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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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.

On May 6, 2020 Harpoon Therapeutics, Inc. (Nasdaq: HARP), a clinical-stage immunotherapy company developing a novel class of T cell engagers, reported financial results for the first quarter ended March 31, 2020 and provided a corporate update (Press release, Harpoon Therapeutics, MAY 6, 2020, View Source [SID1234557135]).

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"Harpoon has made remarkable progress since the beginning of 2020, highlighted by the initiation of clinical development for our third novel TriTAC program, HPN217, for the treatment of multiple myeloma," said Gerald McMahon, Ph.D., President and Chief Executive Officer of Harpoon Therapeutics. "We continue to advance both of our lead clinical programs for HPN424 and HPN536 and plan to present interim clinical data for both of these studies this year. In addition, we are enrolling the clinical trial for HPN217 and are on track to file an IND followed by initiation of our fourth clinical trial for HPN328 in the second half of this year."

First Quarter 2020 Business Highlights and Other Recent Developments

In April, Harpoon announced the first patient was dosed with HPN217 in a Phase 1/2 clinical trial focused on relapsed/refractory multiple myeloma (RRMM). HPN217 is covered by a global development and option agreement with AbbVie Inc. (NYSE: ABBV) and treatment of the first patient in the clinical trial has triggered a $50 million milestone payment to Harpoon. HPN217 targets B-cell maturation antigen (BCMA), a well-validated target expressed on multiple myeloma cells. HPN217 is Harpoon’s third product candidate to enter the clinic and is based on Harpoon’s proprietary Tri-specific T cell Activating Construct (TriTAC) platform designed to recruit a patient’s own immune cells to destroy tumors.

In April, Harpoon appointed Andrew R. Robbins and Joseph S. Bailes, M.D., to its Board of Directors. Among his many achievements, Mr. Robbins is credited with leading the highly successful U.S. launch of BRAFTOVI (encorafenib) + MEKTOVI (binimetinib) in BRAF-mutant metastatic melanoma. Dr. Bailes is a medical oncologist with substantial experience in clinical practice, legislation, public policy and advocacy, and for nearly two decades, served in various executive leadership capacities with the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) including as President.

Patient enrollment and dose escalation continues in the Phase 1 trials for HPN424 in metastatic castration resistant prostate cancer and in the Phase 1/2a trial for HPN536, initially for ovarian and

pancreatic cancers. Harpoon plans to present interim HPN424 data at the ASCO (Free ASCO Whitepaper) 2020 Virtual Meeting (Abstract 5552). The Company will host a virtual event to provide a clinical trial and pipeline update in parallel with the ASCO (Free ASCO Whitepaper) meeting.

Anticipated 2020 Milestones

HPN424 – present interim data from the dose escalation phase of our Phase 1 trial at ASCO (Free ASCO Whitepaper)20 Virtual and initiate expansion cohort in 2020

HPN536 – present interim data from Phase 1/2a trial in the second half of 2020

HPN217 – initiate a Phase 1/2 trial in the first half of 2020 (Completed)

HPN328 – initiate Phase 1/2a trial in the second half of 2020

First Quarter 2020 Financial Results

Harpoon ended the first quarter of 2020 with $138.2 million in cash, cash equivalents, and marketable securities compared to $155.1 million as of December 31, 2019. This figure does not include the $50 million milestone payment achieved through the AbbVie agreement noted above.

Revenue for the first quarter ended March 31, 2020 was $3.3 million compared to $1.1 million for the first quarter ended March 31, 2019. The increase in revenue was primarily due to revenue recognized from the upfront payment under the development and option agreement with AbbVie, signed in November 2019.

Research and development expense for the first quarter ended March 31, 2020 was $12.5 million compared to $9.4 million for the first quarter ended March 31, 2019. The increase primarily arose from clinical development expenses and an increase in personnel-related expenses, which included conducting preclinical studies, the continuation and preparation of the clinical trials for HPN424, HPN536 and HPN217, and manufacturing activities for four TriTAC product candidates in various stages of development.

