On November 2, 2023 Curis, Inc. (NASDAQ: CRIS), a biotechnology company focused on the development of emavusertib, an orally available, small molecule IRAK4 inhibitor for the treatment of hematologic malignancies, reported its business update and financial results for the quarter ended September 30, 2023 (Press release, Curis, NOV 2, 2023, View Source [SID1234636752]).

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"Since the removal of the partial clinical hold, we have re-started patient enrollment in both the Phase 1/2 TakeAim Leukemia monotherapy and TakeAim Lymphoma combination studies and expect to initiate a triplet combination study in AML in the fourth quarter. We also expect to present updated data from the TakeAim Lymphoma combination study at ASH (Free ASH Whitepaper) in December," said James Dentzer, President and CEO of Curis.

Third Quarter 2023 and Recent Operational Highlights

Emavusertib (IRAK4 Inhibitor)

TakeAim Leukemia

Curis is currently enrolling relapsed or refractory (R/R) AML/MDS patients with FLT3 or spliceosome mutation (U2AF1 or SF3B1) who have received < 3 prior lines of treatment. The Company expects additional data from this study in the first half of 2024.
The Company expects to initiate a triplet combination study of emavusertib with azacitidine and venetoclax in the fourth quarter. This Phase 1 study is intended to evaluate emavusertib’s safety, as well as its ability enhance the effectiveness of azacitidine and venetoclax. Initial data from this study is expected in the second half of 2024.
TakeAim Lymphoma

Curis is focusing its lymphoma clinical development efforts on Primary CNS lymphoma (PCNSL), a rare form of extranodal non-Hodgkin lymphoma for which there are limited treatment options. The Company is currently enrolling PCNSL patients in its TakeAim Lymphoma study in which patients are being treated with a combination of emavusertib and ibrutinib. The Company will present updated data from the study at the 65th ASH (Free ASH Whitepaper) Annual Meeting December 9-12, 2023, which will include a PCNSL patient subset.
Upcoming Milestones

Curis expects updated clinical data from the TakeAim Lymphoma combination study with ibrutinib at the ASH (Free ASH Whitepaper) Annual Meeting.
The Company expects updated clinical data from the TakeAim Leukemia monotherapy study in the first half of 2024.
The Company expects initial clinical data from the triplet combination study of emavusertib with azacitidine and venetoclax to treat AML patients in the second half of 2024.
Corporate

In July 2023, Curis completed a registered direct offering with net proceeds of approximately $13.8 million.

On September 28, 2023, the Company effected a 1-for-20 reverse stock split of its common stock.

Third Quarter 2023 Financial Results

For the third quarter of 2023, Curis reported a net loss of $12.2 million or $2.13 per share on both a basic and diluted basis as compared to $13.3 million or $2.83 per share on both a basic and diluted basis, for the same period in 2022. Curis reported a net loss of $35.7 million or $6.96 per share on both a basic and diluted basis, for the nine months ended September 30, 2023 as compared to a net loss of $45.3 million or $9.82 per share on both a basic and diluted basis for the same period in 2022.

Revenues for the third quarters of 2023 and 2022 were both $2.8 million. Revenues for both periods consist of royalty revenues from Genentech and Roche’s sales of Erivedge. Revenues for the nine months ended September 30, 2023 and 2022 were both $7.3 million.

Research and development expenses were $10.4 million for the third quarter of 2023, as compared to $10.8 million for the same period in 2022. The decrease was primarily attributable to lower employee-related costs due to a reduction in headcount, partially offset by increased clinical costs. Research and development expenses were $29.5 million for the nine months ended September 30, 2023, as compared to $34.6 million for the same period in 2022.

General and administrative expenses were $4.8 million for the third quarter of 2023, as compared to $4.6 million for the same period in 2022. The increase was primarily attributable to higher professional, legal, and consulting services costs. General and administrative expenses were $13.8 million for the nine months ended September 30, 2023, as compared to $15.3 million for the same period in 2022.

Other income, net was $0.2 million for the third quarter of 2023, as compared to other expense, net of $0.7 million for the same period in 2022. Other income (expense), net primarily consisted of interest income partially offset by non-cash expense related to the sale of future royalties. Other income, net was $0.4 million for the nine months ended September 30, 2023 compared to other expense, net $2.5 million for the same period in 2022.

Curis’s cash, cash equivalents and investments totaled $68.5 million, and the Company had approximately 5.9 million shares of common stock outstanding. Curis expects its existing cash, cash equivalents and investments to enable its planned operations into 2025.

