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WATERTOWN, Mass. - Disc Medicine, Inc. (NASDAQ:IRON), a biopharmaceutical company, has received positive feedback from the U.S. Food and Drug Administration (FDA) regarding its end-of-Phase 2 meeting for the regulatory path forward for bitopertin in treating Erythropoietic Protoporphyria (EPP), as announced today.
The company detailed that the FDA agreed with all proposed attributes of its upcoming APOLLO study, which is slated to begin by mid-2025. The study will focus on a 60 mg dose of bitopertin over a 6-month treatment period for EPP and X-linked Protoporphyria (XLP) patients aged 12 and older. The primary endpoint will measure the average monthly total time in sunlight without pain during the last month following treatment.
In addition to the primary endpoint, the APOLLO study will include assessments such as changes in protoporphyrin IX (PPIX), occurrence of phototoxic reactions, cumulative total pain-free time in sunlight, and patient global impression of change (PGIC). The FDA’s alignment on these measures reflects the potential for bitopertin to address the significant unmet need in EPP treatment.
The FDA also recognized the potential for accelerated approval based on existing data, with the reduction of PPIX serving as a surrogate endpoint. If the accelerated approval is granted, the APOLLO trial would then act as a confirmatory study.
Disc Medicine plans to meet with the FDA to finalize APOLLO study details, with an update on the discussion and the timeline for a New Drug Application (NDA) filing expected in the first quarter of 2025.
Bitopertin, a GlyT1 inhibitor, has been studied in several clinical trials and is designed to modulate heme biosynthesis. The company acquired global rights to the investigational drug from Roche in May 2021. EPP and XLP are rare conditions that cause severe reactions to sunlight exposure, leading to significant quality of life impacts for patients. Current treatments are limited, with Scenesse® being the only FDA-approved therapy for EPP.
The press release statement also provided an overview of Disc Medicine’s commitment to developing therapies for serious hematologic diseases and noted that bitopertin is not yet approved for therapeutic use in any jurisdiction worldwide.
Investors and interested parties were informed about a conference call hosted by management to discuss these updates, which took place today at 8:00 am EST.
In other recent news, Disc Medicine has made significant strides in its clinical trials and operations. The biopharmaceutical company reported new findings from its Phase 1b study of DISC-0974 in patients with non-dialysis-dependent chronic kidney disease (NDD-CKD) and anemia. The study revealed that a single dose of DISC-0974 significantly reduces hepcidin levels and improves iron mobilization and hemoglobin levels in patients. Disc Medicine has also reported promising Phase 2 results for its lead drug candidate, bitopertin, aimed at treating Erythropoietic Protoporphyria (EPP).
On the analyst front, Disc Medicine received a Buy rating from Jefferies and an Overweight rating from Wells Fargo. H.C. Wainwright maintained its Buy rating and $70.00 stock target for Disc Medicine, while BMO Capital Markets revised its outlook, raising the price target to $70 from $50.
Disc Medicine has also made key appointments, welcoming Dr. Rahul Rajan Kaushik as Chief Technical Officer and Dr. Steve Caffé as Chief Regulatory Officer, both bringing over two decades of experience in the pharmaceutical industry. Furthermore, Disc Medicine announced a public stock offering of approximately $178 million, led by Frazier Life Sciences and Logos Capital. The proceeds will be used to advance research and clinical development of its product candidates, including bitopertin. These are recent developments in the operations of Disc Medicine as it continues to develop treatments for serious hematologic diseases.
InvestingPro Insights
As Disc Medicine (NASDAQ:IRON) advances its regulatory pathway for bitopertin, investors may find additional context in the company's financial metrics and market performance. According to InvestingPro data, Disc Medicine's market capitalization stands at $1.4 billion, reflecting the market's current valuation of the company's potential in the rare disease space.
The company's financial health shows some strengths amidst its developmental stage. An InvestingPro Tip highlights that Disc Medicine holds more cash than debt on its balance sheet, which is crucial for biotechnology companies investing heavily in research and development. This solid cash position could provide the necessary runway for the upcoming APOLLO study and potential NDA filing.
Despite the positive regulatory news, it's important to note that Disc Medicine is not currently profitable, with a negative EBITDA of $110.8 million over the last twelve months as of Q2 2024. This is typical for biopharmaceutical companies in the development phase, and investors often focus on pipeline progress rather than immediate profitability.
Interestingly, the stock has seen a significant price uptick over the last six months, with a total return of 58.21% according to InvestingPro data. This surge may reflect investor optimism about the company's progress with bitopertin and its potential in the EPP market.
For those seeking a deeper dive into Disc Medicine's financials and prospects, InvestingPro offers 10 additional tips that could provide valuable insights for investment decisions in this dynamic biotech stock.
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