On May 6, 2024 GlycoMimetics, Inc. (Nasdaq: GLYC), a late clinical-stage biotechnology company discovering and developing glycobiology-based therapies for cancers and inflammatory diseases, reported topline results from its Phase 3 global pivotal study of uproleselan in 388 patients with R/R AML (Press release, GlycoMimetics, MAY 6, 2024, View Source [SID1234642677]). In the study, uproleselan combined with chemotherapy did not achieve a statistically significant improvement in overall survival in the intent to treat population versus chemotherapy alone.

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Patients treated with uproleselan had a median overall survival of 13 months, compared to 12.3 months in the placebo arm. Adverse events were consistent with known side effect profiles of chemotherapy used in the study.

"While the outcome of our Phase 3 study in R/R AML is not what we hoped, we wish to thank the investigators, the participating patients and their families for their dedication to this large, well-controlled randomized study," said Harout Semerjian, Chief Executive Officer of GlycoMimetics. "We are thoroughly analyzing the data in collaboration with medical, statistical and regulatory experts and are committed to submitting a comprehensive data analysis for presentation at an upcoming medical meeting."

The randomized, double-blind, placebo-controlled Phase 3 clinical study evaluated uproleselan in combination with MEC (mitoxantrone, etoposide and cytarabine) or FAI (fludarabine, cytarabine and idarubicin) in patients with R/R AML. Patients received either uproleselan or placebo for 8 days over 1 cycle of an induction and, if applicable, up to 3 cycles of consolidation. The primary endpoint of the study was overall survival without censoring for transplant. Secondary endpoints included incidence of severe oral mucositis, complete remission rate and remission rate. A total of 388 patients across 70 sites in nine countries were randomized 1:1 between treatment and placebo arms.

The NCI and the Alliance for Clinical Trials in Oncology are conducting an adaptive Phase 2/3 study of uproleselan in adults with newly diagnosed AML who are 60 years or older and fit for intensive chemotherapy. The randomized, controlled study is evaluating the addition of uproleselan to a standard cytarabine/daunorubicin regimen (7+3) versus chemotherapy alone. The Phase 2 portion of the study completed enrollment of 267 patients in December 2021. Results of the pre-planned Phase 2 event free survival interim analysis will be reported when available.

First Quarter 2024 Preliminary Financial Results

Today, the company also disclosed its preliminary financial results for the first quarter of 2024.

Cash position: As of March 31, 2024, GlycoMimetics had cash and cash equivalents of $31.3 million, compared to $41.8 million as of December 31, 2023.
R&D Expenses: The company’s research and development expenses increased to $6.0 million for the quarter ended March 31, 2024, as compared to $5.4 million for the same period in 2023. These increases were due to raw material acquisition costs for future manufacturing batches.
G&A Expenses: The company’s general and administrative expenses decreased to $5.1 million for the quarter ended March 31, 2024, compared to $5.5 million for the same period in 2023. The decrease was due to lower personnel-related and external consulting expenses.
Shares Outstanding: Shares of common stock outstanding as of March 31, 2024, were 64,450,835.
Conference Call Information

The company will host a conference call and webcast today at 8:30 a.m. ET. To access the call by phone, please go to this registration link and you will be provided with dial in details. Participants are encouraged to connect 15 minutes in advance of the scheduled start time.

A live webcast of the call will be available on the "Investors" tab on the GlycoMimetics website. A webcast replay will be available for 30 days following the call.

Please note this call will replace the previously announced First Quarter 2024 Financial Results call scheduled for May 9, 2024 at 8:30 a.m. ET.

About AML

AML is the most common acute leukemia in adults. A cancer of the bone marrow, nearly 21,000 people in the United States are diagnosed with AML each year. Despite the availability of multiple treatments, disease prognosis is poor, and new treatment options are needed to improve outcomes. Newly diagnosed AML has the lowest 5-year survival rate of all leukemias at 31.7%. The five-year survival rate for people with relapsed/refractory disease is only 10%.

About Uproleselan

Discovered and developed by GlycoMimetics, uproleselan (yoo’ pro le’se lan) is an investigational, first-in-class E-selectin antagonist. GlycoMimetics has received Breakthrough Therapy and Fast Track designations from the U.S. Food and Drug Administration (FDA) and Breakthrough Therapy designation from the Chinese National Medical Products Administration for uproleselan as a potential treatment for adult AML patients with relapsed or refractory disease. E-selectin is a leukocyte adhesion molecule constitutively expressed on endothelial cells of the vasculature and bone marrow. In AML, there is evidence that E-selectin–ligand interaction between endothelial cells in the protective niche of the Bone Marrow microEnvironment (BME) and leukemic stem cells and blasts promotes leukemic cell survival and hides them from AML therapies. Uproleselan is designed to disrupt E-selectin binding and prevent leukemic myeloid cells using the protective niche of the BME.

