Fulcrum Therapeutics Announces Recent Business Highlights and Financial Results for Second Quarter 2025
Fulcrum Therapeutics (NASDAQ:FULC) reported Q2 2025 financial results and clinical progress. The company announced promising results from the 12 mg dose cohort of the Phase 1b PIONEER trial for pociredir in sickle cell disease, showing an 8.6% mean increase in fetal hemoglobin and 67% F-cells, with no serious adverse events.
Financial highlights include $214.1 million in cash reserves as of June 30, 2025, extending runway into 2028. Q2 2025 resulted in a net loss of $17.3 million, compared to net income of $55.4 million in Q2 2024. R&D expenses decreased to $13.0 million from $17.3 million year-over-year, while G&A expenses reduced to $6.8 million from $10.2 million.
Fulcrum Therapeutics (NASDAQ:FULC) ha comunicato i risultati finanziari del secondo trimestre 2025 e i progressi clinici. L'azienda ha annunciato risultati promettenti dal gruppo di dosaggio da 12 mg della fase 1b dello studio PIONEER per pociredir nella malattia a cellule falciformi, evidenziando un aumento medio dell'8,6% dell'emoglobina fetale e il 67% di cellule F, senza eventi avversi gravi.
I punti salienti finanziari includono 214,1 milioni di dollari di riserve di liquidità al 30 giugno 2025, che estendono la disponibilità finanziaria fino al 2028. Il secondo trimestre 2025 ha registrato una perdita netta di 17,3 milioni di dollari, rispetto a un utile netto di 55,4 milioni di dollari nel secondo trimestre 2024. Le spese per ricerca e sviluppo sono diminuite a 13,0 milioni di dollari da 17,3 milioni anno su anno, mentre le spese generali e amministrative si sono ridotte a 6,8 milioni di dollari da 10,2 milioni.
Fulcrum Therapeutics (NASDAQ:FULC) informó los resultados financieros del segundo trimestre de 2025 y avances clínicos. La compañía anunció resultados prometedores del grupo de dosis de 12 mg del ensayo Fase 1b PIONEER para pociredir en la enfermedad de células falciformes, mostrando un aumento medio del 8,6% en hemoglobina fetal y 67% de células F, sin eventos adversos graves.
Los aspectos financieros destacados incluyen 214,1 millones de dólares en reservas de efectivo al 30 de junio de 2025, extendiendo la disponibilidad financiera hasta 2028. El segundo trimestre de 2025 resultó en una pérdida neta de 17,3 millones de dólares, en comparación con una ganancia neta de 55,4 millones en el segundo trimestre de 2024. Los gastos en I+D disminuyeron a 13,0 millones de dólares desde 17,3 millones interanual, mientras que los gastos generales y administrativos se redujeron a 6,8 millones de dólares desde 10,2 millones.
Fulcrum Therapeutics (NASDAQ:FULC)는 2025년 2분기 재무 실적과 임상 진행 상황을 발표했습니다. 회사는 겸상적혈구병 치료제 포시레디르의 12mg 용량 그룹에 대한 1b상 PIONEER 임상시험에서 태아 헤모글로빈 평균 8.6% 증가와 67% F-세포 비율을 보이며 유의미한 결과를 발표했고, 심각한 부작용은 없었습니다.
재무 하이라이트로는 2025년 6월 30일 기준 2억 1410만 달러의 현금 보유고를 기록하여 자금 운용 기간을 2028년까지 연장했습니다. 2025년 2분기 순손실은 1730만 달러로, 2024년 2분기 순이익 5540만 달러와 비교됩니다. 연구개발비는 전년 동기 대비 1300만 달러로 감소했고, 일반관리비는 680만 달러로 줄었습니다.
Fulcrum Therapeutics (NASDAQ:FULC) a publié ses résultats financiers du deuxième trimestre 2025 ainsi que ses progrès cliniques. La société a annoncé des résultats prometteurs pour la cohorte de dose de 12 mg de l'essai de phase 1b PIONEER pour le pociredir dans la drépanocytose, montrant une augmentation moyenne de 8,6 % de l'hémoglobine fœtale et 67 % de cellules F, sans événements indésirables graves.
