Interim Results for the Six Months Ended 31 March 2022

On June 23, 2022 Redx (AIM:REDX), the clinical-stage biotechnology company focused on discovering and developing novel, small molecule, highly targeted therapeutics for the treatment of cancer and fibrotic disease, reported its unaudited financial results for the six months ended 31 March 2022 (Press release, Redx Pharma, JUN 23, 2022, View Source [SID1234616229]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Lisa Anson, Chief Executive Officer, Redx Pharma, said: "We have made strong progress across all aspects of our pipeline. Importantly, we have moved our lead oncology asset, RXC004, into Phase 2 clinical studies; reported encouraging Phase 1 clinical results for our lead fibrosis asset, RXC007; and nominated our next development candidate, RXC008. We expect RXC008, a GI targeted ROCK inhibitor with the potential to be a first-in-class treatment for fibrostenotic Crohn’s disease, to be ready to enter the clinic by the end of 2023. Together with the recent acceptance of the IND submission for the pan-RAF inhibitor, JZP815, by our partner, Jazz Pharmaceuticals, Redx has demonstrated strong progress across all aspects of our pipeline-a testament to the world class abilities of our drug discovery team."

"In addition to our strong pipeline progress, we were particularly pleased, post-period, to have completed a £34.3 million (gross) placing of our shares. These proceeds will fund our development plans through the end of 2023. We were delighted to receive strong support from all our existing investors as well as welcoming a new specialist healthcare investor, Invus."

Operational Highlights
· Significant clinical progress on lead oncology asset, RXC004, an oral, potent, selective, small molecule Porcupine inhibitor: o In November 2021, initiated PORCUPINE, a Phase 2 trial in genetically selected MSS metastatic colorectal cancer, with US Investigational New Drug (IND) now open; o In January 2022, initiated PORCUPINE2, a second Phase 2 trial in genetically selected pancreatic cancer and unselected biliary cancer.
· On 10 March 2022, Redx presented encouraging Phase 1 safety data for RXC007, an oral selective Rho Associated Protein Kinase 2 (ROCK2) inhibitor with potential for development in multiple fibrotic conditions: o Data showed an excellent safety and pharmacokinetic profile in both the Single Ascending Dose (SAD) and multiple dose cohorts. 23/06/2022, 10:43 View Source View Source 2/25
· On 30 March 2022 nominated RXC008, a Gastrointestinal (GI) targeted Rho Associated Coiled-Coil Containing Protein Kinase (ROCK) inhibitor, as the Company’s next clinical development candidate: o RXC008 is a potential first-in-class treatment for fibrostenotic Crohn’s disease, which has shown strong anti-fibrotic effects in preclinical models. · Progressed the discovery portfolio with the announcement on 27 January 2022 of the Company’s Discoidin Domain Receptor (DDR) inhibitor fibrosis programme: o Developed potent proprietary DDR inhibitors with drug-like characteristics that are now in the lead optimisation phase.
· Advanced preclinical and clinical collaborations with world-leading institutions to enhance the Company’s research capabilities: o Entered a strategic partnership with Caris Life Sciences in December 2021 to accelerate Phase 2 study recruitment in the US for the RXC004 PORCUPINE clinical trial; o Post-period, in April 2022, expanded our collaboration with the Garvan Institute of Medical Research to investigate novel therapeutic targets in cancer-associated fibrosis.
· Significantly progressed our partnered programmes with AstraZeneca and Jazz Pharmaceuticals, resulting in milestones totalling $19 million during the period: o On 9 December 2021, a $10 million (£7.4 million) milestone was triggered from Jazz Pharmaceuticals for the progress in the oncology research collaboration focused on two cancer targets on the MAPK pathway. Post period, one target under this oncology research collaboration with Jazz Pharmaceuticals is confirmed to continue to progress towards an IND application, whilst a second target has been discontinued due to pipeline prioritisation by Jazz given the evolving competitive landscape; o On 23 December 2021, a $9 million (£6.6 million) milestone was triggered from AstraZeneca as RXC006 entered Phase 1 clinical trials; o Post period, on 15 June 2022, Redx announced a milestone of $5 million from Jazz Pharmaceuticals triggered by the US Food and Drug Administration (FDA) clearance of the IND for pan-RAF inhibitor programme, JZP815, which will represent the fifth clinical programme from Redx’s discovery engine to enter the clinic. · Further strengthened the Board of Directors and management team: o Board appointments of Dr Jane Griffiths as Chair from 1 December 2021 and Dr Rob Scott as Non-Executive Director on 27 January 2022; o Established a Science Committee of the Board on 8 March 2022 to oversee Redx’s progress in achieving its scientific and clinical goals; o Appointed Claire Solk on 17 January 2022 to the newly created position of General Counsel.

