On December 5, 2020 Syros Pharmaceuticals (NASDAQ:SYRS), a leader in the development of medicines that control the expression of genes, reported that it has acquired from Orsenix, LLC (Orsenix) all of its assets related to SY-2101, formerly known as ORH-2014, a novel oral form of arsenic trioxide (ATO) (Press release, Syros Pharmaceuticals, DEC 5, 2020, View Source [SID1234572245]). SY-2101 represents a strategic opportunity to leverage Syros’ expertise and capabilities to advance its growing footprint in hematologic disorders, with a targeted clinical-stage drug candidate that has the potential to dramatically reduce the treatment burden of a standard-of-care regimen for newly diagnosed acute promyelocytic leukemia (APL).

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"It’s rare to talk about cures in cancer," said Nancy Simonian, M.D., Chief Executive Officer of Syros. "The IV formulation of ATO is part of a treatment regimen that cures most APL patients, but it is extremely burdensome with lengthy infusions over a nearly yearlong course of treatment. We believe an oral form that offers similar efficacy while dramatically reducing the treatment burden on these patients could quickly become a new standard-of-care. SY-2101 is also highly complementary to our efforts with SY-1425 in AML and MDS, positioning us for potential new drug applications for both programs in 2024. With the strategic financing announced today, we believe we are well-funded to advance our growing portfolio of product candidates across multiple patient populations to accelerate our vision of becoming a fully integrated biopharmaceutical company."

A Potential New Standard-of-Care Therapy

SY-2101 is in development for the treatment of APL, a subtype of acute myeloid leukemia (AML) defined by a fusion of the RARA and PML genes. An intravenously administered formulation of ATO is approved for use in combination with All-Trans-Retinoic-Acid (ATRA) in newly diagnosed APL and, while curative in approximately 80-90% of patients, its administration requires up to 140 two- to four-hour infusions over the typical course of induction and consolidation treatment.

Because SY-2101 is dosed orally once daily, it has the potential to become the standard-of-care frontline therapy for APL by providing comparable efficacy with a substantially more convenient option that reduces the treatment burden on patients, improving access, and lowering costs to the healthcare system. In a Phase 1 clinical trial, led by investigators at the M.D. Anderson Cancer Center, SY-2101 demonstrated bioavailability, pharmacokinetic (PK) exposures similar to IV ATO, and a generally well-tolerated safety profile.

Clinical Development Plans for SY-2101

Syros plans to initiate a dose confirmation study of SY-2101 in the second half of 2021. Following confirmation of a dose that demonstrates comparable PK to IV ATO, Syros intends to initiate a registration-enabling Phase 3 trial in patients with newly diagnosed APL in 2022. Based on interactions between Orsenix and the U.S. Food and Drug Administration (FDA), Syros believes molecular complete response rate and event-free survival in comparison to historical control data with IV ATO would support accelerated and full approval, respectively. If successful, Syros believes it could file a New Drug Application (NDA) with the FDA in 2024.

SY-2101 has orphan drug designation in the United States for the treatment of APL and Europe for the treatment of AML.

Acquisition Details

Under the terms of the asset purchase agreement, Syros has acquired all assets related to the development and commercialization of SY-2101, including intellectual property, clinical and preclinical data, the regulatory dossier, and product inventory. Syros has made an upfront cash payment of $12 million to Orsenix. In addition, Orsenix is eligible to receive a $6 million regulatory milestone related to the development of SY-2101 in APL and commercial milestones of up to $10 million. Orsenix is also eligible to receive single-digit million milestone payments related to the development of SY-2101 in indications other than APL.

