On November 4, 2021 Oncternal Therapeutics, Inc. (Nasdaq: ONCT), a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies, reported financial results for the third quarter of 2021. Oncternal management will host a webcast today at 5:00 p.m. ET to provide a business update and discuss its third quarter of 2021 financial results (Press release, Oncternal Therapeutics, NOV 4, 2021, View Source [SID1234594421]).

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"We are excited about the progress of our pipeline during this quarter, especially as we continue to advance towards a registration study for our lead asset, zilovertamab, which we believe can play a key role in the treatment paradigm of patients with mantle cell lymphoma," said James Breitmeyer, M.D., Ph.D., Oncternal’s President and CEO. "We are also very pleased to introduce ONCT-534, a pre-clinical first-in-class dual-action androgen inhibitor, as part of our pipeline. Tumor-resistance mechanisms related to the expression of splice variants such as AR-V7 represents an important unmet need for prostate cancer patients, and we are committed to developing new treatment options for them. Finally, our cell therapy program targeting ROR1 continues to move forward, with progress on both our lead candidate ONCT-808, an autologous CAR-T, and potential next-generation therapies based on NK cells."

Recent Highlights

Zilovertamab (ROR1 antibody, formerly cirmtuzumab or UC-961):

Interactions with FDA continue on potential registration pathways.
An abstract titled Phase 1b/2 Study of Cirmtuzumab and Ibrutinib in Mantle Cell Lymphoma (MCL) or Chronic Lymphocytic Leukemia (CLL) (Abstract 3534) (NCT0308887) has been accepted for a poster presentation at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in Atlanta on December 13, 2021. Abstracts are available on the ASH (Free ASH Whitepaper) website at www.hematology.org.
This abstract reflects early data from June 2021, shortly after our interim data was presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2021 Annual Meeting. The results remain encouraging and consistent with previous data releases and will be further updated with additional patients and follow-up at the ASH (Free ASH Whitepaper) meeting.
The investigator-sponsored study of zilovertamab and paclitaxel for metastatic or locally advanced, unresectable breast cancer at UC San Diego (NCT02776917) has completed enrollment and the results are expected to be presented at a scientific conference or in a publication.
The investigator-sponsored study of zilovertamab for treatment of patients with detectable CLL on venetoclax at UC San Diego (NCT04501939) is actively enrolling patients.
ONCT-216 (ETS inhibitor, formerly TK216):

Interim clinical data update for ONCT-216 in patients with relapsed or refractory Ewing sarcoma was accepted for oral presentation at the 2021 Connective Tissue Oncology Society (CTOS) Virtual Annual Meeting on November 13, 2021.
The Phase 2 expansion cohort targeting up to 21 Ewing sarcoma patients is active and enrolling patients, designed to evaluate clinical responses to single agent ONCT-216 using an optimized dosing regimen, treating for 28 days per cycle, to intensify the amount of ONCT-216 administered over time.
ROR1 Cell-Therapy Program:

Selected ONCT-808 as our lead autologous CAR-T targeting ROR1 candidate and made progress on key IND-enabling activities.
Initiated a collaboration with Celularity Inc. to evaluate placental derived T and NK-cell therapies targeting ROR1.
Formed a cell therapy scientific advisory board (SAB) with cross-disciplinary industry and academic experts to support the advancement of our ROR1 cell therapy pipeline.
Joined Karolinska Institutet’s NextGenNK Competence Center for the development of next generation NK cell-based cancer immunotherapies.
Dual-Action Androgen Inhibitor (DAARI) Program:

Selected ONCT-534 (formerly GTx-534) as our lead DAARI preclinical product candidate, based on significant new preclinical data and assessments. We believe ONCT-534 will address unmet medical needs related to tumor resistance to currently approved products for patients with advanced prostate cancer.
Presented preclinical in-vitro and in-vivo data as a virtual poster presentation at the AACR (Free AACR Whitepaper)-NCI-EORTC Virtual AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper) in October. The data presented showed that ONCT-534 binds to the AR-AF-1, or N-terminal domain of the androgen receptor (AR) and induces degradation of both AR full length proteins and AR-SV proteins, including the AR-V7 splice variant. ONCT-534 demonstrated potent anti-tumor activity in AR-V7 expressing tumor xenografts in an animal model.
Corporate:

Appointed Steven Hamburger, Ph.D. as Senior Vice President, Regulatory Affairs and Quality Assurance.
Expected Upcoming Milestones

Zilovertamab (ROR1 antibody) program
Clinical data update for patients with MCL and CLL treated with zilovertamab plus ibrutinib in the ongoing Phase 1b/2 study to be presented at the American Society for Hematology (ASH) (Free ASH Whitepaper) 2021 Annual Meeting in Atlanta in December 2021
Ongoing FDA interactions regarding potential registration pathways for zilovertamab in patients with MCL
ONCT-216 (ETS inhibitor) programs
Clinical data update for patients with relapsed or refractory Ewing sarcoma to be shared in an oral presentation at the Connective Tissue Oncology Society (CTOS) 2021 Virtual Annual Meeting in November 2021
ROR1 CAR-T program
IND submission in the first half of 2022
The Company will host an R&D Day on January 25, 2022 to provide a comprehensive update of its pipeline of product candidates and key development priorities, which will include key opinion leaders in the fields of MCL therapies, cellular immunotherapies, and androgen receptor resistance in prostate cancer
Third Quarter 2021 Financial Results

Our grant revenue was $2.1 million for the third quarter ended September 30, 2021. Our grant revenue is derived from a sub-award under a grant from the California Institute for Regenerative Medicine (CIRM) to UC San Diego, which was awarded to advance our Phase 1b/2 clinical trial evaluating zilovertamab in combination with ibrutinib for the treatment of patients with MCL or CLL, and from two grant awards received from the National Institutes of Health, or NIH, during the third quarter ended September 30, 2021 for up to $2.2 million to support pre-clinical and other research activities for our ONCT-216 and ONCT-534 programs, including $0.7 million payable to subawardees.

