On May 15, 2021 Xencor, Inc. (NASDAQ:XNCR), a clinical-stage biopharmaceutical company developing engineered monoclonal antibodies and cytokines for the treatment of cancer and autoimmune diseases, reported financial results for the first quarter ended March 31, 2021 and provided a review of recent business and clinical highlights (Press release, Xencor, MAY 5, 2021, View Source [SID1234579185]).

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"We continue to expand and mature our clinical portfolio of XmAb drug candidates, recently initiating a Phase 1 study for our second cytokine program, XmAb564, a wholly owned IL-2-Fc fusion protein, in healthy volunteers. We engineered this molecule to preferentially activate regulatory T cells, an emerging mechanism for treating patients with autoimmune diseases," said Bassil Dahiyat, Ph.D., president and chief executive officer at Xencor. "Additionally, at the AACR (Free AACR Whitepaper) meeting, we presented preclinical data from multiple early-stage programs that highlight our protein engineering expertise with our third cytokine, a wholly owned IL-12-Fc fusion protein, as well as the potential of our CD28 platform and XmAb 2+1 bispecific antibody format. Looking ahead, we will continue to present maturing data from our clinical-stage programs, and we have plans to initiate several additional clinical studies this year and early 2022, including a Phase 2 study in prostate cancer with XmAb717, our PD-1 x CTLA-4 bispecific antibody."

Recent Business and Portfolio Highlights

XmAb564 (IL-2-Fc Cytokine): XmAb564 is a wholly owned, monovalent IL-2-Fc fusion protein, engineered to selectively activate and expand regulatory T cells (Tregs) for the potential treatment of patients with autoimmune diseases. XmAb564 is engineered with reduced binding affinity for IL-2’s beta receptor and increased binding affinity for its alpha receptor. In preclinical studies, XmAb564 was well-tolerated, promoted the selective and sustained expansion of Tregs and exhibited a favorable pharmacokinetic profile. In April 2021, the first subject was dosed in a randomized, double-blind, placebo-controlled Phase 1 clinical study evaluating the safety and tolerability of XmAb564, administered subcutaneously in healthy adult volunteers.
Preclinical Presentations at AACR (Free AACR Whitepaper): At the 2021 AACR (Free AACR Whitepaper) Annual Meeting, the Company presented four posters highlighting several preclinical-stage programs, including its IL-12-Fc cytokine program, two XmAb 2+1 bispecific antibodies (Claudin-6 x CD3 and GPC3 x CD3), and a PD-L1 x CD28 bispecific program. Such targeted CD28 bispecific antibodies, a new class of T cell engager, may provide conditional co-stimulation of T cells, for example, to T cells recognizing neoantigens or in concert with CD3 T-cell engaging bispecific antibodies. The Company is also advancing through preclinical development a wholly owned lead CD28 candidate, a B7-H3 x CD28 bispecific antibody, which will be evaluated for the treatment of patients with a range of solid tumors.
New Academic Collaboration with UCLA: In February, the Company entered an agreement with UCLA to develop novel therapeutic antibodies, pairing novel targets proposed by scientists at UCLA and Xencor’s modular suite of XmAb technology platforms. The UCLA Technology Development Group will work with faculty to propose potential antibody drug candidates. For selected candidates, the Company and UCLA expect to use a framework with predefined terms to enter sponsored research agreements and potential license agreements.
Multiple Clinical Studies Planned to Advance Xencor’s Wholly Owned Programs

XmAb717 (PD-1 x CTLA-4): The Company plans to initiate a Phase 2 study in patients with certain molecular subtypes of castration-resistant prostate cancer (CRPC) in mid-2021. This study will evaluate XmAb717 as a monotherapy or in combination depending on the subtype, as these patients represent a high unmet medical need.
Tidutamab (SSTR2 x CD3): The Company plans to initiate a clinical study in patients with Merkel cell carcinoma and small cell lung cancer, SSTR2-expressing tumor types known to be responsive to immunotherapy, in mid-2021.
Plamotamab (CD20 x CD3): In November 2020, the Company entered a strategic clinical collaboration with MorphoSys AG to investigate the chemotherapy-free triple combination of plamotamab, tafasitamab and lenalidomide in patients with relapsed or refractory (r/r) diffuse large B cell lymphoma (DLBCL), first-line DLBCL and r/r follicular lymphoma (FL). The Company plans to initiate the first of these studies, in patients with r/r DLBCL, an aggressive type of non-Hodgkin lymphoma (NHL), in late 2021 or early 2022.
XmAb698 (CD38 x CD3): The Company plans to support investigator-initiated studies of XmAb698 (formerly AMG 424), and a new study is currently being planned to start later in 2021.
XmAb819 (ENPP3 x CD3): XmAb819 is engineered with the multi-valent XmAb 2+1 bispecific antibody format to enable greater tumor selectivity, and it is in development for patients with renal cell carcinoma. The Company plans to submit an investigational new drug (IND) application in 2021 and initiate a Phase 1 study in early 2022.
Progress Across Partnered Programs