Financing Details

Syros also announced today that it has entered into a definitive agreement for the sale of its equity securities in a private placement to a group of institutional accredited investors led by Bain Capital Life Sciences with participation from Ally Bridge Group, Omega Funds, OrbiMed, EcoR1 Capital, and Samsara BioCapital. The agreement provides for the sale of an aggregate of 10,312,500 shares of Syros’ common stock and, in lieu of shares of common stock, pre-funded warrants (Pre-Funded Warrants) to purchase an aggregate of 1,000,000 shares of common stock, and accompanying warrants (Warrants) to purchase an aggregate of up to 2,828,125 additional shares of common stock (or Pre-Funded Warrants in lieu thereof) at a price of $8.00 per share and accompanying Warrant (or $7.99 per Pre-Funded Warrant and accompanying Warrant). The price per Pre-Funded Warrant and accompanying Warrant represents the price of $8.00 per share and accompanying Warrant to be sold in the private placement, minus the $0.01 per share exercise price of each such Pre-Funded Warrant. The exercise price of the Warrants is $11.00 per share, or if exercised for a Pre-Funded Warrant in lieu thereof, $10.99 per Pre-Funded Warrant (representing the Warrant exercise price of $11.00 per share minus the $0.01 per share exercise price of each such Pre-Funded Warrant). The Warrants are exercisable at any time during the period beginning six months following the closing date of the private placement and ending on the fifth anniversary of the closing. The Pre-Funded Warrants are exercisable at any time after their original issuance and will not expire. The gross proceeds from the sales of common stock and Pre-Funded Warrants are expected to be $90.5 million, before deducting offering expenses. The private placement is expected to close on or about December 8, 2020, subject to the satisfaction of customary closing conditions.

The securities to be sold in the private placement have not been registered under the Securities Act of 1933, as amended (Securities Act), or any state or other applicable jurisdiction’s securities laws, and may not be offered or sold in the United States absent registration or an applicable exemption from the registration requirements of the Securities Act and applicable state or other jurisdictions’ securities laws.

This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any offer, solicitation or sale of these securities in any jurisdiction in which such offer, solicitation or sale would be unlawful.

Updated Financial Guidance

As a result of these transactions, Syros expects to have sufficient funds for planned operating expenses and capital expenditure requirements into the second half of 2022.

Conference Call Information

Syros will host a conference call today at 4:30 p.m. ET to discuss its acquisition of SY-2101, as well as the new data for SY-1425, which were presented at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition.

To access the live conference call, please dial 866-5954538 (domestic) or 636-812-6496 (international), and refer to conference ID 1264464. A webcast of the call will also be available on the Investors & Media section of the Syros website at www.syros.com. An archived replay of the webcast will be available for approximately 30 days following the presentation.

About Acute Promyelocytic Leukemia

APL is a well-defined subset of AML caused by the formation of an abnormal fusion gene, PML/RARA. This fusion gene leads to the overproduction of immature white blood cells called promyelocytes and the underproduction of healthy blood cells. Signs, symptoms and complications of APL include abnormal bleeding and bruising, anemia, fatigue, and increased risk of infection. APL makes up about 10% of AML cases, with an annual incidence of approximately 2,000 patients in the United States and Europe.

On December 5, 2020 Syros Pharmaceuticals (NASDAQ:SYRS), a leader in the development of medicines that control the expression of genes, reported new clinical data from its Phase 2 trial evaluating SY-1425, its first-in-class selective retinoic acid receptor alpha (RARα) agonist, in combination with azacitidine in two acute myeloid leukemia (AML) patient populations (Press release, Syros Pharmaceuticals, DEC 5, 2020, View Source [SID1234572244]). The data is being presented today in oral presentations at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting. In a separate poster presentation on Monday, Syros will present translational data highlighting the potential of SY-1425 to benefit newly diagnosed unfit AML patients who may be resistant to the standard-of-care combination of venetoclax plus azacitidine.

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"The data that continue to emerge from the Phase 2 trial of SY-1425 are compelling," said Stéphane De Botton, M.D., Head of Acute Myeloid Malignancies at Institut Gustave Roussy and a clinical investigator in the trial. "SY-1425 in combination with azacitidine demonstrates high response rates, rapid onset of action, meaningful durability and favorable tolerability in RARA-positive newly diagnosed unfit AML patients. Despite recent advances, one-third of unfit AML patients still don’t respond to the standard-of-care regimen in the upfront setting and what we are now learning is that most of these patients may be RARA-positive. Taken together, these data support the potential of SY-1425 to become an important targeted therapy for RARA-positive AML patients, as well as for patients with a closely related hematologic malignancy known as higher-risk myelodysplastic syndrome."

"The new data presented today strengthen our conviction that SY-1425 has the potential to become the foundation of care for all RARA-positive patients," said David A. Roth, M.D., Chief Medical Officer of Syros. "Based on these results, we are excited to launch our planned registration-enabling Phase 3 trial in newly diagnosed higher-risk MDS patients, which puts us on track for a potential new drug application in 2024, and a randomized Phase 2 clinical trial evaluating SY-1425 as part of a triplet regimen with venetoclax and azacitidine in newly diagnosed unfit AML patients."