Our total operating expenses for the third quarter ended September 30, 2021 were $11.8 million, including $1.5 million in non-cash stock-based compensation. Research and development expenses for the quarter totaled $9.0 million, and general and administrative expenses for the quarter totaled $2.8 million. Net loss for the third quarter was $9.6 million, or a loss of $0.19 per share, basic and diluted.

As of September 30, 2021, we had $97.4 million in cash and cash equivalents. We believe these funds will be sufficient to fund our operations into 2023. As of September 30, 2021, we had approximately 49.4 million shares of common stock outstanding.

Management Webcast

As previously announced, Oncternal will host a webcast today, November 4, 2021, at 5:00 p.m. ET (2:00 p.m. PT). The live webcast will be available online at investor.oncternal.com. A replay of the webcast will be available beginning approximately one hour after the conclusion of the call and will remain available for at least 30 days thereafter.

On November 4, 2021 Alector, Inc. (Nasdaq: ALEC), a clinical-stage biotechnology company pioneering immuno-neurology, reported third quarter 2021 financial results and summarized recent portfolio and business updates. As of September 30, 2021, Alector’s cash, cash equivalents and investments totaled $777.9 million (Press release, Alector, NOV 4, 2021, View Source [SID1234594420]).

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"During the third quarter, Alector reached a significant inflection point, validating our immuno-neurology approach and advancing our lead programs as we strive to help patients with neurodegenerative diseases," said Arnon Rosenthal, Ph.D., co-founder and Chief Executive Officer of Alector. "We reported 12-month Phase 2 data for our lead therapeutic candidate, AL001, in which progranulin levels in frontotemporal dementia patients reverted to normal, and for the first time, showed encouraging early evidence of a slowing of disease progression across diverse datapoints. The signing of a collaborative agreement with GSK will further enable us to develop our progranulin franchise programs to their full potential in frontotemporal dementia, amyotrophic lateral sclerosis, Parkinson’s disease and Alzheimer’s disease. These achievements, alongside steady progress across our portfolio, are moving us ever closer to realizing our mission of harnessing the innate immune system to halt the destruction of neurodegenerative diseases."

Clinical Updates

Progranulin Franchise

In September, Alector initiated a Phase 2 clinical trial of AL001 in amyotrophic lateral sclerosis (ALS) patients with a C9orf72 genetic mutation. The six-month, randomized, double-blind, placebo-controlled, multicenter study is expected to enroll approximately 45 adult participants with C9orf72-associated ALS. The primary endpoint of the study is safety, tolerability, pharmacokinetics and pharmacodynamics of AL001, including plasma and cerebrospinal fluid (CSF) progranulin levels. The Phase 2 will also gather data on changes to multiple liquid biomarkers. ALS is the first of several indications beyond frontotemporal dementia where elevating progranulin levels is thought to have potential neuroprotective benefits.

AL001 received orphan drug designation from the European Commission for the treatment of frontotemporal dementia. Orphan drug designation in the EU may be granted for medicines in development for debilitating or life-threatening conditions that affect fewer than five in 10,000 people in the EU and provides benefits to the sponsor such as protocol assistance, reduced regulatory fees and extended market exclusivity. AL001 has previously received Fast Track and Orphan Drug designations from the U.S. Food and Drug Administration.

Twelve-month data from the open-label INFRONT-2 Phase 2 clinical trial of AL001 in patients with frontotemporal dementia with a progranulin mutation (FTD-GRN) were presented at the Alzheimer’s Association International Conference (AAIC) held in July 2021. AL001 showed a favorable safety profile, successful elevation of progranulin levels and encouraging evidence that treatment may slow disease progression.(1)

In July, Alector and GlaxoSmithKline (GSK) entered into a global collaboration to co-develop and co-commercialize AL001 and AL101 for the treatment of neurodegenerative diseases, including FTD-GRN, as well as other forms of FTD, ALS, Alzheimer’s disease and Parkinson’s disease. Under the terms of the agreement, Alector will receive $700 million in upfront payments, $500 million of which was received in August 2021 with a second payment to occur in the first quarter of 2022.
Alzheimer’s Disease Portfolio

Two posters were presented at the 2021 AAIC for Alector’s AL002 program providing data from a Phase 1 study in healthy volunteers and details on the design of the ongoing INVOKE-2 Phase 2 clinical trial. AL002 is Alector’s first-in-class monoclonal antibody that binds to the TREM2 receptor to optimize microglial activity in the brain. Alector is developing AL002 as a potential treatment for Alzheimer’s disease in collaboration with AbbVie.
Corporate News

