With a goal to stamp out a rare disease that takes the lives of about one-fifth of the children it affects, biotechnology company Glycomine is finding deep-pocketed support from investors including Danish pharmaceutical giant Novo Nordisk.
The San Carlos, California company announced recently that it raised $33 million for its experimental therapy for PMM2-CDG (CDG-1a), the most common of a group of inherited diseases that in infants weakens muscle tone, delays development and causes eyes to look in different directions. It kills about 20% of affected children, and is diagnosed in about 1,000 people worldwide. No FDA approved treatment is available.
Novo Holdings A/S, the Danish holding company for drugmaker Novo Nordisk A/S and biotech Novozymes A/S, led the financing round, which will advance clinical trials. Funding also came from Asahi Kasei Pharma Corp. and Mission Bay Capital, and previous investors Sanderling Ventures and Chiesi Ventures. Glycomine raised $12 million in an early funding round in 2016.
“Glycomine’s novel therapy offers great promise to address a critical unmet medical need,” said Kenneth Harrison, a principal at Novo Ventures, Novo Holdings’ venture firm focusing on the life sciences sector. “We believe that Glycomine’s approach has the potential to directly address many of the most debilitating symptoms of PMM2-CDG.”
PMM2-CDG (CDG-1a), which has no known treatment, is a so-called orphan disease, a condition that affects so few people that decades ago it would have been ignored by major drugmakers because there was little chance of making a profit. That changed in 1983, when the U.S. passed a law providing tax credits for costs of clinical research, government grant funding, assistance for clinical research, and a seven-year period of exclusive marketing to encourage development of medicines for orphan diseases.
“Glycomine’s novel therapy offers great promise to address a critical unmet medical need,” said Kenneth Harrison, a principal at Novo Ventures, Novo Holdings’ venture firm focusing on the life sciences sector. “We believe that Glycomine’s approach has the potential to directly address many of the most debilitating symptoms of PMM2-CDG.”
The law has resulted in more than 250 drugs for orphan diseases, according to the Federal Drug Administration.
“There are about 7,000 rare disorders out there, and 95% of them don’t have therapeutic options,” said Agnes Rafalko, Glycomine’s founder and chief executive officer, told the San Francisco Business Times in June. “The technology we’re working on is very interesting and wanted by Big Pharma.”
Other biotechs, such as Arrowhead Pharmaceuticals, are working on treatments for somewhat similar diseases, albeit with different approaches and targets than Glycomine.
Christopher Starr, Glycomine’s executive chairman, said a company he co-founded in 1997, BioMarin Pharmaceutical, had a similar approach to Glycomine’s but was focused on treating a different group of disorders.
Karma Take: While promising, Glycomine’s therapy is in such an early stage of testing that only the savviest — or bravest — investor should take a chance.