MorphoSys AG: In April 2021, MorphoSys initiated the Phase 3 inMIND study to evaluate the addition of tafasitamab to lenalidomide and rituximab in patients with r/r follicular lymphoma or marginal zone lymphoma. Xencor earned $12.5 million for the development milestone and recognized royalty revenue of $1.4 million on net sales of Monjuvi during the first quarter of 2021.
Vir Biotechnology, Inc.: Vir and its partner GlaxoSmithKline plc (GSK) are evaluating VIR-7831 in an extensive ongoing clinical development program. In March 2021, Vir and GSK submitted an emergency use authorization (EUA) application to the U.S. Food and Drug Administration based on an interim analysis of the Phase 3 COMET-ICE (COVID-19 Monoclonal antibody Efficacy Trial – Intent to Care Early) trial, which demonstrated an 85% reduction in hospitalization or death in high-risk adult outpatients with COVID-19 receiving VIR-7831 as monotherapy compared to placebo, the primary endpoint of the trial.
Monjuvi is a registered trademark of MorphoSys AG.

First Quarter Ended March 31, 2021 Financial Results

Cash, cash equivalents and marketable investment securities totaled $577.1 million at March 31, 2021, compared to $604.0 million at December 31, 2020. The decrease reflects royalties, milestone payments and equity received related to licensing agreements, net of cash used to fund operating activities in the first quarter of 2021.

Total revenue for the first quarter ended March 31, 2021 was $34.0 million, compared to $32.4 million for the same period in 2020. Revenues in the first quarter of 2021 included revenues related to the Janssen collaboration, milestone revenue recognized from MorphoSys and the royalty revenue from Alexion and MorphoSys, compared to revenues from the same period in 2020, which were primarily revenue recognized from MorphoSys, royalty revenue from Alexion, and licensing revenue from Aimmune and Gilead.

Research and development expenditures for the first quarter ended March 31, 2021 were $41.4 million, compared to $33.9 million for the same period in 2020. Additional spending on research and development expenses for the first quarter of 2021 was primarily due to increased spending on XmAb306, XmAb564 and XmAb819 programs.

General and administrative expenses for the first quarter ended March 31, 2021 were $8.2 million, compared to $7.2 million in the same period in 2020. Additional spending on general and administrative expenses for the first quarter of 2021 reflects increased spending related to staffing.

Other income for the first quarter ended March 31, 2021 was $13.2 million and included a gain of $12.9 million from equity related to a licensing transaction, compared to $0.7 million for the same period in 2020, which was primarily net interest income earned for the period.

Non-cash, stock-based compensation expense for the first quarter ended March 31, 2021 was $8.3 million, compared to $6.5 million for same period in 2020.

Net loss for the first quarter ended March 31, 2021 was $2.5 million, or $(0.04) on a fully diluted per share basis, compared to net loss of $8.1 million, or $(0.14) on a fully diluted per share basis, for the same period in 2020. The lower net loss reported for first quarter of 2021 compared to the net loss for the same period in 2020 is primarily due to other income recognized related to equity received in the first quarter of 2021 in excess of increased spending on research and development.

The total shares outstanding were 58,221,953 as of March 31, 2021, compared to 57,001,253 as of March 31, 2020.

Financial Guidance

Based on current operating plans, Xencor expects to have cash to fund research and development programs and operations into 2024. Xencor expects to end 2021 with between $425 million and $475 million in cash, cash equivalents and marketable securities.

Conference Call and Webcast

Xencor will host a conference call today at 4:30 p.m. ET (1:30 p.m. PT) to discuss these first quarter 2021 financial results and provide a corporate update.

The live call may be accessed by dialing (877) 359-9508 for domestic callers or +1 (224) 357-2393 for international callers and referencing conference ID number 2378094. A live webcast of the conference call will be available online from the Investors section of Xencor’s website at www.xencor.com. The webcast will be archived on Xencor’s website for 30 days.

