On August 3, 2020 DURECT Corporation (Nasdaq: DRRX) reported financial results for the three months ended June 30, 2020 and provided a corporate update (Press release, DURECT, AUG 3, 2020, View Source [SID1234562703]).

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Total revenues were $25.8 million and net income was $14.3 million for the three months ended June 30, 2020 as compared to total revenues of $4.0 million and net loss of $7.2 million for the three months ended June 30, 2019. Revenues for the three months ended June 30, 2020 included the recognition of $23.1 million in deferred revenue from the $35 million upfront fee and initial milestone payment associated with the termination notice of our agreement with Gilead. This $23.1 million in revenue is non-recurring and has no cash flow impact.

At June 30, 2020, cash and investments were $51.3 million, compared to cash and investments of $64.8 million at December 31, 2019. Debt at June 30, 2020 was $20.5 million, compared to $20.3 million at December 31, 2019.

"We continued to make progress in our clinical programs investigating DUR-928, our lead epigenetic regulator, during the second quarter. We were pleased to report positive topline data from our Phase 1b NASH trial and we look forward to presenting additional data from the trial at a future scientific meeting," stated James E. Brown, D.V.M., President and CEO of DURECT. "We also initiated a Phase 2 trial of DUR-928 in COVID-19 patients with acute liver or kidney injury and are finalizing our preparations to initiate the Phase 2b trial with DUR-928 in alcoholic hepatitis in the upcoming months. The review of our New Drug Application (NDA) for POSIMIR is also ongoing and we continue to respond to the Agency’s requests for information."

Update on Selected Programs:

Epigenetic Regulator Program. DUR-928, the lead product candidate in the Company’s Epigenetic Regulator Program, is an endogenous, orally bioavailable, first-in-class small molecule, which may have broad applicability in acute organ injuries such as alcoholic hepatitis (AH) and coronavirus disease 2019 (COVID-19) patients with acute liver or kidney injury as well as in chronic liver diseases such as non-alcoholic steatohepatitis (NASH).

Clinical Development

Alcoholic Hepatitis (AH)

We are working with the FDA and our advisors to finalize the design of a multi-center, international, randomized, double-blind, placebo-controlled Phase 2b clinical trial of DUR-928 in severe AH patients. Patients in the trial will be randomized to receive 30 mg of DUR-928, 90 mg of DUR-928 or placebo. The primary endpoint will be survival rate for patients treated with DUR-928 compared to those treated with placebo. Further details of the trial design, including the size of the trial and other trial parameters will be provided at a future date. We expect to initiate this trial in the second half of 2020.

During 2019, we completed a Phase 2a clinical trial of DUR-928 in patients with AH. All 19 patients treated with DUR-928 survived the 28-day follow-up period, 74% of patients (14/19) were discharged in ≤ 4 days after receiving a single dose of DUR-928, and there were no drug-related serious adverse events. These results show the potential of DUR-928 as a life-saving therapy for AH patients which currently have an overall historical mortality rate of 26% at 28 days, according to an analysis of 77 studies published between 1971 and 2016, which included data from a total of 8,184 patients.

AH is an acute form of alcoholic liver disease (ALD) associated with long-term heavy intake of alcohol, and often occurs after a recent period of increased alcohol consumption. AH is typically characterized by recent onset jaundice and hepatic failure. According to the most recent data provided by the Agency for Healthcare Research and Quality (AHRQ), a part of the US Department of Health and Human Services (HHS), there were over 117,000 hospitalizations for patients with AH in 2016. From a recent publication analyzing the mortality and costs associated

with AH, the cost per patient is estimated at over $50,000 in the first year. ALD is one of the leading causes of liver transplants in the U.S., costing over $800,000 per patient.