Financial Highlights

· Cash balance at 31 March 2022 of £31.6 million (31 March 2021 £39.9 million) which includes $19 million in milestone payments received from partnered programmes during the period;
· Successful placing of £34.3 million (gross) completed post-period in June 2022, which received strong support from existing investors and included a new specialist healthcare investor, Invus, which funds the Company’s operations through calendar year 2023, including important Phase 2 proof of concept data readouts for RXC004 and RXC007;
· Post period, on 15 June 2022, Redx also triggered a further milestone payment of $5 million from Jazz; · Increasing investment, reflecting the strong progress in our pipeline, led to increased research and development expenses of £12.9 million (H1 2021: £10.5 million);
· Loss for the period of £9.8 million (H1 2021 £12.7 million). 23/06/2022, 10:43 View Source View Source 3/25 The person responsible for the release of this announcement on behalf of the Company is Andrew Booth, Company Secretary.

SCG Cell Therapy Presents Clinical Proof-of-Concept Data at International Liver Congress (ILC) 2022 Annual Meeting

On June 23, 2022 Singapore-based SCG Cell Therapy Pte Ltd ("SCG") reported interim data from an investigator-initiated clinical trial of SCG101, which included data evaluating single dose SCG101 in patients with advanced hepatitis B (HBV) related hepatocellular carcinoma (HCC) (Press release, SCG Cell Therapy, JUN 23, 2022, View Source [SID1234616224]). The data showed significant antiviral activity and tumour control in two of three patients. This set of data was presented at the International Liver Congress (ILC) 2022 Annual Meeting in London on 23 June.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

In the three HLA-A*02:01-positive patients with advanced HBV-related HCC who had received at least two prior cancer therapies, the tumour growth was controlled in two out of three patients with lesion shrinkage observed. In addition, there is rapid and significant serum HBsAg reduction observed in both subjects from a baseline of 1,004.3 IU/mL and 521.6IU/ML to 23.8 IU/mL and 9.1 IU/mL in just 28 days from SCG101 treatment and further reduced to 14.0 IU/m and 0.3 IU/mL within 60 days from treatment.

"These data showed encouraging antiviral activity and tumour control by a unique T cell receptor (TCR) therapeutic targeting hepatitis B surface antigen (HBsAg) which provides the first clinical proof-of-concept," said Prof. Dr Ulrike Protzer, Director of the Institute of Virology at Helmholtz Munich and Technical University of Munich and Scientific Founder of SCG. "These early results support the continued development of SCG101 for patients with HBV-associated hepatocellular carcinoma where we see a high unmet medical need."

The safety profile of SCG101 was consistent with the side effects expected in T cell therapy. There was no neurotoxicity observed and also no treatment-related AE or SAE (Adverse Event or Serious Adverse Event) which led to treatment discontinuation. Treatment-related cytokine release syndrome (CRS) with fever and bloating were observed in all three patients. Transient ALT and AST increase, without bilirubin elevation or clinical symptoms, was observed but expected due to SCG101 mechanisms of target cells clearance.

"The results of SCG101 show for the first time that an HBsAg directed TCR T cell therapy can provide dual benefit of antiviral and tumour control to patients with HBV-related HCC," said Shunda Du, MD, hepatologist, Peking Union Medical College and principal investigator for the trial. "Chronic HBV infection accounts for more than half of all patients with liver cancer worldwide. The development of HBV-directed TCR therapeutic prompts us to reconsider hepatitis B as a factor in diagnosis and treatment planning for patients with liver cancer."

