On January 8, 2024 Foghorn Therapeutics Inc. (Nasdaq: FHTX), a clinical-stage biotechnology company pioneering a new class of medicines that treat serious diseases by correcting abnormal gene expression, reported its strategic objectives for 2024 (Press release, Foghorn Therapeutics, JAN 8, 2024, View Source [SID1234639077]).

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"We enter 2024 with several important milestones ahead of us. We anticipate data from our Phase 1 combination study of FHD-286 in AML in the second half of the year and look forward to continued progress with our unique pipeline, including our BRM selective inhibitor and degrader programs in collaboration with Loxo@Lilly," said Adrian Gottschalk, President and Chief Executive Officer. "We have made significant progress with our preclinical programs, including our selective EP300, CBP and ARID1B degrader programs and are getting ready to share more preclinical data in the first half of the year. This will also include preclinical combination data of FHD-286 with tyrosine kinase inhibitors of EGFR and KRAS. Over the next four years, we anticipate the filing of at least six new INDs, reflecting the productivity of our precision medicine platform. This is all supported by our cash and equivalents position of approximately $259.9 million as of September 30, 2023."

-FHD-286. FHD-286 is a potent, selective inhibitor of the BRG1 and BRM subunits of the BAF chromatin remodeling complex where dependency on BRG1/BRM is well-established preclinically with multiple tumor types, including acute myelogenous leukemia (AML)/myelodysplastic syndrome (MDS), non–small cell lung cancer (NSCLC) and prostate cancer.

oAML Update. Foghorn commenced a Phase 1 study of FHD-286 in combination with decitabine or low-dose cytarabine (LDAC) in relapsed and/or refractory AML patients, with the first patient dosed during the third quarter of 2023. The first clinical data are expected in the second half of 2024.
oTKI Resistance. Recently published data, along with Foghorn’s work, suggest that FHD-286 may play an important role in overcoming resistance in EGFR/KRAS tumors. The company is conducting preclinical work to further explore the opportunity.

-Differentiated Pipeline Advancement. Foghorn continues to expand its platform and pipeline. The Company anticipates the potential for six new investigational new drug (IND) applications in the next four years. The Company continues to progress programs for multiple targets that include chromatin remodeling complexes, transcription factors, helicases and other chromatin-related factors. These targets include selective BRM* and wholly owned programs including CBP, EP300, and ARID1B, as well as other undisclosed targets, which combined could address more than 20 tumor types impacting more than 500,000 new patients annually.
oSelective EP300 and Selective CBP programs. Foghorn presented new preclinical data for its EP300 and CBP selective degrader programs at Hanson Wade’s 6th Annual Targeted Protein Degradation Summit on October 31, 2023.
EP300 selective degraders showed potent cellular antiproliferation and in vivo tumor growth inhibition in an AR+ enzalutamide prostate in vivo model.
CBP selective degraders demonstrated significant tumor growth inhibition in a colorectal cancer in vivo model. Antiproliferative effects were also observed for numerous cancer cell lines, including colorectal, gastric and bladder cancers.
At preclinical efficacious doses, neither the EP300 nor the CBP selective degraders caused thrombocytopenia, a commonly observed safety liability for dual CBP/EP300 inhibitors.
Additional preclinical data will be presented in the first half of 2024.
-*Loxo@Lilly Collaboration. Foghorn is engaged in a strategic collaboration with Loxo@Lilly and continues to advance the BRM selective inhibitor and degrader programs along with other undisclosed programs.
-Strong Balance Sheet and Cash Runway. As of September 30, 2023, the Company had $259.9 million in cash, cash equivalents and marketable securities providing cash runway into the first half of 2026.
About AML
Adult acute myeloid leukemia (AML) is a cancer of the blood and bone marrow and the most common type of acute leukemia in adults. AML is a diverse disease associated with multiple genetic mutations. It is diagnosed in about 20,000 people every year in the United States.

