Hepion Pharma’s NASH drug is set for Phase 2b after clearing the safety bar

Hepion Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on developing therapeutic drugs for the liver disease treatment arising from non-alcoholic steatohepatitis (NASH), declaring positive topline results from its Phase 2a ‘AMBITION’ NASH clinical trial. All primary endpoints of the trial were fulfilled. 

AMBITION was a Phase 2a randomized, multi-center, placebo-controlled, a single-blind trial designed to evaluate once-daily oral administration of CRV431 at doses of 75 mg and 225 mg administered as soft gelatin capsules to expected F2 and F3 NASH subjects for 28 days, followed by a 14-day observation period for safety.

The primary result measure of the AMBITION trial was the incidence of safety and tolerability events of CRV431 versus placebo. CRV431 at both study doses was well tolerated, and there were no severe adverse events, and the few adverse events observed were primarily mild and unrelated to the study drug.

In the AMBITION trial, CRV431 blood concentrations after oral dosing of either 75 mg or 225 mg once daily were in the expected effective range for NASH treatment. The drug reached maximum concentrations within two hours after dosing with an effective half-life of approximately 30 hours, which supports once-daily dosing.

Bicycle Therapeutics enters exclusive license and collaboration deal with Ionis for development of targeted oligonucleotide therapeutics

Bicycle Therapeutics plc, a biotechnology company pioneering a new and differentiated class of therapeutics based on its proprietary bicyclic peptide (Bicycle) technology, declared that Ionis Pharmaceuticals has implemented its option and came into an exclusive global license and collaboration agreement for tissue-targeted delivery of oligonucleotide therapeutics utilizing Bicycles with high affinity to the transferrin receptor (TfR1). 

Ionis had an option for an exclusive license under the terms of a December 2020 evaluation and option agreement. The deal granted Ionis the right to assess tissue-targeting TfR1 binding Bicycles as vehicles to proffer oligonucleotide therapeutics to particular organ systems and an option to secure an exclusive license at the end of the evaluation period.

Bicycle gets a total of USD 45 million upfront, which includes a license fee, an option fee, and a USD 11 million equity investment. Bicycle can receive development, regulatory and commercial milestone payments and royalties for each program developed under the agreement.

Bicycle has recognized binders that it believes to be the first small molecules to aim TfR1 with high specificity without altering TfR1’s natural function. These binders present broad conjugation potential across multiple payloads, resulting in treating diseases in various therapeutic areas, comprising skeletal and cardiac muscles and the central nervous system. Bicycle is currently under agreement with the Dementia Discovery Fund (DDF) to enhance potential TfR1 Bicycles for treating dementia.

Under the terms of the license and collaboration deal, Ionis and Bicycle will collaborate to create a pipeline of oligonucleotide therapeutic product candidates delivered using the tissue-targeting TfR1 Bicycle technology, while Bicycle reserves the rights to use TfR1 Bicycles for all non-oligonucleotide therapeutic purposes, including within its existing collaboration with DDF targeting dementia.

Nimbus raises USD 105 Million for multifront TYK2 clinical trial 

Nimbus Therapeutics raised USD 105 million to clutch an allosteric TYK2 inhibitor in phase II clinical trial. The multi-front mid-stage program is designed to determine if the drug has potential in the range of autoimmune and inflammatory diseases that may be susceptible to TYK2 inhibition.

TYK2 is part of the JAK family. Approved JAK inhibitors such as Eli Lilly’s Olumiant and AbbVie’s Rinvoq have enhanced outcomes for diseases such as rheumatoid arthritis. However, the FDA has warned of an increased risk of serious infections, malignancies, and thrombosis. Selective inhibition of TYK2 may proffer a better risk and benefit profile.

Investor syndicates led by BVF partners have come together to aid Nimbus to see if the outlook is fulfilling its promises. Nimbus has cash from new and existing investors such as RA Capital Management, Atlas Ventures, Access Biotechnology, and Comodor Capital and intends to launch multiple Phase 2 studies in 2021 and 2022. Patients will benefit from this new treatment. 

Bristol Myers Squibb has set a marker for potential TYK2 inhibitors over the past year by proffering Phase 3 data on duclavasitinib in psoriasis. Armed with data, Bristol Myers will look for FDA approval to generate USD 4 billion in annual revenue.

The excitement about TYL2 inhibitors reflects the potential of the molecule to treat a wide range of diseases. Bristol Myers studies duclavasitinib for indications such as lupus nephritis, psoriatic arthritis, systemic lupus erythematosus, and ulcerative colitis. With USD105 million in new funding, Nimbus has the means to study the potential of rival molecules. Bristol Myers has gained the option to acquire Nimbus’ TYK2 program through the acquisition of Celgene.

Regeneron to support ISA Pharma’s HPV program in a USD 30 million funding round

Regeneron is once again backing ISA Pharmaceuticals’ efforts to bring human papillomavirus treatments to market in a USD 30 million funding round.

The Dutch biotechnology will utilize this funding to partner with Regeneron to fund the development of three late-stage clinical trials to treat human papillomavirus type 16 (HPV16) called ISA101b. As per the statement, this HPV strain is responsible for more than 50% of human cervical cancers and more than 85% of HPV-positive head and neck cancers.

ISA Pharma raised funding from existing backers such as Invest-NL and Regeneron, new investors to market immunotherapy.

The company has completed Phase 2 trials to treat vulvar intraepithelial neoplasia, a type of cancer that affects the vulva, and completed Phase 1/2 trials for cervical cancer.

Regeneron has further committed to being a key candidate for ISA Pharma in the funding round after first linking the arm in December 2017. ISA101b is being studied in phase 2 clinical trials combining primary and secondary HPV16-positive head and neck cancer. Uses Regeneron and Sanofi’s PD-1 inhibitor Libetayo. The company said the same combination would be studied in a phase 2 study in patients with cervical cancer.

ISA Pharma is one of several companies testing treatments in combination with Libtayo. Inovio is considering a DNA drug in combination with a drug for patients with brain tumor glioblastoma. BioNTech is working on the intermediate stage study of mRNA cancer immunotherapy with Libtayo in patients with advanced melanoma.