Rhythm Pumps $100M Gained in Voucher Sale Back Into Imcivree
In a $100 million deal, Rhythm Pharmaceuticals is selling its rare pediatric disease priority review voucher (PRV), granted by the U.S. Food and Drug Administration (FDA) with the approval of Imcivree (setmelanotide) to treat three rare genetic obesity disorders.
Imcivree recently became the first therapy approved to treat adults and children 6 and older with obesity caused by the deficiency of POMC, PCSK1, or LEPR, all genes involved in the appetite and weight-regulating melanocortin-4 receptor (MC4R) pathway.
“Capital from the sale of our PRV provides an important source of additional funding to advance the continued development of setmelanotide as a precision medicine for people whose severe obesity and insatiable hunger may be caused by genetic variants associated with the [MC4R] receptor pathway,” David Meeker, MD, president and CEO of Rhythm, said in a press release.
The PRV conveys regulatory incentives to promote the development of new therapies to prevent and treat rare pediatric diseases. Upon issue, a PRV can be redeemed to receive priority review for future marketing applications or sold to other companies without limitation. Under the terms of the definitive agreement announced, Rhythm will receive $100 million upfront when the transaction is concluded.
Imcivree activates the MC4R pathway, boosting appetite regulation and weight control. FDA approval late last year was based on positive results from the largest Phase 3 trials to date to study Imcivree as a treatment for genetic obesity caused by POMC/PCSK1 deficiency (NCT02896192) or LEPR deficiency (NCT03287960).
Top-line data from 21 participants showed that Imcivree, administered by daily subcutaneous (under the skin) injections, reduced weight by at least 10% in 80% of POMC/PCSK1 deficiency patients and 45.5% of those with LEPR deficiency.
Those with POMC/PCSK1 deficiency lost an average of 31.9 kg (70.2 lbs), while participants with LEPR deficiency lost an average of 16.7 kg (36.8 lbs). More than half of the patients experienced a reduction in hunger of at least 25%, and a reduction in body mass index was seen in 22.3% to 49.2% of POMC/PCSK1 deficiency patients, and 10.6% of people with LEPR deficiency.
Another Phase 3 trial (NCT03746522) is assessing Imcivree in 38 participants, ages 6 and older, with moderate to severe obesity caused by two other genetic obesity disorders, Bardet-Biedl syndrome (BBS) or Alström syndrome. One year of Imcivree’s use was seen to significantly significantly reduce weight and appetite in BBS patients 12 and older. In those with Alström syndrome, the data trended toward a reduction in weight and appetite.
Rhythm plans to complete an analysis of full trial results in March, and to present the data soon after.
The company expects Imcivree to be available to eligible patients in the U.S. early this year.
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