On May 6, 2024 Foghorn Therapeutics Inc. (Nasdaq: FHTX), a clinical-stage biotechnology company pioneering a new class of medicines that treat serious diseases by correcting abnormal gene expression, reported a financial and corporate update in conjunction with the Company’s 10-Q filing for the quarter ended March 31, 2024 (Press release, Foghorn Therapeutics, MAY 6, 2024, View Source [SID1234642676]). With an initial focus in oncology, Foghorn’s Gene Traffic Control Platform and resulting broad pipeline have the potential to transform the lives of people suffering from a wide spectrum of diseases.

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"We are excited about the progress and strength of data presented this quarter which includes robust preclinical anti-tumor monotherapy activity at tolerable doses for multiple pipeline programs. We believe these data demonstrate the potential of our platform to generate novel medicines against very challenging targets, as well as our leadership in the development of therapeutics in chromatin biology," said Adrian Gottschalk, President and Chief Executive Officer of Foghorn.

Mr. Gottschalk added, "In the second half of the year, we expect initial data from our FHD-286 combination study in AML. The IND for FHD-909 was recently submitted and would be the first selective BRM inhibitor to enter the clinic, with a Phase 1 trial planned to begin in the second half of this year. Earlier this year, we also shared data from our Selective CBP degrader program demonstrating tumor growth inhibition in solid tumors without thrombocytopenia or anemia that have been observed with dual CBP/EP300 inhibition. Our Selective EP300 degrader program also demonstrated promising preclinical efficacy with no thrombocytopenia, which is often seen in dual approaches. Our cash balance remains strong, and with multiple programs advancing, we are expected to deliver near- and mid-term value for both patients and shareholders."

Corporate Updates

Presented at AACR (Free AACR Whitepaper) Annual Meeting. In April 2024, Foghorn presented preclinical data highlighting pipeline progress from multiple potential first-in-class medicines, including the first presentation of preclinical data for FHD-909, at the 2024 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting held April 5-10, 2024.

Hosted Chromatin Regulation Summit. In April 2024, Foghorn hosted the first Future of Disease and Chromatin Regulation Summit at the Foghorn corporate headquarters in Cambridge, Massachusetts. The live event featured presentations and panel discussions with world-renowned industry and academic key opinion leaders on therapeutic opportunities in chromatin regulatory biology.

Strengthened Financial Leadership. In April 2024, Foghorn appointed Kristian Humer as Chief Financial Officer. Mr. Humer joins Foghorn with over 14 years of diversified financial strategy and business development experience in the life science industry and more than 20 years of experience in the financial industry.

Key Recent Program Updates and Upcoming Milestones

FHD-286. FHD-286 is a potent, selective inhibitor of the BRG1 (SMARCA4) and BRM (SMARCA2) subunits of the BAF chromatin remodeling complex where dependency on BRG1/BRM is well-established preclinically with multiple tumor types, including acute myeloid leukemia (AML)/myelodysplastic syndrome (MDS), non-small cell lung cancer (NSCLC) and prostate cancer.
•AML Update. Foghorn initiated a Phase 1 trial of FHD-286 in combination with decitabine or low-dose cytarabine (LDAC) in relapsed and/or refractory AML patients, with the first patient dosed during the third quarter of 2023. Dose escalation is ongoing with initial clinical data expected in the second half of 2024.
•Tyrosine Kinase Inhibitor Resistance. Data published in Cancer Cell in August 2023, along with Foghorn’s work, suggest that FHD-286 may play an important role in overcoming and delaying resistance in EGFR/KRAS tumors. Preclinical data is expected in the second quarter of 2024.