Sam Jackson, M.D., MBA, and Linda Rubinstein joined Alector’s executive leadership team assuming the roles of interim Chief Medical Officer and interim Chief Financial Officer, respectively.
Dr. Jackson joined Alector in 2020 as Senior Vice President, Clinical Sciences overseeing Alector’s clinical science and clinical operations functions.
Ms. Rubinstein brings significant industry and financial experience to Alector, having served as Chief Financial Officer or interim Chief Financial Officer to numerous biotechnology companies.
Elizabeth Garofalo, M.D., a veteran biopharmaceutical executive with more than 25 years of experience in global clinical development and regulatory affairs with a focus on neurology, was appointed to Alector’s Board of Directors.
Third Quarter 2021 Financial Results

Revenue. Collaboration revenue for the quarter ended September 30, 2021, was $182.4 million, compared to $5.9 million for the same period in 2020. In August, Alector received $500 million as part of the collaboration agreement with GSK. The increase in 2021 third quarter revenue was mainly due to the recognition of $179.8 million of the upfront payment from GSK.

R&D Expenses. Total research and development expenses for the quarter ended September 30, 2021, were $43.1 million, compared to $43.8 million for the quarter ended September 30, 2020. The decrease in R&D expenses was attributable to timing of manufacturing of drug supply and clinical trials start-up costs.

G&A Expenses. Total general and administrative expenses for the quarter ended September 30, 2021, were $13.0 million, compared to $15.8 million for the same period in 2020. This decrease was primarily due to reduced legal fees.

Net Income (Loss). For the quarter ended September 30, 2021, Alector reported a net income of $126.6 million, or $1.56 net income per share, compared to a net loss of $52.7 million, or $0.67 net loss per share, for the same period in 2020.

Cash Position. Cash, cash equivalents, and marketable securities were $777.9 million as of September 30, 2021.

On November 4, 2021 Emergent BioSolutions Inc. (NYSE: EBS) reported financial results for the third quarter ended September 30, 2021 (Press release, Emergent BioSolutions, NOV 4, 2021, View Source [SID1234594419]).

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"Emergent’s core products and service businesses remain strong as evidenced by our accomplishments this quarter," said Robert G. Kramer, president and CEO of Emergent BioSolutions. "We have secured renewals of multiple medical countermeasure supply contracts, made pipeline advancements, implemented organizational enhancements that better serve our customers, and are pursuing new business prospects. We are confident in our 2024 growth plan and remain focused on our mission – to protect and enhance life."

Q3 2021 AND OTHER RECENT BUSINESS

Announced a mutual agreement with the U.S. Department of Health and Human Services (HHS) to terminate the Company’s 2012 Center for Innovation in Advanced Development and Manufacturing (CIADM) contract to establish a public-private partnership for pandemic preparedness, along with all associated task orders, including the 2020 task order to reserve capacity and expand manufacturing for third-party COVID-19 vaccine and therapeutic candidates
Initiated a pivotal Phase 3 safety and immunogenicity study to evaluate CHIKV VLP, the company’s single-dose chikungunya virus virus-like particle vaccine candidate
Secured a multi-year development and manufacturing agreement with Providence Therapeutics, valued at approximately $90 million, for its mRNA COVID-19 vaccine candidate
Received a contract modification to the 2016 AV7909 (Anthrax Vaccine Adsorbed with Adjuvant) development and procurement contract with the U.S. government, valued at approximately $399 million, to deliver doses of AV7909 to the Strategic National Stockpile (SNS) over 18 months
Received Orphan Drug designation from the U.S. Food and Drug Administration (FDA) for AV7909
Announced inclusion of the company’s SARS-CoV-2 Immune Globulin Intravenous (Human) (COVID-HIG) plasma-derived therapy in a Phase 3 safety and efficacy study, INSIGHT-012, sponsored by the National Institute of Allergy and Infectious Diseases of the National Institutes of Health, evaluating hyperimmune intravenous immunoglobulin for outpatient COVID-19 treatment
Q3 2021 FINANCIAL PERFORMANCE (1)

Product Sales, net
NARCAN Nasal Spray
For Q3 2021, revenues from NARCAN (naloxone HCI) Nasal Spray increased $44.5 million as compared to Q3 2020. The increase is driven by continued growth in unit sales to the U.S. public interest and commercial retail markets as well as customer channels in Canada.

ACAM2000
For Q3 2021, revenues from ACAM2000 (Smallpox (Vaccinia) Vaccine, Live) increased $79.7 million as compared to Q3 2020. The increase is largely driven by the timing of deliveries to the U.S. government (USG), specifically the Strategic National Stockpile (SNS). The revenues recognized in Q3 2021 are a result of a recent option exercise in July 2021 by the USG valued at approximately $182 million. The Company expects to deliver the remaining units under this option exercise during the fourth quarter of 2021.

Anthrax vaccines
For Q3 2021, revenues from Anthrax vaccines decreased $58.3 million as compared to Q3 2020. The decrease is largely driven by timing of deliveries to the USG, specifically the SNS. The Company received an AV7909 contract modification in September 2021 wherein the Company expects to deliver additional doses of AV7909 over 18 months from the date of execution of the contract modification valued at approximately $399 million.

Other (4)
For Q3 2021, revenues from other product sales were consistent as compared to Q3 2020. During the quarter, an increase in sales of VIGIV [Vaccinia Immune Globulin Intravenous (Human)] was offset by a decline in sales of BAT [Botulism Antitoxin Heptavalent (A, B, C, D, E, F, G) – (Equine)], largely driven by timing of deliveries to the USG, specifically the SNS.

