On November 9, 2020 Atara Biotherapeutics, Inc. (Nasdaq: ATRA), a pioneer in T-cell immunotherapy, leveraging its novel allogeneic EBV T-cell platform to develop transformative therapies for patients with serious diseases including solid tumors, hematologic cancers and autoimmune disease, reported financial results for the third quarter ended September 30, 2020 and recent business highlights (Press release, Atara Biotherapeutics, NOV 9, 2020, View Source [SID1234570369]).

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"Atara is on track to finish the year strong, meeting each milestone set to date," said Pascal Touchon, President and Chief Executive Officer of Atara. "Following an encouraging interim analysis of the pivotal Phase 3 data for tab-cel in relapsed-refractory EBV+ PTLD and very productive interactions with regulatory authorities, we are preparing for tab-cel submissions, and then approval and launch. We are committed to bringing this potentially life-saving therapy to the patients who acutely need it as they have no other therapeutic solution and face a very limited median life expectancy of only two to three months."

Tab-cel for Post-transplant Lymphoproliferative Disease (PTLD)

An Interim Analysis of the tab-cel ALLELE study showed a 50 percent objective response rate (ORR) to tab-cel with independent oncologic and radiographic assessment (IORA) in patients with relapsed-refractory EBV+ PTLD following hematopoietic cell transplants (HCT) or solid organ transplants (SOT), that had reached at least six months follow-up after achieving a response. This ORR is consistent with previously published investigator assessed data. The tab-cel safety profile is also consistent with previously published data, with no new safety signals.
In October, working under the Breakthrough Therapy Designation (BTD) for tab-cel, Atara presented a comprehensive data package to the FDA. The Company aligned with FDA on several key topics related to the regulatory package, including:
a rolling submission is acceptable for the biologics license application (BLA);
the Company can complete the BLA submission with the ALLELE study’s currently enrolled patients with at least six months follow-up for duration of response; and
the FDA will consider the following as supportive data to the pivotal study in the BLA clinical module: the Phase 2 trials conducted at Memorial Sloan Kettering (MSK); Atara’s Phase 2 multicenter expanded access protocol (201 EAP study); and, the Single Patient Use (SPU) program.
Atara remains on track to initiate a BLA submission for patients with EBV+ PTLD by the end of 2020. The Company will continue engaging with the FDA as part of its rolling BLA and BTD status and expects to finalize the BLA submission in Q3 2021.
Following discussion with the PRIority MEdicines (PRIME) team and after EMA approval of the Pediatric Investigation Plan (PIP) expected in December 2020, Atara is on track to submit an EU marketing authorization application (MAA) for patients with EBV+ PTLD in the second half of 2021.
Data from the ALLELE study will be presented at an appropriate congress in 2021.
Tab-cel for Potential Additional Indications

Atara initiated a tab-cel Phase 2 multi-cohort study in the third quarter of 2020 and expects to enroll the first patient in the fourth quarter of 2020. This study is being initiated concurrently in the U.S. and the EU.
The multi-cohort study is intended to enrich the evidence base with the goal of expanding the potential label for tab-cel in both treatment-naïve and previously treated patients (in six populations, including four within Immunodeficiency-Associated Lymphoproliferative Diseases (IA-LPDs) and two in other EBV-associated diseases).
Data demonstrating tab-cel was well-tolerated and showed encouraging clinical activity in patients with EBV+ AID-LPD and PID-LPD (Acquired and Primary Immunodeficiency LPDs) were featured in an e-poster at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2020 Virtual Congress held in September 2020. Specifically, in patients where previous treatments have failed, the objective response rate, including complete response, were 33.3 percent (three out of nine patients) in AID-LPD and 37.5 percent (three out of eight patients) in PID-LPD groups. Tab-cel was generally well-tolerated with a favorable safety profile consistent with previously published clinical studies.
Data on tab-cel in patients with life-threatening complications stemming from persistent EBV viremia was accepted for presentation at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition being held virtually December 5-8, 2020. These clinical data demonstrate that tab-cel was well-tolerated and showed encouraging clinical activity in this patient population with objective response rate ranging from 50 to 80 percent, warranting further investigation in the Phase 2 multi-cohort study.
ATA188 for Progressive Forms of Multiple Sclerosis (MS)

