On February 24, 2021 G1 Therapeutics, Inc. (Nasdaq: GTHX), a commercial-stage oncology company, reported a corporate and financial update for the fourth quarter and full-year ended December 31, 2020 (Press release, G1 Therapeutics, FEB 24, 2021, View Source [SID1234575538]).

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"With the recent approval of COSELA, we have a tremendous opportunity to introduce an effective and innovative therapy to proactively address chemotherapy-induced myelosuppression in patients with extensive-stage small cell lung cancer," said Jack Bailey, Chief Executive Officer of G1 Therapeutics. "The launch is underway with our partners at Boehringer Ingelheim, and the initial interest among medical oncologists and oncology nurses is extremely promising. COSELA has a broad mechanism of action, and as such, we are taking a tumor-agnostic approach to the development of COSELA as we advance it into and through multiple Phase 2 and registrational studies. We are confident in the potential for COSELA and look forward to delivering on our goal of improving the lives of as many people living with cancer as possible."

Fourth Quarter 2020 and Recent Highlights

Commercial

COSELA Approved by U.S. Food and Drug Administration (FDA): On February 12, 2021, the FDA approved COSELA for injection to decrease the incidence of chemotherapy-induced myelosuppression in adult patients when administered prior to a platinum/etoposide-containing regimen or topotecan-containing regimen for extensive stage small cell lung cancer (ES-SCLC). It is the first and only therapy designed to help protect bone marrow (myeloprotection) when administered prior to the start of chemotherapy. COSELA is expected to be commercially available through G1’s specialty distributor partner network in early March. (Press release here)
Clinical

Initiated Pivotal Trial of COSELA in Patients with First Line Colorectal Cancer in the Fourth Quarter of 2020: Patient enrollment has begun in a ~300 patient randomized, double-blind placebo-controlled pivotal registrational trial of COSELA in colorectal cancer. The primary endpoint is myelosuppression; secondary endpoints include progression free survival, overall survival, and patient reported outcomes. The data readout is expected in the first half of 2023.
Announced New Upcoming Registrational Trial of COSELA in First-line / Second Line Metastatic Triple-Negative Breast Cancer (mTNBC); Initiation Expected in First Half of 2021: The Company expects to initiate a randomized, double-blind placebo-controlled registrational trial of COSELA in mTNBC in the first half of 2021. The trial is expected to enroll approximately 250 participants, with approximately 170 in the first line cohort. The trial will enroll patients who are both PD-L1-positive and PD-L1-negative in the first-line cohort. The primary endpoint is overall survival; secondary endpoints include patient-reported outcomes measures, safety/tolerability, myeloprotective measures, and progression-free survival. (Press release here)
Announced Two Upcoming Phase 2 Trials of COSELA in First-Line Metastatic Bladder Cancer (mUC) and Second Line / Third Line Non-Small Cell Lung Cancer (NSCLC); Initiation Expected in the First Half of 2021: The Company expects to initiate Phase 2 trials of COSELA in first-line treatment of locally advanced or metastatic bladder cancer (locally advanced or metastatic urothelial carcinoma, or mUC) and second- and third-line treatment of NSCLC, both of which are known immunogenic tumors, in the first half of 2021. Both trials are designed to evaluate the anti-tumor efficacy of COSELA.
Entered into Clinical Trial Collaboration for Upcoming First-Line Locally Advanced or Metastatic Bladder Cancer (mUC) Trial: G1 has entered into a clinical trial collaboration with the alliance between Merck KGaA, Darmstadt, Germany and Pfizer whereby the alliance will contribute clinical supply of the checkpoint inhibitor avelumab to the G1-sponsored and funded first-line mUC trial.
Presented Final Data from the Randomized Phase 2 Trial of COSELA in mTNBC at the 2020 San Antonio Breast Cancer Symposium (SABCS): New data presented at the SABCS meeting showed that COSELA significantly improved overall survival (OS) in patients with mTNBC treated with COSELA prior to administration of a chemotherapy regimen of gemcitabine/carboplatin (GC) compared with GC alone, and that COSELA enhanced immune system function. Compared to GC alone (Group 1), statistically significant improvements in OS were achieved in both COSELA arms (Group 2: HR=0.31, p=0.0016; Group 3: HR=0.40, p=0.0004). As of the data cutoff of July 17, 2020, the median OS was 12.6 months in patients receiving GC alone, not yet reached for Group 2, and 17.8 months in Group 3. The median OS for Groups 2 and 3 combined was 19.8 months (HR=0.37, p<0.0001). Patients with both PD-L1-positive and PD-L1-negative tumors treated with COSELA and GC demonstrated improvement in OS compared to patients receiving GC alone, with the PD-L1-positive subset achieving statistically significant improvement. Data from T-cell clonality analysis suggest that administering COSELA prior to chemotherapy enhanced immune system function. (Poster here)
Presented Updated Results from Phase 1b Monotherapy Trial of Rintodestrant in ER+, HER2- Breast Cancer at the 2020 SABCS: In a heavily pre-treated patient population, G1’s oral selective estrogen receptor degrader (SERD) rintodestrant showed evidence of clinical activity as monotherapy, including a clinical benefit rate of 30%. Safety and tolerability findings across all doses, including the 600 mg and 1,000 mg expansion cohorts, were consistent with previously reported data. These findings supported the Company’s decision to move the 800mg dose into the ongoing 40-patient Phase 2 combination trial with CDK4/6 inhibitor palbociclib. The data readout is expected in the second quarter of 2021. (Poster here)
Corporate

