On February 22, 2024 Guardant Health, Inc. (Nasdaq: GH), a leading precision oncology company, reported financial results for the quarter and full year ended December 31, 2023 (Press release, Guardant Health, FEB 22, 2024, View Source [SID1234640387]).

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Fourth Quarter 2023 Financial Highlights
•Revenue of $155.1 million for the fourth quarter of 2023, an increase of 22% over the fourth quarter of 2022
•Reported 46,400 tests to clinical customers and 9,500 tests to biopharmaceutical customers in the fourth quarter of 2023, representing increases of 29% and 16%, respectively, over the fourth quarter of 2022
Full Year 2023 Financial Highlights
•Revenue of $563.9 million for the full year 2023, an increase of 25% over the full year 2022
•Reported 172,900 tests to clinical customers and 29,900 tests to biopharmaceutical customers in the full year 2023, representing an increase of 39% and 15%, respectively, over the full year 2022
•Lowered full year 2023 operating expenses and improved free cash flow compared to full year 2022
•Ended the year with $1.2 billion in cash, cash equivalents and marketable debt securities
Recent Operating Highlights
•Improved ASPs with Guardant360 LDT CMS reimbursement increased from $3,500 to $5,000 effective January 1, 2024
•Published first paper for Shield demonstrating improved adherence with blood-based CRC screening
•Collaborated with US Oncology Network and leading community oncology practices to increase the use of biomarker testing to identify patients who would benefit from therapies
•Appointed Terilyn Juarez Monroe as chief people officer
"2023 marked another strong year for Guardant. This was underscored by our team’s exceptional execution to deliver strong clinical volumes, which grew nearly 40%," said Helmy Eltoukhy, co-founder and co-CEO. "We also surpassed an important milestone by achieving cash flow breakeven in our Therapy Selection business by year-end. Looking ahead to 2024, we are well positioned for continued growth across our oncology product portfolio and have a strong foundation to deliver long-term shareholder value."
"We are continuing to make steady progress with FDA on our PMA submission for Shield and look forward to the upcoming Advisory Committee Panel as the next phase of the review process," said AmirAli Talasaz, co-founder and co-CEO. "2024 will be a pivotal year for our screening business as we prepare to launch our Shield IVD test following expected FDA approval. We are excited to see the impact our test will have to CRC screening when it is broadly launched later this year."
Fourth Quarter 2023 Financial Results
Revenue was $155.1 million for the fourth quarter of 2023, a 22% increase from $126.9 million for the corresponding prior year period. Precision oncology revenue grew 25%, to $142.2 million for the fourth quarter of 2023, from $113.8 million for the corresponding prior year period, driven predominantly by an increase in clinical and biopharma testing volume, which grew 29% and 16%, respectively, over the prior year period. Development services and other revenue was $12.9 million for the fourth quarter of 2023, compared to $13.1 million for the corresponding prior year period.
Gross profit, or total revenue less cost of precision oncology testing and cost of development services and other, was $92.5 million for the fourth quarter of 2023, an increase of $12.8 million from $79.8 million for the corresponding prior year period. Gross margin, or gross profit divided by total revenue, was 60%, as compared to 63% for the corresponding prior year period. Precision oncology gross margin was 60% in the fourth quarter of 2023, as compared to 62% in the prior year period. The reduction is primarily due to changes in product mix. Development services and other gross margin was 60% in the fourth quarter of 2023, as compared to 74% in the prior year period. The change is primarily due to the cost of processing an increased volume of Shield LDT tests, for which we are currently booking minimal revenue.

Operating expenses (research and development expense, sales and marketing expense, and general and administrative expense) were $206.6 million for the fourth quarter of 2023, as compared to $225.9 million for the corresponding prior year period. Other operating expense was $83.4 million for the fourth quarter of 2023, related to a non-recurring legal accrual. No such other operating expense was recorded for the corresponding prior year period. Non-GAAP operating expenses were $183.1 million for the fourth quarter of 2023, as compared to $201.2 million for the corresponding prior year period.
Net loss was $187.0 million for the fourth quarter of 2023, as compared to $139.9 million for the corresponding prior year period. Net loss per share was $1.58 for the fourth quarter of 2023, as compared to $1.36 for the corresponding prior year period. The year-over-year increase in net loss is primarily due to the non-recurring $83.4 million legal accrual, partially offset by a $32.0 million year over year improvement in loss from operations, excluding the legal accrual impact, and a $11.7 million increase in interest income.
Non-GAAP net loss was $75.9 million for the fourth quarter of 2023, as compared to $119.6 million for the corresponding prior year period. Non-GAAP net loss per share was $0.64 for the fourth quarter of 2023, as compared to $1.17 for the corresponding prior year period.
Adjusted EBITDA loss was $78.4 million for the fourth quarter of 2023, as compared to a $109.8 million loss for the corresponding prior year period.
Free cash flow for the fourth quarter of 2023 was negative $82.8 million, as compared to negative $100.8 million for the corresponding prior year period. Cash, cash equivalents and marketable debt securities were $1.2 billion as of December 31, 2023.

