On November 2, 2023 ImmunoGen Inc. (Nasdaq: IMGN), a leader in the expanding field of antibody-drug conjugates (ADCs) for the treatment of cancer, reported recent progress in the business and reported financial results for the quarter ended September 30, 2023 (Press release, ImmunoGen, NOV 2, 2023, View Source [SID1234636766]).

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"Building on the momentum generated in the first half of 2023, we delivered a strong third quarter highlighted by significant ELAHERE revenue growth and the achievement of key operational milestones," said Mark Enyedy, ImmunoGen’s President and Chief Executive Officer. "With a 36% increase in sequential quarterly net sales and over $210 million in product revenue through the first nine months of the year, ELAHERE is tracking towards one of the most successful first product launches in oncology in a decade. We also made important progress in our effort to bring ELAHERE to eligible patients around the world with the acceptance of the MAA by the EMA and the NDA by the NMPA, and the establishment of a collaboration with Takeda to deliver ELAHERE in Japan."

Enyedy continued, "In addition, we advanced the ELAHERE development program and are pleased to report that our PICCOLO trial in platinum-sensitive ovarian cancer has met the primary endpoint of objective response rate based on an interim efficacy assessment. With a number of patients remaining on treatment and longer follow-up required to establish mature response durability of ELAHERE in this patient population, we anticipate an ORR of at least 48% when we report full data in mid-2024. Turning to our second pivotal program, PVEK, we continue to advance our 802 study and look forward to reporting data from our PVEK/VEN/AZA triplet in frontline AML at ASH (Free ASH Whitepaper) in December. We also continued to monitor the NSCLC cohort with IMGC936 and advanced dose escalation with IMGN151, our second-generation ADC targeting FRα. We look forward to a strong finish to the year and a productive 2024 with the continued growth of ELAHERE, important new data for our programs, and geographic expansion in Europe and China."

RECENT PROGRESS

ELAHERE (mirvetuximab soravtansine-gynx)

Generated $105.2 million in ELAHERE net sales for the quarter ended September 30, 2023.
Obtained acceptance of the Marketing Authorization Application (MAA) by the European Medicines Agency (EMA) for ELAHERE in folate receptor alpha (FRα)-positive platinum-resistant ovarian cancer (PROC) to support approval and launch in Europe.
Submitted the supplemental Biologics License Application (sBLA) to the US Food and Drug Administration (FDA) to support conversion to full approval.
Our partner, Huadong Medicine, obtained acceptance of the New Drug Application (NDA) to the National Medical Products Administration (NMPA) of China for ELAHERE in FRα-positive PROC to support approval and launch.
Announced collaboration with Takeda Pharmaceutical Company Limited (Takeda) granting Takeda an exclusive license to develop and commercialize ELAHERE in Japan. The Company received an upfront payment of $23.21 million and is eligible to receive up to approximately $1352 million in regulatory and commercial milestone payments in addition to tiered royalties in the low double-digits to mid-twenties.
Clinical Pipeline and Research

Presented additional subset analyses in prior lines of therapy and prior exposure to PARP inhibitor (PARPi) therapy from MIRASOL in an oral session at the 24th Congress of the European Society of Gynaecological Oncology (ESGO) demonstrating clinical outcomes of safety and efficacy consistent with the overall MIRASOL study population.
Reported that PICCOLO, the ongoing single-arm Phase 2 trial of mirvetuximab in FRα-high, platinum-sensitive ovarian cancer (PSOC) has met the primary endpoint of objective response rate (ORR) based upon an interim assessment with no new safety signals identified. An ORR of at least 48% is expected when full data are reported in mid-2024.
Established a multi-target license and option agreement with ImmunoBiochem Corporation to develop next-generation ADCs.
Corporate

Appointed Lauren White as Senior Vice President and Chief Financial Officer, and Heather Adkins Huet, PhD, as Senior Vice President and Chief Scientific Officer.
ANTICIPATED UPCOMING EVENTS

