On August 6, 2024 Corvus Pharmaceuticals, Inc. (Corvus or the Company) (Nasdaq: CRVS), a clinical-stage biopharmaceutical company, reported a business update and announced financial results for the second quarter ended June 30, 2024 (Press release, Corvus Pharmaceuticals, AUG 6, 2024, View Source [SID1234645414]).

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"We have seen growing evidence supporting the central role of ITK in T cell biology and the significant potential of ITK inhibition as a new mechanism to treat a broad range of immune diseases and cancers," said Richard A. Miller, M.D., co-founder, president and chief executive officer of Corvus. "This includes early data from our Phase 1 clinical trial of soquelitinib in atopic dermatitis and our ongoing preclinical work exploring ITK inhibition in autoimmunity and inflammation. Based on this, we are increasingly optimistic that our ITK platform has the potential to improve clinical outcomes for a range of indications as an oral medication with an attractive tolerability profile. We have leveraged our experience in T cell lymphomas which has allowed us to gain a deeper understanding of the role of ITK in T cell biology that we can now apply to immune diseases. We remain on track to initiate a Phase 3 registrational trial in PTCL in the third quarter."

Business Update and Strategy

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

Soquelitinib for Immune Diseases

Corvus continues to enroll patients at multiple clinical sites in its randomized, placebo-controlled Phase 1 clinical 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.
Initial results, as of July 31, 2024, from three evaluable patients in the first cohort of the trial that completed the 28-day dosing regimen and follow-up visit demonstrated signs of clinical activity and corresponding changes in serum cytokine levels that are consistent with soquelitinib’s mechanism of action. These patients received a dose of 100 mg two-times a day, the lowest dose level planned for the study.
Corvus anticipates interim data from the Phase 1 clinical trial will be presented in the fourth quarter of 2024.
Recent published data from researchers at Cornell University demonstrated that ITK controls the fate of inflammatory Th17 cells. When ITK is inhibited by soquelitinib, the Th17 cells convert or switch to Treg cells that suppress inflammation. Soquelitinib treatment in an asthma model of mice with allergic airway inflammation significantly reduced the percentage of Th17 cells in the lung resulting in an increase in the ratio of Treg to Th17 cells. These studies confirm our understanding of the role of specific ITK inhibition in inflammation and are relevant to many immune diseases.
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 in immune diseases and cancers.

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 July 16, 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 six CRs (26%) and three PRs. Compared to the prior data reported as of May 3, 2024, one patient with continued tumor regression achieved a CR that previously had a PR at the first follow up visit. 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.
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. Recently, the Company received a Pediatric Waiver from the FDA indicating that it would not be required to conduct clinical trials in a pediatric population for this indication.
As recently announced, soquelitinib has received Fast Track designation for treatment of adult patients with relapsed or refractory peripheral T cell lymphoma after at least 2 lines of systemic therapy.

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 July 16, 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.

Collaboration with Kidney Cancer Research Consortium: Ciforadenant (adenosine A2a receptor inhibitor)

Corvus is collaborating with the 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 31, 2024 a total of 32 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 31, 2024, the interim analysis of the clinical trial has met the threshold for efficacy and therefore enrollment continues.
In July 2024, a new U.S. patent was issued (United States Patent No. 12,023,337) by the U.S. Patent and Trademark Office that covers the use of ciforadenant for the treatment of cancer; foreign counterparts are pending.
Partner Led Program: Mupadolimab (anti-CD73)

Angel Pharmaceuticals, Corvus’ partner in China, is enrolling patients in an expansion cohort of a Phase 1/1b clinical trial of mupadolimab in patients with relapsed non-small cell lung cancer (NSCLC). In this clinical trial, patients will receive mupadolimab monotherapy.

Financial Results

As of June 30, 2024, Corvus had cash, cash equivalents and marketable securities of $47.2 million as compared to $27.1 million as of December 31, 2023. During the quarter ended June 30, 2024, the Company completed a registered direct offering in which it sold shares of common stock, pre-funded warrants and common warrants, generating $30.3 million in net proceeds.