"We’re proud to collaborate with our partnered hospitals to deliver the world’s first clinical proof of concept for TCR T cell therapy targeting HBV, which we believe validates the strength of our platform and opens doors for us to explore further breakthrough discoveries for the treatment of other cancers and diseases with high unmet need," said Dr Ke Zhang, Chief Scientific Officer of SCG.

SCG101 was granted clinical trial approvals by China National Medical Products Administration (NMPA) and Singapore Health Science Authority (HSA) in May 2022. It is the first TCR-T cell therapy product approved by the NMPA for the treatment of HCC and the first multi-country clinical trial approval across China and Singapore in the field of cell therapy.

About SCG101

SCG101, an autologous T-cell receptor (TCR) T cell therapy, is an investigational cell therapy product that targets specific epitopes of hepatitis B surface antigen (HBsAg). SCG101 incorporates SCG’s proprietary technology, which allows for redirecting and engineering endogenous T cells using virus-specific TCRs with high sensitivity and avidity selectively against dysfunctional infected and tumour cells. Preclinical studies of SCG101 demonstrated tumour inhibition and HBV cccDNA eradication. In 2022, SCG101 was granted clinical trial approvals by the China National Medical Products Administration (NMPA) and Singapore Health Science Authority (HSA) for the potential treatment of HBV-related HCC. The Phase 1/2 study evaluating SCG101 is underway.

About Liver Cancer

Hepatocellular carcinoma (HCC) is the most common type of liver cancer. It is estimated more than 905,000 new cases of liver cancer and more than 830,100 deaths from the disease globally in 2020, making it one of the leading causes of cancer deaths around the world [1]. Chronic hepatitis B virus (HBV) infection accounts for at least 50% of cases of HCC worldwide [2]. HCC is typically diagnosed at an advanced stage and is associated with a poor prognosis. The five-year survival rate of less than 15%.

CAPS Medical Enrolls Patients from Sheba Medical Center in First Ever Study for Treatment of Bladder Cancer Using Selective Endoscopic Cancer Treatment Technology

On June 23, 2022 CAPS Medical, developer of a highly selective and minimally invasive non-thermal plasma (NTAP) device for cancer treatment, reported the enrollment of the first patients in a multi-center study to assess the safety and technical performance of their NTAP system in the treatment of Non Muscle Invasive Bladder Cancer (NMIBC) (Press release, CAPS Medical, JUN 23, 2022, View Source [SID1234616223]). The cancer patients in the study have been treated at Sheba Medical Center’s Urology Department. This is the first ever worldwide study for minimally invasive treatment of Bladder Cancer using Non Thermal Plasma.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Non-Muscle Invasive Bladder Cancer (NMIBC) impacts 500,000 people in the U.S. alone with over 80,000 new cases added annually. NMIBC is characterized by a high recurrence rate of up to 80%, which means that in the majority of cases NMIBC is not cured. It is treated and retreated –using a Transurethral Resection of Bladder Tumor (TURBT) procedure, which can cause scarring and damage to the bladder. This is followed by a series of intravesical instillations, which cause life-altering side effects. "We hope that CAPS Medical’s NTAP solution will help reduce the cancer recurrence rate by replacing the existing TURBT procedure while also avoiding the typical side effects, damage to the bladder and complications associated with the treatment options available today," said Prof. Zohar Dotan, Chairman of the Department of Urology and the principal investigator of the trial at the Sheba Medical Center. Sheba has been ranked by Newsweek magazine, for the fourth consecutive year, as one of the 10 top hospitals in the world

Non-thermal Atmospheric Plasmas are streams of low ionized gas at room temperature. Their efficacy in selectively destroying cancer cells while preserving surrounding healthy tissue and their ability to trigger an immunological effect and encourage the body to heal itself are well-known and have been demonstrated. To date, however, the high energy required for existing non-thermal plasma applications necessitated the use of large-sized devices. This made using non-thermal plasma to treat cancerous tumors in minimally invasive procedures unfeasible.

