On November 4, 2019 Sierra Oncology, Inc. (SRRA), a late-stage drug development company focused on advancing targeted therapeutics for the treatment of patients with significant unmet needs in hematology and oncology, reported its financial and operational results for the third quarter ended September 30, 2019 (Press release, Sierra Oncology, NOV 4, 2019, View Source [SID1234550244]).

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"During the third quarter, we continued to prepare for the launch of the MOMENTUM Phase 3 clinical trial, which is expected to occur in the fourth quarter of 2019. Data that will be generated from this trial, along with data from more than 800 myelofibrosis patients previously treated with momelotinib, many of them durably benefiting for several years of treatment, will form the basis of our planned regulatory submissions for this drug," said Dr. Nick Glover, President and CEO of Sierra Oncology. "If ultimately approved, momelotinib could represent a significant addition to the limited number of therapeutics available to patients with myelofibrosis, and potentially become the first therapeutic capable of improving anemia in these patients rather than exacerbating it, while also providing meaningful and long-lasting symptom and spleen benefits."

Sierra’s pipeline also includes a portfolio of DNA Damage Response (DDR) assets, SRA737 (Chk1 inhibitor) and SRA141 (Cdc7 inhibitor). Sierra has previously announced plans to prioritize its resources on the development of momelotinib and has launched a campaign exploring non-dilutive strategic options to support the future continued development of these other drug candidates.

About the MOMENTUM Phase 3 Clinical Trial for Patients with Myelofibrosis:

Sierra plans to launch the MOMENTUM Phase 3 clinical trial in the fourth quarter of 2019. The randomized double-blind trial is designed to enroll 180 myelofibrosis patients who are symptomatic and anemic, and who have been treated previously with a JAK inhibitor. Patients will be randomized 2:1 to receive either momelotinib or danazol. Danazol has been selected as an appropriate treatment comparator given its use to ameliorate anemia in myelofibrosis patients, as recommended by NCCN and ESMO (Free ESMO Whitepaper) guidelines. After 24 weeks of treatment, patients on danazol will be allowed to crossover to receive momelotinib.

The Primary Endpoint of the trial is the Total Symptom Score (TSS) response rate of momelotinib compared to danazol at Week 24 (99% power; p-value < 0.05).

Secondary and exploratory endpoints include:

Transfusion Independence (TI) rate at Week 24 (key secondary: >90% powered; p-value < 0.05),
Splenic response rate (SRR) at Week 24 (>90% powered; p-value < 0.05),
Duration of TSS response to Week 48,
Other measures of anemia benefit, including Transfusion Dependence response rate and various measures of cumulative transfusion burden,
Patient Reported Outcome measures of fatigue and physical function.
Dr. Srdan Verstovsek, MD, PhD, Chief, Section for Myeloproliferative Neoplasms, Department of Leukemia, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, Houston, Texas, has been named Chief Investigator of the MOMENTUM Phase 3 trial.

The FDA has granted Fast Track designation to momelotinib for the treatment of patients with intermediate/high-risk myelofibrosis who have previously received a JAK inhibitor.

Third Quarter 2019 Financial Results (all amounts reported in U.S. currency)

Research and development expenses were $10.1 million for the three months ended September 30, 2019, compared to $12.9 million for the three months ended September 30, 2018. The decrease was due to a $3.0 million upfront fee paid to Gilead to acquire momelotinib in the third quarter of 2018 and decreases in SRA737 and SRA141 costs, including a $2.4 million decrease in clinical trial costs primarily related to SRA737, a $1.4 million decrease in third-party manufacturing costs, and a $0.5 million decrease in research and preclinical costs. These decreases were partially offset by costs pertaining to momelotinib including an increase of $3.9 million in clinical trial and development related costs and a $0.6 million increase in third-party manufacturing costs. Research and development expenses included non-cash stock-based compensation of $0.9 million and $1.2 million for the three months ended September 30, 2019 and 2018, respectively.

Research and development expenses were $32.0 million for the nine months ended September 30, 2019, compared to $30.0 million for the nine months ended September 30, 2018. The increase was primarily due to costs related to momelotinib including a $8.2 million increase in clinical trial and development costs, a $1.9 million increase in third-party manufacturing costs and a $2.1 million increase in personnel-related and allocated overhead costs. These increases were partially offset by a $3.0 million upfront fee paid to Gilead to acquire momelotinib in the third quarter of 2018 and decreases in SRA737 and SRA141 costs, including a $3.6 million decrease in third-party manufacturing costs, a $2.4 million decrease in clinical trial costs primarily related to SRA737, and a $1.3 million decrease in research and preclinical costs. Research and development expenses included non-cash stock-based compensation of $3.3 million for the nine months ended September 30, 2019 and 2018.

