Antivirals & Antitoxins Program

David Boucher, PhD Project Officer, CBRN Antivirals and Antitoxins October 29, 2018

October 29-30, 2018 | Grand Hyatt • Washington, D.C. BARDA Chemical Biological Radiological & Nuclear Portfolio at a Glance: Antivirals & Antitoxins Branch IND NDA/BLA

Preclinical Production Discovery Phase I Phase II Phase III Licensure Development & Delivery

Filovirus Antiviral Antiviral Anthrax Antitoxin

Filovirus Antiviral Botulism Antitoxin

Smallpox Antiviral

Saving Lives. Protecting Americans. 2 Anthrax Antitoxin Program

OBJECTIVE Develop safe and effective anthrax antitoxins to treat inhalational anthrax

Approved Products: All three products are in the Strategic National Stockpile and NOV available for deployment 2012 (Emergent, formerly GSK & HGS)

MAR AIG IV (Anthrasil) 2015 (Emergent, formerly Cangene) MAR (Anthim®) 2016 (Elusys)

Saving Lives. Protecting Americans. 3 Anthrax Antitoxins: Program Strategy and Opportunities

Continuing Program Opportunities Efforts Objectives

• Coordinate with industry • Further diversify the • BARDA BAA, AOI #2.1, solicits and ASPR/SNS to ensure portfolio and, in turn, TRL-6 or greater candidates that medical countermeasure our response meet one or more of the following preparedness capabilities requirements: • Extend shelf-life and • Are peptides, small molecules or improve concept of novel compounds; operations of existing • Have a unique mechanism of action; products • Provide a transformational change in • Support completion of operational use; post-marketing • Provide a transformational change in commitments Total Life Cycle Costs

Saving Lives. Protecting Americans. 4 Botulinum Antitoxin Program

OBJECTIVE Develop safe and effective botulism antitoxins to treat botulism intoxication caused by serotypes A-G

Approved Products:

MAR hBAT Emergent, formerly Cangene) 2013

hBAT is in the Strategic National Stockpile and available for deployment

Saving Lives. Protecting Americans. 5 Botulinum Antitoxins: Program Strategy and Opportunities

Continuing Program Opportunities Efforts Objectives

• Coordinate with industry • Further diversify the • BARDA BAA, AOI #2.2, solicits and ASPR/SNS to ensure portfolio and, in turn, candidates that meet one or more of the medical countermeasure our response following requirements: preparedness capabilities • Are mAbs, peptides, small molecules or novel • Support post-marketing compounds; commitments • Have a unique mechanism of action; • Provide a transformational change in operational use; • Provide a transformational change in Total Life Cycle Costs • Candidates at TRL-5 are preferred; however, earlier candidates can be considered based on program impact

Saving Lives. Protecting Americans. 6 Smallpox Antiviral Program OBJECTIVE Develop safe and effective treatment options (multiple antiviral drugs with different mechanisms of action) for individuals with smallpox disease

Approved Products: JUL ST-246/TPOXX ST-246/TPOXX is in the 2018 (SIGA) Strategic National Stockpile and available for deployment

Ongoing Program: CMX-001/ (Chimerix)

Saving Lives. Protecting Americans. 7 Smallpox Antivirals: Program Strategy and Opportunities

Continuing Program Opportunities Efforts Objectives

• Coordinate with industry • Further diversify the • BARDA BAA, AOI #2.3, solicits and ASPR/SNS to ensure portfolio and, in turn, candidates with evidence of antiviral medical countermeasure our response activity against . preparedness capabilities • Candidates at TRL-5 are • Extend shelf-life and preferred; however, earlier improve concept of candidates can be considered operations of existing based on program impact products • Support completion of post-marketing commitments

Saving Lives. Protecting Americans. 8 Filovirus Antiviral Program ( Zaire)

OBJECTIVE Develop safe and effective treatment options for individuals with viral hemorrhagic fever caused by filovirus

ARD Program PBS Program PBS Program /BCX4430 (BioCryst) ZMapp (Mapp ) REGN-EB3 (Regeneron)

Both ZMapp and REGN-EB3 are being made available under EAP/MEURI

Saving Lives. Protecting Americans. 9 Filovirus Antiviral Program (Sudan and )

OBJECTIVE Develop safe and effective treatment options for individuals with viral hemorrhagic fever caused by filovirus infection

Manufacturing material that “Innovation” ARD Program could be used as part of a MBP091 (Mapp Biopharmaceutical) response to • Targets the Marburg MBP134 (Mapp Biopharmaceutical) outbreaks/high-risk • Targets the Sudan virus exposures for which there are no available MCMs is a ARD Program priority MBP134 (Mapp Biopharmaceutical)

Saving Lives. Protecting Americans. 10 Filovirus Antiviral Program Strategy & Opportunities

Continuing Program Opportunities Efforts Objectives

• Advance PBS Programs to • Further diversify the • BARDA BAA, AOI #2.2, solicits licensure and continue to Zaire portfolio candidates at TRL-5 or higher and, make those products for Ebola Zaire, meet one or more of available when needed • Build the Sudan and Marburg portfolios the following requirements: • Continue to support field • Are single mAbs, peptides, small use of filovirus molecules or novel compounds; therapeutics through EAP, RCT & MEURI • Have a unique mechanism of action; • Provide a transformational change in operational use; • Provide a transformational change in Total Life Cycle Costs

Saving Lives. Protecting Americans. 11 BARDA’s Antivirals & Antitoxins Team

Medical Countermeasures Contracts Management & Subject Matter Experts Program Acquisitions

• Chia-Wei Tsai, Branch Chief • Clinical Development • Jason Bell • David Boucher • Pharmaceutical • Brooke Bernold • Clay Hughes Countermeasure • Wendell Conyers • Malen Link Infrastructure • Erin Greninger • Michael Merchlinsky • Non-clinical • Phil Hastings Development • Kimberly Sciarretta • Francine Hemphill • Regulatory & Quality • David Simon Assurance • Ethan Muller • Danielle Turley • Oke Obi • Carol Diaz-Diaz, ORISE Fellow • Matthew Rose

Saving Lives. Protecting Americans. 12 How to contact BARDA

medicalcountermeasures. www.fbo.gov/ https://www.phe.gov/about/ www.drive.hhs.gov gov/home.aspx (“FedBizOpps”) BARDA/Pages/default.aspx DRIVe questions Portal to BARDA: Register to Official announcements and Program description, information, request a TechWatch meeting! info for all government news, announcements contract solicitations

https://www.usajobs.gov/ Join the team!

Saving Lives. Protecting Americans. 13