On May 4, 2021 Deciphera Pharmaceuticals, Inc. (NASDAQ:DCPH) reported financial results for the first quarter ended March 31, 2021, and provided a corporate update (Press release, Deciphera Pharmaceuticals, MAY 4, 2021, View Source [SID1234579074]).

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"We are excited by the continuing successful commercial launch of QINLOCK in the U.S. as we solidify its position among GIST prescribers and patients and expand access to this important medicine globally. We also remain focused on realizing QINLOCK’s potential in earlier lines of therapy," said Steve Hoerter, President and Chief Executive Officer of Deciphera. "We expect the INTRIGUE Phase 3 top-line results in the fourth quarter of this year and believe QINLOCK has the potential to transform the treatment of GIST for this larger, second-line patient population. Building on our commitment to fully explore the potential of QINLOCK to benefit patients with GIST, we are excited to announce today our plans to initiate a Phase 1b/2 study combining QINLOCK with binimetinib, an approved MEK inhibitor. Our enthusiasm for this combination is based on compelling pre-clinical data showing that this combination can induce apoptosis and has the potential to deepen and prolong responses."

Mr. Hoerter continued, "We remain very pleased with the progress and growth for the balance of our pipeline, including the upcoming initiation of the Phase 1 study for our potential first-in-class ULK kinase inhibitor, DCC-3116, in patients with cancers driven by mutant RAS or RAF genes. We look forward to presenting updated data from both the vimseltinib and rebastinib programs in the coming months and plan to finalize registration-enabling studies for both programs before the end of the year."

First Quarter 2021 Highlights and Upcoming Milestones

QINLOCK(ripretinib)
Recorded $20.0 million in QINLOCK net product revenue in the first quarter of 2021, including $19.3 million in U.S. net product revenue.
Received approval in China from the China National Medical Products Administration (NMPA) and from the Hong Kong Department of Health, via our collaboration with Zai Lab, for the treatment of adult patients with fourth-line gastrointestinal stromal tumors (GIST).
Expects potential approval from the European Medicines Agency (EMA) for QINLOCK in the fourth quarter of 2021.
Expects to announce top-line results from the INTRIGUE Phase 3 study in the fourth quarter of 2021.
Expects to present data for QINLOCK patients undergoing intra-patient dose escalation after disease progression in the INVICTUS Phase 3 study at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in June.
Today announced that the Company plans to initiate a Phase 1b/2 study of QINLOCK in combination with binimetinib, an approved MEK inhibitor, to address one of the potential mechanisms of resistance to kinase inhibition, reactivation of the MAPK pathway, in post-imatinib GIST patients.
A publication supporting this approach was recently published online by Molecular Cancer Therapeutics. The results showed that treatment with QINLOCK in combination with MEK inhibitors effectively induced and enhanced apoptotic responses and prevented growth of resistant colonies in both imatinib-sensitive and -resistant GIST cell lines.
Vimseltinib
Expects to present updated data from the ongoing Phase 1/2 study in patients with tenosynovial giant cell tumor (TGCT) in the third quarter of 2021.
Plans to finalize the pivotal development plan for vimseltinib in TGCT in the second half of 2021.
Rebastinib
Expects to present updated data from the ongoing Phase 1b/2 study of rebastinib in combination with paclitaxel in the endometrial cancer cohort at the ASCO (Free ASCO Whitepaper) Annual Meeting in June.
Expects to present updated data from the ongoing Phase 1b/2 study of rebastinib in combination with paclitaxel in the platinum-resistant ovarian cancer cohort in the third quarter of 2021.
Plans to finalize the pivotal development plan for rebastinib in combination with paclitaxel in the second half of 2021.
DCC-3116
Expects to initiate the Phase 1, multicenter, open-label, first-in-human study of DCC-3116 in the second quarter of 2021. The study will evaluate DCC-3116 as a single agent and in combination with trametinib in patients with advanced or metastatic tumors with a mutant RAS or RAF gene. Currently, expansion cohorts are planned in patients with advanced or metastatic pancreatic ductal adenocarcinoma with KRAS or BRAF mutations, non-small cell lung cancer with KRAS, NRAS, or BRAF mutations, colorectal cancer with KRAS, NRAS, or BRAF mutations, and melanoma with NRAS or BRAF mutations.
Upcoming Scientific Congress Presentations