On January 8, 2024 Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to bringing a first-in-class pipeline of induced pluripotent stem cell (iPSC)-derived cellular immunotherapies to patients with cancer and autoimmune disorders, reported the initiation of enrollment for its Phase 1 clinical trial of FT825 / ONO-8250, a multiplexed-engineered, chimeric antigen receptor (CAR) T-cell product candidate targeting human epidermal growth factor receptor 2 (HER2) (Press release, Fate Therapeutics, JAN 8, 2024, View Source [SID1234639076]). The iPSC-derived CAR T-cell product candidate incorporates a novel HER2-targeted antigen binding domain and is designed to overcome unique challenges in treating solid tumors. The Phase 1 study of FT825 / ONO-8250 is being conducted under a strategic collaboration with Ono Pharmaceutical Co., Ltd. (Ono).

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"Since the formation of our partnership with Ono in 2018, we have worked closely together to pioneer the manufacture of CD8 alpha-beta T cells from iPSCs and to discover and integrate novel synthetic controls of cell function into our iPSC-derived CAR T-cell product platform for safe and effective treatment of solid tumors, including functional elements designed to promote cell trafficking, resist immune suppression in the tumor microenvironment, and preferentially target cancer cells," said Scott Wolchko, President and Chief Executive Officer of Fate Therapeutics. "The preclinical data for FT825 / ONO-8250 indicate a highly-differentiated therapeutic profile across a broad range of solid tumors, with the novel HER2-targeted antigen binding domain demonstrating selective targeting of cancer cells expressing HER2 including those with low expression. We are excited to initiate the Phase 1 study in collaboration with Ono and assess the potential to benefit patients with hard-to-treat advanced solid tumors who currently have limited treatment options."

Designed using the Company’s iPSC Product Platform, FT825 / ONO-8250 incorporates seven novel synthetic controls of cell function including a CXCR2 receptor to promote cell trafficking, a chimeric TGFβ receptor to redirect immunosuppressive signals in the tumor microenvironment, and a high-affinity, non-cleavable CD16a receptor to enable antibody-dependent cellular cytotoxicity. Preclinical data of FT825 / ONO-8250 presented at the 2023 Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting demonstrated that the profile of the novel HER2-targeted antigen binding domain is unique and differentiated from that of trastuzumab, exhibiting similar potency with greater specificity for cancer cells expressing HER2.

The Phase 1 study is designed to investigate a single dose of FT825 / ONO-8250 as monotherapy and in combination with monoclonal antibody therapy in previously-treated patients with advanced solid tumors. The dose escalation and dose expansion portions of the trial are expected to evaluate safety, tolerability, and pharmacokinetics as well as anti-tumor activity by overall response rate, duration of response and disease control rate.

Under the terms of its Collaboration and Option Agreement with Ono, Fate will jointly develop and commercialize FT825 / ONO-8250 with Ono in the U.S. and Europe, and Ono maintains exclusive development and commercialization rights for FT825 / ONO-8250 in the rest of the world. Fate is eligible to receive clinical, regulatory and commercial milestone payments as well as tiered royalties on net sales outside of the United States and Europe by Ono. The parties are currently conducting preclinical development of an additional solid tumor program targeting an undisclosed tumor-associated antigen.

About Fate Therapeutics’ iPSC Product Platform
The Company’s proprietary induced pluripotent stem cell (iPSC) product platform enables mass production of off-the-shelf, multiplexed-engineered cell products that are selectively designed, incorporate novel synthetic controls of cell function, and are intended to deliver multiple mechanisms of therapeutic importance to patients. Human iPSCs possess the unique dual properties of unlimited self-renewal and differentiation potential into all cell types of the body. The Company’s platform combines multiplexed engineering and single-cell selection of human iPSCs to create clonal master iPSC lines. Analogous to master cell lines used to mass produce biopharmaceutical drug products such as monoclonal antibodies, the Company utilizes its clonal master iPSC lines as a renewable cell source to manufacture multiplexed-engineered cell products which are well-defined and uniform in composition, can be stored in inventory for off-the-shelf availability, can be combined and administered with other therapies, and can potentially reach a broad patient population. As a result, the Company’s platform is uniquely designed to overcome numerous limitations associated with the manufacture of cell therapies using patient- or donor-sourced cells. Fate Therapeutics’ iPSC product platform is supported by an intellectual property portfolio of over 400 issued patents and 450 pending patent applications.

On January 8, 2024 Dynavax Technologies Corporation (Nasdaq: DVAX), a commercial-stage biopharmaceutical company developing and commercializing innovative vaccines, reported preliminary, unaudited financial highlights for the fourth quarter and full year ended December 31, 2023 (Press release, Dynavax Technologies, JAN 8, 2024, View Source [SID1234639073]).