Full Year 2023 Financial Results

Revenue was $563.9 million for 2023, a 25% increase from $449.5 million for the corresponding prior year period. Precision oncology revenue grew 31%, to $514.2 million for 2023, from $392.0 million for the corresponding prior year period, driven predominantly by an increase in clinical and biopharma testing volume, which grew 39% and 15%, respectively, over the prior year period. Development services and other revenue decreased by 14%, to $49.7 million for 2023, from $57.5 million for the corresponding prior year period, primarily due to the timing and amount of milestones related to our companion diagnostics collaboration projects and other service agreements with biopharmaceutical customers, and a reduction in royalty revenue.

Gross profit, or total revenue less cost of precision oncology testing and cost of development services and other, was $336.9 million for 2023, an increase of $43.7 million from $293.2 million for the corresponding prior year period. Gross margin, or gross profit divided by total revenue, was 60%, as compared to 65% for the corresponding prior year period. Precision oncology gross margin was 60% in the year of 2023, as compared to 62% in the prior year period. The reduction is primarily due to changes in product mix. Development services and other gross margin was 57% in the year of 2023, as compared to 86% in the prior year period. The change is primarily due to the inclusion of the full year 2023 cost of processing Shield LDT samples as part of our screening market development activities, for which we are currently booking minimal revenue, compared to the inclusion of the fourth quarter only cost in 2022.
Operating expenses (research and development expense, sales and marketing expense, and general and administrative expense) were $818.2 million for 2023, as compared to $837.6 million for the corresponding prior year period. Other operating expense was $83.4 million for 2023, related to the non-recurring legal accrual discussed above. No such other operating expense was recorded for the corresponding prior year period. Non-GAAP operating expenses were $729.2 million for 2023, as compared to $736.6 million for the corresponding prior year period.
Net loss was $479.4 million for 2023, as compared to $654.6 million for the corresponding prior year period. Net loss per share was $4.28 for 2023, as compared to $6.41 for the corresponding prior year period. The year-over-year improvement in net loss is primarily due to a $99.8 million fair value adjustment related to the Joint Venture acquisition recorded in 2022, a $87.5 million positive change in unrealized gains and losses, a $63.1 million year over year improvement in loss from operations, excluding the legal accrual impact, and a $29.3 million increase in interest income, partially offset by the $83.4 million legal accrual discussed above, and a $23.8 million increase in impairment on non-marketable equity securities.
Non-GAAP net loss was $352.3 million for 2023, as compared to $435.4 million for the corresponding prior year period. Non-GAAP net loss per share was $3.15 for 2023, as compared to $4.26 for the corresponding prior year period.
Adjusted EBITDA loss was $344.2 million for 2023, as compared to a $403.4 million loss for the corresponding prior year period.
Free cash flow for 2023 was negative $345.5 million, as compared to negative $386.9 million for the corresponding prior year period.

2024 Guidance
Guardant Health expects full year 2024 revenue excluding screening to be in the range of $655 to $670 million, representing growth of 16% to 19% compared to full year 2023, and full year 2024 non-GAAP gross margin excluding screening to be in the range of 60% to 62%. Guardant Health expects total non-GAAP operating expenses to be in the range of $740 to $750 million, representing a 1% to 3% increase compared to full year 2023, and free cash flow to be in the range of negative $320 to $330 million in 2024, an improvement compared to the full year 2023.
Webcast Information
Guardant Health will host a conference call to discuss the fourth quarter and full year 2023 financial results after market close on Thursday, February 22, 2024 at 1:30 pm Pacific Time / 4:30 pm Eastern Time. A webcast of the conference call can be accessed at View Source The webcast will be archived and available for replay for at least 90 days after the event.
Non-GAAP Measures
Guardant Health has presented in this release certain financial information in accordance with U.S. Generally Accepted Accounting Principles (GAAP) and also on a non-GAAP basis, including non-GAAP cost of precision oncology testing, non-GAAP cost of development services and other, non-GAAP cost of screening, non-GAAP gross profit, non-GAAP gross profit excluding cost of screening, non-GAAP research and development expense, non-GAAP sales and marketing expense, non-GAAP general and administrative expense, non-GAAP other operating expense, non-GAAP loss from operations, non-GAAP net loss, non-GAAP net loss per share, basic and diluted, adjusted EBITDA, and free cash flow.

