RHYTHM PHARMACEUTICALS (RYTM) Company Report

Rhythm Pharmaceuticals received a three-month PDUFA extension for setmelanotide in Hypothalamic Obesity (HO) due to a major amendment request from the FDA, pushing the potential approval date to March 2026.
The company views the extended time as advantageous for commercial preparation, particularly for a rare disease launch like HO, which has an estimated 7,000 to 10,000 patients in the U.S..
Rhythm Pharmaceuticals expects to release data for setmelanotide in Prader-Willi Syndrome (PWS) before the Christmas break, with the CEO assigning a 50-50 probability of success due to the disease's complexity.
The company is also developing next-generation assets, including 718, a more potent and specific MC4R agonist, with enrollment for the open-label first part expected to complete in Q1 2026 and data readout by mid-year 2026.

Nov 12, 2025, 2:30 PM

Transcript

Rhythm Pharmaceuticals Provides Update on AHO Approval Timeline and Pipeline

RYTM

Product Launch

New Projects/Investments

Guidance Update

Rhythm Pharmaceuticals' CEO discussed the MC4 axis biology and its benefits in genetic and hypothalamic obesity (AHO), noting that the number of conditions affecting this pathway is larger than initially thought.
The PDUFA date for AHO approval was extended to March 2025 due to an FDA request for a sensitivity analysis, which showed no difference in results. The commercial team is fully hired and the additional three months are considered advantageous for the rare disease launch.
The estimated patient population for AHO in the U.S. is now 7,000-10,000, with growing confidence based on claims analyses. Setmelanotide has demonstrated better efficacy in AHO patients compared to GLP-1s, even in those who previously did not respond to GLP-1s.
The company plans to release Prader-Willi Syndrome (PWS) data before Christmas break and aims to complete enrollment for the open-label part of its next-generation asset, 718, in Q1 2026, with results expected by mid-year.

Nov 12, 2025, 2:30 PM

Transcript

Rhythm Pharmaceuticals Discusses PDUFA Extension and Pipeline Updates

RYTM

Guidance Update

New Projects/Investments

Rhythm Pharmaceuticals received a three-month PDUFA date extension for its AHO application, pushing the decision to March 20, 2026, due to a major amendment request from the FDA. The company views the additional time as advantageous for preparing the commercial organization for launch.
The company estimates the hypothalamic obesity (HO) patient population in the U.S. to be between 7,000 and 10,000, with increasing confidence driven by claims analyses.
Management emphasized the differentiation of setmelanotide from GLP-1s in treating HO, noting that setmelanotide corrects the underlying hormonal deficiency, leading to significant weight loss where GLP-1s often show modest or no response.
Rhythm plans to release Prader-Willi syndrome (PWS) data before the Christmas break and expects to complete enrollment for the open-label part of the 718 trial in Q1 2026, with data readout by mid-year.
The company is also excited about commercial opportunities in Europe and Japan, noting similar epidemiology to the U.S. in Europe and a surprisingly high prevalence in Japan.

Nov 12, 2025, 2:30 PM

Transcript

Rhythm Pharmaceuticals Presents Positive Setmelanotide Data at ObesityWeek 2025

RYTM

New Projects/Investments

Rhythm Pharmaceuticals presented four data sets at ObesityWeek® 2025, highlighting positive results for its lead asset, setmelanotide.
Phase 3 TRANSCEND trial data showed setmelanotide achieved significant BMI reductions in patients with acquired hypothalamic obesity, including a -27.1% mean placebo-adjusted difference when used with GLP-1 therapy and -19.0% without GLP-1 therapy.
The presentations also indicated that setmelanotide treatment led to meaningful improvements in cardiometabolic health and patient-reported outcomes, such as hunger, weight, energy levels, and physical activity.
Setmelanotide (IMCIVREE®) is currently approved in the U.S., EU, and UK for specific rare neuroendocrine diseases causing obesity, and Rhythm is advancing its clinical development for other rare diseases.

Nov 10, 2025, 12:00 PM

Press Release

Rhythm Pharmaceuticals: FDA Extends IMCIVREE Review Period

RYTM

Product Launch

New Projects/Investments

The U.S. Food and Drug Administration (FDA) has extended the review period for Rhythm Pharmaceuticals' supplemental New Drug Application (sNDA) for IMCIVREE® (setmelanotide) for the treatment of acquired hypothalamic obesity.
The Prescription Drug User Fee Act (PDUFA) goal date has been extended by three months, from December 20, 2025 to March 20, 2026.
This extension was due to the FDA requesting additional sensitivity analyses of clinical efficacy data from Rhythm’s Phase 3 pivotal trial, which was deemed a ‘major amendment’.
No new data relating to the safety or manufacturing of setmelanotide was requested by the FDA.