On February 23, 2021, the Board of Directors adopted the G1 Therapeutics, Inc. 2021 Inducement Equity Incentive Plan (the "Plan"). There are 500,000 shares of our common stock reserved under the Plan to be used exclusively for grants of awards to individuals that were not previously employees or directors of G1, as an inducement material to the individual’s entry into employment with G1 within the meaning of Rule 5635(c)(4) of the Nasdaq Listing Rules. The Plan was approved by Board of Directors without stockholder approval pursuant to Rule 5635(c)(4), and the terms and conditions of the Plan are substantially similar to G1’s stockholder-approved 2017 Equity Incentive Plan, as amended.
Fourth Quarter and Full Year 2020 Financial Results

As of December 31, 2020, cash and cash equivalents totaled $207.3 million, compared to $269.2 million as of December 31, 2019.

Subsequent to December 31, 2020, between January 14th, 2021 and February 9th, 2021, we sold 3,513,027 shares of common stock pursuant to our 2018 sales agreement for "at the market offerings" with Cowen and Company, LLC, resulting in $86.4 million in net proceeds. This ATM offering is now closed. In addition, the Company now has access to $30 million of the remaining $80M of our debt financing facility with Hercules Capital upon achievement of the FDA approval of COSELA milestone.

License revenue for the fourth quarter of 2020 was $16.5 million, primarily related to an upfront payment for our license agreement with Simcere recognized following the transfer of the related technology and know-how which occurred during the period. In addition, we recognized revenue for existing inventory transfers related to our license agreements with Genor and EQRx, as well as revenue for reimbursable clinical trial costs due from EQRx. License revenue for the full-year 2020 was $45.3 million.

Operating expenses for the fourth quarter of 2020 were $40.6 million, compared to $36.6 million for the fourth quarter of 2019. GAAP operating expenses include stock-based compensation expense of $4.8 million for the fourth quarter of 2020, compared to $4.5 million for the fourth quarter of 2019. Operating expenses for the full-year 2020 were $141.8 million, compared to $129.0 million for the prior year. Stock-based compensation expense for the full-year 2020 was $18.8 million, compared to $16.4 million for the prior year.

Research and development (R&D) expenses for the fourth quarter of 2020 were $16.4 million, compared to $24.5 million for the fourth quarter of 2019. The decrease in R&D expenses was primarily due to decreases in clinical program costs, external costs related to discovery and preclinical development, and costs for manufacturing pharmaceutical active ingredients. R&D expenses for the full-year 2020 were $73.3 million, compared to $89.0 million for the prior year.

General and administrative (G&A) expenses for the fourth quarter of 2020 were $24.3 million, compared to $12.1 million for the fourth quarter of 2019. The increase in G&A expenses was largely due to an increase in compensation due to increases in headcount, pre-commercialization activities, medical affairs costs, and professional fees and other administrative costs necessary to support our operations. G&A expenses for the full-year 2020 were $68.5 million, compared to $40.0 million for the prior year.

