On January 8, 2021 MANA Therapeutics, a clinical-stage company creating nonengineered, off-the-shelf allogeneic cell therapies that target multiple cancer antigens, reported a $35 million Series A financing (Press release, MANA Therapeutics, JAN 8, 2021, View Source [SID1234573712]). The financing was led by Cobro Ventures and Lightchain Capital, with participation from LifeSci Venture Partners and other undisclosed investors. MANA is using its EDIFY platform to develop a pipeline of proprietary and partnered off-the-shelf cell therapies for cancer patients across a broad range of liquid and solid tumors, with an initial focus on relapsed acute myeloid leukemia (AML).

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MANA’s EDIFY platform leverages natural immune system pathways to educate T-cells to target multiple cell surface and intracellular tumor-associated antigens. The multi-antigen targeting is uniquely achieved without the need for viral or non-viral delivery systems or genetic modifications. MANA was founded based on the research and human proof-of-concept clinical trials conducted by Catherine Bollard, M.D., MBChB and her team at Children’s National Hospital (CNH) along with colleagues at Johns Hopkins Medical Center. The initial CNH and Hopkins-led clinical trials in solid and hematologic tumors supported a strong safety profile, showed immunological anti-tumor activity and validated MANA’s initial set of tumor antigens. The product candidates used in these trials formed the basis of the Phase 1 candidate, MANA-312, an allogeneic donor-derived cell therapy for the treatment of AML in the relapsed/refractory post-hematopoietic stem cell transplant (HSCT) setting, and MANA-412, a preclinical off-the-shelf allogeneic cell therapy being developed for the treatment of transplant-ineligible AML and solid tumors.

"I co-founded MANA with Dr. Bollard and her colleagues from Children’s National Hospital because we believe off-the-shelf, allogeneic approaches are the future of cell therapy that will enable more patients to benefit from this breakthrough in cancer treatment," said Marc Cohen, Co-founder of Cobro Ventures and Co-founder and Executive Chairman of MANA Therapeutics. "MANA is building upon the strong foundational science established at CNH with a unique approach that promises to produce off-the-shelf allogeneic therapies that do not compromise on safety or efficacy. I look forward to continuing to support the MANA team as they advance their internal pipeline for the treatment of AML and select solid tumors, and expand the potential of EDIFY through strategic partnerships focused on new target antigens and cancer types."

MANA’s goal is to develop an inventory of off-the-shelf allogeneic products that will be able to treat the majority of patients in targeted cancer indications based on a simplified HLA matching protocol. Through multiple antigen targeting, MANA’s product candidates are designed to prevent immune escape and could provide superior efficacy to single antigen and other cell therapy approaches. MANA uses a simplified scalable manufacturing process that does not require genetic modification and has a high product yield, with the potential to generate tens of billions of cells from a single partial HLA-matched donor. The high safety profile supported by academic clinical trials and the nonengineered approach may also permit for repeat dosing of patients.

"Over the past decade we have seen tremendous progress in cancer research and treatment and are beginning to unlock the potential of cell therapy for a variety of tumor types," said Dr. Bollard, who chairs MANA’s Scientific Advisory Board. "The human proof-of-concept trials conducted by my team and colleagues showed potential for a nonengineered approach to educating T-cells to attack multiple tumor antigens, which MANA is expanding even further through refinement of the manufacturing process for an allogeneic product and application to a broader set of antigens in a variety of clinical indications and settings."

"This Series A funding is enabling rapid progress with our programs," said Martin Silverstein, M.D., President and CEO of MANA Therapeutics. "We recently initiated our Phase 1 clinical trial for MANA-312 in relapsed/refractory AML with the goal of establishing single-agent activity and safety of higher and multiple doses along with the assessment of key efficacy biomarkers in the post-transplant setting. This important study with an allogeneic donor-derived cell therapy is designed to inform and accelerate trials for our off-the-shelf allogeneic candidate, MANA-412, for which we plan to file an IND in late-2021."

As part of the financing, Drew Dennison of Lightchain Capital has joined the MANA Board of Directors. Locust Walk served as exclusive transaction advisor to MANA.

