.3 million in aggregate net proceeds the Company received from the underwritten public offering of common stock in February 2021.

Research and development expenses for the year ended December 31, 2020 were $61.8 million, compared to $32.4 million for the year ended December 31, 2019. The increase of $29.4 million related to an increase in clinical and pre-clinical expense of $11.3 million, primarily related to the progression of the AT-007 ACTION-Galactosemia adult extension and the AT-001 Phase 3 ARISE-HF clinical studies, as well as the commencement and progression of the AT-007 ACTION-Galactosemia Kids pediatric registrational study; an increase in drug manufacturing and formulation expenses of $15.4 million primarily related to the commencement of the 2020 manufacturing campaigns and the associated raw material deliveries and AT-007 and AT-001 drug product batch releases; an increase in personnel expenses of $0.6 million due to the increase in headcount in support of our clinical program pipeline; a decrease in stock-based compensation of $0.2 million due to the stock option modification expense recognized during the year ended December 31, 2019 for the acceleration of certain options vesting following the IPO; offset by an increase in expense recognized during the year ended December 31, 2020 due to new stock option and restricted stock grants; and an increase of regulatory and other expenses of $2.3 million primarily related to University of Miami license fees and increased clinical consulting fees recognized during the year ended December 31, 2020.

General and administrative expenses were $32.7 million for the year ended December 31, 2020, compared to $13.2 million for the year ended December 31, 2019. The increase of $19.4 million was an increase in professional and legal fees of $4.1 million due to increased operations and costs associated with being a public company for a full year; an increase of $5.7 million related to the establishment of a commercial department; an increase in personnel expenses of $4.2 million and an increase in stock-based compensation of $2.0 million due to an increase in headcount; an increase of insurance expenses of $1.8 million related to increased D&O insurance costs; and an increase in other expenses of $1.6 million, primarily relating to increased costs of rent and other office expenses.

Net loss for the year ended December 31, 2020 was $94.0 million, or $4.28 per basic and diluted common share, compared to a net loss of $45.5 million, or $3.55 per basic and diluted common share, for the year ended December 31, 2019.

On March 18, 2021 Alpine Immune Sciences, Inc. (NASDAQ: ALPN), a leading clinical-stage immunotherapy company focused on developing innovative treatments for cancer and autoimmune/inflammatory diseases, reported financial results for the fourth quarter and year ended December 31, 2020 (Press release, Alpine Immune Sciences, MAR 18, 2021, View Source [SID1234576861]).

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"This past year has been transformative for the company. We are building on our strong momentum to accelerate development of innovative therapies for patients living with cancer and autoimmune/inflammatory disease," said Mitchell H. Gold, M.D., Executive Chairman and Chief Executive Officer of Alpine. "Over the next year we will continue to execute on our clinical-stage programs and plan to initiate an international Phase 2 trial of ALPN-101 in adults with systemic lupus erythematosus (SLE), and further progress our ongoing NEON-1 Phase 1 study of ALPN-202 in advanced malignancies. We also plan to progress key pipeline programs, including the initiation of a Phase 1 healthy volunteer study for ALPN-303. In summary, I believe 2021 will be another year of significant opportunity for Alpine as we advance our pipeline of innovative therapeutic candidates."