The net loss for the fourth quarter of 2020 was $25.3 million, compared to $35.4 million for the fourth quarter of 2019. Net loss for the full-year 2020 was $99.3 million, compared to a net loss of $122.4 million for the prior year. The basic and diluted net loss per share for the fourth quarter of 2020 was $(0.67) compared to $(0.94) for the fourth quarter of 2019. The basic and diluted net loss per share for the full-year 2020 was $(2.62) compared to $(3.27) for the full-year 2019.

Financial Guidance

The Company expects its current financial position to be sufficient to fund its operations and capital expenditures into 2023.

Webcast and Conference Call

G1 will host a webcast and conference call at 4:30 p.m. ET today to provide a corporate and financial update for the fourth quarter and full-year 2020 ended December 31, 2020. The live call may be accessed by dialing (866) 763-6020 (domestic) or (210) 874-7713 (international) and entering the conference code: 5267698. A live and archived webcast will be available on the Events & Presentations page of the company’s website: www.g1therapeutics.com. The webcast will be archived on the same page for 90 days following the event.

IMPORTANT SAFETY INFORMATION

CONTRAINDICATION

COSELA is contraindicated in patients with a history of serious hypersensitivity reactions to trilaciclib.
WARNINGS AND PRECAUTIONS

Injection-Site Reactions, Including Phlebitis and Thrombophlebitis

COSELA administration can cause injection-site reactions, including phlebitis and thrombophlebitis, which occurred in 56 (21%) of 272 patients receiving COSELA in clinical trials, including Grade 2 (10%) and Grade 3 (0.4%) adverse reactions. Monitor patients for signs and symptoms of injection-site reactions, including infusion-site pain and erythema during infusion. For mild (Grade 1) to moderate (Grade 2) injection-site reactions, flush line/cannula with at least 20 mL of sterile 0.9% Sodium Chloride Injection, USP or 5% Dextrose Injection, USP after end of infusion. For severe (Grade 3) or life-threatening (Grade 4) injection-site reactions, stop infusion and permanently discontinue COSELA. Injection-site reactions led to discontinuation of treatment in 3 (1%) of the 272 patients.
Acute Drug Hypersensitivity Reactions

COSELA administration can cause acute drug hypersensitivity reactions, which occurred in 16 (6%) of 272 patients receiving COSELA in clinical trials, including Grade 2 reactions (2%). Monitor patients for signs and symptoms of acute drug hypersensitivity reactions. For moderate (Grade 2) acute drug hypersensitivity reactions, stop infusion and hold COSELA until the adverse reaction recovers to Grade ≤1. For severe (Grade 3) or life-threatening (Grade 4) acute drug hypersensitivity reactions, stop infusion and permanently discontinue COSELA.
Interstitial Lung Disease/Pneumonitis

Severe, life-threatening, or fatal interstitial lung disease (ILD) and/or pneumonitis can occur in patients treated with cyclin-dependent kinases (CDK)4/6 inhibitors, including COSELA, with which it occurred in 1 (0.4%) of 272 patients receiving COSELA in clinical trials. Monitor patients for pulmonary symptoms of ILD/pneumonitis. For recurrent moderate (Grade 2) ILD/pneumonitis, and severe (Grade 3) or life-threatening (Grade 4) ILD/pneumonitis, permanently discontinue COSELA.
Embryo-Fetal Toxicity

Based on its mechanism of action, COSELA can cause fetal harm when administered to a pregnant woman. Females of reproductive potential should use an effective method of contraception during treatment with COSELA and for at least 3 weeks after the final dose.
ADVERSE REACTIONS

Serious adverse reactions occurred in 30% of patients receiving COSELA. Serious adverse reactions reported in >3% of patients who received COSELA included respiratory failure, hemorrhage, and thrombosis.

Fatal adverse reactions were observed in 5% of patients receiving COSELA. Fatal adverse reactions for patients receiving COSELA included pneumonia (2%), respiratory failure (2%), acute respiratory failure (<1%), hemoptysis (<1%), and cerebrovascular accident (<1%).

