On November 3, 2022 Syndax Pharmaceuticals, Inc. (Nasdaq: SNDX), a clinical-stage biopharmaceutical company developing an innovative pipeline of cancer therapies, reported its financial results and provided a business update for the third quarter ended September 30, 2022 (Press release, Syndax, NOV 3, 2022, View Source [SID1234622965]).

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"As we near the end of this transformational year, we are particularly excited to share that updated data from the Phase 1 portion of the ongoing AUGMENT-101 trial of revumenib will be featured during two oral presentations at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December. The rate of patients achieving a complete response (CR/CRh) and the nine-month median duration of CR/CRh response as of the data cutoff, further support the robust clinical profile of revumenib," said Michael A. Metzger, Chief Executive Officer. "The data continue to underscore the potential for revumenib to serve as a first- and best-in-class treatment option for patients with relapsed/refractory (R/R) nucleophosmin 1 (NPM1) mutations and mixed lineage leukemia rearrangements (MLLr). Enrollment continues in AUGMENT-101 and we anticipate reporting topline data from at least one of the pivotal cohorts starting in the third quarter of 2023, with a potential New Drug Application (NDA) filing expected by the end of 2023."

Mr. Metzger added, "We also continue to make significant progress on the development of axatilimab and are pleased to announce that we have completed enrollment in our pivotal AGAVE-201 trial in chronic graft versus host disease (cGVHD). We look forward to announcing topline results from the trial in mid-2023, with a potential biologics license application (BLA) filing expected to follow by the end of 2023."

Recent Pipeline Progress and Anticipated Milestones

Revumenib

Earlier today, the Company announced updated positive data from the Phase 1 portion of the ongoing AUGMENT-101 trial of revumenib in patients with R/R NPM1 mutant or MLLr (also referred to as KMT2Ar) acute leukemia which highlighted a 30% (18/60) CR/CRh rate and a median duration of CR/CRh response of 9.1 months. Additionally, of the 12 patients who achieved a complete response on revumenib treatment and then went on to receive a stem cell transplant, nine (75%) remained in remission as of the data cutoff date, with a median follow-up of 12.3 months. Three patients were treated with revumenib maintenance in the compassionate use setting following stem cell transplant or non-myeloablative stem cell boost, two of whom (67%) remained in remission for over one year. To date, there have been no discontinuations due to treatment-related adverse events. Data reported today will be featured in two oral sessions at the ASH (Free ASH Whitepaper) Annual Meeting on Saturday, December 10, 2022. Copies of both abstracts are available on the ASH (Free ASH Whitepaper) website at www.hematology.org.

The Company also announced today that it will host a conference call and webcast to discuss the ASH (Free ASH Whitepaper) data presentations on Sunday, December 11, 2022 at 8 a.m. CT / 9 a.m. ET. Joining the call will be members of the Syndax management team as well as Principal Investigators from the AUGMENT-101 trial. For additional information on how to access the event, please visit the Investor section of the Company’s website at www.syndax.com.

The pivotal Phase 2 portion of AUGMENT-101 is ongoing, enrolling patients across each of three distinct trial populations: patients with NPM1 mutant acute myeloid leukemia (AML), patients with MLLr AML, and patients with MLLr acute lymphocytic leukemia (ALL). Based on discussions with the U.S. Food and Drug Administration, AUGMENT-101 may serve as the basis for regulatory filings in each of the three distinct populations. The Company expects completion of enrollment in the first trial to extend into the first quarter of 2023 and to report topline data from at least one of the trials starting in the third quarter of 2023. The Company continues to expect to submit its first NDA filing for revumenib by the end of 2023.

Two trials, BEAT-AML and AUGMENT-102, are ongoing and will assess the safety, tolerability, and preliminary anti-leukemic efficacy of revumenib, and establish an appropriate Phase 2 dose when used in combination with other approved agents. BEAT-AML is a front-line combination trial of revumenib with venetoclax and azacitidine being conducted as part of the Leukemia & Lymphoma Society’s Beat AML Master Clinical Trial. AUGMENT-102 is a trial assessing revumenib in combination with chemotherapy in patients with R/R mNPM1 or MLLr acute leukemias.

The Company expects the Australasian Leukaemia and Lymphoma Group (ALLG) to initiate the INTERCEPT trial of revumenib as monotherapy in patients with AML who are minimal residual disease-positive following initial treatment, in the fourth quarter of 2022. The trial is a part of the INTERCEPT AML Master Clinical Trial, a collaborative clinical trial investigating novel therapies to target early relapse and clonal evolution as pre-emptive therapy in AML. Revumenib is the first menin inhibitor to be included in the INTERCEPT AML Master Clinical Trial.