Full Year 2020 and Recent Corporate and Clinical Updates

ALPN-101: Dual CD28/ICOS inhibitor
Announced Option and License Agreement with AbbVie: In June 2020, Alpine announced an exclusive worldwide option and license agreement for ALPN-101. Under the terms of the agreement, Alpine received an upfront payment of $60 million and is eligible to receive up to an aggregate of $805 million for exercise of the option and success-based development, regulatory, and commercial milestones, including up to $75 million in pre-option development milestones. In addition, Alpine is eligible to receive tiered royalties on net sales of ALPN-101.
Preparation for a global Phase 2 study in SLE is ongoing with an intent to initiate the study in mid-2021.
ALPN-202: Conditional CD28 costimulator and dual checkpoint inhibitor
Presentation of NEON-1 trial design at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) Virtual Annual Meeting 2020: The design of NEON-1, our first-in-class conditional CD28 costimulator and dual checkpoint inhibitor, was presented at the AACR (Free AACR Whitepaper) Virtual Annual Meeting I, in the Phase I Trials in Progress Virtual Poster Session.
Enrollment in NEON-1, a first in human monotherapy Phase 1 clinical trial, continues to progress through its dose escalation cohorts. Alpine plans to share interim data at an appropriate scientific forum and to determine expansion cohorts later this year.
Alpine is also targeting the initiation of NEON-2, a Phase 1 combination study of ALPN-202 and a PD-1 inhibitor, later this year.
ALPN-303: Dual APRIL/BAFF inhibitor
Presentation of Preclinical Data on Novel B cell Modulatory Domains, Alone or in Combination with Novel Domains that Inhibit T Cell Costimulation, for Autoimmune Diseases: In June 2020, Alpine presented a poster at the European Congress of Rheumatology (EULAR) E-Congress detailing preclinical work with novel B cell modulatory variant TNF Receptor domains (vTDs), alone or in combination with variant Ig Domains (vIgDtm) that inhibit T cell costimulation. This work included ALPN-303, a novel dual BAFF/APRIL inhibitor, in multiple preclinical models, including the NZB/NZW F1 murine lupus model.
Alpine is targeting completion of activities to support initiation of a Phase 1 healthy volunteer study with ALPN-303 in the fourth quarter of this year.
General Corporate
Raised $60 Million in a Private Placement: In July 2020, Alpine raised $60 million in gross proceeds through a private placement led by Omega Funds with participation from Avidity Partners, EcoR1 Capital, LLC, Invus Public Equities, L.P., and Samsara BioCapital, among others.
Appointed industry veteran Natasha Hernday to the Board of Directors: Ms. Hernday brings more than 20 years of experience in corporate development and corporate strategy, and currently serves as Executive Vice President, Corporate Development and as a member of the Executive Committee for Seagen, Inc.
Promotion of Remy Durand, Ph.D. to Chief Business Officer: Dr. Durand joined Alpine Immune Sciences in 2018 as Vice President, Business Development, and has played a leading role in building the company’s partnerships with AbbVie and Adaptimmune, and has represented the company at investor meetings and conferences.
Appointed Pamela Holland, Ph.D. as Senior Vice President, Research: Dr. Holland is an experienced cancer biologist with a proven track record of successfully discovering and progressing multiple preclinical therapeutics into clinical development, most recently at Surface Oncology and Amgen.
Full Year 2020 Financial Results

As of December 31, 2020, we had cash, cash equivalents, and investments totaling $131.4 million. Net cash provided by operating activities for the year ended December 31, 2020 was $30.1 million compared to net cash used in operating activities of $35.3 million for the year ended December 31, 2019. We recorded net losses of $27.9 million and $41.9 million for the years ended December 31, 2020 and 2019, respectively.

Collaboration revenue for the year ended December 31, 2020 was $9.3 million compared to $1.7 million for the year ended December 31, 2019. The increase was primarily attributable to the revenue recognized under our AbbVie Agreement.

Research and development expenses for the year ended December 31, 2020 were $27.2 million compared to $35.8 million for the year ended December 31, 2019. The decrease was primarily attributable to decreases in contract manufacturing and process development of our product candidates and other direct research activities. The decreases were partially offset by increases in clinical trial activity for NEON-1.

General and administrative expenses for the year ended December 31, 2020 were $10.9 million compared to $9.5 million for the year ended December 31, 2019. The increase was primarily attributable to increases in professional and legal services, stock-based compensation and insurance and facility costs to support the growth and expansion of our business.

Alpine expects that its current cash resources, combined with the potential $75 million in pre-option exercise milestones payable under its option and license agreement with AbbVie, for the development and commercialization of ALPN-101, are sufficient to fund Alpine’s planned operations through 2023.

Fourth Quarter and Full Year 2020 Conference Call and Webcast Details

Alpine will host a conference call and live webcast to discuss the fourth quarter and full year performance today, March 18, 2021 at 4:30 p.m. ET/1:30 p.m. PT.