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 approximately $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 June 30, 2024 totaled $4.1 million compared to $4.0 million for the same period in 2023. The increase of $0.1 million was primarily due to higher clinical trial costs associated with the development of soquelitinib.

The net loss for the three months ended June 30, 2024 was $4.3 million compared to a net loss of $6.5 million for the same period in 2023. Total stock compensation expense for the three months ended June 30, 2024 was $0.8 million compared to $0.5 for the same period in 2023 and the non-cash loss from Corvus’ equity method investment in Angel Pharmaceuticals was $0.6 million for the three months ended June 30, 2024 compared to a loss of $1.3 million for the same period in 2023. In addition, the Company recorded a non-cash gain of $1.8 million from the change in fair value of its warrant liability during the three months ended June 30, 2024 due to the warrants issued in the registered direct offering.

Conference Call Details
Corvus will host a conference call and webcast today, Tuesday, August 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 second 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 August 6, 2024 Cogent Biosciences, Inc. (Nasdaq: COGT), a biotechnology company focused on developing precision therapies for genetically defined diseases, reported financial results for the second quarter ended June 30, 2024 (Press release, Cogent Biosciences, AUG 6, 2024, View Source [SID1234645413]).

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"Cogent has made tremendous progress in the first half of 2024 and we look forward to continuing to execute on our key priorities across the portfolio," said Andrew Robbins, the Company’s President and Chief Executive Officer. "All three of our bezuclastinib registration-directed trials remain on track and we expect to complete enrollment in our PEAK global Phase 3 trial this quarter, with topline data expected from all three trials in 2025. In parallel, our Research team continues to make excellent progress as we develop next-generation programs to build out our pipeline."

Recent Business Highlights

Announced alignment with the U.S. Food and Drug Administration (FDA) on the Company’s novel patient reported outcome measure, Mastocytosis Symptom Severity Daily Diary (MS2D2), for use in Part 2 of the registration-directed SUMMIT trial evaluating bezuclastinib in Nonadvanced Systemic Mastocytosis (NonAdvSM) patients.
Announced additional clinical data from SUMMIT Part 1 at the 2024 European Hematology Association (EHA) (Free EHA Whitepaper) Congress.
As of the cutoff date, December 18, 2023, patients in Part 1 treated at the recommended dose of 100 mg bezuclastinib demonstrated >90% reductions across all markers of mast cell burden. Additional data also showed meaningful reduction in symptom severity and objective measures of disease, including:
Substantial reduction in mast cell reactions (>50%) and patients’ most severe symptoms as measured by MS2D2.
Clinically meaningful reduction in all individual MS2D2 TSS symptoms and domains, as well as additional symptoms including dizziness, diarrhea severity, and brain fog.
Clinically meaningful improvement in skin symptoms as well as objective reduction in skin lesions.
Consistent with results previously reported, the recommended dose of 100 mg demonstrates a favorable safety and tolerability profile.
Presented positive lead-in data from the ongoing Phase 3 PEAK trial at the 2024 ASCO (Free ASCO Whitepaper) annual meeting.
As of the cutoff date, April 1, 2024, 42 patients in Part 1 had been on study for a median of 15.3 months. The median progression-free survival (mPFS) during treatment with bezuclastinib and sunitinib was 10.2 months in all patients.
In a subset of patients with second-line gastrointestinal stromal tumors (GIST) with only prior imatinib, which most closely resembles patients currently enrolling in Part 2 of PEAK, the data demonstrate a mPFS of 19.4 months.
In addition, the objective response rate (ORR) in all patients treated with bezuclastinib and sunitinib was 27.5% and in the subset of second-line patients the ORR was 33.3%, per investigator assessment. Combination treatment resulted in a disease control rate of 80% in all patients and 100% in patients with prior imatinib only.
The combination of bezuclastinib and sunitinib does not appear to add to the severity of adverse events known to be associated with sunitinib monotherapy and is well-tolerated. The majority of treatment-emergent adverse events ("TEAEs") were low-grade and reversible and discontinuations due to TEAEs remain limited.
Announced a new Phase 2 clinical trial of bezuclastinib plus sunitinib in later line GIST patients, sponsored by the Sarcoma Alliance for Research through Collaboration (SARC) and in collaboration with The Life Raft Group and Dana-Farber Cancer Institute.