The Company remains on track to initiate a proof-of-concept clinical trial of revumenib in patients with unresectable metastatic microsatellite stable colorectal cancer in the fourth quarter of 2022.
Axatilimab

The Company and its partner, Incyte, reported completion of enrollment in the pivotal AGAVE-201 trial evaluating axatilimab in patients with cGVHD following two or more prior lines of therapy. The trial is evaluating the safety and efficacy of three dosing regimens of axatilimab. The primary endpoint will assess objective response rate based on the 2014 NIH consensus criteria for cGVHD, with key secondary endpoints including duration of response and improvement in modified Lee Symptom Scale score. The Company expects to report topline data in mid-2023, with the expectation for a BLA filing later in 2023.

The Company plans to initiate a Phase 2b trial to assess the efficacy, safety and tolerability of axatilimab in patients with idiopathic pulmonary fibrosis (IPF) in the fourth quarter of 2022. This 52-week, randomized, double-blind and placebo-controlled trial is expected to enroll approximately 170 patients. The primary endpoint will assess the change from baseline in forced vital capacity, which is the current registrational endpoint in IPF.

The Company is working with its partner, Incyte, to initiate a trial testing axatilimab in combination with ruxolitinib in steroid naive cGVHD. The Phase 1 trial is in preparation and is expected to begin in the first quarter of 2023.
Third Quarter 2022 Financial Results

As of September 30, 2022, Syndax had cash, cash equivalents and short-term investments of $337.8 million and 61.3 million common shares and pre-funded warrants outstanding.

Third quarter 2022 research and development expenses increased to $26.9 million from $25.6 million for the prior year period. The increase was primarily due to increased employee related expenses and professional fees partially offset by decreased clinical and manufacturing expenses, in large part the result of axatilimab cost sharing benefits.

General and administrative expenses for the third quarter 2022 increased to $8.2 million from $6.8 million for the prior year period. The increase is primarily due to increased employee related expenses and professional fees.

For the three months ended September 30, 2022, Syndax reported a net loss attributable to common stockholders of $35.4 million, or $0.58 per share, compared to a net loss attributable to common stockholders of $20.6 million, or $0.40 per share, for the prior year period.

Financial Update and Guidance

For the full year of 2022, the Company is lowering its expectations of both research and development and total operating expenses. The Company now expects research and development expenses to be $115 to $125 million and total operating expenses to be $145 to $155 million. This is a reduction from the Company’s prior guidance for the full year of 2022 of $130 to $140 million in research and development expenses and $160 to $170 million in total operating expenses. The updated guidance reflects the benefits derived from the shared expenses for our axatilimab cGVHD development program.

In September, Syndax made a debt repayment in connection with the termination of a Loan Agreement. This loan repayment has no adverse impact on Syndax’s cash guidance and the Company continues to expect to have sufficient capital to fund operations into the second half of 2024.

Conference Call and Webcast

In connection with the earnings release, Syndax’s management team will host a conference call and live audio webcast at 4:30 p.m. ET today, Thursday, November 3, 2022.

The live audio webcast and accompanying slides may be accessed through the Events & Presentations page in the Investors section of the Company’s website. Alternatively, the conference call may be accessed through the following:

For those unable to participate in the conference call or webcast, a replay will be available on the Investors section of the Company’s website at www.syndax.com approximately 24 hours after the conference call and will be available for 90 days following the call.

On November 3, 2022 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, 2022 (Press release, Dynavax Technologies, NOV 3, 2022, View Source [SID1234622964]).

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"For the third quarter, we demonstrated another quarter of successful execution on our key priorities and remain on track for profitability with record revenues anticipated for both HEPLISAV-B and our CpG 1018 adjuvant in 2022," commented Ryan Spencer, Chief Executive Officer of Dynavax. "We believe we are well capitalized to invest in driving revenue growth for HEPLISAV-B and to deliver progress across our clinical pipeline, focusing on high value vaccine programs where our proven adjuvant may provide meaningful differentiation."

THIRD-QUARTER CORPORATE HIGHLIGHTS

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

HEPLISAV-B vaccine is the first and only adult hepatitis B vaccine approved in the U.S. and EU 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 vaccine achieved net product revenue of $37.5 million for the third quarter of 2022, up 65% compared to $22.7 million for the third quarter of 2021.
Market share in the accounts targeted by the Dynavax field sales team increased to approximately 43%, with total market share increasing to approximately 32% in the third quarter of 2022, up from approximately 32% and 25%, respectively, in the third quarter of 2021.
CpG 1018 Adjuvant Supply for COVID-19 Vaccines

Dynavax has established a global portfolio of CpG 1018 adjuvant commercial supply agreements (CSAs) currently supporting the development of COVID-19 vaccines across a variety of vaccine platforms.

