UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C. 20549
FORM
CURRENT REPORT
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Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§ 230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§ 240.12b-2 of this chapter).
Emerging growth company
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act.
Item 7.01 Regulation FD Disclosure.
On November 4, 2024, Disc Medicine, Inc. (the "Company") issued a press release announcing positive feedback from its end-of-Phase 2 meeting ("EOP2 Meeting") with the U.S. Food and Drug Administration ("FDA") for bitopertin in erythropoietic protoporphyria ("EPP"). The Company will host a previously-announced conference call to discuss the FDA feedback on November 4, 2024 at 8:00 AM ET. An archived webcast will be available following the call for 30 days on the Events & Presentations section of the Company’s website. A copy of the press release is attached as Exhibit 99.1 and a copy of the slide presentation to be presented during the conference call is attached as Exhibit 99.2 to this Current Report on Form 8-K.
The information contained in Item 7.01 of this Current Report on Form 8-K, including Exhibit 99.1 and Exhibit 99.2, is intended to be furnished and shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended, or the Exchange Act, except as expressly provided by specific reference in such filing. The Company undertakes no obligation to update, supplement or amend the material attached hereto as Exhibit 99.1 or Exhibit 99.2.
Item 8.01 Other Events.
On November 4, 2024, the Company announced positive feedback from its EOP2 Meeting with the FDA, supporting the regulatory path forward for bitopertin in EPP. The EOP2 Meeting resulted in the Company and the FDA agreeing on all proposed attributes of the Company’s APOLLO clinical trial of bitopertin in EPP and X-linked protoporphyria ("XLP"), which the Company plans to initiate by mid-2025, including the:
In addition, the FDA agreed with the potential for reduction of PPIX to serve as a surrogate endpoint to support an accelerated approval. Under the accelerated approval pathway, the Company would have the potential to submit a New Drug Application ("NDA") based on the Company's existing data package, and the APOLLO trial would serve as a confirmatory trial. The Company will be meeting with the FDA to finalize the details of APOLLO, and plans to provide an update in the first quarter of 2025 on this discussion as well as timing for a potential NDA filing under an accelerated pathway.
Cautionary Statement Regarding Forward-Looking Statements
This Current Report on Form 8-K contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, express or implied statements regarding the Company’s expectations with respect to its potential APOLLO clinical study of bitopertin in EPP and XLP patients, including the proposed study parameters, the anticipated timeline, and the results thereof; and the possible regulatory path forward for bitopertin in EPP, including the potential to seek approval under the accelerated approval pathway and conduct a confirmatory trial, and the timeline of related discussions with the FDA. The use of words such as, but not limited to, “believe,” “expect,” “estimate,” “project,” “intend,” “future,” “potential,” “continue,” “may,” “might,” “plan,” “will,” “should,” “seek,” “anticipate,” or “could” or the negative of these terms and other similar words or expressions that are intended to identify forward-looking statements. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based on the Company’s current beliefs, expectations and assumptions regarding the future of the Company’s business, future plans and strategies, clinical results and other future conditions. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements.
The Company may not actually achieve the plans, intentions or expectations disclosed in these forward-looking statements, and investors should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in the forward-looking statements as a result of a number of material risks and uncertainties including but not limited to: the adequacy of the Company's capital to support its future operations and its ability to successfully initiate and complete clinical trials; the nature, strategy and focus of the Company; the difficulty in predicting the time and cost of development of the Company's product candidates; the Company's plans to research, develop and commercialize its current and future product candidates; the timing of initiation of the Company's planned preclinical studies and clinical trials; the timing of the availability of data from the Company's clinical trials; the Company's ability to identify additional product candidates with significant commercial potential and to expand its pipeline in hematological diseases; the timing and anticipated results of the Company's preclinical studies and clinical trials and the risk that the results of the Company's preclinical studies and clinical trials may
not be predictive of future results in connection with future studies or clinical trials and may not support further development and marketing approval; and the other risks and uncertainties described in the Company's filings with the Securities and Exchange Commission, including in the “Risk Factors” section of the Company's Annual Report on Form 10-K for the year ended December 31, 2023, and in subsequent Quarterly Reports on Form 10-Q. Any forward-looking statement speaks only as of the date on which it was made. None of the Company, nor its affiliates, advisors or representatives, undertake any obligation to publicly update or revise any forward-looking statement, whether as result of new information, future events or otherwise, except as required by law.
