Significant progress achieved with pipeline focused on rare hematologic diseases and cancers

在治疗罕见血液病和癌症方面取得了重大进展

Positive Phase 1 results in patients with sickle cell disease (SCD) presented at European Hematology Association (EHA) Virtual Congress supporting etavopivat’s potential to significantly impact RBC health and lifespan

在欧洲血液学协会(EHA)虚拟大会上，镰状细胞病(SCD)患者1期阳性结果支持etavopivat显著影响红细胞健康和寿命的潜力

Phase 1 trial of FT-7051 enrolling men with metastatic castration-resistant prostate cancer (mCRPC); initial results to be presented in October at the NCI/AACR/EORTC Virtual International Conference on Molecular Targets and Cancer Therapeutics

FT-7051的1期试验，纳入转移性去雄抵抗前列腺癌(mCRPC)患者;初步结果将于10月在NCI/AACR/EORTC分子靶标和癌症治疗虚拟国际会议上公布

Olutasidenib data in relapsed/refractory acute myeloid leukemia (R/R AML) presented at the American Society of Clinical Oncology (ASCO) and EHA Virtual Congress; new drug application (NDA) preparation ongoing

在美国临床肿瘤学会(ASCO)和EHA虚拟大会上提交的Olutasidenib在复发/难治性急性髓系白血病(R/R AML)中的数据;正在进行新药申请(NDA)准备

WATERTOWN, Mass., August 13, 2021--(BUSINESS WIRE)--Forma Therapeutics Holdings, Inc. (Nasdaq: FMTX), a clinical-stage biopharmaceutical company focused on rare hematologic diseases and cancers, today reported financial results for the second quarter ended June 30, 2021. The company also highlighted recent progress and upcoming milestones for its pipeline programs.

水城,质量。2015年8月13日讯/生物谷bioon /——2015年8月13日讯/生物谷bioon /——2015年8月13日讯/生物谷bioon /生物谷bioon /生物谷bioon /生物谷bioon /生物谷bioon /生物谷bioon /生物谷bioon /生物谷bioon /生物谷bioon.com该公司还强调了其管道项目最近的进展和即将到来的里程碑。

"During the second quarter, we presented positive results from our ongoing Phase 1 trial demonstrating etavopivat’s highly differentiated profile and multimodal mechanism of action to improve markers of sickle cell disease and red blood cell health that are associated with vaso-occlusion," said Frank Lee, president and chief executive officer of Forma. "These results, in addition to the progress on our other clinical programs this quarter, position us well to deliver on our mission of transforming the lives of patients with rare hematologic diseases and cancers."

“在第二季度，我们展示了正在进行的1期试验的积极结果，证明了etavopivat的高分化谱和多模态作用机制，以改善与血管阻塞相关的镰状细胞病和红细胞健康标记物。”Forma总裁兼首席执行官Frank Lee说。“这些结果，加上本季度我们其他临床项目的进展，使我们能够很好地实现我们的使命，改变罕见血液病和癌症患者的生活。”

Key Business and Clinical Highlights

主要业务和临床亮点

PKR Program in Sickle Cell Disease (SCD):Clinical data presented at EHA Virtual Congress support potential of investigational agent etavopivat to significantly impact RBC health and lifespan. Updated results were presented from the two week multiple ascending dose (MAD) cohorts and initial open-label extension (OLE) results administering etavopivat for up to 12 weeks, including:Sustained increases in hemoglobin levels. In the MAD cohorts 73% of patients (11/15) achieved a hemoglobin increase of ≥ 1 g/dL at the end of two-weeks of treatment. In the OLE, hemoglobin levels increased >1g/dL in 88% of patients (7/8) receiving once daily treatment for at least two weeks, and this increase was sustained in those patients receiving continued treatment for up to 12 weeks.

镰状细胞病(SCD)中的PKR计划:EHA虚拟大会上提出的临床数据支持研究药物etavopivat显著影响红细胞健康和寿命的潜力。更新的结果来自两周多次上升剂量(MAD)队列和初始开放标签延长(OLE)结果，给予etavopivat长达12周，包括:血红蛋白水平持续升高。在MAD队列中，73%的患者(11/15)在两周治疗结束时血红蛋白增加≥1 g/dL。在OLE中，88%(7/8)每天接受一次治疗，持续至少两周的患者血红蛋白水平升高>1g/dL，并且这种升高在那些接受持续治疗长达12周的患者中持续。

Improvements in RBC oxygenation and deformability. RBC’s from 14 patients in the MAD cohorts showed increased hemoglobin-oxygen affinity, a significant shift in the point of sickling (POS), and improved deformability.