General and administrative expenses for the quarter ended March 31, 2020 was $3.9 million compared to $5.8 million for the quarter ended March 31, 2019. The decrease was due to higher expenses incurred in the first quarter of 2019 primarily related to legal fees associated with Maverick litigation, and consulting and accounting services, offset by an increase in personnel expenses related to an increase in headcount, and other professional services to support our ongoing operations as a public company.

Net loss for the quarter ended March 31, 2020 was $12.6 million compared to $13.6 million for the quarter ended March 31, 2019.

COVID-19 Update

In response to the COVID19 pandemic, Harpoon notes that it closed its executive offices in compliance with county and state shelter-in-place orders, the result of which is that substantially all of the Company’s employees are currently telecommuting, and there is only a limited the number of staff working in the Company’s laboratory. Harpoon is currently continuing its clinical trials it has underway in sites in the United States, and has not yet experienced any material delays or impacts as a result of the pandemic. In addition, Harpoon’s third-party contract manufacturers continue to operate at or near normal levels and the Company does not currently anticipate material interruptions. Harpoon continues to assess the potential impact of the COVID-19 pandemic on its business and operations, including its programs, expected timelines, expenses, manufacturing and clinical trials. The full extent to which the COVID-19 pandemic may have a negative impact on Harpoon’s business, results of operations and financial condition, and will depend on future developments that are highly uncertain and cannot be accurately predicted.

On May 6, 2020 Constellation Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company using its expertise in epigenetics to discover and develop novel therapeutics, reported its first-quarter 2020 financial results (Press release, Constellation Pharmaceuticals, MAY 6, 2020, View Source [SID1234557134]).

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"In the face of the serious public health and economic impacts of the COVID-19 pandemic, we at Constellation remain focused on our mission of addressing unmet medical needs in cancer and hematological diseases," said Jigar Raythatha, president and chief executive officer of Constellation Pharmaceuticals. "We are continuing to make progress on each of our development programs and are steadily advancing toward our goal of becoming a fully integrated hematology / oncology company with a sustainable product pipeline.

"Our vision is to transform the standard of care in myelofibrosis with CPI-0610. Encouraging preliminary data on CPI-0610 suggest possible disease-modifying effects, including improvement in bone marrow fibrosis, hemoglobin increases, and conversion of transfusion dependence to transfusion independence, as well as spleen volume reductions and symptom improvement – both in combination with ruxolitinib and as a monotherapy.

"We are also working to create novel treatments for patients in other therapeutic areas," Mr. Raythatha concluded. "We believe that our EZH2-inhibitor franchise of CPI-1205 and CPI-0209 provides potential opportunities to treat a wide range of oncology patients, and we aim to achieve important milestones with each of these molecules this year."

Program Updates

CPI-0610

On May 14, we expect three abstracts to publish in association with the European Hematology Association (EHA) (Free EHA Whitepaper). We will provide an update with 12-week data from 29 JAK-inhibitor-naïve (first-line) patients, 24-week data from 15 JAK-inhibitor-naïve patients, and 24-week data from 48 JAK-inhibitor-experienced (second-line) patients.

Data will include preliminary evidence of disease modification, including bone marrow fibrosis, hemoglobin changes, and conversion from transfusion dependence to transfusion independence, in addition to spleen and symptom improvement.

In mid-June, we expect to present a further update in conjunction with the EHA (Free EHA Whitepaper) meeting, including 12-week data from approximately 50 first-line patients, and 24-week data from 25-30 first-line patients and 70-80 second-line patients.

We aim to start a global clinical trial for CPI-0610 in the first-line setting during the second half of 2020.

We continue to plan to meet with the FDA in mid-2020 to discuss the future development of CPI-0610.

