On November 3, 2022 Guardant Health, Inc. (Nasdaq: GH), a leading precision oncology company focused on helping conquer cancer globally through use of its proprietary tests, vast data sets and advanced analytics, reported financial results for the quarter ended September 30, 2022 (Press release, Guardant Health, NOV 3, 2022, View Source [SID1234622972]).

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Recent Highlights
•Revenue of $117.4 million for the third quarter of 2022, an increase of 24% over the corresponding period of 2021
•Reported 32,400 tests to clinical customers and 6,750 tests to biopharmaceutical customers in the third quarter of 2022, representing an increase of 42% and 40%, respectively, over the third quarter of 2021
•Expanded Guardant Reveal , the only tissue-free liquid biopsy test for detection of residual and recurrent disease, to include early-stage breast and lung cancers in addition to colorectal cancer
•Introduced GuardantINFINITY, a next-generation smart liquid biopsy assay that provides broad genomic and epigenomic insights into the complexities of tumor molecular profiles and immune response to advance cancer research and therapy development
•Received FDA approval for Guardant360 CDx liquid biopsy test as a companion diagnostic for ENHERTU for treatment of non-small cell lung cancer patients with activating HER2 mutations
•ECLIPSE readout and PMA submission for Shield assay both expected during the fourth quarter of the year
"Volume growth accelerated sequentially in clinical oncology, propelling us to new records in revenue and volumes. We made additional strides with Guardant Reveal, expanding its use into multiple cancer indications that now include colorectal, lung and breast cancers. In addition, Guardant Infinity, our first smart liquid biopsy offering, has seen a rapid uptake with our biopharma partners. That said, counter to our expectations earlier in the year, clinical volumes continue to be impacted by a challenging backdrop with lingering access restrictions and provider staffing shortages." said Helmy Eltoukhy, co-founder and co-CEO.
"During the quarter we made great progress on the ECLIPSE study and are very close to locking our study database with about 70 CRCs. In parallel, our Shield LDT screening test continues to be well received by physicians and patients and has demonstrated outstanding levels of patient adherence," said AmirAli Talasaz, co-founder and co-CEO. "This positive early response to Shield LDT gives us increased confidence in our ability to develop Shield into a leading non-invasive cancer screening methodology."
Third Quarter 2022 Financial Results
Revenue was $117.4 million for the three months ended September 30, 2022, a 24% increase from $94.8 million for the three months ended September 30, 2021. Precision oncology revenue grew 29%, driven predominantly by an increase in clinical testing volume and biopharma sample volume, which grew 42% and 40%, respectively, over the prior year period. Development services and other revenue slightly decreased by 1%.
Gross profit, or total revenue less cost of precision oncology testing and cost of development services and other, was $76.9 million for the third quarter of 2022, an increase of $12.9 million from $64.0 million for the corresponding prior year period. Gross margin, or gross profit divided by total revenue, was 66%, as compared to 67% for the corresponding prior year period.
Operating expenses were $221.5 million for the third quarter of 2022, as compared to $171.3 million for the corresponding prior year period, an increase of 29%. Non-GAAP operating expenses were $200.5 million for the third quarter of 2022, as compared to $135.1 million for the corresponding prior year period.
Net loss attributable to Guardant Health, Inc. common stockholders was $162.0 million for the third quarter of 2022, as compared to $107.5 million for the corresponding prior year period. Net loss per share attributable to Guardant Health, Inc. common stockholders was $1.58 for the third quarter of 2022, as compared to $1.06 for the corresponding prior year period. Non-GAAP net loss was $120.8 million for the third quarter of 2022, as compared to $70.5 million for the corresponding prior year period. Non-GAAP net loss per share was $1.18 for the third quarter of 2022, as compared to $0.70 for the corresponding prior year period.
Adjusted EBITDA loss was $112.8 million for the third quarter of 2022, as compared to a $65.2 million loss for the corresponding prior year period.
Cash, cash equivalents and marketable debt securities were $1.1 billion as of September 30, 2022.
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2022 Guidance
Given the continued challenging backdrop and reimbursement delays impacting the clinical oncology business, Guardant Health now expects full year 2022 revenue to be in the range of $440 million to $450 million, representing growth of 18% to 20% compared to full year 2021. This compares to previous full year 2022 revenue guidance of $460 million to $470 million.
Webcast Information
Guardant Health will host a conference call to discuss the third quarter 2022 financial results after market close on Thursday, November 3, 2022 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 research and development expense, non-GAAP sales and marketing expense, non-GAAP general and administrative expense, non-GAAP loss from operations, non-GAAP net loss, non-GAAP net loss attributable to Guardant Health, Inc., common stockholders, non-GAAP net loss per share attributable to Guardant Health, Inc. common stockholders, basic and diluted, and Adjusted EBITDA.
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, changes in estimated fair value of noncontrolling interest liability, adjustments relating to redeemable noncontrolling interest, contingent consideration, acquisition related expenses, amortization of intangible assets, fair value adjustments on marketable equity securities, impairment of other assets, and other non-recurring items.
Adjusted EBITDA is defined as net loss attributable to Guardant Health, Inc. common stockholders 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, changes in estimated fair value of noncontrolling interest liability, adjustments relating to redeemable noncontrolling interest and contingent consideration, and, if applicable in a reporting period, acquisition-related expenses, and other non-recurring items.
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 other items because we believe that these income (expenses) do not reflect expected future operating expenses. 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 November 3, 2022 Puma Biotechnology, Inc. (NASDAQ: PBYI), a biopharmaceutical company, reported that financial results for the third quarter ended September 30, 2022 (Press release, Puma Biotechnology, NOV 3, 2022, View Source [SID1234622971]). Unless otherwise stated, all comparisons are for the third quarter of 2022 compared to the third quarter of 2021.

