Fulcrum Therapeutics Announces Recent Business Highlights and Financial Results for Third Quarter 2024
Fulcrum Therapeutics (FULC) reported Q3 2024 financial results and business updates. The company continues to progress with patient enrollment in the Phase 1b PIONEER trial of pociredir for sickle cell disease. Cash position stood at $257.2 million as of September 30, 2024, with runway extending into 2027. Q3 net loss was $21.7 million, compared to $24.0 million in Q3 2023. R&D expenses decreased to $14.6 million from $18.2 million year-over-year. The company suspended losmapimod development following September's REACH trial results and is now focusing on pociredir development and other early discovery programs.
Fulcrum Therapeutics (FULC) ha riportato i risultati finanziari e gli aggiornamenti aziendali del terzo trimestre del 2024. L'azienda continua a progredire con l'arruolamento dei pazienti nello studio di Fase 1b PIONEER di pociredir per la malattia delle cellule falciformi. La posizione di cassa ammontava a 257,2 milioni di dollari al 30 settembre 2024, con una liquidità che si estende fino al 2027. La perdita netta nel terzo trimestre è stata di 21,7 milioni di dollari, rispetto ai 24,0 milioni di dollari del terzo trimestre del 2023. Le spese per R&S sono diminuite a 14,6 milioni di dollari rispetto ai 18,2 milioni di dollari dell'anno precedente. L'azienda ha sospeso lo sviluppo di losmapimod in seguito ai risultati dello studio REACH di settembre e ora si concentra sullo sviluppo di pociredir e su altri programmi di scoperta precoce.
Fulcrum Therapeutics (FULC) reportó los resultados financieros y las actualizaciones comerciales del tercer trimestre de 2024. La compañía continúa avanzando con el reclutamiento de pacientes en el ensayo de Fase 1b PIONEER de pociredir para la enfermedad de células falciformes. La posición de efectivo se situó en 257.2 millones de dólares al 30 de septiembre de 2024, con un financiamiento que se extiende hasta 2027. La pérdida neta en el tercer trimestre fue de 21.7 millones de dólares, en comparación con 24.0 millones de dólares en el tercer trimestre de 2023. Los gastos en I+D disminuyeron a 14.6 millones de dólares desde 18.2 millones de dólares en el año anterior. La compañía suspendió el desarrollo de losmapimod tras los resultados del ensayo REACH de septiembre y ahora se centra en el desarrollo de pociredir y otros programas de descubrimiento temprano.
Fulcrum Therapeutics (FULC)는 2024년 3분기 재무 결과 및 사업 업데이트를 발표했습니다. 이 회사는 겸상적혈구병을 위한 pociredir의 1b 단계 PIONEER 임상 시험에서 환자 모집을 계속 진행 중입니다. 현금 보유액은 2024년 9월 30일 기준으로 2억 5720만 달러였으며, 2027년까지의 자금 여력이 있습니다. 3분기 순손실은 2170만 달러로, 2023년 3분기의 2400만 달러에 비해 감소했습니다. 연구개발 비용은 전년 동기 대비 1820만 달러에서 1460만 달러로 줄어들었습니다. 이 회사는 9월의 REACH 시험 결과에 따라 losmapimod 개발을 중단하고 현재는 pociredir 개발 및 기타 초기 발견 프로그램에 집중하고 있습니다.
Fulcrum Therapeutics (FULC) a publié les résultats financiers et les mises à jour commerciales du troisième trimestre 2024. L'entreprise continue de progresser dans le recrutement des patients pour l'essai PIONEER de phase 1b de pociredir pour la maladie des cellules falciformes. La position de trésorerie s'élevait à 257,2 millions de dollars au 30 septembre 2024, avec une trésorerie prévue jusqu'en 2027. La perte nette du troisième trimestre s'élevait à 21,7 millions de dollars, contre 24,0 millions de dollars au troisième trimestre 2023. Les dépenses de R&D ont diminué à 14,6 millions de dollars, contre 18,2 millions de dollars l'année précédente. L'entreprise a suspendu le développement de losmapimod suite aux résultats de l'essai REACH de septembre et se concentre désormais sur le développement de pociredir et d'autres programmes de découverte précoce.
