On January 6, 2019 Neurocrine Biosciences, Inc. (NASDAQ: NBIX) reported an update on its business performance, including preliminary net product sales results for 2018, and key clinical development programs for 2019 (Press release, Neurocrine Biosciences, JAN 6, 2019, View Source [SID1234532491]). Kevin Gorman, Chief Executive Officer of Neurocrine, will discuss these updates as part of a webcast presentation at the 37th Annual J.P. Morgan Healthcare Conference in San Francisco on Monday, Jan. 7 at 2:30 p.m. PT (5:30 p.m. ET).

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"Our fourth quarter and 2018 results reflect the dedication of our team in educating healthcare providers about tardive dyskinesia and the benefit INGREZZA can bring to patients. We still have a lot of work to do as many people suffering from tardive dyskinesia remain undiagnosed and untreated, and we remain committed to helping the lives of patients through our recently expanded field sales team and disease awareness education," said Kevin Gorman, Ph.D., Chief Executive Officer of Neurocrine Biosciences. "In 2019, we will remain focused on reaching more patients with INGREZZA and making progress in our clinical development programs, including the planned submission of AbbVie’s new drug application for uterine fibroids and our planned submission for Parkinson’s disease, as well as advancing our congenital adrenal hyperplasia program."

Preliminary Fourth Quarter and Full-Year 2018 Net Product Sales Results (Unaudited)

Based on preliminary unaudited financial information, the Company expects net product sales for the three months and full-year ended December 31, 2018 to be approximately $130 million and $409 million, respectively, compared to $64.5 million and $116.6 million for the same periods in 2017.

2019 Expected Milestones

INGREZZA (valbenazine) for Tardive Dyskinesia

"Talk About TD" disease state awareness campaign
Execution of post-marketing clinical studies, including RE-KINECT, the largest real-world study in patients with possible tardive dyskinesia
Presentations at key scientific annual meetings, including American Academy of Neurology (AAN), American Psychiatric Association (APA), International Parkinson and Movement Disorder Society (MDS)
Elagolix in Collaboration with AbbVie

Continued launch of ORILISSA (elagolix) to treat endometriosis by AbbVie
Elagolix for uterine fibroids: planned 2019 New Drug Application (NDA) submission
Opicapone for Parkinson’s Disease

NDA submission in Q2 2019
Preparation for 2020 opicapone commercial launch
Presentations at key scientific annual meetings, including AAN, MDS
Congenital Adrenal Hyperplasia (CAH)/NBI-74788 (In Development)

Phase IIa data for CAH (adults) in Q1 2019
Phase IIa initiation for CAH (pediatric) in Q2/Q3 2019
Pivotal study initiation for CAH (adults) in 2H 2019, pending U.S. Food and Drug Administration (FDA) discussion in Q2
Early Stage Programs

Investigational New Drug submission and initiation of a Phase I trial for a new, internally discovered program
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 in 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.

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.

On January 6, 2019 Ascentage Pharma Group, Inc., and The University of Texas MD Anderson Cancer Center reported a five-year strategic collaboration agreement to advance the development of five potential new cancer therapies (Press release, Ascentage Pharma, JAN 6, 2019, View Source [SID1234532492]).

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The alliance is aimed at developing novel cancer therapeutics based upon Ascentage’s proprietary Protein-Protein Interaction drug discovery technology platform. MD Anderson’s leukemia team will join in efforts to advance the clinical development of Ascentage’s apoptosis-targeted and tyrosine kinase inhibitor candidates, including Bcr-Abl inhibitor HQP1351, Bcl-2/xL inhibitor APG-1252, Bcl-2 selective inhibitor APG-2575, IAP inhibitor APG-1387 and MDM2-p53 inhibitor APG-115. These compounds will be studied as single-agent therapies, as well as in combination with approved or investigational therapeutics against various forms of hematologic malignancies.

The strategic alliance leverages MD Anderson’s translational research and clinical expertise to help accelerate the development of the select candidates, chosen for their potential to target cancer cell apoptosis and other pathways for treating life-threatening cancers, such as Acute Myelocytic Leukemia (AML), Chronic Myeloid Leukemia (CML), Acute Lymphoblastic Leukemia (ALL), Myeloproliferative Neoplasms (MPN), and Myelofibrosis. The alliance will be led by Hagop Kantarjian, M.D., Chair of Department of Leukemia at MD Anderson, whose research and collaborations were the basis for the FDA approvals of over 20 drugs in leukemia.

"MD Anderson is highly dedicated to developing and providing more effective therapies for patients. This strategic alliance is important for our work towards finding cures to treat cancers. We will be investigating this pipeline of candidate therapies, and we are interested in the novel mechanism of their actions," said Dr. Kantarjian.

"We are pleased to announce this important partnership with MD Anderson," said Dr. Dajun Yang, Chairman & CEO of Ascentage Pharma. "We look forward to working closely with the investigators in MDACC in the hopes of accelerating the clinical development of these important candidates to provide new treatment options for cancer patients in the US and worldwide."