Atara’s Phase 1 clinical study of off-the-shelf, allogeneic ATA188 in patients with progressive forms of MS is ongoing, with encouraging data recently reported and more data from the Open Label Extension (OLE) to be periodically presented over the next 12 months including at the European Charcot Foundation (ECF) 28th Annual Meeting November 15-19, 2020.
Twelve-month data from all four cohorts in the Phase 1a portion of the study were presented at the MSVirtual2020: 8th Joint ACTRIMS-ECTRIMS Meeting held in September 2020.
These data demonstrated that ATA188 was well-tolerated across all four dose cohorts; patients who demonstrated sustained disability improvements (SDI) at any timepoint maintained it at all future timepoints; and, a higher proportion of patients showed SDI with increasing dose (42 percent in Cohorts 3 and 4 (higher doses) vs 17 percent in Cohorts 1 and 2 (lower doses)).
No dose-limiting toxicities and no fatal adverse events (AEs) have been reported. The safety profile has remained consistent with previously reported data.
Data from the OLE with redosing at 12 months show that of the three patients enrolled in the OLE at that time that had SDI at 12 months, all maintained SDI at 15 months, including one patient evaluated at both 15 and 18 months who maintained SDI at both time points. A fourth patient demonstrated SDI during the OLE at 24 months.
Atara also presented preclinical translational data at ACTRIMS-ECTRIMS that further support the proposed mechanism of action of ATA188 targeting EBV-infected B cells. These combined analyses of T cells comprising ATA188 are consistent with its proposed mechanism of targeting EBV-infected B cells by recognizing MS-relevant EBV antigens on these cells via defined T cell receptors (TCRs).
The double-blind randomized placebo-controlled trial (RCT), which enrolled its first patient in June 2020 continues active recruitment.
Given encouraging clinical results to date in ATA188 studies and the significant unmet medical need in progressive forms of MS, the Company is increasing its investment in the ATA188 program. Atara is expanding the size of the RCT to at least 64 patients, changing the primary endpoint of the study to disability improvement, and maintaining biological and functional endpoints; the design allows for the addition of the cohort 4 dose if desired.
Atara plans to discuss the Phase 1a data with the FDA by the end of this year, as well as the updated RCT study design, and potential opportunities for accelerated development of ATA188 for MS patients.
CAR T Programs

ATA2271/ATA3271 (Solid Tumors Over-Expressing Mesothelin)

The open-label, single-arm Phase 1 clinical study of ATA2271, the Company’s second-generation autologous CAR T therapy targeting mesothelin (MSLN) that incorporates for the first time both a PD-1 DNR (dominant-negative programmed death-1 receptor) for intrinsic check-point inhibition and novel 1XX co-stimulatory domain, has been initiated for the treatment of advanced mesothelioma. Recently presented preclinical data have shown improved functional persistence and enhanced anti-tumor efficacy superior to first-generation MSLN CAR T. MSK is on track to enroll the first patient in the Phase 1 study in Q4 2020.
ATA3271 is an off-the-shelf, allogeneic CAR T therapy targeting MSLN using a PD-1 DNR and 1XX CAR co-stimulatory signaling domain through Atara’s EBV T-cell platform. In new preclinical data, ATA3271 demonstrates potent anti-tumor activity, functional persistence and significant survival benefit with no evidence of allocytotoxicity in vivo, suggesting that allogeneic MSLN-CAR-engineered EBV T cells are a promising approach for the treatment of MSLN-positive cancers. These data will be presented at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 35th Anniversary Annual Meeting November 11-14, 2020.
ATA3219 (B-cell Malignancies)

A collaborative and successful pre-IND meeting with the FDA in October 2020 provided feedback to guide the IND filing in 2021.
IND-enabling studies are progressing with a package ready to be filed in 2021 for ATA3219, a potent next-generation off-the-shelf allogeneic CD19 CAR T utilizing the 1XX technology without the need for TCR gene editing through Atara’s EBV T-cell platform.
The first abstract to be presented on ATA3219 was accepted for presentation at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition being held virtually December 5-8, 2020. These preclinical data for ATA3219 demonstrate persistence, polyfunctional phenotype and efficient targeting of CD19-expressing tumor cells with no evidence of allocytotoxicity in vivo.
Third Quarter 2020 Financial Results