FHD-909. FHD-909 is a first-in-class oral BRM (SMARCA2) selective inhibitor that has been demonstrated in preclinical studies to have high selectivity over its closely related paralog BRG1 (SMARCA4), two proteins that are the catalytic engines across all forms of the BAF complex. BRG1 mutations are common across tumor types, including approximately 10% of NSCLC, and result in tumors being dependent on BRM activity for their survival. Selectively blocking BRM activity is a promising synthetic lethal strategy intended to induce tumor death while sparing healthy cells.
•In December 2021, Foghorn announced a strategic collaboration with Lilly to create novel oncology medicines. The collaboration includes a US 50/50 co-development and co-commercialization agreement for Foghorn’s Selective BRM oncology program and an additional undisclosed oncology target. In addition, the collaboration includes three discovery programs using Foghorn’s proprietary Gene Traffic Control platform.
•In the first quarter of 2024, Lilly selected FHD-909 for clinical development and in April the investigational new drug (IND) application was submitted. The primary target patient population is BRG1-mutated NSCLC.
•In April 2024, Foghorn and Lilly presented new preclinical data as a poster presentation at the AACR (Free AACR Whitepaper) Annual Meeting demonstrating, at tolerable doses, high BRM selectivity and dose-dependent single agent activity in BRG1-mutated cancers.
•Dosing of first patient in Phase 1 trial for FHD-909 in BRG1-mutated NSCLC is expected in the second half of 2024.

Selective CBP Degrader Program and Selective EP300 Degrader Program. Foghorn is advancing two separate programs targeting either CBP or EP300, both of which are paralog histone acetyltransferases with a synthetic lethal relationship in tumor cells. Attempts to selectively drug CBP or EP300 have been historically challenging due to the high level of similarity between the two proteins. Additionally, dual inhibition of CBP/EP300 is limited by hematopoietic toxicity.
•Selective CBP Degrader Program. In April, Foghorn presented new pharmacodynamic and pharmacokinetic preclinical data at the 2024 AACR (Free AACR Whitepaper) Annual Meeting highlighting:
•Deep and sustained CBP degradation significantly inhibited tumor growth in mouse xenograft solid tumor models.
•Robust monotherapy preclinical anti-tumor activity that was not associated with significant body weight loss, thrombocytopenia or anemia.
•Identification of potent and selective CBP protein degraders with first-in-class potential to address tumors harboring EP300 mutations in many types of cancer, including bladder, gastric and endometrial cancers.
•Long-acting CBP-selective protein degrader formulations with first-in-class potential for patients with tumors harboring EP300 mutations.
•IND-enabling studies on track to initiate by year-end 2024.

•Selective EP300 Degrader Program. In April, Foghorn presented new pharmacodynamic and pharmacokinetic preclinical data at the 2024 AACR (Free AACR Whitepaper) Annual Meeting highlighting:
•No observed thrombocytopenia in vivo.
•Identification of potent and selective EP300 protein degraders with first-in-class potential to address tumors harboring CBP mutations and tumors with dependency on EP300 in several types of cancer, including prostate, multiple myeloma and diffuse large B cell lymphoma (DLBCL).

Selective ARID1B Degrader Program. ARID1A is the most mutated subunit in the BAF Complex and amongst the most mutated proteins in oncology. These mutations lead to a dependency on ARID1B, in several types of cancer, including ovarian, endometrial, colorectal and bladder. Attempts to selectively drug ARID1B have been challenging because of the high degree of similarity between ARID1A and ARID1B and the fact that ARID1B has no enzymatic activity to target.
•In April, Foghorn presented data at the AACR (Free AACR Whitepaper) Annual Meeting demonstrating potent and selective small molecule binders to ARID1B. The Company is in the process of converting these selective binders into heterobifunctional degraders.

Drug Discovery Platform. Foghorn’s synthesis of deep chromatin expertise, protein biochemistry and screening, and degrader expertise uniquely positions the Company to successfully drug historically challenging targets.
•Potential for five additional INDs in the next four years.
•Targets combined have the potential to address more than 20 tumor types impacting over 500,000 new patients annually.

First Quarter 2024 Financial Highlights

•Collaboration Revenues. Collaboration revenue was $5.1 million for the three months ended March 31, 2024, compared to $5.3 million for the three months ended March 31, 2023. The decrease year-over-year was primarily driven by the termination of the Merck collaboration agreement.

•Research and Development Expenses. Research and development expenses were $25.5 million for the three months ended March 31, 2024, compared to $30.0 million for the three months ended March 31, 2023. This decrease was primarily due to lower personnel-related costs.

•General and Administrative Expenses. General and administrative expenses were $7.7 million for the three months ended March 31, 2024, compared to $8.6 million for the three months ended March 31, 2023. This decrease was primarily due to lower personnel-related costs.

•Net Loss. Net loss was $25.0 million for the three months ended March 31, 2024, compared to a net loss of $30.5 million for the three months ended March 31, 2023.

