On May 6, 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 severe diseases including solid tumors, hematologic cancers and autoimmune disease, reported financial results for the first quarter ended March 31, 2020 and recent business highlights (Press release, Atara Biotherapeutics, MAY 6, 2020, View Source [SID1234557139]).

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"It is with tremendous pride that I acknowledge the commitment and resiliency of our entire Atara team. We have remained focused on our mission to serve patients and implemented industry-leading practices to ensure safety while mitigating the impact of COVID-19 on our business," said Pascal Touchon, President and Chief Executive Officer of Atara. "We have made great progress in Q1 toward accomplishing our key objectives and are well-poised to achieve significant milestones throughout the year as we expect to initiate the tab-cel biologics license application (BLA) submission in the second half of 2020 and are eager to present further results from the ATA188 Phase 1a study in progressive multiple sclerosis in Q2. Such momentum in developing innovative off-the-shelf allogeneic T-cell immunotherapies has also supported our ability to attract top talent including a non-executive board chair and general counsel."

Recent Highlights and Anticipated Upcoming Milestones

Operational

COVID-19 Response and Actions

Atara continues to deliver product to patients from our inventory of off-the-shelf, allogeneic tab-cel and ATA188.

Prior to the COVID-19 outbreak, as part of our routine supply planning and operational risk management strategies, the Company had already manufactured significant inventories of tab-cel and process intermediates and procured the required starting materials needed to maintain long-term product supply across tab-cel, ATA188 and other programs.

The Atara clinical study and operational teams have been working closely with sites to ensure the safety of site staff and patients as well as preserve data integrity and access to treatment as appropriate. Where needed, they have established remote study visits, leveraged tele-medicine, home health care, and other methods to ensure continuity of care for patients while preserving key endpoint data.

Atara is closely monitoring the evolving COVID-19 pandemic and continues to assess potential impact on the business and operations, including the timing and execution of clinical and preclinical studies.

Board and Executive Appointments

Atara recently announced the appointment of a Chair of the Board and executive with extensive leadership and management experience in the life sciences industry:

Atara appointed Ron Renaud as Chairman of the Board of Directors. Mr. Renaud has served as Translate Bio’s chief executive officer since 2014 and brings deep and broad experience in strategic and corporate development, partnering, financing, and industry and Wall Street relationships. He has significant prior board experience having served on the boards of both public and private companies.

K. Amar Murugan was named Senior Vice President, General Counsel. Mr. Murugan brings significant expertise in M&A, corporate finance, securities, life science transactions and corporate governance. Mr. Murugan was most recently Senior Vice President and General Counsel of Assertio Therapeutics.

Tab-cel (tabelecleucel)

Atara continues to progress tab-cel Phase 3 development for patients with EBV-associated post-transplant lymphoproliferative disease (EBV+ PTLD).

Atara remains on track to initiate a tab-cel BLA submission for patients with EBV+ PTLD in the second half of 2020.

Atara plans to discuss the totality of tab-cel data with the U.S. Food and Drug Administration (FDA) in a pre-BLA meeting prior to initiating the BLA submission.

In the U.S. and Australia, most of the current 40 clinical study sites are available for enrollment and the Company is preparing to open additional sites in the U.S., Canada and Europe.

The Company’s clinical trial applications (CTAs) in the United Kingdom, Spain, France, and Austria were recently approved, the first European clinical site has opened for enrollment, and we expect to open additional European Phase 3 clinical sites in 2020.

Atara has submitted a Pediatric Investigation Plan (PIP) to the European Medicines Agency (EMA).

Following EMA approval of the PIP, Atara plans to submit a tab-cel EU marketing authorization application (MAA) for patients with EBV+ PTLD in 2021.

Atara continues to see strong tab-cel investigator, physician and patient interest and, for cases in which the Company is not able to enroll patients in its EBV+ PTLD Phase 3 clinical study, Atara is providing tab-cel to patients in need under its expanded access protocol (EAP) and single patient use (SPU) programs.

Studies supporting potential additional tab-cel indications are also advancing.

Based on clinical data from treating a variety of ultra-rare EBV+ diseases, Atara expects to initiate enrollment in the second half of 2020 in a tab-cel Phase 2 multi-cohort study including up to six additional ultra-rare EBV+ patient populations.

ATA188 for Progressive Multiple Sclerosis (MS)

A Phase 1a clinical study of off-the-shelf, allogeneic ATA188 in patients with progressive MS is ongoing across clinical sites in the U.S. and Australia.

Atara expects to present ATA188 Phase 1a six-month clinical results for the dose-escalating cohorts 1-4 and 12-month results for cohorts 1-3 in an appropriate forum in the second quarter of 2020 and expects to present 12-month cohort 4 data in the second half of 2020.

Atara is re-treating patients in the open-label extension (OLE) of the Phase 1a study in an appropriate setting and as determined by the treating physician and patient.

Atara has temporarily paused the screening and enrollment of patients in the Phase 1b randomized placebo-controlled study to ensure sites can focus on meeting the needs of patients with COVID-19 and to protect the safety of study participants, investigators and staff.

