On November 2, 2023 Cidara Therapeutics, Inc. (Nasdaq: CDTX), a biotechnology company focused on developing targeted therapies designed to save lives and improve the standard of care for patients facing serious diseases, reported financial results for the third quarter ended September 30, 2023 and provided an update on its corporate activities and product pipeline (Press release, Cidara Therapeutics, NOV 2, 2023, View Source [SID1234637013]).

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"We are proud of the recent advancements of both the REZZAYO program as well as our Cloudbreak drug-Fc conjugate (DFC) platform," said Jeffrey Stein, Ph.D., president and chief executive officer of Cidara. "The US commercial launch of REZZAYO by our partner Melinta as well as the receipt by our ex-US/ex-Japan partner Mundipharma of the positive opinion from the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) demonstrates continued execution of our rezafungin commercial strategy. In addition, we believe the receipt of an Election to Proceed Notice from Janssen for CD388 for the universal prevention of influenza A and B represents a key validation of the significant potential of this clinical program. Finally, the new data on our oncology DFC programs disclosed at our recently held R&D Day indicate that the advantages of the CD388 program for influenza are translating to our Cloudbreak oncology DFC programs."

Dr. Stein continued, "our oncology clinical development strategy is being led by Nicole Davarpanah, M.D., J.D., who recently joined Cidara as Senior Vice President of Translational Research & Development. Nicole is a practicing oncology physician and joins us from Genentech/Roche where she has had substantial cancer-focused product development experience. Looking ahead, we look forward to filing an Investigational New Drug Application (IND) in mid-2024 for CBO421, a potential best-in-class inhibitor of CD73."

Recent Corporate Highlights

Received Positive CHMP Opinion for Rezafungin for the Treatment of Invasive Candidiasis in Adults: In October 2023, Cidara and Mundipharma Medical Company (Mundipharma) announced that the EMA’s CHMP has adopted a positive opinion for rezafungin (rezafungin acetate) for the treatment of invasive candidiasis in adults. The European Commission (EC) will review the CHMP recommendation and is expected to make a final decision on approval by the end of 2023. In October 2023, Melinta Therapeutics, LLC (Melinta) announced receipt from the Centers for Medicare & Medicaid Services (CMS) of both a product-specific J-Code and a new technology add-on payment (NTAP) for REZZAYO. Outside the U.S. and Japan, Cidara’s development and commercial partner is Mundipharma. Although we have shifted our research focus to our proprietary Cloudbreak platform, we continue to execute on the ongoing ReSPECT Phase 3 pivotal clinical trial for the prevention of invasive fungal infections in adult allogeneic blood and marrow transplant recipients. A significant portion of our future royalties and milestones to be received under both Melinta and Mundipharma licensing agreements are tied to the successful completion of the ReSPECT Phase 3 trial.
Received Election to Proceed Notice from Janssen Pharmaceuticals, Inc. for CD388, which is being developed for the universal prevention of influenza A and B: In September 2023, Janssen Pharmaceuticals, Inc. (Janssen), one of the Janssen Pharmaceutical Companies of Johnson & Johnson, delivered to Cidara its Election to Proceed Notice for CD388. Responsibility for future development, manufacturing and commercialization activities of CD388 will be assumed by Janssen, which intends to transfer its rights and obligations under the agreement to another entity. Cidara received a $7.0 million milestone payment from Janssen for the election to proceed for CD388 and other influenza DFCs. Under the collaboration agreement, Cidara is eligible to potentially receive an additional $685.0 million in development, regulatory and commercial milestones, plus tiered royalties on worldwide sales.
Hosted R&D Day on Cloudbreak development pipeline: In September 2023, Cidara hosted an R&D Day during which the company provided an update on Phase 2a data for CD388, as well as a detailed overview of the potential role of DFCs in oncology. Cidara provided an update on its Cloudbreak pipeline using its oncology DFCs, as well as insight into clinical development considerations for DFCs, focusing on CD73, chemokine receptors, and multispecific DFCs targeting solid tumors.
Presented at IDWeek 2023: In September 2023, Cidara delivered an oral presentation and two poster presentations highlighting the safety and efficacy of CD388.
Presented at the 25th Annual H.C. Wainwright Global Investment Conference: In September 2023, Dr. Stein participated in the 25th Annual H.C. Wainwright Global Investment Conference.
Appointed Dr. Davarpanah as Senior Vice President of Translational Research & Development: In August 2023, Cidara appointed Dr. Davarpanah to lead the company’s oncology efforts, focusing on the strategy for preclinical, translational, and early clinical development activities to identify and advance drug candidates through human proof-of-concept studies. She has a decade of experience in oncology drug development and joined Cidara from Genentech/Roche where Dr. Davarpanah most recently served as Clinical and Translational Lead in Oncology.
Third Quarter 2023 Financial Results

