On November 5, 2020 Arvinas, Inc. (Nasdaq: ARVN), a clinical-stage biopharmaceutical company creating a new class of drugs based on targeted protein degradation, reported financial results for the third quarter ended September 30, 2020 and provided a corporate update (Press release, Arvinas, NOV 5, 2020, View Source [SID1234570114]).

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"Beginning the Phase 2 portion of the ARV-110 clinical trial marks another significant milestone for Arvinas," said John Houston, Ph.D., President and Chief Executive Officer at Arvinas. "Together with the expected initiation of a cohort expansion of ARV-471 in combination with a CDK4/6 inhibitor before the end of the year, we’re gratified by the strong momentum of our lead programs heading into 2021."

"When combined with our recent disclosure of five additional programs in our preclinical pipeline, these clinical milestones further validate our PROTAC Discovery Engine and the opportunity to create PROTAC degraders that meaningfully improve patient care," added Dr. Houston.

Business Highlights and Recent Developments

Dose escalation in each of the Phase 1/2 clinical trials of ARV-110 and ARV-471 continues.
Arvinas has initiated dosing at a first dose level in a Phase 2 cohort expansion for ARV-110.
The company announced platform updates and disclosed five additional programs from its preclinical pipeline. Arvinas’ portfolio encompasses a range of validated and undruggable targets in oncology, immuno-oncology, and neuroscience. The newly disclosed programs were B-cell lymphoma 6 protein (BCL6), Kirsten rat sarcoma (KRAS) protein, c-Myc, hematopoietic progenitor kinase 1 (HPK1), and mutant huntingtin (mHTT).
Arvinas entered into a collaboration and supply agreement with Pfizer in connection with a planned Phase 1b cohort expansion evaluating ARV-471 in combination with Pfizer’s Ibrance (palbociclib), an oral CDK4/6 inhibitor.
Anticipated Milestones and Expectations

2020 Milestones and Expectations

Arvinas expects to provide a program update for ARV-110, including a Phase 1 dose escalation update and an overview of the recently initiated Phase 2 dose expansion, in December 2020.
For the ARV-471 program, Arvinas expects to provide an update from its Phase 1 dose escalation in December 2020.
Arvinas expects to initiate a Phase 1b cohort expansion of ARV-471 in combination with Ibrance (palbociclib) in the fourth quarter of 2020. The study will evaluate the safety and tolerability of ARV-471 in combination with palbociclib and identify the recommended combination dose of ARV-471 for use with palbociclib.
2021 Milestones and Expectations

Arvinas expects to initiate the first of potentially two Phase 1b investigations of ARV-110 in combination with standard of care agents for mCRPC (e.g., abiraterone) in 2021.
Arvinas expects to share interim data from the Phase 2 dose expansion trial of ARV-110 in 2021.
Arvinas expects to initiate a Phase 2 dose expansion of ARV-471 in 2021.
Arvinas expects to share data from the Phase 1b cohort expansion of ARV-471 in combination with Ibrance (palbociclib) in the second half of 2021.
Arvinas expects to file an investigational new drug (IND) application for ARV-766, an androgen receptor degrader, in the first half of 2021.
Financial Guidance

Based on its current operating plan, Arvinas expects its cash, cash equivalents, and marketable securities will be sufficient to fund its planned operating expenses and capital expenditures into 2022.

Financial Highlights

Cash, Cash Equivalents, and Marketable Securities Position: As of September 30, 2020, cash, cash equivalents, and marketable securities were $248.6 million as compared with $280.9 million as of December 31, 2019. The decrease in cash, cash equivalents and marketable securities of $32.3 million for the first nine months of 2020 was primarily related to cash used for operations of $64.8 million and the purchase of lab equipment and leasehold improvements of $4.6 million, partially offset by net proceeds from the issuance of common stock and exercises of stock options of $33.1 million and $4.0 million received from two collaborators.

Research and Development Expenses: Research and development expenses were $30.0 million for the quarter ended September 30, 2020, as compared with $16.6 million for the quarter ended September 30, 2019. The increase in research and development expenses of $13.4 million for the quarter was primarily related to increases in clinical trials and chemistry, manufacturing and controls expenses associated with our AR program of $4.2 million and our ER program of $5.1 million, in addition to increases in expenses of $4.1 million associated with exploratory programs and investments in platform research.