This action will help to preserve study and data integrity as there are numerous assessments that require a specific clinical setting.

Atara expects this pause to be limited and plans to initiate enrollment in this study in the second or third quarter of 2020.

ATA2271/ATA3271 and ATA3219 CAR T Programs

Atara is developing a next generation mesothelin-targeted autologous CAR T immunotherapy (ATA2271) and expects collaborators at Memorial Sloan Kettering Cancer Center (MSK) to submit an Investigational New Drug (IND) application to the FDA for patients with advanced mesothelioma in the second or third quarter of 2020.

ATA2271 is designed to improve efficacy, persistence, and durability of response using a novel 1XX CAR co-stimulatory domain and cell intrinsic checkpoint inhibition technology with a PD-1 dominant negative receptor (DNR).

Data from IND-enabling studies for ATA2271 have been accepted as a late-breaking e-poster at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Virtual Annual Meeting II in June, and the abstract will be released on May 15.

Additional clinical data for the first-generation academic program from MSK are expected to be presented in an upcoming forum in the second half of this year.

Atara is also developing off-the-shelf, allogeneic CAR T immunotherapies targeting mesothelin (ATA3271) and CD19 (ATA3219) using its next-generation technologies and EBV T-cell platform.

The Company has initiated preclinical IND-enabling studies for ATA3271 and ATA3219.

First Quarter 2020 Financial Results

Atara believes that its cash, cash equivalents and short-term investments as of March 31, 2020, are sufficient to fund planned operations into the second quarter of 2021.

Cash, cash equivalents and short-term investments as of March 31, 2020 totaled $214.6 million, as compared to $259.1 million as of December 31, 2019.

The decrease of $44.5 million includes the benefit of the sale of 1,528,216 shares of common stock pursuant to the Company’s at-the-market (ATM) facility in the first quarter of 2020 for net proceeds of $23.1 million.

Net cash used in operating activities was $67.0 million for the first quarter of 2020, as compared to $70.2 million for the same period in 2019.

The number of outstanding shares of common stock and pre-funded common stock warrants as of April 30, 2020 was 58,952,045 shares and 2,888,526 warrants, respectively.

Atara reported net losses of $73.5 million, or $1.20 per share, for the first quarter of 2020 as compared to $66.3 million, or $1.44 per share, for the same period in 2019.

Total operating expenses include non-cash expenses of $14.5 million for the first quarter 2020, as compared to $13.9 million for the same period in 2019.

Research and development expenses were $57.7 million for the first quarter of 2020, as compared to $48.7 million for the same period in 2019. The increase in the first quarter of 2020 was due to costs associated with the Company’s continuing expansion of research and development activities, including:

Clinical study, manufacturing and process performance qualification activities related to tab-cel.

Higher employee-related costs from increased headcount.

Increased facilities and information technology expenses allocated to our research and development function.

Research and development expenses include $7.7 million of non-cash stock-based compensation expenses for the first quarter of 2020, as compared to $6.1 million for the same period in 2019.

General and administrative expenses were $17.0 million for the first quarter of 2020, as compared to $19.2 million for the same period in 2019. The decrease in the first quarter 2020 was primarily due to a decrease in outside services costs and non-cash stock-based compensation expenses, partially offset by an increase in payroll-related costs driven by increased headcount.

General and administrative expenses include $5.0 million of non-cash stock-based compensation expenses for the first quarter of 2020, as compared to $6.2 million for the same period in 2019.

On May 6, 2020 OPKO Health, Inc. (NASDAQ: OPK) reported business highlights and financial results for the three months ended March 31, 2020 (Press release, Opko Health, MAY 6, 2020, View Source [SID1234557138]).

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Business Highlights

BioReference Laboratories launched COVID-19 testing nationwide: On March 13, 2020, BioReference Laboratories launched a Real-Time Reverse Transcription Polymerase Chain Reaction assay with an approximate 24-72 hour turnaround time to promote earlier diagnosis of the SARS-CoV-2 virus to aid in limiting the spread of infection. In addition to its nationwide COVID-19 test offering, BioReference announced testing partnerships with the New York State Department of Health, New York City Health and Hospital Corporation, the State of New Jersey, the State of Florida and the cities of Detroit and Miami, among others. Additionally, Rite Aid Corporation selected BioReference to provide COVID-19 laboratory testing to its drive-up locations with the goal of flattening the curve through widely accessible diagnostic testing. To date, BioReference has run approximately 700,000 COVID-19 tests and currently has a COVID-19 testing capacity of 35,000 tests per day. As of April 27, 2020, BioReference Laboratories started offering COVID-19 antibody testing, a semi-quantitative immunoassay that measures SARS-CoV-2 specific IgG antibody levels, correlating with the patient’s immune response after COVID-19 infection. Within the next two weeks, BioReference expects to expand its capacity to be able to process up to 400,000 tests per day.

