From Rhythm Pharmaceuticals (RYTM) we learned that positive, top-line results from two pivotal Phase 3 clinical trials evaluating setmelanotide, for pro-opiomelanocortin (POMC) and leptin receptor (LEPR) deficiency obesities, have met their primary endpoints and all key secondary endpoints. The product demonstrated a statistically significant and clinically meaningful effect on weight loss and reductions in insatiable hunger, or hyperphagia, in patients with POMC and LEPR deficiency obesities.
Eight of 10 patients with POMC deficiency obesity achieved the primary endpoint of greater than 10% weight loss over approximately one year.
The mean reduction from baseline in body weight for POMC deficiency obesity patients was-25.4%.
The mean reduction from baseline in most hunger rating for POMC deficiency obesity patients was -27.8%.
Of the POMC Deficiency Obesity
- 50% of patients in the trial met or exceeded a 25% improvement in self-reported hunger scores.
- Mean weight loss for these patients was 31.9 kg, or 70.2 pounds, over one year on therapy.
Of the LEPR Deficiency Obesity
- More than half (5 of 11) patients achieved the primary endpoint of greater than 10% weight loss over one year.
- The mean reduction from baseline in body weight for LEPR deficiency obesity patients was -12.5%.
- The mean reduction from baseline in most hunger rating for LEPR deficiency obesity patients was -41.9%.
- In addition, 72.7% of the LEPR deficiency obesity patients in the trial met or exceeded a 25% improvement in self-reported hunger scores.
- Mean weight loss for these patients was 16.7 kg, or 36.8 pounds over one year on therapy.
Setmelanotide vs Placebo
Upon entry into a placebo period the patients have almost immediately gained weight and experienced an increase in hunger; reversing their downward trends in weight loss and hunger scores observed during the first 12 weeks of the treatment period. In both trials, the mean weight increase during the four-week placebo period was approximately 5 kg or more than 11 pounds and this weight gain was accompanied by a worsening in hunger scores.
Consistent with prior clinical experience, setmelanotide was generally well-tolerated in both trials. Treatment-related adverse events (AEs) included: injection site reactions, nausea and vomiting, and increased hyperpigmentation (darkening of the skin); these were consistent with prior clinical trials of setmelanotide.
There were no reports of cardiovascular AEs related to setmelanotide.
One LEPR study patient withdrew before the end of titration due to AE of mild hypereosinophilia.
Bottom line, there were no serious adverse events (SAEs) with setmelanotide.
One LEPR study patient died from injuries unrelated to the study drug. This patient was a passenger in a car accident who died from injuries from the accident.
This is good news for Rhythm Pharmaceuticals; a company that we added to the Prohost Portfolio on October 25, 2017, at $22. As usual, we ignored the traders’ buying and selling games; selling at each and every quarterly financial result. We also ignored the stock decline that used to occur each time the market sinks; and the market has done it several times in 2019.
Today, while losing over 360 points, the market could not prevent Rhythm’s stock gaining $3.55, following the announcement of the impressive results from Phase 3 trial of the firm’s product setmelanotide treating POMC and LEPR deficiency obesities.
We also see the good news that Rhythm is on track to complete submission of a rolling New Drug Application (NDA) to the U.S. FDA that will cover both POMC and LEPR deficiency obesities late in the fourth quarter of 2019 or the first quarter of 2020.
Genetically-driven, severe obesity and insatiable hunger needed effective treatments. Rhythm’s successful, pivotal data makes us believe that Rhythm’s product setmelanotide, and other products in this firm’s pipeline, have the potential for effective treatment for the yet to be treated obesities.
Rhythm anticipates sharing the full data, from these Phase 3 clinical trials in POMC and LEPR deficiency obesities, in a forthcoming publication and at a presentation at an upcoming medical meeting.
This is excellent news for the obese and, from now on, for Rhythm’s shareholders.
The Manipulation or Mishandling of Preclinical Data Has Nothing to Do with the Safety and Efficacy of Its Gene Therapy Zolgensma for Spinal Muscular Atrophy.
Indeed, the sales of the Novartis (NVS) gene therapy drug Zolgensma will not be affected; as the FDA and the company informed that the drug is safe and effective and will remain on the market. The mishandling of the data is related to preclinical trials conducted on mice.
The FDA has decided to continue investigating the mishandling, or mismanagement, of the data, which again, we repeat, has nothing to do with the gene therapy efficacy and its sales. There is nothing in the whole story to cause any losses to Novartis’ shareholders. We also heard that it was Novartis who discovered the mishandling and informed the FDA.
The rush by some to sell the stock has offered an opportunity, for those who believe in Novartis, to accumulate on weaknesses.
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