Rhythm Pharmaceuticals modify design of ongoing MC4r treatment following FDA feedback

First I’ll start with an apology for being MIA; I’ve had the worst spell of migraine attacks. Unfortunately when I have an attack they can last anywhere between a few days to two weeks and I’ve been down for about two weeks. Still not 100% but on the mend.

I’ve had a few things I’ve wanted to talk about, but my brain feels like mush and I can’t recall what they were. However, there is some news in the MC4r world so that’s where I’m going to start because I feel like it’s been a while since we had any.

Our favourite Pharmaceutical company, Rhythm Pharmaceuticals have announced that it is modifying the design of their ongoing clinical studies that are evaluating its lead setmelanotide as a precision medicine for patients with genetic diseases of obesity.

The modified study of phase III trial and the phase II trial will focus on patients with rare genetic related diseases of obesity. The modifications follow recommendations from the FDA who have indicated additional clinical trials may be necessary to support registration of the drug as a treatment.

The decision to focus on rare patients with genetic obesity will reduce the target population of the drug, hurting its prospects. Investors are dismayed by the design of the study and the company saw a 16.3% decline in its share price on April 6.

Overall, not the best news for all my MC4r cohorts out there — I always thought having a rare genetic defect that affected my weight was bad. Only to now find out that the company developing the drug that could help so many people, are now narrowing the field (of what is already a pretty small field) even smaller to target rarer genes. You can understand why investors would start pulling out their money.

NOW, I’m not annoyed at Rhythm Pharmaceuticals because this decision has come off the back of feedback from the FDA. I’m not going to pretend I understand how the world of pharmaceuticals work and how the world of medicine works. But we’ve followed this study for more than a year, and for that time I had hope, not just for me but for a lot of other people and for a future where other children may not have to go through what I did. This news is a little disheartening.

However, if anyone else out there is also feeling a little sad by this. I’m going to give you a silver lining. This is not a loss or a defeat — we’re still at the starting line. Rhythm Pharmaceuticals is one company, and they’ve got the ball rolling, sure they’ve hit a hurdle but that’s all it is. I’d link to think, that they didn’t do ALL this research just to throw it away in the basis of a recommendation (I know I wouldn’t).

While the future of the setmelanotide is undetermined at the moment, let’s all hope that it’s successful. Even if it doesn’t help me, I hope it gets the chance to help someone.

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