Cash, cash equivalents and short-term investments as of September 30, 2020 totaled $327.2 million, as compared to $347.7 million as of June 30, 2020.
September 30, 2020 cash balance of $327.2 million included $34.1 million net proceeds from at-the-market (ATM) facilities during the third quarter of 2020. Third quarter 2020 cash burn was $20.5 million, net of ATM proceeds.
Atara believes that its cash, cash equivalents and short-term investments as of September 30, 2020 are sufficient to fund planned operations into 2022.
Net cash used in operating activities was $53.0 million for the third quarter of 2020, as compared to $52.1 million for the same period in 2019.
The number of outstanding shares of common stock and pre-funded common stock warrants as of September 30, 2020 was 77,220,159 shares and warrants to purchase 5,755,487 shares, respectively.
Atara reported net losses of $74.3 million, or $0.92 per share, for the third quarter of 2020, as compared to $71.9 million, or $1.31 per share, for the same period in 2019.
Total operating expenses include non-cash expenses of $15.4 million for the third quarter 2020, as compared to $13.9 million for the same period in 2019.
Research and development expenses were $59.9 million for the third quarter of 2020, as compared to $53.5 million for the same period in 2019. The increase in the 2020 period was primarily due to costs associated with the Company’s continuing expansion of research and development activities, including:
Clinical trial, manufacturing and process performance qualification activities related to tab-cel.
Higher employee-related costs from increased headcount.
Research and development expenses include $8.2 million of non-cash stock-based compensation expenses for the third quarter of 2020, as compared to $7.0 million for the same period in 2019.
General and administrative expenses were $14.8 million for the third quarter of 2020, as compared to $19.0 million for the same period in 2019. The decrease in the 2020 period was primarily due to decrease in outside services costs and employee-related costs.
General and administrative expenses include $5.1 million of non-cash stock-based compensation expenses for the third quarter of 2020, as compared to $5.1 million for the same period in 2019.

On November 9, 2020 Infinity Pharmaceuticals, Inc. (NASDAQ: INFI) reported its third quarter 2020 financial results and provided an update on the Company, including its third quarter progress with eganelisib (IPI-549), the Company’s first-in-class, oral immuno-oncology product candidate targeting immune-suppressive tumor-associated myeloid cells through selective phosphoinositide-3-kinase-gamma (PI3K-gamma) inhibition (Press release, Infinity Pharmaceuticals, NOV 9, 2020, View Source [SID1234570368]).

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"We are approaching an important inflection point at Infinity, with expected data readouts across our clinical programs in the next few months that demonstrate the benefit of eganelisib across multiple indications, patient populations and treatment settings." said Adelene Perkins, Chief Executive Officer and Chair of Infinity Pharmaceuticals. "We are pleased that the IDMC supports the further exploration of eganelisib in second-line metastatic urothelial cancer. We will continue to follow the forty-nine patients previously enrolled through the remainder of the year and use these data to determine the best path forward, which may include the re-opening of enrollment of MARIO-275 or the initiation of a new study that leverages our clinical and translational insights from the patients enrolled to date."

Ms. Perkins continued, "In addition, encouraging data from the MARIO-3 TNBC cohort suggest that eganelisib has the potential to be an important component of a new treatment regimen in the front-line setting, and we look forward to presenting these data at SABCS next month. This week we also shared data from the melanoma and SCCHN cohorts of MARIO-1 at SITC (Free SITC Whitepaper)."

Key Q3 2020 Updates:

Clinical and Regulatory:

MARIO-275 is the Company’s ongoing controlled, randomized Phase 2 study evaluating eganelisib in combination with Opdivo in platinum-refractory, I/O naïve patients with advanced urothelial cancer (aUC), in collaboration with Bristol Myers Squibb.
The MARIO-275 Independent Data Monitoring Committee (IDMC) determined that the risk/benefit for patients warrants resumption of enrollment after the successful implementation of a dose reduction from 40mg QD to 30mg QD to reduce the reversible liver enzyme elevations, which were reported after the first scheduled MARIO-275 IDMC meeting.
Infinity is continuing to evaluate the forty-nine patients previously enrolled across safety and time-to-event measures including progression free survival and overall survival and will determine next steps by year end. This may include the re-opening of enrollment of MARIO-275 or the initiation of a new study which leverages our findings from the patients enrolled to date.
MARIO-3 is the Company’s ongoing Phase 2 study in collaboration with Roche/Genentech to evaluate eganelisib in a novel triple combination in the front-line setting with Tecentriq and Abraxane in triple negative breast cancer (TNBC) and with Tecentriq and Avastin in renal cell cancer (RCC).
Encouraging data from the TNBC cohort of MARIO-3 will be presented at the San Antonio Breast Cancer Symposium (SABCS) Annual Meeting, December 8-11, 2020.
Fast Track Designation: Infinity received Fast Track Designation for eganelisib in combination with a checkpoint inhibitor and chemotherapy for first-line treatment of advanced TNBC.
Enrollment has been completed in the RCC cohort. In TNBC, we have implemented a number of enrollment initiatives and expect to provide an update on enrollment expectations at SABCS.
MARIO-1-is the Company’s ongoing Phase 1/1b study evaluating eganelisib as a monotherapy and in combination with Opdivo in patients with solid tumors in collaboration with Bristol Myers Squibb.
Presented data from the MARIO-1 melanoma and squamous cell carcinoma of the head and neck (SCCHN) cohorts, which were designed to isolate the clinical benefit of eganelisib by examining clinical activity in patients not expected to respond to checkpoint inhibitor monotherapy due progression on an immediate prior checkpoint inhibitor, at SITC (Free SITC Whitepaper). Both presentations demonstrate that eganelisib had a manageable safety and tolerability profile, provide further validation of the eganelisib mechanism of action of immune modulation, and support the Company’s strategy of moving eganelisib into earlier treatment settings.
Arcus Collaboration: A Phase 1b collaboration study being conducted by Arcus Biosciences is evaluating a checkpoint-inhibitor free, novel triple-combination regimen of eganelisib + etrumadenant (AB928, dual adenosine receptor antagonist) + Doxil in up to approximately 40 advanced TNBC patients.
Data from the study will be presented at SABCS in December 2020.
Third Quarter 2020 Financial Results:

At September 30, 2020, Infinity had total cash, cash equivalents and available-for-sale securities of $41.3 million, compared to $42.7 million at June 30, 2020.
Research and development expense for the third quarter of 2020 was $6.1 million, compared to $7.1 million for the same period in 2019. The decrease is primarily related to a combination drug purchase during the third quarter of 2019.
General and administrative expense was $2.9 million for the third quarter of 2020, compared to $3.6 million for the same period in 2019. The decrease is primarily related to a reduction in professional services and consulting.
Net loss for the third quarter of 2020 was $9.5 million, or a basic and diluted loss per common share of $0.16, compared to a net loss of $11.4 million, or a basic and diluted loss per common share of $0.20 for the same period in 2019.
2020 Financial Outlook:

Net Loss: Infinity expects net loss for 2020 to range from $35 million to $45 million.

Cash and Investments: Infinity expects to end 2020 with a year-end cash, cash equivalents and available-for-sale securities balance ranging from $25 million to $35 million.

Cash Runway: Based on its current operational plans, Infinity expects that its existing cash, cash equivalents and available-for-sale securities, will be adequate to satisfy the Company’s capital needs through 2021. Infinity’s financial guidance does not include potential additional funding or business development activities, a potential $5 million milestone payment from BVF based on PellePharm’s ongoing Phase 3 clinical trial of patidegib topical gel in Gorlin Syndrome, or any milestones from, or the sale of the Company’s equity interest in, PellePharm.

Conference Call Information

Infinity will host a conference call today, November 9, 2020, at 4:30 p.m. ET to discuss these financial results and company updates. A live webcast of the conference call can be accessed in the "Investors/Media" section of Infinity’s website at www.infi.com. To participate in the conference call, please dial (877) 316-5293 (domestic) and (631) 291-4526 (international) five minutes prior to start time. The conference ID number is 1575996. An archived version of the webcast will be available on Infinity’s website for 30 days.

On November 9, 2020 Cellectar Biosciences, Inc. (NASDAQ: CLRB), a late-stage clinical biopharmaceutical company focused on the discovery, development and commercialization of drugs for the treatment of cancer, reported financial results for the third quarter ended September 30, 2020 and provided a corporate update (Press release, Cellectar Biosciences, NOV 9, 2020, View Source [SID1234570367]).