On May 5, 2021 Personalis, Inc. (Nasdaq: PSNL), a leader in advanced genomics for population sequencing and cancer, reported financial results for the first quarter ended March 31, 2021 (Press release, Personalis, MAY 5, 2021, View Source [SID1234579184]).

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First Quarter Highlights

Record quarterly revenue of $20.9 million in the first quarter of 2021 compared with $19.2 million in the first quarter of 2020, a 9% increase
Revenue of $7.7 million from biopharma and all other customers, excluding VA MVP, in the first quarter of 2021 compared with $4.4 million in the first quarter of 2020, a 74% increase
Announced a collaboration with Natera in the field of personalized oncology; Personalis to provide the exome sequence data for Natera to pair with their personalized ctDNA platform, SignateraTM
Announced a collaboration with MapKure, LLC, a company jointly owned by BeiGene, Ltd. and SpringWorks Therapeutics, Inc., LLC for use of the NeXT PlatformTM for clinical trials and companion diagnostic development
Added approximately $162 million of cash to balance sheet from public offering of common stock financing in the first quarter of 2021; ended the quarter with cash, cash equivalents, and short-term investments of $353.4 million as of March 31, 2021
"I’m proud to say that we were able to report record revenue once again this quarter and achieved our nineteenth consecutive quarter of growth, as we converted an increasing number of orders into revenue. Biopharma and all other customer revenue grew 74% year-over-year, and orders from customers exceeded revenues reported once again this quarter," said John West, Chief Executive Officer. "Recently, we announced a partnership with Natera in the field of personalized oncology, and a companion diagnostics collaboration with MapKure. Both collaborations further validate our NeXT platform as a leading front end tissue-sequencing platform, capable of detecting cancer cell mutations that conventional exome tests often miss. Our tissue offering complements our whole exome liquid biopsy product that we launched in August 2020 and NeXT Personal, our Minimal Residual Disease (MRD) offering that we expect to launch in 2021, providing Personalis with access to three distinct revenue opportunities in the rapidly growing cancer monitoring market."

First Quarter 2021 Financial Results

Revenues were $20.9 million in the three months ended March 31, 2021, up 9% from $19.2 million in the same period of the prior year.

Gross margin was 35.6% in the three months ended March 31, 2021, compared with 21.1% in the same period of the prior year.

Operating expenses were $19.9 million in the three months ended March 31, 2021, compared with $13.7 million in the same period of the prior year.

Net loss was $12.4 million in the three months ended March 31, 2021 and net loss per share was $0.29 based on a weighted-average basic and diluted share count of 42.3 million, compared with a net loss of $9.1 million and a net loss per share of $0.29 on a weighted-average basic and diluted share count of 31.3 million in the same period of the prior year.

Business Outlook

Personalis expects the following for the second quarter of 2021:

Total Company revenues to be approximately $21.3 million
Revenues from biopharma and all other customers, excluding VA MVP, to be in the range of $7.3 million to $7.7 million
Net Loss to be in the range of $16 million to $17 million; estimated outstanding shares of 43 million
Personalis expects the following for the full year of 2021:

Total Company revenues to be approximately $85 million
Revenues from biopharma and all other customers, excluding VA MVP, to be in the range of $30 million to $32 million
Net Loss to be in the range of $70 million to $75 million; estimated outstanding shares of 44 million
Webcast and Conference Call Information

Personalis will host a conference call to discuss the first quarter 2021 financial results after market close on Wednesday, May 5, 2021 at 2:00 p.m. Pacific Time / 5:00 p.m. Eastern Time. The conference call can be accessed live over the phone by dialing (866) 220-8061 for U.S. callers or (470) 495-9168 for international callers, using the conference ID: 6587766. The live webinar can be accessed at View Source

On May 5, 2021 Neurocrine Biosciences, Inc. (Nasdaq: NBIX) reported its financial results for the first quarter ended March 31, 2021 and provided revised full-year 2021 financial expense guidance (Press release, Neurocrine Biosciences, MAY 5, 2021, View Source [SID1234579183]).

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"Our first quarter results reflect a lower than normal refill rate per patient due to the typical seasonal payor dynamics for INGREZZA that were exacerbated by COVID. Importantly, we did not see an increase in discontinuations and exited the quarter with more patients on INGREZZA versus the prior quarter. New patient starts did pick up late in the quarter and we are focused on restoring INGREZZA growth as the impact of the pandemic wanes," said Kevin Gorman, Ph.D., Chief Executive Officer of Neurocrine Biosciences. "We made meaningful progress advancing our large, growing and diverse pipeline, including initiating a registrational study for the treatment of pediatric patients with congenital adrenal hyperplasia and a Phase II study in essential tremor. With important commercial products addressing patient needs and on track to initiate nine mid-to-late stage clinical studies this year, we are executing our plan to build a leading neuroscience-based company."