Overcoming these limitations, CAPS Medical is a pioneer in creating a device for NTAP delivery through a working channel of standard minimally invasive tools, making it possible to avoid surgery. "This multi-center study should demonstrate how NTAP could be painlessly and safely used to treat tumors inside the body, while preserving healthy tissue. CAPS Medical intends to continue working to improve patients’ lives through the use of this revolutionary technology which can be tailored to a variety of additional cancer types," said Ilan Uchitel, CEO and Co-Founder of CAPS Medical.

"With its NTAP system, CAPS Medical is overcoming a real barrier within the cancer treatment ecosystem. The NTAP procedure selectively targets cancer cells with greater precision-which should lead to fewer side effects than many current treatment options. We look forward to seeing the company progress through its clinical trials," said Harel Gadot, Executive Chairman of CAPS Medical and Company Group Chairman of MEDX Ventures Group.

RedHill Biopharma Announces Q1/22 Highlights: On Track for Positive Cash from Operations in H2/22

On June 23, 2022 RedHill Biopharma Ltd. (Nasdaq: RDHL) ("RedHill" or the "Company"), a specialty biopharmaceutical company, reported its first quarter 2022 financial results and operational highlights, targeting positive cash from operations1 to start during H2/22 and recent implementation of a comprehensive cost reduction plan, expected to generate operational cost savings of approximately $50 million over the next 18 months (Press release, RedHill Biopharma, JUN 23, 2022, View Source [SID1234616222]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Dror Ben-Asher, RedHill’s Chief Executive Officer, said: "To address the current market realities and operating landscape, RedHill is being decisive about controlling its own destiny and is highly focused on achieving earlier profitability, targeting positive cash from operations to start during H2/221. Our comprehensive cost reduction plan is expected to generate operational cost savings of approximately $50 million over the next 18 months. The majority of these savings result from an approximately one-third reduction of the U.S. commercial team workforce, returning to pre-pandemic levels, streamlined operational expenditure including selling, general and administrative (SG&A) expenses and refining the Company’s R&D strategy to rely mostly on external funding sources based on the promising clinical data generated to date. On behalf of RedHill and its Board of Directors, I would like to express my profound and respectful gratitude to the colleagues who are departing RedHill. We value and are extremely grateful for your immense contributions and dedication which enabled RedHill to build a first-class U.S. commercial organization and launch three innovative products that help patients in need. While difficult, the changes we have made as part of our cost reduction plan were necessary given the current realities."

Mr. Ben-Asher continued: "Improved financial stability resulting from cost savings, a commercial basket including three FDA-approved proprietary drugs, continued prescription growth and healthy sales, improved managed care coverage and the amended credit agreement with HCR, positions RedHill for further growth. To that end, RedHill is in non-binding discussions to acquire a synergetic U.S. FDA-approved, patented GI drug currently generating dozens of millions of dollars, which, if materialized, will help expedite and increase cash generation."

Micha Ben Chorin, Chief Financial Officer at RedHill, added: "RedHill’s commercial team has further strengthened Movantik’s share of the growing PAMORA class to approximately 74%, and Talicia’s approximately 13% quarterly growth further consolidates its position as the leading U.S. brand for H. pylori eradication. The Company’s disciplined cost control measures reduced Q1/22 cash used in operating activities by more than 70% to approximately $4 million, compared to approximately $15 million in Q4/21. Despite the challenging market conditions, RedHill is on its way to achieving company-wide positive cash from operations1 this year, and this, along with the envisaged growth drivers from the pipeline and potential acquisitions, could boost cash generation and further improve cash flow. We are confident that RedHill is well positioned for continuing growth."