2021 ASCO (Free ASCO Whitepaper) Annual Meeting, June 4-8. E-poster presentations will be available on-demand via the ASCO (Free ASCO Whitepaper) Meeting Library beginning on Friday, June 4 at 9:00 AM ET.
QINLOCK
E-poster presentation: Intra-patient dose escalation (IPDE) of ripretinib after disease progression in patients with advanced gastrointestinal stromal tumor (GIST): Analyses from the phase 3 INVICTUS study.
Rebastinib
E-poster presentation: Open-label, multicenter, phase 1b/2 study of rebastinib in combination with paclitaxel to assess safety and efficacy in patients with advanced or metastatic endometrial cancer.
First Quarter Financial Results

Revenue: Total revenue for the first quarter of 2021 was $25.2 million, which includes $20.0 million of net product revenue from sales of QINLOCK and $5.2 million of collaboration revenue. Net product revenues for the first quarter of 2021 included U.S. sales of QINLOCK of $19.3 million and ex-U.S. sales of QINLOCK of $0.7 million. The Company also recognized $5.0 million in collaboration revenue under its license agreement with Zai Lab based on the approval of QINLOCK in China. In the first quarter of 2020, the Company did not generate product revenue.
Cost of Sales: Cost of sales were $0.2 million in the first quarter of 2021. There were no cost of sales in the first quarter of 2020 as no product sales were generated during that period. Cost of sales will not be significant until the initial pre-launch inventory is depleted, and additional inventory is manufactured and sold.
R&D Expenses: Research and development expenses for the first quarter were $55.7 million, compared to $51.4 million for the same period in 2020. The increase was primarily due to personnel and preclinical costs, partially offset by a decrease in clinical trial expenses related to the INTRIGUE Phase 3 study in second-line GIST and the INVICTUS Phase 3 study in fourth-line and fourth-line plus GIST. Non-cash, stock-based compensation was $5.0 million and $3.3 million for the first quarters of 2021 and 2020, respectively.
SG&A Expenses: Selling, general and administrative expenses for the first quarter of 2021 were $30.7 million, compared to $23.9 million for the same period in 2020. The increase was primarily due to personnel costs as well as external spend related to professional fees, including those associated with establishing a targeted commercial infrastructure in key European markets to support a potential launch of QINLOCK in Europe, if approved. Non-cash, stock-based compensation was $6.2 million and $3.7 million for the first quarters of 2021 and 2020, respectively.
Net Loss: For the first quarter of 2021, Deciphera reported a net loss of $61.3 million, or $1.06 per share, compared with a net loss of $72.8 million, or $1.36 per share, for the same period in 2020. The decrease in net loss was primarily a result of product sales during the first quarter of 2021, partially offset by an increase in R&D and SG&A expenses as described above.
Cash Position: As of March 31, 2021, cash, cash equivalents and marketable securities were $502.2 million, compared to $561.3 million as of December 31, 2020. Based on its current operating plans, Deciphera expects its current cash, cash equivalents, and marketable securities together with anticipated product and royalty revenues, but excluding any potential future milestone payments or other payments under its collaboration or license agreements, will enable the Company to fund its operating and capital expenditures into the first half of 2023.
Conference Call and Webcast

Deciphera will host a conference call and webcast to discuss this announcement today, May 4, 2021 at 4:30 PM ET. To access the live call by phone please dial (866) 930-5479 (domestic) or (409) 216-0603 (international); the conference ID is 5470938. A live audio webcast of the event may also be accessed through the "Investors" section of Deciphera’s website at www.deciphera.com. A replay of the webcast will be available for 30 days following the event.

On May 4, 2021, Invitae Corporation (the "Company") reported that entered into a Sales Agreement (the "Agreement") with Cowen and Company, LLC (the "Sales Agent") (Filing, 8-K, Invitae, MAY 4, 2021, View Source [SID1234579100]). The Agreement provides for the issuance and sale by the Company of common stock having an aggregate offering price of up to $400,000,000. Pursuant to the Agreement, the Company may offer and sell the shares in transactions deemed to be an "at-the-market" offering as defined in Rule 415(a)(4) of the Securities Act of 1933, which shares can be sold by the Company from time to time, depending upon market demand, with the Sales Agent acting as an agent for sales.