Permanent discontinuation due to an adverse reaction occurred in 9% of patients who received COSELA. Adverse reactions leading to permanent discontinuation of any study treatment for patients receiving COSELA included pneumonia (2%), asthenia (2%), injection-site reaction, thrombocytopenia, cerebrovascular accident, ischemic stroke, infusion-related reaction, respiratory failure, and myositis (<1% each).

Infusion interruptions due to an adverse reaction occurred in 4.1% of patients who received COSELA.

The most common adverse reactions (≥10%) were fatigue, hypocalcemia, hypokalemia, hypophosphatemia, aspartate aminotransferase increased, headache, and pneumonia.
DRUG INTERACTIONS

COSELA is an inhibitor of OCT2, MATE1, and MATE-2K. Co-administration of COSELA may increase the concentration or net accumulation of OCT2, MATE1, and MATE-2K substrates in the kidney (e.g., dofetilide, dalfampridine, and cisplatin).

On February 24, 2021 Xilio Therapeutics, a biotechnology company developing potent, tumor-selective immunotherapies for patients with cancer, reported the successful closing of a $95 million Series C financing (Press release, Xilio Therapeutics, FEB 24, 2021, View Source [SID1234575554]). Proceeds from the financing will be used to advance Xilio’s lead therapeutic candidates, XTX202 (tumor-selective IL-2) and XTX101 (tumor-selective anti-CTLA4 mAb), into clinical trials . Investigational new drug (IND) applications for XTX202 and XTX101 are expected to be filed with the U.S. Food and Drug Administration (FDA) in 2021.

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The financing was led by Rock Springs Capital and was joined by Bain Capital Life Sciences, Deerfield Management Company and RA Capital Management, among other new investors. Xilio’s existing investors, including Atlas Venture, SV Health Investors, Takeda Ventures, RiverVest Venture Partners, and MRL Ventures Fund, also participated in the financing. Concurrent with the closing, Dave Gardner of Rock Springs Capital and Andrew Hack, M.D., Ph.D, of Bain Capital Life Sciences will join the Xilio Therapeutics Board of Directors.

"The support from Rock Springs Capital and this world-class group of investors is a testament to our unique tumor-selective technology and the expertise of the team we have built, all of whom share our vision to transform the lives of people living with cancer," said Rene Russo, Pharm.D., chief executive officer of Xilio Therapeutics. "This financing provides us with additional capital to execute our clinical development plans for XTX202 and XTX101, as well as to advance our cytokine pipeline, including our tumor-selective IL-12, XTX301."

"Xilio Therapeutics has made remarkable progress in building its promising pipeline of cancer therapies designed to unlock previously unattainable therapeutic potential," said Dave Gardner of Rock Springs Capital. "We are proud to partner with this exceptional team and look forward to seeing the results of their clinical research."

Xilio’s promising candidates, XTX202 (tumor-selective IL-2) and XTX101 (tumor-selective anti-CTLA4 mAb), have demonstrated significant anti-tumor effects with minimal peripheral effects in preclinical models, indicating the potential to achieve substantially higher levels of efficacy compared with currently approved IL-2 and anti-CTLA4 therapies. Tumor selectivity holds the promise of deepening patient responses to these validated therapies, as well as substantially increasing the number of patients who could benefit from them.

"While high dose aldesleukin (rhIL-2) has the potential for durable complete responses, it is only given to a small number of patients due to its life-threatening toxicity. Similarly, approved aCTLA4 therapy produces severe autoimmune toxicity, limiting potential benefit by preventing patients from receiving highly therapeutic doses or completing full courses of treatment," said Martin Huber, M.D., chief medical officer of Xilio Therapeutics. "Xilio’s proprietary technology allows us to mask the therapeutic (IL-2 or aCTLA4) until it is activated in the tumor micro-environment, enabling high dose drug delivery into the tumor to maximize the potential for efficacy."