Mr. Ben-Asher further remarked: "Turning to R&D, in reliance of promising clinical and other data we generated to date, we expect external funding for much of our promising R&D programs, through grants, industry partnerships and participation in platform studies. COVID-19, included in a broader category of ‘pandemic preparedness’, remains an area of high interest and need for novel oral therapeutics such as RedHill’s candidates. Regulatory feedback on opaganib, supportive of a confirmatory study has provided a pathway to potential opaganib submissions for approval in COVID-19. Most recently, opaganib further exhibited its variant-agnostic credentials by demonstrating potent in vitro efficacy against Omicron, while also showing, in a separate study, potent in vitro inhibition of influenza A H1N1, adding to evidence of opaganib’s broader antiviral potential and its promising applicability to ‘pandemic preparedness’. Meanwhile, RedHill’s 2nd COVID-19 therapeutic candidate, RHB-107, successfully met the Part A study primary outcome measure for its Phase 2/3-stage study in non-hospitalized COVID-19, showing, among other promising data, a 100% reduction in hospitalization due to COVID-19. Discussions are ongoing regarding potential external funding through grants, platform studies and other public, private and industry partnerships to support the opaganib and RHB-107 programs, as well as our ongoing Phase 3 study with RHB-204 for NTM disease."

Financial results for the quarter ended March 31, 2022 (Unaudited)[3]

Net Revenues for the first quarter of 2022 were $18.2 million, as compared to $22.1 million in the fourth quarter of 2021, the difference being attributable to typical cyclical trends in Movantik sales and increased gross to net deductions related mainly to increased formulary coverage.

Cost of Revenues for the first quarter of 2022 were $8.0 million, as compared to $19.3 million in the fourth quarter of 2021. The decrease was attributed to recognition of an approximately $9 million impairment related to the intangible asset of Aemcolo for travelers’ diarrhea in the previous quarter.

Gross Profit for the first quarter of 2022 was $10.2 million, as compared to $2.7 million in the fourth quarter of 2021. The increase was attributed to the impairment recognized in the previous quarter, as detailed above.

Research and Development Expenses for the first quarter of 2022 were $3.1 million, as compared to $5.9 million in the fourth quarter of 2021. The decrease was attributed to the ongoing optimization of R&D costs and completion of elements of the opaganib and RHB-107 development programs.

Selling, Marketing and General and Administrative Expenses for the first quarter of 2022 were $20.4 million, as compared to $17.6 million in the fourth quarter of 2021. The increase was mainly attributed to a one-off positive adjustment in quarter four of 2021 and expenses related to professional services and other related expenses in the first quarter of 2022.

Operating Loss for the first quarter of 2022 was $13.2 million, as compared to $20.7 million in the fourth quarter of 2021. The decrease was mainly attributed to the impairment recognized in the previous quarter, as detailed above.

Net Cash Used in Operating Activities for the first quarter of 2022 was $4.2 million, as compared to $14.9 million in the fourth quarter of 2021. The decrease was mainly due to changes in working capital and continued implementation of cost-reduction measures.

Net Cash Used in Financing Activities for the first quarter of 2022 was $4.9 million, as compared to Net Cash Provided by Financing Activities of $17.6 million in the fourth quarter of 2021, comprised mostly from proceeds of equity offerings completed in the fourth quarter of 2021. The additional decrease of $5 million was due to a reduction of Movantik acquisition liabilities.

Liquidity and Capital Resources

Cash Balance1 as of March 31, 2022, was $45.0 million, as compared to $54.2 million as of December 31, 2021.

On June 17, 2022, RedHill Biopharma Inc. signed an amendment to the HCR Credit Agreement reducing the revenue covenant to $75.0 million for the next two quarters, with a 0.5% increase in interest.

The license of opaganib[4] for COVID-19 by Kukbo for South Korea is expected to yield a $1.5 million upfront payment to RedHill and, in addition, up to $5.6 million in milestone payments plus royalties on net sales.

The license of Talicia for H. pylori by Gaelan Medical for the United Arab Emirates, with $2.0 million upfront payment received, is anticipated to yield additional milestones and royalties on net sales.

Discussions with additional potential partners, for both in- and out-licensing partnerships, are ongoing including for potential acquisitions of additional synergetic commercial products with strong cash generation potential.