On January 6, 2019 Roivant Sciences reported its corporate updates across its family of companies in advance of the 37th Annual J.P. Morgan Healthcare Conference (Press release, Roivant Sciences, JAN 6, 2019, View Source [SID1234532490]).

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Launch of Alyvant

Roivant announced the launch of Alyvant, a new Vant focused on technology-enabled pharmaceutical commercialization which has entered into an agreement with a pharmaceutical partner to co-promote three approved branded medicines. Alyvant’s long-range vision is to reduce the cost of commercializing all medicines through technology, generating savings for the healthcare system at large.

Alyvant is led by President Gillian Cannon, who joined the company with over 30 years of leadership experience in the pharmaceutical industry, including at Merck, Otsuka, and UCB. Alyvant’s Chief Technology Officer, Mitchell Mittman, previously worked in senior roles at Amazon, Expedia, Goldman Sachs, and other financial institutions where he built industry-leading software and data science platforms.

"Alyvant’s technology integrates market research, sales force optimization, and digital engagement into a single platform to connect patients and physicians with relevant medicines as cost-effectively as possible," said Benjamin Zimmer, President of Roivant Health. "This technology platform allows us to engage new market segments that are underserved by traditional pharma commercial models, while also reducing the need for expensive and wasteful mass-market DTC campaigns that inflate the cost of delivering new medicines to patients."

Additional Updates

Urovant reported that the first patient has been dosed in its Phase 2a trial of vibegron in patients with abdominal pain due to irritable bowel syndrome
Axovant announced the creation of its Scientific Advisory Board and provided clinical trial timelines for four of its investigational gene therapies
Dermavant and Altavant announced appointments of senior executives
Vivek Ramaswamy, Founder and Chief Executive Officer of Roivant, will provide further business updates, including new data from Immunovant’s ongoing Phase 1 study of subcutaneous anti-FcRn antibody RVT-1401, at the J.P. Morgan Healthcare Conference tomorrow, Monday, January 7th, at 9:30 a.m. PT at the Westin St. Francis. His presentation will be webcast here.

On January 6, 2019 Solasia Pharma K.K. (TOKYO:4597, Headquarters: Tokyo, Japan, President & CEO: Yoshihiro Arai, hereinafter "Solasia") reported the initiation of Phase III clinical trial for PledOx in Japan (Press release, Solasia, JAN 6, 2019, View Source [SID1234532489]).

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In November 2017, Solasia acquired exclusive development and commercialization rights for PledOx in Japan, China, Hong Kong, Macau, South Korea and Taiwan from PledPharma.

This Phase III clinical trial in Asia (Japan, South Korea, Taiwan and Hong Kong) is an expansion of the Global Phase III trial led by PledPharma in the rest of the world where the first patient was included in the United States in November. The Asian region including Japan is now officially part of this Global Phase III clinical trial.
Following Japan, Solasia will also initiate in South Korea, Taiwan and Hong Kong successively.

This trial is for colorectal cancer patients treated with mFOLFOX6 (*1) which contains antioxidant drug "oxaliplatin" and to examine the effect of suppressing the development of peripheral neuropathy by administering PledOx. Oxaliplatin is a platinum-based drug and is indicated for colorectal cancer, pancreatic cancer, gastric cancer etc.
Peripheral neuropathy is known as one of the serious side effects caused by administration of oxaliplatin, and one of the causes is that neurons develop by being damaged due to oxidative stress induced by the drug. Peripheral neuropathy is also known as the main side effect of other platinum-based drugs such as cisplatin. There are currently no drugs approved for the treatment of chemotherapy induced peripheral neuropathy. PledOx is a superoxide dismutase analogue that is an enzyme that degrades active oxygen generated in cells and has the effect of protecting nerve cells from damage caused by drug-induced oxidative stress.
The initiation of this trial is a significant milestone for Solasia as this trial is positioned as a registration trial and its success is expected to contribute to patients suffering from peripheral neuropathy due to cancer chemotherapy.

Study description:

Phase III, International, multicenter, double-blind, randomized, placebo-controlled study
Purpose of the study:

The effect of reduce the peripheral neuropathy associated with administration of oxaliplatin by PledOx administration compared with placebo.
Study design:

POLAR-M study: Colorectal cancer patients who undergo mFOLFOX6 therapy with distant metastases are included.
POLAR-A study: Colorectal cancer patients who undergo mFOLFOX6 therapy as an adjuvant therapy for postoperative surgery are included.
Primary outcome measures:

Both the POLAR-M and POLAR-A studies will evaluate the proportion of patients with moderate or severe chronic peripheral neuropathy at 9 months after (first day of FOLFOX therapy) the initial administration of PledOx.
Estimated enrollment:

POLAR-M study: 420 patients (of which 120 patients in Asian region)
POLAR-A study: 280 patients (of which 80 patients in Asian region)
About chemotherapy induced peripheral neuropathy (CIPN) in Japan

Cancer chemotherapy has side effects such as nausea and vomiting and onset of stomatitis, but peripheral neuropathy is also a serious side effect. Peripheral neuropathy is known to be markedly expressed in major drugs of cancer chemotherapy such as plant alkaloid drugs and platinum-based drugs (*2). FOLFOX therapy including oxaliplatin, is a combination of a chemotherapy for advanced and recurrent cancer and a typical anticancer drug for postoperative adjuvant chemotherapy as a treatment for colorectal cancer, which is difficult to heal surgically. Almost all patients (85% – 95%) of oxaliplatin prescribed patients develop peripheral nerve disorder, and the disorder brings about the following symptoms (*2).

Acute symptoms: abnormal sensations such as hands, feet and lip peripheral parts, strangulation of pharyngeal larynx accompanied with dyspnea and dysphagia
Chronic symptoms: numbness in the periphery of the limbs, decreased sensation, decreased tendon reflexes, sensory ataxia
When such side effects occur, it is considered that some symptom improvement is seen in 80% of patients due to discontinuation of medicine and completely recovered in 6 to 8 months in 40% of cases (*2). However, discontinuation of the drug results in discontinuation of cancer treatment or change in treatment regimen which is an important medical issue. There is currently no approved drug to prevent or treat CIPN.

* 1: mFOLFOX6 therapy refers to cancer chemotherapy using three agents, fluorouracil, folinic acid, and oxaliplatin. It is adopted as standard therapy for high risk Stage II colorectal cancer or postoperative adjuvant chemotherapy for Stage III colorectal cancer and systemic chemotherapy for Stage IV recurrent colorectal cancer.

* 2: Reference: Ministry of Health, Labor and Welfare "Corresponding manual for severe side effects disease Peripheral neuropathy"

About PledOx

PledOx is a "first in class" drug candidate developed to provide patients, that are treated adjuvantly or for metastatic colorectal cancer, prevention against the nerve damage that can occur in conjunction with chemotherapy treatment. The results from a completed Phase IIb trial (PLIANT), where patients with metastatic colorectal cancer were treated with the chemotherapy combination FOLFOX and PledOx, indicates that the patients who received PledOx had a lower risk than the placebo group to suffer from nerve damage during the chemotherapy. The presence of the investigator reported sensory nerve damage, the primary endpoint, was after treatment 38% lower in the group of patients treated with PledOx compared with the placebo group (p = 0.16). This was not statistically significant, but a difference of this magnitude is considered to be clinically relevant. After completion of chemotherapy, the patient-reported incidence of moderate and severe neuropathy was 77% lower in patients treated with PledOx compared to the placebo group (exploratory analysis; p = 0.014). This is considered valuable for the success of the forthcoming POLAR studies, where patient-reported symptoms after completion of treatment will be the primary efficacy parameter. No apparent negative effect on the efficacy of the cancer treatment was observed.

On January 6, 2019 Samsung Bioepis Co., Ltd. reported that its rapidly growing biosimilar business will expand into mainland China through a licensing agreement with 3SBio Inc (Press release, Samsung Bioepis, JAN 6, 2019, View Source [SID1234532488]). The agreement covers multiple biosimilar candidates from Samsung Bioepis, including SB8, a biosimilar candidate referencing AVASTIN 1 (bevacizumab).

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Under the agreement, Samsung Bioepis and 3SBio will collaborate across a number of areas, including clinical development, regulatory registration and commercialization in China. Samsung Bioepis will receive upfront and milestone payments, as well as royalties on sales. Additional financial details were not disclosed.

"We are very excited to expand our biosimilar business into China, where we hope to see our biosimilars play an important role in widening patient access to high-quality healthcare. We are confident we will achieve this goal through our partnership with 3SBio, which brings together Samsung Bioepis’ proven development platform with 3SBio’s strong commercialization platform," said Christopher Hansung Ko, President and Chief Executive Officer, Samsung Bioepis. "At Samsung Bioepis, we will continue to demonstrate our enduring commitment to biosimilars by further strengthening our pipeline and widening their availability for patients and healthcare systems across the world."

Established in February 2012, Samsung Bioepis currently has four biosimilars approved and marketed across Europe, which include the anti-TNF trio of BENEPALI (etanercept), FLIXABI (infliximab) and IMRALDI (adalimumab), as well as an oncologic biosimilar, ONTRUZANT (trastuzumab). In the United States, the company has one biosimilar approved and marketed, RENFLEXIS (infliximab), with both SB5 (adalimumab) and SB3 (trastuzumab) biosimilar candidates currently under regulatory review.

In total, over 100,000 patients across the world are currently under treatment with Samsung Bioepis’ biosimilars, with over 6 million doses administered.