On February 24, 2021 Daiichi Sankyo Company, Limited (hereafter, Daiichi Sankyo) and LYSA-LYSARC-CALYM reported that they have entered a strategic research collaboration to study valemetostat (DS-3201), Daiichi Sankyo’s potential first-in-class EZH1/2 dual inhibitor, in B-cell malignancies starting with a phase 2 study in patients with five subtypes of relapsed/refractory B-cell lymphoma (Press release, Daiichi Sankyo, FEB 24, 2021, https://www.businesswire.com/news/home/20210223006211/en/Daiichi-Sankyo-and-LYSA-LYSARC-CALYM-Enter-Research-Collaboration-for-Valemetostat-in-Patients-with-RelapsedRefractory-B-Cell-Lymphoma [SID1234575573]).

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The collaboration brings together Daiichi Sankyo’s innovative science and the multidisciplinary expertise of the Lymphoma Study Association (LYSA), the Lymphoma Academic Research Organization (LYSARC) and the CALYM research consortium to conduct clinical and translational research that will build upon the ongoing phase 1 study of valemetostat in patients with relapsed/refractory non-Hodgkin lymphoma.1

Lymphoma is a heterogenous disease with more than 90 different subtypes and while new treatment advances have improved outcomes for some patients, management of relapsed/refractory B-cell lymphoma remains a major challenge.2 There are currently no dual EZH1/2 directed therapies approved for cancer treatment.

"We are pleased to join forces with Europe’s largest lymphoma research organization to advance and strengthen the development of valemetostat as a potential novel precision medicine for patients with relapsed/refractory B-cell lymphoma," said Arnaud Lesegretain, Vice President, Global Oncology Development, Alpha Portfolio, Daiichi Sankyo. "LYSA-LYSARC-CALYM together with Daiichi Sankyo have designed a phase 2 study that will enroll patients based on disease subtype and biomarkers in order to further evaluate safety and efficacy, and we are planning a comprehensive translational research program to answer important scientific questions relating to clinical utility, optimal patient selection and mechanisms of resistance."

"We are very happy to engage our cooperative group in a collaboration with Daiichi Sankyo for this promising development program with valemetostat targeting relevant epigenetic factors EZH2 and EZH1 in B-cell lymphomas," said Franck Morschhauser, Professor of Hematology in Lille, France and President of LYSA-LYSARC. "We believe that our extensive multidisciplinary and international expertise will help advance the science behind the novel mechanism of action and we look forward to playing a role in bringing this potential new medicine to patients with lymphoma."

About the Collaboration

Under the agreement, LYSA-LYSARC will execute a multi-center, non-randomized, open-label phase 2 study to evaluate the safety and efficacy of valemetostat in six cohorts of patients with relapsed/refractory B-cell lymphoma.

The study will enroll patients with diffuse large B-cell lymphoma (with and without an EZH2 mutation) that has progressed on at least one prior treatment; follicular lymphoma (EZH2 mutant and EZH2 wild-type), mantle cell lymphoma, and marginal zone lymphoma/other indolent lymphomas that have progressed on two or more prior treatments; and Hodgkin lymphoma that has progressed on three or more prior treatments including checkpoint inhibitors.

The primary endpoint of the study is best overall response rate determined by investigator assessment. Secondary endpoints include complete response rate, progression-free survival, duration of response, time to response and safety measures including adverse events. Exploratory endpoints include overall survival and measures of biomarker expression and treatment response. Pharmacokinetic endpoints will also be assessed.

The study will include approximately 140 patients at 22 sites in France and Belgium and is anticipated to initiate in 2021.

About EZH1 and EZH2

EZH1 (enhancer of zeste homolog 1) and EZH2 (enhancer of zeste homolog 2) enzymes are part of polycomb protein complexes that act through histone methylation to regulate gene expression.3 EZH1 and EZH2 are recurrently highly expressed or mutated in many hematologic malignancies.4 Epigenetic dysregulation of the methylation process is associated with suppression of genes that regulate cancer cell growth and proliferation.4 Research shows that both EZH1 and EZH2 have a role in hematologic cancer progression and that simultaneous inhibition would be effective in targeting the cancers.5 There are no dual EZH1/2 directed therapies approved for treatment of cancer.