Revenue totaled $12.7 million and $46.3 million for the three and nine months ended September 30, 2023, respectively, compared to $40.7 million and $54.1 million for the same periods in 2022, respectively. Revenue for the three and nine months ended September 30, 2023 related to the achievement of milestones and ongoing research and development and clinical supply services provided to Mundipharma, Janssen and Melinta, royalty revenue recognized following initiation of the commercial launch of REZZAYO in the U.S. on July 31, 2023, as well as product revenue related to shipment of REZZAYO naked vials to Melinta. Revenue for the three and nine months ended September 30, 2022 related to the achievement of milestones and ongoing research and development and clinical supply services provided to Mundipharma, Janssen and Melinta, and revenue recognized upon transfer of an intellectual property license to Melinta in August 2022.
Cash and cash equivalents totaled $48.7 million as of September 30, 2023, compared with $32.7 million as of December 31, 2022.
Research and development expenses were $17.3 million and $53.2 million for the three and nine months ended September 30, 2023, respectively, compared to $20.0 million and $55.5 million for the same periods in 2022, respectively. The research and development expenses for all periods primarily relate to clinical expenses associated with the rezafungin clinical trials and certain drug manufacturing costs before FDA approval, as well as clinical expenses and drug manufacturing costs associated with the Cloudbreak platform, including CD388 for which Cidara is fully reimbursed under the Janssen Collaboration Agreement.
General and administrative expenses were $3.6 million and $11.2 million for the three and nine months ended September 30, 2023, respectively, compared to $5.8 million and $15.1 million for the same periods in 2022, respectively. The general and administrative expenses for all periods primarily relate to consulting, personnel and legal costs.
Net loss for the three months ended September 30, 2023 was $8.2 million, compared to a net income of $15.0 million for the same period in 2022. For the nine months ended September 30, 2023 and 2022, net loss was $17.3 million and $16.4 million, respectively.
During the three months ended September 30, 2023, Cidara did not sell shares of common stock pursuant to its at-the-market (ATM) sales agreement.
As of September 30, 2023, Cidara had 90,415,944 shares of common stock outstanding and 2,104,472 shares of Series X Convertible Preferred Stock outstanding, which are convertible into 21,044,720 shares of common stock.

On November 2, 2023 IDRx, Inc., a clinical-stage biopharmaceutical company dedicated to transforming cancer treatment with purpose-built precision therapies, reported preliminary clinical data from the dose escalation portion of the company’s ongoing Phase 1 StrateGIST study of IDRX-42 in patients with metastatic and/or unresectable gastrointestinal stromal tumors (GIST) who have previously received one or more prior lines of tyrosine kinase inhibitor (TKI) therapy (Press release, IDRx, NOV 2, 2023, View Source [SID1234636845]). IDRX-42 is a highly potent, multi-mutation-selective inhibitor of KIT targeting all major classes of primary drivers and resistance mutations in patients with KIT-mutant GIST (including mutations in exons 9, 11, 13 and 17).

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These early data, which demonstrate promising antitumor activity and a favorable safety profile of IDRX-42 in a heavily pre-treated group of patients with advanced GIST after failure of imatinib and other lines of TKI therapies, will be presented by Professor Patrick Schöffski, MD, MPH, head of the Department of General Medical Oncology at the University Hospitals Leuven, in a poster at the Connective Tissue Oncology Society (CTOS) 2023 Annual Meeting, being held November 1‑4, in Dublin, Ireland.

"Advanced GIST generally carries a poor prognosis after failure of imatinib and new therapies such as IDRX-42, that target key primary activating drivers as well as secondary resistance mutations in KIT, are urgently needed," said Prof. Schöffski. "These preliminary data demonstrate exciting and clinically meaningful antitumor activity in a highly refractory and difficult to treat GIST patient population and warrant the investigation of IDRX-42 in earlier lines of therapy."

"We are highly encouraged by these initial data of IDRX-42, supporting a potentially best-in-class profile to improve the outcomes of patients with GIST in all lines of therapy, which we believe remains one of targeted oncology’s great unmet medical needs," said Ben Auspitz, co-founder and chief executive officer of IDRx. "Importantly, these preliminary data will help guide our discussions with physicians regarding development strategies for IDRX-42 in patients with GIST in early lines of therapy to prevent the emergence of genomically-driven resistance mutations. Early suppression of such resistance mutations in KIT is likely to benefit patients with GIST, versus trying to treat clinically heterogeneous disease in any individual with multiple resistant clones."

Presentation Highlights:

Initial data (October 5, 2023 cut-off date) from the dose escalation portion of the ongoing Phase 1 StrateGIST study include 33 patients (all with KIT-mutant GIST) who received IDRX-42 across doses ranging from 120 mg once daily (QD) to 400 mg twice daily (BID). Patients were heavily pretreated, with a median of 4 prior lines of therapy. 28 patients are evaluable for objective response. Median duration on treatment was 16 weeks and continuing, with 70% (23/33) of patients remaining on treatment at time of data cutoff. Maximum tolerated dose (MTD) has not yet been reached.
Confirmed partial responses (PRs) by modified RECIST have been demonstrated to date in four patients (at 120 mg QD, 400 mg QD, and 600 mg QD [n=2]).
Although an MTD has not yet been reached, a 68% (19/28 patients) clinical benefit rate (confirmed complete response/PR or stable disease ≥16 weeks) was observed across the initial doses studied to date. 4/28 patients are on study for <16 weeks and continuing on study drug.
Tumor shrinkage and confirmed partial responses were observed across all major classes of KIT mutational variants, including both primary driver mutations in exons 9 and 11, and secondary resistance mutations in exons 13 and 17. Additionally, notable reductions in mutant allele fraction were observed in circulating tumor DNA across these classes of KIT variants.
Preliminary pharmacokinetic analyses demonstrate dose-dependent increases in exposure, with a long half-life (approximately 125 hours), and a 5 to 8-fold accumulation between single-dose and steady-state at Cycle 2 (Day 28).
IDRX-42 exhibited a favorable safety and tolerability profile with most adverse events being mild (Grade 1) in severity. Treatment related adverse events included diarrhea (70%), nausea (52%), and vomiting (27%). No safety-related treatment discontinuations have been observed to date. Two events coded as dose-limiting toxicities were reported (Grade 3 syncope at 600 mg QD, and Grade 3 vomiting at 400 mg BID); both patients chose to continue on study with dose reduction and are continuing on treatment for 5 months and 2 months, respectively.
Four dose levels (120, 240, 400 and 600 mg QD) have been cleared to date, and dose escalation continues with accrual to the 800 mg (400 mg BID) cohort ongoing.
Presentation Details:

Title: Phase 1 Study of IDRX-42 in Patients with Advanced Gastrointestinal Stromal Tumors Resistant to Prior Systemic Therapy: Early Results
Presenter: Patrick Schöffski, MD, MPH
Abstract Number: P 26
Presentation Type: Poster
Session: CTOS Poster Reception 5:30-6:30 p.m. GMT, Thursday, November 2nd
Location: The Convention Center, Dublin, Ireland

About GIST

Gastrointestinal stromal tumors (GIST) are the most common subtype of soft tissue sarcoma. Approximately 80% of cases arise from gain of function mutations in the KIT receptor tyrosine kinase, driving the malignancy through constitutive activation of aberrant signaling. Resistance mutations in KIT emerge in ~90% of patients treated with imatinib, the current standard of care for GIST. In unresectable or metastatic GIST, clinical benefits from existing treatments can vary by mutation type.

About the StrateGIST Study

StrateGIST1 is an ongoing, open-label, first-in-human Phase 1/1b study designed to evaluate the safety, tolerability, pharmacokinetics, and preliminary antitumor activity of IDRX-42 in patients with metastatic and/or surgically unresectable GIST after failure of imatinib and other approved drugs. The study is currently enrolling patients with documented pathogenic mutation in KIT or any platelet-derived growth factor receptor alpha (PDGFRA) mutation (other than PDGFRA exon 18) at sites in the U.S., Belgium, Germany, and Spain. The Phase 1b portion will include expanded exploratory cohorts based on defined lines of prior TKI therapy.

About IDRX-42

IDRX-42 is a potent, oral, highly selective KIT inhibitor targeting all major categories of activating and resistance mutations in patients with KIT-mutant GIST (including variants in exons 9, 11, 13 and 17). In preclinical studies, IDRX-42 demonstrated superior antitumor activity compared to imatinib, the current first-line of therapy, in GIST human xenograft models expressing mutations in KIT exons 9 and 11. In xenograft models expressing secondary resistance mutations in KIT exon 13 or 17, IDRX-42 treatment resulted in potent and dose-dependent antitumor activity superior to the second-line standard of care agent, sunitinib. IDRX-42 is current being evaluated in a first-in-human Phase 1/1b study.

On November 2, 2023 Incyte (Nasdaq:INCY) reported that more than 40 abstracts highlighting data from eight of its hematology and oncology products will be presented at the upcoming 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting 2023 (ASH 2023), held December 9-12, 2023, in San Diego and virtually (Press release, Incyte, NOV 2, 2023, View Source [SID1234636844]).

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"We have continued to make significant progress in advancing our hematology and oncology pipeline with the goal to deliver better medicines for a range of diseases that have limited treatment options, including myeloproliferative neoplasms (MPNs) and chronic graft-versus-host disease (GVHD)," said Pablo J. Cagnoni, M.D., President and Head of Research and Development, Incyte. "We are excited to showcase the depth of our portfolio and clinical progress at this year’s ASH (Free ASH Whitepaper) congress. In particular, we look forward to the presentation of the axatilimab AGAVE-201 trial results in patients with chronic GVHD at the Plenary Scientific Session, as well as the numerous oral and poster presentations including new data for our mutant CALR, BET, ALK2 and CK0804 programs in MPNs. Additionally, we are proud that the first presentation of data for INCB160058, our new potentially disease modifying JAK2V617F therapy for patients with MPNs, will be at this year’s meeting."

