On November 2, 2023 Dynavax Technologies Corporation (Nasdaq: DVAX), a commercial-stage biopharmaceutical company developing and commercializing innovative vaccines, reported financial results and provided a business update for the quarter ended September 30, 2023 (Press release, Dynavax Technologies, NOV 2, 2023, View Source [SID1234636754]).

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"We’re pleased to report yet another record quarter of HEPLISAV-B revenue driven by continued market share growth and overall expansion of the adult hepatitis B market, demonstrating progress toward our goal of establishing HEPLISAV-B as the leading adult hepatitis B vaccine in the U.S., a market opportunity we believe will continue to expand to over $800 million by 2027," said Ryan Spencer, Chief Executive Officer of Dynavax. "In addition to HEPLISAV-B, we are focused on advancing our pipeline of innovative vaccine candidates, pursuing strategic opportunities to accelerate our growth, and continuing to drive strong financial performance, reflecting the solid foundation we’ve established for sustained success."

BUSINESS UPDATES

HEPLISAV-B [Hepatitis B Vaccine (Recombinant), Adjuvanted]

HEPLISAV-B vaccine is the first and only adult hepatitis B vaccine approved in the U.S., the European Union and Great Britain that enables series completion with only two doses in one month. Hepatitis B vaccination is universally recommended for adults aged 19-59 in the U.S.

HEPLISAV-B achieved net product revenue of $62.3 million for the third quarter of 2023, an increase of 66% compared to $37.5 million for the third quarter of 2022.
HEPLISAV-B total market share in the U.S. increased to approximately 41%, compared to approximately 32% at the end of the third quarter of 2022.
HEPLISAV-B market share in the Integrated Delivery Networks (IDNs) and Large Clinics segment increased to approximately 54% at the end of the third quarter of 2023, compared to approximately 43% for the same quarter in 2022.
HEPLISAV-B market share in the retail pharmacy segment increased to approximately 53% at the end of the third quarter of 2023, compared to 43% for the same quarter in 2022.
A supplemental Biologic License Application (sBLA) for HEPLISAV-B vaccination of adults on hemodialysis is currently under review by the U.S. Food and Drug Administration (FDA) with a Prescription Drug User Fee Act (PDUFA) action date expected on May 13, 2024.
Clinical Pipeline
Dynavax is advancing a pipeline of differentiated product candidates that leverage its CpG 1018 adjuvant, which has demonstrated its ability to enhance the immune response with a favorable tolerability profile in a wide range of clinical trials and real-world commercial use.

Shingles vaccine program:
Z-1018 is an investigational vaccine candidate being developed for the prevention of shingles in adults aged 50 and older.

Dynavax recently received Type B meeting feedback from the FDA on the Z-1018 clinical development plan and expects to submit an Investigational New Drug Application (IND) to the FDA to support the initiation of a Phase 1/2 trial of Z-1018 in the first half of 2024.
Tdap vaccine program:
Tdap-1018 is an investigational vaccine candidate intended for active booster immunization against tetanus, diphtheria, and pertussis (Tdap).

Dynavax plans to submit an Investigational New Drug Application (IND) to the FDA to support the initiation of a Phase 2 human challenge study of Tdap-1018 in mid-2024.
Plague vaccine program:
Dynavax is developing a plague (rF1V) vaccine candidate adjuvanted with CpG 1018 currently in a Phase 2 clinical trial in collaboration with, and fully funded by, the U.S. Department of Defense.

Dynavax and the U.S. Department of Defense recently executed a contract modification to support advancement of the plague vaccine candidate into a nonhuman primate challenge study, which was initiated in August, with the agreement now totaling $33.7 million through 2025.
Dosing has been completed in a randomized, active-controlled Phase 2 clinical trial evaluating immunogenicity, safety, and tolerability, with top line data anticipated in 2024.
THIRD QUARTER 2023 FINANCIAL HIGHLIGHTS

Total Revenues and Net Product Revenue.