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"In 2023, we delivered a record year of revenue for HEPLISAV-B, driven by the expansion of the adult hepatitis B vaccine market in the U.S., and our team’s progress toward establishing HEPLISAV-B as the market leading vaccine. We are extremely pleased with our market share growth in the fourth quarter, which enabled us to achieve our increased product revenue guidance for the year despite the impact of expected seasonality due to increased focus on respiratory disease vaccines during the fall and winter seasons. We believe the seasonal market decline for adult hepatitis B vaccines will be limited to the fourth quarter in line with the administration of the vast majority of influenza and COVID-19 vaccines," said Ryan Spencer, Chief Executive Officer of Dynavax. "Turning to this year, we believe HEPLISAV-B is well-positioned entering 2024, supported by significant market share gains in the total market and in key market segments. We remain extremely confident in the long-term growth of the hepatitis B market, with HEPLISAV-B expected to achieve a majority market share in the U.S. In addition to HEPLISAV-B, we continue to advance our pipeline of innovative vaccine candidates and continue to pursue strategic opportunities to accelerate our growth."

Preliminary Fourth Quarter and Full Year 2023 Financial and Commercial Highlights

Preliminary HEPLISAV-B vaccine net product revenue for the fourth quarter and full year 2023 were approximately $51 million and $213 million, respectively, representing year-over-year growth of approximately 46% and 69% compared to the fourth quarter and full year 2022.
HEPLISAV-B total market share in the U.S. increased to approximately 44%, compared to approximately 35% at the end of 2022.
HEPLISAV-B market share in the retail pharmacy segment increased to approximately 60%, compared to approximately 42% at the end of 2022. HEPLISAV-B market share in the Integrated Delivery Networks (IDNs) and Large Clinics segment increased to approximately 58%, compared to approximately 47% at the end of 2022.
Cash, cash equivalents and marketable securities were approximately $742 million as of December 31, 2023.
The preliminary selected financial results contained herein are unaudited, subject to adjustment, and provided as an estimate in advance of the Company’s announcement of complete financial results, for the three and twelve months ended December 31, 2023. Market share data are preliminary and are as of the latest market data available on December 22, 2023.

Expected Commercial and Pipeline Milestones

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.

Driven by the Centers for Disease Control and Prevention’s Advisory Committee of Immunization Practices (ACIP) universal recommendation for adult hepatitis B vaccination, Dynavax continues to expect the adult hepatitis B vaccine market in the U.S. to expand at an annual growth rate of approximately 10 – 15% over the next several years to a total market of approximately $800 million by 2027, one of the largest adult vaccine markets in the U.S., with HEPLISAV-B well-positioned to achieve a majority market share.
A supplemental Biologic License Application (sBLA) for HEPLISAV-B vaccination of adults on hemodialysis is currently under priority review by the U.S. Food and Drug Administration (FDA) with a Prescription Drug User Fee Act (PDUFA) action date planned for 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 expects to submit an Investigational New Drug Application (IND) to the FDA to support 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 IND to the FDA to support the initiation of a Phase 2 human challenge study of Tdap-1018 in the second half of 2024, upon completion of the independent study conducted by the Canadian Center for Virology to establish the human challenge dose.
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 anticipates top line data for the randomized, active-controlled Phase 2 clinical trial evaluating immunogenicity, safety, and tolerability of the plague vaccine candidate in 2024.
J.P. Morgan Healthcare Conference Presentation Webcast Details
Dynavax will present at the 42nd Annual J.P. Morgan Healthcare Conference on Thursday, January 11 at 11:15 a.m. PT.

The presentation will be webcast and may be accessed through the "Events & Presentations" page on the "Investors" section of the Company’s website at View Source

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 January 8, 2024 Delcath systems presented its corporate presentation (Presentation, Delcath Systems, JAN 8, 2024, View Source [SID1234639072])

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On January 8, 2024 Deciphera Pharmaceuticals, Inc. (NASDAQ: DCPH), a biopharmaceutical company focused on discovering, developing, and commercializing important new medicines to improve the lives of people with cancer, reported its strategic outlook for 2024 and planned 2024 corporate milestones, and announced preliminary unaudited fourth quarter and full year 2023 revenue (Press release, Deciphera Pharmaceuticals, JAN 8, 2024, View Source [SID1234639071]).