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Product revenue, net consists entirely of sales revenue from NERLYNX, Puma’s first commercial product. Product revenue, net in the third quarter of 2022 was $54.3 million, compared to $43.4 million in the third quarter of 2021. Product revenue, net in the first nine months of 2022 was $146.3 million, compared to $138.1 million in the first nine months of 2021.

Based on accounting principles generally accepted in the United States (GAAP), Puma reported a net loss of $0.4 million, or $0.01 per share, for the third quarter of 2022, compared to a net loss of $44.7 million, or $1.09 per share, for the third quarter of 2021. Net income for the first nine months of 2022 was $5.6 million, or $0.13 per basic and diluted share, compared to a net loss of $33.4 million, or $0.82 per share, for the first nine months of 2021.

Non-GAAP adjusted net income was $2.5 million, or $0.05 per basic and diluted share, for the third quarter of 2022, compared to non-GAAP adjusted net loss of $40.4 million, or $0.99 per share, for the third quarter of 2021. Non-GAAP adjusted net income for the first nine months of 2022 was $14.8 million, or $0.33 per basic and diluted share, compared to non-GAAP adjusted net loss of $5.0 million, or $0.12 per share, for the first nine months of 2021. Non-GAAP adjusted net income (loss) excludes stock-based compensation expenses. For a reconciliation of GAAP net income (loss) to non-GAAP adjusted net income (loss) and GAAP net income (loss) per share to non-GAAP adjusted net income (loss) per share, please see the financial tables at the end of this news release.

Net cash provided by operating activities for the third quarter of 2022 was $17.3 million, compared to net cash provided by operating activities of $10.5 million in the third quarter of 2021. Net cash used in operating activities for the first nine months of 2022 was $23.5 million, compared to net cash provided by operating activities of $26.1 million in the first nine months of 2021. On September 30, 2022, Puma had cash, cash equivalents, and marketable securities of $78.0 million, compared to cash, cash equivalents, and marketable securities of $82.1 million at December 31, 2021.

"We are very pleased to report $54.3 million in NERLYNX revenues in the third quarter of 2022," said Alan H. Auerbach, Chairman, Chief Executive Officer, and President of Puma. "This is being driven by the U.S. commercial revenues from NERLYNX and our commercial execution, which is designed to support increased patient access to NERLYNX. This past quarter we also presented updated results from the Phase II SUMMIT basket trial of neratinib for HER2-mutant, recurrent/metastatic cervical cancer at the 2022 European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress and we recently reported Phase II data from the cohort of patients in the SUMMIT basket trial of neratinib in non-small cell lung cancer patients with EGFR exon 18 mutations at the EORTC/NCI/AACR Molecular Targets and Cancer Therapeutics Symposium. Puma remains committed to developing treatments for cancer patients, and in September we were pleased to have in-licensed alisertib, an aurora kinase A inhibitor, which we plan to develop initially for the treatment of hormone receptor-positive breast cancer as well as small cell lung cancer. We will continue evaluating both neratinib and alisertib across a number of cancer indications."