To access the live call by phone, dial (800) 816-3005 (domestic) or (857) 770-0069 (international) and reference conference ID: 2539338. A live webcast of the presentation will be available online in the investor relations section of the company’s website at View Source A replay of the presentation will be available on the company website for 90 days following the webcast.

On March 18, 2021 Alligator Bioscience (Nasdaq Stockholm: ATORX) reported that the Board of Directors has appointed Søren Bregenholt, Ph.D. as the company’s new Chief Executive Officer (CEO), to strengthen Alligator’s business development activities and clinical progress on an international level (Press release, Alligator Bioscience, MAR 18, 2021, View Source [SID1234576860]). Dr. Søren Bregenholt will take up the position on 1 June 2021, succeeding Per Norlén, who will remain in the company. In order to facilitate the transition, Malin Carlsson, Alligator’s COO, is appointed as interim CEO.

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"We’re delighted to have Søren onboard. In line with Alligator’s strategy and increased focus on business development and its clinical assets, Alligator is now entering a phase where it will be commercially crucial to further develop and leverage our international network. We are convinced that Søren’s broad global network across pharma, biotech, venture capital and academia, together with his extensive deal experience from both small and large companies, will be invaluable for Alligator as the company continues to assess favorable partnership and out-licensing opportunities, activities which are highly prioritized for Alligator", said Peter Benson, Chairman of Alligator’s Board of Directors.

Søren Bregenholt has more than 20 years of international experience from operational and strategic leadership positions in global pharma and the biotech industry. He most recently served as CEO and Board Director at Macrophage Pharma Ltd in the UK. Previous experiences include Corporate Vice President positions at Novo Nordisk A/S between 2010 and 2018, and COO at Symphogen A/S where he worked from 2002 to 2010. During his career Søren Bregenholt has negotiated and operationalized numerous licensing, collaboration and co-development agreements. Søren received his PhD from University of Copenhagen and did his post-doctoral training at Institute Pasteur in Paris. He is the author and co-author of more than 45 papers in peer reviewed scientific journals. "I am really looking forward to the new challenge of leading Alligator Bioscience, a very dynamic and respected company in the exciting field of immuno-oncology. I am impressed by the robust quality of the clinical portfolio as well as the research and development capabilities that has brought the company to this stage. It will be truly exciting to lead and work closely with the competent team at Alligator and take the company further towards clinical and commercial success", said Søren Bregenholt.

This information is information that Alligator Bioscience AB is obliged to make public pursuant to the EU Market Abuse Regulation. The information was submitted for publication, through the agency of the contact person set out above, at 7:00 p.m. CET on March 18, 2021.

On March 18, 2021 VAXIMM AG, a Swiss/German biotech company focused on developing an oral plug and play DNA vaccination technology to stimulate patients’ cytotoxic T-cells targeting a wide range of cancer-related antigens, reported that the Company will participate in several scientific and industry events in the coming weeks (Press release, Vaximm, MAR 18, 2021, View Source [SID1234576853]). All conferences will be held virtually.

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15th Annual International Partnering Conference BIO-Europe Spring
March 22-25, 2021
Dr. Heinz Lubenau, Chief Executive Officer and Co-Founder, will give a company presentation and participate in the online partnering system. To request a meeting, please sign up through the event’s partneringONE meeting system.

European Biotech Investor Days 2021
April 7-8, 2021
Dr. Lubenau will give a company presentation at 5pm CEST, 7 April, and be available for one-on-one meetings.

American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting
April 10-15 and May 17-21, 2021
Dr. Lubenau will attend the conference. To schedule a meeting with the Company, please contact info@vaximm.com.

Alliance for Cancer Gene Therapy Summit 2021
April 29, 2021
Dr. Lubenau will attend the conference. To schedule a meeting with the Company, please contact info@vaximm.com.

Upcoming events in which VAXIMM will participate are updated on a regular basis. See the Events section of the Company’s website for more information.

