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This Day That Year: Rhythm Pharma

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Shares of Rhythm Pharmaceuticals Inc. (RYTM) are down nearly 28% from their 52-week high of $37.23 recorded on June 27, 2018.

It was on this day last year, i.e. on October 5, 2017, that Rhythm Pharma debuted on the NASDAQ Global Market by setting a public offering at $17.00 per share.

Rhythm Pharma is a biopharmaceutical company developing therapies for the treatment of rare genetic disorders of obesity. The Company's lead drug candidate is Setmelanotide, under two phase III clinical trials in pro-opiomelanocortin (POMC) and leptin receptor (LEPR) deficiency obesity, two ultra-rare genetic disorders.

POMC deficiency is a rare condition caused by mutations in the POMC gene. This condition is characterized by severe obesity that begins at an early age, low levels of adrenocorticotropic hormone, and red hair and pale skin. (Source: NIH).

Leptin receptor deficiency, a rare cause of obesity, is caused by mutations in the LEPR gene. Affected individuals are of normal weight at birth, but they are constantly hungry and quickly gain weight. (Source: NIH)

The FDA has granted both Breakthrough Therapy Designation and Orphan Drug Designation for Setmelanotide in POMC and LEPR deficiency obesity.

The Company has completed enrollment in the two separate, ongoing, registration-enabling phase III trials of Setmelanotide in pro-opiomelanocortin (POMC) and leptin receptor (LEPR) deficiency obesity.

Initial data from the two phase III trials in both POMC and LEPR deficiency obesity are expected in the third quarter of 2019.

Setmelanotide is also being evaluated in four additional rare genetic disorders of obesity - Bardet-Biedl Syndrome, Alström Syndrome, POMC epigenetic disorders and POMC heterozygous deficiency obesity.

Last month, Rhythm Pharma presented updated data from its ongoing phase II basket studies evaluating Setmelanotide in people with Bardet-Biedl Syndrome (BBS) and Alström Syndrome.

Bardet-Biedl Syndrome and Alström Syndrome are life-threatening, ultra-rare orphan diseases that are closely related. Both cause severe obesity and insatiable hunger, also known as hyperphagia, which often present during childhood.

The updated data from the phase II basket studies showed continued reductions in body weight and decreased appetite in patients with the two rare genetic disorders of obesity.

The Company plans to initiate and enroll the first patients in a combined pivotal phase III trial evaluating Setmelanotide in patients with Bardet-Biedl Syndrome and Alström Syndrome by the end of 2018.

In April of this year, Rhythm Pharma acquired exclusive, worldwide rights from Takeda Pharma to develop and commercialize RM-853, a potent, orally available ghrelin o-acyltransferase (GOAT) inhibitor currently in preclinical development for Prader-Willi Syndrome.

Prader-Willi Syndrome is a rare genetic disorder that results in hyperphagia and early-onset, life-threatening obesity, for which there are no approved therapeutic options.

Rhythm plans to complete preclinical studies of RM-853 and advance RM-853 into clinical studies in the first quarter of 2020.

Key Numbers:

Accumulated deficit: $141.12 million
Cash on hand: about $287.6 million
% held by Institutions: 89.82%*
% held by Insiders: 9.57%*

(As on June 30, 2018, *Data provided by Thomson Reuters)

Shares of Rhythm Pharma have traded in a range of $16.80 to $37.23 over the last 1 year. The stock closed Thursday's trading at $26.98, down 6.51%.

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