Mr. Auerbach added, "We anticipate the following key milestones over the next 12 months: (i) reporting Phase II TBCRC-022 trial data from Cohort 4B and 4C of the combination of Kadcyla plus neratinib in patients with HER2-positive breast cancer with brain metastases who have previously been treated with Kadcyla (H2 2022); (ii) publication of the biomarker studies from the randomized trial of alisertib plus fulvestrant versus alisertib alone in hormone receptor positive, HER2 negative breast cancer (Q4 2022); (iii) biomarker data from the randomized trial of alisertib plus paclitaxel versus paclitaxel alone in hormone receptor positive, HER2 negative breast cancer (H1 2023); (iv) reporting data from an ongoing investigator sponsored Phase I/II trial of alisertib plus pembrolizumab for the treatment of patients with Rb-deficient head and neck squamous cell cancer (2023); (v) conducting a meeting with the FDA to discuss the registration pathway of neratinib in HER2-mutated HR-positive breast cancer (H1 2023); (vi) conducting a meeting with the FDA to discuss the registration pathway for neratinib in non-small cell lung cancer patients with EGFR exon 18 mutations who have previously been treated with an EGFR tyrosine kinase inhibitor (H1 2023); and (vii) conducting a meeting with the FDA to discuss the registration pathway for alisertib in hormone receptor positive, HER2 negative breast cancer and small cell lung cancer (H1 2023)."

Revenue

Total revenue consists of product revenue, net from sales of NERLYNX, Puma’s first commercial product, license revenue from Puma’s sub-licensees and royalty revenue. For the third quarter of 2022, total revenue was $57.1 million, of which $54.3 million was product revenue, net and $2.8 million was royalty revenue. This compares to total revenue of $46.2 million in the third quarter of 2021, of which $43.4 million was product revenue, net and $2.8 million was royalty revenue. For the first nine months of 2022, total revenue was $162.4 million, of which $146.3 million was product revenue, net and $16.1 million was royalty revenue. This compares to total revenue of $197.8 million for the first nine months of 2021, of which $138.1 million was product revenue, net, $50.3 million was license revenue received from Puma’s sub-licensees, which included a $50 million upfront payment for providing development, manufacturing, and commercial rights to NERLYNX in Greater China to Pierre Fabre, and $9.4 million was royalty revenue.

Operating Costs and Expenses

Total operating costs and expenses were $54.7 million for the third quarter of 2022, compared to $55.2 million for the third quarter of 2021. Operating costs and expenses in the first nine months of 2022 were $148.7 million, compared to $203.3 million in the first nine months of 2021.

Cost of Sales

Cost of sales was $12.5 million for the third quarter of 2022, compared to $10.3 million for the third quarter of 2021. Cost of sales was $38.3 million for the first nine months of 2022, compared to $51.8 million for the first nine months of 2021, of which $20.0 million was a termination fee paid to a former sub-licensee for the return of commercial rights to NERLYNX in Greater China.

Selling, General and Administrative Expenses

Selling, general and administrative (SG&A) expenses were $24.0 million for the third quarter of 2022, compared to $26.1 million for the third quarter of 2021. SG&A expenses for the first nine months of 2022 were $64.9 million, compared to $93.8 million for the first nine months of 2021. The $28.9 million decrease in SG&A expenses for the first nine months of 2022 compared to the first nine months of 2021 resulted primarily from a decrease in payroll and related costs of approximately $8.9 million, consisting of approximately $7.0 million from lower headcount and a $2.0 million payroll tax credit under the CARES Act; and a decrease in stock-based compensation expense of approximately $17.0 million, primarily due to the $13.6 million incremental expense resulting from the modification to the term of Mr. Auerbach’s warrant in 2021, as well as approximately $3.4 million due to the impact of lower headcount. Additionally, professional fees and expenses decreased by approximately $2.8 million, due primarily to a decrease of approximately $6.3 million in consultancy efforts related to marketing and commercialization support, partially offset by an increase of approximately $3.8 million in legal fees.

