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.

On January 8, 2024 Day one presented its corporate presentation (Presentation, Day One, JAN 8, 2024, View Source [SID1234639070]).

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On January 8, 2024 CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, reported its strategic priorities and 2024 outlook as the Company enters its next phase of growth (Press release, CRISPR Therapeutics, JAN 8, 2024, View Source [SID1234639069]).

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"We had a landmark year in 2023, marked by the first-ever approval of a CRISPR-based gene-editing therapy in addition to entering the clinic with our in vivo therapies," said Samarth Kulkarni, Ph.D., Chairman and Chief Executive Officer of CRISPR Therapeutics. "As we look ahead to 2024, we continue to drive forward our programs and expand our pipeline with the goal of delivering paradigm-shifting gene editing therapies to patients. We are well positioned to execute on our clinical trials across various therapeutic areas, including oncology, autoimmune, cardiovascular and diabetes, setting up a catalyst-rich 12-18 months for the company. In parallel, we are continuously innovating on our platform with next-generation gene editing and delivery technologies that could enable us to address even more diseases with potentially curative medicines. Even in this challenging macroeconomic environment for biotech companies, our strong capital position and efficient operating model provides us with a competitive advantage to expand upon our leadership in the space."

Strategic Priorities and 2024 Outlook

CASGEVY (exagamglogene autotemcel [exa-cel])

Received regulatory approvals for CASGEVY in the fourth quarter of 2023 in the U.S. for sickle cell disease (SCD) and in Great Britain and Bahrain for the treatment of SCD and transfusion-dependent beta thalassemia (TDT); also received a positive opinion from the European Medicines Agency’s (EMA’s) Committee for Medicinal Products for Human Use (CHMP) for CASGEVY for both SCD and TDT from the European Medicines Agency (EMA). Exa-cel is the first therapy to emerge from a strategic partnership between CRISPR Therapeutics and Vertex Pharmaceuticals. Vertex leads global development, manufacturing, regulatory and commercialization of CASGEVY with support from CRISPR Therapeutics.
The FDA has assigned a Prescription Drug User Fee Act (PDUFA) action date of March 30, 2024, for CASGEVY in TDT. Additional regulatory submissions for CASGEVY are currently under review in Switzerland and the Kingdom of Saudi Arabia, with the submission in Canada planned for the first half of 2024.
Completed enrollment in two global Phase 3 studies of CASGEVY in patients 5 to 11 years of age with SCD or TDT.
Vertex is engaging with experienced hospitals to establish a network of authorized treatment centers (ATCs) throughout the U.S. to offer CASGEVY to patients. Nine ATCs have been activated in the U.S. and three in Europe, with the goal of activating approximately 50 ATCs in the U.S. and 25 in the EU. Additionally, Vertex announced an agreement with a major medication contracting organization, Synergie Medication Collective, which covers nearly 100 million lives in the U.S., to provide access to CASGEVY through an outcomes-based contract.
Hemoglobinopathies

CRISPR Therapeutics has two next-generation research focuses that each have the potential to expand the addressable population with SCD and TDT significantly. First, the Company continues to advance its internal targeted conditioning program, an anti-CD117 (c-Kit) antibody-drug conjugate (ADC), through preclinical studies.
Second, the Company has ongoing research efforts to enable in vivo editing of hematopoietic stem cells. This work, supported in part by a $14.5 million grant from the Bill & Melinda Gates Foundation, could obviate the need for conditioning altogether, expand geographic reach, and enable the treatment of multiple additional other diseases beyond SCD and TDT.
Immuno-Oncology and Autoimmune Disease

CRISPR Therapeutics’ next-generation allogeneic CAR T candidates reflect the Company’s mission of innovating continuously to bring potentially transformative medicines to patients as quickly as possible. Clinical trials are ongoing for CRISPR Therapeutics’ next-generation CAR T product candidates, CTX112 and CTX131, targeting CD19 and CD70, respectively. Emerging pharmacology data, including pharmacokinetics, indicate that the novel potency gene edits in CTX112 and CTX131 can lead to significantly higher CAR T cell expansion and functional persistence in patients compared to the first-generation candidates. Focusing efforts on these candidates will enable the Company to advance these potentially best-in-class CAR T therapies more efficiently and rapidly. The Company expects to provide a clinical update in 2024 for these next-generation candidates.
CRISPR Therapeutics plans to initiate a clinical trial of CTX112 in systemic lupus erythematosus (SLE) in the first half of 2024, with the potential to expand into additional autoimmune indications in the future. Early clinical studies have shown that CD19-directed autologous CAR T therapy can produce long-lasting remissions in multiple autoimmune indications.
CRISPR Therapeutics is expanding trials of CTX131 into hematologic malignancies, including T- and B-cell malignancies, in addition to the ongoing clinical trial in solid tumors.
In Vivo