We define our non-GAAP measures as the applicable GAAP measure adjusted for the impacts of stock-based compensation and related employer payroll tax payments, contingent consideration, amortization of intangible assets, fair value adjustments on marketable equity securities, impairment of non-marketable equity securities and other related assets, fair value adjustments of noncontrolling interest liability, and other non-recurring items.
Adjusted EBITDA is defined as net loss adjusted for interest income; interest expense; other income (expense), net; provision for (benefit from) income taxes; depreciation and amortization expense; stock-based compensation expense and related employer payroll tax payments; contingent consideration; fair value adjustments of noncontrolling interest liability; and other non-recurring items. Free cash flow is defined as net cash used in operating activities in the period less purchase of property and equipment in the period.

We believe that the exclusion of certain income and expenses in calculating these non-GAAP financial measures can provide a useful measure for investors when comparing our period-to-period core operating results, and when comparing those same results to that published by our peers. We exclude certain items because we believe that these income and expenses do not reflect expected future operating performance. Additionally, certain items are inconsistent in amounts and frequency, making it difficult to perform a meaningful evaluation of our current or past operating performance. We use these non-GAAP financial measures to evaluate ongoing operations, for internal planning and forecasting purposes, and to manage our business.

These non-GAAP financial measures are not intended to be considered in isolation from, as substitute for, or as superior to, the corresponding financial measures prepared in accordance with GAAP. There are limitations inherent in non-GAAP financial measures because they exclude charges and credits that are required to be included in a GAAP presentation, and do not present the full measure of our recorded costs against its revenue. In addition, our definition of the non-GAAP financial measures may differ from non-GAAP measures used by other companies.

On February 22, 2024 Elevation Oncology, Inc. (Nasdaq: ELEV), an innovative oncology company focused on the discovery and development of selective cancer therapies to treat patients across a range of solid tumors with significant unmet medical needs, reported that it has expanded its ongoing Phase 1 clinical trial of EO-3021 outside the United States, dosing the first patient in Japan (Press release, Elevation Oncology, FEB 22, 2024, View Source;utm_medium=rss&utm_campaign=elevation-oncology-expands-ongoing-phase-1-clinical-trial-of-eo-3021-globally-dosing-first-patient-in-japan [SID1234640386]). This trial is evaluating the safety, tolerability and preliminary anti-tumor activity of EO-3021 in patients with advanced, unresectable or metastatic solid tumors likely to express Claudin 18.2, including gastric, gastroesophageal junction, pancreatic or esophageal cancers.

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"We are pleased to expand our clinical development efforts outside of the United States and into Japan where there is a large number of patients with gastric cancer who could benefit from EO-3021," said Valerie Malyvanh Jansen, M.D., Ph.D., Chief Medical Officer of Elevation Oncology. "Claudin 18.2 is expressed in most gastric adenocarcinomas and is increasingly recognized as an important therapeutic target, which can be effectively addressed with an antibody drug conjugate (ADC). We are excited to have dosed our first patient in Japan, as we continue to execute on our mission of delivering novel, selective cancer therapies that can offer better outcomes to patients."

"Despite recent advancements in the treatment of gastric cancer and improvements in mortality, there remains a significant need for new treatment options that offer patients improved outcomes," said Kohei Shitara, M.D., Chief, Department of Gastrointestinal Oncology, National Cancer Center Hospital East in Kashiwa, Japan and principal investigator on the Phase 1 clinical trial, "Based on preclinical and early clinical data, I believe EO-3021 has the potential to overcome the limitations of other therapeutic modalities to deliver benefit to patients with varying levels of Claudin 18.2 expression. I am excited to dose patients with EO-3021 and to have access to this much-needed option for patients in Japan."

Elevation Oncology’s Phase 1 clinical trial (NCT05980416) is an open-label, multi-center dose escalation and expansion study to evaluate the safety, tolerability and preliminary anti-tumor activity of EO-3021. An additional objective of the study will be to assess the association of Claudin 18.2 expression and objective response. The study is recruiting patients with advanced unresectable or metastatic solid tumors likely to express Claudin 18.2, including gastric, gastroesophageal junction, pancreatic or esophageal cancers, at multiple sites in the United States and Japan. Elevation Oncology expects to provide an update from the trial in mid-2024, with additional data expected in the first half of 2025.