Select key abstract presentations from Incyte-developed and partnered programs include:

Plenary Scientific Session

Axatilimab

Safety and Efficacy of Axatilimab at 3 Different Doses in Patients with Chronic Graft-Versus-Host Disease (AGAVE-201)1 (Abstract #1. Plenary Scientific Session. Sunday, December 10, 5:00 p.m. – 7:00 p.m. ET)

Oral Presentations

Ruxolitinib (MPN)

A Real-World Evaluation of Risk Factors for Disease Progression in Patients with Polycythemia Vera (PV) Enrolled in REVEAL (Abstract #385. Session: 906. Outcomes Research – Myeloid Malignancies: Risk Factors and Health Disparities. Saturday, December 9, 7:00 p.m. ET)

Phase 1/2 Study of the Activin Receptor-Like Kinase-2 Inhibitor Zilurgisertib (INCB000928, LIMBER-104) as Monotherapy or with Ruxolitinib in Patients with Anemia Due to Myelofibrosis (Abstract #624. Session: 634. Myeloproliferative Syndromes: Clinical and Epidemiological: Charting The Future Of MPN Therapies. Sunday, December 10, 8:45 p.m. ET)

Bromodomain and Extra-Terminal (BET) Inhibitor INCB057643 (LIMBER-103) in Patients with Relapsed or Refractory Myelofibrosis (R/R MF) and Other Advanced Myeloid Neoplasms: A Phase 1 Study (Abstract #750. Session: 634. Myeloproliferative Syndromes: Clinical and Epidemiological: Treatment and Outcomes in MPNs. Monday, December 11, 2:45 p.m. ET)

Ruxolitinib (GVHD)

Ruxolitinib in Patients with Chronic Graft-Versus-Host Disease: Three-Year Final Analysis of Efficacy and Safety of the Phase 3 REACH3 Study2 (Abstract #654. Session: 722. Allogeneic Transplantation: Acute and Chronic GVHD, Immune Reconstitution: Innovative Approaches to GVHD Prevention and Treatment. Sunday, December 10, 8:45 p.m. ET)

Tafasitamab

Tafasitamab for the Treatment of Relapsed/Refractory (R/R) Diffuse Large B-cell Lymphoma (DLBCL) in the U.S. Real-World Setting (Abstract #265. Session: 905. Outcomes Research – Lymphoid Malignancies: Outcomes Research in Lymphoma/CLL: Biomarkers, Dosing Strategies, and Big-Data. Saturday, December 9, 5:00 p.m. ET)

Itacitinib

Itacitinib for the Prevention of Immune Effector Cell Therapy-Associated Cytokine Release Syndrome: Results from the Phase 2 INCB 39110-211 Placebo-Controlled, Randomized Cohort (Abstract #356. Session: 705. Cellular Immunotherapies: Late Phase and Commercially Available Therapies: Prediction and Management of CAR-T Cell Related Toxicity. Saturday, December 9, 7:15 p.m. ET)

INCB160058

Preclinical Evaluation of INCB160058 – A Novel and Potentially Disease-Modifying Therapy for JAK2V617F Mutant Myeloproliferative Neoplasms (Abstract #860. Session: 631. Myeloproliferative Syndromes and Chronic Myeloid Leukemia: Basic and Translational: Lineage Tracing and Novel Target Discovery. Monday, December 11, 6:00 p.m. ET)

Poster Presentations

Ruxolitinib (MPN)

Effect of New or Worsening Anemia on Clinical Outcomes in 2,233 Patients with Myelofibrosis (MF) Treated with Ruxolitinib in the Expanded-Access JUMP Study (Abstract #5174. Session: 906. Outcomes Research—Myeloid Malignancies: Poster III. Monday, December 11, 9:00 p.m. – 11:00 p.m. ET)

Ruxolitinib Treatment in Polycythemia Vera Results in Reduction in JAK2 Allele Burden in Addition to Improvement in Hematocrit Control and Symptom Burden (Abstract #4553. Session: 634. Myeloproliferative Syndromes: Clinical and Epidemiological: Poster III. Monday, December 11, 9:00 p.m. – 11:00 p.m. ET)

High Rate of Disease Progression in Patients with Low-Risk Myelofibrosis (MF) Enrolled in the Prospective, Real-World, MOST Study Abstract #3803. Session: 906. Outcomes Research—Myeloid Malignancies: Poster II. Sunday, December 10, 9:00 p.m. – 11:00 p.m. ET)

Progression to Myelofibrosis in Patients with Essential Thrombocythemia: A Real-World Analysis from the Prospective MOST Study (Abstract #2433. Session: 906. Outcomes Research—Myeloid Malignancies: Poster I. Saturday, December 9, 8:30 p.m. – 10:30 p.m. ET)

Clinical and Disease Characteristics of Patients With Myelofibrosis and Essential Thrombocythemia that Harbor a Calreticulin (CALR) Gene Mutation: Subanalysis of the MOST Study (Abstract #3812. Session: 906. Outcomes Research—Myeloid Malignancies: Poster II. Sunday, December 10, 9:00 p.m. – 11:00 p.m. ET)

Comparison of the Enzymatic and Cellular Profiles of Clinical JAK2 Inhibitors for the Treatment of Myelofibrosis (Abstract #4532. Session: 631. Myeloproliferative Syndromes and Chronic Myeloid Leukemia: Basic and Translational: Poster III. Monday, December 11, 9:00 p.m. – 11:00 p.m. ET)

ALK2 and JAK2 Inhibition for Improved Treatment of Anemia in Myelofibrosis Patients: Preclinical Profile of an ALK2 Inhibitor Zilurgisertib in Combination with Ruxolitinib (Abstract #1789. Session: 631. Myeloproliferative Syndromes and Chronic Myeloid Leukemia: Basic and Translational: Poster I. Saturday, December 9, 8:30 p.m. – 10:30 p.m. ET)