Item 9.01 Financial Statements and Exhibits.
(d) Exhibits
Exhibit No. |
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Description |
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99.1 |
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Press release issued by Disc Medicine, Inc. on November 4, 2024, furnished herewith |
99.2 |
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Disc Medicine, Inc. presentation, dated November 4, 2024, furnished herewith |
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Cover Page Interactive Data File (embedded within the Inline XBRL document) |
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
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DISC MEDICINE, INC. |
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Date: |
November 4, 2024 |
By: |
/s/ John Quisel, J.D., Ph.D. |
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Name: Title: |
John Quisel, J.D., Ph.D. |
Exhibit 99.1
Disc Medicine Announces Successful End of Phase 2 Meeting with FDA for Bitopertin in Erythropoietic Protoporphyria (EPP), Including Potential for Accelerated Approval
WATERTOWN, Mass. November 4, 2024 -- Disc Medicine, Inc. (NASDAQ:IRON), a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of novel treatments for patients suffering from serious hematologic diseases, today announced positive feedback from its end-of-Phase 2 meeting with the U.S. Food and Drug Administration (FDA), supporting the regulatory path forward for bitopertin in EPP.
“We are thrilled with the outcome of our end-of-Phase 2 meeting with the FDA, which provides us with a clear development path forward for bitopertin. Importantly, the FDA agreed with all attributes of our study design, including a primary endpoint we feel is statistically robust and would fully capture the potential benefit of bitopertin in EPP,” said John Quisel, J.D., Ph.D., President and Chief Executive Officer of Disc. “We’re particularly excited by the potential to file under the Accelerated Approval Program based on our existing data and use of PPIX reduction as a surrogate endpoint. This is a testament to the significant unmet need in EPP and the strength of the bitopertin data package, and we look forward to engaging further with the FDA on this pathway.”
The meeting resulted in agreement on all proposed attributes of the APOLLO study, which the company plans to initiate by mid-2025, including the:
In addition, the FDA also agreed with the potential for reduction of PPIX to serve as a surrogate endpoint to support an accelerated approval. Under this pathway, Disc would have the potential to submit an NDA based on the existing data package and the APOLLO trial would serve as a confirmatory trial. Disc will be meeting with the FDA to finalize the details of APOLLO and plans to provide an update in Q1 2025 on this discussion as well as timing for NDA filing under an accelerated pathway.
Management will host a call to discuss these updates on Monday, November 4 at 8:00 am EST. Please register for the event on the Events and Presentations page of Disc’s website (https://ir.discmedicine.com/).
About Bitopertin
Bitopertin is an investigational, clinical-stage, orally administered inhibitor of glycine transporter 1 (GlyT1) that is designed to modulate heme biosynthesis. GlyT1 is a membrane transporter expressed on developing red blood cells and is required to supply sufficient glycine for heme biosynthesis and support erythropoiesis. Disc is planning to develop bitopertin as a potential treatment for a range of hematologic diseases including erythropoietic porphyrias, where it has potential to be the first disease-modifying therapy. Bitopertin has been studied in multiple clinical trials in patients with EPP, including the Phase 2 open-label BEACON trial, the Phase 2 double-blind, placebo-controlled AURORA trial, and an open-label extension HELIOS trial.
Bitopertin is an investigational agent and is not approved for use as a therapy in any jurisdiction worldwide. Disc obtained global rights to bitopertin under a license agreement from Roche in May 2021.
About Erythropoietic Protoporphyria (EPP) and X-linked Protoporphyria (XLP)
Erythropoietic protoporphyria (EPP) and X-linked Protoporphyria (XLP) are rare, debilitating and potentially life-threatening diseases caused by mutations that affect heme biosynthesis, resulting in the accumulation of a toxic, photoactive intermediate called protoporphyrin IX (PPIX). This causes severe reactions when patients are exposed to sunlight, characterized by excruciating pain, edema, burning sensations and potential blistering and disfigurement. PPIX also accumulates in the hepatobiliary system and can result in complications including gallstones, cholestasis, and liver damage in 20-30% of patients and in extreme cases liver failure. Current standard of care involves extreme measures to avoid sunlight, including restricting outdoor activities to nighttime, use of protective clothing and opaque shields, and pain management. This has a significant impact on the psychosocial development, quality of life, and daily activities of patients, particularly in young children and families. There is currently no cure for EPP and only one FDA-approved therapy, a surgically implanted synthetic hormone designed to stimulate melanin production called Scenesse® (afamelanotide).