Research and Development Expenses

Research and development (R&D) expenses were $11.2 million for the third quarter of 2022, compared to $18.8 million for the third quarter of 2021. R&D expenses for the first nine months of 2022 were $38.5 million, compared to $57.7 million for the first nine months of 2021. The $19.2 million decrease in R&D expenses for the first nine months of 2022 compared to the first nine months of 2021 resulted primarily from a decrease in clinical trial expense of approximately $8.5 million, primarily due to the reduction in the number of patients in certain clinical trials; a decrease in internal R&D expenses of approximately $6.3 million; a decrease in consultant and contractor expense of approximately $2.4 million, primarily due to the close of the CONTROL study and a reduction in the number of patients being treated in the SUMMIT study; and a decrease in stock-based compensation expense of approximately $2.1 million, primarily due to the impact of lower headcount.

Acquired In-Process Research and Development Expense

In September 2022, the Company entered into an exclusive license agreement with Takeda Pharmaceutical Company Limited to in-license the worldwide research and development and commercial rights to alisertib. The Company recorded acquired in-process research and development expense related to the up-front payment of $7.0 during the three months ended September 30, 2022.

Total Other Income (Expenses)

Total other expenses were $2.7 million for the third quarter of 2022, compared to $35.7 million for the third quarter of 2021. Total other expenses were $7.9 million for the first nine months of 2022, compared to $27.7 million for the first nine months of 2021. The $19.8 million decrease for the first nine months of 2022 compared to the first nine months of 2021 resulted primarily from net reductions in legal verdict expense of $9.8 million, $8.1 million in loss on debt extinguishment related to our debt refinancing in July 2021, and a decrease of $1.8 million in lower interest expense related to our outstanding debt.

Full Year 2022 Financial Outlook

Puma increased its financial guidance for full year 2022 to reflect improvements in U.S. demand for NERLYNX and changes to the timing of shipments to Puma’s international partners.

Conference Call

Puma Biotechnology will host a conference call to report its third quarter 2022 financial results and provide an update on the Company’s business and outlook at 1:30 p.m. PDT/4:30 p.m. EDT on Thursday, November 3, 2022. The call may be accessed by dialing (877) 709-8150 (domestic) or (201) 689-8354 (international). Please dial in at least 10 minutes in advance and inform the operator that you would like to join the "Puma Biotechnology Conference Call." A live webcast of the conference call and presentation slides may be accessed on the Investors section of the Puma Biotechnology website at View Source A replay of the call will be available shortly after completion of the call and will be archived on Puma’s website for 90 days.

On November 3, 2022 Corcept Therapeutics Incorporated (NASDAQ: CORT), a commercial-stage company engaged in the discovery and development of medications to treat severe endocrine, oncologic, metabolic and neurological disorders by modulating the effects of the hormone cortisol, reported its results for the quarter ended September 30, 2022 (Press release, Corcept Therapeutics, NOV 3, 2022, View Source [SID1234622970]).

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Financial Results

Revenue of $101.7 million, compared to $96.1 million in third quarter 2021
Tightened 2022 revenue guidance of $400 – $410 million
Diluted net income per common share of $0.30, compared to $0.24 in third quarter 2021
Cash and investments of $401.2 million, compared to $382.0 million at June 30, 2022
Corcept’s third quarter 2022 revenue was $101.7 million, compared to $96.1 million in the third quarter of 2021. Third quarter operating expenses were $69.8 million, compared to $59.9 million in the third quarter of 2021, due to increased clinical trial and sales and marketing activities and expenses to support the expansion of our clinical development and commercial teams. Net income was $34.6 million in the third quarter of 2022, compared to $30.5 million in the third quarter of 2021.

Cash and investments of $401.2 million at September 30, 2022 reflects the acquisition in the third quarter of $15.5 million of Corcept common stock in connection with the exercise of employee stock options and vesting of restricted stock grants.

"Diagnosing and treating patients with a complex disease such as Cushing’s syndrome requires frequent in-person contact with physicians. Our revenue in the third quarter was affected by fewer than expected in-person interactions as many physician practices have not returned to pre-pandemic patterns of activity. To reflect this near-term challenge, we are tightening our 2022 revenue guidance to $400 to $410 million," said Joseph K. Belanoff, MD, Corcept’s Chief Executive Officer. "We remain extremely optimistic about the present and the future of our Cushing’s syndrome business. Korlym is an excellent treatment for patients with Cushing’s syndrome and there are many eligible patients who have yet to receive it. We are making substantial investments to improve the screening and treatment of these patients and we are confident that these initiatives will contribute to our results in the coming quarters."