The Association between Blood Cell Counts and Thrombotic Events in Japanese Patients with Polycythemia Vera: A Retrospective Database Study2 (Abstract #3191. Session: 634. Myeloproliferative Syndromes: Clinical and Epidemiological: Poster II. Sunday, December 10, 9:00 p.m. – 11:00 p.m. ET)

Tafasitamab

Real-World Use of Tafasitamab (tafa) for Relapsed or Refractory (R/R) Diffuse Large B-cell Lymphoma (DLBCL) Among Racial and Ethnic Minorities in the United States (Abstract #2415. Session: 905. Outcomes Research – Lymphoid Malignancies: Poster I. Saturday, December 9, 8:30 – 10:30 p.m. ET)

Tafasitamab in Combination with a CD20xCD3 Bispecific T-cell Engager Significantly Prolongs Survival in Preclinical Lymphoma Models3 (Abstract #2813. Session: 605. Molecular Pharmacology and Drug Resistance: Lymphoid Neoplasms: Poster II. Sunday, December 10, 9:00 – 11:00 p.m. ET)

Pemigatinib

Deep and Durable Cytogenetic and Molecular Responses with Pemigatinib in Myeloid/Lymphoid Neoplasms with Fibroblast Growth Factor Receptor 1 Rearrangement: The FIGHT-203 Study (Abstract #4551. Session: 634. Myeloproliferative Syndromes: Clinical and Epidemiological: Poster III. Monday, December 11, 9:00 p.m. – 11:00 p.m. ET)

Ponatinib

Long-term Results From the OPTIC Trial: A Dose-Optimization Study of 3 Starting Doses of Ponatinib4 (Abstract #3164. Session: 632. Chronic Myeloid Leukemia: Clinical and Epidemiological: Poster II. Sunday, December 10, 9:00 – 11:00 p.m. ET)

Ponatinib Versus Imatinib in Patients with Newly Diagnosed Ph+ ALL: Subgroup Analysis of the Phase 3 PhALLCON Study4 (Abstract #2871. 614. Acute Lymphoblastic Leukemias: Therapies, Excluding Transplantation and Cellular Immunotherapies: Poster II. Sunday, December 10, 9:00 – 11:00 p.m. ET)

Itacitinib

Janus Kinase (JAK) 1 Inhibition Results in Significant Changes in Serum Proteins and Peripheral T-Cell Populations that Correlated with Clinical Scores in Chronic Graft-Versus-Host Disease (GVHD) Patients (an Analysis from GRAVITAS-309) (Abstract #2197. Session: 722. Allogeneic Transplantation: Acute and Chronic GVHD, Immune Reconstitution: Poster I. Saturday, December 9, 8:30 – 10:30 p.m. ET)

Axatilimab

Axatilimab Ameliorates Inflammation and Fibrosis by Targeting the Macrophages in a Preclinical Model of Chronic GVHD (Abstract #2540. Session: 201. Granulocytes, Monocytes, and Macrophages: Poster II. Sunday, December 10, 9:00 – 11:00 p.m. ET)

CK0804

A Phase 1b, Open-Label Study of Add on Therapy with CK0804 in Participants with Myelofibrosis and Suboptimal Response to Ruxolitinib5 (Abstract #1813. Session: 634. Myeloproliferative Syndromes: Clinical and Epidemiological: Poster I. Saturday, December 9, 8:30 – 10:30 p.m. ET)

More information regarding the congress is available on the ASH (Free ASH Whitepaper) website: View Source This in-person event will be broadcast virtually and access to the meeting’s virtual platform is included with registration.

Conference Call and Webcast
Incyte will host an in-person analyst and investor event on Monday, December 11, 2023, from 12:00-1:30 p.m. PT (3:00–4:30 p.m. ET) to discuss the key data presentations at ASH (Free ASH Whitepaper). The event will be webcasted and can be accessed via the Events and Presentations tab of the Investor section of Incyte.com and it will be available for replay for 30 days.

Conference call details will be provided on our website.

About Jakafi (ruxolitinib)
Jakafi (ruxolitinib) is a JAK1/JAK2 inhibitor approved by the U.S. FDA for treatment of polycythemia vera (PV) in adults who have had an inadequate response to or are intolerant of hydroxyurea; intermediate or high-risk myelofibrosis (MF), including primary MF, post-polycythemia vera MF and post-essential thrombocythemia MF in adults; steroid-refractory acute graft-versus-host disease (GVHD) in adult and pediatric patients 12 years and older; and chronic GVHD after failure of one or two lines of systemic therapy in adult and pediatric patients 12 years and older6.

Jakafi is marketed by Incyte in the United States and by Novartis as Jakavi (ruxolitinib) outside the United States. Jakafi is a registered trademark of Incyte Corporation. Jakavi is a registered trademark of Novartis AG in countries outside the United States.

About Iclusig (ponatinib) tablets
Ponatinib (Iclusig) targets not only native BCR-ABL but also its isoforms that carry mutations that confer resistance to treatment, including the T315I mutation, which has been associated with resistance to other approved TKIs.