Fulcrum Therapeutics (FULC) hat die finanziellen Ergebnisse und Geschäftsupdates für das dritte Quartal 2024 bekannt gegeben. Das Unternehmen schreitet beim Patientenrekrutierung in der Phase 1b PIONEER-Studie von pociredir zur Sichelzellenkrankheit voran. Die liquiden Mittel beliefen sich zum 30. September 2024 auf 257,2 Millionen US-Dollar, mit einer Planungsbasis bis 2027. Der Nettoverlust im dritten Quartal betrug 21,7 Millionen US-Dollar im Vergleich zu 24,0 Millionen US-Dollar im dritten Quartal 2023. Die F&E-Ausgaben sanken im Jahresvergleich von 18,2 Millionen US-Dollar auf 14,6 Millionen US-Dollar. Das Unternehmen stellte die Entwicklung von Losmapimod nach den Ergebnissen der REACH-Studie im September ein und konzentriert sich nun auf die Entwicklung von pociredir und anderen frühen Entdeckungsprogrammen.
- Strong cash position of $257.2 million, up from $236.2 million at end of 2023
- Extended cash runway into 2027
- Reduced net loss to $21.7 million from $24.0 million year-over-year
- R&D expenses decreased by $3.6 million compared to Q3 2023
- Received $80.0 million upfront payment from Sanofi in Q2 2024
- Collaboration revenue dropped to zero from $0.8 million in Q3 2023
- Suspended development of losmapimod following unsuccessful REACH trial
- Expected cash burn of $55.0-65.0 million for full year 2025
Insights
The Q3 results reveal mixed financial performance. While
The suspension of losmapimod development following unsuccessful REACH trial results represents a setback, but the company's strategic pivot to focus on pociredir for SCD and other rare disease programs demonstrates adaptability. The
The PIONEER trial progression for pociredir in sickle cell disease gains increased significance following OXBRYTA's global withdrawal, creating a larger market opportunity. The trial's design, evaluating escalating doses of 12 mg and 20 mg daily in cohorts of up to 10 patients each, appears well-structured to determine optimal dosing. The company's planned expansion into Phase 1 studies with healthy volunteers, following FDA interactions, suggests a comprehensive development approach.
The pipeline refocus on inherited aplastic anemias, including Diamond-Blackfan anemia, Shwachman-Diamond syndrome and Fanconi anemia, represents a strategic concentration on high-unmet-need rare diseases where targeted therapies could have significant impact.
― Patient enrollment and site activation progressing in Phase 1b PIONEER trial of pociredir in sickle cell disease (SCD) ―
― Ended the third quarter of 2024 with
― Conference call and webcast scheduled for 8:00 a.m. ET today ―
CAMBRIDGE, Mass., Nov. 13, 2024 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc.® (Fulcrum) (Nasdaq: FULC), a clinical-stage biopharmaceutical company focused on developing small molecules to improve the lives of patients with genetically defined rare diseases, today reported financial results for the third quarter of 2024 as well as a business update.
“In the third quarter, we made progress enrolling patients and activating new sites in the Phase 1b PIONEER trial of pociredir in sickle cell disease and advancing our preclinical pipeline,” said Alex C. Sapir, Fulcrum’s president and chief executive officer. “The need for effective therapeutic options for patients with sickle cell disease has become even more urgent due to the recent withdrawal of OXBRYTA® globally. Based on the initial data generated in the Phase 1b trial, we believe that pociredir has the potential to increase fetal hemoglobin to levels that could ameliorate symptoms of sickle cell disease. While we were disappointed with the results of the Phase 3 REACH trial announced in September, we remain committed to improving the lives of patients with genetically defined diseases in areas of high unmet medical need like sickle cell disease. We are focused on progressing the development of pociredir as expeditiously as possible and remain on track to provide data from the PIONEER trial in 2025.”
Recent Business Highlights
- Patient enrollment and site activations continue to progress in the Phase 1b PIONEER trial evaluating pociredir in patients with SCD. Cohort 3 of the Phase 1b trial is evaluating pociredir at the 12 mg once daily dose, which will be followed by Cohort 4 at the 20 mg once daily dose. Each cohort is expected to enroll up to 10 patients. Fulcrum plans to share clinical data from the PIONEER trial in 2025.
- Following recent interactions with the FDA, Fulcrum plans to initiate Phase 1 clinical studies in healthy volunteers in support of the overall pociredir development program.