Les points financiers clés incluent 214,1 millions de dollars en réserves de trésorerie au 30 juin 2025, prolongeant la trésorerie jusqu'en 2028. Le deuxième trimestre 2025 s'est soldé par une perte nette de 17,3 millions de dollars, contre un bénéfice net de 55,4 millions au deuxième trimestre 2024. Les dépenses en R&D ont diminué à 13,0 millions de dollars contre 17,3 millions en glissement annuel, tandis que les dépenses administratives et générales sont passées à 6,8 millions de dollars contre 10,2 millions.
Fulcrum Therapeutics (NASDAQ:FULC) berichtete über die Finanzergebnisse und den klinischen Fortschritt im zweiten Quartal 2025. Das Unternehmen gab vielversprechende Ergebnisse aus der 12 mg-Dosiskohorte der Phase-1b-Studie PIONEER für Pociredir bei Sichelzellanämie bekannt, mit einem durchschnittlichen Anstieg des fetalen Hämoglobins um 8,6% und 67% F-Zellen, ohne schwerwiegende Nebenwirkungen.
Finanzielle Highlights umfassen 214,1 Millionen US-Dollar an Barreserven zum 30. Juni 2025, was die Finanzierung bis 2028 sichert. Im zweiten Quartal 2025 wurde ein Nettoverlust von 17,3 Millionen US-Dollar verzeichnet, im Vergleich zu einem Nettogewinn von 55,4 Millionen US-Dollar im zweiten Quartal 2024. Die F&E-Ausgaben sanken von 17,3 Millionen auf 13,0 Millionen US-Dollar, während die Verwaltungs- und Gemeinkosten von 10,2 Millionen auf 6,8 Millionen US-Dollar reduziert wurden.
- Promising Phase 1b PIONEER trial results with 8.6% mean HbF increase and 67% F-cells
- Strong cash position of $214.1M with runway extended into 2028
- Reduced R&D and G&A expenses by $4.3M and $3.4M respectively year-over-year
- No serious adverse events in pociredir trial with all treatment-related AEs at Grade 1
- Net loss of $17.3M in Q2 2025 compared to net income of $55.4M in Q2 2024
- Zero collaboration revenue vs $80M in Q2 2024
- Cash position decreased by $26.9M from December 2024
Insights
Fulcrum's pociredir shows promising clinical results for sickle cell disease with robust HbF increases and favorable safety profile.
The Phase 1b PIONEER trial results for pociredir represent a significant clinical advancement in sickle cell disease treatment. At the 12 mg dose, the drug demonstrated a mean HbF increase of 8.6% with 67% F-cells, indicating robust pan-cellular distribution. This is particularly noteworthy as HbF levels above 8-10% are generally associated with milder disease phenotypes in SCD.
The 0.9 g/dL increase in total hemoglobin alongside improvements in hemolysis markers suggests meaningful clinical benefit beyond just HbF induction. For context, even a 1 g/dL hemoglobin increase can substantially improve quality of life and reduce complications in SCD patients.
The safety profile appears excellent with no drug-related serious adverse events, no discontinuations due to adverse events, and all treatment-related events being Grade 1 (mild). This favorable tolerability is crucial for a chronic disease requiring lifelong treatment.
While the trends in VOC reductions are described as "encouraging" without specific numbers, this preliminary signal suggests potential for reducing the most debilitating aspect of SCD. The upcoming 20 mg cohort data will be critical to establish dose-response relationships and optimal therapeutic dosing.
Pociredir's oral, once-daily administration represents a potential advantage over existing gene therapies and monthly infusions, potentially addressing the significant treatment burden in SCD. The data supports advancing to later-stage trials, though confirmation in larger populations with longer follow-up will be essential to validate these promising early results.
Fulcrum shows strong clinical progress while maintaining solid financial position with $214.1M cash extending runway into 2028.
Fulcrum's financial position remains robust with $214.1 million in cash and equivalents, providing runway into 2028—a substantial extension from previous guidance. This cash preservation is particularly impressive given the advancement of their clinical programs, suggesting efficient capital deployment.