First Quarter Net Product Sales and Commercial Highlights:

INGREZZA net product sales for the first quarter of 2021 were $230 million and $227 million on an inventory adjusted basis
Annual seasonal payor dynamics resulted in lower refill rates per existing patient as compared to historical norms due to the COVID-19 pandemic and related disruption on timing of patient insurance reauthorizations
Exiting the first quarter, commercial activity and weekly new prescriptions both achieved their highest levels since the start of the pandemic reflecting an improved environment for diagnosis of tardive dyskinesia (TD). We expect second quarter inventory adjusted sales sequential dollar growth to be similar to 2020.
INGREZZA direct-to-consumer advertising campaign, "TD Spotlight", to be launched in May to educate patients about tardive dyskinesia and the benefits of INGREZZA
Financial Highlights:

First quarter 2021 GAAP net income and diluted earnings per share were approximately $32 million and $0.33, respectively, compared with approximately $37 million and $0.39, respectively, in the first quarter of 2020
First quarter 2021 non-GAAP net income and diluted earnings per share were approximately $48 million and $0.49, respectively, compared with approximately $79 million and $0.82, respectively, in the first quarter of 2020
Difference between first quarter 2021 GAAP and non-GAAP net income and diluted earnings per share vs. the first quarter of 2020 were driven by:
Increased Research and Development (R&D) expense primarily due to increased investment across our expanded pipeline programs and higher headcount costs
Increased Selling, General and Administrative (SG&A) expense primarily due to increased investment in commercial initiatives
At March 31, 2021, the Company had cash, cash equivalents and debt securities available-for-sale of $1.1 billion
A reconciliation of GAAP to non-GAAP financial results can be found in Table 3 and Table 4 at the end of this earnings release.

Provision for Income Taxes:

First quarter 2021 benefit from income taxes was $4.9 million, compared with a provision for income taxes of $1.5 million in the first quarter of 2020. Our effective tax rate for the first quarter of 2021 was lower than federal and state statutory rates primarily due to excess tax benefits related to stock compensation. On December 31, 2020, the Company released substantially all of its valuation allowance against its net operating losses and other deferred tax assets. Beginning in the first quarter of 2021, the Company began recording a provision for income taxes using an effective tax rate approximating federal and state statutory rates. Due to our ability to offset our pre-tax income against previously benefited federal net operating losses, no federal cash tax is expected in 2021.

Recent Events

In February 2021, the Mitsubishi Tanabe Pharma Corporation (MTPC) reported positive top-line results from the J-KINECT Phase III Study, designed to evaluate the efficacy and safety of valbenazine in tardive dyskinesia. Detailed results from this trial will be presented at a future medical conference. In April 2021, MTPC submitted a Marketing Authorization Application (MAA) with the Ministry of Health and Welfare in Japan for valbenazine for the treatment of tardive dyskinesia. MTPC submission of valbenazine triggered a milestone payment of $15 million, to be paid by MTPC to Neurocrine Biosciences and recognized as collaboration revenue in the second quarter of 2021.
In February 2021, the Company notified Voyager Therapeutics, Inc. (Voyager) of the Company’s termination of the NBIb-1817 (VY-AADC) development program in Parkinson’s disease (the Program). The Company will work to transfer the rights to the Program to Voyager by August 2, 2021.
On March 2, 2021, the Company announced that investigational drug luvadaxistat (NBI-1065844/TAK-831) did not meet its primary endpoint in the Phase II INTERACT study in adults with negative symptoms of schizophrenia. Luvadaxistat met both secondary endpoints of cognitive assessment. The Company plans to initiate a Phase II study for the treatment of cognitive impairment associated with schizophrenia (CIAS) by the end of 2021.
In April 2021, the U.S. Food and Drug Administration (FDA) approved a 60 mg INGREZZA capsule for the treatment of adults with tardive dyskinesia. The 60 mg capsule of INGREZZA is expected to be available to patients by late second quarter 2021.
Previously, the Company expected combined GAAP R&D and SG&A expenses in the range of $800 million to $850 million and combined non-GAAP R&D and SG&A expenses in the range of $675 million to $725 million
Increase to GAAP and Non-GAAP expense guidance range primarily driven by investment in INGREZZA direct-to-consumer marketing campaign, "TD Spotlight"
GAAP-only guidance includes approximately $130 million of share-based compensation. GAAP-only guidance does not include any potential milestones or in-process research and development costs associated with current collaborations or future business development activities.
Conference Call and Webcast Today at 4:30 PM Eastern Time
Neurocrine Biosciences will hold a live conference call and webcast today at 4:30 p.m. Eastern Time (1:30 p.m. Pacific Time). Participants can access the live conference call by dialing 800-895-3361 (US) or 785-424-1062 (International) using the conference ID: NBIX. The webcast can also be accessed on Neurocrine Biosciences’ website under Investors at www.neurocrine.com. A replay of the webcast will be available on the website approximately one hour after the conclusion of the event and will be archived for approximately one month.