CpG 1018 adjuvant revenue for the third quarter of 2022 was $126.3 million, up 50% compared to $84.3 million for the third quarter of 2021.
The Company reiterates its expectation of 2022 full-year CpG 1018 adjuvant COVID-19 supply revenue to be between $550 million and $600 million, based on committed orders under our CSAs, with an anticipated full-year gross margin of approximately 60%.
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.

Tetanus, diphtheria and pertussis (Tdap) vaccine program:

In October, the Company presented adult and adolescent safety data from the Phase 1 clinical trial demonstrating the Tdap vaccine candidate was well tolerated without safety concerns. Immunogenicity in adults was consistent with the Company’s expectations and support its plan to continue advancement of this clinical program. These clinical results were presented at ID Week.
Shingles vaccine program:

In August, enrollment was completed in the ongoing randomized Phase 1 clinical trial evaluating the safety, tolerability, and immunogenicity in adults of the Company’s shingles vaccine candidate adjuvanted with CpG 1018 compared to the leading marketed shingles vaccine in the U.S.
Data from this clinical trial is anticipated before the end of 2022.
Plague vaccine candidate:

In August, the first participant was dosed in the Phase 2 clinical trial evaluating the immunogenicity, safety and tolerability in adults of a plague (rF1V) vaccine candidate adjuvanted with CpG 1018. The clinical trial is being conducted in collaboration with, and funded by, the U.S. Department of Defense.
THIRD-QUARTER FINANCIAL HIGHLIGHTS

Total Revenues and Product Revenue, Net.

Total revenues for the third quarter of 2022 were $167.7 million, compared to $108.3 million for the third quarter of 2021.

HEPLISAV-B vaccine product revenue, net was $37.5 million for the third quarter of 2022, compared to $22.7 million for the third quarter of 2021.
CpG 1018 adjuvant product revenue, net was $126.3 million in the third quarter of 2022 compared to $84.3 million in the third quarter of 2021.
Selected financial highlights from CpG 1018 adjuvant product supply partnerships for COVID-19 vaccines and vaccine candidates:
The Company recorded approximately $87.5 million in CpG 1018 adjuvant product revenue under its CSA with Clover.
The Company recorded approximately $27.6 million in CpG 1018 adjuvant product revenue under its CSA with Biological E.
The Company recorded approximately $11.2 million in CpG 1018 adjuvant product revenue under its CSA with Bio Farma.
Cost of Sales – Product. Cost of sales – product for the third quarter of 2022 increased to $61.3 million, compared to $60.1 million for the third quarter of 2021. The increase was due to manufacturing costs for increased volumes of CpG 1018 adjuvant sold to COVID-19 supply partners, an inventory write-down of $14.5 million related to the reduction in demand for CpG 1018 and increased HEPLISAV-B vaccine sales volume.

Research and Development Expenses (R&D). R&D expenses for the third quarter of 2022 increased to $13.0 million, compared to $6.2 million for the third quarter of 2021. The increase was primarily driven by increased headcount-related compensation and personnel costs, including non-cash stock-based compensation, as well as investments in product candidates utilizing CpG 1018 adjuvant for TDAP, shingles and plague.

Selling, General, and Administrative Expenses (SG&A). SG&A expenses for the third quarter of 2022 increased to $32.0 million, compared to $26.9 million for the third quarter of 2021. The increase was primarily driven by compensation and related personnel costs, including non-cash stock-based compensation coupled with increased external commercial and marketing activities related to the universal recommendation for hepatitis B vaccination.

Net Income. GAAP net income was $63.8 million, or $0.50 per share (basic) and $0.43 per share (diluted) in the third quarter of 2022, compared to GAAP net loss of $28.4 million, or $0.24 per share (basic) and $0.24 per share (diluted) in the third quarter of 2021.

Cash and Marketable Securities. Cash and marketable securities were $586.5 million as of September 30, 2022.

2022 Financial Guidance

Dynavax anticipates 2022 revenues, operating expenses, and other costs to be in the ranges shown below, consistent with the Company’s previous financial guidance provided on August 4, 2022:

Full-year CpG 1018 adjuvant net product revenues of between $550 million and $600 million, with an associated gross margin anticipated to be approximately 60%
Research and development expenses to be between approximately $50-$60 million
Selling, general and administrative expenses to be between approximately $130-$140 million
Interest expense of approximately $7 million
Conference Call and Webcast Information

Dynavax will host a conference call and live audio webcast on Thursday, November 3, 2022, 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 3, 2022 Gritstone bio, Inc. (Nasdaq: GRTS), a clinical-stage biotechnology company working to develop the world’s most potent vaccines, reported financial results for the third quarter ended September 30, 2022 and reviewed business highlights (Press release, Gritstone Oncology, NOV 3, 2022, View Source [SID1234622963]).