About Valemetostat

Valemetostat (DS-3201) is a potential first-in-class small molecule oral EZH1/2 dual inhibitor currently in clinical development in the Alpha portfolio of Daiichi Sankyo for several hematologic cancers. Valemetostat targets epigenetic regulation by inhibiting both the EZH1 and EZH2 enzymes. Valemetostat has displayed antitumor activity in various hematological malignancies in preclinical models.5,6

The development program of valemetostat includes a pivotal phase 2 trial in patients with relapsed or refractory adult T-cell leukemia/lymphoma (ATL) in Japan; a phase 1 study in patients with several types of non-Hodgkin lymphomas (NHL) including B-cell lymphoma, adult T-cell leukemia-lymphoma (ATL) and peripheral T-cell lymphoma (PTCL) in the U.S. and Japan; and a phase 1 study in patients with acute lymphocytic leukemia (ALL) and acute myeloid leukemia (AML) in the U.S.

In April 2019, valemetostat received SAKIGAKE Designation for the treatment of adult patients with relapsed or refractory PTCL by the Japan Ministry of Health, Labour and Welfare (MHLW).

Valemetostat is an investigational agent that has not been approved for any indication in any country. Safety and efficacy have not been established.

About B-Cell Lymphoma

Lymphoma is the most common type of blood cancer.7 There were more than 627,000 new cases of lymphoma diagnosed globally and more than 283,000 deaths from the disease in 2020.8

There are more than 90 different lymphoma subtypes, which occur in varying frequencies in different geographic regions around the world.7 Most lymphomas originate in B-cell lymphocytes, with the most common types being diffuse large B-cell lymphoma (about 33 percent), follicular lymphoma (about 20 percent), mantle cell lymphoma (about 5 percent) and marginal zone lymphoma (5 to 10 percent).9 Treatment recommendations and prognosis vary for different subtypes of B-cell lymphoma.10 New treatment advances have improved outcomes for some patients with certain types of B-cell lymphoma, but management of relapsed or refractory lymphoma remains a major challenge and new and novel treatments are needed.2

On February 24, 2021 Vincerx Pharma, Inc. (Nasdaq: VINC), a biopharmaceutical company aspiring to address the unmet medical needs of patients with cancer through paradigm-shifting therapeutics, reported that it will be hosting a key opinion leader (KOL) meeting on VIP152 for the treatment of hematologic and solid tumors on Friday, March 5, 2021 at 12:00pm Eastern Time (Press release, Vincerx Pharma, FEB 24, 2021, View Source [SID1234575523]).

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The event will feature presentations by KOLs Ian Flinn, M.D., Ph.D., Director of Sarah Cannon Center for Blood Cancer, John Byrd, M.D., D. Warren Brown Chair of Leukemia Research, The Ohio State University Comprehensive Cancer Center, and Howard "Skip" Burris III, M.D., President and Chief Medical Officer of Sarah Cannon, who will discuss the CDK9 inhibitor landscape in hematologic and solid tumors. Drs. Flinn, Byrd, and Burris will be available to answer questions following the formal presentations.

Vincerx’s management team will also discuss the development plan for their lead product VIP152, a potent and highly selective CDK9 inhibitor optimized for intermittent intravenous treatment. VIP152’s differentiated profile for selectivity, potency, and durability has translated to early signals of clinical activity in Phase 1, notably in patient populations with high unmet medical needs including double-hit DLBCL. In addition, VIP152 has demonstrated on-target disruption of PTEFb function with reductions in kinase activity and mRNA levels of key oncogenes including MYC and MCL-1.

KOLs
Ian Flinn, M.D., Ph.D. is an internationally recognized clinical investigator whose research focuses on the development of new therapies for patients with lymphoma and chronic lymphocytic leukemia. This research includes first in human to phase 3 trials with novel approaches such as immune effector cell therapies, inhibitors of the B cell receptor pathway, and BCL-2 inhibitors amongst others. He is an author of approximately 200 articles in journals such as the New England Journal of Medicine, Lancet, Journal of Clinical Oncology and Blood. Dr Flinn joined Tennessee Oncology and Sarah Cannon in 2006 and serves as Director of Lymphoma Research. In his role, he oversees lymphoma research throughout Sarah Cannon and its affiliates. Dr. Flinn also serves as the director for the Sarah Cannon Center for Blood Cancer at Tennessee Oncology.