On March 18, 2021 RedHill Biopharma reported Fourth Quarter and Full Year 2020 Financial Results and Operational Highlights (Press release, RedHill Biopharma, MAR 18, 2021, View Source [SID1234576852])

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Financials:

Full year 2020 net revenues of approximately $64 million, with gross profit of approximately $27.5 million

Solid cash balance of approximately $100 million as of March 4, 2021

Planned commercial operational breakeven by the end of 2021

Commercial Highlights:

Talicia: Consistent month-over-month prescription growth despite pandemic conditions, with 52% new prescription growth in Q4/2020 compared to Q3/2020

Movantik: Market leadership position holding strong and well-positioned for further growth in 2021

R&D Highlights:

Two advanced programs at the forefront of global COVID-19 novel therapeutics development:

Opaganib: Ongoing global Phase 2/3 study in hospitalized patients approximately two thirds enrolled, data expected Q2/2021; Positive U.S. Phase 2 data reported

RHB-107: Ongoing U.S. Phase 2/3 study in symptomatic non-hospitalized patients
RHB-204: Ongoing Phase 3 study for pulmonary NTM disease as oral first-line treatment

Management to host webcast today, at 8:30 a.m. EDT

RedHill Biopharma Ltd. (Nasdaq: RDHL) ("RedHill" or the "Company"), a specialty biopharmaceutical company, today reported its financial results and operational highlights for the year ended December 31, 2020.

Dror Ben-Asher, RedHill’s Chief Executive Officer, said: "2020 was a year that our team looks back on with a sense of immense achievement. While navigating the challenging conditions caused by the pandemic, we have delivered broad commercial growth culminating in a very strong end to 2020 for both Movantik and Talicia". Mr. Ben-Asher continued: "At the same time, we have rapidly progressed two novel oral COVID-19 therapies to Phase 3 stage development, covering both hospitalized and non-hospitalized patients and have reported positive clinical and preclinical data, positioning RedHill at the forefront of novel COVID-19 therapeutics development worldwide. With strong momentum across both commercial and R&D operations, we expect 2021 to be a breakout year."

Micha Ben Chorin, Chief Financial Officer at RedHill, added: "RedHill is delivering on a clear strategy designed to enable us to achieve fast growth and increased profit margin. We have been diligent in maintaining a solid balance sheet and we expect to achieve commercial operational breakeven by the end of this year."

Financial highlights for the year ended December 31, 2020

Net Revenues were approximately $64 million for the year ended December 31, 2020, an increase of $58 million compared to the year ended December 31, 2019, attributed to revenues generated from the commercialization of Movantik and Talicia initiated in 2020. Net revenues for the fourth quarter of 2020 were approximately $21.5 million, an increase of $0.5 million compared to the third quarter of 2020, with a 12% increase in product delivery.

Gross Profit was approximately $27.5 million for the year ended December 31, 2020, an increase of $23.5 million compared to the year ended December 31, 2019, primarily due to the increase in net revenues.

Research and Development Expenses were approximately $16.5 million for the year ended December 31, 2020, mainly attributable to the development of our COVID-19 therapeutics and the Phase 3 study of RHB-204 for pulmonary NTM disease, and were, in total, lower than the research and development expenses for the year ended December 31, 2019.

Selling, Marketing and Business Development Expenses were approximately $49 million for the year ended December 31, 2020, compared to approximately $18 million for the year ended December 31, 2019. The increase was attributable to the expansion of our U.S. sales force and marketing activities, in support of the launch of Talicia and post-acquisition commercialization of Movantik.

General and Administrative Expenses were approximately $25 million for the year ended December 31, 2020, compared to approximately $11 million for the year ended December 31, 2019. The increase was mainly attributable to the expansion of commercialization activities related to the Talicia launch and the Movantik acquisition from AstraZeneca.

Operating Loss was approximately $64 million for the year ended December 31, 2020, compared to approximately $43 million for the year ended December 31, 2019. The increase was attributable to the expansion of our commercial operations.

Net Loss was approximately $76 million for the year ended December 31, 2020, compared to approximately $42 million for the year ended December 31, 2019. The increase was attributable to factors mentioned above, as well as interest expenses mainly related to the royalty and debt financing in the first quarter of 2020.