Clinical Development

"Our clinical trials continue to advance and generate data supporting cortisol modulation’s broad therapeutic potential," added Dr. Belanoff. "We are very excited about our most recently initiated studies: ROSELLA, our confirmatory Phase 3 trial in platinum-resistant ovarian cancer; and DAZALS, our Phase 2 trial in ALS. We are also looking forward to important readouts from our two Phase 2 trials in antipsychotic-induced weight gain by the end of this year."

Oncology

Enrollment continues in ROSELLA – 360-patient pivotal Phase 3 trial of relacorilant plus nab-paclitaxel in patients with recurrent, platinum-resistant ovarian cancer
Enrollment continues in open-label, Phase 1b trial of relacorilant plus
pembrolizumab in patients with adrenal cancer with cortisol excess
Randomized, placebo-controlled Phase 2 trial of relacorilant plus enzalutamide in patients with prostate cancer to begin next year in collaboration with the University of Chicago
"We and our investigators are excited that ROSELLA is active and enrolling patients," said Bill Guyer, PharmD, Corcept’s Chief Development Officer. "Our goal in this trial is to replicate the positive results of our successful Phase 2 study, which demonstrated improvements in progression-free survival, duration of response and overall survival, all without increasing side effect burden. Women with platinum-resistant ovarian cancer do not have good treatment options and relacorilant plus nab-paclitaxel has the potential to become a new standard of care."

Amyotrophic Lateral Sclerosis (ALS)

Enrollment continues in DAZALS – 198-patient, randomized, double-blind, placebo-controlled Phase 2 trial of dazucorilant in patients with ALS
"ALS, also known as Lou Gehrig’s disease, is a devastating neuromuscular illness with an urgent need for better treatment," said Dr. Guyer. "Dazucorilant showed great promise in animal models of ALS – improving motor performance and reducing neuroinflammation and muscular atrophy. We are excited to initiate this important study in collaboration with TRICALS, the leading ALS academic consortium in Europe, to investigate dazucorilant’s potential to significantly improve the lives of patients with ALS."

Metabolic Diseases

Enrollment completed in GRATITUDE and GRATITUDE II – two double-blind, placebo-controlled Phase 2 trials of miricorilant to reverse recent and long-standing antipsychotic-induced weight gain (AIWG) – data from both trials expected this quarter
Enrollment continues in Phase 1b dose-finding trial of miricorilant in patients
with presumed non-alcoholic steatohepatitis (NASH) – data expected in the first half of 2023
"We expect our double-blind, placebo-controlled GRATITUDE and GRATITUDE II trials in patients with AIWG to build on the positive data from our Phase 1 studies," said Dr. Guyer. "There is a substantial opportunity to improve the medical care of these patients as weight gain and other adverse metabolic effects caused by antipsychotic medications dissuade many patients from adhering to their treatment regimen, pose serious health risks and reduce the life expectancy of millions of patients."

"The goal of our Phase 1b dose-finding study in patients with NASH is to identify a dosing regimen that captures the unprecedented rapidity and magnitude of liver fat reduction observed in our prior study without causing excessive liver irritation," added Dr. Guyer.

Cushing’s Syndrome

Enrollment continues in Phase 3 GRACE trial of relacorilant as a treatment for patients with all etiologies of Cushing’s syndrome – new drug application (NDA) submission expected
in second half 2023
Enrollment continues in Phase 3 GRADIENT trial of relacorilant as a treatment for patients
with Cushing’s syndrome caused by adrenal adenomas
"We advanced relacorilant to Phase 3 in Cushing’s syndrome based on its extremely promising Phase 2 efficacy and safety data. We expect our GRACE trial to serve as the basis for relacorilant’s NDA in Cushing’s syndrome, which we plan to submit in the second half of 2023," said Dr. Guyer. "The Phase 3 GRADIENT trial will produce valuable data about an etiology of Cushing’s syndrome that has not been subject to rigorous, controlled study, but affects many patients."

Conference Call

We will hold a conference call on November 3, 2022, at 5:00 p.m. Eastern Time (2:00 p.m. Pacific Time). Participants must register in advance of the conference call by clicking here. Upon registering, each participant will receive a dial-in number, and a unique access PIN. Each access PIN will accommodate one caller. Additionally, a listen-only webcast will be available by clicking here. A replay of the call will be available on the Investors / Events tab of www.corcept.com.