改善红细胞氧合和变形能力。MAD队列中14例患者的红细胞显示血红蛋白-氧亲和力增加，镰状点(POS)发生显著变化，变形性改善。

Significant reduction in hemolysis with markers approaching normal levels. Reticulocyte counts were reduced in 100% of patients (15/15), with normalization in some patients at the end of 2 weeks of treatment. The majority of patients demonstrated a marked decrease in lactate dehydrogenase levels (LDH) and indirect bilirubin levels as compared to baseline levels.

溶血明显减少，指标接近正常水平。100%的患者(15/15)网织红细胞计数下降，一些患者在治疗2周结束时恢复正常。与基线水平相比，大多数患者的乳酸脱氢酶水平(LDH)和间接胆红素水平显著下降。

Reduction in systemic biomarkers related to inflammation and hypercoagulability. Initial results from the OLE showed improvement in systemic biomarkers such as lower levels of TNF-alpha, a marker of inflammation and decreases in prothrombin 1.2 and D-dimer, markers of coagulation activation.

与炎症和高凝性相关的全身生物标志物减少。OLE的初步结果显示，全身的生物标志物如炎症标志物tnf - α水平降低，凝血酶原1.2和凝血激活标志物d -二聚体水平降低。

Etavopivat was well tolerated with a safety profile consistent with underlying sickle cell disease. Etavopivat was well tolerated at doses up to 600mg daily (150% of the maximum dose in the ongoing Phase 2/3 Hibiscus Study).

Etavopivat耐受性良好，安全性与潜在镰状细胞病一致。Etavopivat的耐受性良好，每日剂量达600mg(为正在进行的2/3期木槿研究中最大剂量的150%)。

CPB/p300 Program in Prostate Cancer: FT-7051 Phase 1 clinical trial enrollment is ongoing. In January 2021, Forma announced the first patient dosed in the ongoing Phase 1 clinical trial evaluating FT-7051 for the treatment of mCRPC. The trial is a multicenter, open-label evaluation of the safety and tolerability, pharmacokinetics/pharmacodynamics (PK/PD), and preliminary anti-tumor activity, of FT-7051 in men with mCRPC who have progressed despite prior therapy with at least one anti-androgen therapy. The adaptive trial design is intended to accelerate the dose escalation to potentially therapeutic doses and yield important safety information, as well as to identify biomarkers of clinical benefit such as PSA response. Genetic mutation analysis will be conducted to correlate genetic changes with resistance to standard-of-care and will also evaluate expression of the AR-v7 splice variant, for which there are no approved therapies.

前列腺癌的CPB/p300计划:FT-7051 1期临床试验正在进行中。2021年1月，Forma宣布了正在进行的评估FT-7051治疗mCRPC的1期临床试验中的首位患者。该试验是一项多中心、开放标签的安全性、耐受性、药代动力学/药理学(PK/PD)和初步抗肿瘤活性的评估，在mCRPC患者中，尽管之前接受过至少一种抗雄激素治疗，但仍有进展。适应性试验设计的目的是加速剂量升级到潜在的治疗剂量，并获得重要的安全信息，以及识别临床受益的生物标志物，如PSA反应。将进行基因突变分析，将基因变化与对标准护理的耐药性联系起来，并将评估AR-v7剪接变体的表达，目前还没有批准的治疗方法。