Promising New Data from Phase 2 Trial of SY-1425 in RARA-positive AML
Syros presented new data from its fully enrolled Phase 2 trial evaluating the safety and efficacy of SY-1425 in combination with azacitidine in newly diagnosed AML patients who are not suitable candidates for standard chemotherapy, as well as in RARA-positive relapsed or refractory (R/R) AML patients.

Mature Data in Newly Diagnosed Unfit AML
As of October 1st, 51 newly diagnosed unfit AML patients, including both RARA-positive and RARA-negative patients, were eligible for a safety analysis. Eighteen RARA-positive were evaluable for clinical response. In those patients, the data showed that:

Overall response rate (ORR) was 67% (12/18), with a composite CR rate of 61%, (11/18) including nine patients (50%) achieving a complete response (CR), two patients (11%) achieving a complete response with incomplete blood count recovery (CRi).
89% (8/9) of CRs were deep molecular or cytogenetic CRs.
Responses were seen across AML risk groups, including patients with mutations that are typically associated with poor outcomes.
Median time to initial response was 1.2 months.
Median duration of response was 10.8 months, and median overall survival (OS) among patients who achieved a CR or CRi was 18 months.
86% (6/7) of patients who were transfusion dependent at baseline became transfusion independent, and 67% (12/18) of patients achieved or maintained transfusion independence.
SY-1425 in combination with azacitidine was generally well-tolerated with no evidence of increased toxicity relative to either as a single agent, including rates of myelosuppression that were comparable to single-agent azacitidine.
Syros will also present new translational data demonstrating that most RARA-positive newly diagnosed unfit AML patients have a monocytic disease phenotype that is highly correlated with resistance to upfront treatment with venetoclax and azacitidine. These data suggest that the RARA biomarker not only selects for patients who are more likely to respond to treatment with SY-1425 but also for patients who are less likely to respond to treatment with venetoclax and azacitidine.

Data in Relapsed or Refractory AML
As of October 1st, 28 RARA-positive R/R AML patients were eligible for safety analysis and 21 were evaluable for clinical response. In those patients, the data showed:

ORR was 19% (4/21), consisting of one CRc, two CRi and one MLFS.
In HMA and venetoclax naïve patients, the ORR was 43% (3/7 patients).
Median OS was 5.9 months.
30% (6/20) achieved or maintained transfusion independence, including 27% (3/11) of patients who were transfusion dependent at baseline.
SY-1425 in combination with azacitidine was also generally well-tolerated in this patient population.
Advancing SY-1425 in Newly Diagnosed HR-MDS and Unfit AML
Based on the encouraging clinical activity and favorable safety and tolerability profile of SY-1425 in combination with azacitidine as well as an assessment of ongoing areas of high unmet need within the evolving treatment landscape, Syros plans to advance SY-1425 in combination with azacitidine into a registration-enabling Phase 3 trial in RARA-positive newly diagnosed higher-risk myelodysplastic syndrome (HR-MDS) patients. HR-MDS is a hematologic malignancy that is closely related to AML, and as in AML, about 30 percent of HR-MDS patients are RARA-positive.

Based on feedback from the U.S. Food and Drug Administration, Syros plans to enroll approximately 190 RARA-positive newly diagnosed HR-MDS patients in the double-blind placebo-controlled trial, randomized 2:1 to receive SY-1425 in combination with azacitidine or placebo with azacitidine, respectively. The primary endpoint of the trial will be the CR rate, which, depending on the data outcome, could support accelerated or full approval in this patient population. Syros expects to initiate the Phase 3 trial in the first quarter of 2021.

In addition, Syros plans to advance SY-1425 in combination with venetoclax and azacitidine in RARA-positive newly diagnosed unfit AML patients. The trial is designed with a single-arm safety lead-in to confirm the dosing regimen of the triplet to be used in the randomized portion of the Phase 2 trial, which will evaluate the safety and efficacy of SY-1425 in combination with venetoclax and azacitidine compared to venetoclax and azacitidine in approximately 80 patients randomized 1:1. The trial will also evaluate the triplet as a salvage therapy in patients who don’t respond to venetoclax and azacitidine. Syros expects to initiate the Phase 2 trial in the second half of 2021.