The quarterly cash burn of approximately $13.5 million (derived from the $26.9M six-month decrease) appears sustainable given their extended runway projection. R&D expenses decreased 24.9% year-over-year to $13.0 million, while G&A expenses dropped 33.3% to $6.8 million, reflecting disciplined cost management following their 2024 workforce reduction.
The dramatic shift from $55.4 million net income in Q2 2024 to a $17.3 million net loss in Q2 2025 is primarily due to the non-recurring $80 million Sanofi collaboration payment recognized in the prior year period. Excluding this one-time revenue, the underlying operational loss has actually improved year-over-year.
The pipeline diversification strategy is progressing with plans to submit an IND for Diamond-Blackfan anemia in Q4 2025, potentially creating additional value drivers beyond pociredir. The positive clinical data for pociredir in SCD represents significant value creation, as this indication represents a multi-billion dollar market opportunity where effective oral therapies could capture substantial market share.
The extended cash runway into 2028 provides sufficient capital to potentially advance pociredir through pivotal trials without immediate financing pressure, significantly reducing dilution risk for current shareholders while allowing full value capture from upcoming clinical catalysts.
― Announced results from the 12 mg dose cohort (n=16) of the Phase 1b PIONEER trial of pociredir in sickle cell disease (SCD); pociredir was generally well-tolerated with no treatment-related serious adverse events (SAEs) ―
― Observed robust and rapid pan-cellular increases in fetal hemoglobin (HbF); meaningful improvements in key markers of hemolysis and anemia; encouraging trends in vaso-occlusive crises (VOCs) ―
― On track to provide clinical data from the 20 mg dose cohort by the end of 2025 ―
― Ended Q2 2025 with
CAMBRIDGE, Mass., July 29, 2025 (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 second quarter of 2025 and provided a business update.
“Fulcrum has made substantial progress this quarter, having reported very promising results from the 12 mg cohort of the PIONEER trial,” said Alex C. Sapir, Fulcrum’s President and Chief Executive Officer. “We believe that this data demonstrates that pociredir has the potential to increase fetal hemoglobin to levels that could ameliorate SCD symptomology and transform the standard of care with a once daily oral treatment option. We look forward to reporting the 20 mg data later this year and progressing pociredir into later-stage development.”
Recent Business Highlights
- Announced results from the 12 mg dose cohort of the PIONEER trial, following conclusion of the 12-week treatment period. Results included a robust mean increase of
8.6% in HbF, evidence of pan-cellular induction of HbF shown by a mean of67% F-cells, improvements in markers of hemolysis and a 0.9 g/dL increase in total hemoglobin, and encouraging trends in VOC reductions. Pociredir was generally well-tolerated, with no drug-related SAEs and no discontinuations due to treatment-emergent adverse events through the completion of the 12 mg dose cohort. In addition, all treatment-related AEs through completion of the 12 mg dose cohort were Grade 1. - The 20 mg dose cohort is ongoing, and Fulcrum plans to share data from this cohort by the end of 2025.
- Two abstracts were presented at the 2025 European Hematology Association (EHA) Congress in Milan, Italy, which took place June 12-15, 2025. The abstracts highlight preclinical target engagement and gene expression reversibility data of pociredir and clinical data from our previously completed Phase 1 healthy volunteer study.
- Fulcrum continues to advance its program for the potential treatment of inherited aplastic anemias, such as Diamond-Blackfan anemia (DBA), Shwachman-Diamond syndrome, and Fanconi anemia, and plans to submit an investigational new drug application (IND) for DBA during the fourth quarter of 2025.