Commercial Highlights

Movantik (naloxegol)[5]

The Company’s focus and determination to drive both Movantik and the PAMORA class as a whole have delivered important results, with Movantik’s Q1/22 performance matching the record pace set in Q4/21. This represents an 8.6% increase in new prescriptions compared to Q1/21, more than doubling the already strong growth of the overall PAMORA class in the same period and contributing to a further increase in market share for Movantik, now up to almost 74% of the PAMORA class.

Nearly 92% of insurance plans provide access for Movantik – best-in-class coverage – and as of January 1, 2022, Movantik has been approved for inclusion as a preferred and unrestricted brand on a major National Medicare Part D formulary serving more than 10 million Americans. Movantik’s total commercial coverage now extends to 151 million American patients’ lives and has grown to 46 million Medicare lives, with over 93% coverage of Medicare Part D lives.

With best-in-class payer coverage, focused execution in the pain segment and continuing the PAMORA class market development, Movantik is well-positioned for continued growth in 2022 and beyond.

Talicia (omeprazole magnesium, amoxicillin and rifabutin)[6]

Talicia reached new TRx heights in Q1/22, growing a further 12.8% compared to the previous record levels set in Q4/21. This growth represents an 80% increase in new prescriptions from Q1/21, further cementing Talicia’s position as the most prescribed branded H. pylori therapy in the U.S. Significant increase in prescription volume in March of this year, coupled with a growing prescriber base, improving payor coverage and continuing promotional focus is expected to result in further growth acceleration.

Medi-Cal, California’s Medicaid Health Care program, which added Talicia last year to its Contract Drug List (CDL) for H. pylori treatment, with no prior authorization required, expanded coverage to 14 million beneficiaries on January 1, 2022. Florida Medicaid coverage started in April and another large Part D plan coverage became effective earlier this quarter. The Company also expects a major new coverage win to initiate July 1, 2022. As of May 2022, total Talicia coverage stood at nearly 200 million American lives, equating to seven out of ten lives.

Outside of the U.S., in January 2022, the Company announced that it had entered into an exclusive license agreement with Gaelan Medical Trade LLC, a wholly owned subsidiary of the Ghassan Aboud Group (GAG), for Talicia in the United Arab Emirates (UAE). Under the terms of the agreement, RedHill received an upfront payment of $2 million and is eligible for additional milestone payments as well as tiered royalties up to mid-teens on net sales of Talicia in the UAE if marketing authorization is received and Talicia is commercialized. Gaelan Medical received the exclusive rights to Talicia in the UAE, as well as a right of first refusal in relation to Talicia in the Gulf Cooperation Council region (Saudi Arabia, Kuwait, Qatar, Bahrain and Oman) for a pre-determined period.

Aemcolo (rifamycin)[7]

The Company continues to be ready for post-COVID-19 returning travel opportunities for Americans.

R&D Highlights

Opaganib (ABC294640)

COVID-19:

COVID-19, included in a broader category of ‘pandemic preparedness’, remains an area of high interest and need for novel oral therapeutics. Data from prespecified analyses of opaganib’s Phase 2/3 study (NCT04467840), announced in January and February 2022, demonstrated that opaganib improved viral RNA clearance, achieved faster time to recovery and reduced mortality in key subpopulations of moderate to severe hospitalized patients with COVID-19. Additionally, a post-hoc analysis identified a biomarker, the fraction of inspired oxygen (FiO2), to select patients that showed superior outcomes with opaganib vs. placebo. Opaganib also demonstrated potent in vitro efficacy against the Omicron SARS-CoV-2 variant and is expected to remain effective against sub-variants BA.2, XE and other emerging and future variants. Based on regulatory guidance, a positive confirmatory study constitutes the likely pathway to potential opaganib submissions for approval in the U.S., EU, and multiple other territories.

Data from the global Phase 2/3 study has now been published on MedRxiv and will also be the subject of a "Late-Breaker" oral presentation at the joint CDC / Task Force for Global Health-organized International Conference on Emerging Infectious Diseases, to be held in August.