About Disc Medicine
Disc Medicine is a clinical-stage biopharmaceutical company committed to discovering, developing, and commercializing novel treatments for patients who suffer from serious hematologic diseases. We are building a portfolio of innovative, potentially first-in-class therapeutic candidates that aim to address a wide spectrum of hematologic diseases by targeting fundamental biological pathways of red blood cell biology, specifically heme biosynthesis and iron homeostasis. For more information, please visit www.discmedicine.com.
Disc Cautionary Statement Regarding Forward-Looking Statements
This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, express or implied statements regarding Disc’s expectations with respect to its potential APOLLO clinical study of bitopertin in EPP and XLP patients, including the proposed study parameters, the anticipated timeline, and the results thereof; and the possible regulatory path forward for bitopertin in EPP, including the potential to seek approval under the Accelerated Approval pathway and conduct a confirmatory trial, and the timeline of related discussions with the FDA. The use of words such as, but not limited to, “believe,” “expect,” “estimate,” “project,” “intend,” “future,” “potential,” “continue,” “may,” “might,” “plan,” “will,” “should,” “seek,” “anticipate,” or “could” or the negative of these terms and other similar words or expressions that are intended to identify
forward-looking statements. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based on Disc’s current beliefs, expectations and assumptions regarding the future of Disc’s business, future plans and strategies, clinical results and other future conditions. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements.
Disc may not actually achieve the plans, intentions or expectations disclosed in these forward-looking statements, and investors should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in the forward-looking statements as a result of a number of material risks and uncertainties including but not limited to: the adequacy of Disc’s capital to support its future operations and its ability to successfully initiate and complete clinical trials; the nature, strategy and focus of Disc; the difficulty in predicting the time and cost of development of Disc’s product candidates; Disc’s plans to research, develop and commercialize its current and future product candidates; the timing of initiation of Disc’s planned preclinical studies and clinical trials; the timing of the availability of data from Disc’s clinical trials; Disc’s ability to identify additional product candidates with significant commercial potential and to expand its pipeline in hematological diseases; the timing and anticipated results of Disc’s preclinical studies and clinical trials and the risk that the results of Disc’s preclinical studies and clinical trials may not be predictive of future results in connection with future studies or clinical trials and may not support further development and marketing approval; and the other risks and uncertainties described in Disc’s filings with the Securities and Exchange Commission, including in the “Risk Factors” section of our Annual Report on Form 10-K for the year ended December 31, 2023, and in subsequent Quarterly Reports on Form 10-Q. Any forward-looking statement speaks only as of the date on which it was made. None of Disc, nor its affiliates, advisors or representatives, undertake any obligation to publicly update or revise any forward-looking statement, whether as result of new information, future events or otherwise, except as required by law.
Media Contact
Peg Rusconi
Deerfield Group
peg.rusconi@deerfieldgroup.com
Investor Relations Contact
Christina Tartaglia
Precision AQ
christina.tartaglia@precisionaq.com
Bitopertin End of Phase 2 Meeting Feedback November 4, 2024 Exhibit 99.2
Disclaimer and FLS This presentation contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, express or implied statements regarding Disc’s expectations with respect to its proposed APOLLO clinical trial of bitopertin, including the primary endpoint and other study parameters, its AURORA Phase 2, BEACON Phase 2 and HELIOS Phase 2 clinical trials of bitopertin and the results thereof, its Phase 1b/2 clinical trial of DISC-0974 in patients with MF and NDD-CKD patients with anemia, and its HVOL MAD data in its Phase 1 clinical trial of DISC-3405 in healthy volunteers; projected timelines for the initiation and completion of its clinical trials, anticipated timing of release of data, and other clinical activities; the possible regulatory path for bitopertin in EPP, including the potential to seek approval under the Accelerated Approval pathway and the timeline of related discussions with the FDA; and commercialization plans for bitopertin. The use of words such as, but not limited to, “believe,” “expect,” “estimate,” “project,” “intend,” “future,” “potential,” “continue,” “may,” “might,” “plan,” “will,” “should,” “seek,” “anticipate,” or “could” or the negative of these terms and other similar words or expressions that are intended to identify forward-looking statements. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based on Disc’s current beliefs, expectations and assumptions regarding the future of Disc’s business, future plans and strategies, clinical results and other future conditions. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements. Disc may not actually achieve the plans, intentions or expectations disclosed in these forward-looking statements, and investors should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in the forward-looking statements as a result of a number of material risks and uncertainties including but not limited to: the adequacy of Disc’s capital to support its future operations and its ability to successfully initiate and complete clinical trials; the nature, strategy and focus of Disc; the difficulty in predicting the time and cost of development of Disc’s product candidates; Disc’s plans to research, develop and commercialize its current and future product candidates; the timing of initiation of Disc’s planned preclinical studies and clinical trials; the timing of the availability of data from Disc’s clinical trials; Disc’s ability to identify additional product candidates with significant commercial potential and to expand its pipeline in hematological diseases; the timing and anticipated results of Disc’s preclinical studies and clinical trials and the risk that the results of Disc’s preclinical studies and clinical trials may not be predictive of future results in connection with future studies or clinical trials and may not support further development and marketing approval; and the other risks and uncertainties described in Disc’s filings with the Securities and Exchange Commission, including in the “Risk Factors” section of our Annual Report on Form 10-K for the year ended December 31, 2023, and in subsequent Quarterly Reports on Form 10-Q. Any forward-looking statement speaks only as of the date on which it was made. None of Disc, nor its affiliates, advisors or representatives, undertake any obligation to publicly update or revise any forward-looking statement, whether as result of new information, future events or otherwise, except as required by law.
Agenda 01 Introduction and Summary John Quisel, J.D., PhD, Chief Executive Officer 02 Detailed Review of End of Phase 2 Feedback Steve Caffé, M.D., Chief Regulatory Officer Proposed APOLLO Study Parameters Will Savage, M.D., PhD, Chief Medical Officer 03 Closing Remarks John Quisel, J.D., PhD, Chief Executive Officer 04 Q&A Session 05
Alignment with the FDA on all proposed study parameters FDA acknowledged that EPP is a serious and potentially life-threatening disease with significant unmet medical need FDA agreed that average monthly time in sunlight without pain at the end of a 6-month treatment period can be used as a primary endpoint Key Takeaways from Positive End of Phase 2 Meeting Proceeding to APOLLO, a 6-month study with a 60 mg dose of bitopertin in EPP and XLP patients ages 12+ PPIX reduction may be sufficient as a surrogate endpoint supportive of accelerated approval
EPP Phase 2 Development Program BEACON, AURORA, and HELIOS Studies Successful end of Phase 2 meeting with the FDA acknowledging the high unmet need in EPP and supporting our chosen trial parameters; clear development path to registration EPP and XLP; N = 26 (22 adults, 4 adolescents) Australia Open-label, randomized, 24-week study EPP; N = 75 adults United States Double-blind, randomized, placebo-controlled, 17-week study EPP and XLP; adults and adolescents US and Australia Open-label extension study (>80% rollover from BEACON and AURORA)
Significant reductions in PPIX 40% reduction vs baseline Time-dependent, improvements in pain-free time in sunlight vs placebo 2x more light time vs baseline Significant 75% reduction in rate of phototoxic reactions vs placebo Phototoxic reaction-free in last 60 days Significant improvement in PGIC vs placebo 86% reported EPP was ‘much better’ Clear association between PPIX reduction and clinical endpoints Summary of AURORA Results Bitopertin 60 mg Placebo Bitopertin 60 mg Phototoxic Reactions = Phototoxic Reaction = 2 Phototoxic Reactions
Agenda 01 Introduction and Summary John Quisel, J.D., PhD, Chief Executive Officer 02 Detailed Review of End of Phase 2 Feedback Steve Caffé, M.D., Chief Regulatory Officer Proposed APOLLO Study Parameters Will Savage, M.D., PhD, Chief Medical Officer 03 Closing Remarks John Quisel, J.D., PhD, Chief Executive Officer 04 Q&A Session 05