Conference Call Information

Curis management will host a conference call today, November 2, 2023, at 8:30 a.m. ET, to discuss the business update and these financial results.

To access the live conference call, please dial 1-888-390-0546 from the United States or 1-416-764-8688 from other locations, shortly before 8:30 a.m. ET. The conference call can also be accessed on the Curis website in the Investors section.

On November 2, 2023 Corvus Pharmaceuticals, Inc. (NASDAQ: CRVS), a clinical-stage biopharmaceutical company, reported that it will present new interim data from its Phase 1/1b trial of soquelitinib in patients with relapsed peripheral T cell lymphoma (PTCL), at the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition, which is taking place in-person and virtually from December 9-12, 2023 (Press release, Corvus Pharmaceuticals, NOV 2, 2023, View Source [SID1234636751]).

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Details regarding the poster presentation, which will be available in the poster hall and via the virtual event platform, are as follows:

Date and Time: Saturday, December 9, 2023, 5:30 PM PT

Title: Dynamic Single-Cell Profiling Reveals Novel Immune Regulatory Mechanism of ITK Inhibitor Soquelitinib in Refractory T Cell Lymphoma

Abstract #: 1442

Presenter: Dr. Ning Ding, Peking University Cancer Hospital & Institute, Beijing, China

On November 2, 2023 Cogent Biosciences, Inc. (Nasdaq: COGT), a biotechnology company focused on developing precision therapies for genetically defined diseases, reported recent business highlights and financial results for the third quarter ended September 30, 2023 (Press release, Cogent Biosciences, NOV 2, 2023, View Source [SID1234636749]).

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"This quarter was marked by meaningful progress as we furthered our efforts developing bezuclastinib for the AdvSM, NonAdvSM and GIST patient populations," said Andrew Robbins, Cogent’s President and Chief Executive Officer. "We look forward to the opportunity to present clinical data from both SUMMIT, selected for an oral presentation, and APEX clinical trials at the 2023 American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting in December, and are pleased with the updated PEAK lead-in data we are sharing at the 2023 Connective Tissue Oncology Society (CTOS) annual meeting this weekend. With these important advances coupled with our cash runway into 2026, we believe we are well positioned to further build on our momentum to bring best-in-class therapies to patients with genetically defined diseases."

Business Highlights & Milestones

Oral presentation of initial clinical data from SUMMIT Part 1a at the ASH (Free ASH Whitepaper) annual meeting on Saturday, December 9, 2023. SUMMIT is a randomized, global, multicenter, double-blind, placebo-controlled, multi-part Phase 2 trial evaluating bezuclastinib in patients with nonadvanced systemic mastocytosis (NonAdvSM). Cogent will host an investor webcast to review the results on Monday, December 11, 2023 at 8:00 a.m. ET. Details will be provided closer to the event.
Presentation will include safety, tolerability, pharmacokinetics, biomarker and symptomatic improvement results from the 20 patients enrolled in SUMMIT Part 1a.
Measures of symptomatic improvement will include clinical data collected from multiple patient reported outcome measures including MAS, MCQoL, PGIS and PGIC.
SUMMIT Part 1 enrollment completed in Q3 2023, including over-enrollment at 54 patients across Part 1a and Part 1b. Clinical results from the entire Part 1 patient population are planned for presentation at a scientific conference in Q1 2024.
SUMMIT Part 2, a registration-directed, global, randomized placebo-controlled trial is on track for initiation in 1H 2024, ahead of schedule.
Updated clinical data from the lead-in portion of the ongoing PEAK Phase 3 study to be shared in an oral presentation at the 2023 CTOS annual meeting on November 4, 2023. PEAK is an ongoing, multi-part Phase 3 randomized, global, multicenter trial evaluating bezuclastinib in combination with sunitinib in patients with imatinib-resistant gastrointestinal stromal tumors (GIST).
Safety and tolerability data from 42 patients enrolled in Part 1a and Part 1b are consistent with results shared at the 2023 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting, demonstrating the combination of bezuclastinib and sunitinib was well tolerated and the adverse event profile was similar to sunitinib monotherapy.
Updated clinical activity from a subset of 2nd-line GIST patients demonstrates a 33% confirmed overall response rate (ORR) with ongoing median duration of therapy greater than 14 months. Together with clinical data previously reported from a Phase 1/2 trial, 4 of 10 evaluable 2nd-line GIST patients treated with the combination have reached confirmed partial response status.
Phase 3 portion of the PEAK study remains on track to complete enrollment by the end of 2024, with over 100 active sites globally.
Updated clinical results from Part 1 of the ongoing APEX study to be presented at the 2023 ASH (Free ASH Whitepaper) annual meeting on Monday, December 11, 2023. APEX is a global, multi-part Phase 2 trial evaluating bezuclastinib in patients with advanced systemic mastocytosis (AdvSM).
Presentation will include safety, tolerability, pharmacokinetics, biomarker and response assessments from 33 patients enrolled in APEX Part 1.
APEX Part 2 is on track to complete enrollment by the end of 2024. An additional APEX cohort was initiated in Q3 and is designed to allow concomitant administration of bezuclastinib with azacitadine in patients with systemic mastocytosis with an associated hematologic neoplasm (SM-AHN).
Presented preclinical data at the 2023 ENA annual meeting on the Company’s next-generation, reversible, non-covalent fibroblast growth factor receptor 2 (FGFR2) program, which exhibited low nanomolar potency on WT FGFR2 and FGFR2 mutations and is selective against the kinome and a panel of channels and receptors.
Exploratory pharmacokinetics (PK) studies conducted across species showed CGT4859 to be a low-clearance compound with high oral bioavailability. Further, in a mutant-driven mouse model, CGT4859 demonstrated dose-responsive tumor growth inhibition with complete regressions at 5 mg/kg PO and was well-tolerated. Cogent anticipates filing an Investigational New Drug (IND) Application and beginning a clinical trial in 2024.
Upcoming Scientific Presentations