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"Gritstone’s relentless execution continues to generate novel clinical data," said Andrew Allen, M.D., Ph.D., Co-founder, President and Chief Executive Officer of Gritstone. "The KRAS neoantigen-directed data presented at ESMO (Free ESMO Whitepaper) 2022 in September, from our off-the-shelf neoantigen program (SLATE), show a molecular response rate of approximately 40%, which was also associated with extended overall survival in advanced lung cancer patients. These results are consistent with prior results from GRANITE in advanced colorectal cancer patients. The scientific foundations of the GRANITE program, common to SLATE, are illuminated in our recent publication of GRANITE Phase 1/2 results in Nature Medicine. This technology platform is establishing its potential to transform the treatment of a large number of solid tumor patients who currently do not respond to checkpoint inhibitors. Through the CORAL program, we continue to demonstrate proof-of-concept for self-amplifying mRNA (samRNA) as a novel, differentiated, scalable, and widely applicable platform technology against COVID-19 and other viral pathogens. As we enter 2023 with a strong balance sheet, our focus remains on driving our clinical programs toward meaningful data catalysts and continuing to unlock the value of our platforms across oncology and infectious diseases."

Clinical Program Updates
Oncology Programs
GRANITE – Individualized neoantigen-directed immunotherapy

In August 2022, interim results from the Phase 1/2 trial of GRANITE were published in Nature Medicine (here). The paper describes how Gritstone’s neoantigen-directed vaccination approach (referred to as "prime-boost") led to both priming and boosting of tumor-specific T cells, with associated molecular responses in approximately half of treated advanced colorectal cancer (CRC) patients. Follow-up data from the Phase 1/2 study have suggested a correlation between molecular response and extended overall survival (MOS of 18+ months among responders vs. 7.8 months in non-responders; latest overall survival results reported in May 2022). These data catalyzed the launch of GRANITE-CRC-1L, a randomized, controlled Phase 2/3 maintenance trial in newly diagnosed metastatic CRC patients that is ongoing and has registrational intent.

Preliminary data from the Phase 2 portion of GRANITE-CRC-1L are expected in 4Q 2023.
SLATE – "Off-the-shelf" shared neoantigen-directed immunotherapy

In September 2022, Gritstone presented positive data from a Phase 1/2 study evaluating KRAS-directed SLATE at ESMO (Free ESMO Whitepaper). The data included initial results with SLATE-KRAS, a shared mutant KRAS-specific neoantigen vaccine candidate, and updated data using the first version of the vaccine candidate (SLATE v1) which contains both KRAS and non-KRAS neoantigens. In 38 patients with advanced solid tumors, SLATE v1 (n =26) and SLATE-KRAS (n=12) demonstrated a 39% molecular response rate (MRR). In a mature dataset of NSCLC subjects who had progressed on prior chemo-immunotherapy, a positive association was observed between molecular response and overall survival, a signal similar to that seen in the Phase 1/2 study of GRANITE (individualized immunotherapy for advanced solid tumors).
Infectious Disease Programs
CORAL – Second-generation SARS-CoV-2 vaccine program. This program serves as proof-of-concept for Gritstone’s infectious disease approach and the potential broad application of samRNA in infectious diseases.

In October 2022, Gritstone shared interim positive results from the ongoing Phase 1 CORAL-BOOST (NCT05148962) and CORAL-CEPI (NCT05435027) trials at a company-sponsored webinar. Collectively, these results showed Gritstone’s samRNA vaccine candidates to be well-tolerated and capable of driving strong, potentially durable and broad immunogenicity across several subject populations and settings.

In September 2022, enrollment was completed in the CORAL-NIH trial (NCT04776317), which is sponsored and executed by the National Institute of Allergy and Infectious Disease (NIAID).
HIV – Collaboration with Gilead Sciences, Inc. (Gilead) under Gilead’s HIV Cure Program to research and develop vaccine-based HIV immunotherapy treatment.