IDH1 Program in AML and Glioma:Phase 2 registrational results for olutasidenib in R/R AML were presented at scientific conferences. Olutasidenib data in R/R AML were presented at both the annual ASCO and EHA meetings in June 2021. The primary efficacy evaluable population, comprised of 123 patients, received 150 mg of olutasidenib twice daily for at least six months prior to the planned interim analysis. The primary endpoint, a composite complete remission (CR) or CR plus CR with partial hematologic recovery (CRh), was achieved in 33.3% (30% CR and 3% CRh) of patients. While the median duration of response was not yet reached, in a sensitivity analysis with hematopoietic stem cell transplant considered as the end of a response, the median duration was 13.8 months. The median overall survival (OS) was 10.5 months. Although a median OS has not yet been reached for the CR/CRh population, 18-month survival is estimated at 87% for that response category, and median survival is 15.0 months for non-CR/CRh responders. In addition, among patients with a CR who were transfusion-dependent at baseline, 56-day transfusion independence was achieved in 100% of patients as measured by platelets and 80% as measured by RBC’s. Olutasidenib was well-tolerated, and adverse events were consistent with the late stage disease in this heavily pre-treated patient population. Based upon these results, Forma is preparing an NDA for the R/R AML indication.

急性髓系白血病和胶质瘤的IDH1项目:olutasidenib在R/R急性髓系白血病中的2期注册结果已在科学会议上公布。在2021年6月的ASCO和EHA年度会议上，Olutasidenib在R/R AML中的数据都得到了展示。主要疗效评估人群包括123名患者，在计划的中期分析前至少6个月接受了150 mg olutasidenib，每天两次。33.3% (30% CR和3% CRh)患者达到了主要终点，即复合完全缓解(CR)或CR + CR +部分血液学恢复(CRh)。虽然还没有达到缓解的中位持续时间，但在以造血干细胞移植作为缓解结束的敏感性分析中，中位持续时间为13.8个月。中位总生存期(OS)为10.5个月。尽管CR/CRh人群的中位OS尚未确定，但18个月生存率估计为87%，非CR/CRh应答者的中位生存期为15.0个月。此外，在基线时输血依赖的CR患者中，100%的血小板和80%的RBC患者实现了56天的输血独立。Olutasidenib耐受性良好，不良事件与晚期疾病的严重预处理患者群体一致。基于这些结果，Forma正在为R/R AML适应症准备一份NDA。

CorporateIn June 2021, Forma announced the appointment of John E. Bishop, Ph.D., as chief technology officer. Dr. Bishop leads chemistry, manufacturing and control (CMC)-related functions and quality, encompassing Forma’s early pipeline through commercial product. Dr. Bishop’s background includes extensive expertise with CMC development in oncology and hematology. Prior to joining Forma, Dr. Bishop served as senior vice president of pharmaceutical sciences at Epizyme, Inc., where he was a member of the executive team and held overall responsibility for the CMC and quality assurance functions.

2021年6月，Forma宣布任命John E. Bishop博士为首席技术官。Bishop博士领导化学，制造和控制(CMC)相关的功能和质量，包括Forma的早期管道到商业产品。Bishop博士的背景包括肿瘤和血液学CMC发展的广泛专业知识。在加入Forma之前，他曾担任Epizyme, Inc.制药科学高级副总裁，在那里他是执行团队的成员，并全面负责CMC和质量保证职能。

Upcoming MilestonesScientific conference presentation of updated Phase 1 etavopivat results in SCD. Updated results of safety, clinical activity, and biomarkers from the 12-week OLE are expected to be presented at a scientific congress in late 2021. Up to 20 patients are being administered etavopivat 400mg once daily and assessed for hematologic and hemolytic response, improvements in RBC oxygenation and deformability, and systemic markers of SCD.

即将召开的里程碑科学会议介绍了SCD中更新的一期etavopivat结果。为期12周的OLE的安全性、临床活性和生物标志物的更新结果预计将在2021年底的科学大会上公布。多达20例患者每天服用一次etavopivat 400mg，评估其血液学和溶血反应，红细胞氧化和变形能力的改善，以及SCD的全身标志物。

Initiation of etavopivat trials in thalassemia and pediatric sickle cell patients. Enrollment in a Phase 2 trial of etavopivat in thalassemia patients is expected to begin prior to the end of the year, with results anticipated in 2022. The trial may enroll up to 60 patients with either thalassemia or SCD who are receiving chronic red blood cell transfusions, or thalassemia without chronic red blood cell transfusions. A trial in pediatric sickle cell disease patients is planned to begin in the first half of 2022.