- Following the REACH trial results disclosed in September 2024, Fulcrum suspended development of losmapimod and has focused its research and development activities on advancing pociredir for the treatment of SCD, as well as advancing novel therapeutic agents for the potential treatment of inherited aplastic anemias, such as Diamond-Blackfan anemia, Shwachman-Diamond syndrome, and Fanconi anemia, and its early discovery programs.
Third Quarter 2024 Financial Results
- Cash Position: As of September 30, 2024, cash, cash equivalents, and marketable securities were
$257.2 million , as compared to$236.2 million as of December 31, 2023. The increase is due to the$80.0 million upfront payment received from Sanofi in the second quarter of 2024, partially offset by cash used to fund operating activities in 2024. - Collaboration Revenue: Collaboration revenue was zero for the three months ended September 30, 2024, as compared to
$0.8 million for the three months ended September 30, 2023. The decrease of$0.8 million was due to the completion of research services under the collaboration agreement with MyoKardia during the fourth quarter of 2023. - R&D Expenses: Research and development expenses were
$14.6 million for the three months ended September 30, 2024, as compared to$18.2 million for the three months ended September 30, 2023. The decrease of$3.6 million was primarily due to the reimbursement from the global development cost sharing under the collaboration with Sanofi for losmapimod, partially offset by increased costs related to the advancement of the Phase 1b PIONEER trial. - G&A Expenses: General and administrative expenses were
$8.4 million for the three months ended September 30, 2024, as compared to$10.0 million for three months ended September 30, 2023. The decrease of$1.6 million was primarily due to decreased employee compensation costs as a result of the reduction in workforce implemented in the third quarter of 2024. - Net Loss: Net loss was
$21.7 million for the three months ended September 30, 2024, as compared to a net loss of$24.0 million for the three months ended September 30, 2023.
Cash Guidance
Based on its current operating plans, Fulcrum expects to end 2024 with approximately
Conference Call and Webcast
Fulcrum Therapeutics, Inc. will host a conference call and webcast today at 8:00 a.m. ET to review the third quarter 2024 financial results and recent business highlights. Individuals may register for the conference call by clicking the link here. Once registered, participants will receive dial-in details and unique PIN which will allow them to access the call. An audio webcast will be accessible through the Investor Relations section of Fulcrum's website at www.fulcrumtx.com or by clicking here. Following the live webcast, an archived replay will also be available.
About Fulcrum Therapeutics
Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on developing small molecules to improve the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum’s lead program in clinical development is pociredir, a small molecule designed to increase expression of fetal hemoglobin and in development for the treatment of SCD. Fulcrum uses proprietary technology to identify drug targets that can modulate gene expression to treat the known root cause of gene mis-expression. For more information, visit www.fulcrumtx.com and follow us on Twitter/X (@FulcrumTx) and LinkedIn.
About Pociredir
Pociredir is an investigational oral small-molecule inhibitor of Embryonic Ectoderm Development (EED) that was discovered using Fulcrum’s proprietary discovery technology. Inhibition of EED leads to potent downregulation of key fetal globin repressors, including BCL11A, thereby causing an increase in fetal hemoglobin (HbF). Pociredir is being developed for the treatment of SCD. Initial data in SCD demonstrated proof-of-concept and achieved absolute levels of HbF increases associated with potential overall patient benefit. In clinical trials conducted prior to the clinical hold, which was lifted by the FDA in August 2023, pociredir was generally well-tolerated in people with SCD with up to three months of exposure, with no serious treatment-related adverse events reported. Pociredir has been granted FDA Fast Track designation and Orphan Drug Designation for the treatment of SCD. To learn more about these trials please visit ClinicalTrials.gov.
About Sickle Cell Disease
Sickle cell disease (SCD) is a genetic disorder of the red blood cells caused by a mutation in the HBB gene. This gene encodes a protein that is a key component of hemoglobin, a protein complex whose function is to transport oxygen in the body. The result of the mutation is less efficient oxygen transport and the formation of red blood cells that have a sickle shape. These sickle shaped cells are much less flexible than healthy cells and can block blood vessels or rupture cells. People with SCD typically suffer from serious clinical consequences, which may include anemia, pain, infections, stroke, heart disease, pulmonary hypertension, kidney failure, liver disease, and reduced life expectancy.