General and administrative expenses were $3.1 million for both the three months ended September 30, 2019 and 2018. General and administrative expenses included non-cash stock-based compensation of $0.4 million and $0.7 million for the three months ended September 30, 2019 and 2018, respectively.

General and administrative expenses were $10.0 million for the nine months ended September 30, 2019, compared to $10.7 million for the nine months ended September 30, 2018. This decrease was due to decreases in personnel-related and allocated overhead costs of $0.3 million, professional fees of $0.2 million and business development related costs of $0.2 million. General and administrative expenses included non-cash stock-based compensation of $1.5 million and $1.8 million for the nine months ended September 30, 2019 and 2018, respectively.

For the three months ended September 30, 2019, Sierra incurred a net loss of $12.9 million compared to a net loss of $15.6 million for the three months ended September 30, 2018. For the nine months ended September 30, 2019, Sierra incurred a net loss of $40.8 million compared to a net loss of $39.1 million for the nine months ended September 30, 2018.

Cash and cash equivalents totaled $67.7 million as of September 30, 2019, compared to $106.0 million as of December 31, 2018. At September 30, 2019, there were 74,688,283 shares of common stock issued and outstanding, an additional 13,222,900 issuable upon exercise of stock options and warrants, and a term loan of $5.0 million.

On November 4, 2019 Puma Biotechnology, Inc. (NASDAQ: PBYI), a biopharmaceutical company, reported that Alan H. Auerbach, Chairman, Chief Executive Officer, President and Founder of Puma, will provide an overview of the Company at 10:55 a.m. MST (9:55 a.m. PST, 12:55 p.m. EST) on Tuesday, November 12, at the Credit Suisse 28th Annual Healthcare Conference (Press release, Puma Biotechnology, NOV 4, 2019, View Source [SID1234550243]). The conference will be held at The Phoenician Resort in Scottsdale, Arizona.

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A live webcast of the presentation will be available on the Company’s website at www.pumabiotechnology.com. The presentation will be archived on the website and available for 30 days.

On November 4, 2019 PharmaCyte Biotech, Inc. (OTCQB: PMCB), a biotechnology company focused on developing cellular therapies for cancer and diabetes using its signature live-cell encapsulation technology, Cell-in-a-Box, reported that it is closer to submitting an Investigational New Drug application (IND) to the U.S. Food & Drug Administration (FDA) (Press release, PharmaCyte Biotech, NOV 4, 2019, View Source [SID1234550242]). PharmaCyte and its partner, Austrianova Singapore (Austrianova), has successfully completed encapsulation of the cells from PharmaCyte’s Master Cell Bank (MCB) in the second of two staggered and back-to-back manufacturing runs for the production of PharmaCyte’s clinical trial product. This product will be used for PharmaCyte’s planned clinical trial in patients with locally advanced, inoperable pancreatic cancer (LAPC).

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PharmaCyte’s Chief Executive Officer, Kenneth L. Waggoner, commented, "The encapsulation process was performed last week at Austrianova’s manufacturing facility in Thailand. After reviewing the first pictures of the encapsulated cells, we’re confident that this manufacturing run is progressing as well as our first successful manufacturing run.

"We are anxiously awaiting the completion of the second of the two back-to-back manufacturing runs and the results from the FDA required testing on each of those runs. Once the information from that testing is available, it will be incorporated into our Investigational New Drug application (IND) for submission to the FDA."

Most of the work needed for PharmaCyte to submit an IND to the FDA has been completed. The rate limiting factor is and always has been for Austrianova to complete successfully two back-to-back manufacturing runs. During PharmaCyte’s recent shareholder update call, the company reported on Austrianova’s work to encapsulate successfully PharmaCyte’s genetically modified human cells that will be used to treat patients suffering from LAPC.

After months of extensive research and development (R&D) by a team of experts from Austrianova and PharmaCyte, a total of eight different changes were made to the manufacturing process. It was not until the eighth and final change was made that the encapsulated cells grew as well in Austrianova’s manufacturing facility in Thailand as they grew at Austrianova’s R&D facility in Singapore.