Net Cash Used in Operating Activities was approximately $49 million for the year ended December 31, 2020, compared to approximately $41 million for the year ended December 31, 2019. The increase was attributable to the increase in operating loss, as described above.

Net Cash Used in Investing Activities was approximately $36 million for the year ended December 31, 2020, primarily related to the $52.5 million upfront payment to AstraZeneca for the acquisition of Movantik, partially offset by inflow from current bank deposits and financial assets at fair value through profit or loss.

Net Cash Provided by Financing Activities was approximately $84 million for the year ended December 31, 2020, comprised primarily from financing inflow of approximately $102 million, mainly in debt and equity, partially offset by $16 million classified as restricted cash.

Liquidity and Capital Resources

Cash Balance[1] as of December 31, 2020, was approximately $46 million. Cash balance as of March 4, 2021 was approximately $100 million.

Commercial Highlights

Movantik (naloxegol)[2]
The Company has completed three full quarters of Movantik promotion following its acquisition from AstraZeneca, achieving: A reversal of the trend of declining new prescriptions prior to the acquisition, maintaining Movantik’s position as a segment-leading brand and ending 2020 strongly recording the second highest monthly new prescription volume of 2020 in December.

RedHill acquired the global rights to Movantik from AstraZeneca, excluding Europe and Canada, subsequently adding Israel rights, and replaced a co-commercialization agreement with Daiichi Sankyo (assigned under the agreement with AstraZeneca), with a new royalty-bearing agreement that resulted in RedHill assuming full control over brand strategy and commercialization activities for Movantik in the U.S. and increasing gross margin.

Talicia (omeprazole magnesium, amoxicillin and rifabutin)[3]
Despite the challenging pandemic environment, the Company has persisted in its efforts to support the launch and rapid growth of Talicia, in particular in expanding the prescriber base. This has resulted in the strong accumulation of new Talicia prescribers in the second half of 2020 and consistent month-over-month prescription growth despite pandemic conditions. Talicia ended 2020 strongly, with 52% new prescription growth in the fourth quarter of 2020, as compared to the previous quarter, and achieving its highest weekly new prescription volume in December.

This growth is supported by the addition of Talicia as a preferred brand on leading national formularies, approaching 70% U.S. commercial coverage in the fourth quarter, with further formulary additions expected to add to the previously announced listings of Talicia on the national formularies of Prime Therapeutics, EnvisionRx, and Express Scripts.

Aemcolo (rifamycin)[4]
The Company has implemented plans to support, and build on, the initial momentum that Aemcolo was generating pre-COVID-19 travel restrictions. The Company expects that these plans will drive a resurgence of interest in Aemcolo once travel restrictions are lifted and international travel from the U.S. returns to significant levels.

R&D Highlights

COVID-19 Program: Opaganib (ABC294640, Yeliva)[5]
The late-stage development program for novel, orally-administered, opaganib in patients with severe COVID-19 pneumonia is progressing rapidly. Opaganib has demonstrated dual anti-inflammatory and antiviral activity, targeting a human cell component involved in viral replication and therefore expected to be effective against emerging viral strains with mutations in the spike protein.

The global Phase 2/3 randomized, double-blind, parallel-arm, placebo-controlled study of opaganib in patients with severe COVID-19 pneumonia requiring hospitalization and treatment with supplemental oxygen (NCT04467840), is rapidly advancing in a total of 8 countries and approximately 40 recruiting sites, with additional expansion ongoing. The study has passed three Data Safety Monitoring Board reviews, including a futility review, which is suggestive that the study is progressing as expected. The 464-patient study has already enrolled approximately two thirds of the patients and is expected to deliver top-line data in the second quarter of 2021.

In December 2020, the Company reported positive top-line safety and efficacy data from the U.S. Phase 2 study with opaganib in patients with COVID-19 pneumonia, in which opaganib demonstrated greater improvement in reducing oxygen requirement by end of treatment on Day 14, on top of standard-of-care. The Phase 2 data also showed no material safety differences between the opaganib and placebo treatment arms – further adding to the growing opaganib safety database.