About EO-3021

EO-3021 (also known as SYSA1801) is a differentiated, clinical-stage antibody drug conjugate (ADC) with best-in-class potential comprised of an immunoglobulin G1 (IgG1) monoclonal antibody (mAb) that targets Claudin 18.2. EO-3021 is site-specifically conjugated to the monomethyl auristatin E (MMAE) payload via a cleavable linker with a drug-to-antibody ratio (DAR) of 2. Claudin 18.2 is a specific isoform of Claudin 18 that is normally expressed in gastric epithelial cells. During malignant transformation, the tight junctions may become disrupted, exposing Claudin 18.2 and allowing them to be accessible by Claudin 18.2 targeting agents. Elevation Oncology is evaluating EO-3021 in a Phase 1 study (NCT05980416) in patients with advanced, unresectable or metastatic solid tumors likely to express Claudin 18.2 including gastric, gastroesophageal junction, pancreatic or esophageal cancers.

Elevation Oncology has the exclusive rights to develop and commercialize EO-3021 in all global territories outside Greater China.

On February 22, 2024 Dynavax Technologies Corporation (Nasdaq: DVAX), a commercial-stage biopharmaceutical company developing and commercializing innovative vaccines, reported financial results for the fourth quarter and full year ended December 31, 2023 (Press release, Dynavax Technologies, FEB 22, 2024, View Source [SID1234640385]).

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"2023 was characterized by record revenue growth for HEPLISAV-B, and the achievement of becoming the market share leader in the two largest growth segments, demonstrating important progress toward our goal of establishing HEPLISAV-B as the leading vaccine in the U.S. adult hepatitis B vaccine market," said Ryan Spencer, Chief Executive Officer of Dynavax. "We expect 2024 to be an important year in building a vaccine portfolio of best-in-class products, including further growing the HEPLISAV-B brand as well as advancing our pipeline programs into clinical trial initiations and data readouts. Importantly, our strong financial position provides us with the optionality to continue to build value across our business, including through investing to drive the HEPLISAV-B market opportunity, advancing and expanding our R&D efforts, and pursuing strategic opportunities to accelerate our growth."

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 vaccine net product revenue for the fourth quarter and full year 2023 were approximately $51.1 million and $213.3 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 42% at the end of 2023, compared to approximately 35% at the end of 2022.
HEPLISAV-B market share in the retail pharmacy segment increased to approximately 58% at the end of 2023, 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 56% at the end of 2023, compared to approximately 47% at the end of 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 planned for May 13, 2024.
Driven by the Centers for Disease Control and Prevention’s Advisory Committee of Immunization Practices (ACIP) universal recommendation for adult hepatitis B vaccination, the hepatitis B vaccine market continues to expand in the U.S. and Dynavax believes the U.S. market has the potential to grow to approximately $800 million by 2027, with HEPLISAV-B well-positioned to achieve a majority market share.
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 submitted an Investigational New Drug Application (IND) to the U.S. 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 U.S. 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 Vaccinology 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.
FOURTH QUARTER AND FULL YEAR 2023 FINANCIAL HIGHLIGHTS

Total Revenues and Net Product Revenue.

HEPLISAV-B vaccine net product revenue was $51.1 million for the fourth quarter of 2023, compared to $34.9 million for the fourth quarter of 2022, and $213.3 million for the full year 2023, compared to $125.9 million for the full year 2022.
Other revenue was $4.5 million for the fourth quarter of 2023, compared to $2.3 million for the fourth quarter of 2022, and $19.0 million for the full year 2023, compared to $9.0 million for the full year 2022. Other revenue primarily includes revenue from the 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 fourth quarter and full year 2023, compared to $147.2 million and $587.7 million in the same periods of 2022, respectively, due to completion of all obligations and product delivery under the Company’s CpG 1018 adjuvant COVID-19 collaboration agreements as of the end of 2022.
Total revenues for the fourth quarter of 2023 were $55.6 million, compared to $184.5 million for the fourth quarter of 2022, and $232.3 million for the full year 2023, compared to $722.7 million for the full year 2022.
Cost of Sales – Product. Cost of sales – product for HEPLISAV-B the fourth quarter of 2023 decreased to $8.7 million, compared to $12.4 million for the fourth quarter of 2022, and $50.2 million for the full year 2023, compared to $40.1 million for the full year 2022.