•Cash, Cash Equivalents and Marketable Securities. As of March 31, 2024, the Company had $206.7 million in cash, cash equivalents and marketable securities, which providing expected cash runway into the first half of 2026.

About FHD-286
FHD-286 is a potent, selective, allosteric, and orally available small-molecule, enzymatic inhibitor of BRG1 (SMARCA4) and BRM (SMARCA2), two highly similar proteins that are the ATPases, or the catalytic engines, of the BAF complex, one of the key regulators within the chromatin regulatory system. In preclinical studies, FHD-286 has shown anti-tumor activity across a broad range of malignancies including both hematologic and solid tumors.

About AML
Adult acute myeloid leukemia (AML) is a cancer of the blood and bone marrow and the most common type of acute leukemia in adults. AML is a diverse disease associated with multiple genetic mutations. It is diagnosed in about 20,000 people every year in the United States.

About FHD-909
FHD-909 (a.k.a. LY4050784) is a potent, allosteric and orally available small molecule that selectively inhibits the ATPase activity of BRM (SMARCA2) over its closely related paralog BRG1 (SMARCA4), two proteins that are the catalytic engines across all forms of the BAF complex, one of the key regulators of the chromatin regulatory system. In preclinical studies, tumors with mutations in BRG1 rely on BRM for BAF function. FHD-909 has shown significant anti-tumor activity across multiple BRG1-mutant lung tumor models.

On May 6, 2024 Delcath Systems, Inc. (Nasdaq: DCTH), an interventional oncology company focused on the treatment of primary and metastatic cancers of the liver, reported the publication of results from the pivotal Phase 3 FOCUS study of HEPZATO KIT (melphalan/Hepatic Delivery System) in patients with unresectable metastatic Uveal Melanoma (mUM) on May 5, 2024 in the journal Annals of Surgical Oncology (Press release, Delcath Systems, MAY 6, 2024, View Source [SID1234642675]).

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The Food and Drug Administration (FDA) approved HEPZATO KIT on August 14, 2023 based on the results from the pivotal FOCUS study. A total of 91 patients with unresectable mUM were treated with HEPZATO KIT at 23 treatment centers in the US and Europe. Preliminary results from the FOCUS study were presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in 2022.

The FOCUS study was designed to provide a robust evaluation of efficacy and safety of HEPZATO KIT treatment, and enrolled a heterogeneous mUM patient population, including treatment-naïve and pretreated patients, patients with and without extrahepatic disease and patients with a range of baseline tumor burden.

The primary efficacy endpoint of the FOCUS study was Overall Response Rate (ORR), which was 36.3%, including 7.7% of patients with Complete Response (CR), as determined by an Independent Review Committee. 37.4% of patients had Stable Disease (SD). ORR achieved in the FOCUS study was compared to a Meta-analysis of historic data, encompassing 16 published clinical studies with a total of 476 mUM patients treated with contemporary immunotherapy drugs. ORR of 36.3% in the FOCUS study was statistically significantly better than the pooled ORR estimate (a weighted mean of the observed ORR) of 5.5% in the historical control group.

Secondary efficacy endpoints included Duration of Response (DOR), median Progression-free Survival (mPFS) and median Overall Survival (mOS), which were 14, 9 and 20.5 months, respectively.

Safety and tolerability of HEPZATO KIT treatment reported in the FOCUS study was comparable with published clinical experience with Chemosat in Europe. Median number of administered HEPZATO KIT treatment cycles in the FOCUS study was 4. The most common serious treatment-emergent adverse events (SAE) were thrombocytopenia (15.8%) and neutropenia (10.5%), managed with standard supportive care and resolved with no ongoing complications. No treatment-related deaths were observed.

"My team at the Moffitt Cancer Center and I are very excited about the publication of results from the FOCUS study. The study results are consistent with data obtained from previous clinical studies, as well as our own experience with percutaneous hepatic perfusion (PHP) with more than 200 treatments performed at Moffitt over the past 15 years," said Dr. Jonathan Zager, MD Chief Academic Officer and Director of Regional Therapies at Moffitt Cancer Center. "Since HEPZATO KIT became available in January, the treatment has become the liver directed standard of care for appropriate patients with mUM at our institution."