The open label, single-arm Phase 2 trial sponsored by SARC and in collaboration with The Life Raft Group and Dana-Farber Cancer Institute is designed to evaluate the mPFS as well as the safety and tolerability of bezuclastinib plus sunitinib in 40 patients with GIST who have previously progressed on sunitinib.
Appointed Cole Pinnow Chief Commercial Officer.
Mr. Pinnow joined Cogent from Pfizer, where he held increasing roles of responsibility, including President and Managing Director of Pfizer Canada and most recently as Global Franchise Lead, Genitourinary and Breast Cancer Business where he led a global commercial team accountable for the growth of a $5B innovative cancer portfolio in prostate, kidney, bladder and breast therapies. Prior to Pfizer, he held several leadership roles with Hospira and founded the company’s commercial development organization. Mr. Pinnow began his pharmaceutical career as a scientist at Abbott Laboratories.
Initiated IND-enabling studies for the potent, selective CNS-penetrant ErbB2 program following presentation of new preclinical data at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2024 Annual Meeting.
The poster described CGT4255’s exceptional stability in human whole blood and liver cytosol fractions and high oral bioavailability and low clearance across preclinical species.
In addition, CGT4255 demonstrated 80% brain penetrance in mice and was well-tolerated at 10 fold maximally efficacious concentration, resulting in mouse tumor regression, suggesting potential best-in class performance.
Anticipated Upcoming Milestones

Complete enrollment in the global, Phase 3 PEAK trial in patients with GIST in Q3 2024.
Provide additional safety, tolerability, and patient-reported outcomes data from the open label extension portion of SUMMIT Part 1 by the end of 2024.
Complete enrollment in the registration-directed APEX Phase 2 trial in patients with Advanced Systemic Mastocytosis (AdvSM) by the end of 2024 and report top-line results by mid-2025.
Complete enrollment in SUMMIT Part 2 in the second quarter of 2025 and deliver top-line results by the end of 2025.
Initiate a Phase 1 trial of the first Cogent-discovered pipeline program, designed as a potent, selective, reversible FGFR2 inhibitor with best-in-class potential in the second half of 2024.
Select lead candidate and initiate IND-enabling studies from ongoing PI3Kα program, designed to potently and selectively target the H1047R driver mutation, which affects >30,000 cancer patients each year.
Second Quarter 2024 Financial Results

Cash Position: As of June 30, 2024, cash, cash equivalents and marketable securities were $389.9 million, as compared to $435.7 million as of March 31, 2024. The company expects its existing cash, cash equivalents and marketable securities will be sufficient to fund its operating expenses and capital expenditure requirements into 2027 and through clinical readouts from ongoing SUMMIT, PEAK, and APEX registration-directed trials.

R&D Expenses: Research and development expenses were $54.3 million for the second quarter of 2024 as compared to $38.9 million for the second quarter of 2023. The increase was primarily due to costs associated with accelerating enrollment in both SUMMIT and PEAK clinical trials, on-going APEX costs and costs related to development of the research pipeline. R&D expenses include non-cash stock compensation expense of $4.7 million for the second quarter of 2024 as compared to $3.5 million for the second quarter of 2023. In the quarter, an additional $4.5 million was incurred to support sunitinib clinical supply for the PEAK trial due to faster than expected enrollment.

G&A Expenses: General and administrative expenses were $10.1 million for the second quarter of 2024 as compared to $8.2 million for the second quarter of 2023. The increase was primarily due to the growth of the organization. G&A expenses include non-cash stock compensation expense of $5.3 million for the second quarter of 2024 as compared to $3.6 million for the second quarter of 2023.

Net Loss: Net loss was $59.0 million for the second quarter of 2024 as compared to a net loss of $44.1 million for the same period of 2023.