2023 ASH (Free ASH Whitepaper) – Oral Presentation
Title: Initial Results from Summit: An Ongoing, 3-Part, Multi-Center, Randomized, Double-Blind, Placebo-Controlled Phase 2 Clinical Study of Bezuclastinib in Adult Patients with NonAdvanced Systemic Mastocytosis (NonAdvSM)
Date: Saturday, December 9, 2023
Session Time: 9:30 a.m. – 11:00 a.m. PT/12:30 p.m. – 2:00 p.m. ET
Presenter: Dr. Prithviraj Bose, MD Anderson Cancer Center, Houston, Texas
2023 CTOS – Oral Presentation
Title: Peak Study: A Phase 3, Randomized, Open-Label, Multicenter Clinical Study of Bezuclastinib (CGT9486) and Sunitinib Combination Versus Sunitinib in Patients with Gastrointestinal Stromal Tumors (GIST)
Date: November 4, 2023
Session Time: 9:00 a.m. – 10:00 a.m. GMT/5:00 a.m. – 6:00 a.m. ET
Presenter: Dr. Neeta Somaiah, MD Anderson Cancer Center, Houston, Texas
2023 ASH (Free ASH Whitepaper) – Poster Presentation
Title: Safety and Efficacy of Bezuclastinib (CGT9486), a Novel, Highly Selective, Potent KIT D816V Tyrosine Kinase Inhibitor, in Patients with Advanced Systemic Mastocytosis (AdvSM): Results From Part 1 of the Phase 2 Apex Trial
Date: Monday, December 11, 2023
Session Time: 6:00 p.m. – 8:00 p.m. PT/9:00 p.m. – 11:00 p.m. ET
Presenter: Dr. Pankit Vachhani, University of Alabama, Birmingham
Upcoming Investor Conferences
A live webcast of the following events can be accessed on the Investors & Media page of Cogent’s website at investors.cogentbio.com/events. A replay will be available approximately two hours after completion of the events and will be archived for up to 30 days.

Jefferies London Healthcare Conference on Wednesday, November 15, 2023 at 12:00 p.m. GMT (7:00 a.m. ET).
Piper Sandler 35th Annual Healthcare Conference on Tuesday, November 28, 2023 at 2:30 p.m. ET.
Third Quarter 2023 Financial Results

Cash Position: As of September 30, 2023, cash, cash equivalents and marketable securities were $312.8 million, as compared to $350.9 million as of June 30, 2023. The company believes that its cash, cash equivalents and marketable securities will be sufficient to fund its operating expenses and capital expenditure requirements into 2026.

R&D Expenses: Research and development expenses were $50.1 million for the third quarter of 2023 as compared to $29.9 million for the third quarter of 2022. R&D expenses include non-cash stock compensation expense of $4.0 million for the third quarter of 2023 compared to $2.1 million for the third quarter of 2022. In the quarter, $6.6 million in non-recurring charges were incurred for third party CDMOs to prepare for bezuclastinib pre-commercialization and to support sunitinib clinical supply for the PEAK study. Additional increases resulted from costs associated with the acceleration of APEX, SUMMIT and PEAK clinical trials and the continued development of our research pipeline.