Non-Alcoholic Steatohepatitis (NASH)

In May 2020, we reported positive topline results from a Phase 1b randomized and open-label clinical study conducted in the U.S. to evaluate safety, pharmacokinetics and signals of biological activity (including clinical chemistry and biomarkers as well as liver fat content and liver stiffness by imaging) of DUR-928 in NASH patients with stage 1-3 fibrosis. A total of 65 patients completed the study. DUR-928 was orally administered daily at 50 mg (n=23), 150 mg (n=21), or 600 mg (300 mg BID (n=21)). Patients in this trial were dosed daily for 4 weeks and followed up for an additional 4 weeks.

As shown in the table below, significant reductions from baseline (pre-treatment) levels were seen in liver enzymes, liver stiffness as measured by imaging and serum lipids. The Company believes that this demonstration of multiple important liver enzymes, plasma lipids and imaging results moving in the same direction, especially given the short treatment course of four weeks, is a promising indication of DUR-928’s potential in NASH.

DUR-928 was well tolerated at all three doses evaluated. There were no serious adverse events reported during the study. Pharmacokinetic (PK) parameters after repeat dosing were comparable to those after a single dose (from a prior study), indicating no accumulation after repeat dosing.

Results, including biomarker data, are still being analyzed. DURECT plans to present additional results and data analyses at a future scientific meeting.

Non-alcoholic fatty liver disease (NAFLD) is the most common form of chronic liver disease in both children and adults. It is estimated that NAFLD affects approximately 30% to 40% of adults and 10% of children in the United States. NASH, a more severe and progressive form of NAFLD, is one of the most common chronic liver diseases worldwide, with an estimated prevalence of 3-5% globally. No drug is currently approved for NAFLD or NASH.

COVID-19

We have begun to recruit patients for a randomized, double-blind, placebo-controlled, multi-center Phase 2 study to evaluate the safety and efficacy of DUR-928 in hospitalized COVID-19 infected patients with acute liver or kidney injury.

A total of approximately 80 patients are planned to be enrolled into two study treatment groups in a 3:1 (DUR-928: placebo) ratio. Patients will receive a dose of 150 mg of DUR-928 or placebo by intravenous infusion on day 1 and day 4 in combination with standard of care therapy, which will be determined by the principal investigator (PI) at each clinical trial site. The primary efficacy endpoint is a composite of survival and being free of acute organ failure (free of mechanical ventilation, free of liver failure events and free of renal replacement therapy) at day 28. Patients will be followed for 60 days. Should any drug product be determined by the FDA to be safe and effective for the treatment of COVID-19 while the trial is ongoing, such treatments may be offered, at each PI’s discretion, to any remaining and future patients in this trial.

COVID-19 is an infectious disease caused by severe acute respiratory syndrome coronavirus (SARS-COV-2). The rapid spread of the disease has resulted in a pandemic with millions of confirmed cases and hundreds of thousands of deaths worldwide. While most cases result in mild symptoms, including fever, cough and shortness of breath, some rapidly progress into acute respiratory distress syndrome (ARDS), multi-organ failure, and death. Many of these patients experience a rapid elevation of inflammation-inducing signaling molecules (cytokine storm) that trigger acute injuries in multiple organs including the liver and the kidney. Organ injury may also occur in hospitalized COVID-19 patients as the result of other complications of the viral infection. In a study of 1,059 adult cases of confirmed hospitalized COVID-19, 62% of patients presented with at least one elevated liver enzyme. In another study, 36.6% of 5,449 patients admitted with COVID-19 had or developed acute kidney injury (AKI).

The reasons for investigating DUR-928 in this patient population include:

DUR-928 has demonstrated, both in vitro and in vivo, its ability to stabilize mitochondria, modulate inflammatory responses, and promote cell survival and tissue regeneration, which may render it to be effective in preventing or treating acute organ injury.

Patients with severe COVID-19 can develop multi-organ injury, including acute kidney, liver and/or cardiac injury, in addition to lung injury and ARDS. Therefore, preventing or successfully treating acute organ injury by alleviating acute cell injury, regulating inflammation, promoting cell survival, and stimulating tissue regeneration could potentially save lives of those hospitalized patients with COVID-19.