In the EU, Iclusig is approved for the treatment of adult patients with chronic phase, accelerated phase or blast phase chronic myeloid leukemia (CML) who are resistant to dasatinib or nilotinib; who are intolerant to dasatinib or nilotinib and for whom subsequent treatment with imatinib is not clinically appropriate; or who have the T315I mutation, or the treatment of adult patients with Philadelphia-chromosome positive acute lymphoblastic leukemia (Ph+ ALL) who are resistant to dasatinib; who are intolerant to dasatinib and for whom subsequent treatment with imatinib is not clinically appropriate; or who have the T315I mutation.

Click here to view the Iclusig EU Summary of Medicinal Product Characteristics.

Incyte has an exclusive license from Takeda Pharmaceuticals International AG to commercialize ponatinib in the European Union and 29 other countries, including Switzerland, UK, Norway, Turkey, Israel and Russia. Iclusig is marketed in the U.S. by Millennium Pharmaceuticals, Inc., a wholly owned subsidiary of Takeda Pharmaceutical Company Limited.

About Monjuvi/Minjuvi (tafasitamab)
Monjuvi/Minjuvi (tafasitamab) is a humanized Fc-modified CD19 targeting immunotherapy. In 2010, MorphoSys licensed exclusive worldwide rights to develop and commercialize tafasitamab from Xencor, Inc. Tafasitamab incorporates an XmAb engineered Fc domain, which mediates B-cell lysis through apoptosis and immune effector mechanism including Antibody-Dependent Cell-Mediated Cytotoxicity (ADCC) and Antibody-Dependent Cellular Phagocytosis (ADCP).

In the United States, Monjuvi (tafasitamab-cxix) is approved by the U.S. Food and Drug Administration in combination with lenalidomide for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) not otherwise specified, including DLBCL arising from low grade lymphoma, and who are not eligible for autologous stem cell transplant (ASCT). This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). Please see the U.S. full Prescribing Information for Monjuvi for important safety information.

In Europe, Minjuvi (tafasitamab) received conditional marketing authorization in combination with lenalidomide, followed by Minjuvi monotherapy, for the treatment of adult patients with relapsed or refractory DLBCL who are not eligible for ASCT.

Tafasitamab is being clinically investigated as a therapeutic option in B-cell malignancies in several ongoing combination trials. Its safety and efficacy for these investigational uses have not been established in pivotal trials.

Monjuvi and Minjuvi are registered trademarks of MorphoSys AG. Tafasitamab is co-marketed by Incyte and MorphoSys under the brand name Monjuvi in the U.S., and marketed by Incyte under the brand name Minjuvi in Europe and Canada.

XmAb is a registered trademark of Xencor, Inc.

About Pemazyre (pemigatinib)
Pemazyre is a kinase inhibitor indicated in the United States for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or other rearrangement as detected by an FDA-approved test7. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

Pemazyre is also the first targeted treatment approved for use in the United States for treatment of adults with relapsed or refractory myeloid/lymphoid neoplasms (MLNs) with FGFR1 rearrangement.

In Japan, Pemazyre is approved for the treatment of patients with unresectable biliary tract cancer (BTC) with a fibroblast growth factor receptor 2 (FGFR2) fusion gene, worsening after cancer chemotherapy.

In Europe, Pemazyre is approved for the treatment of adults with locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement that have progressed after at least one prior line of systemic therapy.

Pemazyre is a potent, selective, oral inhibitor of FGFR isoforms 1, 2 and 3 which, in preclinical studies, has demonstrated selective pharmacologic activity against cancer cells with FGFR alterations.

Pemazyre is marketed by Incyte in the United States, Europe and Japan.

Pemazyre is a trademark of Incyte Corporation.

About Axatilimab
Axatilimab is an investigational monoclonal antibody that targets colony stimulating factor-1 receptor, or CSF-1R, a cell surface protein thought to control the survival and function of monocytes and macrophages. In pre-clinical models, inhibition of signaling through the CSF-1 receptor has been shown to reduce the number of disease-mediating macrophages along with their monocyte precursors, which has been shown to play a key role in the fibrotic disease process underlying diseases such as chronic graft-versus-host disease (GVHD) and idiopathic pulmonary fibrosis (IPF). Phase 1/2 data of axatilimab in chronic GVHD demonstrating its broad activity and tolerability was last presented at the 63rd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting. Axatilimab was granted Orphan Drug Designation by the U.S. Food and Drug Administration for the treatment of patients with chronic GVHD and IPF. In September 2021, Syndax and Incyte entered into an exclusive worldwide co-development and co-commercialization license agreement for axatilimab. Axatilimab is being developed under an exclusive worldwide license from UCB entered into between Syndax and UCB in 2016.

Enrollment in the Company’s global pivotal Phase 2 AGAVE-201 Phase 2 study evaluating the efficacy, safety, and tolerability of axatilimab in patients with recurrent or refractory active chronic GVHD who have received at least two prior lines of systemic therapy is complete, and topline data is expected mid-2023. Additionally, a Phase 1 combination trial of ruxolitinib and axatilimab, led by Incyte, is in preparation and expected to initiate by end of the first quarter of 2023, and a Phase 2b trial of axatilimab in patients with idiopathic pulmonary fibrosis led by Syndax is expected to begin in the fourth quarter of 2022.