Somatrogon abstract to be presented at Endocrine Society’s ENDO Online 2020: On April 22, 2020, OPKO announced the data from its two abstracts regarding the global Phase 3 pediatric trial evaluating somatrogon dosed once weekly in pre-pubertal children with growth hormone deficiency will be combined into a single presentation at ENDO Online 2020, a virtual event being held June 8 through 22 featuring on-demand and live programming. The results of the pivotal Phase 3 study will be delivered on June 8, 2020 at 11:00 a.m. Eastern time by Dr. Cheri Deal, the Principal Investigator of the pediatric study. The two abstracts entitled "Somatrogon Growth Hormone in the Treatment of Pediatric Growth Hormone Deficiency: Results of the Pivotal Phase 3" and "Interpretation of Insulin-like Growth Factor (IGF-1) Levels Following Administration of Somatrogon (a long-acting Growth Hormone-hGH-CTP)" will be published online in the April-May supplemental issue of the Journal of Endocrine Society.

Somatrogon global regulatory submissions: Somatrogon regulatory submission in the U.S. is anticipated to occur in the second half of 2020. In Europe, regulatory submission will follow completion of the open-label study demonstrating benefit and compliance with reduced treatment burden, which is expected to be completed in the third quarter of 2020. The registration study in Japan for pediatric growth hormone deficiency patients was completed in early March and topline data is expected in mid-2020.

RAYALDEE total prescriptions reported by IQVIA increased 78% in the first quarter of 2020 compared with the first quarter in 2019: Total prescriptions for the three months ended March 31, 2020 increased to approximately 18,327, compared with approximately 10,307 during the comparable period of 2019.

Interim results from two ongoing RAYALDEE studies reported: On March 25, 2020, OPKO announced positive preliminary data from the ongoing Phase 4 comparative trial, which suggest that RAYALDEE may be more effective in raising serum total 25-hydroxyvitamin D to the level required to effectively suppress elevated plasma intact parathyroid hormone (iPTH) in stage 3 or 4 chronic kidney patients. Final results are expected in the second half of 2020. In addition, OPKO announced positive proof-of-concept data from the ongoing Phase 2 trial in patients with stage 5 chronic kidney disease demonstrating RAYALDEE may be useful in treating secondary hyperparathyroidism in dialysis patients. The Phase 2 trial in hemodialysis patients is on track to complete enrollment in the third quarter of 2020 with full topline data expected in the first quarter of 2021.

Nearly 16,000 4Kscore tests performed during the first quarter of 2020: Novitas Solutions issued its final Local Coverage Determination for Medicare payments for the 4Kscore test with defined coverage criteria, effective December 30, 2019. With Medicare reimbursement in place, the Company began its salesforce expansion for the 4Kscore during the first quarter of 2020; however, COVID-19 impacted utilization during the month of March.

Financial Results

Consolidated revenues for the first quarter of 2020 were $211.5 million compared with $222.5 million for the comparable period of 2019. The net loss for the first quarter of 2020 was $59.1 million, or $0.09 per share, compared with a net loss of $80.8 million, or $0.14 per share, for the comparable period of 2019.

Diagnostics: Revenue from services in the first quarter of 2020 was $170.8 million compared with $178.9 million for the comparable period in 2019. Although revenue from services was positively affected by increased reimbursement amounts and improved operational procedures, in the last two weeks of March 2020, the Company experienced a decline in testing volumes net of COVID-19 testing services due to the COVID-19 pandemic. Total costs and expenses were $189.0 million in the first quarter of 2020 compared with $212.5 million for the comparable period in 2019 with the reduction primarily attributable to lower selling, general and administrative expenses due to cost-reduction initiatives. In addition, the 2019 period included a $10.6 million legal accrual. As a result, operating loss was $18.1 million in the first quarter of 2020 compared with $33.6 million in the first quarter of 2019.

Pharmaceuticals: Revenue from products in the first quarter of 2020 was $31.1 million compared with $25.3 million in the first quarter of 2019 with the increase primarily attributable to higher sales of RAYALDEE of $9.9 million in the first quarter of 2020 compared with $5.8 million in the prior year period. Revenue from licensing and intellectual property was $9.6 million in the first quarter of 2020 compared to $18.3 million in the first quarter of 2019 with the reduction primarily due to a decrease in the amortization of payments received from Pfizer, OPKO’s commercial partner for its long-acting human growth hormone product, Somatrogon. Total cost and expenses were $54.8 million in the first quarter of 2020 compared with $73.0 million for the prior year period, with the decline primarily attributable to lower research and development expenses due to the completion of the pediatric Phase 3 study for somatrogon. The operating loss was $14.1 million in the first quarter of 2020 compared to $29.5 million in the first quarter of 2019.

Cash and equivalents: Cash, cash equivalents and marketable securities were $34.5 million as of March 31, 2020. In addition, the Company has an unutilized $100 million credit facility which provides the company with access to incremental capital on a non-dilutive basis. In April 2020, the Company also accessed additional capital available under the CARES Act which provided the company with approximately $30 million of short-term liquidity through various provisions under the Act.

