RedHill Biopharma Ltd (RDHL)

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RedHill Biopharma Announces Positive New Data with Opaganib for Nuclear Radiation Injury

https://finance.yahoo.com/news/redhill-biopharma-announces-positive-data-133000587.html

Positive in vivo results from a new study of opaganib in radiation-induced hematologic and renal toxicity reaffirms opaganib's potential protective impact on hematological and kidney function following total body irradiation (TBI)

These new preclinical results are consistent with recently published, U.S. government funded, in vivo efficacy data suggestive of opaganib's potential as a nuclear radiation injury therapeutic for homeland security medical countermeasures (MCM)

Opaganib is an oral, highly stable, novel small molecule pill potentially suitable, if approved, for central stockpiling by governments against mass casualty nuclear radiation incidents

Following prior FDA guidance specific to opaganib, and subject to further FDA guidance, development of opaganib as a homeland security nuclear medical countermeasure is expected to follow the Animal Rule under which human efficacy studies may not be required

Opaganib, if approved, may be eligible for a medical countermeasure Priority Review Voucher

Discussions regarding further support and pathway to potential approval have been initiated with U.S. and other governments

Development continues for COVID-19, other pandemic preparedness antiviral indications and oncology, strongly positioning opaganib as a pipeline-in-a-product

TEL AVIV, Israel and RALEIGH, NC, Nov. 17, 2022 /PRNewswire/ -- RedHill Biopharma Ltd. (Nasdaq: RDHL) ("RedHill" or the "Company"), a specialty biopharmaceutical company, today announced positive in vivo results from a new pre-clinical study evaluating the effects of opaganib[1] on radiation-induced hematologic and renal toxicity, undertaken by RedHill and its partner Apogee Biotechnology Corporation ("Apogee"). The results, suggesting that opaganib exerts a protective impact on key hematological and kidney function parameters following total body irradiation (TBI), are consistent with recently published U.S. government-funded in vivo opaganib data[2] and are further supportive of opaganib's planned development under the Animal Rule for nuclear radiation protection.

Lynn W. Maines, PhD., Apogee's VP of Research, said: "These results, as well as the established scientific understanding that disruption of S1P production reduces damaging pathologic inflammation, further support the hypothesis that opaganib's inhibition of SK2, and subsequent reduction S1P production, suppresses Heme-ARS and kidney damage by lethal total body irradiation, and may play a key role in protecting against generalized radiation injury."

"These new results add to, and are consistent with, the positive indications towards opaganib's radioprotective capabilities reported in the recent peer-reviewed International Journal of Molecular Sciences (IJMS) publication and support further studies of opaganib in this indication," said Dr Mark Levitt, MD, PhD, Chief Scientific Officer at RedHill. "Our assessment of the pathway towards approval, and following recent discussions with key government agencies, gives us an understanding of the progress already made, and of the areas of ongoing work still to be completed, which we believe can be done in an expedited manner given the prevailing need for new radioprotective treatment options."

Recently published, U.S. government funded results, from eight preclinical studies and additional experiments, indicate opaganib's potential as a nuclear radiation injury therapeutic for homeland security material threat medical countermeasures (MCM) and for antitumor radiotherapy. These results indicate that opaganib may protect normal tissue from damage due to ionizing radiation exposure or cancer radiotherapy, improve antitumor activity and response to chemoradiation, and enhance tolerability and survival.

Opaganib is a novel oral small molecule sphingosine kinase 2 (SK2) inhibitor that is potentially suitable to current government research priorities for material threat medical countermeasures suitable for central stockpiling for use in mass casualty nuclear radiation incidents. Opaganib can be administered 24 hours or later after radiation exposure, is highly stable with a more than five-year shelf-life, easy to administer and distribute, and demonstrated its clinical safety profile in various human clinical studies and expanded access uses in other indications.

Multiple discussions with government agencies in the U.S. and internationally are ongoing regarding funding and medical countermeasure development pathways.

Sponsors of approved medical countermeasures product applications may be eligible for a medical countermeasure Priority Review Voucher.

About Opaganib (ABC294640)

Opaganib a new chemical entity, is an orally administered, first-in-class proprietary selective inhibitor of sphingosine kinase-2 (SK2) with suggested anti-inflammatory, anticancer, radioprotective and antiviral activity.

Opaganib is thought to work through the inhibition of multiple pathways, the induction of autophagy and apoptosis, and disruption of viral replication, through simultaneous inhibition of three sphingolipid-metabolizing enzymes in human cells (SK2, DES1 and GCS).

In an oncology & radiological setting, opaganib has been observed to elevate certain ceramides and reduces sphingosine 1-phosphate (S1P) in cells, conditions that increase the antitumor efficacy of radiation while concomitantly suppressing inflammatory damage to normal tissue, leading to the potential to suppress toxicity from unintended ionizing radiation (IR) exposure and improve patient response to chemoradiation. Opaganib has received Orphan Drug designation from the U.S. FDA for the treatment of cholangiocarcinoma and is being evaluated in a Phase 2a study in advanced cholangiocarcinoma. Patient accrual, treatment and analysis in a prostate cancer study is ongoing. Opaganib has a Phase 1 chemoradiotherapy study protocol ready for IND submission.

Opaganib has demonstrated broad-acting, host-directed, antiviral activity against SARS-CoV-2, multiple variants, and several other viruses, such as Influenza A. Being host-targeted, and based on data accumulated to date, opaganib is expected to maintain effect against emerging viral variants. In prespecified analyses of Phase 2/3 clinical data in hospitalized patients with moderate to severe COVID-19, oral opaganib demonstrated improved viral RNA clearance, faster time to recovery and significant mortality reduction in key patient subpopulations versus placebo on top of standard of care. Data from the opaganib global Phase 2/3 study has been submitted for peer review and recently published in medRxiv.

Opaganib has also shown positive preclinical results in renal fibrosis, and has the potential to target multiple oncology, radioprotection, viral, inflammatory, and gastrointestinal indications.