On August 6, 2024 Caribou Biosciences, Inc. (Nasdaq: CRBU), a leading clinical-stage CRISPR genome-editing biopharmaceutical company, reported financial results for the second quarter 2024 and reviewed recent pipeline progress (Press release, Caribou Biosciences, AUG 6, 2024, View Source [SID1234645412]).

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"We are advancing our lead off-the-shelf CAR-T cell therapy, CB-010, in the ANTLER phase 1 trial with a partial HLA matching strategy with the objective of developing an allogeneic CAR-T cell therapy that can meaningfully rival the autologous CAR-T cell therapies," said Rachel Haurwitz, PhD, Caribou’s president and chief executive officer. "We are enrolling approximately 20 second-line and 10 prior CD19 relapsed LBCL patients, and we plan to present initial data for both patient cohorts in the first half of 2025. For CB-011, we expect to report initial dose escalation data in patients with relapsed or refractory multiple myeloma by the end of this year. For CB-012, dose level 1 was cleared, and we are enrolling patients at dose level 2 in the AMpLify Phase 1 trial. We continue to focus our efforts and resources on rapidly advancing our four oncology and autoimmune disease clinical-stage programs through multiple clinical data milestones expected in 2024 and 2025."
Clinical highlights
CB-010, a clinical-stage allogeneic anti-CD19 CAR-T cell therapy for B cell non-Hodgkin lymphoma
•In June 2024, Caribou presented clinical data from the ongoing ANTLER Phase 1 clinical trial that indicate a single dose of CB-010 has the potential to rival the safety and efficacy of approved autologous CAR-T cell therapies. The clinical results were presented during a poster presentation (View Source) at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting.
•At ASCO (Free ASCO Whitepaper), Caribou presented data on the first 46 patients enrolled in ANTLER. Three dose levels of CB-010 were evaluated (40×106, 80×106, and 120×106 CAR-T cells) and 80×106 CAR-T cells was selected as the recommended Phase 2 dose (RP2D). In dose escalation, 16 patients with multiple subtypes of aggressive relapsed or refractory B cell non-Hodgkin lymphoma (r/r B-NHL) were enrolled, and, in dose expansion, 30 patients with second-line large B cell lymphoma (2L LBCL) were enrolled. As of the April 1, 2024 data cutoff date, results demonstrated:
◦CB-010 was generally well tolerated. No Grade 3 or higher cytokine release syndrome (CRS) and no graft-versus-host disease (GvHD) was observed.
◦A retrospective analysis of all patient data demonstrated that patients who received a dose of CB-010 manufactured from a healthy donor who shared four or more matching human leukocyte antigen ("HLA") alleles with the patient (referred to as partial HLA matching) showed the potential for improved efficacy.
◦Pharmacokinetic (PK) data showed that partial HLA matching correlated with increased CAR-T cell expansion and persistence. Pharmacodynamic (PD) data showed extended B cell aplasia and rapid recovery of patients’ endogenous T and NK cells.
•Based on these data, Caribou has begun dosing a cohort of approximately 20 2L LBCL patients to prospectively confirm that partial HLA matching may improve patient outcomes.
•Caribou also is enrolling a cohort of up to 10 patients who have relapsed following any prior CD19-targeted therapy in a proof-of-concept cohort in this population of unmet need. This cohort will also incorporate partial HLA matching between donors and patients.
•Caribou plans to initiate a pivotal Phase 3 trial in the second half of 2025, should data confirm improved outcomes for patients receiving a partially HLA matched dose of CB-010 and following agreement with the FDA on a pivotal trial design.

CB-010, a clinical-stage allogeneic anti-CD19 CAR-T cell therapy for lupus
•Caribou plans to initiate the GALLOP Phase 1 clinical trial to evaluate a single infusion of CB-010 in adult patients with lupus nephritis (LN) and extrarenal lupus (ERL). The trial will incorporate partial HLA matching between donors and patients.
•Caribou plans to initiate the GALLOP Phase 1 clinical trial in adult patients with LN and ERL by year-end 2024.