"Results from the pivotal FOCUS study demonstrate that the PHP procedure, whether utilizing the FDA approved HEPZATO KIT or melphalan delivered by the Chemosat device available in Europe, is an important treatment option for patients with liver-dominant mUM," said Dr. Vojo Vukovic, MD, PhD Delcath’s Chief Medical Officer. "Delcath is looking forward to publishing additional results from the FOCUS study later this year."

The vast majority of patients with mUM will have liver metastases, often leading to liver failure, and the National Comprehensive Cancer Network guidelines recommend liver-directed therapies be considered for the treatment of mUM. HEPZATO is the only liver-directed treatment for unresectable mUM patients approved by the FDA.

On May 6, 2024 Corvus Pharmaceuticals, Inc. (Corvus or the Company) (Nasdaq: CRVS) (GLOBAL NEWSWIRE), a clinical-stage biopharmaceutical company, reported a business update and announced financial results for the first quarter ended March 31, 2024 (Press release, Corvus Pharmaceuticals, MAY 6, 2024, View Source [SID1234642674]).

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"We continue to make strong progress with our two key priorities for the year – advancing soquelitinib for PTCL and generating early data for soquelitinib for atopic dermatitis," said Richard A. Miller, M.D., co-founder, president and chief executive officer of Corvus. "We are encouraged that two newly evaluable PTCL patients from the soquelitinib Phase 1 trial achieved objective responses – one complete response and one partial response – as we move forward with our plans for a registration Phase 3 trial that is on track to be initiated in the third quarter. We strengthened our position with a recent financing that gives us runway through several expected clinical readouts for our pipeline in 2024 and 2025, including early Phase 1 data with soquelitinib in atopic dermatitis in late 2024 and final data in 2025, soquelitinib initial Phase 1 data in solid tumors in the second half of 2025, and additional ciforadenant data later this year."

"We are excited by the initial clinical results from our Phase 1b/2 trial of ciforadenant, which are consistent with our 2018 publication of preclinical models demonstrating that anti-CTLA-4 is a promising agent to combine with adenosine antagonists," said Dr. Miller. "We believe we have identified an important, novel mechanism leveraging the combination of adenosine antagonism and checkpoint inhibitors working together to enhance anti-tumor efficacy."

"We are encouraged by the early results achieved with ciforadenant in this trial," said Kathryn Beckermann, M.D., PhD, Assistant Professor of Medicine, Division of Hematology Oncology at Vanderbilt University Medical Center and lead investigator of the ciforadenant Phase 1b/2 clinical trial. "Immunotherapies have made a large impact on the treatment of metastatic renal cell cancer, however, most patients do not achieve deep tumor responses and ultimately experience disease relapse. Ciforadenant adds a new immunotherapeutic mechanism that may be synergistic to our standard regimen, providing additional benefit by helping more patients achieve a deep response, which in our experience has significantly improved their long-term outlook."

Financing Update

On May 6, 2024, we closed a registered direct offering that resulted in gross proceeds of approximately $30.6 million. The financing consisted of the sale of 13,512,699 shares of common stock and accompanying common warrants to purchase 13,078,509 shares of common stock (or pre-funded warrants in lieu thereof) for a combined offering price of $1.7312 per share, and pre-funded warrants to purchase 4,144,085 shares of common stock and accompanying common warrants to purchase 4,010,927 shares of common stock (or pre-funded warrants in lieu thereof) at a combined offering price of $1.7311 per share. The pre-funded warrants have an exercise price of $0.0001 per share of common stock and the common warrants have an exercise price of $3.50 per share of common stock (or $3.4999 per pre-funded warrant). The common warrants and pre-funded warrants are immediately exercisable, subject to certain ownership limitations, and the common warrants expire on June 30, 2025.

Business Update and Strategy

Prioritized Program: Soquelitinib (formerly CPI-818, Corvus’ selective ITK inhibitor)