John C. Byrd, M.D., is an internationally known researcher and clinical specialist in leukemia and other hematologic malignancies at Ohio State University’s Comprehensive Cancer Center – James Cancer Hospital and Solove Research Institute, where he serves as Senior Advisor for Cancer Experimental Therapeutics. He is a Distinguished University Professor of Medicine and Medicinal Chemistry and holds the D. Warren Brown Chair in Leukemia Research. Dr. Byrd is also the Chief Medical Officer for BEAT AML. Dr. Byrd received his medical degree from the University of Arkansas for Medical Sciences. His education continued in hematology and oncology at Walter Reed Army Medical Center and Johns Hopkins University before moving to Columbus to join the faculty at Ohio State University. Dr. Byrd has over 575 publications in leukemia and experimental therapeutics research. He runs a highly translational laboratory focused on drug development in chronic lymphocytic leukemia and related lymphoproliferative disorders. He has been part of the successful development of multiple therapeutics in acute myeloid leukemia and chronic lymphocytic leukemia.

Howard A. "Skip" Burris III, M.D., FASCO, FACP serves as President and Chief Medical Officer of Sarah Cannon, as well as the Executive Director, Drug Development for Sarah Cannon Research Institute. He is an associate of Tennessee Oncology, PLLC, where he practices medical oncology. Dr. Burris’ clinical research career has focused on the development of new cancer agents with an emphasis on first-in-human therapies, having led the trials of many novel antibodies, small molecules, and chemotherapies now FDA approved. In 1997, he established in Nashville the first community-based early phase drug development program, which grew into the Sarah Cannon Research Institute. He has authored over 400 publications and 700 abstracts. Dr. Burris served as President of ASCO (Free ASCO Whitepaper) in 2019-2020 and is serving as Chair of the Board for the 2020-2021 term. He also currently serves on the Board of ASCO (Free ASCO Whitepaper)’s Conquer Cancer Foundation. Additionally, in 2014, Dr. Burris was selected by his peers as a Giant of Cancer Care for his achievements in drug development. Dr. Burris completed his undergraduate education at the United States Military Academy at West Point, his medical degree at the University of South Alabama, and his internal medicine residency and oncology fellowship at Brooke Army Medical Center in San Antonio. While in Texas, he also served as the Director of Clinical Research at The Institute for Drug Development of the Cancer Therapy and Research Center and The University of Texas Health Science Center. He attained the rank of lieutenant colonel in the US Army, and among his decorations, he was awarded a Meritorious Service Medal with oak leaf cluster for his service in Operation Joint Endeavor.

On February 24, 2021 Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, reported fourth quarter and full year 2020 financial results and recent business highlights (Press release, Sangamo Therapeutics, FEB 24, 2021, View Source [SID1234575539]).

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"We are very pleased with our execution and the progress we made in the challenging year of 2020 during the pandemic, where we advanced our hemophilia A product candidate into a Phase 3 clinical trial with Pfizer, dosed patients in our Phase 1/2 clinical study evaluating our wholly owned product candidate treating Fabry disease, entered into transformational neuroscience collaborations with Biogen and Novartis, brought in-house AAV manufacturing online in our Brisbane headquarters, and built a strong cash position," said Sandy Macrae, Chief Executive Officer of Sangamo. "In 2021, we will continue to build on our momentum with a focus on clinical execution and bringing our in-house cell therapy manufacturing facilities online."

Fourth Quarter Updates and Recent Business Highlights

Presented with collaborator Pfizer updated follow-up data from the Phase 1/2 Alta study of giroctocogene fitelparvovec, a gene therapy product candidate to treat hemophilia A, which was generally well-tolerated and demonstrated sustained Factor VIII activity levels in the therapeutic range through one year for the five patients in the highest dose cohort. The data were presented at the 62nd American Society for Hematology Annual Meeting.
Announced with Pfizer that the first participant has been dosed in the registrational Phase 3 AFFINE trial of giroctocogene fitelparvovec.
Completed dosing in February 2021 of the third patient in the Phase 1/2 STAAR study evaluating ST-920 gene therapy for Fabry disease. This is the first patient in the second dose cohort.
Brought in-house AAV manufacturing capabilities online in Sangamo’s Brisbane, California headquarters at the end of 2020.
Announced global collaboration with Novartis to develop and commercialize gene regulation therapies to treat three neurodevelopmental targets, including genes linked to autism spectrum disorder and intellectual disability.
Announced global collaboration with Biogen to develop and commercialize gene regulation therapies for Alzheimer’s, Parkinson’s, neuromuscular and other neurological diseases.
Fourth Quarter and Full Year 2020 Financial Results