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Third Quarter and Recent Corporate Highlights

·Held FDA Type B guidance meeting to define the registrational pathway for our priority adult hematology oncology indications and planned initiation of the pivotal study for our lead indication in the fourth quarter

·Announced CLR 131 achieved a 40% overall response rate (ORR) in Triple Class Refractory Multiple Myeloma (MM) patients with an administered total body dose (TBD) of 60mCi or greater from the Phase 2 CLOVER-1 study

·Interim results from the Phase 2 COVER-1 study at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) Virtual Meeting: Advances in Malignant Lymphoma demonstrated a 100% ORR and a 75% major response rate (MRR) in LPL/WM. Mean duration of response exceeding 17 months (8.4 – 31.7 months); duration of response continues to increase for all patients

·Strengthened the management team with the appointment of Dr. John Friend, chief medical officer

"We continue to make good progress towards the fourth quarter initiation of the CLR 131 pivotal study in our lead heme-oncology indication. Our recent FDA guidance meeting was most encouraging and we look forward to providing greater details in the near-term," said James Caruso, president and CEO of Cellectar. "Additional data from our Phase 2 CLOVER-1 study remain strong, with patients in WM achieving a 100% ORR and a 75% MRR in patients failing a BTKi and a 40% ORR in the challenging to treat triple class refractory MM patient population."

Third Quarter 2020 Financial Highlights

·Cash and Cash Equivalents: As of September 30, 2020, the company had cash and cash equivalents of $18.8 million compared to $10.6 million at December 31, 2019. Cash used in operating activities was approximately $10.1 million during the nine months ended September 30, 2020 as compared to $9.0 million during the nine months ended September 30, 2019.

·Research and Development Expense: R&D expense for the three months ended September 30, 2020 was $2.7 million, compared to $2.7 million for the three months ended September 30, 2019. The cumulative R&D spending for the first nine months of 2020 was $7.8 million as compared to $6.8 million for the first nine months of 2019. The increase in R&D expense year-to-date in 2020 was primarily a result of higher general research and development costs resulting from increased personnel related costs and clinical study costs. Manufacturing and related costs decreased because of a reduction in materials production processes and related costs.

·General and Administrative Expense: G&A expense for the three months ended September 30, 2020 was $1.2 million compared to $1.3 million for the three months ended September 30, 2019. The cumulative G&A spending for the first nine months of 2020 were of $3.7 million as compared to $4.0 million for the first nine months of 2019. The decrease in G&A expense year-to-date in 2020 was primarily a result of lower stock-based compensation expense.

·Net Loss: The net loss attributable to common stockholders for the three months ended September 30, 2020 was ($3.9) million, or ($0.15) per share, compared to ($3.9) million, or ($0.42) per share, in 2019. Net loss attributable to common stockholders for the nine months ended September 30, 2020 was ($11.5) million, or ($0.69) per share, compared to ($10.7) million, or ($1.51) per share, in 2019.

On November 9, 2020 Neurocrine Biosciences, Inc. (Nasdaq: NBIX) reported its financial results for the third quarter ended September 30, 2020 and provided revised full-year 2020 financial expense guidance (Press release, Neurocrine Biosciences, NOV 9, 2020, View Source [SID1234570366]).

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"Our focus remains on helping as many patients with tardive dyskinesia as possible and we are adapting to the challenges posed by the pandemic, including the slow recovery of in-person visits with psychiatrists. We are encouraged by recent trends and remain confident in the intermediate and long-term opportunity of INGREZZA to help many more patients with tardive dyskinesia," said Kevin Gorman, Ph.D., Chief Executive Officer of Neurocrine Biosciences. "We are pleased to expand our reach with neurologists with the addition of ONGENTYS to our movement disorder portfolio and continue to advance our development programs focused on neurological, endocrine and psychiatric disorders."