Detailed FDA Feedback FDA agreed to all study parameters Disc requested prior to the meeting CONFIDENTIAL Primary Endpoint Average monthly total time in sunlight without pain between 10:00 and 18:00 after 6 months of treatment is clinically meaningful and can serve as a primary endpoint Additional Endpoints Change in PPIX, occurrence of phototoxic reactions, cumulative total pain-free time in sunlight, and patient global impression of change (PGIC) Dose and Duration Proceed with 60 mg dose of bitopertin and 6-month study duration Study Population Patients aged 12 years and older with EPP, including XLP Disc Request FDA Agreement
Accelerated Approval CONFIDENTIAL FDA acknowledged PPIX reduction, as demonstrated in AURORA, may serve as surrogate endpoint to support accelerated approval An accelerated NDA package would include data from BEACON, AURORA, HELIOS, and a >4,000 participant safety database Will meet with FDA to finalize confirmatory trial design Expect to provide an update on this process in Q1 2025, while simultaneously moving forward with preparations for trial initiation by mid-2025 Accelerated approval pathway allows for earlier marketing authorization using a surrogate endpoint. Full approval is subject to demonstration of clinical benefit in a confirmatory trial
Agenda 01 Introduction and Summary John Quisel, J.D., PhD, Chief Executive Officer 02 Detailed Review of End of Phase 2 Feedback Steve Caffé, M.D., Chief Regulatory Officer Proposed APOLLO Study Parameters Will Savage, M.D., PhD, Chief Medical Officer 03 Closing Remarks John Quisel, J.D., PhD, Chief Executive Officer 04 Q&A Session 05
Primary Endpoint: Average Monthly Time in Light without Pain After 6 Months of Treatment Robust Endpoint Longitudinal analysis leverages robust model that demonstrated significance in AURORA Accounts for time-dependent PPIX lowering effects with bitopertin and for waning of a placebo effect Endpoint has >80% power with 150 patients Strong Study Design Rigorous evaluation of baseline light tolerance required during screening and factored into analysis of the primary endpoint Stratification by geography to minimize confounding factors affecting light exposure across study arms 2-Week Aggregated Time (hr) AURORA: Sunlight Tolerance Over Time = last month of study = last 2-weeks of study p=0.013 vs PBO* p=0.03 vs PBO* Post-hoc longitudinal analysis adjusted for baseline * nominal p value
APOLLO Study Parameters N Size ~150 patients across sites in the US and Europe Trial Duration 6-month treatment period Trial Design Randomized 1:1, double-blind, placebo-controlled Trial Population EPP and XLP patients ages 12+, stratified by baseline light tolerance and geography Dose 60 mg Primary Endpoint Average monthly total time in sunlight without pain between 10:00 and 18:00 after 6 months of treatment Additional Endpoints Change from baseline in whole blood metal-free PPIX Occurrence of phototoxic reactions Patient global impression of change (PGIC) Cumulative total pain-free time in sunlight Safety and tolerability Pending regulatory feedback on final protocol design
Agenda 01 Introduction and Summary John Quisel, J.D., PhD, Chief Executive Officer 02 Detailed Review of End of Phase 2 Feedback Steve Caffé, M.D., Chief Regulatory Officer Proposed APOLLO Study Parameters Will Savage, M.D., PhD, Chief Medical Officer 03 Closing Remarks John Quisel, J.D., PhD, Chief Executive Officer 04 Q&A Session 05
Key Takeaways from End of Phase 2 Meeting Endpoint Alignment Alignment with FDA on clinically meaningful endpoints Target Population Included Study can be conducted in adults and adolescents (ages 12+) with EPP and XLP Desired Dose Endorsed Endorsement of 60 mg dose for 6-month study Accelerated Approval Potential PPIX may serve as a surrogate endpoint to support accelerated approval
ASN: Presented positive Phase 1 SAD data for DISC-0974 in CKD Additional data updates by EOY Bitopertin: Additional analyses from BEACON and AURORA DISC-0974: Complete Phase 1b data in MF anemia DISC-3405: Phase 1 HVOL MAD data Q4 2024 Catalysts Discussion of confirmatory trial design with FDA, with updates provided in Q1 2025 Trial initiation by mid-2025 European protocol assistance and confirmation of regulatory path with EMA Commercialization and launch preparation Next Steps and Upcoming Catalysts Bitopertin Next Steps HVOL = healthy volunteers; SAD = single ascending dose; MF = myelofibrosis; MAD = multiple ascending dose
Agenda 01 Introduction and Summary John Quisel, J.D., PhD, Chief Executive Officer 02 Detailed Review of End of Phase 2 Feedback Steve Caffé, M.D., Chief Regulatory Officer Proposed APOLLO Study Parameters Will Savage, M.D., PhD, Chief Medical Officer 03 Closing Remarks John Quisel, J.D., PhD, Chief Executive Officer 04 Q&A Session 05