Hypercortisolism

Hypercortisolism, often referred to as Cushing’s syndrome, is caused by excessive activity of the hormone cortisol. Endogenous Cushing’s syndrome is an orphan disease that most often affects adults aged 20-50. In the United States, an estimated 20,000 patients have Cushing’s syndrome, with about 3,000 new patients diagnosed each year. Symptoms vary, but most patients experience one or more of the following manifestations: high blood sugar, diabetes, high blood pressure, upper-body obesity, rounded face, increased fat around the neck, thinning arms and legs, severe fatigue and weak muscles. Irritability, anxiety, cognitive disturbances and depression are also common. Hypercortisolism can affect every organ system and can be lethal if not treated effectively. Corcept holds patents directed to the composition of relacorilant and the use of cortisol modulators, including Korlym, in the treatment of patients with hypercortisolism.

On November 3, 2022 Cytokinetics, Incorporated (Nasdaq: CYTK) reported financial results for the third quarter of 2022 (Press release, Cytokinetics, NOV 3, 2022, View Source [SID1234622969]). Net loss for the third quarter was $142.3 million, or $1.52 per share, compared to net loss for the third quarter of 2021 of $76.1 million, or $0.95 per share. Cash, cash equivalents and investments totaled $896.2 million at September 30, 2022.

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"During the third quarter, we continued to execute towards our potential transformation to commercialization with expansion of teams and readiness activities in preparation for our upcoming FDA Advisory Committee meeting and the potential launch of omecamtiv mecarbil," said Robert I. Blum, Cytokinetics’ President and Chief Executive Officer. "At the same time, the development program for aficamten is proceeding with continued conduct of Cohort 4 of REDWOOD-HCM and SEQUOIA-HCM, alongside start up activities for our second Phase 3 clinical trial of aficamten. We also are continuing COURAGE-ALS following its first interim analysis marking progress aligned with our commitment to ALS. We have entered the fourth quarter with a strong balance sheet ahead of key corporate milestones."

Q3 and Recent Highlights

Cardiac Muscle Programs

omecamtiv mecarbil (cardiac myosin activator)

Engaged in further interactions with the U.S. Food and Drug Administration (FDA) related to our New Drug Application (NDA) for omecamtiv mecarbil. Continued discussions with FDA regarding matters related to substantial evidence of efficacy, benefit-risk, and dosing.

Conducted meetings with assigned rapporteurs to discuss the planned submission of a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA).

Continued commercial preparations for the potential U.S. launch of omecamtiv mecarbil including convening meetings with national and regional payers, advancing wholesaler agreements for distribution, finalizing third party logistics agreement, completing strategic sourcing of drug substance and drug product contract manufacturing organizations, continuing development of patient services hub, and beginning the final wave of hiring for sales force leaders.

Expanded the headquarters- and field-based medical affairs team, continued support of independent medical education activities at medical conferences, and initiated planning for our Medical Contact Center.

Launched "The Heart of Contractility," a disease state education campaign for healthcare providers to build awareness of worsening heart failure and the importance of contractility.
aficamten (cardiac myosin inhibitor)

Continued clinical trial site activation and enrollment of patients with obstructive HCM in SEQUOIA-HCM (Safety, Efficacy, and Quantitative Understanding of Obstruction Impact of Aficamten in HCM), our first Phase 3 trial of aficamten, with 70 sites now enrolling in the U.S. and Europe.

Completed screening for patients with non-obstructive HCM to enroll in Cohort 4 of REDWOOD-HCM (Randomized Evaluation of Dosing With CK-274 in Obstructive Outflow Disease in HCM).

Renamed the open-label extension clinical study of aficamten in patients with hypertrophic cardiomyopathy (HCM), previously known as REDWOOD-HCM OLE (Randomized Evaluation of Dosing With CK-274 in Obstructive Outflow Disease in HCM Open Label Extension) to FOREST-HCM (Five-Year, Open-Label, Research Evaluation of Sustained Treatment with Aficamten in HCM) to reflect the entry of patients from additional clinical trials of aficamten including SEQUOIA-HCM.

Presented data from FOREST-HCM (previously known as REDWOOD-HCM OLE) at the Heart Failure Society of America (HFSA) Annual Scientific Meeting showing that treatment with aficamten was associated with substantial and significant symptom improvements as measured by the change in Kansas City Cardiomyopathy Questionnaire (KCCQ) scores.

Presented data from FOREST-HCM at the 2022 HCM Society Scientific Sessions demonstrating in patients treated with aficamten the successful reduction or withdrawal of standard of care therapies.