Potential FDA approval of ELAHERE’s sBLA in H1 2024.
Report full data from the single-arm Phase 2 PICCOLO trial of mirvetuximab in FRα-high PSOC in mid-2024.
Potential EMA approval of ELAHERE in late 2024 to support launch in Europe.
Our partner, Huadong Medicine, is planning for NMPA approval of ELAHERE by the end of 2024 to support launch in China.
Report data from the 802 trial, evaluating the pivekimab sunirine (pivekimab) triplet with Venclexta (venetoclax) and Vidaza (azacitidine) in frontline acute myeloid leukemia (AML) at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2023.
Report top-line data from the pivotal frontline de novo cohort in the Phase 2 CADENZA trial of pivekimab in blastic plasmacytoid dendritic cell neoplasm (BPDCN) in 2024.
Provide an update on the IMGC936 non-small cell lung cancer (NSCLC) cohort following a prespecified interim analysis.
FINANCIAL RESULTS

Total revenues were $113.4 million for the quarter ended September 30, 2023, including $105.2 million of net product revenues from sales of ELAHERE, compared to $15.4 million in total revenues for the quarter ended September 30, 2022. The increase was driven by ELAHERE net sales, partially offset by $7.4 million of license fees recorded as revenue in the prior year period pursuant to the Company’s collaboration agreements with Eli Lilly and Company and Novartis Institutes for BioMedical Research, Inc.

Research and development expenses were $47.6 million for the quarter ended September 30, 2023 compared to $59.2 million for the quarter ended September 30, 2022. The decrease was primarily driven by ELAHERE supply costs expensed in the prior quarter versus capitalized in the current period and lower hiring expenses. Partially offsetting these decreases, salaries and benefits increased driven largely by the expansion of our medical affairs organization.

Selling, general and administrative expenses were $37.7 million for the quarter ended September 30, 2023 compared to $33.6 million for the quarter ended September 30, 2022. The increase was due primarily to greater expenses in support of the US launch of ELAHERE, including costs related to the addition of our commercial organization and sales and marketing activities.

Net income for the third quarter of 2023 was $30.7 million, or $0.10 per basic and diluted share, compared to a net loss of $77.8 million, or $0.31 per basic and diluted share, for the third quarter of 2022.

ImmunoGen had $605.5 million in cash and cash equivalents and $130.7 million in accounts receivable as of September 30, 2023, compared with $275.1 million in cash and cash equivalents and $12.6 million in accounts receivable as of December 31, 2022. Cash used in operations was $137.7 million for the first nine months of 2023 compared with cash used in operations of $169.6 million for the same period in 2022. Capital expenditures were $1.6 million and $1.1 million for the first nine months of 2023 and 2022, respectively.

FINANCIAL GUIDANCE

ImmunoGen’s full year financial guidance for 2023 remains unchanged; the Company continues to expect:

revenues, excluding product revenue from ELAHERE, between $45 million and $50 million; and
operating expenses between $350 million and $365 million.
The Company continues to expect that its existing cash and cash equivalents, together with anticipated future product and collaboration revenues, will fund operations for more than two years.

CONFERENCE CALL INFORMATION

ImmunoGen will hold a conference call today at 8:00 a.m. ET to discuss these results. To access the live call by phone, please register here. A dial-in and unique PIN will be provided to join the call. The call may also be accessed through the Investors and Media section of the Company’s website, www.immunogen.com. Following the call, a replay will be available at the same location.

ABOUT ELAHERE

ELAHERE (mirvetuximab soravtansine-gynx) is a first-in-class ADC comprising a folate receptor alpha-binding antibody, cleavable linker, and the maytansinoid payload DM4, a potent tubulin inhibitor designed to kill the targeted cancer cells.

ELAHERE is indicated for the treatment of adult patients with folate receptor-alpha (FRα) positive, platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer, who have received one to three prior systemic treatment regimens. Select patients for therapy based on an FDA-approved test.

This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

Eye problems are common with ELAHERE and can be severe. ELAHERE also can cause severe or life-threatening inflammation of the lungs that may lead to death and patients may develop nerve problems called peripheral neuropathy during treatment. Please see full Prescribing Information, including Boxed Warning, and Medication Guide for ELAHERE.