Research and Development Expenses (R&D). R&D expenses for the fourth quarter of 2023 increased to $14.1 million, compared to $12.9 million for the fourth quarter of 2022, and $54.9 million for the full year 2023, compared to $46.6 million for the full year 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 fourth quarter of 2023 increased to $41.3 million, compared to $31.0 million for the fourth quarter of 2022, and $152.9 million for the full year 2023, compared to $131.4 million for the full year 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 $0.2 million, or less than $0.01 per share (basic and diluted) in the fourth quarter of 2023, compared to GAAP net income of $67.7 million, or $0.53 per share (basic) and $0.45 per share (diluted) in the fourth quarter of 2022. GAAP net loss was $6.4 million, or $0.05 per share (basic and diluted) for the full year 2023, compared to GAAP net income of $293.2 million, or $2.32 per share (basic) and $1.97 per share (diluted) for the full year 2022.

Cash and Marketable Securities. Cash, cash equivalents and marketable securities were $742.3 million as of December 31, 2023.

2024 FINANCIAL GUIDANCE

Dynavax is providing the following full year 2024 financial guidance, based on the Company’s current operating plan:

HEPLISAV-B net product revenue between approximately $265 – $280 million, including approximately $3 million in ex-U.S. sales through commercialization agreement with Bavarian Nordic in Germany
HEPLISAV-B gross margin of approximately 80% for full year 2024
Research and development expenses between approximately $60 – $75 million
Selling, general and administrative expenses between approximately $160 – $180 million
Expect to be cash flow positive for full year ended December 31, 2024
Conference Call and Webcast Information

Dynavax will host a conference call and live audio webcast on Thursday, February 22, 2024, 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 February 22, 2024 C4 Therapeutics, Inc. (C4T) (Nasdaq: CCCC), a clinical-stage biopharmaceutical company dedicated to advancing targeted protein degradation science, reported financial results for the year ended December 31, 2023, as well as recent business updates (Press release, C4 Therapeutics, FEB 22, 2024, View Source [SID1234640384]).

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"2023 was an important year for C4T as we executed across three clinical trials, entered into two new collaborations, generated positive dose escalation data from our CFT7455 program for patients with relapsed/refractory multiple myeloma, and meaningfully extended our cash runway," said Andrew Hirsch, president and chief executive officer of C4 Therapeutics. "We began 2024 with positive momentum across our portfolio and are looking forward to sharing data from our two lead programs, CFT7455 and CFT1946, in the second half of the year, as well as supporting our partner, Betta Pharmaceuticals, with trial start-up activities for the Phase 1 trial of CFT8919 in Greater China this year. As we continue to advance our portfolio, we are well-positioned with a strong balance sheet to deliver on our goals and execute through and beyond meaningful value inflection points in order to bring new therapeutic options to patients with difficult-to-treat diseases."

FOURTH QUARTER 2023 AND RECENT ACHIEVEMENTS

CFT7455: CFT7455 is an oral degrader of IKZF1/3 for the potential treatment of relapsed/refractory (R/R) multiple myeloma (MM) and R/R non-Hodgkin’s lymphomas (NHL).

Presented Positive Data from the Ongoing Phase 1/2 Trial in R/R MM. In December 2023, presented positive clinical data from the ongoing CFT7455 Phase 1/2 trial in R/R MM showing that the 14 days on/14 days off schedule is optimal. Additionally, the data demonstrated anti-myeloma activity, including International Myeloma Working Group (IMWG) responses in patients who have undergone numerous lines of prior therapy for MM, including BCMA therapies.
Advanced the Phase 1/2 Clinical Trials. The dose escalation portion of the Phase 1/2 trials evaluating CFT7455 in combination with dexamethasone for R/R MM and as a monotherapy for R/R NHL continues to progress. As of February 2024, two dose levels are open for enrollment in the Phase 1/2 trial for R/R MM and one dose level open for enrollment in the Phase 1/2 trial for R/R NHL.
CFT1946: CFT1946 is an oral degrader targeting BRAF V600X mutations for the potential treatment of solid tumors including non-small cell lung cancer (NSCLC), colorectal cancer (CRC) and melanoma.

Shared Encouraging Initial Pharmacokinetic (PK) and Pharmacodynamic (PD) Data. In January 2024, shared PK and PD data from the initial escalation cohorts of the CFT1946 Phase 1/2 trial demonstrating dose proportional exposure and oral bioavailability, which are associated with deep degradation of BRAF V600E, a clinically validated target.
New Preclinical Data Accepted as a Poster at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2024. Accepted to present CFT1946 preclinical data demonstrating differentiated activity in preclinical models of BRAF V600X melanoma, CRC, NSCLC and brain metastasis.
Advanced the Phase 1/2 Clinical Trial. The dose escalation portion of the CFT1946 Phase 1/2 trial for BRAF V600X mutations, including NSCLC, CRC and melanoma, continues to progress. As of February 2024, three escalation cohorts are complete and dose escalation continues with a fourth dose level currently enrolling.
Collaborations:

Merck

In December 2023, C4T and Merck entered into a license and research collaboration to discover and develop degrader antibody conjugates. Under the terms of the agreement, C4T and Merck will collaborate to develop degrader antibody conjugates directed to an initial undisclosed oncology target exclusive to the collaboration; in January 2024, C4T received the $10 million upfront payment for this initial target. C4T is eligible to receive milestone payments totaling approximately $600 million, as well as tiered royalties on future sales, for degrader antibody conjugates directed to this initial target. The agreement also provides Merck with the option to extend the collaboration to include three additional targets that would be exclusive to the collaboration, which could yield option exercise payments as well as potential milestones and royalties. If Merck exercises all of its options to extend the collaboration, C4T would be eligible to receive up to approximately $2.5 billion in potential payments across the entire collaboration.
Betta Pharmaceuticals

In January 2024, the previously announced $25 million stock purchase by a subsidiary of partner Betta Pharmaceuticals was completed.
In December 2023, Betta Pharmaceuticals received approval from the Chinese National Medical Products Administration for the Clinical Trial Application of CFT8919, which is being evaluated in patients with EGFR L858R NSCLC.
Corporate Updates:

In January 2024, C4T announced 2024 strategic priorities focused on advancing product candidates CFT7455 and CFT1946, delivering on discovery collaborations, and streamlined internal research efforts, which resulted in an approximately 30% reduction in the company’s workforce.
As announced in January 2024, C4T sold approximately 13.7 million shares under the company’s at the market (ATM) offering arrangement, at an average price of $5.42 per share, resulting in approximately $72 million of new equity capital, net of commissions and fees. As of December 31, 2023, 11.2 million of these shares had settled for net proceeds of $57.7 million.
In November 2023, C4T appointed Owen Hughes to its board of directors. Mr. Hughes is an accomplished life sciences executive with nearly three decades of experience in investing, operations and corporate governance.
KEY UPCOMING MILESTONES

CFT7455:

Present updated data from the ongoing Phase 1 dose escalation trial in R/R MM in 2H 2024.
Present data from the ongoing Phase 1 dose escalation trial in R/R NHL in 2H 2024.
Complete Phase 1 dose exploration in R/R MM and R/R NHL by year-end 2024.
CFT1946:

Present preclinical data demonstrating differentiated activity in preclinical models of BRAF V600X NSCLC, CRC, melanoma and brain metastasis at the AACR (Free AACR Whitepaper) Annual Meeting taking place April 5 – 10, 2024 in San Diego, CA.
Present clinical data from the ongoing Phase 1 dose escalation trial in NSCLC, CRC, melanoma and other cancers with BRAF V600X mutations in 2H 2024.
UPCOMING INVESTOR EVENTS

March 4, 2024: Management will present at TD Cowen’s 44th Annual Health Care Conference taking place March 4 – 6, 2024, at the Marriott Copley Place in Boston, MA.
March 11, 2024: Management will participate in a fireside chat at the Leerink Partners Global Biopharma Conference taking place March 11 – 13, 2024, at the Fontainebleau in Miami, FL.
FULL YEAR 2023 FINANCIAL RESULTS

Revenue: Total revenue for the year ended December 31, 2023 was $20.8 million, compared to $31.1 million for the year ended December 31, 2022. The decrease in revenue was primarily due to the collaboration agreement with Calico ending in January 2023 and completion of research activities for a target under the collaboration agreement with Biogen, partially offset by the completion of research activities for select targets under the collaboration agreement with Roche. 2023 revenue reflects amounts recognized under our collaboration agreements with Biogen, Calico, and Roche.

Research and Development (R&D) Expense: R&D expense for the year ended December 31, 2023 was $117.7 million, compared to $117.8 million for the year ended December 31, 2022. R&D expense was relatively flat year over year as preclinical costs decreased and clinical costs increased with the transition of CFT1946 to clinical development.

General and Administrative (G&A) Expense: G&A expense for the year ended December 31, 2023 was $42.1 million, compared to $42.8 million for the year ended December 31, 2022. The decrease in G&A expense was primarily attributable to a decrease in professional fees.

Net Loss and Net Loss per Share: Net loss for the year ended December 31, 2023 was $132.5 million, compared to $128.2 million for the year ended December 31, 2022. Net loss per share for the year ended December 31, 2023 was $2.67, compared to $2.62 for the year ended December 31, 2022.