在地中海贫血和儿童镰状细胞患者中启动etavopivat试验。一项etavopivat用于地中海贫血患者的2期临床试验预计将在今年年底前开始注册，结果预计将于2022年公布。该试验可能纳入多达60名接受慢性红细胞输血的地中海贫血或SCD患者，或未接受慢性红细胞输血的地中海贫血患者。一项针对儿童镰状细胞病患者的试验计划于2022年上半年开始。

Scientific conference presentation of initial Phase 1 FT-7051 clinical results in mCRPC. An abstract from this ongoing trial has been accepted for presentation at the NCI/AACR/EORTC Virtual International Conference on Molecular Targets and Cancer Therapeutics taking place Oct. 7-10, 2021. The presentation will include preclinical data and initial clinical results on safety, tolerability and PK/PD from patients undergoing dose escalation.

mCRPC 1期FT-7051临床初步结果的科学会议报告。这项正在进行的试验的摘要已被接受在2021年10月7日至10日举行的NCI/AACR/EORTC分子靶标和癌症治疗虚拟国际会议上发表。演示将包括临床前数据和初步临床结果的安全性，耐受性和PK/PD患者接受剂量增加。

Possibility of COVID-19 impact remains. The COVID-19 pandemic remains a factor in the successful completion of these milestones and ongoing clinical trials. Many clinical trials across the biopharma industry, including Forma’s, have been impacted by the COVID-19 pandemic. Clinical trial sites implementing new policies in response to COVID-19 may result in potential delays to enrollment of clinical trials or changes in the ability to access sites participating in clinical trials.

2019冠状病毒病影响的可能性仍然存在。COVID-19大流行仍然是成功完成这些里程碑和正在进行的临床试验的一个因素。包括Forma在内的生物制药行业的许多临床试验都受到了COVID-19大流行的影响。实施应对COVID-19新政策的临床试验站点可能会导致临床试验登记延迟或访问参与临床试验站点的能力发生变化。

Financial ResultsCash Position: Cash, cash equivalents and marketable securities were $570.8 million as of June 30, 2021, as compared to $645.6 million as of Dec. 31, 2020. Current cash runway is projected through the third quarter of 2024.

现金状况:截至2021年6月30日，现金、现金等价物和有价证券为5.708亿美元，而截至2020年12月31日，为6.456亿美元。目前的现金跑道预计将持续到2024年第三季度。

Research and Development (R&D) Expenses: R&D expenses were $31.6 million for the quarter ended June 30, 2021, compared to $20.5 million for the quarter ended June 30, 2020. The increase was primarily attributable to etavopivat development, as well as increases in staff and stock-based compensation.

研发费用:截至2021年6月30日的季度，研发费用为3160万美元，而截至2020年6月30日的季度为2050万美元。增加的主要原因是etavopivat的发展以及工作人员和股票薪酬的增加。

General and Administrative (G&A) Expenses: G&A expenses were $12.5 million for the quarter ended June 30, 2021, compared to $6.4 million for the quarter ended June 30, 2020. The increase in was primarily attributable to increased stock-based compensation, executive and staff hiring, professional fees, and insurance.

一般和行政费用:截至2021年6月30日的季度，一般和行政费用为1250万美元，而截至2020年6月30日的季度为640万美元。增加的主要原因是股票薪酬、高管和员工招聘、专业费用和保险增加。

Net Loss: Net loss was $43.6 million for the quarter ended June 2021, compared to net loss of $25.4 million for the quarter ended June 30, 2020.

净亏损:截至2021年6月的季度净亏损4360万美元，而截至2020年6月30日的季度净亏损2540万美元。

Forma will conduct a conference call and webcast Aug.13 at 8:00 a.m. Eastern Daylight Time (EDT) to discuss second quarter 2021 results and business updates. The call can be accessed by dialing (833) 301-1146 in the U.S., and (914) 987-7386 internationally, with conference ID 9155938.

Forma将于8月13日上午8点举行电话会议和网络直播。东部夏令时(EDT)讨论2021年第二季度业绩和业务更新。在美国可以拨打(833)301-1146，在国际上拨打(914)987-7386，会议号码为9155938。

The live webcast will be available in the "News & Investors" section of Forma’s website www.formatherapeutics.com.

现场直播将在Forma的网站www.formatherapeutics.com的“新闻与投资者”部分进行。

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