On June 21, 2022, and adding to opaganib’s expanding patent suite, RedHill was granted an additional U.S. patent directed to a method for the treatment of COVID-19 in patients with moderate to severe COVID-19 related pneumonia using opaganib.

RedHill is pursuing multiple public and private external funding sources for our programs including grants in the U.S., UK and EU, government sponsored platform studies and industry partnerships. In March 2022, the Company announced that it had entered into an exclusive license agreement with Kukbo Co. Ltd., a South Korean corporation, for the exclusive rights to commercialize opaganib for the treatment of COVID-19 in South Korea. Under the terms of the agreement, RedHill is entitled to receive an upfront payment of $1.5 million and is eligible for $5.6 million in additional milestone payments, as well as low double-digit royalties on net sales of opaganib if marketing authorization is received and opaganib is commercialized.

Other indications under investigation:

Influenza A H1N1: Opaganib demonstrated potent in vitro inhibition of influenza A H1N1, at low concentrations and with no evidence of toxicity at these levels. The results were obtained in Normal Human Bronchial Epithelial Cells (NHBE) assay, the natural human target of the virus, making it a realistic model. These results add to previous data demonstrating antiviral activity in several in vitro and in vivo viral infection models including SARS-CoV-2, influenza and Ebola, providing further evidence of opaganib’s potential broad-spectrum antiviral effect. Discussions with NIH are ongoing regarding next steps for investigating opaganib’s broader antiviral potential and its promising applicability to ‘pandemic preparedness’.

Oncology:

The Company continues to advance opaganib’s development program in oncology with the cholangiocarcinoma (CCA) Phase 2 study analysis expected in Q3/2022

RHB-107 (upamostat)[8]

COVID-19:

In March 2022, the Company announced positive top-line results from Part A of the two- stage Phase 2/3 study of novel, once-daily, orally-administered, antiviral drug candidate, RHB-107. The study, evaluating RHB-107 for the treatment of non-hospitalized patients with symptomatic COVID-19 in the early course of the disease (NCT04723537), who do not require supplemental oxygen (the vast majority of COVID-19 patients) was predominantly conducted in the U.S. (60/61 patients) as well as South Africa.

Results showed that RHB-107 met the primary outcome measure, demonstrating a favorable safety and tolerability profile and showed a 100% reduction in hospitalization due to COVID-19, with zero patients (0/41) on the RHB-107 arms versus 15% (3/20) hospitalized due to COVID-19 on the placebo-controlled arm (nominal p-value=0.0317). The study also showed an approximately 88% reduction in reported new severe COVID-19 symptoms after treatment initiation, with only one patient in the RHB-107 treated group 2.4%, (1/41) versus 20% (4/20) of patients in the placebo-controlled arm. Further analysis of study data also showed a faster recovery from severe COVID-19 symptoms for patients in the RHB-107 arm, with a median of 3 days to recovery with RHB-107 vs. 8 days with placebo.

RHB 107’s unique human host-targeted, suggested antiviral mechanism is expected to act independently of viral spike protein mutations and remain effective against Omicron and sub-variants BA.2, XE and other emerging and future variants.

Next steps for the study are expected following ongoing discussions with regulators.

RHB-204 – Pulmonary Nontuberculous Mycobacteria (NTM) Disease[9]

A U.S. Phase 3 study is ongoing in the U.S. to evaluate the efficacy and safety of RHB-204 in adults with pulmonary NTM disease caused by Mycobacterium avium Complex (MAC) infection (NCT04616924). The waning of COVID-19 is expected to accelerate the advancement of the program

The study protocol provides for 6 months co-primary endpoint of sputum culture conversion (SCC) and clinical outcome (patient-reported outcomes – PRO) in a randomized placebo-controlled design, followed by open label active treatment with RHB-204 for 12 months from conversion.

RHB-204 has been granted Orphan Drug designation and QIDP status – providing for Fast Track and Priority Review and resulting in eligibility for 12 years post-approval market exclusivity.