Consolidated net loss for the fourth quarter ended December 31, 2020 was $40.7 million, or $0.29 per share, compared to net income of $4.5 million, or $0.04 per share, for the same period in 2019. For the year ended December 31, 2020, consolidated net loss was $121.1 million, or $0.90 per share, compared to consolidated net loss of $95.4 million, or $0.85 per share, for the year ended December 31, 2019.

Revenues

Revenues for the fourth quarter ended December 31, 2020 were $25.8 million, compared to $54.9 million for the same period in 2019. The decrease in revenue was due primarily to milestones achieved under our collaboration agreements in the fourth quarter of 2019, which included $25.0 million from Pfizer for the completion of the investigational new drug, or IND, transfer for giroctocogene fitelparvovec and $7.5 million from Sanofi for dosing of the first patient in our Phase 1/2 clinical study evaluating our BIVV003 product candidate to treat sickle cell disease.

Revenues were $118.2 million in 2020, compared to $102.4 million in 2019. The increase in revenues was primarily due to recognition of upfront license fees under the Biogen and Novartis collaboration agreements entered into in 2020. This increase was partially offset by a decrease in revenues from our giroctocogene fitelparvovec collaboration agreement with Pfizer following the IND transfer in December 2019.

Operating expenses

Three Months Ended December 31,

Year Ended December 31,

(In millions)
2020

2019

2020

2019

Research and development
$

52.4

$

38.3

$

180.6

$

145.9

General and administrative

16.8

15.1

67.1

61.7

Total operating expenses

69.2

53.4

247.7

207.6

Stock-based compensation expense

(6.6

)

(5.2

)

(25.7

)

(19.3

)

Non-GAAP operating expenses
$

62.6

$

48.2

$

222.0

$

188.3

Total operating expenses for the fourth quarter ended December 31, 2020 were $69.2 million compared to $53.4 million for the same period in 2019. Stock-based compensation expense for the fourth quarter ended December 31, 2020 was $6.6 million, compared to $5.2 million for the same period in 2019. Non-GAAP operating expenses, which exclude stock-based compensation expense, for the fourth quarter ended December 31, 2020 were $62.6 million, compared to $48.2 million for the same period in 2019.

Total operating expenses in 2020 were $247.7 million, compared to $207.6 million in 2019. Stock-based compensation expense in 2020 was $25.7 million, compared to $19.3 million in 2019. Non-GAAP total operating expenses, which exclude excluding stock-based compensation expense, were $222.0 million and $188.3 million in 2020 and 2019, respectively.

The increase in operating expenses in the full year and fourth quarter was due primarily to headcount growth and facilities expansion to support the advancement of our clinical trials and manufacturing capabilities. The full year and fourth quarter increase was partially offset by a decrease in travel and corporate costs arising from the COVID-19 pandemic.

Cash, cash equivalents and marketable securities

Cash, cash equivalents and marketable securities as of December 31, 2020 were $692.0 million, compared to $384.3 million as of December 31, 2019. The balance as of December 31, 2020 includes a $30.0 million milestone from Pfizer for the initiation of the AFFINE trial for giroctocogene fitelparvovec and a $5.0 million milestone from Pfizer for our C9ORF72 collaboration with Pfizer.

In August 2020, we entered into an Open Market Sale Agreement with Jefferies LLC providing for the sale of up to $150.0 million of our common stock from time to time in ‘at-the-market’ offerings under our shelf registration statement. Through February 19, 2021, we sold 1,034,762 shares of our common stock pursuant to this agreement for net proceeds of approximately $15.7 million.

Initial Financial Guidance for 2021

On a GAAP basis, we expect total operating expenses in the range of approximately $285 million to $305 million in 2021, which includes non-cash stock-based compensation expense.

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

Conference Call

Sangamo will host a conference call today, February 24, 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 1795427. 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 1795427.