About EDIFY

MANA Therapeutics’ EDIFY platform constitutes the next-generation cell therapy approach by leveraging natural immune system pathways to educate T-cells to target the unique sets of antigens expressed by tumors, without the need for genetic modification. The EDIFY platform uses dendritic cells as antigen presenting cells. The dendritic cells are loaded with ManaMix antigens and stimulate and expand T-cells to generate product candidates. Product candidates developed from the EDIFY platform are designed to increase efficacy through multiple antigen targeting and to support a strong safety profile by utilizing a nonengineered approach that could permit for repeat dosing of patients. The EDIFY platform uses a simplified, high-yield manufacturing process.

On January 8, 2021 OncoSec Medical Incorporated (NASDAQ:ONCS) (the "Company" or "OncoSec") reported the first patient was dosed in OMS-104, an investigator-initiated Phase 2 trial evaluating TAVO (tavokinogene telseplasmid), the Company’s intratumoral DNA plasmid-based interleukin-12 (IL-12) therapy administered using its gene delivery platform (gene electrotransfer), in combination with the anti-PD-1 checkpoint inhibitor OPDIVO (nivolumab) as a neoadjuvant therapy prior to surgery in patients with operable, locally or regionally advanced melanoma (Press release, OncoSec Medical, JAN 8, 2021, View Source [SID1234573711]). The trial is designed to evaluate if the addition of TAVO can improve clinical outcomes already observed when using nivolumab alone as a neoadjuvant therapy.

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Anti-PD1 checkpoint inhibitors, when administered as a neoadjuvant therapy, have shown encouraging clinical results, but rapid recurrence remains an issue for many patients. TAVO in combination with OPDIVO may drive deep anti-tumor immune responses and complete elimination of tumors prior to surgery, leading to improved long-term clinical outcomes for a significant proportion of treated patients. TAVO in combination with another anti-PD-1 checkpoint inhibitor, KEYTRUDA (pembrolizumab), has already been shown to enhance overall response rate and partial tumor responses in patients with anti-PD-1 checkpoint-refractory metastatic melanoma in OncoSec’s KEYNOTE-695 registration directed Phase 2 clinical trial.

"While studies have shown relapse and overall survival advantages when checkpoint inhibitors are given alone following surgery, there is a need to investigate novel immunotherapeutic agents such as TAVO that can be given preoperatively in order to further enhance the clinical efficacy of immunotherapy in patients with advanced melanoma," said Armad A. Tarhini, M.D., Ph.D., Leader of the OMS-104 trial and Senior Member and Professor at the H. Lee Moffitt Cancer Center and Research Institute and the University of South Florida Morsani College of Medicine. "The neoadjuvant approach utilizing TAVO in combination with checkpoint inhibitors as being tested in this study may improve operability, pathologic tumor response and long-term disease control, which is highly desirable for these patients, who continue to have a high risk of recurrence and progression despite the use of standard therapy after surgery."

OMS-104 (NCT04526730) is a Phase 2 open-label, single arm study investigating intratumoral TAVO delivered by gene electrotransfer, or short electric pulses, plus nivolumab as neoadjuvant therapy in patients with operable locally-regionally advanced melanoma. The trial aims to enroll 33 patients and consists of three phases:

1) Neoadjuvant phase, where TAVO will be administered intratumorally using gene electrotransfer in three cycles on days one and eight every four weeks and nivolumab will be administered after TAVO on day eight of each cycle via 30-minute intravenous (IV) infusion;

2) Surgical phase consisting of a definitive surgery that will be scheduled 2-4 weeks after the last dose of nivolumab following radiologic and clinical assessment; and

3) Adjuvant phase, where nivolumab monotherapy will begin 2-4 weeks after surgery and will be administered for up to nine four-week cycles.

The primary endpoint is pathological complete response, estimated based on the proportion of participants with no viable tumor on histologic assessment at definitive surgery after the 12-week neoadjuvant period.

Daniel J. O’Connor, President and Chief Executive Officer of OncoSec, added, "TAVO delivers DNA plasmid-based IL-12 directly into the tumor using gene electrotransfer, which demonstrably enhances the immunogenicity of the treated tumors to yield productive ‘in situ’ vaccines. This principle has yielded striking results in post-PD-1 patients and is likely relevant in this earlier clinical setting. We look forward to exploring the utility of TAVO as a potential neoadjuvant therapy in a variety of solid tumor settings for patients in need of more effective treatment options."