Soquelitinib for T Cell Lymphoma

Corvus continues to follow patients with relapsed T cell lymphoma in its Phase 1/1b clinical trial (no longer enrolling new patients) evaluating single agent therapy with soquelitinib. Updated interim data as of May 3, 2024:
A total of 25 patients were enrolled in the Phase 1/1b trial at the optimum 200 mg two-times a day dose and meet the eligibility criteria for the planned registrational Phase 3 clinical trial based on ≥1 and ≤3 prior therapies, including 23 evaluable patients.
For the 23 evaluable patients, objective responses (complete response, CR plus partial response, PR) were seen in nine patients (39%), including five CRs (22%) and four PRs. Compared to the prior data reported as of January 22, 2024, two additional patients have responded at the first follow up visit, and both of these patients are continuing on therapy. These two patients had each failed two prior therapies and had multiple sites of disease. See waterfall plot below.
Disease control (CR, PR and stable disease) was seen in 14 of 23 patients (61%). The stable disease group included five patients who achieved tumor reductions that did not meet the criteria for a PR. Several patients experiencing tumor regression are continuing to receive therapy.
Corvus anticipates initiating a registrational Phase 3 clinical trial of soquelitinib in patients with relapsed PTCL in the third quarter of 2024. There are currently no FDA fully approved agents for the treatment of relapsed PTCL and the FDA has granted Orphan Drug Designation for soquelitinib for the treatment of T cell lymphoma.
Waterfall Plot for Patients in the 200 mg Dose Cohort of the Soquelitinib Phase 1/1b Clinical Trial for Peripheral T Cell Lymphoma. The plot shows the best percent change in tumor volume in the 23 evaluable patients (eligible patient population), as of May 3, 2024, that were measurable by CT scan or by Modified Severity-Weighted Assessment Tool (mSWAT) for patients with cutaneous involvement. PTCL-NOS, peripheral T cell lymphoma not otherwise specified; CTCL, cutaneous T cell lymphoma of either Sezary or mycosis fungoides type; NKTCL, natural killer cell T cell lymphoma; ALCL, anaplastic large cell lymphoma; AITL, angioimmunoblastic T cell lymphoma.

Soquelitinib for Immune Diseases

Corvus is enrolling patients at multiple clinical sites in its randomized, placebo-controlled Phase 1 trial of soquelitinib in patients with moderate to severe atopic dermatitis. The trial is planned to enroll 64 patients that have failed at least one prior therapy across four different 28-day dosing regimens of soquelitinib compared to a placebo group. The endpoints include safety and improvement in Eczema Area and Severity Index. Patients and physicians will be blinded to treatment assignment. The company anticipates early interim data from the Phase 1 trial by year-end 2024.
Corvus continues to advance its next-generation ITK inhibitor preclinical product candidates, which were designed to deliver precise T-cell modulation that is optimized for specific immunology indications. The next-generation ITK inhibitor candidates are part of the Company’s ongoing business development efforts to maximize the potential of the Company’s ITK inhibitor programs and other programs.
Collaboration with Kidney Cancer Research Consortium: Ciforadenant (adenosine A2a receptor inhibitor)

Corvus is collaborating with Kidney Cancer Research Consortium in a Phase 1b/2 clinical trial evaluating ciforadenant as a potential first line therapy for metastatic renal cell cancer (RCC) in combination with ipilimumab (anti-CTLA-4) and nivolumab (anti-PD-1). The efficacy endpoint for the trial is deep response rate, defined as CR plus PRs of greater than 50% tumor volume reduction. The clinical trial is expected to enroll up to 60 patients and as of May 2, 2024 a total of 27 patients were enrolled in the trial. The protocol defined, interim pre-specified statistical threshold for efficacy is a 50% increase above the 32% deep response rate seen with previous ipilimumab/nivolumab combination trials in RCC conducted by investigators at the Kidney Cancer Research Consortium. As of May 2, 2024, the interim analysis of the clinical trial has met the threshold for efficacy and therefore enrollment continues.
Partner Led Program: Mupadolimab (anti-CD73)

Angel Pharmaceuticals, Corvus’ partner in China, is enrolling patients in a Phase 1/1b clinical trial of mupadolimab in patients with non-small cell lung cancer (NSCLC) and head and neck squamous cell cancers (HNSCC). In this clinical trial, patients will receive mupadolimab monotherapy or in combination with pembrolizumab.
Financial Results

As of March 31, 2024, Corvus had cash, cash equivalents and marketable securities of $22.1 million as compared to $27.1 million as of December 31, 2023. Cash as of March 31, 2024 does not include approximately $30.6 million of cash received in a financing completed on May 6, 2024. Corvus expects full year 2024 net cash used in operating activities to be between approximately $24 million and $27 million, resulting in a projected cash balance of between $31 million and $34 million at December 31, 2024. Based on its current plans, Corvus expects its cash to fund operations into the fourth quarter of 2025.

Research and development expenses for the three months ended March 31, 2024 totaled $4.1 million compared to $4.6 million for the same period in 2023. The decrease of $0.5 million was primarily due to lower manufacturing costs associated with the development of soquelitinib.