CDMO Services
For Q3 2021, revenue from contract development and manufacturing services increased $59.5 million as compared to Q3 2020. This increase is largely due to arrangements with innovator manufacturers to address the COVID-19 pandemic, specifically Johnson & Johnson, as well as out-of-period adjustments (see discussion below entitled "Out-of-Period Adjustments").

CDMO Leases
During Q3 2021, the Company determined that it was necessary to classify the public-private partnership with the Biomedical Advanced Research and Development Authority (BARDA) as a lease rather than a stand-ready arrangement. This change has been considered as part of the immaterial out-of-period adjustment (see discussion below entitled "Out-of-Period Adjustments"). As such, the Company is now separately disclosing lease revenues on the statement of operations. For Q3 2021, revenue from contract development and manufacturing leases decreased $175.0 million largely due to a reduction in lease revenues associated with the public-private partnership with BARDA as the Company recognized revenue of $85.9 million in Q3 2020 and recorded a reversal of revenue of $86.0 million during Q3 2021 based on the lack of cash collections under the arrangement in recent months. In November 2021, the Company and BARDA mutually terminated this arrangement ending the public-private partnership with BARDA. As a result of the termination, the Company expects to record CDMO lease revenue in the fourth quarter 2021 to reflect the remaining unrecognized contract value and associated payments of approximately $155.7 million.

Contracts and Grants
For Q3 2021, revenues from contracts and grants decreased $9.0 million as compared to Q3 2020. The decrease is primarily due to a decrease in activities associated with the COVID-HIG therapeutic product candidate. As a result of the CIADM base contract termination, the Company expects to record approximately $60.0 million of contracts and grants revenue during the fourth quarter 2021.

Operating Expenses

Cost of Product Sales
For Q3 2021, cost of product sales decreased $17.0 million as compared to Q3 2020. The decrease in cost is primarily due to significant items in the prior year that did not recur in the current period offset by higher volume of product sales, specifically NARCAN Nasal Spray and ACAM2000. During Q3 2020, the Company incurred charges of $30.2 million related to the Company’s contingent consideration liabilities and $13.8 million related to a write-down of inventory balances related to the Company’s travel health vaccines.

Cost of CDMO
For Q3 2021, cost of CDMO increased $85.5 million as compared to Q3 2020. The increase in cost is primarily due to increases in CDMO services and additional investments in manufacturing and quality systems and capabilities, largely from the Company’s arrangements to address the COVID-19 pandemic and out-of-period adjustments (see discussion below entitled "Out-of-Period Adjustments").

Research and Development
For Q3 2021, research and development expenses decreased $34.8 million as compared to Q3 2020. The decrease is primarily due to a decline in costs associated with the Company’s COVID-HIG therapeutic product candidate and a non-recurring charge for an impairment of the Company’s in-process research and development (IPR&D) intangible asset of $29.0 million during Q3 2020.

Selling, General and Administrative
For Q3 2021, selling, general and administrative expenses increased $6.6 million due to organizational growth in headcount and professional services in support of the expansion of the Company’s business operations.

Out-of-Period Adjustments
During the three months ended September 30, 2021, the Company made immaterial out-of-period adjustments related to its revenue recognition policy for contract development and manufacturing (CDMO) services and classification of the BARDA public-private partnership as a lease. These adjustments resulted in out-of-period increases of $38.3 million in CDMO service revenue, $36.9 million of costs of product sales and CDMO services for the three months ended September 30, 2021 with a net impact to income before income taxes of $1.4 million.

Additional Financial Information
Product Margin and Adjusted Product Margin (2)

For Q3 2021, product margin increased $85.3 million as compared to Q3 2020. Adjusted product margin increased $42.2 million as compared to Q3 2020. The increase in gross margin is primarily due to the $44.0 million of charges related to inventory write-offs of the travel health vaccines and the Company’s contingent consideration liabilities which were not recurring in Q3 2021. Adjusted product margin percent is consistent from Q3 2021 as compared to Q3 2020.

For Q3 2021, CDMO margin decreased $26.0 million as compared to Q3 2020. Adjusted CDMO margin decreased $29.1 million as compared to Q3 2020. The decline in CDMO margin and adjusted CDMO margin is primarily due to an increase in CDMO service activities, increase in costs due to out-of-period adjustments (see discussion below entitled "Out-of-Period Adjustments") and additional costs to support remediation efforts for our COVID-19 manufacturing activities.

For Q3 2021, capital expenditures increased largely due to the Company’s continued investments associated with increased capacity and capabilities at the Company’s Rockville facility. The increase in gross capital expenditures was offset by reimbursements of $5.7 million related to arrangements funded by the USG.