Conference Call and Webcast Information:

The Company will host a conference call and webcast today, Thursday, June 23, 2022, at 8:30 a.m. EDT, during which it will present key highlights for the first quarter of 2022.

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

Fusion Pharmaceuticals Announces FDA Clearance of IND for FPI-2059, an Investigational Small Molecule-Based Radiopharmaceutical Targeting Solid Tumors Expressing NTSR1

On June 23, 2022 Fusion Pharmaceuticals Inc. (Nasdaq: FUSN), a clinical-stage oncology company focused on developing next-generation radiopharmaceuticals as precision medicines, reported that the U.S. Food and Drug Administration (FDA) has cleared the Company’s Investigational New Drug (IND) applications for [225Ac]-FPI-2059 (FPI-2059) and the corresponding imaging analogue [111In]-FPI-2058 (FPI-2058). FPI-2059 is a targeted alpha therapy (TAT) designed to use a small molecule to target and deliver actinium-225 to tumor sites expressing neurotensin receptor 1 (NTSR1), a protein that is overexpressed in multiple solid tumor types, including colorectal, pancreatic, gastric, neuroendocrine differentiated prostate, head and neck squamous cell carcinoma, and Ewing sarcoma cancers (Press release, Fusion Pharmaceuticals, JUN 23, 2022, View Source [SID1234616221]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"The FPI-2059 program showcases Fusion’s ability to use our platform technology and R&D expertise to efficiently convert different classes of targeting molecules into TATs against innovative targets that are designed to address cancers with high unmet need," said John Valliant, Ph.D. "With FPI-2059, we believe there is significant opportunity to address multiple solid tumor types, including neuroendocrine differentiated prostate cancer where PSMA expression is typically low and therefore patients are not adequately treated by existing radioligand therapies. We look forward to progressing FPI-2059, Fusion’s first small molecule-based TAT and third clinical program, into a Phase 1 study."

Fusion acquired [177Lu]-IPN-1087 (IPN-1087), a lutetium-based beta-emitting radiopharmaceutical, from Ipsen in April 2021, and converted the compound to the alpha-emitting [225Ac]-FPI-2059. In clinical studies, IPN-1087 showed promising early safety data and good uptake in multiple tumor types. In a head-to-head in vivo comparison of therapeutic efficacy in a mouse xenograft model of colorectal cancer between FPI-2059 and IPN-1087, results show tumor regression with FPI-2059 is achieved at doses of approximately 1500 times lower than IPN-1087.

Fusion plans to initiate a Phase 1, non-randomized, open-label clinical trial in patients with solid tumors expressing NTSR1, intended to investigate safety, tolerability and pharmacokinetics and to establish the recommended Phase 2 dose. The study will prioritize six solid tumor indications, including head and neck squamous cell carcinoma, pancreatic, neuroendocrine prostate, colorectal, gastric and Ewing sarcoma. The study employs a 3 + 3 dose escalation design to evaluate multiple ascending doses of FPI-2059. As part of the screening process, patients will be administered an imaging analogue of FPI-2059, FPI-2058, and only those who meet predefined tumor uptake and safety criteria will go on to receive FPI-2059.

Radiopharmaceuticals are a precision medicine in that the alpha therapeutic can be converted into a corresponding imaging analogue with a different radionuclide (in this case indium), used to screen for a biomarker in patients with tumors that express the cancer target, increasing the likelihood of response to therapy. Fusion plans to provide additional guidance on timelines for the FPI-2059 program following initial experience with patient screening in order to better predict the cadence of patient enrollment.

About FPI-2059
[225Ac]-FPI-2059 (FPI-2059) is a targeted alpha therapy combining actinium-225 with a small molecule designed to target neurotensin receptor 1 (NTSR1), in development as a potential treatment for various solid tumors. NTSR1 is a promising target for cancer treatment that is overexpressed in multiple solid tumors including colorectal, pancreatic, gastric, neuroendocrine differentiated prostate, head and neck squamous cell carcinoma and Ewing sarcoma cancers. FPI-2059 is currently being evaluated in a Phase 1 study.