Once the recently encapsulated cells in this second of two manufacturing runs have completely filled the capsules, they will be placed into PharmaCyte’s clinical trial syringes and then frozen. A representative sample of those syringes will be thawed and undergo the FDA required "release testing." All "release testing" related to safety of the encapsulated cells is being outsourced by PharmaCyte to independent third-party laboratories in Europe. All "release testing" related to functionality of the encapsulated cells is being handled by Austrianova at its GMP facility in Thailand.

To learn more about PharmaCyte’s pancreatic cancer treatment and how it works inside the body to treat locally advanced inoperable pancreatic cancer, the company encourages you to watch its documentary video complete with medical animations at: View Source

On November 4, 2019 Perrigo Company plc (NYSE; TASE: PRGO), a leading global provider of "Quality, Affordable Self-care Products", reported that its Board of Directors declared a quarterly dividend of $0.21 per share, payable on December 17, 2019 to shareholders of record on November 29, 2019 (Press release, Perrigo Company, NOV 4, 2019, View Source [SID1234550241]).

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On November 4, 2019 Neurocrine Biosciences, Inc. (NASDAQ: NBIX) reported its financial results for the quarter ended September 30, 2019 and provided an update on the launch of INGREZZA (valbenazine) and its clinical development programs (Press release, Neurocrine Biosciences, NOV 4, 2019, View Source [SID1234550240]).

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"We are pleased that a record number of new patients initiated treatment with INGREZZA as healthcare providers continue to recognize and treat the involuntary movements associated with tardive dyskinesia," said Kevin Gorman, Ph.D., Chief Executive Officer of Neurocrine Biosciences. "Our development programs continue to progress including engagement with regulatory agencies on the adult CAH pivotal trial design. We remain focused on providing patients with access to INGREZZA and preparing for the approval of opicapone in the U.S., while investing strategically to position the company as a leading global biopharmaceutical organization."

Financial Results

Total revenues for the three and nine months ended September 30, 2019, were $222.1 million and $544.1 million, respectively, compared to $151.8 million and $319.7 million for the same periods in 2018.

Total revenues were comprised of the following (unaudited):

Collaboration revenue reflects event-based milestones and royalties earned under the Company’s collaboration agreement with AbbVie. During the third quarter of 2019, the Company recognized a $20 million event-based milestone as revenue upon the U.S. Food & Drug Administration (FDA) acceptance of AbbVie’s New Drug Application (NDA) submission of elagolix for the treatment of uterine fibroids. During the third quarter of 2018, the Company recognized a $40 million event-based milestone as revenue upon the FDA approval of ORILISSA (elagolix) for the management of endometriosis with associated moderate to severe pain.

For the third quarter of 2019, the Company reported net income of $53.8 million, or $0.56 diluted earnings per share, compared to net income of $50.8 million, or $0.52 diluted earnings per share, for the same period in 2018. The increase in net income is due to increased INGREZZA net product sales partially offset by continued INGREZZA investment and a $28.5 million unrealized loss on the Company’s Voyager Therapeutics equity investment. For the nine months ended September 30, 2019, the Company reported net income of $3.0 million, or $0.03 diluted earnings per share, compared to net income of $3.0 million, or $0.03 diluted earnings per share, for the same period in 2018. Net income for the first nine months of 2019 reflects increased INGREZZA net product sales offset by $118.1 million of in-process research and development (IPR&D) in connection with the strategic collaboration with Voyager.

Research and development (R&D) expenses for the three and nine months ended September 30, 2019, were $45.3 million and $144.6 million, respectively, compared to $35.5 million and $121.4 million for the same periods in 2018. The increase in R&D expenses for both periods is primarily due to funding of development activities in connection with the Voyager transaction.

In further connection with the Voyager collaboration, the Company recognized IPR&D of $118.1 million during the first nine months of 2019. In addition, the Company made an equity investment in Voyager which is required to be marked to market each quarter, resulting in an unrealized loss of $28.5 million and $5.8 million for the third quarter and first nine months of 2019, respectively, and is reflected in Other Expense.

Sales, general and administrative (SG&A) expenses for the three and nine months ended September 30, 2019, were $84.5 million and $252.9 million, respectively, compared to $60.4 million and $180.0 million for the same periods in 2018. The increase in SG&A expenses for both periods is primarily due to the sales force expansion completed in the third quarter of 2018, the national launch of a patient-focused disease state awareness campaign, Talk About TD, and an increase in the Branded Pharmaceutical Drug fee expense.

As of September 30, 2019, the Company’s cash and available-for-sale investments was $875.0 million.

