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Mark Cuban Unexpected Leader in Orphan Drug Development Reform

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Copyright © DrugPatentWatch. Originally published at Mark Cuban Unexpected Leader in Orphan Drug Development Reform

Businessman and investor Mark Cuban is probably best known as the owner of the Dallas Mavericks and as an investor on the reality television series Shark Tank. His business and investment history encompasses many industries, including software, media, and a range of technology startups.

JD Lasica /

Mark Cuban has invested in a San Francisco startup that is developing treatments for rare diseases.

Cuban also feels passionately that drug prices are too high, and that pricing strategies for drugs need to change. But rather than just sounding off about high drug prices, he recently invested in San Francisco biotech firm Perlara, a company that works to develop both near-term treatments and cures for rare diseases – those that are often left behind in a world where drug patents are seen as vehicles for maximizing profitability.

Challenges in Developing Drugs for Rare Diseases

In addition to investing a quarter million dollars into Perlara’s last fundraising round, Mark Cuban invested an undisclosed amount into one of the company’s “PerlQuests,” a project designed to find treatments for Leigh syndrome, an extremely rare and devastating neurological disorder that affects infants. 

PerlQuest CEO Ethan Perlstein says the company stakes its brand on bringing down drug pricing, and though the company is interested in pursuing drug patents for novel treatments of rare diseases, it’s also seeking nearer-term solutions. 

PerlQuest: A Different Approach to Treating Rare Diseases

Perlstein is well aware of the outstanding promise of gene therapy, but knows that medical treatments based on gene therapy aren’t financially feasible for most people. Therefore, PerlQuest will also seek nearer-term solutions at lower cost by testing existing drugs to see if they may have beneficial therapeutic activity against rare diseases. 

Repurposing existing compounds only costs a fraction of what developing new drugs does. Perlstein’s hope is that therapies can be developed to treat rare diseases to buy time while research on cures also proceeds. Cuban is enthusiastic about their approach, telling CNBC of his pledge to “significantly reduce the pricing of all orphan drugs we invest in.”

Cuban: Expect ROI, but Not Necessarily Maximum ROI

Expecting ROI on rare drug treatments makes sense, says Cuban, but not maximizing ROI without regard to human life.

Mark Cuban would not have achieved his level of wealth by forgetting about ROI. But as for his investment in Perlara, he says, “[W]e aren’t looking to maximize ROI. We are looking to maximize health and wellness.”

He went into a bit more detail in a prior interview: “I’m all for capitalism at its purest, but it requires capitalists acting like human beings first.” Otherwise, healthcare becomes unaffordable and people die or otherwise don’t get the care they need. He also says that when capitalism proceeds without a grounding sense of humanity, the ultimate result is increased government regulation – something markets and investors want to avoid. 

Getting “Bad Actors” in Pharmaceutical Space in Line

Getting the “bad actors” in pharmaceuticals in line, says Cuban, won’t work if you appeal to empathy, because what he calls “inhumane capitalism” does not respond to requests for empathy. Rather, he says, pressure from shareholders, insurance companies, and the general public, plus the threat of limiting legislation will be what it takes to align strong pharmaceutical research with fair drug price schemes. He doesn’t consider the pressure to act with basic humanity as a constraint on investment, because billions can be made in pharmaceuticals, and nothing is going to change that. 

Drug patents and drug prices – especially for treatments for rare diseases – present challenges to scientists, investors, and the FDA. Mark Cuban’s investment in Perlara indicates that he sees promise in how some startups approach rare disease treatment, an approach that blends shorter-term treatments based on repurposed existing drugs, as well as development of the high-tech drugs that may someday offer cures.

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Copyright © DrugPatentWatch. Originally published at Mark Cuban Unexpected Leader in Orphan Drug Development Reform
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