When politics, climate change or international conflicts have me down, I turn to biotech instead. There’s always a new scientific discovery, a new drug for a rare disease, a new medical device for a common ailment.
Here are ten real biomedical wins from 2018 (with a contentious #10):
- Alnylam* won approval for the first drug that uses RNAi, a new modality to silence mutations that cause genetic diseases like hATTR amyloidosis.
- Catalent won approval for a novel opioid-withdrawal treatment, a critical success near to my heart in the midst of a relentless American opioid crisis.
- Immuno-oncology (IO) went global. Last week, I met a teacher who’d had stage III melanoma; the 3rd patient in a Sydney trial of nivo-ipi. Melanoma killed her cousin in 2013, but she was cured after just two IO infusions.
- For the first time, >50% of novel drug approvals targeted rare diseases.
- GW Pharmaceuticals* won approval for the first marijuana-derived drug, which treats two rare, deadly forms of epileptic seizures in young children.
- Moderna, the top biotech unicorn (discounting Samumed), had its IPO. The Form S-1 substantively discloses Moderna’s notoriously secret work on mRNA therapeutics, a boon to other researchers in an important new field.
- SIGA won approval for the first drug that treats smallpox, in case the virus ever escapes its holdouts in biowarfare arsenals and BSL-4 research labs.
- Martin Shkreli went to prison; Elizabeth Holmes will likely follow. House-cleaning bolsters the industry’s reputation and discourages malfeasance.
- Amarin* scored unexpectedly positive data for a purified fish oil therapy to lower triglyceride levels, reducing often lethal cardiac events by 25%.
- Scientists completed the first use of CRISPR to prevent a disease in babies, a key advance despite well-justified criticism. The team lacked competency and ethics and should be sanctioned. But if the babies are healthy, with a hopefully reduced risk of HIV, the pursuit will ultimately be a net positive.
So whatever else happened in 2018, the biomedical endeavor continues its steady progress, to the benefit of us all.
*Disclosure: I’ve owned shares in starred companies within the past 12 months.