Most relevant to COVID-19 patients with acute liver or kidney injury are results from the recently completed Phase 2a study in AH patients (see above). As in AH patients, COVID-19 patients, especially those with acute liver or kidney injury, are at risk of septic shock and eventually multi organ failure and death. All 19 AH patients dosed with DUR-928 survived the 28-day study, while the historical 28-day mortality rate in AH patients is 26% on average.

POSIMIR (bupivacaine extended-release solution) Post-Operative Pain Relief Depot. POSIMIR is DURECT’s investigational post-operative pain relief depot that uses the Company’s patented SABER technology and is designed to deliver bupivacaine to provide up to 3 days of pain relief after surgery.

Since the Anesthetic and Analgesic Drug Products Advisory Committee (AADPAC) meeting on January 16, 2020, we have continued to interact with the FDA, including answering information requests, as they continue their review of the POSIMIR NDA.

Conference Call

We will host a conference call today at 4:30 p.m. Eastern Time / 1:30 p.m. Pacific Time to discuss second quarter 2020 results and provide a corporate update:

Monday, August 3 @ 4:30 p.m. Eastern Time / 1:30 p.m. Pacific Time

Toll Free:877-407-0784

International:201-689-8560

Conference ID:13706541

Webcast:View Source

A live audio webcast of the presentation will also be available by accessing DURECT’s homepage at www.durect.com and clicking "Investors." If you are unable to participate during the live webcast, the call will be archived on DURECT’s website under "Event Calendar" in the "Investors" section.

On August 3, 2020 Aduro Biotech, Inc. (NASDAQ: ADRO), a clinical-stage biopharmaceutical company focused on developing therapies targeting the A Proliferation Inducing Ligand (APRIL) and Stimulator of Interferon Genes (STING) pathways for the treatment of cancer, autoimmune and inflammatory diseases, reported financial results for the second quarter ended June 30, 2020 (Press release, Aduro Biotech, AUG 3, 2020, View Source [SID1234562702]).

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"The second quarter of 2020 was highlighted by the announcement of our planned merger with Chinook Therapeutics as well as significant progress in our BION-1301 program for IgA nephropathy (IgAN). We recently dosed the first IgAN patient with BION-1301 in Part 3 of our ongoing Phase 1 study and presented positive data from Parts 1 and 2 of this study in healthy volunteers at the 57th ERA-EDTA Virtual Congress. The data indicated BION-1301 was well-tolerated, had a half-life of approximately 33 days, achieved over 90% target engagement with a single 450 mg dose of BION-1301 and demonstrated dose-dependent and durable reductions in IgA and IgM levels, and to a lesser extent, IgG levels," said Stephen T. Isaacs, chairman, president and chief executive officer of Aduro. "We continue to enroll patients in our Phase 2 study of ADU-S100 in combination with pembrolizumab in squamous cell carcinoma of the head and neck and make progress on our cGAS-STING antagonist research collaboration with Lilly." Isaacs continued, "We ended the second quarter of 2020 with a cash position of $186.1 million, which we believe will enable us to continue our ongoing STING and APRIL programs in the near-term and also meet our net cash requirements at the close of the merger with Chinook."

Recent Highlights

Announced definitive merger agreement with Chinook Therapeutics, which is expected to close in the second half of 2020, subject to the satisfaction or waiver of the conditions to completion of the merger. Following completion of the merger, the combined company will operate as Chinook Therapeutics and advance a pipeline of precision medicines for kidney diseases, led by atrasentan and BION-1301 in IgAN, assuming satisfaction of the conditions to closing the merger.

Presented healthy volunteer data from Part 1 (single ascending dose) and Part 2 (multiple ascending dose) of the ongoing Phase 1 study of BION-1301 at the 57th ERA-EDTA Virtual Congress.

Presented nonclinical toxicology studies of BION-1301 evaluating intravenous administration for up to six months and subcutaneous administration for up to one month at the 57th ERA-EDTA Virtual Congress.

Dosed the first patient with IgAN in Part 3 of the ongoing Phase 1 study of BION-1301.