On November 2, 2023 Ichnos Sciences Inc., a global clinical-stage biotechnology company developing innovative multispecific immune cell engager antibodies in oncology, reported dosing of the first patient in the Phase 1 first-in-human clinical trial of ISB 2001, a BCMA x CD38 x CD3 TREAT trispecific antibody1, for the treatment of multiple myeloma (Press release, Ichnos Sciences, NOV 2, 2023, View Source [SID1234636765]). This milestone follows the company’s approval from the Human Research Ethics Committee (HREC) in Australia and IND clearance from the U.S. Food and Drug Administration (U.S. FDA). Additionally, the trispecific antibody received orphan drug designation (ODD) from the U.S. FDA in July, making it Ichnos’ third clinical-stage asset to receive the designation.

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ISB 2001 is the company’s first T-cell engaging trispecific antibody based on the company’s proprietary BEAT platform2,which enables the development of immune cell engagers. It features three proprietary Fab arms binding to CD3 on T cells, as well as BCMA and CD38 on multiple myeloma cells. By targeting two tumor-associated antigens, ISB 2001 exhibits heightened specificity in recognizing and binding to tumor cells. This avidity-based binding demonstrates increased tumor cell killing in vitro compared to teclistamab, alnuctamab, and EM-801 across variable levels of BCMA and CD38 expression.

Additionally, ISB 2001 exhibits higher potency in vitro when compared to the combination of daratumumab and teclistamab currently under clinical investigation, as demonstrated by data presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper)’s (AACR) (Free AACR Whitepaper) 2023 Annual Meeting.

"We are proud to advance our third oncology asset to clinical trials—a milestone made more significant because it is our first clinical-stage trispecific antibody," said Cyril Konto, M.D., president and CEO of Ichnos. "Our novel approach to targeting multiple molecules intends to enhance specificity, engage the immune system, and offer potential synergistic effects in our ongoing pursuit of groundbreaking cancer treatments."

"Not only does the advancement of ISB 2001 demonstrate our shared commitment to providing curative therapies that may extend and improve lives, but it’s also a testament to the operational excellence that enables us to run concurrent clinical programs in pursuit of our vital mission," Konto added.

This study is a first-in-human, Phase 1, open-label study that will evaluate safety and anti-myeloma activity of ISB 2001 in an estimated 80 participants with relapsed/refractory multiple myeloma (R/R MM) across multiple centers, including the U.S. and Australia. The multicenter study will be conducted in two parts: dose escalation and dose expansion.

"The unique triple targeting of BCMA, CD38, and CD3 showcases Ichnos’ scientific ingenuity underlying ISB 2001, offering a promising approach in the treatment of multiple myeloma," said Lida Pacaud, M.D., Chief Medical Officer of Ichnos. "We look forward to advancing our understanding of its safety and efficacy profile as we progress through this first-in-human study, driven by a commitment to pushing the boundaries of what is possible in the field of hemato-oncology."

More information about the trial, including investigational site locations, site-specific contacts and eligibility criteria for participants, is available on ClinicalTrials.gov.

With the start of this first-in-human clinical study of ISB 2001, Ichnos now has three clinical-stage oncology assets under investigation for the treatment of relapsed/refractory multiple myeloma and has initiated one new Phase 1 clinical study per year for three consecutive years.

Additional details about ISB 2001 and Ichnos’ pipeline of oncology assets can be found at ichnos.com/pipeline/.

On November 2, 2023 Helix BioPharma Corp. (TSX: "HBP") ("Helix" or the "Company"), a clinical-stage biopharmaceutical company developing unique therapies in the field of immuno-oncology, based on its proprietary technological platform DOS47, reported that it has closed the first tranche of private placement financing for gross proceeds of CAD $2,998,000 from the issuance of 16,655,557 common shares at a price of $0.18 per common share (Press release, Helix BioPharma, NOV 2, 2023, View Source [SID1234636764]).

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On November 2, 2023 Heidelberg Pharma AG (FSE: HPHA) reported that it will present new findings of its clinical Phase I/IIa study with the proprietary ADC candidate HDP-101 at the 65th Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) (Press release, Heidelberg Pharma, NOV 2, 2023, View Source [SID1234636763]).

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Dr. András Strassz, Chief Medical Officer at Heidelberg Pharma, commented: "We are very pleased with the progress of patient enrollment in our clinical trial with HDP-101. So far, HDP-101 has shown to be safe and well tolerated, and we will continue dose escalation in the fifth patient cohort (100 µg/kg) as planned."