CRISPR Therapeutics is advancing a pipeline of in vivo gene editing programs using lipid nanoparticle (LNP) delivery of Cas9 mRNA and a guide RNA (gRNA) to the liver. Its first two in vivo programs, CTX310 and CTX320, each aim to reduce expression of a validated target for cardiovascular disease. Beginning with these programs, CRISPR Therapeutics aims to transform the treatment paradigm for cardiovascular indications and beyond with potential one-time therapies that could recapitulate the proven benefit of targets validated by natural human genetics and other therapeutic modalities.
A Phase 1 clinical trial is ongoing for CTX310, targeting angiopoietin-like 3 protein (ANGPTL3). In humans, naturally occurring loss-of-function variants in ANGPTL3 are associated with reduced levels of serum lipids and reduced risk of atherosclerotic cardiovascular disease. The Phase 1 trial is enrolling patients with mixed dyslipidemias, homozygous familial hypercholesterolemia, and severe hypertriglyceridemia.
CRISPR Therapeutics has initiated a Phase 1 clinical trial for CTX320, an investigational program targeting lipoprotein(a) (Lp(a)). Elevated Lp(a), which is associated with an increased risk of atherosclerotic cardiovascular disease, is present in approximately one in five people in the United States and around the world.
CRISPR Therapeutics expects to nominate additional in vivo programs targeting both rare and common diseases this year, to be disclosed in mid-2024.
Regenerative Medicine

CRISPR Therapeutics announced today that ViaCyte, Inc. (a subsidiary of Vertex Pharmaceuticals, Inc.) has elected to opt-out of the collaboration with CRISPR Therapeutics for the co-development and co-commercialization of gene-edited stem cell therapies for the treatment of diabetes. Per the opt-out terms, the on-going collaboration assets will now be wholly owned by CRISPR Therapeutics, subject to a royalty on future sales owed to ViaCyte. The opt-out will become effective in early February. The ViaCyte collaboration assets include CTX211 (formerly VCTX211), an allogeneic, gene-edited, immune-evasive, stem cell derived product candidate that is transplanted into patients in a device and intended to produce insulin in a glucose-dependent manner. CRISPR Therapeutics continues to advance a Phase 1 clinical trial for CTX211 for the treatment of Type 1 Diabetes (T1D). CRISPR Therapeutics remains committed to its goal of developing a beta-cell replacement product that does not require chronic immunosuppression.
Separate from the ViaCyte collaboration, Vertex continues to have non-exclusive rights to certain CRISPR Therapeutics’ CRISPR/Cas9 technology to accelerate development of potentially curative cell therapies for T1D. Vertex paid $170 million to CRISPR Therapeutics in upfront and milestone payments in 2023 as part of that licensing agreement, and CRISPR Therapeutics remains eligible for an additional $160 million in research and development milestones and would receive royalties on any future products resulting from this agreement.
Manufacturing

CRISPR Therapeutics’ state-of-the-art Good Manufacturing Practice (GMP) facility located in Framingham, MA is fully operational, and continues to support the production of the Company’s various investigational therapies. Potential benefits of this facility include significantly lower cost of goods, increased flexibility and greater scalability.
The Company’s next-generation allogeneic CAR T candidates manufactured at its internal GMP facility exhibit increased manufacturing robustness, yield and scalability.
Cash Position and Operating Efficiencies

CRISPR Therapeutics begins 2024 in a strong financial position with approximately $1.9 billion in cash, cash equivalents and marketable securities, which is inclusive of a $200 million milestone payments received in January related to the FDA approval of CASGEVY.
CRISPR Therapeutics continues to focus on resource efficiency and return on invested capital as it advances multiple clinical programs across its pipeline.
About CASGEVY (exagamglogene autotemcel [exa-cel])

CASGEVY is a genome-edited cellular therapy consisting of autologous CD34+ hematopoietic stem cells (HSCs) edited by CRISPR/Cas9 technology at the erythroid-specific enhancer region of the BCL11A gene. CASGEVY is intended for one time administration via a hematopoietic stem cell transplant procedure where the patient’s own CD34+ cells are modified to reduce BCL11A expression in erythroid lineage cells, leading to increased fetal hemoglobin (HbF) production. HbF is the form of the oxygen-carrying hemoglobin that is naturally present during fetal development, which then switches to the adult form of hemoglobin after birth. CASGEVY has been shown to reduce or eliminate vaso-occlusive crises for patients with SCD.

CASGEVY is approved in the U.S. to treat people aged 12 years and older with SCD who have recurrent VOCs. CASGEVY was granted a conditional marketing authorization in Great Britain by the U.K. Medicines and Healthcare products Regulatory Agency and by the National Health Regulatory Authority in Bahrain for patients 12 years of age and older with SCD characterized by recurrent vaso-occlusive crises or transfusion-dependent beta thalassemia (TDT), for whom hematopoietic stem cell transplantation is appropriate and a human leukocyte antigen matched related hematopoietic stem cell donor is not available. CASGEVY is currently under review by the European Medicines Agency and the Saudi Food and Drug Authority for both SCD and TDT.

The use of CASGEVY for the treatment of TDT in the U.S. remains investigational. Vertex has submitted a BLA to the U.S. FDA for the potential use of CASGEVY for patients 12 years and older with TDT and has been assigned a Prescription Drug User Fee Act (PDUFA) target action date of March 30, 2024.