Financial Results

Cash Position – Cash, cash equivalents and marketable securities totaled $186.1 million at June 30, 2020, compared to $213.6 million at December 31, 2019. Cash spend year to date was offset by the receipt of a $10 million development milestone payment from Merck in the first quarter of 2020.

Revenue – Revenue was $5.6 million for the second quarter of 2020 and $19.5 million for the six months ended June 30, 2020, compared to $4.9 million and $8.8 million, respectively for the same periods in 2019. The increase in revenue for the quarter was primarily due to fluctuations in revenue recognized under our Novartis collaboration which is dependent on clinical timelines and progress under the research and collaboration agreement. In addition to the Novartis collaboration, the increase in revenue for the year to date period included the recognition of the $10.0 million development milestone payment received under our license and research collaboration agreement with Merck.

Research and development expenses were $11.1 million for the second quarter of 2020 and $26.9 million for the six months ended June 30, 2020, compared to $16.7 million and $34.2 million, respectively, for the same periods in 2019. The decrease in expense from 2019 to 2020 was primarily due to 2019 costs related to the deprioritized programs that were substantially wound down in 2019 offset by higher costs for our STING and APRIL programs. The decrease was also attributable to lower compensation and related personnel costs as well as stock-based compensation as compared to 2019 due to reduced headcount as a result of the January 2020 restructuring.

General and administrative expenses were $9.3 million for the second quarter of 2020 and $17.1 million for the six months ended June 30, 2020, compared to $7.8 million and $16.1 million, respectively, for the same periods in 2019. The quarter and year to date increases were due to higher professional services expenses associated with the merger transaction, the increase was offset by lower personnel and stock-based compensation expense, as compared to 2019, due to reduced headcount as a result of the January 2020 restructuring.

Restructuring and related expenses were $2.0 million for the second quarter of 2020 and $6.4 million for the six months ended June 30, 2020, compared to $0.4 million and $3.4 million, respectively, for the same periods in 2019. The year to date restructuring and related expenses consisted of severance and employee retention costs as well as the impairment of property and equipment associated with the planned closure of the Aduro Biotech Europe facility in Oss, The Netherlands as part of the January 2020 corporate restructuring plan. The $3.4 million restructuring and related expenses recorded in 2019, which included employee severance and retention payments, related to the January 2019 strategic reset.

Net Loss – Net loss for the second quarter of 2020 was $16.6 million or $0.21 per share and $24.2 million or $0.30 per share for the six months ended June 30, 2020, compared to net loss of $18.6 million or $0.23 per share and $42.0 million or $0.53 per share, respectively, for the same periods in 2019. In addition to the factors described above, the net loss was offset by approximately $5.6 million of income tax benefit related to an income tax carryback claim allowed by the Coronavirus Aid, Relief, and Economic Security Act (CARES Act). The income tax refund is expected to be received in the second half of 2020.

On August 3, 2020 Veracyte, Inc. (Nasdaq: VCYT) (the "Company") reported that it has commenced an underwritten public offering of 6,000,000 shares of its common stock (Press release, Veracyte, AUG 3, 2020, View Source [SID1234562701]). All of the shares are being offered by Veracyte. In addition, the Company expects to grant the underwriters a 30-day option to purchase up to 900,000 additional shares of its common stock at the public offering price, less underwriting discounts and commissions.

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Veracyte intends to use the net proceeds from the offering for working capital and other general corporate purposes. The Company may also use a portion of the net proceeds from the offering to acquire or invest in complementary businesses, technologies or other assets, although it has no present commitments or agreements to do so.

Goldman Sachs & Co. LLC and SVB Leerink LLC are acting as joint lead book-running managers for the offering, William Blair & Company, L.L.C. is acting as a book-running manager and BTIG, LLC, Needham & Company, LLC, and Lake Street Capital Markets, LLC are acting as co-managers.