2021 FINANCIAL FORECAST

For full year 2021, the Company’s updated forecast includes the following financial metrics:

The Company’s financial forecast for 2021 includes the following additional considerations:

Revised Considerations

Gross margin reflects the impact of the Q3 2021 performance as well as expectations for the remainder of the year.
CDMO services revenue reflects the impact of the mutual agreement with HHS to end the Company’s involvement in the CIADM program and to close out remaining obligations under the CIADM base contract and related task orders. This agreement reduces the total contract value to be realized under the 2020 task order to $470.9 million from $650.8 million.
Unchanged Considerations

Narcan Nasal Spray revenues assume the naloxone market remains competitive and incorporates the impact of at least one new branded entrant into the market (one branded competitor entered the market during the third quarter of 2021), as well as no generic entrant into the market prior to the anticipated appellate decision related to the pending patent litigation, which is expected by the end of 2021.
Anthrax vaccines revenue is expected to continue to primarily reflect procurement of AV7909 under the terms of the Company’s existing contract with BARDA at a more normalized annual level.
ACAM2000vaccine revenues incorporate the expected full delivery of product under the $182 million option exercise received in July 2021 as well as other international sales.
CDMO services revenue reflects the continued manufacturing of Johnson & Johnson’s COVID-19 vaccine bulk drug substance. On July 29th, the Company announced that it was informed by the FDA that it can resume production at its Bayview manufacturing facility.
Total revenues, specifically other product sales, are expected to be impacted due to the Company’s assumption that a new raxibacumab contract will be awarded later than previously planned.
R&D expenses are expected to reflect continued pipeline progress across the portfolio, including the assumption of at least one Phase 3 launch and one Biologics License Application (BLA)/Emergency Use Authorization (EUA) filing.
Capital expenditures, net of reimbursement, are expected to be in a range of 8% to 9% of total revenues, reflecting ongoing investments in capacity and capability expansions in support of the Company’s CDMO services business and product portfolio.
FOOTNOTES

(1) All financial information incorporated within this release is unaudited.
(2) See "Reconciliation of Net Income to Adjusted Net Income," "Reconciliation of Net Income to Adjusted EBITDA," "Reconciliation of Product Margin and Adjusted Product Margin," "Reconciliation of CDMO Margin and Adjusted CDMO Margin" and "Reconciliation of Net Research and Development Expenses" for a definition of terms and the reconciliation tables.
(3) Product sales, net are reported net of variable consideration including returns, rebates, wholesaler fees and prompt pay discounts.
(4) Other can include a combination of sales of any of the following products: BAT, VIGIV, Anthrasil, raxibacumab, RSDL, Trobigard, Vivotif, and Vaxchora.
(5) CDMO backlog is defined as estimated remaining contract value as of the indicated period pursuant to signed contracts, the majority of which is expected to be recognized over the next 24 months.
(6) CDMO new business is defined as initial value of contracts secured as well as incremental value of existing contracts modified within the indicated period and is incorporated into Backlog.
(7) CDMO opportunity funnel is defined as proposal values from new work with new customers, new work with existing customers and extensions/expansions of existing contracts with existing customers that, if converted to new business, the majority of which is expected to be realized over the next 24 months. This excludes any value associated with an extension of the commercial supply agreement (CSA) with Johnson & Johnson.

CONFERENCE CALL, PRESENTATION SUPPLEMENT AND WEBCAST INFORMATION

Company management will host a conference call at 5:00 pm (Eastern Time) today, November 4, 2021, to discuss these financial results. The conference call and presentation supplement can be accessed from the Company’s website or through the following

On November 4, 2021 Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment of cancer, reported third quarter 2021 financial results and provided a corporate update (Press release, Kura Oncology, NOV 4, 2021, View Source [SID1234594418]).

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"I am pleased by the progress our team has made over the past quarter, highlighted by accelerated enrollment in the Phase 1b expansion cohorts for KO-539," said Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology. "We continue to be encouraged by preliminary results from the Phase 1b study, with evidence of activity at both doses and a favorable safety and tolerability profile. Pending determination of a recommended Phase 2 dose, we are designing a development strategy that builds on the potential to register KO-539 as a monotherapy, while giving us the flexibility to get to larger opportunities in combination more quickly, including in earlier lines of therapy. We look forward to providing a more comprehensive update, including results from the Phase 1a and Phase 1b studies, at a future medical meeting."

Recent Highlights

Continued enrollment in Phase 1b expansion cohorts for KO-539 – Kura is currently enrolling two expansion cohorts in the Phase 1b portion of its KOMET-001 trial – a lower dose of 200 mg and a higher dose of 600 mg. Each cohort is comprised of patients with NPM1-mutant or KMT2A-rearranged relapsed or refractory acute myeloid leukemia (AML). Approximately half of an estimated 40 global sites are now actively screening patients for enrollment in the study. The Company maintains its enrollment guidance of 12 evaluable patients in each cohort by the first quarter of 2022. Once enrolled, patients in each cohort will be assessed for safety and tolerability, pharmacokinetics and efficacy to determine the recommended Phase 2 dose for KO-539.

Comprehensive clinical development strategy for KO-539 – Pending determination of a recommended Phase 2 dose, Kura intends to conduct a comprehensive clinical development plan for KO-539, both as a monotherapy and in combination. This development strategy builds on the potential to register KO-539 as a monotherapy while giving the Company the flexibility to address larger opportunities in combination more efficiently, including earlier lines of therapy.

ASH presentation to highlight potential for synergistic activity of KO-539 with venetoclax – Encouraging pre-clinical data has been generated through a research collaboration with Dr. Kapil Bhalla at the MD Anderson Cancer Center highlighting the molecular mechanisms of anti-leukemic activity for KO-539 and its potential for synergistic activity in combination with venetoclax in KMT2A-rearranged and NPM1-mutant AML models. These data have been accepted for presentation at the upcoming American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2021. The abstract was published earlier today and is now available on the ASH (Free ASH Whitepaper) website.