Conference Call Information
Syros will host a conference call today at 4:30 p.m. ET to discuss these data, as well as its acquisition of SY-2101, a clinical-stage drug candidate for the treatment of acute promyelocytic leukemia, from Orsenix, LLC, which was also announced today.

To access the live conference call, please dial 866-595-4538 (domestic) or 636-812-6496 (international), and refer to conference ID 1264464. A webcast of the call will also be available on the Investors & Media section of the Syros website at www.syros.com. An archived replay of the webcast will be available for approximately 30 days following the presentation.

On December 5, 2020 Gamida Cell Ltd. (Nasdaq: GMDA), an advanced cell therapy company committed to cures for blood cancers and serious hematologic diseases, reported in an oral presentation the updated and expanded results from a Phase 1 clinical study of GDA-201, an investigational, natural killer (NK) cell-based cancer immunotherapy for the treatment of patients with non-Hodgkin lymphoma (NHL) and multiple myeloma (MM), at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition, which is being held virtually December 5–8 (Press release, Gamida Cell, DEC 5, 2020, View Source [SID1234572243]).

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GDA-201 was well-tolerated and no dose-limiting toxicities were observed in 35 patients (19 with NHL and 16 with MM). The data show that therapy using GDA-201 with monoclonal antibodies demonstrated significant clinical activity in heavily pretreated patients with advanced NHL. Of the 19 patients with NHL, 13 complete responses and one partial response were observed, with an overall response rate of 74 percent and a complete response rate of 68 percent. The maximum tolerated dose was not achieved, as no dose limiting toxicities were observed in patients who received the maximum target dose (2 x 108 cells/kg).

"Data from an expanded group of patients in this Phase 1 clinical study for GDA-201 show that NK cell therapies continue to exhibit impressive therapeutic potential to treat relapsed and refractory patients with lymphomas, while maintaining a favorable safety profile," said Veronika Bachanova, M.D., Ph.D., Professor of Medicine in the Division of Hematology, Oncology and Transplantation at the University of Minnesota and principal investigator of the study. "Despite recent advancements in therapies for patients with hematologic malignancies, too many patients progress to develop refractory or resistant disease. I look forward to the continued clinical development of this novel investigational therapy."

NK cell immunotherapies are thought to offer tremendous potential for transforming the care of hematologic malignancies. With GDA-201, Gamida Cell is pioneering a novel approach that harnesses the power of its cell expansion technology to improve antibody-dependent cellular cytotoxicity (ADCC) and tumor targeting of NK cells.

"These additional results again show that GDA-201 has striking signs of efficacy and safety in patients with heavily pre-treated NHL," said Julian Adams, Ph.D., chief executive officer of Gamida Cell. "With these results in hand, we plan to initiate a Phase 2 clinical study, with the goal of submitting an IND in 2021."

GDA-201 Phase 1 Clinical Data

The presentation, "Results of a Phase 1 Trial of GDA-201, Nicotinamide-Expanded Allogeneic Natural Killer (NK) Cells in Patients with Refractory Non-Hodgkin Lymphoma (NHL) and Multiple Myeloma," described results from the Phase 1 clinical study of GDA-201 in heavily pre-treated patients with advanced NHL and MM. Preliminary results from this study were presented at the 2019 ASH (Free ASH Whitepaper) Annual Meeting.

In the study, cell therapy using GDA-201 with monoclonal antibodies was shown to be safe; there were no dose-limiting toxicities, neurotoxic events, confirmed cytokine release syndrome, graft versus host disease or marrow aplasia. Overall survival and progression-free survival at one year in the NHL cohort suggest durable disease control, with a median follow-up of ten months (range 1–28 months). The most common adverse events were decreased neutrophil count, febrile neutropenia, anemia and low platelet counts.

In the NHL cohort, durable complete responses were observed in patients with both follicular and diffuse large B cell lymphoma, with an overall response rate of 74 percent. Future development of GDA-201 may include cryopreservation and the exploration of multiple treatment cycles in a multi-center Phase 2 trial in patients with NHL.