(Press release, Neurocrine Biosciences, MAY 5, 2021, View Source [SID1234579183])

On May 5, 2021 Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for patients with cancer, reported business highlights and financial results for the first quarter ended March 31, 2021 (Press release, Fate Therapeutics, MAY 5, 2021, View Source [SID1234579182]).

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"During the first quarter of 2021, we strengthened our balance sheet by raising $460 million and successfully positioned our off-the-shelf, iPSC-derived NK cell pipeline to achieve significant clinical milestones across our disease franchises throughout the remainder of the year. We look forward to sharing Phase 1 clinical data from our FT516 and FT538 programs in relapsed / refractory AML at an investor event to be held alongside the ASGCT (Free ASGCT Whitepaper) conference. We are also pleased with the clinical expansion of our FT538 program into solid tumors, where we plan to combine with FDA-approved monoclonal antibodies targeting EGFR, HER2, and PDL1," said Scott Wolchko, President and Chief Executive Officer of Fate Therapeutics. "While we are disappointed that the PROTECT study of ProTmune did not meet its primary endpoint for prevention of acute graft-versus-host disease following allogeneic stem cell transplant, we will now turn our full attention and resources to our deep pipeline of off-the-shelf, iPSC-derived cancer immunotherapies. We would like to sincerely thank the patients, caregivers and investigators who participated in the clinical investigation of ProTmune, and we intend to share our clinical findings with that community."

AML Disease Franchise

Interim Phase 1 Clinical Data for FT516 and FT538 Programs to be Featured at Upcoming Investor Event. On May 13, the Company plans to host a virtual investor event to highlight interim Phase 1 clinical data from its FT516 (hnCD16 engineered iPSC-derived NK cell) and FT538 (hnCD16 + IL-15RF + CD38KO engineered iPSC-derived NK cell) programs for the treatment of relapsed / refractory acute myeloid leukemia (AML). The Phase 1 study of FT516 as a monotherapy (NCT04023071) has enrolled the first and second dose cohorts (90 million and 300 million cells per dose, respectively), and dose escalation is ongoing with enrollment in the third dose cohort (900 million cells per dose). The Phase 1 study of FT538 as a monotherapy (NCT04614636) is enrolling in the first dose cohort (100 million cells per dose). The Company is also working with investigators from the Masonic Cancer Center, University of Minnesota to initiate a Phase 1 clinical trial of FT538 in combination with the CD38-targeted monoclonal antibody daratumumab in patients with relapsed / refractory AML, a therapeutic strategy designed to exploit the product candidate’s proprietary high-affinity, non-cleavable (hnCD16) receptor and CD38 knock-out (CD38KO) to target and eliminate CD38+ leukemic blasts.
ASGCT Symposium to Highlight Adaptive NK Cell Features and Functionality of FT538. At the 24th Annual American Society of Gene & Cell Therapy Meeting (ASGCT) (Free ASGCT Whitepaper) to be held virtually from May 11-14, Dr. Jeffrey S. Miller, Professor of Medicine, University of Minnesota and Deputy Director of the Masonic Cancer Center, plans to present preclinical data demonstrating that the metabolic, transcriptional and functional properties of FT538 are substantially similar to those of adaptive NK cells, a discrete subset of memory-like NK cells with superior effector function. First described as a well-defined subset of NK cells that expand in cytomegalovirus (CMV) seropositive individuals (Gumá et al., 2004), adaptive NK cells exhibit increased effector function, enhanced persistence, resistance to oxidative stress, and potent serial cytotoxicity. Dr. Miller’s presentation is scheduled to be held on May 11 during a session entitled Recent Advances and Future Directions of Gene and Cellular Therapies in Immune Oncology.
B-cell Malignancy Disease Franchise