Final results from Phase 2 study of tipifarnib in T-cell lymphoma at ASH (Free ASH Whitepaper) – Final results from a Phase 2 study of tipifarnib in patients with relapsed or refractory peripheral T-cell lymphoma (PTCL) have been accepted for oral presentation by Dr. Thomas Witzig, Hematologist at Mayo Clinic and the study’s lead investigator, at the upcoming ASH (Free ASH Whitepaper) Annual Meeting. The abstract is now available on the ASH (Free ASH Whitepaper) website. The clinical benefit demonstrated in patients with certain subtypes of T-cell lymphoma, including an overall response rate of 56.3% (18/32) and a median overall survival of 32.8 months in patients with angioimmunoblastic T-cell lymphoma (AITL), underscores the potential to target farnesyl transferase to drive clinical benefit in patients with cancer.

First clinical site activated in Phase 1/2 trial of tipifarnib plus alpelisib in HNSCC – In July, Kura announced a clinical collaboration with Novartis to evaluate the combination of tipifarnib and the PI3Kα inhibitor alpelisib in patients with head and neck squamous cell carcinoma (HNSCC). The Company believes this combination has the potential to increase the total addressable population for tipifarnib to as much as 50% of patients with HNSCC and is now beginning a Phase 1/2 clinical trial (KURRENT) of tipifarnib in combination with alpelisib in patients who have HRAS- and/or PIK3CA-dependent HNSCC. Kura has now activated the first clinical site and expects to dose the first patient in the trial by the end of 2021.

Next-generation FTI program focused on delaying onset of drug resistance in large solid tumor indications – Last quarter, Kura nominated KO-2806 as the lead development candidate in its next-generation farnesyl transferase inhibitor (FTI) program. The Company’s next-generation FTI program is designed to target innovative biology and address large solid tumor indications of high unmet need through rational combinations, with a focus on delaying the onset of drug resistance. IND-enabling studies of KO-2806 are ongoing.
Financial Results and Guidance

Research and development expenses for the third quarter of 2021 were $22.4 million, compared to $16.6 million for the third quarter of 2020.

General and administrative expenses for the third quarter of 2021 were $11.3 million, compared to $7.6 million for the third quarter of 2020.

Net loss for the third quarter of 2021 was $33.4 million, compared to a net loss of $23.8 million for the third quarter of 2020. This included non-cash share-based compensation expense for the third quarter of 2021 of $6.1 million, compared to $3.4 million for the same period in 2020.

Cash, cash equivalents and short-term investments totaled $543.4 million as of September 30, 2021, compared with $633.3 million as of December 31, 2020.

Operating expenses for the full year 2021 are expected to be in the range of $130 million to $140 million.

Net cash used in operating activities for the full year 2021 is expected to be $105 million to $115 million.

Management expects that current cash, cash equivalents and short-term investments will be sufficient to fund current operations into 2024.
Upcoming Milestones

Dose first patient in the KURRENT Phase 1/2 study of tipifarnib in combination with alpelisib by the end of 2021.

Complete enrollment of 24 evaluable patients in the KOMET-001 Phase 1b expansion cohorts by the first quarter of 2022.

Determine the recommended Phase 2 dose of KO-539 by the first quarter of 2022.

Submit an IND application for KO-2806 by the end of 2022.
Conference Call and Webcast

Kura’s management will host a webcast and conference call at 4:30 p.m. ET / 1:30 p.m. PT today, November 4, 2021, to discuss the financial results for the third quarter 2021 and provide a corporate update. The live call may be accessed by dialing (866) 269-4260 for domestic callers and (323) 347-3277 for international callers and entering the conference code: 9395801. A live webcast and archive of the call will be available online from the investor relations section of the company website at www.kuraoncology.com.

On November 4, 2021 DYNAVAX TECHNOLOGIES CORPORATION (Nasdaq: DVAX), a biopharmaceutical company focused on developing and commercializing novel vaccines, reported financial results for the third quarter ended September 30, 2021 and provided a business update (Press release, Dynavax Technologies, NOV 4, 2021, View Source [SID1234594417]).

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"This quarter we continued to make strong progress with both our lead commercial vaccine, HEPLISAV-B, and our CpG 1018 vaccine adjuvant platform," commented Ryan Spencer, Chief Executive Officer of Dynavax. "Our belief in the significant value of both assets is reinforced by the continued growth in market share and revenue for HEPLISAV-B along with multiple positive data readouts for adjuvanted COVID-19 vaccine candidates demonstrating the capabilities of CpG 1018 to help drive efficacy and high levels of antibodies while maintaining a favorable tolerability profile. Strong execution on a thoughtful, timely strategy has resulted in $244 million in year-to-date total revenue and $215 million in cash flow from operations, generating approximately $414 million in cash and equivalents at the end of this quarter which further enables our ability to continue to make investments which we believe will drive long-term value."

THIRD QUARTER AND RECENT BUSINESS UPDATE

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

HEPLISAV-B achieved another quarterly high with $22.7 million in revenue during the third quarter of 2021, compared to $11.6 million for the third quarter 2020. This increase was primarily driven by continued success in the field targeted accounts. Market share in the accounts targeted by the field sales team increased to 33.5%, up from 23% in the third quarter of 2020. With a consistent seroprotection rate of over 90% across all patients and the only FDA-approved two-dose hepatitis B vaccine for adults that is completed in one month, in a market where three-dose compliance is known to be a significant challenge, the Company believes HEPLISAV-B can protect more adults against hepatitis B than all other competitor vaccines.