CONFERENCE CALL & WEBCAST INFORMATION

OPKO’s senior management will provide a business update and discuss results in greater detail in a conference call and live audio webcast at 4:30 p.m. Eastern time today, May 6, 2020. The conference call dial-in and webcast information is as follows:
DOMESTIC DIAL-IN:

(877) 783-8475
INTERNATIONAL DIAL-IN:

(614) 999-1827
PASSCODE:

9095275
WEBCAST:

OPKO 1Q20 Results Conference Call
For those unable to participate in the live conference call or webcast, a replay will be available beginning approximately two hours after the close of the conference call. To access the replay, dial 855-859-2056 or 404-537-3406. The replay passcode is 9095275. The replay can be accessed for a period of time on OPKO’s website at OPKO 1Q20 Results Conference Call.

On May 6, 2020 OPKO Health, Inc. (NASDAQ: OPK) reported that business highlights and financial results for the three months ended March 31, 2020 (Press release, Opko Health, MAY 6, 2020, View Source [SID1234557138]).

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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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Business Highlights

BioReference Laboratories launched COVID-19 testing nationwide: On March 13, 2020, BioReference Laboratories launched a Real-Time Reverse Transcription Polymerase Chain Reaction assay with an approximate 24-72 hour turnaround time to promote earlier diagnosis of the SARS-CoV-2 virus to aid in limiting the spread of infection. In addition to its nationwide COVID-19 test offering, BioReference announced testing partnerships with the New York State Department of Health, New York City Health and Hospital Corporation, the State of New Jersey, the State of Florida and the cities of Detroit and Miami, among others. Additionally, Rite Aid Corporation selected BioReference to provide COVID-19 laboratory testing to its drive-up locations with the goal of flattening the curve through widely accessible diagnostic testing. To date, BioReference has run approximately 700,000 COVID-19 tests and currently has a COVID-19 testing capacity of 35,000 tests per day. As of April 27, 2020, BioReference Laboratories started offering COVID-19 antibody testing, a semi-quantitative immunoassay that measures SARS-CoV-2 specific IgG antibody levels, correlating with the patient’s immune response after COVID-19 infection. Within the next two weeks, BioReference expects to expand its capacity to be able to process up to 400,000 tests per day.

Somatrogon abstract to be presented at Endocrine Society’s ENDO Online 2020: On April 22, 2020, OPKO announced the data from its two abstracts regarding the global Phase 3 pediatric trial evaluating somatrogon dosed once weekly in pre-pubertal children with growth hormone deficiency will be combined into a single presentation at ENDO Online 2020, a virtual event being held June 8 through 22 featuring on-demand and live programming. The results of the pivotal Phase 3 study will be delivered on June 8, 2020 at 11:00 a.m. Eastern time by Dr. Cheri Deal, the Principal Investigator of the pediatric study. The two abstracts entitled "Somatrogon Growth Hormone in the Treatment of Pediatric Growth Hormone Deficiency: Results of the Pivotal Phase 3" and "Interpretation of Insulin-like Growth Factor (IGF-1) Levels Following Administration of Somatrogon (a long-acting Growth Hormone-hGH-CTP)" will be published online in the April-May supplemental issue of the Journal of Endocrine Society.

Somatrogon global regulatory submissions: Somatrogon regulatory submission in the U.S. is anticipated to occur in the second half of 2020. In Europe, regulatory submission will follow completion of the open-label study demonstrating benefit and compliance with reduced treatment burden, which is expected to be completed in the third quarter of 2020. The registration study in Japan for pediatric growth hormone deficiency patients was completed in early March and topline data is expected in mid-2020.

RAYALDEE total prescriptions reported by IQVIA increased 78% in the first quarter of 2020 compared with the first quarter in 2019: Total prescriptions for the three months ended March 31, 2020 increased to approximately 18,327, compared with approximately 10,307 during the comparable period of 2019.

Interim results from two ongoing RAYALDEE studies reported: On March 25, 2020, OPKO announced positive preliminary data from the ongoing Phase 4 comparative trial, which suggest that RAYALDEE may be more effective in raising serum total 25-hydroxyvitamin D to the level required to effectively suppress elevated plasma intact parathyroid hormone (iPTH) in stage 3 or 4 chronic kidney patients. Final results are expected in the second half of 2020. In addition, OPKO announced positive proof-of-concept data from the ongoing Phase 2 trial in patients with stage 5 chronic kidney disease demonstrating RAYALDEE may be useful in treating secondary hyperparathyroidism in dialysis patients. The Phase 2 trial in hemodialysis patients is on track to complete enrollment in the third quarter of 2020 with full topline data expected in the first quarter of 2021.

Nearly 16,000 4Kscore tests performed during the first quarter of 2020: Novitas Solutions issued its final Local Coverage Determination for Medicare payments for the 4Kscore test with defined coverage criteria, effective December 30, 2019. With Medicare reimbursement in place, the Company began its salesforce expansion for the 4Kscore during the first quarter of 2020; however, COVID-19 impacted utilization during the month of March.

Financial Results

Consolidated revenues for the first quarter of 2020 were $211.5 million compared with $222.5 million for the comparable period of 2019. The net loss for the first quarter of 2020 was $59.1 million, or $0.09 per share, compared with a net loss of $80.8 million, or $0.14 per share, for the comparable period of 2019.