Second Quarter 2025 Financial Results
- Cash Position: As of June 30, 2025, cash, cash equivalents, and marketable securities were
$214.1 million , as compared to$241.0 million as of December 31, 2024. The decrease of$26.9 million is primarily due to cash used to fund operating activities in 2025. - Collaboration Revenue: Collaboration revenue was zero for the three months ended June 30, 2025, as compared to
$80.0 million for the three months ended June 30, 2024. The decrease of$80.0 million was primarily due to the recognition of the$80.0 million upfront license payment received from Sanofi during the second quarter of 2024. - R&D Expenses: Research and development expenses were
$13.0 million for the three months ended June 30, 2025, as compared to$17.3 million for the three months ended June 30, 2024. The decrease of$4.3 million was primarily due to decreased employee compensation costs as a result of the reduction in workforce implemented in the third quarter of 2024 as well as decreased costs associated with the discontinuation of our losmapimod program, partially offset by increased costs related to the advancement of the Phase 1b PIONEER trial of pociredir. - G&A Expenses: General and administrative expenses were
$6.8 million for the three months ended June 30, 2025, as compared to$10.2 million for the three months ended June 30, 2024. The decrease of$3.4 million was primarily due to decreased professional services costs as well as decreased employee compensation costs as a result of the reduction in workforce implemented in the third quarter of 2024. - Net Loss: Net loss was
$17.3 million for the three months ended June 30, 2025, as compared to net income of$55.4 million for the three months ended June 30, 2024.
Updated Cash Runway Guidance
Based on its current operating plans, Fulcrum now expects that its current cash, cash equivalents, and marketable securities will be sufficient to fund its operating requirements into 2028.
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 clinical program is pociredir, a small molecule designed to increase expression of HbF 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 HbF. Pociredir is being developed for the treatment of SCD. Initial data in SCD in the PIONEER Phase 1b clinical trial showed proof-of-concept and achieved absolute levels of HbF increases associated with potential overall patient benefit. Through the completion of the 12 mg dose cohort, pociredir was demonstrated to be generally well-tolerated in people with SCD with up to three months of exposure, with no treatment-related serious adverse events reported. Pociredir has been granted FDA Fast Track designation and Orphan Drug Designation for the treatment of SCD. To learn more about clinical trials of pociredir please visit ClinicalTrials.gov.
About 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 planned data announcement for such trial; the potential of pociredir to increase HbF to levels that could ameliorate symptoms of SCD and transform the standard of care; Fulcrum’s ability to progress its early stage development programs and planned IND filings related thereto; and its projected cash runway, 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; realizing 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) | ||||||||
June 30, 2025 | December 31, 2024 | |||||||
Cash, cash equivalents, and marketable securities | $ | 214,111 | $ | 241,021 | ||||
Working capital(1) | 210,388 | 238,879 | ||||||
Total assets | 228,838 | 260,718 | ||||||
Total stockholders’ equity | 214,378 | 243,034 | ||||||
(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 June 30, | Six Months Ended June 30, | |||||||||||||||
2025 | 2024 | 2025 | 2024 | |||||||||||||
Collaboration revenue | — | 80,000 | — | 80,000 | ||||||||||||
Operating expenses: | ||||||||||||||||
Research and development | 12,987 | 17,261 | 26,391 | 37,034 | ||||||||||||
General and administrative | 6,828 | 10,247 | 13,827 | 20,308 | ||||||||||||
Total operating expenses | 19,815 | 27,508 | 40,218 | 57,342 | ||||||||||||
(Loss) income from operations | (19,815 | ) | 52,492 | (40,218 | ) | 22,658 | ||||||||||
Other income, net | 2,519 | 2,917 | 5,267 | 5,881 | ||||||||||||
Net (loss) income | $ | (17,296 | ) | $ | 55,409 | $ | (34,951 | ) | $ | 28,539 | ||||||
Net (loss) income per share, basic | $ | (0.28 | ) | $ | 0.89 | $ | (0.56 | ) | $ | 0.46 | ||||||
Net (loss) income per share, diluted | $ | (0.28 | ) | $ | 0.87 | $ | (0.56 | ) | $ | 0.45 | ||||||
Weighted-average common shares outstanding, basic | 62,544 | 62,205 | 62,506 | 62,095 | ||||||||||||
Weighted-average common shares outstanding, diluted | 62,544 | 63,587 | 62,506 | 63,684 | ||||||||||||
Contact:
Alan Musso
Chief Financial Officer
amusso@fulcrumtx.com
