SpringWorks spins out of Pfizer on rare disease mission

A new biotech spun out of Pfizer is to develop four drugs for rare and serious diseases.

SpringWorks is the brainchild of Lara Sullivan, a former Pfizer R&D strategy executive who gained Pfizer's blessing to set up the firm with four lower-priority assets from the company's pipeline.

The biotech starts out with a hefty $103 million in backing from Pfizer, the charity LifeArc (formerly known as MRC Technology), and investors Bain Capital Life Sciences, Bain Capital Double Impact and OrbiMed. SpringWorks aims to develop the four Pfizer drugs, and also plans to expand its pipeline by linking up with other companies and academic institutions.

Pfizer is hailing SpringWorks as a "groundbreaking new model" to accelerate drug development and beyond. The move is certainly unusual for big pharma, but is part of a wider pharma trend to increase the chances of overlooked or low priority molecules to be developed and reach patients.

[caption id="attachment_32120" align="alignnone" width="300"] Freda Lewis-Hall[/caption]

"We hope that our investment in SpringWorks Therapeutics will, over time, enable us to realise even more value for patients and society, said Freda Lewis-Hall, executive vice president and chief medical officer, Pfizer.

"SpringWorks Therapeutics started as an idea about a new way to get things done with and for patients, it's been a tremendous team effort, and we and our partners are excited to see it become a reality."

It will use links with a variety of stakeholders including scientists, biotechs, patient groups, funders and philanthropists.

Pfizer has granted licences that pay milestones and royalties on the experimental therapies.

While two of the drugs have shown promise in mid-stage trials, the other two have so far proved safe but have not produced satisfactory results in other uses.

The drugs under development are:

Nirogacestat (PF-03084014), which SpringWorks plans to take into phase 3 for desmoid tumours, a rare non-metastatic tumour of connective tissue cells that can cause severe morbidity, pain and loss of function in children adults. Current treatments include thermal ablation and surgery, which can be costly and dangerous.
The MEK 1/2 inhibitor PD-0325901 for neurofibromatosis, which cause tumors to grow on nerves throughout the body and can lead to blindness, deafness, disfigurement, cancer, bone abnormalities, learning disabilities and severe pain. SpringWorks plans phase 3 trials in collaboration with the Children’s Tumor Foundation.
Senicapoc (PF-05416266) for the rare genetic disorder, hereditary xerocytosis (HX), where red blood cells become dehydrated due to loss of potassium and cell water. After demonstrating good safety and tolerability in phase 1-3 studies in other uses, SpringWorks will assess its potential activity in HX.
The FAAH inhibitor PF-044578, which SpringWorks aims to develop for post-traumatic stress disorder (PTSD). SpringWorks is to join with Cohen Veterans Biosciences to assess patient populations that could benefit.

[caption id="attachment_32125" align="alignnone" width="180"] Lara Sullivan[/caption]

SpringWorks will be led by a team of industry veterans including executive chairman Daniel Lynch, with more than 25 years of industry experience, and vice president of clinical R&D Mary Smith, who led the development and approval of United Therapeutics’ neuroblastoma drug Unituxin.