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The Company will pay the Sales Agent a commission of up to 3.0% of the gross proceeds from the sale of shares of common stock by it as agent under the Agreement. The Agreement provides that the Company will provide customary indemnification rights to the Sales Agent. The Company has no obligation to sell any shares of common stock pursuant to the Agreement and may at any time suspend sales pursuant to the Agreement. Either party may terminate the Agreement pursuant to the terms of the Agreement without liability of any party.

The shares of common stock will be sold pursuant to the Company’s automatic shelf registration statement on Form S-3 (File No. 333-230053), that was filed with the Securities and Exchange Commission and became automatically effective on March 4, 2019, including the related prospectus, dated March 4, 2019, as supplemented by the prospectus supplement dated May 4, 2021. This Current Report on Form 8-K shall not constitute an offer to sell or the solicitation of an offer to buy nor shall there be any sale of these securities in any state in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state.

Under the Agreement, the Company may also sell shares of common stock to the Sales Agent as principal for its own account, at a price to be agreed upon at the time of sale. If the Company sells shares to the Sales Agent as principal, the Company will enter into a separate terms agreement with the Sales Agent, and the Company will describe the agreement in a separate prospectus supplement or pricing supplement.

The foregoing description of the Agreement does not purport to be complete and is subject to and qualified in its entirety by reference to the Agreement, a copy of which is attached hereto as Exhibit 1.1 and is incorporated herein by reference.

On May 4, 2021 Veracyte, Inc. (Nasdaq: VCYT) reported that new data relating to the Prosigna Breast Cancer Gene Signature Assay will be presented at the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) Breast Cancer Virtual Congress 2021 taking place May 5-8 (Press release, Veracyte, MAY 4, 2021, View Source [SID1234579116]).

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"New data accepted for presentation at this year’s ESMO (Free ESMO Whitepaper) Breast Cancer Congress provide further evidence for the importance of genomic testing in breast cancer, particularly to help physicians and patients make better informed treatment decisions based on the unique biology of individual patients’ tumors," said Bonnie Anderson, Veracyte’s chairman and chief executive officer. "We believe such data strengthen the backdrop for our Prosigna Breast Cancer Gene Signature Assay, and greatly appreciate the breast cancer experts who led and participated in these studies."

Following are details of the Prosigna-related posters accepted for presentation at the ESMO (Free ESMO Whitepaper) Breast Cancer Virtual Congress. These abstracts are available to meeting registrants on demand:

Title: Influence of PAM50 on therapeutic decisions in patients with early-stage Luminal Breast Cancer in a single centre

Poster: 62P

First Author: Alejandro Olivares-Hernandez, M.D., M.Sc., IBSAL – Instituto de Investigación Biomédica de Salamanca, Salamanca, Spain

Title: Consensus on the utility of breast cancer multigene signatures in routine clinical practice among European Breast Cancer clinicians – The PROCURE project

Poster: 24P

First Author:

Giuseppe Curigliano, M.D., Ph.D., IEO – Istituto Europeo di Oncologia, Milan, Italy

The first poster will present findings from a prospective study conducted at the University Hospital of Salamanca (Spain), evaluating the influence of the PAM50 gene signature, the foundation of the Prosigna assay, on therapeutic decision-making for 143 patients with early-stage breast cancer.

The second poster will feature initial findings and consensus from the first wave of the PROCURE (Prosigna Clinical Utility Review) project, a European study utilizing the Delphi methodology to generate consensus regarding the clinical utility of genomic tests, including the Prosigna Breast Cancer Gene Signature Assay, in breast cancer treatment. The study is led by an independent scientific committee of eight breast cancer experts with input from 141 breast cancer clinicians practicing in nearly a dozen European countries.