Diagnostics: Revenue from services in the first quarter of 2020 was $170.8 million compared with $178.9 million for the comparable period in 2019. Although revenue from services was positively affected by increased reimbursement amounts and improved operational procedures, in the last two weeks of March 2020, the Company experienced a decline in testing volumes net of COVID-19 testing services due to the COVID-19 pandemic. Total costs and expenses were $189.0 million in the first quarter of 2020 compared with $212.5 million for the comparable period in 2019 with the reduction primarily attributable to lower selling, general and administrative expenses due to cost-reduction initiatives. In addition, the 2019 period included a $10.6 million legal accrual. As a result, operating loss was $18.1 million in the first quarter of 2020 compared with $33.6 million in the first quarter of 2019.

Pharmaceuticals: Revenue from products in the first quarter of 2020 was $31.1 million compared with $25.3 million in the first quarter of 2019 with the increase primarily attributable to higher sales of RAYALDEE of $9.9 million in the first quarter of 2020 compared with $5.8 million in the prior year period. Revenue from licensing and intellectual property was $9.6 million in the first quarter of 2020 compared to $18.3 million in the first quarter of 2019 with the reduction primarily due to a decrease in the amortization of payments received from Pfizer, OPKO’s commercial partner for its long-acting human growth hormone product, Somatrogon. Total cost and expenses were $54.8 million in the first quarter of 2020 compared with $73.0 million for the prior year period, with the decline primarily attributable to lower research and development expenses due to the completion of the pediatric Phase 3 study for somatrogon. The operating loss was $14.1 million in the first quarter of 2020 compared to $29.5 million in the first quarter of 2019.

Cash and equivalents: Cash, cash equivalents and marketable securities were $34.5 million as of March 31, 2020. In addition, the Company has an unutilized $100 million credit facility which provides the company with access to incremental capital on a non-dilutive basis. In April 2020, the Company also accessed additional capital available under the CARES Act which provided the company with approximately $30 million of short-term liquidity through various provisions under the Act.

CONFERENCE CALL & WEBCAST INFORMATION

OPKO’s senior management will provide a business update and discuss results in greater detail in a conference call and live audio webcast at 4:30 p.m. Eastern time today, May 6, 2020. The conference call dial-in and webcast information is as follows:
DOMESTIC DIAL-IN:

(877) 783-8475
INTERNATIONAL DIAL-IN:

(614) 999-1827
PASSCODE:

9095275
WEBCAST:

OPKO 1Q20 Results Conference Call
For those unable to participate in the live conference call or webcast, a replay will be available beginning approximately two hours after the close of the conference call. To access the replay, dial 855-859-2056 or 404-537-3406. The replay passcode is 9095275. The replay can be accessed for a period of time on OPKO’s website at OPKO 1Q20 Results Conference Call.

On May 6, 2020 AnaptysBio, Inc. (Nasdaq: ANAB), a clinical-stage biotechnology company developing first-in-class antibody product candidates focused on emerging immune control mechanisms applicable to inflammation and immuno-oncology indications, reported operating results for the first quarter ended March 31, 2020 and provided pipeline updates (Press release, AnaptysBio, MAY 6, 2020, View Source [SID1234557137]).

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"Our wholly-owned pipeline continues to advance with three Phase 2 clinical trial readouts anticipated in the remainder of 2020," said Hamza Suria, president and chief executive officer of AnaptysBio. "AnaptysBio’s capital-efficient business model has resulted in the advancement of 7 internally-generated antibodies to the clinic to date, and we anticipate continued discovery and development of novel antibodies in the upcoming future. We also look forward to significant revenues associated with regulatory filing related milestone payment and future royalties upon FDA approval of dostarlimab under our GSK partnership later this year."

Etokimab (ANB020 Anti-IL-33) Program

AnaptysBio is conducting a randomized, placebo-controlled Phase 2 trial in 100 adult patients with chronic rhinosinusitis with nasal polyps, also referred to as the ECLIPSE trial. Patients are being treated with two multi-dosing frequencies of subcutaneously-administered etokimab or placebo, each in combination with mometasone furoate nasal spray as background therapy. We have over-enrolled this trial beyond the first 100 patients to compensate for lost patient visits due to the COVID-19 pandemic, and hence anticipate interim top-line data from this trial to be available in the third quarter of 2020.

As previously announced, the Company has decided to postpone the initiation of its planned Phase 2b etokimab clinical trial in eosinophilic asthma, a multi-dose, randomized, double-blinded, placebo-controlled trial in 300-400 patients, until results are available from the ECLIPSE trial.