An investigational new drug application (IND) was cleared in December 2021, and the Phase 1 clinical study is ongoing.
Corporate Highlights

In August 2022, Dr. Larry Corey, an internationally renowned expert in virology, immunology and vaccine development and Emeritus President and Director of Fred Hutch, joined Gritstone’s Board of Directors.
In October 2022, Gritstone closed a private investment in public equity financing of $45 million of common stock and pre-funded warrants to support development of its ongoing and future preclinical and clinical programs.
Third Quarter 2022 Financial Results
Cash, cash equivalents, marketable securities and restricted cash were $151.8 million as of September 30, 2022, compared to $223.5 million as of December 31, 2021.

Research and development expenses were $26.4 million for the three months ended September 30, 2022, compared to $24.4 million for the three months ended September 30, 2021. The increase of $2.0 million for the three months ended September 30, 2022 compared to the three months ended September 30, 2021 was primarily due to increases of $1.6 million in personnel-related expenses, $1.0 million in outside services, consisting primarily of clinical trial and other chemistry, manufacturing and controls ("CMC") related expenses, and $0.6 million in facilities-related costs, offset by a decrease of $1.2 million in laboratory supplies.

General and administrative expenses were $6.5 million for the three months ended September 30, 2022, compared to $6.4 million for the three months ended September 30, 2021. The increase of $0.1 million was primarily attributable to an increase of $0.7 million in personnel-related expenses, offset by decreases of $0.4 million in outside services and $0.2 million in facilities-related costs.

Collaboration, license, and grant revenues were $3.0 million for the three months ended September 30, 2022, compared to $2.6 million for the three months ended September 30, 2021. The $0.4 million increase was primarily attributable to revenue recognized under the CEPI Funding Agreement in the amount of $2.1 million, offset by a decrease of $1.4 million related to collaboration revenue recognized under the Gilead Collaboration Agreement.

About Gritstone’s Oncology Programs
Gritstone’s two clinical stage oncology programs are developing Tumor-Specific Neoantigen (TSNA)-directed vaccine-based immunotherapies that use an adenoviral priming vector and samRNA boost vector ("prime-boost" approach) to deliver relevant neoantigens in combination with immune checkpoint blockade (ICB). GRANITE, which is "individualized" and SLATE, which is "off-the-shelf," aim to induce a substantial neoantigen-specific CD8+ T cell response using neoantigen-containing immunotherapies. GRANITE patients receive a product candidate made specifically for them, based upon their tumor DNA/RNA sequence. In contrast, SLATE patients receive an off-the-shelf product candidate made for common driver mutations present in the patient’s tumor as well as the patient having a HLA allele that can present the common driver mutation.

About Gritstone’s Infectious Disease Programs
Gritstone’s infectious disease programs aim to deliver vaccines that drive durable and broad B cell and T cell immunity to deliver long-lasting clinical protection against viral disease, a clear unmet need in the field. All programs utilize Gritstone’s self-amplifying mRNA (samRNA) platform.

About the CORAL Program
Gritstone’s CORAL program is evaluating the company’s infectious disease approach, which is designed to drive both B cell and T cell immunity using self-amplifying mRNA (samRNA) against SARS-CoV-2. The program currently includes three ongoing Phase 1 trials: CORAL-BOOST, which is evaluating one construct in a boost setting (following primary series of currently-approved COVID-19 vaccines); CORAL-CEPI, which is evaluating multiple constructs in virus-naïve, convalescent, and HIV+ subjects in South Africa; and CORAL-NIH, which is being run by the National Institute of Allergy and Infectious Disease (NIAID) and is evaluating multiple constructs in previously vaccinated healthy volunteers. The program serves as proof-of-concept for the application of Gritstone’s platform against coronaviruses and other infectious diseases and is supported by the Bill & Melinda Gates Foundation, NIAID and the Coalition for Epidemic Preparedness Innovations (CEPI).

On November 3, 2022 Corvus Pharmaceuticals, Inc. (Corvus or the Company) (Nasdaq: CRVS), a clinical-stage biopharmaceutical company, reported financial results for the third quarter ended September 30, 2022 (Press release, Corvus Pharmaceuticals, NOV 3, 2022, View Source [SID1234622962]).

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"We anticipate several important milestones for all three of our clinical programs in the near-term, headlined by the continued advancement of CPI-818 for T cell lymphoma and the initiation of studies for CPI-818 in autoimmune disease," said Richard A. Miller, M.D., co-founder, president and chief executive officer of Corvus. "We look forward to sharing new data on CPI-818 at the ASH (Free ASH Whitepaper) meeting in December, which will provide an update on its activity in patients with T cell lymphoma along with data on its effects on normal T cell function related to its potential in autoimmunity and allergy. CPI-818 continues to be well positioned as the most advanced program targeting ITK inhibition and we are increasingly confident in its biologic activity and potential broad applications in immune mediated diseases."