G&A Expenses: General and administrative expenses were $9.5 million for the third quarter of 2023 as compared to $6.9 million for the third quarter of 2022. G&A expenses include non-cash stock compensation expense of $4.8 million for the third quarter of 2023 compared to $2.6 million for the third quarter of 2022.

Net Loss: Net loss was $55.4 million for the third quarter of 2023 as compared to a net loss of $35.1 million for the same period of 2022.

On November 2, 2023 Cerus Corporation (Nasdaq: CERS) reported its financial results for the third quarter ended September 30, 2023 (Press release, Cerus, NOV 2, 2023, View Source [SID1234636748]).

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Recent highlights include:

Third quarter 2023 total revenue of $47.3 million was comprised of total product revenue of $39.8 million and government contract revenue of $7.5 million.
Completed Phase 3 ReCePI study enrollment in cardiovascular surgery patients, with top-line data readout on track for Q1 2024.
Attended first in-person AABB Annual Meeting since 2019, where presentations from transfusion medicine leaders highlighted the growing experience with the benefits of INTERCEPT-treated products, including INTERCEPT platelets and INTERCEPT Fibrinogen Complex (IFC).
Cash, cash equivalents, and short-term investments were $79.0 million at September 30, 2023.
Reaffirming commitment to achieve non-GAAP adjusted EBITDA breakeven in the fourth quarter of 2023.
"We continued to make progress on multiple fronts in the third quarter," said William "Obi" Greenman, Cerus’ president and chief executive officer. "We completed patient enrollment in our U.S. Phase 3 ReCePI study and continue to plan for a top-line data readout from the study in the first quarter of next year."

"On the top line, product revenues in the quarter returned to prior year levels, and we expect continued growth from here through the end of the year, with the near-term growth trajectory influenced by the system-wide roll-out of INTERCEPT platelets at Canadian Blood Services," continued Greenman. "Due primarily to the timing of our recent execution of an IFC sales agreement with one of the largest U.S. producers of cryoprecipitate, we are adjusting our full-year 2023 product revenue guidance to a range of $155 million to $158 million. The growth we are expecting in the second half of this year reflects the ongoing global demand for INTERCEPT-treated blood components and the growing use of IFC by blood center and hospital customers, as evidenced at the recent AABB Annual Meeting."

Revenue

Product revenue during the third quarter of 2023 was $39.8 million, compared to $39.6 million during the prior year period.

Third-quarter 2023 government contract revenue was $7.5 million, compared to $6.8 million during the prior year period. Reported government contract revenue in the third quarter 2023 increased versus the prior year period primarily due to funding associated with development of LyoIFC as well as research and development (R&D) activities related to the INTERCEPT Blood System for Red Blood Cells. In addition to this funding, the Company’s government contract revenue was comprised of funding associated with efforts related to the development of next-generation pathogen reduction technology to treat whole blood.

Product Gross Profit & Margin

Product gross profit for the third quarter of 2023 was $21.8 million, which is consistent with the prior year period. Product gross margin for the third quarter of 2023 was 54.9% compared to 55.4% for the third quarter of 2022. The Company continues to expect stability in gross margin percentage for the balance of the year. The Company’s margin expansion efforts are ongoing with the goal of realizing further margin expansion in the future.

Operating Expenses

Total operating expenses for the third quarter of 2023 were $34.5 million compared to $36.1 million for the same period of the prior year, reflecting a year-over-year decrease of 4%.

Selling, general, and administrative (SG&A) expenses for the third quarter of 2023 totaled $16.2 million, compared to $19.9 million for the third quarter of 2022. The year-over-year decrease in SG&A expenses for the third quarter was tied to decreased headcount and decreased non-cash stock-based compensation.

R&D expenses for the third quarter of 2023 were $16.8 million, compared to $16.2 million for the third quarter of 2022. The small year-over-year increase in R&D expenses in the third quarter was tied to the development of our next-generation illuminator and increased clinical research activities.

As previously described, the Company entered into a plan to restructure certain functions and reduce its real estate footprint during the second quarter of 2023. For the third quarter of 2023, the plan resulted in an additional $1.6 million restructuring charge. Of the $1.6 million, $1.1 million primarily relates to the write off of operating lease assets* which will be paid down over the course of the operating lease, and $0.5 million relates to non-cash charges associated with leasehold improvements which were written off. The Company excludes the restructuring charge from its non-GAAP adjusted EBITDA measure presented below.