ANB019 (Anti-IL-36 Receptor) Program

In September, AnaptysBio announced positive topline data from an interim analysis of its Phase 2 clinical trial of ANB019 monotherapy in moderate-to-severe generalized pustular psoriasis, or GPP, also known as the GALLOP trial. In this interim analysis, both patients achieved the primary endpoint of disease score improvement at Day 29 and Day 113 without requiring rescue therapy, demonstrated rapid and sustained mJDA score improvement, with reduction of 58% at Day 8 and 63% at Day 113, and showed complete clearance of skin pustules by Day 8 and through Day 113, with CRP levels decreasing to nearly normal. The Company anticipates additional clinical data and a regulatory strategy update for the development of ANB019 in GPP during the second half of 2020.

The Company is also conducting a randomized, placebo-controlled, multi-dose Phase 2 trial in 50 patients with palmoplantar pustulosis, or PPP, also known as the POPLAR trial, with topline data anticipated in the second half of 2020.

Enrollment of the GALLOP and POPLAR trials has been enhanced by expanding clinical sites and geographies involved. While the Company does not believe that the aforementioned timelines are materially impacted by the COVID-19 pandemic at this point, some of the sites involved in the GALLOP and POPLAR trials have been affected by the COVID-19 pandemic, and the Company will continue to monitor the situation over the upcoming months.
ANB030 (Anti-PD-1 Agonist) Program

ANB030 is a wholly-owned antibody that binds PD-1 in an agonistic manner, leading to reduced T cell activity and anti-inflammatory effects in vivo. Genetic mutations in the PD-1 pathway are associated with increased susceptibility to various inflammatory conditions and we believe ANB030 has the potential to suppress inflammatory diseases by restoring insufficient PD-1-mediated negative signaling on activated T cells. The Company plans to focus future clinical development of ANB030 on certain autoimmune diseases where PD-1 checkpoint receptor function may be under-represented. Our Investigational New Drug Application (IND) for ANB030 has been cleared by the FDA and we plan to initiate a Phase 1 clinical trial in the first half of 2020. Preclinical data from the ANB030 was presented in June at the 2019 FOCIS Annual Meeting.
ANB032 (Anti-BTLA Modulator) Program

Our fourth wholly-owned program is an anti-BTLA modulator antibody, known as ANB032, which is broadly applicable to human inflammatory diseases associated with lymphoid and myeloid immune cell dysregulation. Mutations in the BTLA signaling pathway are associated with human inflammatory disease and we believe ANB032 silences pro-inflammatory signaling by modulating BTLA binding to HVEM. We anticipate filing an IND for ANB032 in the second half of 2020.
Dostarlimab (Anti-PD-1 Antagonist) Program Partnered with GSK

In the first quarter of 2020, the FDA accepted the first Biologics License Application (BLA) filing for dostarlimab, an AnaptysBio-generated PD-1 antagonist antibody under partnership with GSK, for the treatment of endometrial cancer. AnaptysBio received a $10.0 million cash milestone payment upon this acceptance, and anticipates an additional $20.0 million cash milestone upon first FDA approval of dostarlimab during 2020. Also in the first quarter of 2020, the EMA accepted GSK’s Marketing Authorization Application (MAA) for approval of dostarlimab in the EU for endometrial cancer, for which AnaptysBio has earned a $5.0 million milestone payment. Including additional cash milestones due upon future development and commercialization of dostarlimab, TSR-022, an AnaptysBio-generated TIM-3 antibody, and TSR-033, an AnaptysBio-generated LAG-3 antibody, AnaptysBio can potentially receive a total of $1.1 billion in aggregate milestone payments under this GSK partnership. In addition, AnaptysBio is due a 4% to 8% royalty from GSK, tiered upon global sales, for each of the aforementioned programs.
First Quarter Financial Results

Cash, cash equivalents and investments totaled $412.7 million as of March 31, 2020 compared to $428.5 million as of December 31, 2019, for a decrease of $15.8 million. The decrease relates primarily to cash used for operating activities.

Collaboration revenue was $15.0 million for the three months ended March 31, 2020, which related to milestone payments for successful BLA and MAA filings for dostarlimab, compared to zero for the three months ended March 31, 2019.

Research and development expenses were $21.0 for the three months ended March 31, 2020, compared to $20.6 million for the three months ended March 31, 2019. The increase was due primarily to continued advancement of the Company’s preclinical programs.

General and administrative expenses were $4.3 million for the three months ended March 31, 2020, compared to $4.1 million for the three months ended March 31, 2019. The increase was due primarily to personnel-related expenses, including share-based compensation.

Net loss was $8.3 million for the three months ended March 31, 2020, or a net loss per share of $0.30, compared to a net loss of $22.1 million for the three months ended March 31, 2019, or a net loss per share of $0.82.
Financial Guidance
AnaptysBio expects its net cash burn in 2020 will be approximately $60.0 million, and that its cash, cash equivalents and investments will fund its current operating plan at least into 2023.

On May 6, 2020 Neurocrine Biosciences, Inc. (Nasdaq: NBIX) reported its financial results for the first quarter ended March 31, 2020 and provided revised full-year 2020 financial expense guidance (Press release, Neurocrine Biosciences, MAY 6, 2020, View Source [SID1234557136]).