Nov 7, 2025, 1:09 PM

8-K

Rhythm Pharmaceuticals Announces FDA Review Extension for IMCIVREE

RYTM

Product Launch

New Projects/Investments

The U.S. Food and Drug Administration (FDA) has extended the review period for Rhythm Pharmaceuticals' supplemental New Drug Application (sNDA) for IMCIVREE® (setmelanotide), intended for the treatment of acquired hypothalamic obesity.
The Prescription Drug User Fee Act (PDUFA) goal date has been moved by three months, from December 20, 2025, to March 20, 2026.
This extension is due to the FDA's request for additional sensitivity analyses of clinical efficacy data from Rhythm's Phase 3 pivotal trial, which was deemed a 'major amendment'.
No new data related to the safety or manufacturing of setmelanotide was requested by the FDA.

Nov 7, 2025, 12:30 PM

Press Release

Rhythm Pharmaceuticals Secures Public Reimbursement for IMCIVREE in Canada

RYTM

Product Launch

New Projects/Investments

Rhythm Pharmaceuticals (Nasdaq: RYTM) has entered into Product Listing Agreements for the public reimbursement of IMCIVREE in six Canadian provinces (Ontario, Alberta, British Columbia, Saskatchewan, Nova Scotia) and with the Federal Non-Insured Health Benefits (NIHB) Program.
These agreements provide access to IMCIVREE for weight management in eligible adult and pediatric patients aged 6 years and older with clinically or genetically confirmed Bardet-Biedl syndrome (BBS) and obesity.
IMCIVREE was approved by Health Canada in May 2023 and was recently added to the common list of new drugs for rare diseases as part of the Government of Canada's National Strategy for Drugs for Rare Diseases.

Nov 5, 2025, 9:01 PM

Press Release

Rhythm Reports Q3 2025 Results, Anticipates Key Regulatory Decisions and Data Readouts

RYTM

Earnings

Guidance Update

Product Launch

Rhythm (RYTM) reported Q3 2025 global InSibiri revenue of $51.3 million, marking a 6% sequential increase from Q2 2025, driven by a 10% global increase in patients on reimbursed therapy.
The company ended Q3 2025 with $416.1 million in cash on hand, providing at least 24 months of runway, following a $189.2 million equity offering completed in July.
Rhythm anticipates a December 20th PDUFA date for FDA approval of InSibiri in acquired hypothalamic obesity (HO) and expects preliminary phase 2 data from its Prader-Willi syndrome (PWS) trial by the end of 2025.
For fiscal year 2025, the company tightened its non-GAAP operating expense guidance to $295-$315 million.

Nov 4, 2025, 1:00 PM

Transcript

Rhythm Pharmaceuticals Reports Strong Q3 2025 Growth and Prepares for Hypothalamic Obesity Launch

RYTM

Earnings

Product Launch

Guidance Update

Rhythm Pharmaceuticals reported Q3 2025 global InSibiri revenue of $51.3 million, marking a 6% sequential increase from Q2 2025 and a 54% year-over-year increase from Q3 2024, driven by a 10% increase in patients on reimbursed therapy globally.
The company concluded Q3 2025 with $416.1 million in cash on hand, which is projected to fund planned operations for at least 24 months.
Preparations are underway for the launch of setmelanotide in acquired hypothalamic obesity (HO), with the FDA PDUFA date set for December 20th and potential European marketing authorization anticipated in the second half of 2026.
For fiscal year 2025, Rhythm updated its non-GAAP operating expense guidance to $295 million-$315 million, with non-GAAP R&D expenses projected at $150 million-$165 million and non-GAAP SG&A expenses at $145 million-$150 million.
Preliminary results from the exploratory phase two trial in Prader-Willi syndrome are expected by the end of 2025.

Nov 4, 2025, 1:00 PM

Transcript

Rhythm Pharmaceuticals Reports Q3 2025 Results and Provides Updates on Pipeline and HO Launch Preparations

RYTM

Earnings

Guidance Update

Product Launch

Rhythm Pharmaceuticals reported global Imcivree revenue of $51.3 million for Q3 2025, marking a 6% sequential increase from Q2 2025, driven by a 10% increase in patients on reimbursed therapy globally.
The company concluded Q3 2025 with a strong balance sheet, holding $416.1 million in cash on hand, which is projected to provide a cash runway of at least 24 months.
Preparations are underway for the potential launch of Imcivree for acquired hypothalamic obesity (HO), with an FDA PDUFA date set for December 20th and anticipated European approval in the second half of 2026.
Preliminary results from the exploratory Phase II trial in Prader-Willi syndrome are expected to be reported by the end of 2025.
For fiscal year 2025, Rhythm Pharmaceuticals tightened its non-GAAP operating expense guidance to a range of $295 million to $315 million.

Nov 4, 2025, 1:00 PM

Transcript

Earnings Transcripts

Quarterly earnings call transcripts for RHYTHM PHARMACEUTICALS.

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