New FT516 Phase 1 Clinical Data for B-cell Lymphoma to be Presented at ASCO (Free ASCO Whitepaper). Dose escalation is ongoing with enrollment in the fourth dose cohort (900 million cells per dose) in the Phase 1 clinical trial to assess the safety and determine the maximum dose of FT516 in combination with CD20-targeted monoclonal antibody therapies for the treatment of relapsed / refractory B-cell lymphoma (BCL) (NCT04023071). The Company plans to present new clinical data from the Phase 1 study as part of a poster session at the 2021 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting (ASCO) (Free ASCO Whitepaper) being held virtually from June 4-8. In December 2020, the Company reported positive interim data from the second and third dose cohorts (90 million and 300 million cells per dose, respectively), with three of four patients achieving an objective response including two complete responses.
Submitted FT596 Protocol Amendment to FDA to Assess Multi-dose Treatment Schedule. The Phase 1 clinical trial of FT596 (CAR19 + hnCD16 + IL-15RF engineered iPSC-derived NK cell) is designed to assess the safety and determine the maximum dose of FT596 as a monotherapy and in combination with CD20-targeted monoclonal antibody therapies for the treatment of relapsed / refractory BCL and chronic lymphocytic leukemia (CLL) (NCT04245722). Dose escalation of the single-dose treatment schedule is ongoing with enrollment in the third dose cohort (300 million cells) as monotherapy and in combination with rituximab for the treatment of BCL, and in the first dose cohort (30 million cells) as a monotherapy for the treatment of CLL. The Company has submitted a protocol amendment to the U.S. Food and Drug Administration (FDA) to also assess administration of multi-dose treatment schedules for FT596.
FT819 Product Attributes to be Featured in Oral Presentation at ASGCT (Free ASGCT Whitepaper). The Company is preparing to initiate a multi-center Phase 1 clinical trial of FT819, the first-ever off-the-shelf, allogeneic CAR T-cell therapy derived from a clonal master iPSC line, for patients with BCL, CLL, or acute lymphoblastic leukemia (ALL). In an oral presentation at ASGCT (Free ASGCT Whitepaper), the Company plans to describe the generation of the clonal master engineered iPSC line for FT819 using its proprietary iPSC platform, and to present preclinical data of FT819 characterizing its phenotypic and functional profile, including its expression of memory, activation and exhaustion markers, and its anti-tumor activity in comparison to primary CAR T cells. FT819 is engineered with several first-of-kind features designed to improve the safety and efficacy of CAR T-cell therapy including a novel 1XX CAR signaling domain targeting CD19+ malignancies (1XX-CAR19) that extends T-cell effector function without eliciting exhaustion; integration of the CAR transgene directly into the T-cell receptor alpha constant (TRAC) locus, which promotes uniform CAR expression and enhances T-cell potency; and complete bi-allelic disruption of T-cell receptor expression to prevent graft-versus-host disease.
Multiple Myeloma Franchise

Phase 1 Study for FT538 in Combination with Daratumumab Set for Initiation. The Phase 1 clinical trial is designed to assess three once-weekly doses of FT538 in combination with the CD38-targeted monoclonal antibody, daratumumab, for patients with relapsed / refractory multiple myeloma (NCT04614636). The Company will initiate enrollment at 100 million cells per dose upon clearance of the first dose cohort in the study’s AML regimen assessing FT538 as monotherapy.
FT576 Preclinical Data Presented at AACR (Free AACR Whitepaper) Demonstrate Super Anti-tumor Activity. At the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2021 (AACR) (Free AACR Whitepaper) in April, the Company presented preclinical data for FT576 (CAR-BCMA + hnCD16 + IL-15RF + CD38KO engineered iPSC-derived NK cells), demonstrating superior in vivo persistence and anti-tumor activity as compared to cytokine-expanded primary NK cells in a disseminated xenograft model of multiple myeloma. In addition, the Company observed that multi-antigen targeting of FT576 in combination with daratumumab exhibits greater in vivo efficacy compared to primary CAR T cells in combination with a gamma secretase inhibitor. FT576 is derived from a clonal master iPSC line engineered with four functional components designed to enable multi-antigen targeting of myeloma cells, augment antibody-dependent cellular cytotoxicity (ADCC), promote NK cell activation without exogenous cytokine support, enhance cell persistence and prevent anti-CD38 monoclonal antibody-induced fratricide. The Company is preparing to initiate a multi-center Phase 1 clinical trial to assess single-dose and multi-dose treatment regimens of FT576 as monotherapy and in combination with CD38-targeted monoclonal antibody therapy for the treatment of relapsed / refractory multiple myeloma.
Solid Tumor Franchise