Poster title: HDP-101, an Anti-BCMA Antibody-Drug Conjugate with a Novel Payload Amanitin in Patients with Relapsed Multiple Myeloma, Initial Findings of the First in Human Study

Presentation details

Abstract: #3334

Session: 652. Multiple Myeloma: Clinical and Epidemiological: Poster II

Time and location: Sunday, 10th December 2023, 6:00 pm – 8:00 pm PST, Hall G-H

Dr. Strassz will present the poster showing safety data and preliminary findings from four patient cohorts of the ongoing open-label, multicenter Phase I/IIa trial evaluating HDP-101 in multiple myeloma. He will also be available to answer questions.

HDP-101 is a BCMA antibody-Amanitin conjugate for the treatment of relapsed or refractory multiple myeloma, a bone marrow cancer with high unmet medical need. The first part of the trial is a Phase I dose escalation study to determine an optimal and safe dose of HDP-101 for the Phase II part of the study.

The first four patient cohorts and dose levels of the clinical study have been completed and proved to be safe and well tolerated. Currently, the trial is enrolling patients in the fifth cohort.

On November 2, 2023 Geron Corporation (Nasdaq: GERN), a late-stage clinical biopharmaceutical company developing a first-in-class telomerase inhibitor, imetelstat, to treat hematologic malignancies, reported business highlights and financial results for the third quarter of 2023 (Press release, Geron, NOV 2, 2023, View Source [SID1234636762]).

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"This quarter, we continued to make important progress and build momentum along our planned path to develop and commercialize imetelstat, which is now the first telomerase inhibitor to be under review by both the FDA and EMA for potential regulatory approval," said John A. Scarlett, M.D., Chairman and Chief Executive Officer. "We see lower risk MDS as a very compelling commercial opportunity, with few durable treatment options and significant need for large patient segments such as patients without sideroblasts (RS-) and those with high transfusion burden. We believe, if approved, that imetelstat could play a meaningful role in this treatment landscape."

Dr. Scarlett continued, "We believe that we are in a strong position to execute upon a potential launch in the U.S., bolstered by an experienced leadership team and with our talented commercial and medical affairs leadership teams fully onboarded. Additionally, with approximately $382 million on the balance sheet as of the end of the quarter, and expected available resources, we have the financial resources to fund a potential successful launch in the U.S. and our planned operations through the end of Q3 2025."

Business Highlights

Received acceptance from the U.S. Food & Drug Administration (FDA) of the New Drug Application (NDA) submitted for imetelstat for the treatment of transfusion-dependent anemia in adult patients with low- to intermediate-1 risk myelodysplastic syndromes (MDS), or lower risk MDS, who have failed to respond, or have lost response to, or are ineligible for erythropoiesis-stimulating agents (ESAs). The FDA assigned a Prescription Drug User Fee Act (PDUFA) action date of June 16, 2024. In addition, the FDA informed the Company that it is currently planning to hold an advisory committee meeting as part of the NDA review.
Submitted the Marketing Authorization Application (MAA) for imetelstat in the same lower risk MDS indication as in the NDA and received validation from the European Medicines Agency (EMA) that the application is under regulatory review by the European Committee for Medicinal Products for Human Use (CHMP) under the centralized procedure. Review of the MAA is expected to be completed by the end of 2024.
Presented encore data and analyses from the IMerge Phase 3 clinical trial evaluating imetelstat in patients with lower risk MDS at the Society of Hematologic Oncology Annual Meeting. New analyses from this trial are also planned at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, on which a separate press release will be issued.
Escalated to the second dose cohort in the Phase 1 ImproveMF study evaluating imetelstat as a combination therapy with ruxolitinib in patients with frontline myelofibrosis (MF) following a unanimous decision by the study’s Safety Evaluation Team (SET), who reviewed the first cohort (3 patients) data and identified no dose-limiting toxicities.
Appointed Michelle Robertson as Executive Vice President, Chief Financial Officer and Treasurer, following Olivia Bloom’s retirement. Ms. Robertson brings to Geron over 30 years of financial and commercial operations experience. Prior to joining, she served as the Chief Financial Officer and Treasurer of Editas Medicine, a CRISPR genome editing company, where she raised $500M in capital over three years to support the company’s research transition into late-stage clinical development. Before that, she served as Chief Financial Officer of Momenta Pharmaceuticals, Inc. from 2018 until 2020, leading the finance team through a strategic restructure, before its acquisition by Johnson & Johnson. Prior to joining Momenta, Ms. Robertson held multiple finance and commercial operations roles of increasing responsibility.
Third Quarter 2023 Financial Results