Cash Position and Financial Guidance: Cash, cash equivalents and marketable securities as of December 31, 2023 were $281.7 million, compared to $337.1 million as of December 31, 2022. Cash and cash equivalents as of December 31, 2023 do not include $25 million in proceeds from the sale of shares of our common stock to a subsidiary of Betta Pharmaceuticals, $14.1 million of proceeds in connection with the settlement of 2,500,601 shares under our ATM program, and the $10.0 million upfront payment related to our collaboration agreement with Merck, all of which were received in January 2024. The company expects that its cash, cash equivalents and marketable securities as of December 31, 2023, together with these amounts received in January 2024, which result in a proforma balance of approximately $330 million, will enable the company to fund its operating plan into 2027.

On February 22, 2024 BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio or the Company), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, reported its financial results for the fourth quarter and full year ended December 31, 2023, and provided an update on the Company’s operations (Press release, BridgeBio, FEB 22, 2024, View Source [SID1234640383]).

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"Our focus this year is executing on the launch of acoramidis for patients with ATTR cardiomyopathy," said Neil Kumar, Ph.D., founder and CEO of BridgeBio. "At the same time, we are also focused on fully enrolling three ongoing Phase 3 clinical trials by the end of 2024. Finally, we hope that reading out potentially exciting data from our Phase 1/2 trial in congenital adrenal hyperplasia later this year will let us take the next step in serving that patient community."

BridgeBio’s key programs:

Acoramidis (AG10) – Transthyretin (TTR) stabilizer for transthyretin amyloid cardiomyopathy (ATTR-CM):
The Company filed an NDA for acoramidis for the treatment of ATTR-CM with the US FDA; the NDA was accepted for review with a PDUFA date of November 29, 2024. The Company has also filed a Marketing Authorization Application for acoramidis with the EMA, which has been accepted for review.
The regulatory filings were based on data from the Phase 3 ATTRibute-CM study, which met its primary endpoint (Win Ratio of 1.8) with a highly statistically significant p-value (p<0.0001). Additional results from ATTRibute-CM include:
An 81% survival rate on acoramidis, which approaches the survival rate in the age-matched U.S. database (~85%), and a 0.29 mean annual CVH rate on acoramidis, which approaches the annual hospitalization rate observed in the broader U.S. Medicare population (~0.26);
Improvements from baseline observed for a large proportion of participants treated with acoramidis on laboratory and functional measures including n-terminal prohormone of brain natriuiretic peptide (NT-proBNP) and 6-minute walk distance;
Rapid clinical benefit on the composite endpoint of ACM and CVH in participants treated with acoramidis, demonstrated by placebo and acoramidis time-to-first event Kaplan-Meier curves for a composite of ACM and CVH that separated at Month 3 and continued to diverge steadily through Month 30 as presented at the American Heart Association Scientific Sessions in November 2023; and
Acoramidis was well-tolerated with no safety signals of potential clinical concern identified.
The Company also shared positive results of an open-label, single-arm Phase 3 study conducted in Japan by licensing partner Alexion, AstraZeneca Rare Disease, including that no mortality was reported over the 30 month acoramidis treatment period.
Additional detailed results of ATTRibute-CM are planned for presentation at 2024 medical meetings.
Low-dose infigratinib – FGFR1-3 inhibitor for achondroplasia and hypochondroplasia:
In December 2023, the Company announced the dosing of the first child in PROPEL 3, its global Phase 3 registrational study of infigratinib in achondroplasia.
In February 2024, the Company announced a partnership with Kyowa Kirin wherein the Company grants Kyowa Kirin an exclusive license to develop and commercialize infigratinib for achondroplasia, hypochondroplasia, and other skeletal dysplasias in Japan; in exchange, the Company will receive an upfront payment of $100 million as well as royalties up to the high-twenties percent on sales of infigratinib in Japan, with the potential for additional milestone-based payments.
The Company is committed to exploring the potential of infigratinib on the wider medical and functional impacts of achondroplasia, hypochondroplasia and other skeletal dysplasias, and anticipates initiating its clinical program for hypochondroplasia in 2024.
BBP-418 – Glycosylation substrate for limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9):
FORTIFY, the global Phase 3 registrational trial of BBP-418, continues to enroll in the U.S. with clinical trial sites planned for Europe and Australia. Full enrollment of the interim analysis population is expected in 2024. The Company believes there is potential to pursue Accelerated Approval for BBP-418 based on recent interactions with the FDA on the use of glycosylated αDG levels as a surrogate endpoint.
Encaleret – Calcium-sensing receptor (CaSR) inhibitor for autosomal dominant hypocalcemia type 1 (ADH1):
CALIBRATE, the Phase 3 clinical trial of encaleret, continues to enroll; the Company anticipates sharing topline data from CALIBRATE in 2025.
Recent Corporate Updates:

·Secured up to $1.25 billion of capital from Blue Owl and CPP Investments: The raise includes $500 million in cash from Blue Owl and CPP Investments available upon FDA approval of acoramidis in exchange for a 5% royalty on future global net sales of acoramidis, as well as a $450 million credit facility from Blue Owl that refinanced existing senior secured credit, extending maturity from 2026 to 2029 subject to certain conditions.

