Big Pharma needs more—not fewer—bold bets on rare and difficult diseases after Novartis’ efforts to invest in muscular dystrophy and ALS hit major roadblocks this month.
If I had $12 billion, I’d like to think that—after refinishing my kitchen and perhaps buying a Birkin bag—I might do what Novartis did in buying Avidity Biosciences.
Here was a biotech developing a drug for a devastating genetic disorder called Myotonic dystrophy type 1 (DM1) that robs children and adults of muscle control. The disease weakens the muscles and can impact the gastrointestinal tract, uterus and the muscles used for breathing. Life expectancy is dramatically shortened in people with this form of the disease.
Avidity offered a shot at an effective treatment with del-desiran, an antibody-oligonucleotide conjugate that targets a disease-causing protein called DMPK. Unfortunately, almost a year after the buyout, the therapy failed a Phase 3 trial, unable to significantly improve hand opening time in patients.
Much of the deal’s value was attributed to this asset. Although Avidity offered a deeper pipeline including candidates for Duchenne muscular dystrophy (DMD) and facioscapulohumeral muscular dystrophy (FSHD), analysts hailed the del-desiran miss as a “$12B disappointment.”
But this deal is what we hope for: a company like Novartis getting into a rare disease and putting the massive Big Pharma machine behind the effort.
To its credit, Novartis has also devoted resources toward another devastating rare disease, ALS. Unfortunately, the pharma suffered a blow to that program this week as well, with the TREM2 stabilizer lifonebart failing to improve survival without a ventilator.
The failure is disappointing, but think about that endpoint for a minute: survival without a ventilator. The stakes couldn’t be higher.
These failures are more than just business bloopers. They’re a damn shame. But they are not a red flag to stop trying.
Yes, rare disease is a big risk, but we want biotechs, investors, entrepreneurs and Big Pharma to get the signal that it’s viable. We need more big swings or else we will never have more to offer patients like those with DM1.
To be clear, there is no unimportant disease. Disease is human suffering, and if you believe in the promise of the life sciences industry, you must believe that its role is to alleviate human suffering of all kinds.
But many diseases have decent treatment options now. That’s exciting; it’s a triumph. We need that same spirit to address those ailments that still carry a high unmet need.
Novartis tried. Others have too. I implore these companies not to give up.
I know that pharma is a business and investors need to see returns to recycle their money back into the industry. But if any of you are reading this, I beg of you, keep betting. Keep exploring what’s possible. Sometimes you won’t succeed, but the day you do, you’ll have achieved much more than a fat return. You might just give a patient and their family hope, relief—even a second chance at life.
