Rising Genetic Diagnosis Will Expand Rare Disease Treatment Pipelines

Key Takeaways

• Expansion into new indications and age groups, alongside global diagnostic trends, positions Rhythm for sustained patient base and revenue growth.
  
• Strong intellectual property and regulatory advantages ensure high margins, pricing power, and long-term resilience against competitive threats.
  
• Heavy losses, high spending, overreliance on a single drug, regulatory risks, and early-stage global expansion threaten revenue growth, margin improvement, and long-term value.

Catalysts

A commercial-stage biopharmaceutical company, focuses on the rare neuroendocrine diseases.

• Rhythm is well-positioned to benefit from the rising global prevalence and improved diagnosis rates of rare genetic obesity disorders, supported by increasing physician education and advances in genetic testing-trends that are likely to drive sustained expansion of its addressable patient base and future revenue growth.
  
• Upcoming potential regulatory approvals and launches for setmelanotide (IMCIVREE) in new indications like acquired hypothalamic obesity and Prader-Willi syndrome, alongside expansion into younger age groups, are set to materially grow Rhythm's commercial opportunity and topline over the next several years.
  
• Extension of intellectual property protection to 2034 for the lead asset and to 2040+ for next-generation compounds, along with orphan drug exclusivity incentives in the U.S. and EU, create a multi-year window of strong pricing power and high net margins, protecting earnings from generic/biosimilar erosion.
  
• Accelerated regulatory pathways and strong reimbursement environments for rare disease therapies, as evidenced by early access programs and favorable payor positioning in multiple countries, enhance Rhythm's ability to rapidly scale international sales and diversify revenue streams, supporting long-term earnings resiliency.
  
• Progress in the development and potential future approval of next-generation assets (bivamelagon, RM-718) underscores Rhythm's potential to expand its rare disease pipeline, improve product lifecycle, and increase both revenue mix and long-term profitability through more specialized, higher-margin therapies.

Rhythm Pharmaceuticals Future Earnings and Revenue Growth

Assumptions

• Analysts are assuming Rhythm Pharmaceuticals's revenue will grow by 68.1% annually over the next 3 years.
• Analysts assume that profit margins will increase from -120.6% today to 29.8% in 3 years time.
• Analysts expect earnings to reach $221.2 million (and earnings per share of $3.33) by about August 2028, up from $-188.4 million today.
• In order for the above numbers to justify the analysts price target, the company would need to trade at a PE ratio of 45.7x on those 2028 earnings, up from -32.6x today. This future PE is greater than the current PE for the US Biotechs industry at 16.9x.
• Analysts expect the number of shares outstanding to grow by 4.55% per year for the next 3 years.
• To value all of this in today's terms, we will use a discount rate of 6.78%, as per the Simply Wall St company report.

Rhythm Pharmaceuticals Future Earnings Per Share Growth

Risks

• Rhythm Pharmaceuticals continues to report operating losses despite increased revenues, and ongoing high R&D and SG&A expenses suggest persistent negative net earnings and potential future dilutive capital raises, which can pressure shareholder value and limit net margin improvement.
  
• The company's heavy reliance on its lead asset, setmelanotide, introduces substantial risk; any safety, efficacy, or competitive challenges (e.g., from new drugs or generics post-2032/2034 patent expiration) could rapidly erode revenues and future earnings streams.
  
• Rhythm's international expansion is still in early stages with limited commercial infrastructure; unpredictable named patient sales, reliance on country-specific reimbursement and early access programs, and variable launch timelines outside core markets may constrain revenue scaling and impact medium-term profitability.
  
• Rising regulatory scrutiny, potential global drug pricing reforms, and movement toward value-based healthcare, particularly affecting high-cost rare disease therapies, could compress Rhythm's pricing power, threaten reimbursement terms, and put sustained revenue and long-term net margins at risk.
  
• Emerging competition from alternative biotechnologies (e.g., gene & RNA therapies), uncertainty in advancing pipeline programs (successful Phase II/III transition rates), and the risk of shifting payer/provider preferences away from current peptide-based modalities may divert demand and reduce future earnings potential.

Valuation

• The analysts have a consensus price target of $110.714 for Rhythm Pharmaceuticals based on their expectations of its future earnings growth, profit margins and other risk factors. However, there is a degree of disagreement amongst analysts, with the most bullish reporting a price target of $135.0, and the most bearish reporting a price target of just $80.0.
  
• In order for you to agree with the analyst's consensus, you'd need to believe that by 2028, revenues will be $742.1 million, earnings will come to $221.2 million, and it would be trading on a PE ratio of 45.7x, assuming you use a discount rate of 6.8%.
  
• Given the current share price of $92.48, the analyst price target of $110.71 is 16.5% higher.
  
• We always encourage you to reach your own conclusions though. So sense check these analyst numbers against your own assumptions and expectations based on your understanding of the business and what you believe is probable.