As of September 30, 2023, the Company had $381.9 million in cash, cash equivalents, and marketable securities. In the third quarter of 2023, the Company received $28.3 million upon the cash exercise of outstanding warrants. As of September 30, 2023, warrants remaining outstanding are exercisable for potential future proceeds of $3.2 million. Based on the Company’s current operating plans and expectations regarding the timing of regulatory approval and commercialization of imetelstat in the United States (U.S.) in the first half of 2024, Geron projects that its existing financial resources, together with projected revenues from U.S. sales of imetelstat, proceeds from the exercise of outstanding warrants, and funding under the Company’s loan facility, will be sufficient to fund its projected operating requirements through the end of Q3 2025.

Revenues for the three and nine months ended September 30, 2023, were $164,000 and $214,000, respectively, compared to $297,000 and $493,000 for the comparable 2022 periods. Revenues in both years primarily reflect estimated royalties from sales of cell-based research products from the Company’s divested stem cell assets.

Total operating expenses for the three and nine months ended September 30, 2023, were $47.8 million and $139.9 million, respectively, compared to $40.2 million and $97.1 million for the comparable 2022 periods.

Research and development expenses for the three and nine months ended September 30, 2023, were $29.4 million and $92.1 million, respectively, compared to $24.6 million and $67.3 million for the comparable 2022 periods. The increase in research and development expenses for the three and nine months ended September 30, 2023, compared to the same periods in 2022 primarily reflects higher clinical trial costs related to supporting IMerge Phase 3 and IMpactMF, increased personnel-related expenses for additional headcount, higher consulting costs to support regulatory submissions and greater imetelstat manufacturing costs in preparation for potential commercialization in lower risk MDS.

General and administrative expenses for the three and nine months ended September 30, 2023, were $18.4 million and $47.7 million, respectively, compared to $15.6 million and $29.8 million for the comparable 2022 periods. The increase in general and administrative expenses for the three and nine months ended September 30, 2023, compared to the same periods in 2022, primarily reflects new costs for commercial preparatory activities and higher personnel-related expenses for additional headcount.

Interest income was $5.0 million and $13.6 million for the three and nine months ended September 30, 2023, respectively, compared to $852,000 and $1.3 million for the same periods in 2022. The increase in interest income for the three and nine months ended September 30, 2023, compared to the same periods in 2022, primarily reflects higher yields on the Company’s marketable securities as a result of rising interest rates, as well as a larger investment portfolio with the cash proceeds from the January 2023 public offering and warrant exercises in the first nine months of 2023.

Interest expense was $2.0 million and $6.0 million for the three and nine months ended September 30, 2023, respectively, compared to $1.8 million and $4.9 million for the same periods in 2022. The increase in interest expense for the three and nine months ended September 30, 2023, compared to the same periods in 2022, primarily reflects higher interest rates. Currently, the Company has $50.0 million in principal debt outstanding.

Projected 2023 Financial Guidance

For fiscal year 2023, under generally accepted accounting principles (GAAP), the Company continues to expect total expenses in the range of approximately $200 million to $210 million, which includes non-cash items such as stock-based compensation expense, amortization of debt discounts and issuance costs, and depreciation and amortization.

The fiscal year 2023 financial guidance reflects costs to support regulatory submissions with the FDA and EMA in 2023; continued support of ongoing clinical trials, IMerge Phase 3, IMpactMF, ImproveMF, and the investigator-led Impress trial, as well as preclinical studies in lymphoid malignancies and discovery research for a next generation telomerase inhibitor; manufacturing of commercial inventory of imetelstat; preparations for potential U.S. commercial launch of imetelstat in lower risk MDS; projected increases in headcount and interest payments on outstanding debt.