"We also continue to advance ciforadenant, our adenosine 2a receptor inhibitor, with the recent initiation of a Phase 1b/2 clinical trial in first line renal cell cancer. This study is partnered with the Kidney Cancer Research Consortium and has the potential to provide near-term clinical data. Outside of the United States, our Chinese partner Angel Pharmaceuticals intends to initiate a Phase 1/1b trial evaluating mupadolimab as a monotherapy and together with pembrolizumab in patients with lung cancer and head and neck cancer. Altogether, our portfolio is positioned to deliver clinical results in multiple diseases in the coming year."

Business Update and Strategy

CPI-818 (selective ITK inhibitor)
Corvus and Angel Pharmaceuticals are enrolling patients with relapsed T cell lymphomas (TCL) in a Phase 1/1b clinical trial evaluating single agent therapy with CPI-818. A 200 mg dose of CPI-818 given orally twice per day has been identified as the optimal dose and the companies are enrolling additional patients with TCL in a 200 mg dose cohort of the clinical trial. Angel Pharmaceuticals is responsible for all expenses related to conducting the clinical trial in China. Recent developments include:

Monitoring of immune modulation of normal T cells as well as safety and anti-tumor activity are being assessed in the clinical trial, with new data to be presented at the 64th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition in December 2022.
As of July 22, 2022, 12 patients were enrolled in the 200 mg cohort and eight were evaluable for response. In this group, there was one complete response (CR) lasting 25 months; one nodal CR lasting 16 months; one partial response (PR) ongoing at two months follow up. Five patients had stable disease (SD), two of the patients with SD had been on treatment for approximately 12 weeks and continued on study. Two additional patients were on treatment and had not yet had their disease monitoring assessments. An additional patient in the 600 mg cohort also had a PR.
Analysis of blood in four of four patients treated in the 200 mg cohort showed increases in Th1 cells compared to baseline and increases in terminally differentiated T effector memory cells; these are T cells that are antigen primed and capable of destroying tumor cells. Tumor biopsy from one patient taken during response demonstrated an increase in these cells in the tumor.
Three of three patients in the 200 mg cohort with high baseline, pretreatment eosinophil counts demonstrated reductions in circulating eosinophils during treatment with CPI-818. Eosinophils are white blood cells that play a key role in allergic and autoimmune diseases, and they are often elevated in patients with TCL.
Corvus is also developing CPI-818 for autoimmune and allergic diseases and is preparing to initiate clinical trials for certain autoimmune diseases in the near term.

CPI-818 has demonstrated activity in various animal models of autoimmunity including models of systemic lupus erythematosus, psoriasis, inflammatory bowel disease, lung fibrosis and graft versus host disease. Some of this research was presented at the annual meetings of the American Society of Hematology (ASH) (Free ASH Whitepaper) in 2020 and 2021.
Corvus’ research and development team has been working to prioritize and prepare protocols for new CPI-818 clinical studies in autoimmune diseases, with the announcement of next clinical program anticipated in the near-term.
Ciforadenant (adenosine 2a receptor inhibitor) for first line renal cell cancer

Corvus is collaborating with the KCRC in an open-label Phase 1b/2 clinical trial evaluating ciforadenant as a first line therapy for metastatic RCC in combination with ipilimumab (anti-CTLA-4) and nivolumab (anti-PD-1). The clinical trial is expected to enroll up to 60 patients and is intended to evaluate the potential for ciforadenant to generate increased complete responses and deep responses in the first line setting.
The KCRC is comprised of a group of leading cancer centers in the United States led by investigators at The University of Texas MD Anderson Cancer Center. The trial design is based on Corvus’ preclinical research published in 2018 in Cancer Immunology Research that demonstrated impressive antitumor control and complete tumor elimination in several animal models using ciforadenant in combination with anti-CTLA-4 and anti-PD-1.
Mupadolimab (anti-CD73)

In September 2022, the Center for Drug Evaluation of the China National Medical Products Administration approved an IND application to initiate a Phase 1/1b clinical trial with mupadolimab alone and together with pembrolizumab in patients with relapsed refractory non-small cell lung cancer and head and neck squamous cell cancers. Corvus anticipates that the study will be initiated by Angel Pharmaceuticals in the near-term.
R&D Conference Call and Webcast on December 12, 2022
The Company will host a conference call on Monday, December 12, 2022 from 4:30 – 5:30 pm ET to provide an overview of the CPI-818 data that will be presented at the ASH (Free ASH Whitepaper) meeting, along with providing an update on the Company’s development programs. A webcast of the event will be available on the Corvus website at www.corvuspharma.com.