General and Administrative Expenses: General and administrative expenses were $9.3 million for the quarter ended September 30, 2020, as compared with $8.0 million for the quarter ended September 30, 2019. The increase of $1.3 million was primarily related to an increase in personnel and facility related costs of $2.4 million, offset by a reduction in legal costs of $1.1 million.

Revenues: Revenue related to the license and rights to technology fees and research and development activities related to the collaboration and license agreement with Bayer (Bayer Collaboration Agreement) that was initiated in July 2019, the collaboration and license agreement with Pfizer (Pfizer Collaboration Agreement) that was initiated in January 2018, and the amended and restated option, license and collaboration agreement with Genentech that was initiated in November 2017 (collectively Collaboration Revenue) was $7.6 million for the quarter ended September 30, 2020, as compared with $5.4 million for the quarter ended September 30, 2019. The increase in Collaboration Revenue of $2.2 million primarily related to the revenues from the Bayer Collaboration Agreement and the Pfizer Collaboration Agreement. Total revenue of $30.1 for the three months ended September 30, 2019 included $24.7 million for the contribution of a license to Oerth Bio LLC (the Joint Venture).

Loss from Equity Method Investment: Loss from equity method investment for the quarter ended September 30, 2019 was $24.7 million. This loss was generated from the Joint Venture’s expensing the values associated with the contributed intellectual property from the Joint Venture partners.

Net Loss: Net loss was $30.8 million for the quarter ended September 30, 2020, as compared with $17.7 million for the quarter ended September 30, 2019. The increase in net loss for the quarter was primarily due to increased research and development expenses and increased general and administrative expenses.

About ARV-110
ARV-110 is an orally bioavailable PROTAC protein degrader designed to selectively target and degrade the androgen receptor (AR). ARV-110 is being developed as a potential treatment for men with metastatic castration-resistant prostate cancer.

ARV-110 has demonstrated activity in preclinical models of AR mutation or overexpression, both common mechanisms of resistance to currently available AR-targeted therapies.

About ARV-471
ARV-471 is an orally bioavailable PROTAC protein degrader designed to specifically target and degrade the estrogen receptor (ER) for the treatment of patients with locally advanced or metastatic ER+/HER2- breast cancer.

In preclinical studies, ARV-471 demonstrated near-complete ER degradation in tumor cells, induced robust tumor shrinkage when dosed as a single agent in multiple ER-driven xenograft models, and showed superior anti-tumor activity when compared to a standard of care agent, fulvestrant, both as a single agent and in combination with a CDK4/6 inhibitor.

On November 5, 2020 Cogent Biosciences, Inc. (Nasdaq: COGT), a biotechnology company focused on developing precision therapies for genetically defined diseases, reported that Andrew Robbins, Chief Executive Officer and President, will virtually present a corporate overview at the Piper Sandler 32nd Annual Healthcare Conference taking place December 1-3, 2020 (Press release, Cogent Biosciences, NOV 5, 2020, View Source [SID1234570113]).

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A recording of the presentation will be available to view after the conference under the "Events" tab on the investor relations section of the Cogent Biosciences website at: View Source

On November 5, 2020 VBL Therapeutics (Nasdaq: VBLT), a clinical-stage biotechnology company focused on the discovery, development and commercialization of first-in-class treatments for cancer, reported that Prof. Dror Harats, M.D., Chief Executive Officer, will provide a corporate overview, in a format of a fireside chat with analyst Swayampakula Ramakanth, Ph.D. (RK), at the H.C. Wainwright 6th Annual Israel Conference to be held virtually on November 12th, 2020 (Press release, VBL Therapeutics, NOV 5, 2020, View Source [SID1234570112]).

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An archive recording will be available for listening after the event, on the Investor Relations page of VBL’s corporate website, under "Events & Presentations."

On November 5, 2020 Sierra Oncology, Inc. (SRRA), a late-stage biopharmaceutical company focused on the Phase 3 execution, registration and potential commercialization of momelotinib, a novel drug that may address serious unmet needs in myelofibrosis, reported its financial and operational results for the third quarter ended September 30, 2020 (Press release, Sierra Oncology, NOV 5, 2020, View Source [SID1234570111]).