About TAVO
OncoSec’s gene delivery technology combines TAVO (tavokinogene telseplasmid), a DNA plasmid-based interleukin-12 (IL-12), with an intra-tumoral gene delivery platform (gene electrotransfer) to achieve endogenous IL-12 production in the tumor microenvironment that enables the immune system to target and attack tumors throughout the body. TAVO has demonstrated a local and systemic anti-tumor response in several clinical trials, including the pivotal Phase 2b trial KEYNOTE-695 for metastatic melanoma and the KEYNOTE-890 Phase 2 trial in triple negative breast cancer (TNBC). TAVO has received both Orphan Drug and Fast-Track Designation by the U.S. Food & Drug Administration for the treatment of metastatic melanoma.

On January 8, 2021 KemPharm, Inc. (NASDAQ: KMPH), a specialty pharmaceutical company focused on the discovery and development of proprietary prodrugs, reported the pricing of an underwritten public offering of 7,692,307 shares of its common stock (or pre-funded warrants to purchase common stock in lieu thereof) and accompanying warrants to purchase up to 7,692,307 shares of common stock (Press release, KemPharm, JAN 8, 2021, View Source [SID1234573710]). Each share of common stock (or pre-funded warrant in lieu thereof) is being sold together with one warrant to purchase one share of common stock at a combined public offering price of $6.50. The aggregate gross proceeds from the offering are expected to total $50.0 million, before deducting the underwriting discounts and commissions and estimated offering expenses payable by KemPharm and without giving effect to proceeds from any subsequent exercise of warrants.

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KemPharm’s common stock will be listed on The Nasdaq Capital Market and will trade under the ticker symbol "KMPH" beginning today, January 8, 2021. The offering is expected to close on or about January 12, 2021, subject to customary closing conditions. In addition, KemPharm has granted to the underwriter a 45-day option to purchase up to 1,153,846 additional shares of its common stock and/or warrants to purchase up to 1,153,846 shares of its common stock, in any combination thereof, at the public offering price, less the underwriting discount.

Roth Capital Partners is acting as sole manager for the offering.

The securities described above are being sold by KemPharm pursuant to a registration statement filed by KemPharm with the Securities and Exchange Commission (the "SEC"), which was declared effective on January 7, 2021. The securities will be sold only by means of a prospectus, forming a part of the effective registration statement. Electronic copies of the accompanying prospectus may be obtained, when available, by contacting Roth Capital Partners, 888 San Clemente, Newport Beach, CA 92660, Attn: Prospectus Department, telephone: 800-678-9147, or email at [email protected], or by visiting the SEC’s website at View Source

On January 8, 2021 argenx (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases and cancers, reported its 2021 corporate priorities and highlighted recent achievements from its late-stage immunology pipeline driven by its FcRn antagonist, efgartigimod (Press release, argenx, JAN 8, 2021, View Source,of%20its%20clinical%2Dstage%20autoimmune [SID1234573709]). Additionally, the Company announced interim data from the Phase 2 CULMINATE trial of cusatuzumab in development with Cilag GmbH International, an affiliate of Janssen, and provided financial guidance for 2021.

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argenx previously announced an exclusive license agreement with Zai Lab Limited ("Zai Lab") for the development and commercialization of efgartigimod in Greater China and the acceleration of efgartigimod development through Phase 2 proof-of-concept trials in new autoimmune indications. Zai Lab will also contribute Chinese patients to argenx’s global Phase 3 trials of efgartigimod. Under the terms of the agreement, argenx will receive $175 million in collaboration payments comprised of upfront Zai Lab equity, a guaranteed development cost-sharing payment, and a milestone payment upon U.S. efgartigimod approval. argenx will also be eligible for tiered royalties based on annual net sales of efgartigimod in Greater China.

"We are excited to enter a new chapter for argenx as we look toward commercialization and achieving our mission of reaching patients with debilitating rare diseases. We’ve submitted a BLA to the FDA for efgartigimod in gMG and expect to have global efgartigimod trials ongoing this year in six indications and two formulations. We hope to continue to demonstrate the broad opportunity of our FcRn antagonist within autoimmune diseases in 2021 and beyond," said Tim Van Hauwermeiren, Chief Executive Officer of argenx. "In parallel, establishing global commercial infrastructure within the U.S. and Japan continues to be a top priority. Now through our collaboration with Zai Lab in China and with the appointment of a general manager in Europe, we’ve solidified and accelerated our capabilities to bring efgartigimod and our future immunology candidates to patients worldwide."

