PTC Therapeutics has signed a strategic financing partnership worth up to $1bn with funds managed by Blackstone to back the development of the former’s innovative product pipeline. Under the deal, funds managed by Blackstone Life Sciences and Blackstone Credit will make initial funding worth $500m to PTC, which comprises $350m in low-cost, low-dilution capital at closing.

This alliance takes advantage of the several therapeutic platforms of PTC and its established track record of providing transformative therapies to patients with rare diseases. Translarna, Emflaza, Upstaza, sepiapterin and vatiquinone are used as collateral for the tailored senior secured term loan investment from Blackstone.

The World Health Organization (WHO) has granted prequalification for Sinovac Biotech’s live attenuated varicella vaccine. With the latest development, the vaccine became the first Chinese varicella shot to be prequalified by the WHO. It is also the company’s fourth vaccine to receive approval from the health agency. 

According to the data from a Phase III efficacy clinical trial, the seroconversion rate was found to be 97.1% in a group of children aged one to 12 years old who received the vaccine. The vaccine also demonstrated an efficacy of 87.1% and 89.2% against varicella and breakthrough varicella, respectively.

The US FDA has granted Fast Track designation for Dyne Therapeutics’ Dyne-251 to treat Duchenne muscular dystrophy (DMD) mutations amenable to exon 51 skipping. Dyne-251 comprises a phosphorodiamidate morpholino oligomer (PMO) conjugated to a fragment antibody (Fab) that attaches to the transferrin receptor 1 (TfR1) expressed highly on muscle.

It can permit targeted muscle tissue delivery and boost exon skipping in the nucleus, aiding muscle cells to produce a shortened, functional dystrophin protein for halting or reversing the progression of the disease. It attained exon skipping, particularly in the heart and diaphragm, muscles in DMD patients.  At present, DYNE-251 is being analysed in the international Phase I/II DELIVER trial.

Viatris has signed definitive agreements to acquire Oyster Point Pharma and Famy Life Sciences in a deal totalling nearly $700m-$750m in cash to establish an ophthalmology franchise. According to the deal, all outstanding shares of Oyster Point will be acquired by Viatris for a price of $11 for each share in cash at closing, plus a non-tradeable contingent value right (CVR) for up to $2 for each share.

A commercial-stage biopharmaceutical firm, Oyster Point discovers, develops and commercialises therapies for treating ophthalmic ailments. Oyster Point Pharma introduced Tyrvaya, a nasal spray approved by the US FDA to treat dry eye disease, in November last year.

The US Food and Drug Administration (FDA) has accepted Seres Therapeutics’ Biologics License Application (BLA) for SER-109 to prevent recurrent C. difficile infection (rCDI) for priority review. 

A decision from the regulatory agency on approval for the therapy is anticipated on 26 April next year. Additionally, the FDA said that it is not presently planning to organise an Advisory Committee Meeting to discuss the BLA for SER-109. An investigational, oral microbiome therapy candidate, SER-109 has purified Firmicutes spores to treat rCDI. In July last year, the company and Nestlé Health Science signed an agreement to co-market SER-109 in the US and possibly in Canada.

Ayala Pharmaceuticals has signed a definitive merger agreement with biotechnology company Advaxis. The latter’s immunotherapies are based on a technology that leverages engineered Listeria monocytogenes.

The business combination would result in a merged company that will mainly focus on developing and marketing Ayala’s AL102 and Advaxis’ ADXS-504. A lead candidate of Ayala, AL102 is intended to treat desmoid tumours, while ADXS-504 is being developed for prostate cancer. AL102 is being assessed in the Phase II/III RINGSIDE trial for desmoid tumours.