Published a manuscript entitled "A Phase 1 Dose-Escalation Study of the Cardiac Myosin Inhibitor Aficamten in Healthy Participants" in JACC: Basic to Translational Science.
Skeletal Muscle Program

reldesemtiv (fast skeletal muscle troponin activator (FSTA))

Announced that the Data Monitoring Committee (DMC) for COURAGE-ALS (Clinical Outcomes Using Reldesemtiv on ALSFRS-R in a Global Evaluation in ALS) convened to review unblinded data from the clinical trial and recommended that conduct of the Phase 3 trial continue.
Pre-Clinical Development and Ongoing Research

Continued to advance new muscle directed compounds and conduct IND-enabling studies with the expectation of our potentially moving 1-2 drug candidates into clinical development in the next year.

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

Raised $523.6 million in net proceeds from a 3.50% convertible senior notes offering (due 2027), after deducting underwriters’ discounts and transaction fees, and before repurchasing approximately $117 million of previously outstanding 4.00% convertible senior notes (due 2026).

Announced a new release of the Pooled Resource Open-Access ALS Clinical Trials (PRO-ACT) database updated with clinical data from Cytokinetics’ completed clinical trials in ALS.

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

Cardiac Muscle Programs

omecamtiv mecarbil (cardiac myosin activator)

Participate in Advisory Committee meeting to review the NDA for omecamtiv mecarbil on December 13, 2022.

Launch omecamtiv mecarbil in the U.S. subject to FDA approval in Q1 2023.

Submit Marketing Authorization Application (MAA) to the European Medicines Agency by the end of 2022.
aficamten (cardiac myosin inhibitor)

Continue enrolling patients with obstructive HCM in SEQUOIA-HCM through 1H 2023 with results expected in 2H 2023.

Complete enrolling patients with non-obstructive HCM in Cohort 4 of REDWOOD-HCM with data expected in 1H 2023.

Begin second Phase 3 clinical trial of aficamten in obstructive HCM in Q4 2022.
CK-3828136 (CK-136) (cardiac troponin activator)

Begin Phase 1 study of CK-136 in Q4 2022.
Skeletal Muscle Program

reldesemtiv (fast skeletal muscle troponin activator (FSTA))

Expect the Data Monitoring committee to conduct the second interim analysis from COURAGE-ALS in 1H 2023, which will assess for futility and allow for a fixed increase in total enrollment, if deemed necessary, to augment the statistical power of the trial.

Continue enrolling patients with ALS in COURAGE-ALS and expect to complete enrollment in 1H 2023.
Financials

Revenues for the three and nine months ended September 30, 2022 were $2.5 million and $92.6 million, respectively, compared to $5.4 million and $14.8 million for the corresponding period in 2021. The increase in revenues for the nine months ended September 30, 2022 was primarily due to the recognition of $87.0 million of deferred revenue for royalties on the net sales of products containing mavacamten as a result of the extinguishment of royalty obligations.

Research and development expenses for the three and nine months ended September 30, 2022 increased to $62.7 million and $165.8 million, respectively, compared to $48.4 million and $116.4 million for the same period in 2021. The changes were primarily due to increases in spending for clinical development activities for COURAGE-ALS and SEQUOIA-HCM, and for our other cardiac muscle inhibitor and early research programs.

General and administrative expenses for the three and nine months ended September 30, 2022 increased to $48.2 million and $124.0 million, respectively, from $26.2 million and $63.0 million for the same period in 2021 due primarily to higher outside services spending in anticipation of the potential commercial launch of omecamtiv mecarbil, and an increase in personnel related costs including stock-based compensation.

During the quarter, we recognized a loss of $22.2 million related to the conversion and partial settlement of our 2026 Convertible Notes.

Conference Call and Webcast Information

The conference call will be simultaneously webcast and can be accessed from the homepage and in the Investors & Media section of Cytokinetics’ website at www.cytokinetics.com. The live audio of the conference call can also be accessed by telephone by registering in advance at the following link: Cytokinetics Q3 2022 Earnings Conference Call. Upon registration, participants will receive a dial-in number and a unique passcode to access the call. An archived replay of the webcast will be available via Cytokinetics’ website for twelve months.

On November 3, 2022 Cellectis (the "Company") (Euronext Growth: ALCLS – NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, reported the release of two abstracts, which were accepted for presentation at the 64th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting taking place from December 10 to 13, 2022 (Press release, Cellectis, NOV 3, 2022, View Source [SID1234622968]).