2021 Corporate Priorities and Recent Progress

The Company will continue its transition to a fully integrated immunology company by executing on three corporate priorities in 2021, including: preparation for the potential FDA approval and U.S. commercial launch of efgartigimod for the treatment of patients with gMG; the progression of its clinical-stage autoimmune pipeline; and the continued growth of its broad and differentiated pipeline through its Immunology Innovation Program.

Preparation for potential FDA approval and global commercial launch of efgartigimod for the treatment of patients with gMG

Submitted BLA to FDA for efgartigimod for treatment of gMG and continued preparations for other global regulatory submissions

On track to submit application to Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) in first half of 2021
On track to submit to European Medicines Agency (EMA) in second half of 2021
Submission in China expected to occur shortly following potential approval in U.S.
Initiated bridging study for SC efgartigimod in gMG based on association between total IgG reduction and clinical benefit, and feedback from the FDA. The study is a registrational, non-inferiority trial comparing the pharmacodynamic effect of 1000mg SC efgartigimod with 10mg/kg IV efgartigimod and is expected to enroll approximately 50 patients.
Commercial preparation activities are underway and on track for potential 2021 launch, including continued engagement with key stakeholders, commercial inventory build, milestone-based hiring of field force around potential BLA acceptance and FDA approval, and development of a patient services program.
Appointed Anant Murthy, Ph.D., as General Manager of argenx Europe. In this role, Dr. Murthy will establish the commercial infrastructure for a European launch and lead market development activities in advance of a potential European Medicines Agency (EMA) approval of efgartigimod. Dr. Murthy brings ~20 years of international experience to argenx, most recently as Head of Market Access for EMEA and Canada and the General Manager of multiple European countries for Alnylam Pharmaceuticals.
Progress clinical-stage autoimmune pipeline, including seven expected global trials of efgartigimod and Phase 1 trial of first-in-class C2 antibody ARGX-117

ADVANCE (IV) and ADVANCE SC trials ongoing evaluating IV and SC efgartigimod in patients with primary immune thrombocytopenia (ITP); global program expected to support registration of both formulations
ADDRESS registrational trial ongoing evaluating SC efgartigimod in patients with pemphigus vulgaris (PV) and pemphigus foliaceous (PF)
ADHERE trial ongoing evaluating SC efgartigimod in chronic inflammatory demyelinating polyneuropathy (CIDP);

Completed enrollment of first 30 patients
Decision whether to expand enrollment up to 130-140 patients expected in first quarter of 2021
Clinical trials in fifth and sixth indications of efgartigimod to begin enrollment in 2021
Ongoing Phase 1 healthy volunteer trial of IV and SC ARGX-117, a first-in-class C2 antibody, to evaluate safety and tolerability and establish dosing regimen

Data expected in mid-2021, after which argenx plans to launch Phase 2 proof-of-concept trials in severe autoimmune diseases, including multifocal motor neuropathy (MMN)
Continued investment in broad and differentiated pipeline through Immunology Innovation Program

Preclinical work ongoing in early-stage pipeline, including continued progress on ARGX-118 and ARGX-119, and the optimization of ARGX-120
Commitment to expand pipeline at cadence of one new candidate per year from Immunology Innovation Program

Interim Data from Cusatuzumab Phase 2 CULMINATE Trial

Development of cusatuzumab in acute myeloid leukemia (AML) remains ongoing as part of a global collaboration and license agreement with Cilag GmbH International, an affiliate of Janssen.

The Phase 2 CULMINATE trial (NCT04023526) is evaluating cusatuzumab in combination with azacitidine in newly-diagnosed, elderly AML patients who are ineligible for intensive chemotherapy. A total of 103 patients were randomized to receive either 10mg/kg (n=51) or 20mg/kg (n=52) cusatuzumab plus azacitidine as part of a dose identification. The 20mg/kg dose has been selected for ongoing and future trials.

A pre-planned interim analysis was conducted of the 52 patients (46.2% adverse ELN risk classification) receiving 20mg/kg cusatuzumab plus azacitidine treatment (intent-to-treat population (ITT)). The results from the ITT analysis showed a complete remission (CR) rate of 27% (14/52) and composite complete remission (CRc), including CRs with incomplete hematologic recovery, rate of 40% (21/52). The 30-day mortality rate of the ITT population was 9.6% (5/52). In a cohort where patients received at least two treatment cycles (20mg/kg cusatuzumab plus azacitidine), 42% (14/33) achieved CR and 64% (21/33) achieved CRc.