Forward-Looking Statements
This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties. All statements, other than statements of historical facts, contained in this press release are forward-looking statements, including express or implied statements regarding Fulcrum’s Phase 1b PIONEER clinical trial of pociredir, including status of enrollment, number of patients per cohort and planned data announcement for such trial; the potential of pociredir to increase fetal hemoglobin to levels that could ameliorate symptoms of SCD; resumption of Phase 1 clinical studies in healthy volunteers ; Fulcrum’s ability to progress its early stage development programs; the effects of the reduction in workforce; and its projected year end cash position, cash runway and cash burn for 2025, among others. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with Fulcrum’s ability to continue to advance its product candidates in clinical trials; initiating and enrolling clinical trials on the timeline expected or at all; obtaining and maintaining necessary approvals from the FDA and other regulatory authorities; replicating in clinical trials positive results found in preclinical studies and/or earlier-stage clinical trials; obtaining, maintaining or protecting intellectual property rights related to its product candidates; managing expenses; realize the anticipated benefits of the workforce reduction and strategic realignment and managing risks associated therewith; and raising the substantial additional capital needed to achieve its business objectives, among others. For a discussion of other risks and uncertainties, and other important factors, any of which could cause Fulcrum’s actual results to differ from those contained in the forward-looking statements, see the “Risk Factors” section, as well as discussions of potential risks, uncertainties, and other important factors, in Fulcrum’s most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent Fulcrum’s views as of the date hereof and should not be relied upon as representing Fulcrum’s views as of any date subsequent to the date hereof. Fulcrum anticipates that subsequent events and developments will cause Fulcrum’s views to change. However, while Fulcrum may elect to update these forward-looking statements at some point in the future, Fulcrum specifically disclaims any obligation to do so.
| Fulcrum Therapeutics, Inc. Selected Consolidated Balance Sheet Data (In thousands) (Unaudited) | ||||||||
| September 30, 2024 | December 31, 2023 | |||||||
| Cash, cash equivalents, and marketable securities | $ | 257,234 | $ | 236,221 | ||||
| Working capital(1) | 252,980 | 228,524 | ||||||
| Total assets | 279,008 | 257,694 | ||||||
| Total stockholders’ equity | 257,291 | 235,193 | ||||||
(1) Fulcrum defines working capital as current assets minus current liabilities.
| Fulcrum Therapeutics, Inc. Consolidated Statements of Operations (In thousands, except per share data) (Unaudited) | ||||||||||||||||
| Three Months Ended September 30, | Nine Months Ended September 30, | |||||||||||||||
| 2024 | 2023 | 2024 | 2023 | |||||||||||||
| Collaboration revenue | $ | — | $ | 759 | $ | 80,000 | $ | 1,934 | ||||||||
| Operating expenses: | ||||||||||||||||
| Research and development | 14,639 | 18,238 | 51,673 | 52,802 | ||||||||||||
| General and administrative | 8,424 | 9,961 | 28,732 | 31,804 | ||||||||||||
| Restructuring expenses | 2,063 | — | 2,063 | — | ||||||||||||
| Total operating expenses | 25,126 | 28,199 | 82,468 | 84,606 | ||||||||||||
| Loss from operations | (25,126 | ) | (27,440 | ) | (2,468 | ) | (82,672 | ) | ||||||||
| Other income, net | 3,430 | 3,423 | 9,311 | 10,093 | ||||||||||||
| Net (loss) income | $ | (21,696 | ) | $ | (24,017 | ) | $ | 6,843 | $ | (72,579 | ) | |||||
| Net (loss) income per share, basic | $ | (0.35 | ) | $ | (0.39 | ) | $ | 0.11 | $ | (1.19 | ) | |||||
| Net (loss) income per share, diluted | $ | (0.35 | ) | $ | (0.39 | ) | $ | 0.11 | $ | (1.19 | ) | |||||
| Weighted-average common shares outstanding, basic | 62,409 | 61,823 | 62,200 | 61,121 | ||||||||||||
| Weighted-average common shares outstanding, diluted | 62,409 | 61,823 | 63,688 | 61,121 | ||||||||||||
Contact:
Chris Calabrese
LifeSci Advisors, LLC
ccalabrese@lifesciadvisors.com
917-680-5608
FAQ
What were Fulcrum Therapeutics (FULC) Q3 2024 financial results?
What happened to FULC's losmapimod development program?
When will Fulcrum (FULC) share data from the PIONEER trial?
What is FULC's cash runway projection?