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The Company will present, in an oral session on December 12, preliminary clinical data from its AMELI-01 clinical trial (evaluating UCART123) in patients with relapsed/refractory acute myeloid leukemia (r/r AML). Amsterdam University Medical Center (location VUmc), in collaboration with Cellectis, will also present, in a poster session on December 10, preclinical data supporting anti-tumor activity for Cellectis’ UCARTCS1 product candidate, which is being evaluated in clinical trial, MELANI-01, for patients with relapsed/refractory multiple myeloma (r/r MM).

"Cellectis is excited to share preliminary clinical data from our AMELI-01 clinical trial, evaluating UCART123 in patients with relapsed and/or refractory acute myeloid leukemia. This trial addresses a patient population with severe unmet medical needs and no additional therapeutic options. We hope our off-the-shelf approach through gene editing will serve as the next step in improving outcomes in patients with this disease," said Mark Frattini, M.D., Ph.D., Chief Medical Officer at Cellectis.

Cellectis’ oral presentation on AMELI-01:

AMELI-01 investigating UCART123 product candidate in r/r AML

The abstract includes preliminary clinical data from the Phase 1, open-label, dose-escalation trial, AMELI-01, in patients with r/r AML having received UCART123 following lymphodepletion (LD) with either fludarabine and cyclophosphamide (FC) or FC with alemtuzumab (FCA). The data show that adding alemtuzumab to the FC regimen was associated with improved LD and significantly higher UCART123 cell expansion, which correlated with improved activity.

UCART123 is a novel and genetically modified allogeneic T-cell product manufactured from healthy donor cells. Donor-derived T-cells are transduced using a lentiviral vector to express the anti-CD123 chimeric antigen receptor (CAR) and are further modified using Cellectis’ TALEN technology to disrupt the T-cell receptor alpha constant (TRAC) and CD52 genes to minimize risk of graft-vs-host disease (GvHD) and allow use of anti-CD52–directed therapy as a component of the LD regimen, respectively.

These data are encouraging and support the continued enrollment into the study.

Presentation Details:
Title: AMELI-01: A Phase I Trial of UCART123v1.2, an Anti-CD123 Allogeneic CAR-T Cell Product, in Adult Patients with Relapsed or Refractory (R/R) CD123+ Acute Myeloid Leukemia (AML)
Publication Number: 981
Presenter: David A. Sallman, MD, Moffitt Cancer Center, Department of Malignant Hematology, Tampa, FL
Session Name: 704. Cellular Immunotherapies: Early Phase and Investigational Therapies: Acute Leukemia and Hodgkin Lymphoma
Date, Time, Location: Monday, December 12, 2022; 5:00PM; Ernest N. Morial Convention Center, Hall E

Link to abstract, here.

Poster Presentation on UCARTCS1, in collaboration with Amsterdam UMC

The abstract includes preclinical data evaluating in vitro activity of UCARTCS1 against MM cell lines and bone marrow samples from MM patients, as well as in vivo activity in a MM mouse model. The potential impact of previous therapy and tumor characteristics on the in vitro efficacy of UCARTCS1 was also investigated.

The preclinical data that will be presented demonstrates anti-tumor activity in vitro and in vivo, supporting the potential benefit of UCARTCS1 first in-human study of, MELANI-01 a Phase 1, open-label, dose-escalation trial, for patients with r/r MM.

UCARTCS1 is a genetically modified allogeneic T-cell product manufactured from healthy donor cells. Donor-derived T-cells are transduced using a lentiviral vector to express the anti-CS1 CAR and are further modified using Cellectis’ TALEN gene editing technology to disrupt the T-cell receptor alpha constant (TRAC) and CS1 genes to minimize risk of graft-vs-host disease (GvHD) and avoid fratricide during production, respectively.

Presentation Details/Poster Abstract Session:

Title: Preclinical Activity of Allogeneic CS1-Specific CAR T-Cells (UCARTCS1) in Multiple Myeloma
Publication Number: 1833
Presenter: C.L.B.M. Korst, Amsterdam UMC location Vrije Universiteit Amsterdam, Department of Hematology
Session Name: 651. Multiple Myeloma and Plasma Cell Dyscrasias: Basic and Translational: Poster I
Date, Time, Location: Saturday, December 10, 2022; 5:30 PM – 7:30 PM; Ernest N. Morial Convention Center, Hall D