About GDA-201

Gamida Cell applied the capabilities of its NAM-based cell expansion technology to develop GDA-201, an innate natural killer (NK) cell immunotherapy for the treatment of hematologic and solid tumors in combination with standard of care antibody therapies. GDA-201 addresses key limitations of NK cells by increasing the cytotoxicity and in vivo retention and proliferation in the bone marrow and lymphoid organs of NK cells expanded in culture. GDA-201 is in Phase 1 development through an investigator-sponsored study in patients with refractory non-Hodgkin lymphoma and multiple myeloma.1 For more information on the clinical study of GDA-201, please visit www.clinicaltrials.gov.

GDA-201 is an investigational therapy, and its safety and efficacy have not been established by the U.S. Food and Drug Administration or any other health authority.

About the NAM Therapeutic Platform

Gamida Cell’s proprietary NAM-based cell expansion platform is designed to enhance the number and functionality of donor cells in culture, enabling the creation of potentially transformative therapies that move beyond what is possible with existing approaches. The NAM therapeutic platform leverages the unique properties of nicotinamide to enable the expansion of multiple cell types — including stem cells and natural killer (NK) cells — with appropriate growth factors to maintain the cells’ original phenotype and potency. This can enable the administration of a therapeutic dose of cells with the potential to improve patient outcomes.

On December 5, 2020 Rhizen Pharmaceuticals, a clinical-stage oncology-focussed biopharmaceutical company, reported presentation of the interim results of a Phase I/II combination study of Tenalisib with Romidepsin for relapsed/refractory T-Cell Lymphoma patients at the upcoming 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting and exposition, to be held virtually from December 5 – 8, 2020 (Press release, Rhizen Pharmaceuticals, DEC 5, 2020, View Source [SID1234572242]).

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The poster presentation at ASH (Free ASH Whitepaper), 2020 includes interim data from a multi-center, open label, Phase I/II study in patients with T cell lymphoma (NCT03770000) with the objective of determining the MTD/optimal dose of Tenalisib given in combination with Romidepsin and assessing the preliminary efficacy of the combination. No dose limiting toxicities were reported and Tenalisib 800 mg BID plus Romidepsin 14 mg/m2 was determined to be the maximum tolerated dose for the expansion cohorts. Overall, the Tenalisib and Romidepsin combination was well tolerated across all patients (N=33) with no unexpected adverse events or increased frequency of adverse events previously reported for the individual agents.

As of the cut-off date (5th Nov 2020) for data analysis, seven PTCL patients had completed their first efficacy assessment (C3D1), of which four patients showed a complete response, one demonstrated a partial response and two had stable disease. The duration of response ranged from 1.4+ to 8.16+ months. Of the eleven CTCL patients who had completed their first efficacy assessment (C3D1), one patient showed a complete response, three demonstrated a partial response and five had stable disease. The duration of response ranged from 1.20+ to 10.6+ months.

Swaroop Vakkalanka, the company’s President and CEO stated, "We are extremely encouraged by the interim data being presented today which reinforces Tenalisib’s stellar safety and impressive efficacy when studied in combination with Romidepsin. Both PTCL and CTCL are difficult to treat indications and patients quickly run out of options, so we are excited with the preliminary results. We expect this study to eventually support a pivotal trial of the combination and we plan to approach the FDA when the full study results are available.

The Tenalisib poster session details at ASH (Free ASH Whitepaper) 2020 are noted below:

Poster Title: A Multi-Center, Open Label, Phase I/II Study to Assess the Safety and Efficacy of Tenalisib Given in Combination with Romidepsin in Patients with Relapsed/Refractory T-Cell Lymphoma (Publication Number: 1155)
Session: 624. Hodgkin Lymphoma and T/NK Cell Lymphoma—Clinical Studies: Poster I
Date: Saturday, December 5, 2020; 7:00 AM – 3:30 PM (Pacific Time)
Presenter: Swaminathan P Iyer, MD, Professor, Department of Lymphoma/Myeloma, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, Houston, TX
About Tenalisib (RP 6530):

Tenalisib (RP6530) is a highly selective next generation orally active dual PI3K δ/γ inhibitor, which is in Phase 2 clinical development for hematological malignancies and solid tumors. Tenalisib has been granted US FDA Fast Track Designations for treatment of relapsed/refractory peripheral T-cell lymphoma and cutaneous T-cell lymphoma (R/R PTCL and R/R CTCL), in addition to Orphan-Drug Designations for treatment of peripheral and cutaneous T-cell lymphoma (PTCL and CTCL).