Updated 2019 SG&A and R&D Expense Guidance

SG&A, IPR&D, and R&D expenses for 2019 are expected to be $658 million to $668 million. Ongoing SG&A and R&D expenses for 2019, excluding IPR&D, are now expected to approximate $540 million to $550 million, which compares to the prior SG&A and R&D expense guidance of $540 million to $570 million.

Pipeline Highlights

Valbenazine Update – Chorea Associated with Huntington’s Disease

In September 2019, the Company initiated KINECT-HD, a Phase III study of valbenazine for the treatment of chorea associated with Huntington’s disease. This is a multicenter, randomized, double-blind, placebo-controlled study to assess the efficacy, safety and tolerability of once-daily valbenazine in up to 120 adult patients over 12 weeks of treatment. The primary endpoint of this study is the comparison of the change from baseline of the Total Maximal Chorea sub-score of the Unified Huntington’s Disease Rating Scale between placebo and active treatment groups using the average of week 10 and week 12 scoring. Top-line data are expected in 2021.

Opicapone Update

In February 2017, the Company entered into an exclusive licensing agreement with

BIAL – Portela & CA, S.A. (BIAL) for the development and commercialization of opicapone in

the United States and Canada. Opicapone is a once-daily, oral, selective catechol-O-methyltransferase inhibitor, being developed as an adjunctive therapy to levodopa/carbidopa in patients with Parkinson’s disease experiencing OFF episodes. The Company met with the FDA in January 2018 and based upon the BIPARK-1 and BIPARK-2 pivotal Phase III studies conducted by BIAL, the FDA did not require additional Phase III trials to form an NDA submission. The NDA for opicapone was submitted to the FDA during the second quarter of 2019. The NDA was accepted by the FDA with a Prescription Drug User Fee Act (PDUFA) target action date of April 26, 2020.

Elagolix Update

On July 24, 2018, AbbVie, in collaboration with Neurocrine Biosciences, announced FDA approval and in October 2018 Health Canada approval for ORILISSA for the management of endometriosis with associated moderate to severe pain.

AbbVie provided positive top-line efficacy data from two Phase III studies in women with uterine fibroids in the first quarter of 2018 and from the associated six-month safety extension study during the third quarter of 2018. The ELARIS UF-I and UF-II studies of elagolix met all primary and ranked secondary endpoints at month six. The NDA for uterine fibroids was submitted to the FDA and accepted during the third quarter of 2019 with a PDUFA target action date in the second quarter of 2020. With the FDA acceptance of the NDA, a $20 million event-based milestone was recognized as revenue in the third quarter with a payment to be made by AbbVie during the fourth quarter of 2019.

AbbVie initiated a Phase II study of elagolix in women with polycystic ovary syndrome (PCOS) during the third quarter of 2019.

Congenital Adrenal Hyperplasia (CAH) Program (NBI-74788) Update

The Company began an adaptive, Phase II proof-of-concept study examining the pharmacokinetics, pharmacodynamics, and safety of NBI-74788 in adults with classic 21-hydroxylase deficiency congenital adrenal hyperplasia (CAH) in November 2017. This study evaluates the safety and tolerability of NBI-74788 in patients with CAH together with the relationship between exposure and specific steroid hormone levels in these patients. In March 2019, positive interim results from this ongoing study demonstrated a clinically meaningful reduction in key biomarkers associated with the management of CAH. NBI-74788 was shown to be well tolerated with no serious adverse events reported to date.

In July 2019, the Company initiated an adaptive, Phase II proof-of-concept study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of NBI-74788 in pediatric patients with classic CAH. In Q3 2019, the Company engaged with the FDA and the European Medicines Agency (EMA) to discuss the registrational trial design for the adult program.

Voyager Collaboration and VY-AADC Program

During the first quarter of 2019, Neurocrine Biosciences formed a strategic collaboration with Voyager Therapeutics focused on the development and commercialization of Voyager’s gene therapy programs, VY-AADC for Parkinson’s disease and VY-FXN01 for Friedreich’s ataxia, as well as rights to two programs to be determined. This collaboration combines Neurocrine

Biosciences’ expertise in neuroscience, drug development and commercialization with Voyager’s innovative gene therapy programs targeting severe neurological diseases.

Based on the results from the VY-AADC Phase I programs in Parkinson’s disease, RESTORE-1, a Phase II, randomized, placebo-surgery controlled, double-blinded, multi-center, clinical trial was initiated to evaluate the safety and efficacy of VY-AADC in patients who have been diagnosed with Parkinson’s disease for at least four years, are not responding adequately to oral medications, and have at least three hours of OFF time during the day as measured by a validated self-reported patient diary.

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.

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.