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"We continue to make good progress in our pivotal MOMENTUM Phase 3 trial for momelotinib and anticipate completing target enrollment by approximately mid-2021 and reporting top-line data in H1 2022, subject to any unforeseen impact from COVID-19," said Dr. Stephen Dilly, President and CEO of Sierra Oncology. "In addition, we anticipate presenting new analyses from previously completed Phase 3 studies of momelotinib at the American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting in December 2020."

"We are also developing our internal capabilities to support potential future regulatory filings and commercialization of momelotinib in the U.S., with key hires made recently in Regulatory Affairs, Technical Operations, Legal, Pharmacovigilance, Commercial, Business Development and Medical Affairs," added Dr. Dilly. "We remain well capitalized to complete enrollment for the MOMENTUM trial, report top-line results and to prepare for filing and pre-commercialization."

Third Quarter 2020 Financial Results (all amounts reported in U.S. currency)
Research and development expenses were $10.4 million for the three months ended September 30, 2020 compared with $10.1 million for the three months ended September 30, 2019. The increase was primarily due to a $1.5 million increase in clinical trial costs for momelotinib and a $0.3 million increase in personnel-related costs pertaining to an increase in stock-based compensation. These increases were offset by a $1.5 million decrease in clinical trial, third-party manufacturing, and research and preclinical costs for SRA737. Research and development expenses included non-cash stock-based compensation of $1.2 million and $0.9 million for the three months ended September 30, 2020 and 2019, respectively.

Research and development expenses were $32.2 million for the nine months ended September 30, 2020, compared with $32.0 million for the nine months ended September 30, 2019. The increase was primarily due to a $7.6 million increase in clinical trial and development costs for momelotinib, and a non-cash charge of $1.5 million pertaining to the change in fair value of an obligation to issue common stock and a warrant to Gilead Sciences, Inc. (Gilead), which were issued during the first quarter of 2020. These increases were offset by a $6.6 million decrease in clinical trial, third-party manufacturing, research and preclinical costs for SRA737, a $1.8 million decrease in personnel-related and allocated overhead costs, and a $0.5 million decrease in third-party manufacturing costs for momelotinib. Research and development expenses included non-cash stock-based compensation of $2.7 million and $3.3 million for the nine months ended September 30, 2020 and 2019, respectively.

General and administrative expenses were $4.1 million for the three months ended September 30, 2020, compared to $3.2 million for the three months ended September 30, 2019. The increase was due to an increase in personnel-related and allocated overhead costs of $0.7 million, primarily relating to an increase in stock-based compensation, and an increase of $0.2 million in professional fees. General and administrative expenses included non-cash stock-based compensation of $0.9 million and $0.4 million for the three months ended September 30, 2020 and 2019, respectively.

General and administrative expenses were $14.9 million for the nine months ended September 30, 2020, compared to $10.0 million for the nine months ended September 30, 2019. The increase was due to a $3.8 million increase in personnel-related and allocated overhead costs, including a non-cash $2.2 million stock-based compensation charge pertaining to the resignation of an executive and $1.0 million of severance charges, and an increase of $1.1 million in professional fees primarily relating to pre-commercial planning costs for momelotinib. General and administrative expenses included non-cash stock-based compensation of $4.0 million and $1.5 million for the nine months ended September 30, 2020 and 2019, respectively.

Other income (expense), net was $22,000 of other expense, net for the three months ended September 30, 2020, compared to $0.3 million of other income, net for the three months ended September 30, 2019. The difference was primarily attributable to a decrease in interest income due to lower interest rates. Other income (expense), net was $15.7 million of other expense, net for the nine months ended September 30, 2020, compared to $1.0 million of other income, net for the nine months ended September 30, 2019. The difference was primarily attributable to a non-cash charge of $16.2 million related to the change in fair value of warrant liabilities which were reclassified to equity in January 2020.