About Prosigna

The Prosigna Breast Cancer Gene Signature Assay is a prognostic genomic test, built from PAM50 molecular subtypes, which combines tumor gene expression with clinicopathologic factors to better inform treatment decisions. The assay is indicated in female breast cancer patients who have undergone either mastectomy or breast-conserving therapy in conjunction with locoregional treatment consistent with standard of care, either as a prognostic indicator for distant recurrence-free survival at 10 years in post-menopausal women with Hormone Receptor-Positive (HR+), lymph node-negative, Stage I or II breast cancer, or lymph node-positive (1–3 positive nodes, or 4 or more positive nodes), Stage II or IIIA breast cancer to be treated with adjuvant endocrine therapy alone, when used in conjunction with other clinicopathological factors. Outside of the United States, the Prosigna Breast Cancer Gene Signature Assay also provides the intrinsic subtypes of the tumor tissue within three groups, low, intermediate and high, by the nCounter Analysis System through decentralized performance.

The Prosigna test is FDA 510(k) cleared in the United States for use on the nCounter Analysis System and is available for use when ordered by a physician. The Prosigna test has received CE Mark and is available for use by healthcare professionals in the European Union and other countries that recognize the CE Mark, as well as in Canada, Israel, Australia, New Zealand and Hong Kong. The assay is covered by Medicare and leading private payers in the United States, and is widely covered by government and private payers in the countries where it is available.

On May 4, 2021 Vivace Therapeutics, Inc., a small molecule discovery and development company developing first-in-class therapies targeting the Hippo pathway, reported the completion of enrollment of the first cohort of patients being treated with VT3989 in its Phase 1 clinical study (Press release, Vivace Therapeurtics, MAY 4, 2021, https://www.prnewswire.com/news-releases/vivace-therapeutics-announces-dosing-of-first-cohort-of-patients-with-its-first-in-class-tead-inhibitor-301283520.html [SID1234579133]). The Company anticipates enrolling the next higher dose cohort in the second part of May.

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The VT3989 Phase 1 study is now open at centers in both the US and Australia (View Source) to evaluate the safety, tolerability, PK and biological activity of VT3989 in patients with refractory metastatic solid tumors, including refractory pleural malignant mesothelioma. The study is currently in dose escalation, to be followed by a dose expansion phase in which patients with NF2 mutant tumors will be enrolled after an optimal dose is determined. "To the best of our knowledge, we are the first company to test a small molecule in the clinic targeting this important pathway. Our preclinical work has shown that tumors with NF2 mutations are quite sensitive to VT3989, especially NF2-deficient mesothelioma," said Leonard Post, Ph.D., Chief Scientific Officer of Vivace Therapeutics.

"The Phase 1 clinical study is designed to test the translatability of our preclinical observations in cancer patients as quickly as possible. We are enrolling patients with a variety of signaling abnormalities during dose escalation and will allow NF2-mutated patients to backfill any previously cleared dose cohorts. In addition, our study design allows for intra-patient dose escalation so that patients who are tolerating VT3989 at a given dose may increase to the highest well-tolerated dose," said Andrew Dorr, M.D., Chief Medical Officer of Vivace Therapeutics.

The Company has filed patent applications across a diverse set of chemotypes, all of which inhibit palmitoylation of members of the transcriptional enhanced associate domain (TEAD) protein family. The latest publication from Vivace Therapeutics titled "Small Molecule Inhibitors of TEAD Auto-palmitoylation Selectively Inhibit Proliferation and Tumor Growth of NF2-deficient Mesothelioma" in Molecule Cancer Therapeutics (View Source) presented some of the preclinical work done by the company.

On May 4, 2021 Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, reported recent business highlights and first quarter 2021 financial results (Press release, Sangamo Therapeutics, MAY 4, 2021, View Source [SID1234579172]).

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"This quarter, we have continued our focus on advancing our lead programs through clinical execution, regulatory interactions, and collaborations with our partners and investigators. We are pleased that enrollment has completed for Pfizer’s lead-in study for the hemophilia A Phase 3 AFFINE trial. Additionally, the EMA granted Orphan Designation and the FDA granted Fast Track Designation to BIVV003, now known as SAR445136, our cell therapy product candidate treating sickle cell disease partnered with Sanofi. Also, we initiated the STEADFAST study for our product candidate treating renal transplant rejection, which we believe is the first-in-human CAR-Treg clinical study," said Sandy Macrae, Chief Executive Officer of Sangamo. "Further, our research engine continued to be highly productive this quarter, advancing both our CAR-Treg programs for autoimmune disorders and our transcriptional regulation therapies for neurological diseases."