CB-011, a clinical-stage allogeneic anti-BCMA CAR-T cell therapy for multiple myeloma
•Caribou is enrolling patients with relapsed or refractory multiple myeloma (r/r MM) in the dose escalation portion of the ongoing CaMMouflage Phase 1 clinical trial (View Source).
•Caribou plans to present initial dose escalation data from the ongoing CaMMouflage Phase 1 clinical trial by year-end 2024.

CB-012, a clinical-stage allogeneic anti-CLL-1 CAR-T cell therapy for acute myeloid leukemia
•In June 2024, a poster (View Source) was presented at ASCO (Free ASCO Whitepaper) on the AMpLify Phase 1 trial design for CB-012 in adults with relapsed or refractory acute myeloid leukemia (r/r AML).
•Caribou is enrolling patients with r/r AML in the dose escalation portion of the ongoing AMpLify Phase 1 clinical trial (View Source?term=cb-012&" target="_blank" title="View Source?term=cb-012&" rel="nofollow">View Source;rank=1&tab=table). Enrollment has concluded for dose level 1 (25×106 CAR-T cells, N=3) and patients are being enrolled at dose level 2 (75×106 CAR-T cells).
Corporate updates
Appointed autoimmune expert to Caribou’s scientific advisory board (View Source)
•In July 2024, Terri Laufer, MD, was appointed to Caribou’s scientific advisory board. Dr. Laufer is a leading rheumatologist known for her extensive research into immune cell regulation and dysfunction that leads to autoimmune diseases. She is an emeritus associate professor of medicine at the Perelman School of Medicine at the University of Pennsylvania and an attending rheumatologist at the Penn Presbyterian Medical Center and Philadelphia VA Medical Center.

Extended cash runway into H2 2026
•In July 2024, Caribou discontinued the preclinical research activities associated with its allogeneic CAR-NK platform and reduced its workforce by approximately 12%. The workforce reduction, together with other cost containment measures, are expected to extend the cash runway by at least 6 months, into H2 2026. The Company will incur approximately $0.5 million to $1.0 million in one-time costs consisting primarily of cash severance costs, benefits, and transition support services for impacted employees.
Anticipated milestones
•CB-010 ANTLER: Caribou plans to present initial data from both the additional HLA-matched 2L and prior CD19 relapsed LBCL patient cohorts in H1 2025. Caribou plans to initiate a pivotal Phase 3 clinical trial in H2 2025 should data confirm improved outcomes for patients receiving a partially HLA matched dose of CB-010.
•CB-010 GALLOP: Caribou plans to initiate the GALLOP Phase 1 clinical trial in adult patients with LN and ERL by year-end 2024.
•CB-011 CaMMouflage: Caribou plans to present initial dose escalation data from the ongoing CaMMouflage Phase 1 clinical trial by year-end 2024.
•CB-012 AMpLify: Caribou plans to provide updates on dose escalation as the AMpLify Phase 1 clinical trial in r/r AML advances.
Second quarter 2024 financial results
Cash, cash equivalents, and marketable securities: Caribou had $311.8 million in cash, cash equivalents, and marketable securities as of June 30, 2024, compared to $372.4 million as of December 31, 2023. Caribou expects these cash, cash equivalents, and marketable securities will be sufficient to fund its current operating plan into H2 2026.

Licensing and collaboration revenue: Revenue from Caribou’s licensing and collaboration agreements was $3.5 million for the three months ended June 30, 2024, compared to $3.8 million for the same period in 2023. The decrease was primarily due to the now-terminated AbbVie Collaboration and License Agreement, partially offset by an increase in revenue recognized under the Information Rights Agreement Caribou entered into with Pfizer on June 29, 2023. Licensing and collaboration revenue for the three months ended June 30, 2024, includes $1.6 million in a one-time receipt of non-cash equity consideration from one of Caribou’s licensees.
R&D expenses: Research and development expenses were $35.5 million for the three months ended June 30, 2024, compared to $26.5 million for the same period in 2023. The increase was primarily due to costs to advance pipeline programs, including the CB-010 ANTLER, CB-011 CaMMouflage, and CB-012 AMpLify Phase 1 clinical trials; personnel-related expenses, including stock-based compensation, due to headcount increases; and facilities and other allocated expenses.