Cusatuzumab was observed to be well-tolerated and the safety profile was consistent with prior studies. Final results from the CULMINATE trial will be presented in a peer-reviewed forum.

The decision to initiate additional studies in the development of cusatuzumab, under the collaboration, will be determined following review of data from the ongoing Phase 1b ELEVATE trial (NCT04150887), which is evaluating cusatuzumab in combination with venetoclax and azacitidine in newly-diagnosed, elderly patients with AML who are ineligible for intensive chemotherapy.

Financial Guidance

As of December 31, 2020, argenx had approximately $2.0 billion in cash, cash equivalents and current financial assets. This preliminary cash balance does not include expenses or proceeds from recently announced business development transactions, including the purchase of a priority review voucher from Bayer HealthCare Pharmaceuticals, Inc. and the exclusive license agreement with Zai Lab for efgartigimod in Greater China.

Based on current plans to fund anticipated operating expenses and capital expenditures, argenx expects its cash burn to increase significantly in 2021, approximately doubling compared to 2020. The increased spend will support the Company’s transition to an integrated immunology company in 2021, including the build-out of global commercial infrastructure and drug product inventory ahead of the expected launch of efgartigimod in gMG in the U.S, the advancement of its clinical-stage pipeline, including seven expected global trials of efgartigimod, and the continued investment in its Immunology Innovation Program.

J.P. Morgan Healthcare Conference Presentation and Webcast

argenx CEO Tim Van Hauwermeiren, will present these updates at the virtual 39th Annual J.P. Morgan Healthcare Conference on Monday, January 11, 2020 at 8:20 a.m. ET, followed by a question and answer session.

The live webcast of the presentation and question and answer session that follows may be accessed on the homepage of the argenx website at www.argenx.com. A replay of the webcast will be available for 90 days on the argenx website.

About Efgartigimod

Efgartigimod is an investigational antibody fragment designed to reduce disease-causing immunoglobulin G (IgG) antibodies and block the IgG recycling process. Efgartigimod binds to the neonatal Fc receptor (FcRn), which is widely expressed throughout the body and plays a central role in rescuing IgG antibodies from degradation. Blocking FcRn reduces IgG antibody levels representing a logical potential therapeutic approach for several autoimmune diseases known to be driven by disease-causing IgG antibodies, including: myasthenia gravis (MG), a chronic disease that causes muscle weakness; pemphigus vulgaris (PV), a chronic disease characterized by severe blistering of the skin; immune thrombocytopenia (ITP), a chronic bruising and bleeding disease; and chronic inflammatory demyelinating polyneuropathy (CIDP), a neurological disease leading to impaired motor function.

On January 8, 2021 Neurocrine Biosciences, Inc. (Nasdaq: NBIX) reported an update on its business performance, including preliminary net product and inventory adjusted sales results of INGREZZA (valbenazine) for 2020, and key commercial and clinical development milestones for 2021 (Press release, Neurocrine Biosciences, JAN 8, 2021, View Source [SID1234573708]). Kevin Gorman, Chief Executive Officer of Neurocrine Biosciences, will discuss these updates as part of a webcast presentation at the 39th Annual J.P. Morgan Healthcare Conference to be held virtually on Monday, January 11 at 2:00 p.m. Eastern Time, followed by a Question and Answer session at approximately 2:20 p.m. Eastern Time.

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Preliminary Fourth Quarter and Full-Year 2020 INGREZZA (valbenazine) Net Product Sales and Inventory Adjusted Net Product Sales (Unaudited)

Based on preliminary unaudited financial information, the Company expects INGREZZA net product sales for the three months and full-year ended December 31, 2020 to be approximately $240 million and $993 million respectively. Preliminary analysis of INGREZZA net product sales suggests:

– INGREZZA inventory adjusted net product sales for the fourth quarter were approximately $258 million reflecting an $18 million channel inventory decrease in Q4

– New prescriptions and refills increased in the fourth quarter of 2020 vs. the third quarter of 2020