IND Application Allowed by FDA for FT538 in Combination with Monoclonal Antibody Therapy for Solid Tumors. The Company plans to initiate a multi-center Phase 1 clinical trial to assess the safety and determine the maximum dose of FT538 in combination with monoclonal antibody therapy for the treatment of a broad array of solid tumors. The novel therapeutic strategy is designed to exploit the product candidate’s hnCD16 receptor to promote ADCC, a potent anti-tumor mechanism by which NK cells recognize, bind and kill antibody-coated cancer cells. The clinical protocol includes combination with three monoclonal antibodies: EGFR-targeted cetuximab; HER2-targeted trastuzumab; and PDL1-targeted avelumab. Each patient is eligible to receive up to two FT538 treatment cycles, with each cycle consisting of three days of outpatient lympho-conditioning, three once-weekly infusions of FT538, and monoclonal antibody therapy.
Preclinical Studies of FT536 CAR MICA/B Program Demonstrate Superiority over Primary NK Cells. At AACR (Free AACR Whitepaper), the Company highlighted its development of FT536, a novel CAR NK cell product candidate targeting the alpha-3 domain of the pan-tumor associated stress antigens MICA and MICB. In preclinical studies designed to increase the density of stress antigen expression and sensitivity of tumor cells to NKG2D-mediated recognition and killing by NK cells, FT536 demonstrated superior anti-tumor activity compared to cytokine-expanded primary NK cells across a broad array of cancer cell lines. The Company plans to submit an IND application in the second half of 2021 to initiate a Phase 1 clinical trial of FT536 for the treatment of solid tumors.
Novel B7H3-targeted Binding Domain Selected for Development of iPSC-derived CAR NK Cell Program. At AACR (Free AACR Whitepaper), the Company presented preclinical data demonstrating anti-tumor activity of its novel B7H3 binding domain, including when incorporated in a tri-specific NK cell engager (TriKE), a primary CAR T cell, and an iPSC-derived CAR NK cell. B7H3 is an immune checkpoint molecule of the B7 protein superfamily with broad expression in cancer and its upregulation is associated with metastatic cancer and poor prognosis. The Company is conducting preclinical development of multiplexed-engineered, iPSC-derived CAR B7H3 NK cell and T-cell product candidates for solid tumors.
ProTmune

Phase 2 PROTECT Study Did Not Meet Primary Endpoint for Prevention of Acute GvHD. The randomized, controlled and double-blinded PROTECT study was designed to assess the safety and efficacy of ProTmune, a patient-specific hematopoietic cell graft manufactured on-site at local transplant centers using the Company’s ex vivo small molecule modulation technology, for patients undergoing allogeneic stem cell transplant. The results failed to show a statistically-significant difference between ProTmune and control with respect to the primary endpoint of cumulative incidence of Grades 2-4 acute GvHD by Day 100, and the Company is discontinuing development of ProTmune.
Other Corporate Highlights

Completed $460 Million Public Offering. In January 2021, the Company completed an underwritten public offering of 5.1 million shares of its common stock priced at $85.50 per share and, in lieu of common stock to certain investors, pre-funded warrants to purchase 0.3 million shares of its common stock priced at $85.499 per pre-funded warrant.
First Quarter 2021 Financial Results

Cash & Investment Position: Cash, cash equivalents and investments as of March 31, 2021 were $888.4 million. This amount includes net proceeds to the Company of approximately $432 million from the January 2021 underwritten public offering.
Total Revenue: Revenue was $11.1 million for the first quarter of 2021, which was derived from the Company’s collaborations with Janssen and Ono Pharmaceutical.
R&D Expenses: Research and development expenses were $44.9 million for the first quarter of 2021, which includes $8.5 million of non-cash stock-based compensation expense.
G&A Expenses: General and administrative expenses were $12.5 million for the first quarter of 2021, which includes $4.5 million of non-cash stock-based compensation expense.
Shares Outstanding: Common shares outstanding were 93.9 million, and preferred shares outstanding were 2.8 million, as of March 31, 2021. Each preferred share is convertible into five common shares.
Today’s Conference Call and Webcast

The Company will conduct a conference call today, Wednesday, May 5, 2021 at 5:00 p.m. ET to review financial and operating results for the quarter ended March 31, 2021. In order to participate in the conference call, please dial 800-708-4539 (toll free) or 847-619-6396 (toll) and refer to conference ID 50156207. The live webcast can be accessed under "Events & Presentations" in the Investors section of the Company’s website at www.fatetherapeutics.com. The archived webcast will be available on the Company’s website beginning approximately two hours after the event.