The shares will be issued pursuant to an effective shelf registration statement filed with the Securities and Exchange Commission (SEC) on May 2, 2019. A preliminary prospectus supplement and accompanying prospectus relating to the offering will be filed with the SEC and will be available on the SEC’s website at www.sec.gov. Copies of the preliminary prospectus supplement and accompanying prospectus relating to the offering, when available, may be obtained by contacting Goldman Sachs & Co. LLC, Attention: Prospectus Department, 200 West Street, New York, NY 10282, by email at [email protected], or by telephone at (866) 471-2526; or SVB Leerink LLC, Attention: Syndicate Department, One Federal Street, 37th Floor, Boston, Massachusetts 01220, by email at [email protected], or by telephone at (800) 808-7525, ext. 6218.

On August 3, 2020 Selecta Biosciences, Inc. (NASDAQ: SELB), a clinical-stage biotechnology company focused on unlocking the full potential of biologic therapies based on its immune tolerance platform, ImmTOR, reported the appointment of Peter G. Traber, MD, to the position of Chief Medical Officer (Press release, Selecta Biosciences, AUG 3, 2020, View Source [SID1234562700]). Dr. Traber has been serving in the same position in an interim capacity, and joins the Company full-time, effective August 1, 2020.

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"Since becoming part of our organization, Peter has brought a wealth of expertise from his experience in large pharma, biotech, and academia that has substantially strengthened our clinical development capabilities, and we are extremely pleased that he will be joining us full-time as Chief Medical Officer," said Carsten Brunn, Ph.D., President and CEO of Selecta. "We’re confident that he will play a critical role in elevating the strength of our science and translating it clinically, as we explore new applications for ImmTOR in biologics, gene therapy, and immunotherapies for autoimmune diseases."

"I am proud to be part of an organization that is pioneering innovations that may advance the treatment of a number of challenging diseases with unmet needs," said Dr. Traber. "Selecta has the capabilities and scientific acumen to become the undisputed leader in targeted and specific immune tolerance, and I’m excited to help advance ImmTOR and explore its role in driving innovation that will ultimately help patients."

As Chief Medical Officer, Dr. Traber will oversee medical affairs, program management, and all aspects of clinical development and strategy, as well as provide scientific and clinical guidance for potential business development initiatives. He is the founder and president of PGT Life Sciences Consulting, a biopharmaceutical consultancy for which he provides drug development strategy, clinical trial design, and scientific oversight for a broad range of companies. In a previous role, Dr. Traber was the President, Chief Executive Officer, and Chief Medical Officer of Galectin Therapeutics. During his tenure, he successfully built a full drug development team and created the strategy to progress its lead compound forward in multiple indications, including a phase 3-ready program in NASH cirrhosis. Prior to Galectin, he served as the Chief Medical Officer and Senior Vice President, Clinical Development & Medical Affairs at GSK. Previously, he served as the President and CEO of the Baylor College of Medicine, and Chairman of Medicine and CEO of the University of Pennsylvania Health System. His experience also includes managing an academic research laboratory, serving on the editorial boards of medical journals, and he has authored more than 100 research articles, reviews, and book chapters. He received his medical degree from Wayne State University School of Medicine, holds a Bachelor of Science in Chemical Engineering from the University of Michigan, and earned a certificate in Medical Leadership from Wharton Business School. He is currently an Adjunct Professor of Medicine at the University of Pennsylvania and serves on the board of Caladrius Biosciences.

On August 3, 2020 Neurocrine Biosciences, Inc. (Nasdaq: NBIX) reported its financial results for the second quarter ended June 30, 2020 and provided revised full-year 2020 financial expense guidance (Press release, Neurocrine Biosciences, AUG 3, 2020, View Source [SID1234562698]).

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"I want to thank healthcare providers and our employees for the perseverance they showed during the second quarter to ensure patients had uninterrupted access to INGREZZA under the challenging circumstances caused by the COVID-19 pandemic. As we move into the second half of 2020, we remain focused on improving the diagnosis and treatment rates for people with tardive dyskinesia, preparing to make ONGENTYS available to people living with Parkinson’s disease, and advancing our programs in clinical development," said Kevin Gorman, Ph.D., Chief Executive Officer of Neurocrine Biosciences. "With four U.S. FDA approved treatments that address four unique patient populations and a diverse and expanding pipeline, Neurocrine Biosciences is well positioned to be a leading neuroscience-focused biopharmaceutical company."

