Regrow's cell therapy product gets orphan drug status from USFDA

Start-up in talks with MNCsto out-license Ossgrow for developed markets

Mumbai-based biotech start-up Regrow Biosciences that developed cell-therapy based solution for orthopaedic joint issues is in talks with multinational firms to out-license its product for commercialisation in the US and other developed markets. It has recently received orphan drug designation (ODD) from the US drug regulator and expects the same from its European counterpart this month. 

The development would further fuel Regrow’s ambitions to fetch higher value for its innovation which it claims is the only approved product (using the stem cell therapy) to treat early-stage avascular necrosis of bone joints. Necrosis is basically death of bone tissue due to interruptions in blood supply. Regrow expects to fetch something around $500 million in the out-licensing deal for the ODD product in the US. 

Orphan drug designation is given by the US Food and Drug Administration (USFDA) for rare diseases that affect less than 200,000 patients in the US. Since the market for drugs for rare diseases is small, some special incentives are extended to companies involved in the discovery and development of such drugs - which include a seven-year marketing exclusivity, waiver of prescription drug user fee (about $1.5 million per indication), and also some specific tax credits. 

Besides, these drugs may also be eligible for fast-track approval process that would facilitate an early market launch. Indian pharma majors like Hyderabad-based Natco Pharma, Ahmedabad’s Cadila Pharmaceuticals, apart from the largest Indian drug firm in the US, Sun Pharma, have earlier received ODD from the USFDA for some of their products (mostly in cancer therapy). Regrow is possibly the first Indian start-up to receive the same. 

Globally, Belgium’s Bone Therapeutics is working on a similar product based on bone cell therapy. According to recent media reports, the company stopped phase 3 trials of its autologous cell therapy for “futility”. The University of California, Davis, enabled start-up Molecular Matrix received FDA approval for a carbohydrate-based polymer that can help injured bones heal and regrow. It is, however, a synthetic bone graft substitute unlike Regrow’s natural product. 

The eight-year-old start-up is close to raising around $25 million from a US-based investor who is picking up a 21 per cent stake in Regrow Biosciences (thus valuing the firm at roughly around $125 million). Founder Yash Sanghavi (who had a pharmaceutical distribution business) said the funds would be used for conducting clinical trials in the US. “We would need around $20-21 million for the ‘Ossgrow’ clinical trials. We aim to start the trials around November this year,” he explained. 

Ossgrow is the trade name for the product to treat early stage avascular necrosis of the hip joint while Cartigrow is the product that helps treat cartilage loss in the knees, or early stage osteoarthritis. Cartilage loss in the knee, on the other hand, can occur on account of injury, early osteoarthritis etc. 

Getting the orphan drug status would be crucial for Regrow’s growth plans including out-licensing deals and pipeline product development. “We do not have the marketing muscle in the US to sell the product which could be done better by an MNC. Further, we have another four innovative products lined up and we would need funds for the development of those,” Sanghavi said. 

Back home Regrow has treated over 350 patients after it got the license from the Drug Controller General of India in 2017. The bootstrapped start-up has invested Rs 75 crore so far. 

 Sanghavi claimed that Ossgrow, Cartigrow etc can be called “living drugs”. 

The process is simple. One would take a bone marrow sample from the patient, then ship it through cold chain logistics, develop the tissue in its Lonavala laboratory, and then ship it back to the hospital where through a simple arthroscopic surgery the patient’s own cells are given back to the affected area that has seen either bone or cartilage damage. Unlike conventional joint implants, the process Sanghavi claimed was far more easier and had no side effects as the solution is naturally developed. After the bone cell implantation, new bone formation has been witnessed in the patients. 

These products have been developed in-house by a team of scientists led by Sanghavi’s son Satyen Sanghavi who studied stem cell research in the UK. In India the company has already applied for patent for the products

The firm has tied up with major hospital chains across India and leading orthopaedic surgeons. The product was launched in 2017 by Prathap C Reddy, founder chairman of Apollo. Both Ossgrow and Cartigrow are priced at Rs 140,000 for a single procedure. In the US, however, it is closing in on tying up with FDA approved contract manufacturers to manufacture Ossgrow.