HEPLISAV-B vaccine net product revenue was $62.3 million for the third quarter of 2023, compared to $37.5 million for the third quarter of 2022, representing year-over-year growth of 66%. The increase was primarily due to higher sales volume driven by both continued improvement in market share and higher utilization of adult hepatitis B vaccines related to the ACIP universal recommendation.
Other revenue was $7.2 million for the third quarter of 2023, compared to $3.9 million in the same period of 2022. Other revenue primarily includes revenue from our plague vaccine agreement with the U.S. Department of Defense. The increase was primarily driven by the advancement into a nonhuman primate challenge study.
No CpG 1018 adjuvant product revenue was recorded in the third quarter of 2023, compared to $126.3 million in the third quarter of 2022, due to completion of all obligations and product delivery under the Company’s CpG 1018 adjuvant COVID-19 collaboration agreements as of December 31, 2022.
Total revenues for the third quarter of 2023 were $69.5 million, compared to $167.7 million for the third quarter of 2022.
Cost of Sales – Product. Cost of sales – product for HEPLISAV-B in the third quarter of 2023 increased to $13.2 million, compared to $11.5 million for the third quarter of 2022. The increase was primarily due to higher sales volume driven by continued improvement in HEPLISAV-B market share, offset by lower per-unit manufacturing costs as the result of previous process improvements. Total cost of sales – product for the third quarter of 2023 decreased to $13.2 million, compared to $61.3 million in the third quarter of 2022. The decrease is primarily due to no CpG 1018 adjuvant cost of sales – product for the third quarter of 2023 as a result of completing all obligations and product delivery under the prior CpG 1018 adjuvant collaboration agreements as of December 31, 2022.

Research and Development Expenses (R&D). R&D expenses for the third quarter of 2023 increased to $14.1 million, compared to $13.0 million for the third quarter of 2022. The increase was primarily driven by continued investments in advancing our clinical and preclinical development programs and collaborations.

Selling, General, and Administrative Expenses (SG&A). SG&A expenses for the third quarter of 2023 increased to $38.1 million, compared to $32.0 million for the third quarter of 2022. The increase was primarily driven by higher compensation and related personnel costs and an overall increase in targeted commercial and marketing efforts designed to increase HEPLISAV-B market share and maximize the opportunities presented by the ACIP’s universal recommendation.

Net income. GAAP net income was $14.3 million, or $0.11 per share (basic) and $0.10 per share (diluted) in the third quarter of 2023, compared to GAAP net income of $63.8 million, or $0.50 per share (basic) and $0.43 per share (diluted) in the third quarter of 2022.

Cash and Marketable Securities. Cash, cash equivalents and marketable securities were $720.4 million as of September 30, 2023.

2023 FINANCIAL GUIDANCE
Full year 2023 financial guidance has been revised to consist of the following expectations:

HEPLISAV-B net product revenue between approximately $210 – $220 million, compared to the prior range of approximately $200 – $215 million.
Research and development expenses between approximately $50 – $60 million, compared to the prior range of approximately $55 – $70 million.
Selling, general and administrative expenses between approximately $145 – $155 million, compared to the prior range of approximately $135 – $155 million.
Conference Call and Webcast Information
Dynavax will host a conference call and live audio webcast on Thursday, November 2, 2023, at 4:30 p.m. ET/1:30 p.m. PT. The live audio webcast may be accessed through the "Events & Presentations" page on the "Investors" section of the Company’s website at View Source A replay of the webcast will be available for 30 days following the live event.

To dial into the call, participants will need to register for the call using the caller registration link. It is recommended that participants dial into the conference call or log into the webcast approximately 10 minutes prior to the call.

Important U.S. Product Information
HEPLISAV-B is indicated for the prevention of infection caused by all known subtypes of hepatitis B virus in adults aged 18 years and older.

For full U.S. Prescribing Information for HEPLISAV-B, click here.

Important U.S. Safety Information (ISI)
Do not administer HEPLISAV-B to individuals with a history of a severe allergic reaction (e.g., anaphylaxis) after a previous dose of any hepatitis B vaccine or to any component of HEPLISAV-B, including yeast.

Appropriate medical treatment and supervision must be available to manage possible anaphylactic reactions following administration of HEPLISAV-B.

Immunocompromised persons, including individuals receiving immunosuppressant therapy, may have a diminished immune response to HEPLISAV-B.