Financial Results
As of September 30, 2022, Corvus had cash, cash equivalents and marketable securities totaling $49.6 million. This compared to cash, cash equivalents and marketable securities of $69.5 million as of December 31, 2021. Consistent with last quarter, Corvus expects full year 2022 net cash used in operating activities to be between $27 million and $29 million, resulting in a projected cash balance of between $40.5 million and $42.5 million as of December 31, 2022. Based on its current plans, Corvus expects its cash to fund operations into early 2024.

Research and development expenses for the three months ended September 30, 2022 totaled $10.4 million compared to $7.0 million for the same period in 2021. The increase of $3.4 million was primarily related to a $5.5 million increase in drug manufacturing costs, which was partially offset by a $2.2 million reduction in clinical trial costs.

The net loss for the three months ended September 30, 2022 was $14.8 million compared to a net loss of $10.7 million for the same period in 2021. Total stock compensation expense for the three months ended September 30, 2022 was $0.7 million compared to $1.1 million for the same period in 2021 and the non-cash loss from the Company’s equity method investment in Angel Pharmaceuticals was $2.7 million for the three months ended September 30, 2022 compared to $1.7 million in the same period in 2021.

Conference Call Details
Corvus will host a conference call and webcast today, Thursday, November 3, 2022, at 4:30 p.m. ET (1:30 p.m. PT), during which time management will provide a business update and discuss the third quarter 2022 financial results. The conference call can be accessed by dialing 1-844-825-9789 (toll-free domestic) or 1-412-317-5180 (international) and using the conference ID 10170960. 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 November 3, 2022 Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, reported recent business highlights and third quarter 2022 financial results (Press release, Sangamo Therapeutics, NOV 3, 2022, View Source [SID1234622961]).

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"This has been a year marked by progress across our pipeline. In the third quarter, we continued to advance our clinical trials and preclinical activities while maintaining fiscal discipline and operational excellence," said Sandy Macrae, Chief Executive Officer of Sangamo. "We were proud to present promising updated preliminary data from our wholly owned Fabry study, to resume our partnered Hemophilia A pivotal trial, and to continue dosing in our renal transplant rejection and sickle cell studies. Our pipeline progress is expected to yield additional data in Q4 and into 2023. As we look to next year and beyond, I am confident in Sangamo’s ability to carry out our mission of developing transformational therapies for patients in need."

Recent Business Highlights

Fabry disease – Reported data updates from the Phase 1/2 STAAR study’s dose escalation phase; Dose expansion phase underway and dosing commenced; Phase 3 planning progresses.

Presented updated preliminary data from the Phase 1/2 STAAR study evaluating isaralgagene civaparvovec, our wholly owned gene therapy product candidate for the treatment of Fabry Disease at three separate conferences, most notably at the 29th Congress of the European Society of Gene & Cell Therapy (ESGCT), presenting updated data as of July 21, 2022.
Preliminary data showed all nine patients from the dose escalation phase exhibited sustained elevated α-Gal A activity, ranging from nearly 2-fold to 30-fold of mean normal, for up to 23 months post dosing, as of the last date of measurement.
Four patients were withdrawn from enzyme replacement therapy (ERT) and maintained significantly elevated levels of α-Gal A activity up to 28 weeks post withdrawal. Since the cutoff date, the fifth and final patient in the dose escalation phase who started the study on ERT has been withdrawn from ERT. All patients withdrawn have remained off ERT.
The Phase 1/2 STAAR study has transitioned into the expansion phase, with the first five expansion patients dosed at the 5e13 vg/kg dose level, including the first two female patients.
We expect to present additional clinical updates from the STAAR study, including the first data from the expansion cohort, in the first half of 2023.
We continue to actively prepare for a potential pivotal Phase 3 trial.
Sickle cell disease – Dosed sixth patient, the second with a product candidate manufactured using improved methods; Phase 3 planning progresses.

We dosed the sixth patient in the Phase 1/2 PRECIZN-1 study of BIVV003, a zinc finger nuclease gene-edited cell therapy candidate for the treatment of sickle cell disease. This is the second patient in the study to receive a product candidate manufactured using improved methods that have been shown in internal experiments to increase the number of long-term progenitor cells in the final product.
Received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for BIVV003.
We expect to present updated data from the Phase 1/2 PRECIZN-1 study via a poster presentation at the 64th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition on December 10-13, 2022 in New Orleans, Louisiana.
Phase 3 study design, enabling activities and manufacturing readiness are in progress.
Renal Transplant Rejection – Dosed the second patient in the Phase 1/2 STEADFAST study; progressed clinical activities in preparation for patient three.