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"First and foremost, I would like to thank our employees, healthcare providers and suppliers for their commitment and dedication in managing these tough times. Together, we remain focused on ensuring that patients with tardive dyskinesia, many of whom are managing their involuntary movements with a mental health condition, are well supported and have uninterrupted access to INGREZZA," said Kevin Gorman, Ph.D., Chief Executive Officer of Neurocrine Biosciences. "Even during these challenging times, we continue to see strong persistency and compliance with INGREZZA."

"With the recent FDA approval of ONGENTYS for patients with Parkinson’s disease, we’re excited to add another treatment for movement disorders to our portfolio and look forward to making it available to patients later this year. In addition, we are well positioned to launch clinical studies of several compounds in our pipeline and plan to have on-going studies in three registrational programs and four mid-stage programs by the end of this year," Gorman added. "The company is in a strong financial position, enabling us to navigate through this pandemic while continuing to execute on our mission to discover, develop and deliver important medicines to treat patients with neurological, endocrine, and psychiatric disorders."

First Quarter Net Product Sales Highlights:

INGREZZA net product sales for the first quarter 2020 were $231 million, representing a year-over-year increase of 69%.

Successfully navigated through the annual seasonal payor dynamics driving steady persistency and strong momentum in the second half of the first quarter.

End of first quarter 2020 days-on-hand channel inventory slightly increased relative to the fourth quarter 2019 resulting in an approximate $4 million benefit to net product sales.

Financial Highlights:

Research and Development (R&D) investment increased in the first quarter of 2020 versus the first quarter of 2019 primarily due to the Company’s increased investment in its gene therapy programs in collaboration with Voyager Therapeutics and increased activity to support advancing its expanded clinical portfolio.

Selling, General and Administrative (SG&A) investment increased in the first quarter of 2020 versus the first quarter of 2019, primarily due to increased headcount costs and continued investment in the patient-focused disease state awareness campaign, "Talk About TD".

First quarter of 2020 GAAP net income and diluted earnings per share were $37 million and $0.39, respectively, compared to a net loss and net loss per share of $102 million and $1.12, respectively, in the first quarter of 2019, largely attributable to IPR&D expense of $113.1 million.

First quarter of 2020 non-GAAP net income and diluted earnings per share were $79 million and $0.82, respectively, compared to $28 million and $0.29, respectively, in the first quarter of 2019.

At March 31, 2020, the Company had cash, cash equivalents and debt securities available-for-sale over $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

On April 3, 2020, the Company provided an update on its business operations as a result of the COVID-19 pandemic as part of its commitment to prioritize the safety, health and well-being of patients, their caregivers, healthcare providers and employees. Please refer to the press release for additional information pertaining to the status of our programs and ongoing company initiatives through the COVID-19 crisis. Key updates included:

Employees, including customer-facing teams, have been working from home since March and have been utilizing remote technologies to ensure continued support for patients and healthcare professionals.

Patients taking INGREZZA have assurance of continued supply, and specialty and mental health pharmacies remain engaged with patients to ensure they receive continuity of care and support their overall health and well-being.

Enrollment has been paused for clinical studies and will be reinstated when it is safe and appropriate to do so. In parallel, the Company continues preparations to launch new clinical studies in the second half of the year. By the end of 2020, Neurocrine Biosciences plans to have studies ongoing in three registrational and four mid-stage programs.

On April 24, 2020, the FDA approved 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. The FDA approval of ONGENTYS for Parkinson’s disease triggered a $20 million milestone under the terms of the collaboration agreement with BIAL. The commercial launch of ONGENTYS is expected to occur later in 2020.

Previously, the Company expected combined GAAP R&D and SG&A expenses in the range of $740 million to $770 million and combined non-GAAP R&D and SG&A expenses in the range of $620 million to $650 million.

GAAP and non-GAAP expense guidance range reflects increased investment in R&D programs including three registrational programs, meaningful investments across early stage programs including Voyager and Xenon collaborations, continued investment in INGREZZA and marketing costs associated with the anticipated launch of ONGENTYS.

GAAP-only guidance includes approximately $105 million of share-based compensation and a $20 million milestone to BIAL connected with the approval of ONGENTYS by the FDA. GAAP-only guidance does not include any other 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 800-894-5910 (US) or 785-424-1052 (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.

About INGREZZA (valbenazine) Capsules

INGREZZA, a selective vesicular monoamine transporter 2 (VMAT2) inhibitor, is the first FDA-approved product indicated for the treatment of adults with tardive dyskinesia, a condition associated with uncontrollable, abnormal and repetitive movements of the face, torso, and/or other body parts.

INGREZZA is thought to work by reducing the amount of dopamine released in a region of the brain that controls movement and motor function, helping to regulate nerve signaling in adults with tardive dyskinesia. VMAT2 is a protein in the brain that packages neurotransmitters, such as dopamine, for transport and release from presynaptic neurons. INGREZZA, developed in Neurocrine’s laboratories, is novel in that it selectively inhibits VMAT2 with no appreciable binding affinity for VMAT1, dopaminergic (including D2), serotonergic, adrenergic, histaminergic, or muscarinic receptors. Additionally, INGREZZA can be taken for the treatment of tardive dyskinesia as one capsule, once-daily, together with psychiatric medications such as antipsychotics or antidepressants.