Fourth Quarter and Full Year 2023 Financial Results:

Cash, Cash Equivalents, Marketable Securities and Short-term Restricted Cash

Cash, cash equivalents and short-term restricted cash, totaled $392.6 million as of December 31, 2023, compared to cash, cash equivalents, marketable securities and short-term restricted cash of $466.2 million as of December 31, 2022. The net decrease of $73.6 million in cash, cash equivalents, marketable securities and short-term restricted cash was primarily attributable to net cash used in operating activities of $527.7 million and $6.9 million in repurchase of shares to satisfy tax withholdings, primarily offset by net proceeds received of $449.8 million from various equity financings, $6.0 million from stock option exercises, and $3.4 million from common stock issuances under our employee stock purchase plan during the year ended December 31, 2023.

Revenue

Revenue for the three months and year ended December 31, 2023 were $1.7 million and $9.3 million, respectively, as compared to $1.9 million and $77.6 million for the same periods in the prior year, respectively. The net decreases of $0.2 million and $68.3 million for the three months and year ended December 31, 2023, respectively, compared to the same periods in the prior year, were primarily due to license revenue recognized in 2022 upon the transfer of the license in accordance with the Navire-BMS License Agreement which was entered into in May 2022.

Operating Costs and Expenses

Operating costs and expenses for the three months and year ended December 31, 2023 were $179.2 million and $616.7 million, respectively, compared to $131.1 million and $589.9 million, for the same periods in the prior year, respectively.

The overall increase of $48.1 million in operating costs and expenses for the three months ended December 31, 2023, compared to the same period in the prior year, was primarily due to an increase of $39.3 million in research and development and other expenses (R&D) to advance the Company’s pipeline of development programs, an increase of $15.7 million in selling, general and administrative (SG&A) expenses to support commercialization readiness efforts, offset by a decrease of $6.9 million in restructuring, impairment and related charges given that the majority of the restructuring initiatives occurred in the prior year.

The overall increase of $26.8 million in operating costs and expenses for the year ended December 31, 2023 , compared to the same period in the prior year, was primarily due to an increase of $55.2 million in R&D expenses to advance the Company’s pipeline of development programs, an increase of $7.4 million in SG&A expenses to support commercialization readiness efforts, offset by a decrease of $35.8 million in restructuring, impairment and related charges given that the majority of the restructuring initiatives occurred in the prior year.

Restructuring, impairment and related charges for the three months and year ended December 31, 2023, amounted to $0.8 million and $7.9 million, respectively. These charges primarily consisted of winding down, exit costs, and severance and employee-related costs. Restructuring, impairment and related charges for the same periods in the prior year were $7.7 million and $43.8 million, respectively. These charges primarily consisted of impairments and write-offs of long-lived assets, severance and employee-related costs, and exit and other related costs.

Stock-based compensation expenses included in operating costs and expenses for the three months ended December 31, 2023 were $37.1 million, of which $22.5 million is included in R&D expenses, and $14.6 million is included in SG&A expenses. Stock-based compensation expenses included in operating costs and expenses for the same period in the prior year were $22.6 million, of which $8.9 million is included in R&D expenses, and $13.6 million is included in SG&A expenses.

Stock-based compensation expenses included in operating costs and expenses for the year ended December 31, 2023 were $115.0 million, of which $61.6 million is included in R&D expenses, and $53.4 million is included in SG&A expenses. Stock-based compensation expenses included in operating costs and expenses for the same period in the prior year were $93.8 million, of which $38.0 million is included in R&D expenses, $54.7 million is included in SG&A expenses, and $1.2 million is included in restructuring, impairment and related charges.

"Coming off of our recent royalty financing, we find ourselves well capitalized to launch acoramidis this year alongside strong new partners who share our confidence in acoramidis’ potential in the ATTR-CM market," said Brian Stephenson, Ph.D., CFA, Chief Financial Officer of BridgeBio. "We are excited for this launch, as well as for the continued advancement of our late stage pipeline, which we hope will allow us to serve patients with genetic diseases both directly with the advancement of those medicines towards the market as well as by diversifying our top line revenue and enabling reinvestment into the R&D and business development opportunities that will allow us to be sustainable in the long term."