The ProSTAR trial is fully enrolled and we plan to determine next steps for CPI-1205 after taking a mid-year data cut. As we previously discussed, our plans for any potential Phase 3 program for CPI-1205 will depend on our assessment of these data on duration of effect, as well as other considerations.

The Phase 1 clinical trial for CPI-0209 is proceeding as planned, and we expect to determine a recommended Phase 2 dose in the second half of 2020. Once we have established the Phase 2 dose we plan to start a broad-based expansion study in solid tumors.

Impact of COVID-19

Execution of ongoing clinical trials. Patient safety remains paramount in the execution of our clinical trials. In the face of the COVID-19 pandemic, we continue to treat patients in our MANIFEST, ProSTAR, and CPI-0209 clinical trials.

Patient enrollment in MANIFEST began to slow toward the end of first quarter of 2020. Prior to the pandemic, we had met or exceeded our internal enrollment goal for MANIFEST, and we continue to assess what impact the pandemic could have on our MANIFEST trial timeline. Similarly, while we have had incidences of incomplete data collection to date, we are utilizing provisions of the protocol and recent regulatory guidance that provide potential flexibility in the time and place of data collection, and we will continue to monitor the situation. We expect to provide a data update at EHA (Free EHA Whitepaper) similar to our plans prior to the COVID-19 outbreak.

To date, we have not seen a significant impact of COVID-19 on clinical trials for CPI-1205 or CPI-0209. The ProSTAR trial for CPI-1205 is proceeding, and we continue to expect to do a data cut in mid-2020 and to provide an update shortly thereafter. Our CPI-0209 Phase 1 trial continues as planned before the COVID-19 outbreak and we continue to expect to determine a recommended Phase 2 dose in the second half of 2020.

CPI-0610 Phase 3 clinical trial. Conditions at clinical trial sites caused by COVID-19 may impact the timing of the start of our Phase 3 clinical trial for CPI-0610. However, we still aim to begin this trial in the second half of 2020.

Manufacturing. We have experienced some disruption in our supply chain due to COVID-19. However, supply chain disruptions have not impacted our overall timelines for conducting clinical trials to date, and we continue to manufacture batches for our ongoing clinical trials.

Milestones

The Company anticipates achieving the following milestones during 2020:

CPI-0610 – Provide MANIFEST program update at EHA (Free EHA Whitepaper) in June

CPI-0610 – Initiate Phase 3 clinical trial in second half of 2020

CPI-0610 – Provide additional MANIFEST program update by end of year

CPI-1205 – Provide ProSTAR program update and determine next steps mid-year

CPI-0209 – Provide program update, including recommended Phase 2 dose, by end of year

First Quarter 2020 Financial Results

Cash, cash equivalents, and marketable securities as of March 31, 2020, were $358.8 million, a decrease of 6.5% compared to December 31, 2019, primarily due to operating expenses.

Research and development (R&D) expenses increased 28.1% year over year to $20.1 million in the first quarter of 2020, mainly due to increased clinical trial expenses.

General and administrative (G&A) expenses grew 33.4% year over year to $5.9 million in the first quarter of 2020, primarily due to building out the organization of the company.

The net loss attributed to common shareholders increased 31.0% year over year to $25.4 million for the first quarter of 2020, mainly due to increased R&D and G&A expenses. The net loss per share attributable to common shareholders decreased 18.7% to $0.61 per share due to an increase in shares outstanding as a result of the private placement in October 2019 and the public offering in December 2019, offset in part by the increased net loss.

Financial Guidance

Constellation expects that its current cash, cash equivalents, and marketable securities will enable it to fund operations into the second half of 2022.

Constellation will host a conference call at 8:00 AM EDT on May 6, 2020, to discuss its clinical programs and financial results. The event will be webcast live and can be accessed on the Investor Relations section of Constellation’s website at View Source To participate in the live question-and-answer session, please dial (877) 473-2077 (domestic) or (661) 378-9662 (international) and refer to conference ID 4372778.