Dosed the second patient in the Phase 1/2 STEADFAST study evaluating TX200, our wholly owned autologous CAR-Treg cell therapy treating patients receiving an HLA-A2 mismatched kidney from a living donor.
The product candidate continues to be generally well tolerated in both patients.
Progressed clinical activities in preparation for the third patient.
We plan to provide guidance on timing for dosing for the third patient once the kidney transplant has been scheduled.
Hemophilia A – Announced, with Pfizer, the resumption of recruitment in the Phase 3 AFFINE trial; dosing is expected to resume shortly; pivotal data read-out expected in the first half of 2024.

Pfizer and Sangamo announced that recruitment has re-opened in the Phase 3 AFFINE trial of giroctocogene fitelparvovec, an investigational gene therapy we are developing with Pfizer for patients with moderately severe to severe hemophilia A.
Trial sites resumed enrollment in September, and dosing is expected to resume shortly.
A pivotal readout is expected in the first half of 2024.
We expect to present updated data from the Phase 1/2 ALTA study via a poster presentation at the ASH (Free ASH Whitepaper) Annual Meeting in December.
Third Quarter 2022 Financial Results

Consolidated net loss for the third quarter ended September 30, 2022 was $53.2 million, or $0.34 per share, compared to a net loss of $47.7 million, or $0.33 per share, for the same period in 2021.

Revenues

Revenues for the third quarter ended September 30, 2022 were $26.5 million, compared to $28.6 million for the same period in 2021.

The decrease of $2.1 million in revenues was primarily attributable to a decrease of $1.9 million and $1.6 million related to our collaboration agreements with Novartis and Biogen respectively, and a decrease of $1.1 million due to the termination of our collaboration agreement with Sanofi. These decreases were partially offset by a $1.9 million adjustment to revenue during 2021 related to the collaboration agreement with Sanofi and an increase of $0.5 million in revenue related to our collaboration agreement with Kite.

Total operating expenses on a GAAP basis for the third quarter ended September 30, 2022 were $81.3 million, compared to $77.0 million for the same period in 2021. Non-GAAP operating expenses, which exclude stock-based compensation expense, for the third quarter ended September 30, 2022 were $73.5 million, compared to $69.1 million for the same period in 2021.

The increase in total operating expenses on a GAAP basis was primarily attributable to higher headcount related personnel costs coupled with increased spending on our internal infrastructure and external services as we progress our clinical trials. These increases were partially offset by reimbursement of certain research and development expenses by Sanofi under the termination agreement.

Cash, cash equivalents and marketable securities

Cash, cash equivalents and marketable securities as of September 30, 2022, were $350.3 million, compared to $464.7 million as of December 31, 2021. We have raised approximately $75.1 million in net proceeds under our at-the-market offering program from January 1, 2022 through October 31, 2022.

Financial Guidance for 2022 Narrowed (initial guidance provided on February 24, 2022)

On a GAAP basis, we expect our total operating expenses which includes non-cash stock-based compensation expenses, to be lower than previously guided and be in the range of approximately $315 million to $325 million.

We expect our non-GAAP total operating expenses, excluding estimated non-cash stock-based compensation expenses of approximately $35 million, to be in the range of approximately $280 million to $290 million.

Upcoming Events

Sangamo plans to participate in the following events in the fourth quarter:

Scientific / Medical Conferences

64th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition, December 10-13, New Orleans, Louisiana
Investor Conferences

31st Annual Credit Suisse Healthcare Conference, November 8, 2022
Barclays Gene Editing & Gene Therapy Summit, November 14, 2022
Stifel Healthcare Conference, November 15, 2022
Jefferies London Healthcare Conference, November 16, 2022
EvercoreISI HealthCONxConference, November 29 – December 1, 2022
Access links for available webcasts for these investor conferences will be available on the Sangamo Therapeutics website in the Investors and Media section under Events and Presentations. Available materials will be found on the Sangamo Therapeutics website after the event.

Conference Call to Discuss Third Quarter 2022 Results

The Sangamo management team will discuss these results on a conference call today, Thursday November 3, 2022, at 4:30 p.m. Eastern Time.

Participants should register for, and access, the call using this link. While not required, it is recommended you join 10 minutes prior to the event start. Once registered, participants will be given the option to either dial into the call with the number and unique passcode provided or to use the dial-out option to connect their phone instantly.

The link to access the live webcast can also be found on the Sangamo Therapeutics website in the Investors and Media section under Events and Presentations.

A replay will be available following the conference call, accessible under Events and Presentations.