For more information about AGAVE-201, please visit View Source

About Itacitinib
Itacitinib (INCB039110) is a novel and selective JAK1 inhibitor currently in clinical studies for the first-line treatment of patients with acute and chronic graft-versus-host disease (GVHD).

Itacitinib was discovered at Incyte, and Incyte holds the global development and commercialization rights for itacitinib with the exception of China, where the rights to develop and commercialize itacitinib have been licensed to Innovent Biologics, Inc.

On November 2, 2023 MEI Pharma, Inc. (Nasdaq: MEIP), a clinical-stage pharmaceutical company focused on advancing new therapies for cancer, reported that an abstract highlighting clinical data from the monotherapy dose escalation stage of the ongoing Phase 1 study evaluating voruciclib, a selective oral cyclin-dependent kinase 9 (CDK9) inhibitor, alone and in combination with venetoclax (Venclexta), a B-cell lymphoma 2 ("BCL2") inhibitor, in patients with acute myeloid leukemia (AML) or B-cell malignancies, will be presented during a poster session at the upcoming 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition to be held December 9 – 12, 2023 (Press release, MEI Pharma, NOV 2, 2023, View Source [SID1234636843]).

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Presentation Title: A Phase 1 Study of the Oral CDK9 Inhibitor Voruciclib in Relapsed/Refractory (R/R) B-Cell Lymphoma (NHL) or Acute Myeloid Leukemia (AML)
Session Title: Acute Myeloid Leukemias: Investigational Therapies, Excluding Transplantation and Cellular Immunotherapies: Poster III (616)
Presenter: Mathew Davids, MD, MMSc., Director, Clinical Research, Division of Lymphoma, Dana Farber Cancer Institute
Date: Monday, December 11, 2023, 6:00-8:00 PM (Pacific Time)
Publication Number: 4286

About the Phase 1 Study

The Phase 1 study is a two stage, open-label, 3+3 dose escalation and expansion study evaluating voruciclib, a CDK9 inhibitor, as a monotherapy and in combination with venetoclax (marketed as Venclexta), a BCL2 inhibitor. Inhibition of CDK9 blocks the production of Mcl-1, which is an established resistance mechanism to the BCL2 inhibitor venetoclax. The primary objectives of the study are to determine the safety and biologic effective dose of voruciclib monotherapy or voruciclib in combination with venetoclax. Secondary objectives of the study include assessing the preliminary efficacy, pharmacokinetics, pharmacodynamics, and biomarkers of voruciclib monotherapy or voruciclib in combination with venetoclax.

The first stage of the study, evaluating the dose and schedule of voruciclib as a single-agent in patients with relapsed and refractory ("R/R") acute myeloid leukemia ("AML") or B-cell malignances after failure of standard therapies, is now completed and the final results presented in the abstract. Stage 2 of the study is evaluating voruciclib in combination with standard dose venetoclax in patients with R/R AML.

About Voruciclib

Voruciclib is an orally administered cyclin-dependent kinase 9 ("CDK9") inhibitor with potential to treat both hematological malignancies and solid tumors. It is in clinical development for acute myeloid leukemia and B-cell malignancies. Applications in solid tumors are also being considered.

The CDK family of proteins are important cell cycle regulators responsible for the control of cell proliferation, differentiation, apoptosis, and DNA repair. CDK9, one of several members of the CDK family of proteins, functions as a gene transcription controller and is also involved in regulating protein degradation. Specifically, CDK9 is a promising target to treat a range of cancers because of its role in controlling two other proteins often dysregulated in cancerous cells: myeloid leukemia cell differentiation protein ("Mcl-1") and the MYC proto-oncogene protein ("MYC")

Mcl-1 is a member of the family of anti-apoptotic proteins which, when elevated, may prevent the cell from undergoing cell death. Inhibition of CDK9 blocks the production of Mcl-1, which is an established resistance mechanism to the B-cell lymphoma 2 ("BCL2") inhibitor venetoclax (marketed as Venclexta).

MYC regulates cell proliferation and growth. Upregulation of MYC is implicated in many human cancers and is frequently associated with poor prognosis and unfavorable patient survival. CDK9, in addition to being a transcription factor for MYC, also decreases phosphorylation of MYC protein that is implicated in stabilizing MYC in KRAS mutant cancers. Targeting MYC directly has historically been difficult, but CDK9 is a promising approach to target this oncogene.

On November 2, 2023 A2 Biotherapeutics, Inc. (A2 Bio), a clinical-stage cell therapy company developing first-in-class logic-gated cell therapies, reported new highlights from the BASECAMP-1 (NCT04981119) master pre-screening protocol in an oral presentation Friday, November 3, at the 38th Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting November 1-5, 2023, in San Diego (Press release, A2 Biotherapeutics, NOV 2, 2023, View Source [SID1234636842]). Additionally, A2 Bio is presenting two posters on November 4, 2023, detailing the BASECAMP-1 master pre-screening trial and EVEREST-1 CAR T interventional trial.