Net Product Sales Highlights:
•INGREZZA net product sales for the third quarter of 2020 were $254 million, representing a year-over-year increase of 28%. Inventory adjusted net sales for the third quarter of 2020 were approximately $248 million.
•New prescriptions increased slightly in the third quarter of 2020 vs. the second quarter of 2020.
•Refill and persistency rates for existing INGREZZA patients in the third quarter 2020 moderated slightly vs. the second quarter of 2020 and remained at the higher end of historical averages.
•ONGENTYS launched in the United States in late September 2020 and net product sales for the third quarter of 2020 were approximately $0.1 million.
Financial Highlights:
•Third quarter 2020 GAAP net loss and loss per share were approximately $58 million and $0.62, respectively, compared with net income and diluted earnings per share of approximately $54 million and $0.56, respectively, in the third quarter of 2019, primarily driven by higher In-Process Research and Development (IPR&D) costs and operating expenses, offset by higher INGREZZA sales.
•Third quarter 2020 non-GAAP net income and diluted earnings per share were approximately $96 million and $0.97, respectively, compared with approximately $87 million and $0.90, respectively, in the third quarter of 2019 driven by higher INGREZZA sales.
•Research and Development (R&D) expense increased in the third quarter of 2020 versus the third quarter of 2019, primarily due to increased investment across our expanded pipeline programs and higher headcount costs.
•Selling, General and Administrative (SG&A) expense increased in the third quarter of 2020 versus the third quarter of 2019, primarily due to increased investment in marketing initiatives and higher headcount costs.
•At September 30, 2020, the Company had cash, cash equivalents and debt securities available-for-sale of $1.1 billion.
A reconciliation of GAAP to non-GAAP quarterly financial results can be found in Table 3 at the end of this earnings release.
Recent Events
•In September 2020, the Company launched ONGENTYS (opicapone), the first and only once-daily COMT inhibitor, as an adjunctive treatment to levodopa/carbidopa in patients with Parkinson’s disease experiencing "off" episodes – periods of time when motor symptoms such as tremor, slowed movement and difficulty walking occur. ONGENTYS also increases "on" time without troublesome dyskinesia, the time when the motor symptoms of a patient with Parkinson’s disease are better controlled.
•In October 2020, the U.S. Food and Drug Administration (FDA) provided feedback on an Investigational New Drug (IND) application submitted by Neurocrine Biosciences in support of a Phase II clinical trial of NBI-921352 in pediatric SCN8A developmental and epileptic encephalopathy syndrome patients. As part of its review of the IND, the FDA is requesting additional non-clinical data to support dose justification in this pediatric study. Together with Xenon, the Company will engage with the FDA to address the feedback received with the goal of initiating a Phase II clinical trial in 2021. In parallel, the Company is advancing clinical plans to develop NBI-921352 for the treatment of adult focal epilepsy.
•In November 2020, the Company announced the initiation of a Phase II study of NBI-827104 (formerly ACT-709478) in CSWS, a rare pediatric epilepsy. NBI-827104 was licensed from Idorsia and is a potent, selective, orally-active and brain penetrating T-type calcium channel blocker.
•In November 2020, the Data Safety Monitoring Board (DSMB) for the RESTORE-1 Phase II clinical trial reviewed patient imaging data from the ongoing trial. The DSMB requested additional data and recommended the Company pause dosing of subjects in RESTORE-1 until the DSMB can review these additional data, which is expected by year-end. The DSMB informed the Company and Voyager Therapeutics that they could continue screening patients for potential enrollment into the trial. However, as previously announced, trial sites participating in RESTORE-1 are not currently screening and enrolling patients as a result of the COVID-19 pandemic. In response to the DSMB’s recommendation, the Company and Voyager Therapeutics have decided to delay the planned resumption this quarter of patient screening in the RESTORE-1 trial until the DSMB is able to complete its evaluation. The Company is preparing an expedited safety report that will be submitted to the FDA within the 15-day reporting window.
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 877-830-2596 (US) or 785-424-1744 (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.

On November 9, 2020 Neurocrine Biosciences, Inc. (Nasdaq: NBIX) reported its financial results for the third quarter ended September 30, 2020 and provided revised full-year 2020 financial expense guidance (Press release, Neurocrine Biosciences, NOV 9, 2020, View Source [SID1234570365]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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"Our focus remains on helping as many patients with tardive dyskinesia as possible and we are adapting to the challenges posed by the pandemic, including the slow recovery of in-person visits with psychiatrists. We are encouraged by recent trends and remain confident in the intermediate and long-term opportunity of INGREZZA to help many more patients with tardive dyskinesia," said Kevin Gorman, Ph.D., Chief Executive Officer of Neurocrine Biosciences. "We are pleased to expand our reach with neurologists with the addition of ONGENTYS to our movement disorder portfolio and continue to advance our development programs focused on neurological, endocrine and psychiatric disorders."