For the three months ended September 30, 2020, Sierra incurred a Generally Accepted Accounting Principles (GAAP) net loss of $14.5 million compared to a GAAP net loss of $12.9 million for the three months ended September 30, 2019. For the nine months ended September 30, 2020, Sierra incurred a GAAP net loss of $62.9 million compared to a GAAP net loss of $40.8 million for the nine months ended September 30, 2019. The GAAP net loss for the nine months ended September 30, 2020 includes a non-cash charge of $16.2 million related to the change in fair value of warrant liabilities included in other income (expense), net and a $1.5 million non-cash charge pertaining to the obligation to issue securities to Gilead included in research and development expenses as mentioned above.

Non-GAAP adjusted net loss was $12.4 million for the three months ended September 30, 2020, compared with a non-GAAP adjusted net loss of $11.6 million for the three months ended September 30, 2019. Non-GAAP adjusted net loss for the three months ended September 30, 2020 and 2019 excludes expenses related to stock-based compensation. For the nine months ended September 30, 2020, Sierra incurred a non-GAAP adjusted net loss of $38.5 million compared to a non-GAAP adjusted net loss of $36.1 million for the nine months ended September 30, 2019. Non-GAAP adjusted net loss for the nine months ended September 30, 2020 excludes expenses related to the change in fair value of warrant liabilities, the change in fair value of the securities issuance obligation, and stock-based compensation. Non-GAAP adjusted net loss for the nine months ended September 30, 2019 excludes expenses related to stock-based compensation. See "Non-GAAP Financial Measures" and "Reconciliation of GAAP to Non-GAAP Financial Measures" below for a reconciliation of this GAAP and non-GAAP financial measure.

Cash and cash equivalents totaled $109.0 million as of September 30, 2020, compared to $147.5 million as of December 31, 2019.

As of September 30, 2020, there were 10,495,732 total shares of common stock outstanding and warrants to purchase 11,102,251 shares of common stock, with an exercise price equal to $13.20 per share. There were 4,246,167 shares issuable upon exercise of stock options and an additional warrant to purchase 1,839 shares.

On November 5, 2020 Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, reported business highlights and financial results for the third quarter ended September 30, 2020 (Press release, Agios Pharmaceuticals, NOV 5, 2020, View Source [SID1234570110]).

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"In the third quarter, we made significant strides toward executing against our key 2020 priorities and getting our medicines to patients who need them," said Jackie Fouse, Ph.D., chief executive officer at Agios. "Most notably, we generated mature overall survival data from our Phase 3 ClarIDHy study of TIBSOVO in previously treated cholangiocarcinoma, prepared for topline data readouts from our two global Phase 3 studies of mitapivat in pyruvate kinase deficiency and advanced our pivotal plans for mitapivat in thalassemia and sickle cell disease. We look forward to building on these achievements with a catalyst-rich fourth quarter and 2021."

THIRD QUARTER 2020 & RECENT HIGHLIGHTS

TIBSOVO (ivosidenib tablets) net sales of $31.7 million, an increase of 15% from the second quarter of 2020.
Reported topline mature overall survival results from ClarIDHy Phase 3 study of TIBSOVO in previously treated IDH1-mutant cholangiocarcinoma patients; submitted final data for presentation at the virtual American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Gastrointestinal Cancers Symposium (ASCO GI).
Withdrew European Marketing Authorization Application (MAA) for TIBSOVO in IDH1-mutant relapsed or refractory acute myeloid leukemia (AML) as a result of feedback from European Medicine Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP).
Initiated Phase 1 healthy volunteers study of AG-946, a next-generation, orally available, small molecule activator of the metabolic enzyme pyruvate kinase-R (PKR).
Appointed Jonathan Biller as chief financial officer, head of legal and corporate affairs.
KEY UPCOMING MILESTONES

Rare Genetic Diseases

Report topline data from ACTIVATE, the company’s global pivotal trial for mitapivat in adults with pyruvate kinase (PK) deficiency who do not receive regular transfusions, by the end of 2020.
Report topline data from ACTIVATE-T, the company’s global pivotal trial for mitapivat in adults with pyruvate kinase (PK) deficiency who receive regular transfusions, in Q1 2021.
Finalize pivotal development plan for mitapivat in thalassemia, including both α-and β-thalassemia, as well as transfusion dependent and non-transfusion dependent patient populations, by the end of 2020.
Finalize pivotal development plan for mitapivat in sickle cell disease in the first half of 2021.
Hematologic Malignancies and Solid Tumors