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"There is a high unmet need for solid tumor cancer treatments. I am excited to be an Investigator with the BASECAMP-1 pre-screening study, which identifies patients early in their clinical course for treatment with A2 Bio’s novel cell therapy platform," said Diane Simeone, M.D., Laura and Isaac Perlmutter Professor of Surgery at NYU.

"BASECAMP-1 provides patients an early opportunity to be identified and triaged to the appropriate EVEREST logic-gate Tmod CAR T trial. BASECAMP-1 is an innovative approach to preserve T cells for subsequent CAR-T manufacturing, in a patient’s clinical course before they are exposed to extensive chemotherapy," said William Go, M.D., Ph.D., Chief Medical Officer of A2 Bio.

BASECAMP-1 is a novel pre-screening protocol to identify a diverse set of patients with the genetic loss of HLA-A*02 in solid tumors, which is required to enroll in A2 Bio interventional trials.

A2 Bio has partnered with Tempus, a leader in precision medicine, to bring the latest technology to patient screening. In BASECAMP-1, tumor tissue of patients at A2 clinical sites is tested through Tempus’ xT NGS diagnostic to identify patients with permanent genetic loss of HLA-A*02 in their tumors. In addition, Tempus connects patients with HLA loss screened through standard of care to A2 clinical investigators through the AWARE program.

Once eligibility is met, patients from BASECAMP-1 can be leukapheresed with their cells stored for future manufacturing for a current or future A2 Bio interventional trial. BASECAMP-1 is open at 13 sites across the United States.

EVEREST-1 (NCT05736731) is a seamless Phase 1/2 study for A2B530, the first autologous logic-gated cell therapy developed from A2 Bio’s proprietary Tmod platform. The Tmod platform utilizes a dual-receptor design consisting of an activator that targets tumor cells and a blocker that protects normal cells. A2B530 consists of an activator that targets carcinoembryonic antigen (CEA) and a blocker that targets HLA-A*02.

The EVEREST-1 poster will highlight the study design for A2B530, which is actively dosing patients with pancreatic, colorectal and lung cancers. If a patient is ready for treatment, A2B530 can be manufactured for a patient from their apheresis that has already been collected and stored at A2 Bio via the BASECAMP-1 study. EVEREST-1 is open at 8 sites across the United States.

EVEREST-2 (NCT06051695), A2 Bio’s second clinical program, is a seamless Phase 1/2 study for A2B694, which targets mesothelin (MSLN) with a blocker that targets HLA-A*02. Patients with pancreatic, lung, colorectal, ovarian and mesothelioma are currently being pre-screened through the BASECAMP-1 study.

Details of the presentations are below:

Oral presentation

BASECAMP-1: A master prescreening study to identify patients with high-risk or metastatic solid tumors with HLA loss of heterozygosity (LOH) in preparation for Tmod CAR T-cell therapy trials
Presenter: Diane Simeone, M.D. – NYU
Session: Rapid Oral Abstract-Clinical | Session 105b
Date and time: Friday, November 3; 12:15pm – 1:15pm PT
Location: Ground Level – Exhibit Hall C – San Diego Convention Center

Poster presentations

EVEREST-1: A seamless phase 1/2 study of CEA logic-gated Tmod CAR T-cell therapy (A2B530) in patients with solid tumors associated with CEA expression also exhibiting HLA loss of heterozygosity (LOH)
Abstract: 634
Presenter: Salman Punekar, M.D. – NYU
Date and time: Saturday, November 4, 2023; 9am – 8:30pm PT
Location: Exhibit Halls A and B1 – San Diego Convention Center

BASECAMP-1: A master prescreening study to identify patients with high-risk or metastatic solid tumors with HLA loss of heterozygosity (LOH) in preparation for Tmod CAR T-cell therapy trials
Abstract: 636
Presenter: Diane Simeone, M.D. – NYU
Date and time: Saturday, November 4, 2023; 9am – 8:30pm PT
Location: Exhibit Halls A and B1 – San Diego Convention Center

The two posters will be available on the A2 Bio website after November 4, 2023.

About A2B530

A2B530 consists of an activator that targets carcinoembryonic antigen (CEA) and a blocker that targets HLA-A*02. CEA is a tumor-associated antigen expressed at high levels in colorectal, pancreatic and non-small cell lung cancers, but also in healthy gut tissues. HLA-A*02 is expressed in normal tissues and permanently lost via genetic deletion in tumor tissues in the patient population eligible for EVEREST-1. A2B530’s dual-receptor design is intended to provide selective killing of tumor tissues that express CEA and have lost the HLA-A*02 gene permanently.

About A2B694

A2B694 consists of an activator that targets mesothelin (MSLN) and a blocker that targets HLA-A*02. MSLN is a tumor-associated antigen expressed at high levels in mesothelioma, ovarian, colorectal, pancreatic, and non-small cell lung cancers, but also in healthy tissue such as the lining of the heart and lung. HLA-A*02 is expressed in normal tissues and permanently lost via genetic deletion in tumor tissues in the patient population eligible for EVEREST-2. A2B694’s dual-receptor design is intended to provide selective killing of tumor tissues that express MSLN and have lost the HLA-A*02 gene permanently.