G&A expenses: General and administrative expenses were $11.5 million for the three months ended June 30, 2024, compared to $10.1 million for the same period in 2023. The increase was primarily due to personnel-related expenses, including stock-based compensation, due to headcount increases, and legal expenses and other service-related expenses. These increases were partially offset by a decrease in patent prosecution and maintenance fees.

Net loss: Caribou reported a net loss of $37.7 million for the three months ended June 30, 2024, compared to $29.5 million for the same period in 2023.
About CB-010
CB-010 is the lead clinical-stage product candidate from Caribou’s allogeneic CAR-T cell therapy platform, and it is being evaluated in patients with relapsed or refractory B cell non-Hodgkin lymphoma (r/r B-NHL) in the ongoing ANTLER Phase 1 clinical trial and will be evaluated in patients with lupus nephritis (LN) and extrarenal lupus (ERL) in the GALLOP Phase 1 clinical trial. In the ANTLER clinical trial, Caribou is enrolling second-line (2L) patients with large B cell lymphoma (LBCL) comprised of different subtypes of aggressive r/r B-NHL (DLBCL NOS, PMBCL, HGBL, tFL, and tMZL) who have never received prior CD19-targeted therapy as well as LBCL patients who have relapsed on a prior CD19-targeted therapy. To Caribou’s knowledge, CB-010 is the first allogeneic CAR-T cell therapy in the clinic with a PD-1 knockout, a genome-editing strategy designed to improve activity against diseases by limiting premature CAR-T cell exhaustion. CB-010 is also, to Caribou’s knowledge, the first anti-CD19 allogeneic CAR-T cell therapy to be evaluated in the 2L LBCL setting and, for r/r B-NHL, CB-010 has been granted Regenerative Medicine Advanced Therapy (RMAT), Fast Track, and Orphan Drug designations by the FDA. Additional information on the ANTLER trial (NCT04637763) can be found at clinicaltrials.gov (View Source).
About CB-011
CB-011 is a product candidate from Caribou’s allogeneic CAR-T cell therapy platform and is being evaluated in patients with relapsed or refractory multiple myeloma (r/r MM) in the CaMMouflage Phase 1 trial. CB-011 is an allogeneic anti-BCMA CAR-T cell therapy engineered using Cas12a chRDNA genome-editing technology. To Caribou’s knowledge, CB-011 is the first allogeneic CAR-T cell therapy in the clinic that is engineered to improve antitumor activity through an immune cloaking strategy with a B2M knockout and insertion of a B2M–HLA-E fusion protein to blunt immune-mediated rejection. CB-011 has been granted Fast Track and orphan drug designations by the FDA. Additional information on the CaMMouflage trial (NCT05722418) can be found at clinicaltrials.gov (View Source).

About CB-012
CB-012 is a product candidate from Caribou’s allogeneic CAR-T cell therapy platform and is being evaluated in the AMpLify Phase 1 clinical trial in patients with relapsed or refractory acute myeloid leukemia (r/r AML). CB-012 is an anti-CLL-1 CAR-T cell therapy engineered with five genome edits, enabled by Caribou’s patented next-generation CRISPR technology platform, which uses Cas12a chRDNA genome editing to significantly improve the specificity of genome edits. To Caribou’s knowledge, CB-012 is the first allogeneic CAR-T cell therapy with both checkpoint disruption, through a PD-1 knockout, and immune cloaking, through a B2M knockout and B2M–HLA-E fusion protein insertion; both armoring strategies are designed to improve antitumor activity.
Caribou has exclusively in-licensed from Memorial Sloan Kettering Cancer Center (MSKCC) in the field of allogeneic CLL-1-targeted cell therapy a panel of fully human scFvs targeting CLL-1, from which the company has selected a scFv for the generation of the company’s CAR.
Additional information on the AMpLify trial (NCT06128044) can be found at clinicaltrials.gov (View Source).