In September 2020, RedHill announced that opaganib demonstrated potent inhibition of SARS-CoV-2, achieving complete blockage of viral replication, as measured after three days incubation, in an in vitro model of human bronchial tissue, comparing favorably with remdesivir, the positive control in the study. Furthermore, treatment of cells infected with SARS-CoV-2 with opaganib did not compromise cell membrane integrity, a measure of cell viability and drug safety, further demonstrating opaganib’s promising potential for treating patients with COVID-19. On top of its anti-inflammatory mechanism, opaganib is also one of very few orally available broad-spectrum antivirals in advanced clinical evaluation for treating COVID-19.

The Company also signed collaborations with several U.S., European and Canadian suppliers, including with Cosmo Pharmaceuticals NV (SIX: COPN) for large-scale ramp-up of opaganib manufacturing, further strengthening manufacturing capabilities and capacity for opaganib.

The Company continues its discussions with U.S. and other government agencies and non-governmental organizations around potential funding to support the rapid advancement of opaganib toward potential emergency use applications and manufacturing scale-up.

COVID-19 Program: RHB-107 (upamostat)[6]
In February 2021, RedHill announced dosing of the first patient in the U.S. Phase 2/3 COVID-19 outpatient study with novel, orally-administered, RHB-107 (upamostat). The study with once-daily RHB-107 is evaluating treatment of patients with symptomatic COVID-19 who do not require hospitalization – the vast majority of COVID-19 patients. The study allows patients to remain in the comfort of their home yet be monitored at a level previously possible only in a hospital setting, enabling increased compliance and retention rates.

RHB-107 is a novel, orally-administered, serine protease inhibitor targeting human cell factors involved in viral entry and is therefore expected to be effective against emerging viral variants with mutations in the spike protein. In previously announced in vitro results, RHB-107 strongly inhibited SARS-CoV-2 viral replication.

RHB-204[7] – Pulmonary Nontuberculous Mycobacteria (NTM) Disease
In November 2020, RedHill commenced its U.S. Phase 3 study to evaluate the efficacy and safety of RHB-204 in adults with pulmonary NTM disease caused by Mycobacterium avium Complex (MAC) infection.

The FDA also granted Fast Track designation for RHB-204 in January 2021, providing early and frequent communications and a rolling review of any New Drug Application (NDA). Having already been granted Qualified Infectious Disease Product (QIDP) designation, RHB-204 is also eligible for NDA Priority Review and Accelerated Approval.

In October 2020, the Company announced that RHB-204 had been granted FDA Orphan Drug Designation. This, along with the previously granted QIDP designation, extends U.S. market exclusivity for RHB-204 to a potential total of 12 years upon FDA approval.

Opaganib – Cholangiocarcinoma and Prostate Cancer
The Phase 2a study evaluating the activity of opaganib in advanced cholangiocarcinoma (bile duct cancer) is ongoing. 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.

In light of preclinical findings demonstrating tumor regression following combination treatment with opaganib and RHB-107 (upamostat), RedHill plans to add an additional cohort to the ongoing Phase 2a study, evaluating opaganib in combination with RHB-107, subject to discussions with the FDA. Opaganib has been granted FDA Orphan Drug Designation for the treatment of cholangiocarcinoma.

An additional Phase 2 study with opaganib in prostate cancer is ongoing at the Medical University of South Carolina (MUSC). The study is supported by a National Cancer Institute grant awarded to MUSC with additional support from RedHill.

COVID-19 Impact Update

RedHill’s primary concern during the COVID-19 pandemic continues to be the safety and health of its employees, patients, colleagues, and the communities to which we belong.

Operationally, the actions the Company took to mitigate the impact of the COVID-19 pandemic continue to serve us well, with minimal effect on our ongoing operational and supply chain activities. Promotional activity has now been largely re-instated, where safe to do so, and in adherence to social distancing and other public health guidelines. RedHill will continue to assess the potential impact of COVID-19 on its business and operations.

Conference Call and Webcast Information:

The Company will host a conference call and live webcast today, Thursday, March 18, 2021, at 8:30 a.m. EDT to present the fourth quarter and full year 2020 financial results and operational highlights.

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