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"2023 was a year of important progress across our organization, in which we demonstrated our ability to drive commercial growth while advancing our clinical pipeline and strategically investing in key earlier-stage programs," said Steve Hoerter, President and Chief Executive Officer of Deciphera Pharmaceuticals. "Thanks to our late-stage clinical execution and global commercial excellence, we have the potential to reach $1 billion in peak revenue with QINLOCK and vimseltinib, and we look forward to continuing this exciting evolution as we work to become a self-sustaining, multi-product company."

Planned 2024 corporate milestones and business updates include:

QINLOCK (ripretinib)

Continue enrolling the INSIGHT pivotal Phase 3 clinical study of QINLOCK versus sunitinib in second-line gastrointestinal stromal tumor (GIST) patients with mutations in KIT exon 11 and 17/18 only.
Publication in Nature Medicine in January 2024 of the results of a ctDNA analysis from the INTRIGUE Phase 3 study demonstrating substantial clinical benefit of QINLOCK in second-line GIST patients with mutations in KIT exon 11 and 17/18 only.
Continue the geographic expansion of QINLOCK in fourth-line GIST, with planned commercial launches following conclusion of pricing and reimbursement negotiations in European and international markets.
The Company has entered into a supply and distribution agreement with GENESIS Pharma, a leading regional biopharma company, in Central and Eastern Europe under which GENESIS Pharma will be the exclusive distributor of QINLOCK in 14 countries in the European Union with a combined population of 118 million including Czech Republic, Greece, Hungary, Romania and Poland.
Vimseltinib

Submit an NDA to the U.S. Food and Drug Administration (FDA) in the second quarter of 2024 and a MAA to the European Medicines Agency in the third quarter of 2024 for vimseltinib, an investigational, orally administered, potent, and highly selective switch-control kinase inhibitor of CSF1R, for the potential treatment of TGCT.
Present additional results from Part 1 of the pivotal Phase 3 MOTION study of vimseltinib at a medical meeting in the second quarter of 2024.
Present updated data from the Phase 1/2 study of vimseltinib in TGCT in the second half of 2024.
Initiate a Phase 2 proof-of-concept study of vimseltinib for the treatment of cGVHD in the fourth quarter of 2024.
Early-Stage Pipeline

DCC-3116

The Company expects to select a recommended Phase 2 dose for expansion cohort(s) for DCC-3116, an investigational switch-control kinase inhibitor of ULK1/2 designed to inhibit autophagy, in 2024.
The Company has prioritized the development of DCC-3116 in combination with sotorasib and with QINLOCK and discontinued development of the encorafenib and cetuximab combination cohort prior to enrollment in any clinical studies as well as the two MEK combination cohorts.
DCC-3084

Initiate a Phase 1 study for DCC-3084, a potential best-in-class pan-RAF inhibitor, in the first half of 2024.
DCC-3009

Submit an investigational new drug (IND) application with the FDA for DCC-3009, a potential best-in-class pan-KIT inhibitor, in the first half of 2024 and initiate a Phase 1 study in the second half of 2024.
Preliminary 2023 Financial Results

Based on preliminary unaudited financial information, Deciphera expects total fourth quarter 2023 revenue to be approximately $47 million and total full year 2023 revenue to be approximately $162 million. QINLOCK net product revenue is estimated to be approximately $46 million in the fourth quarter 2023, including approximately $35 million in U.S. net product revenue and approximately $11 million in international net product revenue, in addition to approximately $1 million in collaboration revenue.

In addition, preliminary unaudited cash, cash equivalents, and marketable securities was approximately $352 million as of December 31, 2023. Based on its current operating plans, Deciphera expects its current cash, cash equivalents, and marketable securities together with anticipated product, royalty, and supply revenues, but excluding any potential future milestone payments under its collaboration or license agreements, will enable the Company to fund its operating and capital expenditures into the second half of 2026.

Preliminary selected financial information presented in this release are unaudited, subject to adjustment, and provided as an approximation in advance of the Company’s announcement of complete financial results expected in February 2024.