On August 6, 2024 Black Diamond Therapeutics, Inc. (Nasdaq: BDTX), a clinical-stage oncology company developing MasterKey therapies that target families of oncogenic mutations in patients with cancer, reported financial results for the second quarter ended June 30, 2024, and provided a corporate update (Press release, Black Diamond Therapeutics, AUG 6, 2024, View Source [SID1234645411]).

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"We continue to execute on enrollment of patients with EGFR mutant NSCLC into second/third-line and first-line Phase 2 cohorts, and remain on track to announce initial results later in the third quarter of this year and in the first quarter of 2025, respectively", said Mark Velleca, M.D., Ph.D., Chief Executive Officer of Black Diamond Therapeutics. "We also look forward to sharing analyses of real world data at the 2024 ESMO (Free ESMO Whitepaper) Congress in September 2024 on treatment practices and therapeutic outcomes for newly diagnosed NSCLC patients with non-classical EGFR mutations that demonstrate a significant unmet medical need."

Recent Developments & Upcoming Milestones:

BDTX-1535:

In April 2024, Black Diamond described real world evidence of the evolving EGFR mutation landscape in patients with non-small cell lung cancer (NSCLC) and the potential of BDTX-1535 to address a broader range of mutations compared to existing therapies at the 2024 American Association for Cancer Research (AACR) (Free AACR Whitepaper) annual meeting. The analyses revealed a spectrum of previously underappreciated non-classical mutations, as well as an increased prevalence of the acquired resistance mutation C797S. These non-classical EGFR mutations were present in 20-30% of newly diagnosed epidermal growth factor receptor mutation positive (EGFRm) NSCLC patients.
In June 2024, Black Diamond presented additional data from the Phase 1 dose escalation trial of BDTX-1535 in patients with relapsed/recurrent glioblastoma (GBM), and initial intratumoral pharmacokinetic data from a "window of opportunity" (also known as a Phase 0/1 "Trigger") trial sponsored by the Ivy Brain Tumor Center, in patients with recurrent high-grade glioma (HGG), at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. Safety and tolerability data in the Phase 1 trial were consistent with BDTX-1535 clinical data in patients with NSCLC previously presented in October 2023 at the European Organization for Research and Treatment of Cancer-National Cancer Institute-American Association for Cancer Research (AACR-NCI-EORTC) (Free AACR-NCI-EORTC Whitepaper) AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper). Among 19 efficacy evaluable patients, several experienced stable disease with promising durability. Results from the investigator-sponsored trial demonstrated that BDTX-1535 penetrates the blood brain barrier with clinically meaningful unbound drug concentration in gadolinium non-enhancing regions of the brain and inhibition of corresponding pharmacodynamic markers. Eight out of nine patients exceeded the pre-specified threshold for drug concentration in the brain tumor tissue and continued on study.
Black Diamond anticipates the following upcoming key milestones for BDTX-1535:
Disclosure of initial Phase 2 data in 2L/3L EGFRm NSCLC patients with non-classical mutations or the acquired resistance C797S mutation remains on track for later in Q3 2024.
Disclosure of initial Phase 2 data in 1L EGFRm NSCLC patients with non-classical mutations remains on track for Q1 2025 (NCT05256290).
An abstract has been accepted for presentation at the 2024 European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress titled "Real World Evidence of Treatment Practices and Therapeutic Outcomes for Newly Diagnosed NSCLC Patients with Non-classical EGFR Mutations Demonstrates High Unmet Medical Need", which will detail an analysis of Guardant Inform data on treatment outcomes for newly diagnosed NSCLC patients with tumors expressing non-classical mutations.
BDTX-4933:

BDTX-4933 is a brain-penetrant oral inhibitor of oncogenic alterations in KRAS, NRAS and BRAF.
Enrollment of patients with BRAF and select RAS/MAPK mutation-positive cancers, with an emphasis on patients with KRAS mutant NSCLC, is progressing through escalating doses in a Phase 1 trial (NCT05786924). An update from this trial is on track for Q4 2024.
Corporate