As of September 30, 2023, the Company had 137 employees. The Company plans to grow to a total of approximately 160 employees by year-end 2023.

Conference Call

Geron will host a conference call at 9:00 am ET on Thursday, November 2, 2023 to discuss business updates and third quarter 2023 financial results.

A live webcast of the conference call and related presentation will be available on the Company’s website at www.geron.com/investors/events. An archive of the webcast will be available on the Company’s website for 30 days.

Participants may access the webcast by registering online using the following link, View Source

About Imetelstat

Imetelstat is a novel, first-in-class investigational telomerase inhibitor exclusively owned by Geron and being developed in hematologic malignancies. Data from non-clinical studies and clinical trials of imetelstat provide strong evidence that imetelstat targets telomerase to inhibit the uncontrolled proliferation of malignant stem and progenitor cells in myeloid hematologic malignancies, resulting in malignant cell apoptosis and suggesting potential disease-modifying activity. Imetelstat has been granted Fast Track designation by the U.S. Food and Drug Administration for both the treatment of adult patients with transfusion dependent anemia due to Low or Intermediate-1 risk MDS that is not associated with del(5q) who are refractory or resistant to an erythropoiesis stimulating agent, and for adult patients with Intermediate-2 or High-risk myelofibrosis (MF) whose disease has relapsed after or is refractory to janus associated kinase (JAK) inhibitor treatment. Imetelstat is currently not approved by any regulatory authority.

About IMerge Phase 3

The Phase 3 portion of the IMerge Phase 2/3 study is a double-blind, 2:1 randomized, placebo-controlled clinical trial to evaluate imetelstat in patients with IPSS Low or Intermediate-1 risk (lower risk) transfusion dependent MDS who were relapsed after, refractory to, or ineligible for, erythropoiesis stimulating agent (ESA) treatment, had not received prior treatment with either a HMA or lenalidomide and were non-del(5q). To be eligible for IMerge Phase 3, patients were required to be transfusion dependent, defined as requiring at least four units of packed red blood cells (RBCs), over an eight-week period during the 16 weeks prior to entry into the trial. The primary efficacy endpoint of IMerge Phase 3 is the rate of red blood cell transfusion independence (RBC-TI) lasting at least eight weeks, defined as the proportion of patients without any RBC transfusion for at least eight consecutive weeks since entry to the trial (8-week TI). Key secondary endpoints include the rate of RBC-TI lasting at least 24 weeks (24-week TI), the duration of TI and the rate of hematologic improvement erythroid (HI-E), which is defined under 2006 IWG criteria as a rise in hemoglobin of at least 1.5 g/dL above the pretreatment level for at least eight weeks or a reduction of at least four units of RBC transfusions over eight weeks compared with the prior RBC transfusion burden. A total of 178 patients were enrolled in IMerge Phase 3 across North America, Europe, Middle East and Asia.

About ImproveMF

IMproveMF is a single arm, open label, two-part Phase 1 study to evaluate the safety, pharmacokinetics, pharmacodynamics and clinical activity of imetelstat in combination with ruxolitinib as a frontline treatment in patients with Intermediate-2 or High-risk MF (frontline MF). In both parts, patients will receive ruxolitinib followed by imetelstat, a dosing schedule that showed synergistic and additive effects of the two agents in preclinical experiments. Part 1 will enroll up to 20 frontline MF patients who, at the time of enrollment, have received an optimized dose of ruxolitinib, to which imetelstat treatment will be added at increasing dose levels based on safety and tolerability. The primary purpose of Part 1 is to identify a safe dose for treating frontline MF patients with a combination of imetelstat and ruxolitinib. If a safe dose is identified in Part 1, participants in Part 2 will be JAK inhibitor naïve and will receive treatment with ruxolitinib after screening and enrollment at a starting dose based on standard-of-care or local prescribing information. Treatment with single-agent ruxolitinib will continue for at least 12 weeks, including four consecutive weeks at a stable dose prior to the addition of imetelstat. Part 2 is designed to confirm the safety profile of imetelstat in combination with ruxolitinib and to evaluate for preliminary clinical activity of the combination.