The net loss for the three months ended March 31, 2024 was $5.7 million compared to a net loss of $7.9 million for the same period in 2023. Total stock compensation expense for the three months ended March 31, 2024 was $0.7 million compared to $0.5 for the same period in 2023 and the non-cash income from Corvus’ equity method investment in Angel Pharmaceuticals was $0.2 million for the three months ended March 31, 2024 compared to a loss of $1.7 million for the same period in 2023.

Conference Call Details
Corvus will host a conference call and webcast today, Monday, May 6, 2024, at 4:30 p.m. ET (1:30 p.m. PT), during which time management will provide a business update and discuss the first quarter 2024 financial results. The conference call can be accessed by dialing 1- 800-717-1738 (toll-free domestic) or 1- 646-307-1865 (international) or by clicking on this link for instant telephone access to the event. The live webcast may be accessed via the investor relations section of the Corvus website. A replay of the webcast will be available on Corvus’ website for 90 days.

On May 6, 2024 Celldex Therapeutics, Inc. (NASDAQ:CLDX) reported financial results for the first quarter ended March 31, 2024 and provided a corporate update (Press release, Celldex Therapeutics, MAY 6, 2024, View Source [SID1234642673]).

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"During the first quarter we presented impressive best-in-field data from a large, randomized Phase 2 study in CSU," said Anthony Marucci, Co-founder, President and Chief Executive Officer of Celldex Therapeutics. "These results added to a rapidly growing body of data across multiple indications that support barzolvolimab’s impressive potential to treat mast cell mediated diseases. This year, we continue to expand barzolvolimab’s profile and, based on its unique mechanism of action and demonstrated improvement in pruritus, we are actively planning for the initiation of a Phase 2 study in atopic dermatitis, a setting where mast cell numbers are known to be increased and activated in the lesions associated with the disease and where, despite established systemic therapies, a significant unmet need still exists."

Mr. Marucci continued, "As the year progresses, we look forward to building on our leadership in the development of mast cell-targeted therapeutics. Importantly, the Company is well capitalized with more than $820M in cash to support the continued advancement and expansion of the barzolvolimab program and the introduction of our first bispecific for inflammatory diseases later this year."

Recent Program Highlights

Barzolvolimab – KIT Inhibitor Program

Barzolvolimab is a humanized monoclonal antibody developed by Celldex that binds the KIT receptor with high specificity and potently inhibits its activity. The KIT receptor tyrosine kinase is expressed in a variety of cells, including mast cells, which mediate inflammatory responses such as hypersensitivity and allergic reactions. KIT signaling controls the differentiation, tissue recruitment, survival and activity of mast cells.

Celldex is conducting Phase 2 clinical studies of barzolvolimab for the treatment of chronic spontaneous urticaria (CSU) and the two most common forms of chronic inducible urticaria (CIndU) – cold urticaria (ColdU) and symptomatic dermographism (SD). These randomized, double-blind, placebo-controlled Phase 2 studies are evaluating the efficacy and safety profile of multiple dose regimens of barzolvolimab in patients who remain symptomatic despite antihistamine therapy, to determine the optimal dosing strategies.

Celldex is currently planning two Phase 3 studies of barzolvolimab in CSU, which are expected to initiate this summer.

In February 2024, 12 week treatment results were reported from the Phase 2 CSU study at the American Academy of Allergy, Asthma & Immunology (AAAAI) Annual Meeting in a late breaking oral presentation. Barzolvolimab achieved the primary efficacy endpoint of the study, with a statistically significant mean change from baseline to week 12 of UAS7 (weekly urticaria activity score) compared to placebo across multiple dosing groups and was well tolerated. Secondary and exploratory endpoints in the study, including ISS7 (weekly itch severity score) and HSS7 (weekly hives severity score) and responder analyses strongly support the primary endpoint results. Importantly, barzolvolimab demonstrated rapid, durable and clinically meaningful responses in patients with moderate to severe CSU refractory to antihistamines, including patients with prior omalizumab treatment. Approximately 20% (n=41) of enrolled patients received prior treatment with omalizumab and more than half of these patients had omalizumab-refractory disease. These patients experienced a similar clinical benefit as the overall treated population within their individual dosing groups consistent with the barzolvolimab mechanism of action. Patients on study will continue to receive barzolvolimab for 52 weeks and the Company plans to report 52 week data in the second half of 2024.

Data from this study on the impact of barzolvolimab on angioedema have been accepted for an oral presentation at the European Academy of Allergy and Clinical Immunology (EAACI) Congress 2024 on Saturday, June 2nd from 3:00-4:30 CET in Valencia, Spain (9:00-10:30 am ET).