The Centers for Disease Control and Prevention’s (CDC) Advisory Committee on Immunization Practices (ACIP) at its November 2021 meeting voted unanimously to recommend that all adults 19 to 59 years of age should receive hepatitis B vaccination. This recommendation greatly simplifies the identification of patients who need a hepatitis B vaccine compared to the current risk-based recommendation, and significantly expands the number of adults in the United States who should be vaccinated against hepatitis B.

CpG 1018 (Vaccine Adjuvant)

The Company’s strategy to expand the use of its CpG 1018 adjuvant platform and proven technology in multiple modalities of vaccine development continued to generate positive results in the third quarter. Through global partnerships, multiple data read outs for late-stage clinical trials of COVID-19 vaccine candidates adjuvanted with CpG 1018 generated impressive efficacy, immunogenicity and tolerability results. These clinical results enhance the data supporting CpG 1018’s ability to help enable new and improved vaccines that are effective and well-tolerated.

Net product revenue for CpG 1018 adjuvant during the third quarter of 2021 was $84.3 million, compared to $1.7 million for the third quarter 2020. The increase was associated with commercial supply agreements for COVID-19 collaborations. With the meaningful progress made across our COVID-19 partnership portfolio, the Company now expects 2021 full-year CpG 1018 revenue to be approximately $375- $425 million.

In July, Medigen Vaccine Biologics Corporation received Emergency Authorization (EUA) from the Taiwan Food and Drug Administration for MVC-COV1901, their COVID-19 vaccine adjuvanted with CpG 1018, and began vaccinating Taiwan residents in late August.
In July, Dynavax and Biological E (Bio E) entered into a commercial supply agreement for the use of CpG 1018 adjuvant in the commercial production of Bio E’s subunit COVID-19 vaccine candidate, CORBEVAX. Upon completion of their Phase 2/3 clinical trial in India and subsequent EUA, Bio E stated India’s Union Ministry of Health has reserved 300 million doses of CORBEVAX.
In September, Clover Biopharmaceuticals reported positive data for their protein-based COVID-19 vaccine candidate, SCB-2019 (CpG 1018/Alum) adjuvanted with Dynavax’s CpG 1018, which achieved the primary and secondary efficacy endpoints in SPECTRA, a global pivotal Phase 2/3 clinical trial that enrolled over 30,000 participants. Vaccine efficacy was successfully demonstrated in an environment where 100% of SARS-CoV-2 strains observed in the efficacy analysis were variants. SCB-2019 (CpG 1018/Alum) demonstrated a favorable safety and tolerability profile. Subject to receiving Emergency Use Listing (EUL) from the World Health Organization (WHO), Clover said it plans to supply up to 414 million doses of its COVID-19 vaccine candidate globally through the COVAX Facility.
In September, Dynavax and the U.S. Department of Defense (DOD) executed an agreement for approximately $22 million over two and a half years to develop a recombinant plague vaccine adjuvanted with CpG 1018. Enrollment for the Phase 2 clinical trial is expected to commence in 2022.
In October, Valneva SE reported positive topline data for their inactivated whole virus COVID-19 vaccine candidate, VLA2001, adjuvanted with Dynavax’s CpG 1018, in Cov-Compare, a comparative immunogenicity PHASE 3 trial in approximately 4,000 adults. The trial successfully met both co-primary endpoints of superior neutralizing antibody titer levels compared to the active comparator vaccine, AstraZeneca’s AZD1222 (ChAdOx1-S), and neutralizing antibody seroconversion rate above 95%. VLA2001 was well-tolerated, demonstrating a statistically significant better tolerability profile compared to AZD1222.
Corporate Updates

In October, Scott Myers was appointed to Board of Director and elected Chairman.
Upcoming Milestones

Multiple CpG 1018 COVID-19 collaboration partners’ regulatory submissions for emergency or conditional use authorization are expected by the end of 2021 and may provide additional revenue opportunity in 2022.
Data from Tdap-1018 in the ongoing Phase 1 clinical trial for an improved tetanus, diphtheria, and acellular pertussis booster vaccine candidate adjuvanted with CpG 1018 are expected in the first quarter of 2022.
FINANCIAL RESULTS FOR THE THIRD QUARTER

Product Revenue, Net.

Total revenue for the third quarter of 2021 was $108.3 million.

HEPLISAV-B product revenue, net was $22.7 million in the third quarter of 2021 compared to $11.6 million in the same period in 2020.
CpG 1018 product revenue, net was $84.3 million in the third quarter of 2021 compared to $1.7 million in the same period in 2020. As CpG 1018 revenues are generally recorded upon shipment to a customer, there may be fluctuations in revenues between quarters, as shipments often consist of large-sized batches.
Cost of Sales – Product. Cost of sales – product for the third quarter 2021 increased to $60.1 million, compared to $4.0 million for the third quarter of 2020. The increase was primarily due to manufacturing costs for increased volumes of CpG 1018 and HEPLISAV-B sold to customers.

Research and Development Expenses (R&D). R&D expenses for the third quarter of 2021 decreased to $6.2 million, compared to $8.5 million for the third quarter of 2020. The decrease is primarily associated with certain non-recurring expenses incurred in the third quarter of 2020 associated with the wind-down of the Company’s legacy immuno-oncology business.