Net Loss Attributable to Cerus Corporation

Net loss attributable to Cerus Corporation for the third quarter of 2023 was $7.3 million, or $0.04 per basic and diluted share, compared to a net loss attributable to Cerus Corporation of $8.5 million, or $0.05 per basic and diluted share, for the third quarter of 2022.

On November 2, 2023 Cellectis (the "Company") (Euronext Growth: ALCLS – NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, reported that preliminary results of the Phase I NATHALI-01 clinical trial evaluating UCART20x22 in patients with relapsed or refractory non-Hodgkin lymphoma (r/r NHL) and updated results of the Phase I BALLI-01 clinical trial evaluating UCART22 in patients with relapsed or refractory CD22+ B-cell acute lymphoblastic leukemia, (r/r B-ALL) will be presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) 65th Annual Meeting (ASH 2023), that will take place on December 9-12, 2023 in San Diego (CA) and online (Press release, Cellectis, NOV 2, 2023, View Source [SID1234636747]).

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These data will be presented in two poster sessions:

Poster Presentation (P2110)

Title: Preliminary Results of Nathali-01: A First-in-Human Phase I/IIa Study of UCART20x22, a Dual Allogeneic CAR-T Cell Product Targeting CD20 and CD22, in Relapsed or Refractory (R/R) Non-Hodgkin Lymphoma (NHL)

Session Name: 704. Cellular Immunotherapies: Early Phase and Investigational Therapies: Poster I

Presenter: Dr. Jeremy Abramson (Massachusetts General Hospital Cancer Center)

Date/Time: Saturday, December 9, 2023 at 5:30 – 7:30 PM PT at San Diego Convention Center, Halls G-H

The poster presentation highlights the following data:

as of July 1, 2023, 3 patients were enrolled and treated at dose level 1 (50 million cells) with product manufactured in-house by Cellectis. Cytokine release syndrome (CRS) Grade 1 or 2 occurred in all patients, and all CRS resolved with treatment.
No immune effector cell associated neurotoxicity (ICANS) or graft versus host disease (GvHD) was observed. There were no UCART20x22 dose limiting toxicities (DLTs), and there was 1 DLT in connection with CLLS52 (alemtuzumab).
All patients responded at Day 28, with 1 partial metabolic response and 2 complete metabolic responses in patients who had failed prior autologous CD19 CAR T-cell therapies.
UCART20x22 expansion correlated with increases in serum cytokine and inflammatory marker levels as well as with CRS.
These initial data support the continued clinical trial evaluating UCART20x22 in R/R NHL.
Poster Presentation (P4847)

Title: Updated Results of the Phase I BALLI-01 Trial of UCART22 Process 2 (P2), an Anti-CD22 Allogeneic CAR-T Cell Product Manufactured By Cellectis Biologics, in Patients with Relapsed or Refractory (R/R) CD22+ B-Cell Acute Lymphoblastic Leukemia (B-ALL)

Session Name: 704. Cellular Immunotherapies: Early Phase and Investigational Therapies: Poster III

Presenter: Dr. Nitin Jain (University of Texas MD Anderson Cancer Center)

Date/Time: Monday, December 11, 2023 at 6:00 – 8:00 PM PT at San Diego Convention Center, Halls G-H

The poster presentation highlights the following data:

in vitro comparability studies suggested that UCART22 Process 2 (P2) (manufactured in-house by Cellectis) is more potent than UCART22 Process 1 (P1) (manufactured by an external CDMO), and as of July 1, 2023, 3 patients were enrolled into the first UCART22 P2 cohort at dose level 2 (1 million cells/kg).
UCART22 P2 was administered after fludarabine, cyclophosphamide, and alemtuzumab (FCA) lymphodepletion regimen and was well tolerated. No DLTs or ICANS was observed, and the CRS observed was Grade 1 or 2.
There was a higher preliminary response rate (67%) at dose level 2 (1 million cells/kg) with UCART22 P2 (manufactured in-house by Cellectis) compared to 50% at dose level 3 (5 million cells/kg) with UCART22 P1 (manufactured by an external CDMO).
UCART22 expansion was observed in the responding patients and correlated with increases in serum cytokines and inflammatory markers.
The study continues to enroll patients at dose level 2i (2.5 million cells/kg) with UCART22 P2.