San Francisco-based RDMD Inc. netted $14 million in series A money fueling an approach designed to generate clinical evidence that will speed drug development in rare diseases. “We’re building the platform that can connect the patients and researchers,” co-founder and CEO Nancy Yu told BioWorld. In a scenario with “only maybe thousands or tens of thousands of patients, a handful of researchers and no drugs previously approved by the FDA, everyone in the community is starting from scratch,” she said.

Not anymore. RDMD signs up patients who volunteer to share their data and then does “all the work behind the scenes to contact their facilities [and] to structure the data into a useable format that drug researchers can use in a de-identified way,” Yu said, adding that the company is “not just relying on survey questions. We’re looking at all the deep clinical evidence from within the medical record, from the physician’s notes, from progress notes, from lab reports, genetics reports – everything.” Although the firm is focused first on neurological, genetic and metabolic disorders, “the list continues to expand,” she said. “At the end of the day, what we’re really trying to build is a platform that can characterize new conditions in a way that’s standardized, in a way that’s ethical and private. We really want to make this a replicable platform and model.”

In rare diseases, “it’s difficult to go the traditional way to get information from hospitals, because patients are seeing various different specialists and they’re all across the country and world,” Yu said. “Our model tries to empower the patients who want to be engaged, who want to be involved, who are willing to participate, and give them that option. We’re very transparent with patients up front. We are working with drug companies to share their de-identified information – they know that from day one.” In most cases, RDMD works with patient advocacy groups, though the firm has “jumpstarted” conditions where no such group exists, she said. “There are usually a few doctors who can lead the charge,” as well as patient ambassadors who get busy rounding up more participants via social media.

The patient volunteer aspect is important, Yu said, and RDMD’s philosophy is similar to that of genetic testing firm 23andme Inc., the San Francisco concern where she previously worked. In the summer of 2018, 23andme and London-based Glaxosmithkline plc (GSK) kicked off a four-year collaboration, with an option to extend into a fifth year, that included a $300 million equity investment by the pharma player. GSK became 23andme's exclusive collaborator for drug target discovery programs. A joint team is using the two entities' combined resources to find new targets, based on collected data and partly on findings already made by 23andme. The latter had started programs in autoimmune disease, cancer immunotherapy, cardiovascular disease, osteoarthritis and liver disease. More recently, 23andme placed its might behind an effort in COVID-19. The company said it is “collecting data through baseline and longitudinal surveys targeted to both symptomatic and asymptomatic individuals.” Next, researchers will conduct a genome-wide association study to determine if any genetic variants are associated with severity of COVID-19 symptoms or disease susceptibility.

RDMD will use the infusion of series A money to expand into 20 more rare diseases and launch studies in the new conditions, with an eye to better understanding the natural progression of each. Partnerships with biopharmaceutical customers are expected to grow, too, and key leadership hires are in the offing. There’s an international push as well. “We recently expanded into Canada, and we’re planning to expand into Europe,” Yu said, citing “a lot of challenges when you’re looking at research in particular,” including matters related to privacy, research ethics and institutional review boards, not to mention simple translation. Asked how soon RDMD might need to raise more money, she pointed to estimates commonly made by other firms of 18 to 24 months as “probably similar to how we’re thinking about it, but it’s too early to say.”

The company was founded in 2018 with $3 million in seed funding. Regarding RDMD’s name, Yu said officials were “trying to make it have multiple meanings. In rare disease [RD], we’re looking at what’s collected during routine clinical care [by M.D.s] and using that to drive research.” Such doctors “really in some ways are doing research, because there is no treatment,” she noted.

A decade ago, the field of rare diseases was “much more quiet,” Yu said. “Since then, a lot of things have driven these efforts,” such as FDA incentives that include longer marketing timelines and the allowance of smaller trials to gain approval. But what’s powering the research most forcefully, she said, is better science, with lower-cost sequencing that permits identification and diagnosing of rare diseases, 80% of which are genetic.

Spark Capital led the series A round, joined by existing seed investors Lux Capital, Village Global and Garuda Ventures, and new backer Maveron Capital. Angel investors representing a diverse group of executives from various health care technology, biopharmaceutical and technology companies, as well as patient foundations, also chipped in. Nabeel Hyatt, general partner at Spark, joined the company’s board.

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