Important Safety Information

Contraindications

INGREZZA is contraindicated in patients with a history of hypersensitivity to valbenazine or any components of INGREZZA. Rash, urticaria, and reactions consistent with angioedema (e.g., swelling of the face, lips, and mouth) have been reported.

Warnings & Precautions

Somnolence

INGREZZA can cause somnolence. Patients should not perform activities requiring mental alertness such as operating a motor vehicle or operating hazardous machinery until they know how they will be affected by INGREZZA.

QT Prolongation

INGREZZA may prolong the QT interval, although the degree of QT prolongation is not clinically significant at concentrations expected with recommended dosing. INGREZZA should be avoided in patients with congenital long QT syndrome or with arrhythmias associated with a prolonged QT interval. For patients at increased risk of a prolonged QT interval, assess the QT interval before increasing the dosage.

Parkinsonism

INGREZZA may cause Parkinsonism in patients with tardive dyskinesia. Parkinsonism has also been observed with other VMAT2 inhibitors. Reduce the dose or discontinue INGREZZA treatment in patients who develop clinically significant parkinson-like signs or symptoms.

Adverse Reactions

The most common adverse reaction (≥5% and twice the rate of placebo) is somnolence. Other adverse reactions (≥2% and >placebo) include: anticholinergic effects, balance disorders/falls, headache, akathisia, vomiting, nausea, and arthralgia.

Please see INGREZZA full Prescribing Information at www.INGREZZA.com/PI.

About ONGENTYS (opicapone) Capsules

ONGENTYS is a novel, once-daily, oral, peripheral, selective and reversible catechol-O-methyltransferase (COMT) inhibitor approved by the U.S. Food and Drug Administration (FDA) as an add-on treatment to levodopa/carbidopa in patients with Parkinson’s disease experiencing "off" episodes. ONGENTYS inhibits the COMT enzyme, which breaks down levodopa, making more levodopa available to reach the brain.

Important Safety Information

Contraindications

ONGENTYS is contraindicated in patients with:

Concomitant use of non-selective monoamine oxidase (MAO) inhibitors.

Pheochromocytoma, paraganglioma, or other catecholamine secreting neoplasms.

Warnings & Precautions

Cardiovascular Effects with Concomitant Use of Drugs Metabolized by Catechol-O-Methyltransferase (COMT)

Possible arrhythmias, increased heart rate, and excessive changes in blood pressure may occur with concomitant use of ONGENTYS and drugs metabolized by COMT, regardless of the route of administration (including inhalation). Monitor patients treated concomitantly with ONGENTYS and drugs metabolized by COMT.

Falling Asleep During Activities of Daily Living and Somnolence

Patients treated with dopaminergic medications and medications that increase levodopa exposure, including ONGENTYS, have reported falling asleep while engaged in activities of daily living, including the operation of motor vehicles, which sometimes has resulted in accidents. If a patient develops daytime sleepiness or somnolence, consider discontinuing ONGENTYS or adjusting other dopaminergic or sedating medications and advise patients to avoid driving and other potentially dangerous activities.

Hypotension/Syncope

Monitor patients for hypotension and advise patients about the risk for syncope. If these adverse reactions occur, consider discontinuing ONGENTYS or adjusting the dosage of other medications that can lower blood pressure.

Dyskinesia

ONGENTYS potentiates the effects of levodopa which may result in dyskinesia or exacerbate pre-existing dyskinesia. Reducing the patient’s levodopa dosage or the dosage of another dopaminergic drug may reduce dyskinesia that occurs during treatment with ONGENTYS.

Hallucinations and Psychosis

Consider stopping ONGENTYS if hallucinations or psychotic-like behaviors occur. Patients with a major psychotic disorder should ordinarily not be treated with ONGENTYS.

Impulse Control/Compulsive Disorders

Patients may experience intense urges (e.g., gambling, sexual, spending money, binge eating) and the inability to control them. It is important for prescribers to specifically ask patients or their caregivers about the development of new or increased urges. Re-evaluate the patient’s current therapies for Parkinson’s disease and consider stopping ONGENTYS if a patient develops such urges while taking ONGENTYS.

Withdrawal-Emergent Hyperpyrexia and Confusion

A symptom complex resembling neuroleptic malignant syndrome (elevated temperature, muscular rigidity, altered consciousness, and autonomic instability), has been reported in association with rapid dose reduction or withdrawal of drugs that increase central dopaminergic tone. There were no reports of neuroleptic malignant syndrome in ONGENTYS controlled clinical studies. When discontinuing ONGENTYS, monitor patients and consider adjustment of other dopaminergic therapies as needed.

Adverse Reactions

The most common adverse reactions (incidence at least 4% and greater than placebo) were dyskinesia, constipation, blood creatine kinase increase, hypotension/syncope, and weight decrease.

Please see ONGENTYS full Prescribing Information at www.neurocrine.com/ongentyspi.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit MedWatch at www.fda.gov/medwatch or call 1-800-FDA-1088.