Second Quarter Net Product Sales Highlights:
•INGREZZA net product sales for the second quarter of 2020 were $268 million, representing a year-over-year increase of 48%.
•Continued strength in refill and persistency rates for existing INGREZZA patients.
•End of second quarter 2020 days-on-hand channel inventory increased relative to end of first quarter 2020, resulting in an approximate $12 million benefit to net product sales.
Financial Highlights:
•Second quarter 2020 GAAP net income and diluted earnings per share were approximately $80 million and $0.81, respectively, compared with approximately $51 million and $0.54, respectively, in the second quarter of 2019, primarily driven by higher INGREZZA sales offset by higher in-process Research and Development (IPR&D) costs and operating expenses.
•Second quarter 2020 non-GAAP net income and diluted earnings per share were approximately $139 million and $1.42, respectively, compared with approximately $67 million and $0.71, respectively, in the second quarter of 2019 driven by higher INGREZZA sales.
•Research and Development (R&D) expense increased in the second quarter of 2020 versus the second quarter of 2019, primarily due to milestone payments to BIAL associated with the approval of ONGENTYS and increased headcount costs.
•Selling, General and Administrative (SG&A) expense increased in the second quarter of 2020 versus the second quarter of 2019, primarily due to increased headcount costs.
•At June 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 April 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 payment to BIAL. The commercial launch of ONGENTYS is expected to occur later in 2020.
•In May 2020, AbbVie received approval from the FDA for ORIAHNNTM (elagolix, estradiol, and norethindrone acetate capsules; elagolix capsules) for the management of heavy menstrual bleeding associated with uterine fibroids in pre-menopausal women. FDA approval for ORIAHNN for uterine fibroids resulted in the achievement of a $30 million milestone. The Company will receive royalties at tiered percentage rates on net sales of ORIAHNN.
•In May 2020, the Company exercised its option with Idorsia Pharmaceuticals Ltd. paying $45 million to license the global rights to NBI-827104 (ACT-709478), a potent, selective, orally active and brain penetrating T-type calcium channel blocker, in clinical development for the treatment of a rare pediatric epilepsy. The option also included a research collaboration to discover novel T-type calcium channel blockers.
•In June 2020, the Company reported positive Phase II data for crinecerfont in adults with congenital adrenal hyperplasia (CAH) and highlighted the resumption of enrollment in the Phase IIa pediatric study in adolescents with classic CAH. In July 2020, the Company initiated the CAHtalyst Study (www.cahtalyststudy.com), a single, global registrational study of crinecerfont in adult patients with classic CAH.
•In June 2020, the Company entered an exclusive license with Takeda Pharmaceutical Company Limited, or Takeda, for the right to develop and commercialize certain compounds in Takeda’s early-to-mid-stage psychiatry pipeline. Specifically, Takeda granted the Company an exclusive license to seven pipeline programs, including three clinical-stage assets for negative effects of schizophrenia, treatment-resistant depression, and anhedonia. The agreement became effective in July 2020, upon expiration of the applicable waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, as amended, at which time the Company paid $120 million upfront (minus an earnest money deposit already paid by the Company to Takeda) to gain the exclusive license.

•Previously, the Company expected combined GAAP R&D and SG&A expenses in the range of $675 million to $725 million and combined non-GAAP R&D and SG&A expenses in the range of $550 million to $600 million.
•The $175 million increase in GAAP expense guidance range primarily reflects $45 million paid to Idorsia upon exercising the option to license the global rights to NBI-827104 (ACT-709478) and $120 million paid to Takeda for the exclusive license for the right to develop and commercialize certain compounds in Takeda’s early-to-mid-stage psychiatry pipeline.
•GAAP-only guidance includes approximately $105 million of share-based compensation. GAAP-only guidance does not include any other potential milestones or in-process research and development costs associated with current collaborations or potential 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 877-876-9173 (US) or 785-424-1667 (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.