About T-Cell Lymphomas:

T-Cell Lymphomas (TCL) are a group of cancers that originate in T-cells and develop in lymphoid tissues such as the lymph nodes and spleen, or outside of lymphoid tissues (i.e., gastrointestinal tract, liver, nasal cavity, skin, and others). TCL constitute ~7-15% of all NHL cases and can be generally classified based on their presentation, as indolent or aggressive.

Peripheral T-Cell Lymphoma (PTCL) describes a heterogeneous group of lymphoproliferative disorders arising from mature T-Cells and accounts for ~10% of all NHL cases. PTCL is an aggressive disease that most commonly presents in patients over the age of 60 and usually has a worse prognosis than diffuse large B cell lymphoma.

Cutaneous T-Cell Lymphoma (CTCL) describes a group of typically indolent lymphomas that appear on, and are most often confined to, the skin and accounts for ~3% of all NHL cases and usually affects adults. CTCL subtypes include the more common and indolent Mycosis Fungoides (MF), which is largely confined to the skin, and the less common but more severe Sezary Syndrome (SS) that affects both the skin and blood and has poor prognosis.

On December 5, 2020 Imago BioSciences, Inc., ("Imago") a clinical-stage biotechnology company developing innovative treatments for myeloid diseases, reported interim data from 49 patients in its ongoing Phase 2b study evaluating bomedemstat (IMG-7289) for the treatment of myelofibrosis (Press release, Imago BioSciences, DEC 5, 2020, View Source [SID1234572241]). The data demonstrated sustained clinical activity and a favorable tolerability profile for the investigational treatment. The data were presented in an oral session by Kristen M. Pettit, M.D., assistant professor of medicine at the University of Michigan Rogel Cancer Center, during the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition.

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The interim results included an analysis of total symptom score (TSS) reduction at 12 weeks in 32 patients, with 78% of patients showing an improvement. In addition, 86% of patients evaluable (N=14) for spleen volume showed a reduction at 12 weeks. In this patient population in which 51% were classified as high molecular risk for evolution to AML, a serial analysis of mutant alleles revealed that 32% of patients experienced a decrease in the frequency of mutant alleles from baseline and 55% remained at stable frequencies. In patients followed for up to 570 days, no patient acquired new mutations in any of the 261 genes associated with myeloid malignancies and none to date have developed leukemia.

"As we continue to advance bomedemstat through Phase 2b clinical trials in myelofibrosis and essential thrombocythemia, we are encouraged by the interim results presented today," said Hugh Young Rienhoff, Jr. MD, CEO, Imago BioSciences. "There is a grave unmet need today among patients with myeloid diseases, and we look forward to realizing the promise of bomedemstat as a new therapeutic option with the potential to alter the natural history of these diseases."

Safety data were presented showing a total of 917 adverse events including 6 serious adverse events each occurring once deemed by Investigators as possibly related to IMG-7289. There have been no safety signals, dose-limiting toxicities, or deaths related to the drug.

About Bomedemstat (IMG-7289)

Bomedemstat is an orally available small molecule discovered and developed by Imago BioSciences that inhibits lysine-specific demethylase 1 (LSD1 or KDM1A), an enzyme shown to be vital in cancer stem/progenitor cells, particularly neoplastic bone marrow cells. In non-clinical studies, bomedemstat demonstrated robust in vivo anti-tumor efficacy across a range of myeloid malignancies as a single agent and in combination with other therapeutic agents. Bomedemstat is an investigational agent currently being evaluated in ongoing clinical trials (ClinicalTrials.gov Identifier: NCT03136185, NCT04262141, NCT04254978 and NCT04081220).

Bomedemstat has U.S. FDA Orphan Drug and Fast Track Designation for the treatment of myelofibrosis and essential thrombocythemia, Orphan Drug Designation for treatment of acute myeloid leukemia and PRIME designation by the European Medicines Agency for the treatment of MF.

Bomedemstat is being evaluated in three open-label Phase 2 clinical trials for the treatment of advanced myelofibrosis (MF), polycythemia vera (PV) and essential thrombocythemia (ET), bone marrow cancers that interfere with the production of blood cells. MF patients who are resistant to a Janus Kinase (JAK) inhibitor are eligible for the study of bomedemstat. ET patients who have failed one standard of care treatment are eligible for the bomedemstat ET study.