– Full-year 2020 total INGREZZA prescriptions grew 32% to approximately 175,700 versus 2019 total prescriptions of approximately 132,700

"I am very proud of our team’s resilience and adaptability to bring INGREZZA to patients living with tardive dyskinesia this past year and we remain encouraged by the continued strength in persistence and refill rates, which is a testament to the many benefits of INGREZZA. We also continue to focus on healthcare provider educational initiatives, patient outreach programs and investing in telemedicine capabilities to improve diagnosis and treatment rates for the estimated 80% of patients with tardive dyskinesia who have not yet been diagnosed," said Kevin Gorman, Ph.D., Chief Executive Officer of Neurocrine Biosciences. "Adding to our movement disorder portfolio, we launched our second commercial treatment, ONGENTYS, and continue to make great progress in our development programs with plans to initiate seven mid-to-late stage clinical studies in 2021 focused on neurological, endocrine and psychiatric disorders."

2021 Expected Milestones and Key Activities

Program

Indication

2021 Milestones / Key Activities

Valbenazine

Chorea in Huntington Disease

Phase III Top-Line Data Expected in Q4 2021

Neurological Indication

Initiate Phase III

Psychiatric Indication

Initiate Phase II

Crinecerfont

Congenital Adrenal Hyperplasia (Adult)

Continue Phase III Enrollment

Congenital Adrenal Hyperplasia (Pediatric)

Initiate Phase III

NBI-1065844

Negative Symptoms of Schizophrenia

Phase II Top-Line Data Expected in

1st Half of 2021

NBI-1065845

Treatment Resistant Depression

Initiate Phase II

NBI-1065846

Anhedonia in Depression

Initiate Phase II

NBI-827104

Rare Pediatric Epilepsy:

Epileptic Encephalopathy with Continuous Spike and Wave During Sleep

Continue Phase II Enrollment

Neurological Indication

Initiate Phase II

NBI-921352

Focal Onset Seizure in Adults

Initiate Phase II

Rare Pediatric Epilepsy: SCN8A-DEE

Ongoing Dialogue with FDA

NBIb-1817

Gene Therapy for Parkinson’s Disease

Determine Regulatory Path with FDA

About Tardive Dyskinesia (TD)
Tardive dyskinesia (TD) is a movement disorder that is characterized by uncontrollable, abnormal and repetitive movements of the face, torso and/or other body parts, which may be disruptive and negatively impact patients. The condition is caused by prolonged use of treatments that block dopamine receptors in the brain, such as antipsychotics commonly prescribed to treat mental illnesses such as schizophrenia, bipolar disorder and depression, and certain anti-nausea medications. In patients with TD, these treatments are thought to result in irregular dopamine signaling in a region of the brain that controls movement. The symptoms of TD can be severe and are often persistent and irreversible. TD is estimated to affect at least 500,000 people in the U.S.

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 Biosciences’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 Information
Approved Use
INGREZZA (valbenazine) capsules is a prescription medicine used to treat adults with movements in the face, tongue, or other body parts that cannot be controlled (tardive dyskinesia).

It is not known if INGREZZA is safe and effective in children.

Important Safety Information
Do not take INGREZZA if you are allergic to valbenazine, or any of the ingredients in INGREZZA.

INGREZZA may cause serious side effects, including:

Sleepiness (somnolence). Do not drive, operate heavy machinery, or do other dangerous activities until you know how INGREZZA affects you.
Heart rhythm problems (QT prolongation). INGREZZA may cause a heart problem known as QT prolongation.
Symptoms of QT prolongation may include: fast, slow, or irregular heartbeat, shortness of breath, dizziness or fainting.
Parkinson-like symptoms. Symptoms include: shaking, body stiffness, trouble moving or walking, or keeping your balance.
Tell your healthcare provider right away if you have a change in your heartbeat (a fast or irregular heartbeat), or if you faint.

Before taking INGREZZA, tell your healthcare provider about all of your medical conditions including if you: have liver or heart problems, are pregnant or plan to become pregnant, or are breastfeeding or plan to breastfeed.

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins and herbal supplements.

The most common side effect of INGREZZA is sleepiness (somnolence). Other side effects include changes in balance (balance problems, dizziness) or an increased risk of falls, headache, feelings of restlessness, dry mouth, constipation, and blurred vision.

These are not all of the possible side effects of INGREZZA. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.