Selling, General and Administrative Expenses (SG&A). SG&A expenses for the third quarter of 2021 increased to $26.9 million, compared to $21.5 million for the third quarter of 2020. This increase is primarily driven by compensation and related personnel costs, including non-cash stock-based compensation, associated with higher headcount.

Income (loss) from Operations and Net Income (loss). Income from operations for the third quarter of 2021 was $16.1 million compared to a loss from operations of $13.8 million in the third quarter of 2020. Net loss for the third quarter of 2021 was $28.4 million compared to net income of $4.4 million for the third quarter of 2020.

Other income (expense). Other income (expense) includes the change in fair value of warrant liability which is a non-cash adjustment to fair value each reporting period. The change in fair value of warrant liability for the third quarter of 2021 resulted in a loss of $45.1 million, compared to a gain of $21.2 million in the third quarter of 2020.

Earnings per share. Basic and diluted net loss per share were ($0.24), for the third quarter of 2021, compared to basic net income per share of $0.04 and diluted net loss per share of ($0.15) in the third quarter of 2020.

Cash Position and cash flow from operations. Cash, cash equivalents and marketable securities totaled $414.2 million on September 30, 2021. Cash flow from operations for the nine months ended September 30, 2021 was approximately $215.0 million.

CONFERENCE CALL AND WEBCAST INFORMATION

Dynavax will hold a conference call today 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 www.dynavax.com. Alternatively, participants may dial (866) 420-4066 or (409) 217-8237 and refer to conference ID 5994808. A replay of the webcast will be available for 30 days following the live event.

Please see Important Safety Information below.

For more information about HEPLISAV-B, visit View Source

About Hepatitis B

Hepatitis B is a viral disease of the liver that can become chronic and lead to cirrhosis, liver cancer and death. The hepatitis B virus is 50 to 100 times more infectious than HIV,I and transmission is on the rise. There is no cure for hepatitis B, but effective vaccination can prevent the disease.

In adults, hepatitis B is spread through contact with infected blood and through unprotected sex with an infected person. The U.S. Centers for Disease Control (CDC) recommends vaccination for those at high risk for infection due to their jobs, lifestyle, living situations and travel to certain areas.II Because people with diabetes are particularly vulnerable to infection, the CDC recommends vaccination for adults age 19 to 59 with diabetes as soon as possible after their diagnosis, and for people age 60 and older with diabetes at their physician’s discretion.III Approximately 20 million U.S. adults have diabetes, and 1.5 million new cases of diabetes are diagnosed each year.IV

About HEPLISAV-B

HEPLISAV-B is an adult hepatitis B vaccine that combines hepatitis B surface antigen with Dynavax’s proprietary Toll-like Receptor (TLR) 9 agonist CpG 1018 to enhance the immune response. Dynavax has worldwide commercial rights to HEPLISAV-B.

Important U.S. Product Information

HEPLISAV-B is indicated for prevention of infection caused by all known subtypes of hepatitis B virus in adults age 18 years and older.

Safety and effectiveness of HEPLISAV-B have not been established in adults on hemodialysis.

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 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%).

Important EU/EEA Product Information

HEPLISAV B is indicated for active immunisation against hepatitis B virus infection (HBV) caused by all known subtypes of hepatitis B virus in adults 18 years of age and older.

The use of HEPLISAV B should be in accordance with official recommendations.

It can be expected that hepatitis D will also be prevented by immunization with HEPLISAV B as hepatitis D (caused by the delta agent) does not occur in the absence of hepatitis B infection.

For full EU/EEA. Prescribing Information for HEPLISAV-B, click here.

Important EU/EEA Safety information

Do not receive HEPLISAV B if you have had a sudden life-threatening, allergic reaction after receiving HEPLISAV B in the past, or if you are allergic to any of components of this vaccine, including yeast. Signs of an allergic reaction may include itchy skin, rash, shortness of breath and swelling of the face or tongue.

Appropriate medical treatment and supervision should be readily available in case of rare anaphylactic reactions following the administration of the vaccine.

The administration of HEPLISAV B should be postponed in subjects suffering from acute severe febrile illness.

Immunocompromised persons may have a diminished immune response to HEPLISAV B.

Because of the long incubation period of hepatitis B, it is possible for unrecognised HBV infection to be present at the time of immunisation. HEPLISAV B may not prevent HBV infection in such cases.

There are very limited data on the immune response to HEPLISAV B in individuals who did not mount a protective immune response to another hepatitis B vaccine.

As a precautionary measure, it is preferable to avoid the use of HEPLISAV B during pregnancy. Vaccination during pregnancy should only be performed if the risk-benefit ratio at the individual level outweighs possible risks for the fetus.

The most common patient-reported side effects reported within 7 days of vaccination were pain, swelling or redness at the injection site, feeling tired, headache, muscle aches, feeling unwell and fever.

About CpG 1018 Adjuvant

CpG 1018 is the adjuvant used in HEPLISAV-B. Dynavax developed CpG 1018 adjuvant to provide an increased vaccine immune response, which has been demonstrated in HEPLISAV-B. CpG 1018 adjuvant provides a well- developed technology and a significant safety database, potentially accelerating the development and large-scale manufacturing of novel or improved vaccines.