Chief Business Officer & Chief Financial Officer, Fang Ni, Pharm.D, will participate in a panel discussion at the Wedbush PacGrow Healthcare Conference taking place August 13-14, 2024, in New York, NY.
Financial Highlights

Cash Position: Black Diamond ended the second quarter of 2024 with approximately $123.0 million in cash, cash equivalents, and investments compared to $131.4 million as of December 31, 2023. Net cash used in operations was $14.7 million for the second quarter of 2024 compared to $14.4 million for the second quarter of 2023.
Research and Development Expenses: Research and development (R&D) expenses were $12.6 million for the second quarter of 2024, compared to $13.2 million for the same period in 2023. The decrease in R&D expenses was primarily due to workforce efficiencies and reduced spending on early discovery projects.
General and Administrative Expenses: General and administrative (G&A) expenses were $9.6 million for the second quarter of 2024, compared to $6.9 million for the same period in 2023. The increase in G&A expenses was primarily due to an increase in consulting and other professional fees.
Net Loss: Net loss for the second quarter of 2024 was $19.9 million, as compared to $19.2 million for the same period in 2023.
Financial Guidance

Black Diamond ended the second quarter of 2024 with approximately $123.0 million in cash, cash equivalents and investments which the Company believes is sufficient to fund its anticipated operating expenses and capital expenditure requirements into the fourth quarter of 2025.

On August 6, 2024 BioXcel Therapeutics, Inc. (Nasdaq: BTAI), a biopharmaceutical company utilizing artificial intelligence to develop transformative medicines in neuroscience and immuno-oncology, reported its financial results for the second quarter of 2024 (Press release, BioXcel Therapeutics, AUG 6, 2024, View Source [SID1234645410]).

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"We are on track with our business priorities as we focus on bringing BXCL501 to the greatest number of patients in need," said Vimal Mehta, Ph.D., CEO of BioXcel Therapeutics. "We are pleased with the progress with our SERENITY and TRANQUILITY programs and our focused market-access strategy for IGALMI. Our confidence in our lead neuroscience asset is underpinned by its broad therapeutic potential across multiple neuropsychiatric conditions and its growing intellectual property portfolio."

Late-Stage Clinical Programs

· SERENITY At-Home* Pivotal Phase 3 Trial: designed to evaluate the safety of a 120 mcg dose of BXCL501 in the at-home setting for agitation associated with bipolar disorders or schizophrenia.

o Recently received feedback on protocol from U.S. Food and Drug Administration (FDA).

· TRANQUILITY In-Care Pivotal Phase 3 Trial: designed to evaluate the efficacy and safety of a 60 mcg dose of BXCL501 for agitation associated with Alzheimer’s dementia (AAD).

o Protocol being finalized for planned submission to FDA.

IGALMI (dexmedetomidine) Sublingual film

Post-marketing Requirement (PMR) Study

· Reported positive topline results from PMR study evaluating PRN (as-needed) treatment of IGALMI for agitation associated with bipolar disorders or schizophrenia.

o Study achieved its objective and demonstrated no evidence of tachyphylaxis, tolerance, or withdrawal with 180 mcg dose (highest approved dose).

o Although this PMR study was not statistically powered to evaluate repeat dose efficacy, a reduction in agitation was observed for each episode occurring during the seven-day study period, and no serious adverse events were reported following treatment.

Commercialization

· IGALMI net revenue grew 90% in Q2 2024 over Q1 2024 driven by focused market-access strategy and increased contracting with psychiatric care clinics and behavioral health facilities using a small commercial team.

Patent Portfolio

The Company continues to strengthen its intellectual property portfolio for IGALMI.

· Recently received a U.S. Patent and Trademark Office (USPTO) Notice of Allowance for U.S. Patent Application No. 18/526,686 for IGALMI. Once issued by the USPTO, the patent is expected to have an expiration date of January 12, 2043, and will be submitted for listing in the FDA’s Approved Drug Products with Therapeutic Equivalence Evaluations (commonly known as the "Orange Book").

· This is expected to be the 11th listed U.S. patent for IGALMI in the Orange Book.