Hepatitis B has a long incubation period. HEPLISAV-B may not prevent hepatitis B infection in individuals who have an unrecognized hepatitis B infection at the time of vaccine administration.

The most common patient-reported adverse reactions reported within 7 days of vaccination were injection site pain (23% to 39%), fatigue (11% to 17%), and headache (8% to 17%).

On November 2, 2023 Cytokinetics, Incorporated (Nasdaq: CYTK) reported financial results for the third quarter of 2023 (Press release, Cytokinetics, NOV 2, 2023, View Source [SID1234636753]). Net loss for the third quarter was $129.4 million, or $1.35 per share, compared to net loss for the third quarter of 2022 of $142.3 million, or $1.52 per share. Cash, cash equivalents and investments totaled $554.7 million on September 30, 2023.

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"During the third quarter we made considerable progress across our specialty cardiology franchise, with aficamten remaining our top priority. Of note, the baseline characteristics of patients enrolled in SEQUOIA-HCM met our objectives for the trial and align with our goal to assess aficamten as a potential next-in-class cardiac myosin inhibitor in a population with a substantial deficit in exercise capacity and significant symptom burden despite existing standard of care," said Robert I. Blum, Cytokinetics’ President and Chief Executive Officer. "In addition, we recently shared longer-term data from FOREST-HCM that demonstrate durable reductions in pressure gradients and cardiac biomarkers as well as improved symptoms in patients with obstructive HCM. During the quarter we also started ACACIA-HCM, a pivotal Phase 3 clinical trial of aficamten in patients with non-obstructive HCM. Alongside these increasing commitments to aficamten, we are approaching the end of 2023 in a strong position with the resources and pipeline to execute on our goals in service to both patients and shareholders."

Q3 and Recent Highlights

Cardiac Muscle Programs

aficamten (cardiac myosin inhibitor)

•
Presented baseline characteristics from SEQUOIA-HCM (Safety, Efficacy, and Quantitative Understanding of Obstruction Impact of Aficamten in HCM), the first Phase 3 trial of aficamten in obstructive hypertrophic cardiomyopathy (HCM), at the HCM Society Scientific Sessions 2023.

•
Shared new long-term data from FOREST-HCM (Follow-up, Open-Label, Research Evaluation of Sustained Treatment with Aficamten in HCM) the open-label extension clinical study of aficamten in patients with HCM, at the Company’s recent Investor and Analyst Day, demonstrating sustained reductions in left ventricular outflow tract (LVOT) gradients with no treatment interruptions for low left ventricular ejection fraction (LVEF) due to aficamten. Additionally, patients experienced sustained reductions in cardiac biomarkers and improved symptoms. Aficamten has been generally well-tolerated, with no treatment-related serious adverse events (SAEs) as assessed by investigators, and no patient deaths.

•
Announced the start of ACACIA-HCM (Assessment Comparing Aficamten to Placebo on Cardiac Endpoints In Adults with Non-Obstructive HCM), a pivotal Phase 3 clinical trial of aficamten in patients with non-obstructive HCM.

•
Published a manuscript entitled "Aficamten in Patients with Drug-Refractory Obstructive Hypertrophic Cardiomyopathy Receiving Disopyramide: REDWOOD-HCM Cohort 3 Analysis" in the Journal of Cardiac Failure.

•
Published a manuscript entitled "Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single and Multiple Doses of Aficamten in Healthy Chinese Participants: a Randomized, Double-blind, Placebo-controlled, Phase 1 Study" in the Frontiers of Pharmacology.

•
Published a manuscript entitled "Exercise Capacity in Patients With Obstructive Hypertrophic Cardiomyopathy: SEQUOIA-HCM Baseline Characteristics and Study Design" in the Journal of the American College of Cardiology: Heart Failure.

omecamtiv mecarbil (cardiac myosin activator)

•
Submitted a Formal Dispute Resolution Request to the Office of New Drugs (OND) of the U.S. Food and Drug Administration (FDA) regarding the Complete Response Letter (CRL) for omecamtiv mecarbil with objective to appeal the FDA’s conclusion, as stated in the CRL, that
substantial evidence of effectiveness had not been established to support approval of omecamtiv mecarbil.