Deliver full-year 2020 U.S. revenue for TIBSOVO of $113-115 million; the company had previously provided guidance of $105-115 million.
Submit a supplemental new drug application for TIBSOVO in previously treated cholangiocarcinoma in Q1 2021.
Research

Achieve at least one new development candidate by year-end 2020.
UPCOMING INVESTOR EVENTS

November 19, 2020: Agios will host a webinar focused on its pyruvate kinase-R (PKR) activation clinical programs, including Agios’ leadership in elucidating the PKR activation mechanism, the commercial opportunity for each program and the company’s preparations for its first potential launch in a rare genetic disease, expected in 2022.
December 8, 2020: Agios will host a webinar to share updated data from the Phase 1 trial of mitapivat in sickle cell disease, which is being conducted in collaboration with the National Institutes of Health (NIH), as well as an overview of the company’s Phase 3 development plans for mitapivat in patients with thalassemia.
THIRD QUARTER 2020 FINANCIAL RESULTS

Revenue: Total revenue for the third quarter of 2020 was $34.7 million, which includes $31.7 million of net product revenue from sales of TIBSOVO, $2.3 million in collaboration revenue and $0.7 million in royalty revenue from net global sales of IDHIFA under our collaboration agreement with Celgene, now a wholly owned subsidiary of Bristol Myers Squibb. This compares to revenue of $26.0 million for the third quarter of 2019. Total revenue increased 33% from the same period last year, driven by an 82% increase in TIBSOVO net product revenue and offset by a decrease in collaboration revenue due to completion of the research and development services performance obligation with Celgene in the second quarter, as well as an adjustment in sales reserves taken in the third quarter by Bristol Myers Squibb.

Cost of Sales: Cost of sales were $0.6 million for the third quarter of 2020 compared to $0.4 million for the third quarter of 2019.

Research and Development (R&D) Expenses: R&D expenses were $89.6 million for the third quarter of 2020 compared to $101.7 million for the third quarter of 2019. The decrease in R&D expense was primarily attributable to a decrease in TIBSOVO clinical development costs, including winding down the ClarIDHy Phase 3 study.

Selling, General and Administrative (SG&A) Expenses: SG&A expenses were $34.8 million for the third quarter of 2020 compared to $33.0 million for the third quarter of 2019. The increase in SG&A expense was primarily attributable to increased workforce expenses, offset by a decrease in external spending due to COVID-19 and cost savings initiatives.

Net Loss: Net loss was $99.0 million for the third quarter of 2020 compared to $106.2 million for the third quarter of 2019.

Cash Position and Guidance: Cash, cash equivalents and marketable securities as of September 30, 2020 were $722.4 million, compared to $540.5 million as of September 30, 2019. The company expects that its cash, cash equivalents and marketable securities as of September 30, 2020, together with anticipated product revenue, anticipated interest income and anticipated expense reimbursements under our collaboration and license agreements, but excluding any additional program-specific milestone payments, will enable the company to fund its anticipated operating expenses and capital expenditure requirements to the end of 2022.

CONFERENCE CALL INFORMATION
Agios will host a conference call and live webcast with slides today at 8:00 a.m. ET to discuss its third quarter 2020 financial results and recent business activities. To participate in the conference call, please dial 1-877-377-7098 (domestic) or 1-631-291-4547 (international) and refer to conference ID 7578909. The live webcast can be accessed under "Events & Presentations" in the Investors section of the company’s website at www.agios.com. The archived webcast will be available on the company’s website beginning approximately two hours after the event.

About the Agios/Celgene Collaboration
In 2010, Agios and Celgene Corporation, now a wholly owned subsidiary of Bristol Myers Squibb, entered into a collaboration agreement focused on cancer metabolism. Under the terms of the agreement, Celgene has worldwide development and commercialization rights for IDHIFA (enasidenib). Celgene and Agios are currently co-commercializing IDHIFA in the U.S., and Agios continues to conduct certain clinical development activities within the IDHIFA development program. Agios is eligible to receive a $25 million payment upon achievement of a specified ex-U.S. commercial milestone event, as well as reimbursement for costs incurred for its co-commercialization efforts and development activities.