Net Product Sales Highlights:

INGREZZA net product sales for the third quarter of 2020 were $254 million, representing a year-over-year increase of 28%. Inventory adjusted net sales for the third quarter of 2020 were approximately $248 million.
New prescriptions increased slightly in the third quarter of 2020 vs. the second quarter of 2020.
Refill and persistency rates for existing INGREZZA patients in the third quarter 2020 moderated slightly vs. the second quarter of 2020 and remained at the higher end of historical averages.
ONGENTYS launched in the United States in late September 2020 and net product sales for the third quarter of 2020 were approximately $0.1 million.
Financial Highlights:

Third quarter 2020 GAAP net loss and loss per share were approximately $58 million and $0.62, respectively, compared with net income and diluted earnings per share of approximately $54 million and $0.56, respectively, in the third quarter of 2019, primarily driven by higher In-Process Research and Development (IPR&D) costs and operating expenses, offset by higher INGREZZA sales.
Third quarter 2020 non-GAAP net income and diluted earnings per share were approximately $96 million and $0.97, respectively, compared with approximately $87 million and $0.90, respectively, in the third quarter of 2019 driven by higher INGREZZA sales.
Research and Development (R&D) expense increased in the third quarter of 2020 versus the third quarter of 2019, primarily due to increased investment across our expanded pipeline programs and higher headcount costs.
Selling, General and Administrative (SG&A) expense increased in the third quarter of 2020 versus the third quarter of 2019, primarily due to increased investment in marketing initiatives and higher headcount costs.
At September 30, 2020, the Company had cash, cash equivalents and debt securities available-for-sale of $1.1 billion.
A reconciliation of GAAP to non-GAAP quarterly financial results can be found in Table 3 at the end of this earnings release.

Recent Events

In September 2020, the Company launched ONGENTYS (opicapone), the first and only once-daily COMT inhibitor, as an adjunctive treatment to levodopa/carbidopa in patients with Parkinson’s disease experiencing "off" episodes – periods of time when motor symptoms such as tremor, slowed movement and difficulty walking occur. ONGENTYS also increases "on" time without troublesome dyskinesia, the time when the motor symptoms of a patient with Parkinson’s disease are better controlled.
In October 2020, the U.S. Food and Drug Administration (FDA) provided feedback on an Investigational New Drug (IND) application submitted by Neurocrine Biosciences in support of a Phase II clinical trial of NBI-921352 in pediatric SCN8A developmental and epileptic encephalopathy syndrome patients. As part of its review of the IND, the FDA is requesting additional non-clinical data to support dose justification in this pediatric study. Together with Xenon, the Company will engage with the FDA to address the feedback received with the goal of initiating a Phase II clinical trial in 2021. In parallel, the Company is advancing clinical plans to develop NBI-921352 for the treatment of adult focal epilepsy.
In November 2020, the Company announced the initiation of a Phase II study of NBI-827104 (formerly ACT-709478) in CSWS, a rare pediatric epilepsy. NBI-827104 was licensed from Idorsia and is a potent, selective, orally-active and brain penetrating T-type calcium channel blocker.
In November 2020, the Data Safety Monitoring Board (DSMB) for the RESTORE-1 Phase II clinical trial reviewed patient imaging data from the ongoing trial. The DSMB requested additional data and recommended the Company pause dosing of subjects in RESTORE-1 until the DSMB can review these additional data, which is expected by year-end. The DSMB informed the Company and Voyager Therapeutics that they could continue screening patients for potential enrollment into the trial. However, as previously announced, trial sites participating in RESTORE-1 are not currently screening and enrolling patients as a result of the COVID-19 pandemic. In response to the DSMB’s recommendation, the Company and Voyager Therapeutics have decided to delay the planned resumption this quarter of patient screening in the RESTORE-1 trial until the DSMB is able to complete its evaluation. The Company is preparing an expedited safety report that will be submitted to the FDA within the 15-day reporting window.
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 877-830-2596 (US) or 785-424-1744 (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.