Indication intelligence, analysis and sector perception

Juno Therapeutics’ JUNO) and Celgene’s (CELG) Investigational Drug JCAR017 granted Breakthrough Therapy Designation from FDA and Priority Medicines Eligibility from EMA for Relapsed/Refractory Diffuse Large B-cell Lymphoma

December 20, 2016 9:36am

JUNO closed at $19.84 and was UP +$1.13 in the pre-open and +$0.64 at the open - Maintaining BUY

The U.S. FDA has granted Breakthrough Therapy designation to investigational drug JCAR017 for the treatment of patients with relapsed/refractory (r/r) aggressive large B-cell non-Hodgkin lymphoma (NHL), including diffuse large B-cell lymphoma (DLBCL), not otherwise specified (de novo or transformed from indolent lymphoma), Primary Mediastinal B-cell Lymphoma (PMBCL) or Grade 3B Follicular Lymphoma.

In addition, the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) and Committee for Advanced Therapies (CAT) have granted JCAR017 access to the Priority Medicines (PRIME) scheme for r/r DLBCL.

JCAR017 uses a defined CD4:CD8 cell composition and 4-1BB as the costimulatory domain, which differentiates it from other CD19-directed CAR T product candidates in clinical development. Earlier this month, data from multiple phase I studies with JCAR017 in non-Hodgkin lymphoma and pediatric acute lymphoblastic leukemia were presented at the 58th American Society of Hematology Annual Meeting.

The Bottom Line: The Breakthrough Therapy designation and PRIME eligibility were granted by the FDA and EMA, respectively, on the basis of early clinical results with JCAR017 in r/r DLBCL. According to the FDA, Breakthrough Therapy designation is intended to expedite the development and review of medicines with early evidence of potential clinical benefit in serious diseases. PRIME was launched by the EMA earlier this year and enables accelerated assessment of therapeutic applications that target an unmet medical need, focusing on medicines that may offer a major therapeutic advantage over existing treatments, or benefit patients with no treatment options.

JCAR017 is not approved in any country. FDA’s Breakthrough Therapy designation and access to EMA’s Priority Medicines scheme does not constitute or guarantee a future approval.

JUNO closed at $19.84 and was UP +$1.13 in the pre-open - BUY

35 companies, 1 interpreter!

Insight, foresight and recommendation

My motto … never leave an investor uninformed! I say today what others won’t, so you can do what others can’t.

My name is Henry McCusker; I endorse these opinions under my own name and with a subscription based membership.

Do you have an opinion you would like to share with us? We would like to hear from you.

Please e-mail us at hwm@regmedinvestors.com.

Editor and Publisher ... Henry McCusker enters his tenth (10th) year at RegMed Investors with past experience in sell-side analysis, buy-side management, corporate management and due diligence.

RegMed Investors (RMi) or Regmedinvestors.com content is the intellectual property of Henry McCusker. Any copying, republication or redistribution of RMI’s content, including by framing or similar means, is expressly prohibited without the prior written consent of Scimitar; who also shall not be liable for any errors or delays in content, or for any actions taken in reliance thereon. For additional information on other Scimitar media services or reposting please email hwm@regmedinvestors.com.

The information included above as well as individual companies and/or securities mentioned should be construed as guidance in RMi reviews.

The facts stated and the opinions given in this publication are based on data and information considered to be reliable and have been carefully worked into my qualitative analyses and prognoses. However, no guarantee can be given as to their fairness, accuracy or completeness.

Henry McCusker, the editor and publisher of RegMed Investors does not hold or have positions in securities referred to in this publication.