Enrollment to the Phase 2 CIndU study has been completed and 12 week primary endpoint data from this study are expected to be reported in the second half of 2024. The study enrolled 196 patients—97 patients with cold urticaria and 99 patients with symptomatic dermographism.
A Phase 2 subcutaneous study in prurigo nodularis (PN) was initiated in early 2024. This randomized, double-blind, placebo-controlled, parallel group study is evaluating the efficacy and safety profile of barzolvolimab in approximately 120 patients with moderate to severe PN who had inadequate response to prescription topical medications, or for whom topical medications are medically inadvisable, including patients who received prior biologics. Patients are receiving barzolvolimab injections of 150 mg Q4W after an initial loading dose of 450 mg, 300 mg Q4W after an initial loading dose of 450 mg, or placebo during a 24‑week Treatment Phase.
In July 2023, the first patient was dosed in the Phase 2 randomized, double-blind, placebo-controlled study in eosinophilic esophagitis (EoE); enrollment is ongoing. To optimize potential efficacy signal in this difficult to treat indication, in early 2024, the protocol was amended to dose 300 mg every 4 weeks rather than 8 weeks. Approximately 75 patients will be enrolled in total.
Atopic Dermatitis has been selected as the fifth indication for the development of barzolvolimab. Atopic dermatitis (AD) is a chronically relapsing, inflammatory skin disease that is typified by pruritus (itching), eczematous lesions, dry skin, thickening of the skin and an increase in skin markings. AD is the most common inflammatory skin condition, with a lifetime prevalence of up to 20% and has a high itch burden—almost 90% of individuals with AD experience daily itching and 60% describe their itching as severe or unbearable. Barzolvolimab’s novel mast cell depleting mechanism could play an important role in addressing patients with moderate to severe AD who do not achieve complete disease control on currently available systemic therapies. Mast cell numbers are increased and activated in AD lesions and produce key TH2 cytokines and neuropeptides that trigger disease progression and itch. Barzolvolimab has demonstrated impressive anti-pruritic effects in other itch driven conditions, including chronic urticarias and prurigo nodularis. Celldex plans to initiate a Phase 2 study in AD by year end.
Bispecific Antibody Platform

CDX-585 – Bispecific ILT4 & PD-1

CDX-585 combines highly active PD-1 blockade with anti-ILT4 blockade to overcome immunosuppressive signals in T cells and myeloid cells. ILT4 is emerging as an important immune checkpoint on myeloid cells.

In May 2023, the first patient was dosed in the Phase 1 study of CDX-585. This open-label, multi-center study of CDX-585 is evaluating patients with advanced or metastatic solid tumors that have progressed during or after standard of care therapy. Enrollment is ongoing in the dose-escalation portion of the study.
First Quarter 2024 Financial Highlights and 2024 Guidance

Cash Position: Cash, cash equivalents and marketable securities as of March 31, 2024 were $823.8 million compared to $423.6 million as of December 31, 2023. The increase was primarily driven by net proceeds of $432.3 million from our March 2024 underwritten public offering, partially offset by cash used in operating activities of $40.6 million. At March 31, 2024, Celldex had 65.9 million shares outstanding.

Revenues: Total revenue was $0.2 million in the first quarter of 2024, compared to $1.0 million for the comparable period in 2023. The decrease in revenue was primarily due to a decrease in services performed under our manufacturing and research and development agreements with Rockefeller University.

R&D Expenses: Research and development (R&D) expenses were $31.7 million in the first quarter of 2024, compared to $26.8 million for the comparable period in 2023. The increase in R&D expenses was primarily due to an increase in barzolvolimab clinical trial and personnel expenses, partially offset by a decrease in barzolvolimab contract manufacturing expenses.

G&A Expenses: General and administrative (G&A) expenses were $9.1 million in the first quarter of 2024, compared to $6.6 million for the comparable period in 2023. The increase in G&A expenses was primarily due to an increase in stock-based compensation and barzolvolimab commercial planning expenses.

Net Loss: Net loss was $32.8 million, or ($0.56) per share, for the first quarter of 2024, compared to a net loss of $29.4 million, or ($0.62) per share, for the comparable period in 2023.

Financial Guidance: Celldex believes that the cash, cash equivalents and marketable securities at March 31, 2024 are sufficient to meet estimated working capital requirements and fund current planned operations through 2027.