On May 5, 2021 Supernus Pharmaceuticals, Inc. (Nasdaq: SUPN), a biopharmaceutical company focused on developing and commercializing products for the treatment of central nervous system (CNS) diseases, reported financial results for the first quarter of 2021, and associated Company developments (Press release, Supernus, MAY 5, 2021, View Source [SID1234579181]).

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"The approval of Qelbree provides pediatric patients living with ADHD a therapy with proven efficacy and a tolerable safety profile, and that is not a controlled substance," said Jack Khattar, President and CEO of Supernus Pharmaceuticals. "Our Qelbree commercial launch activities are ongoing and include engagement with both physicians and patient groups who have expressed great interest in this unique new alternative for the treatment of ADHD."

Net Product Sales

First quarter 2021 net product sales were $128.4 million, 39% higher than the same period in 2020.

Qelbree Launch Update

In April 2021, the U.S. Food and Drug Administration (FDA) approved Qelbree for the treatment of attention-deficit hyperactivity disorder (ADHD) in pediatric patients 6 to 17 years of age. The Company plans to make Qelbree available in the U.S. during the second quarter of 2021.
Supernus will conduct post-marketing commitment studies, including a new study of Qelbree in preschool aged children with ADHD, 4 to 5 years of age. The completion of these studies responds to a written request from the FDA and should therefore result in the FDA granting an additional 6 months of market exclusivity.
Product Pipeline Update

Qelbree (viloxazine, extended-release capsules) – Novel non-stimulant for the treatment of ADHD in adults

In December 2020, the Company announced positive results from a Phase III trial in adult patients with ADHD and plans to submit a supplemental New Drug Application (sNDA) to the FDA for Qelbree in adults in the third quarter of 2021.
SPN-830 (apomorphine infusion pump) – Continuous treatment of motor fluctuations ("on-off" episodes) in PD

The company recently met with the FDA to discuss the path forward for resubmission of the SPN-830 NDA. The FDA provided additional clarity related to the contents of the November 2020 Refusal to File (RTF) letter and the requirements for resubmission. The Company now plans to resubmit the SPN-830 NDA in the second half of 2021.
SPN-820 – Novel first-in-class activator of mTORC1

SPN-820 has advanced to a Phase II clinical program in treatment-resistant depression following the successful completion of a multiple-ascending dose (MAD) study in healthy volunteers. In the MAD study, SPN-820 exhibited a favorable safety and tolerability profile across a broad range of potentially therapeutic doses.
The Company expects to initiate a randomized Phase II clinical study in treatment-resistant depression by the end of 2021.
Financial Highlights

First quarter 2021 operating earnings were $13.2 million, as compared to $29.0 million in the first quarter 2020. In the first quarter of 2021, the Company recorded non-cash research and development expense of $15 million related to the equity investment in Navitor as a result of the accounting impact of the March 2021 Navitor corporate restructuring and non-cash contingent consideration expense of $1 million associated with the 2020 USWM acquisition. Operating earnings for the first quarter of 2021 included amortization of intangible assets expense of $6.0 million, compared to $1.3 million in the first quarter of 2020.

First quarter 2021 net earnings and diluted earnings per share were $5.7 million and $0.11, respectively, as compared to $21.5 million, or $0.40 per diluted share, in the same period last year.

As of March 31, 2021, the Company had $807.7 million in cash, cash equivalents and marketable securities, compared to $772.9 million as of December 31, 2020.

Full Year 2021 Financial Guidance

For full year 2021, the Company reiterates its prior financial guidance and added full year 2021 effective tax rate guidance as set forth below:

1) Total revenues includes net product sales and royalty revenue. Includes $10 million for Qelbree net product sales.
2) Combined research and development and selling, general and administrative expenses.
3) Operating earnings include amortization of intangible assets and contingent consideration expense.
4) The full year 2021 effective tax rate guidance of 28% – 31% is above the normally expected range of 26% – 28% due to the effect of discrete tax items in the period.

Conference Call Details

The Company will hold a conference call hosted by Jack Khattar, President and Chief Executive Officer and Jim Kelly, Executive Vice President and Chief Financial Officer, to discuss these results at 4:30 p.m. Eastern Time, today, May 5, 2021.

Please refer to the information below for conference call dial-in information and webcast registration. Callers should dial in approximately 10 minutes prior to the start of the call.