•
Submitted responses to the Day 120 questions to the European Medicines Agency (EMA) in connection with its review of the marketing application for omecamtiv mecarbil for the treatment of advanced or worsening heart failure with reduced ejection fraction (HFrEF).

•
Ji Xing Pharmaceuticals submitted a request for voluntary withdrawal of the NDA for omecamtiv mecarbil to the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) of the People’s Republic of China, subject to potential re-submission upon receipt of favorable feedback from EMA or FDA with regard to potential drug approval for omecamtiv mecarbil in the EU or U.S., respectively.

CK-3828136 (CK-136, cardiac troponin activator)

•
Completed the single ascending dose (SAD) cohorts of the Phase 1 study of CK-136 in healthy participants. Initiated analyses of the SAD data to inform potentially proceeding to the multiple ascending dose (MAD) cohorts of the Phase 1 study.

CK-4021586 (CK-586, cardiac myosin inhibitor)

•
Completed the SAD cohorts of the Phase 1 study of CK-586 in healthy participants. Completed analyses of the SAD data which are supportive of proceeding to the MAD cohorts of the Phase 1 study in Q4 2023.

Pre-Clinical Development and Ongoing Research

•
Continued research activities directed to our other muscle biology research programs.

Corporate

•
Announced a call for proposals for the sixth annual Cytokinetics Communications Grant Program. The program awards five grants worth $20,000 each to patient advocacy organizations serving the HCM, heart failure or ALS communities, and is intended to support increased capacity in communications and outreach.

Upcoming Corporate Milestones

Cardiac Muscle Programs

aficamten (cardiac myosin inhibitor)

•
Expect topline results from SEQUOIA-HCM in late December.

•
Continue enrollment of MAPLE-HCM.

•
Continue enrollment of ACACIA-HCM.

•
Continue advancing go-to-market strategy for aficamten.

omecamtiv mecarbil (cardiac myosin activator)

•
Continue to pursue potential approval for omecamtiv mecarbil in Europe.

CK-3828136 (CK-136, cardiac troponin activator)

•
Analyze SAD data from the Phase 1 study of CK-136 to inform potentially proceeding with the MAD cohorts in the Phase 1 study.

CK-4021586 (CK-586, cardiac myosin inhibitor)

•
Proceed to the MAD cohorts in the Phase 1 study of CK-586.

Financials

Revenues for the three and nine months ended September 30, 2023 were $0.4 million and $5.9 million (inclusive of $2.5 million milestone, in the nine months ended, from Ji Xing Pharmaceuticals upon the start of ACACIA-HCM), respectively, compared to $2.5 million and $92.6 million (inclusive of $87 million from the sale of our royalty on mavacamten) for the corresponding periods in 2022.

Research and development expenses for the three and nine months ended September 30, 2023 increased to $82.5 million and $245.2 million, respectively, compared to $62.7 million and $165.8 million for the same periods in 2022, respectively, due primarily to increased spending for our cardiac myosin inhibitor programs.

General and administrative expenses for the three months ended September 30, 2023 decreased to $40.1 million from $48.2 million from the three months ended September 20, 2022, primarily due to lower outside services spend. General and administrative expenses for the nine months ended September 30, 2023 increased to $129.5 million from $124.0 million from the nine months ended September 20, 2022,

primarily due to higher personnel related cost including stock-based compensation offset by lower outside service spend.

During the quarter we also received a $50 million cash milestone payment from Royalty Pharma plc upon the start of ACACIA-HCM, treated as a liability on our balance sheet in accordance with GAAP.

Conference Call and Webcast Information

Members of Cytokinetics’ senior management team will review the company’s third quarter 2023 results on a conference call today at 4:30 PM Eastern Time. The conference call will be simultaneously webcast and can be accessed from the Investors & Media section of Cytokinetics’ website at www.cytokinetics.com. The live audio of the conference call can also be accessed by telephone by registering in advance at the following link: Cytokinetics Q3 2023 Earnings Conference Call. Upon registration, participants will receive a dial-in number and a unique passcode to access the call. An archived replay of the webcast will be available via Cytokinetics’ website for twelve months.

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.