Recent Business Highlights

Completed enrollment of patients in Pfizer’s lead-in study to the registrational Phase 3 AFFINE clinical trial of giroctocogene fitelparvovec, a gene therapy product candidate for the treatment of severe hemophilia A, developed in collaboration with Pfizer.
Received Fast Track Designation from the FDA for BIVV003, now known as SAR445136, our cell therapy product candidate for the treatment of sickle cell disease, developed in collaboration with Sanofi. Also, the EMA granted Orphan Designation to SAR445136 based on early clinical data from three treated patients.
Initiated the Phase 1/2 STEADFAST clinical study evaluating TX200, a wholly-owned autologous HLA-A2 CAR-Treg cell therapy product candidate treating patients receiving a HLA-A2 mismatched kidney from a living donor. The first patient is expected to be enrolled in this study by the end of this year.
Published preclinical data on tau- and alpha-synuclein-targeted zinc finger transcriptional repressors in Science Advances and at the 15th International Conference on Alzheimer’s & Parkinson’s Diseases (ADPD), respectively. Also, announced upcoming preclinical alpha-synuclein and C9ORF72 abstracts to be presented at the upcoming 24th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) on May 11, 2021.
First Quarter 2021 Financial Results

Consolidated net loss for the first quarter ended March 31, 2021 was $45.9 million or $0.32 per share, compared to a net loss of $42.9 million or $0.37 per share for the same period in 2020.

Revenues

Revenues for the first quarter ended March 31, 2021 were $26.3 million, compared to $13.1 million for the same period in 2020. The increase of $13.2 million in revenues was primarily due to the recognition of upfront license fees and research reimbursements under our collaboration agreements with Biogen and Novartis, which became effective in April and July 2020, respectively. These increases were partially offset by a decrease in revenue related to our hemophilia A collaboration with Pfizer, as a result of completion of our reimbursable activities in the fourth quarter of 2020.

GAAP and Non-GAAP operating expenses

Total operating expenses on a GAAP basis for the first quarter ended March 31, 2021 were $72.6 million compared to $57.6 million for the same period in 2020. Non-GAAP operating expenses, which exclude stock-based compensation expense, for the first quarter ended March 31, 2021 were $65.1 million compared to $52.0 million for the same period in 2020.

The increase in total operating expenses on a GAAP basis was primarily driven by our higher clinical and manufacturing supply expenses to support the advancement of our clinical trials and our new collaborations along with our increased headcount.

Cash, cash equivalents and marketable securities

Cash, cash equivalents and marketable securities as of March 31, 2021 were $629.5 million compared to $692.0 million as of December 31, 2020.

Guidance for 2021 Reiterated (initial guidance provided on February 24, 2021)

On a GAAP basis, we expect total operating expenses, including non-cash stock-based compensation expenses, to be in the range of approximately $285 million to $305 million.

On a non-GAAP basis, we expect total operating expenses, excluding estimated non-cash stock-based compensation expense of approximately $30 million, to be in the range of approximately $255 million to $275 million.

Conference Call

Sangamo will host a conference call today, May 4, 2021, at 5:00 p.m. Eastern Time, which will be open to the public. The call will also be webcast with live Q&A and can be accessed via a link on the Sangamo Therapeutics website in the Investors and Media section under Events and Presentations.

The conference call dial-in numbers are (877) 377-7553 for domestic callers and (678) 894-3968 for international callers. The conference ID number for the call is 5714729. Participants may access the live webcast via a link on the Sangamo Therapeutics website in the Investors and Media section under Events and Presentations. A conference call replay will be available for one week following the conference call. The conference call replay numbers for domestic and international callers are (855) 859-2056 and (404) 537-3406, respectively. The conference ID number for the replay is 5714729.