Panel Discussions

Wednesday, June 20, 2018

Gene Editing: From Science to Reality

8:00 AM

Moderator:
Liisa Bayko, Senior Analyst, Biotechnology

Panelists:
Samarth Kulkarni, Ph.D., Chief Executive Officer, CRISPR Therapeutics AG (CRSP)
Andrew Hack, Chief Financial Officer, Editas Medicine, Inc. (EDIT)
Thomas Barnes, Senior Vice President, Innovative Sciences, Intellia Therapeutics, Inc. (NTLA)

Description:
Join us for a discussion with the leading edge companies in gene editing to discuss the path from the bench to the clinic and the state of the art technology at play.

Emerging Landscape of Treatment Options in PAH


9:00 AM

Moderator:
Jason Butler, Senior Analyst, Biotechnology

Panelists:
Amit Munshi, President & Chief Executive Officer , Arena Pharmaceuticals, Inc. (ARNA)
Fabian Tenenbaum, Chief Executive Officer, Bellerophon Therapeutics, Inc. (BLPH)

Description:
This panel will focus on differentiated approaches for pulmonary arterial hypertension (PAH), a serious condition with a high morbidity and mortality burden. A number of important drugs are approved for PAH, yet an unmet need for greater efficacy and tolerability remains. We estimate a total U.S. market opportunity of >$4Bn. Given the remaining unmet need and large market potential, we believe this will continue to be an interesting and important area for investors.

Assessing the Market Opportunity for Peanut Allergy Immunotherapy

10:00 AM

Moderator:
Liisa Bayko, Senior Analyst, Biotechnology

Panelists:
Stephen Dilly, Special Advisor, Aimmune Therapeutics, Inc. (AIMT)
Susanna Mesa, Chief Business Officer, DBV Technologies S.A. (DBVT)
Mary Jane Marchisotto, Senior Vice President of Research and International Operations, F.A.R.E.

Description:
With two late-stage peanut allergy immunotherapies going in front of FDA this year, many are trying to get a better grasp of the market opportunity. Peanut allergy is undoubtedly a large, untapped market with >2M cases in the U.S. alone and no FDA-approved products, but only time will tell how many of these patients will eventually adopt these newer treatment options. This panel will discuss commercial dynamics ahead of what could be an exciting new drug category.

Current Trends in Therapeutic and Aesthetic Dermatology

11:30 AM

Moderator:
Don Ellis, Senior Analyst, Specialty Pharmaceuticals

Panelists:
Robert Kaplan, MD, Dermatologist
Neal Walker, President and Chief Executive Officer, Aclaris Therapeutics, Inc. (ACRS)
Mihael Polymeropoulos, President and Chief Executive Officer, Vanda Pharmaceuticals Inc. (VNDA)

Description:
This panel will focus on the treatment of both aesthetic and therapeutic conditions with Dr. Robert Kaplan. Dr. Kaplan is a board certified Dermatologist in Memphis, Tennessee. The panel will feature Aclaris Therapeutics and Vanda Pharmaceuticals. The panel will discuss the recent launch of ESKATA including Dr. Kaplan’s initial impressions of efficacy. We will also discuss the market opportunities in molluscum contagiosum and common warts. Aclaris is also developing JAK (1/3) inhibitors for alopecia and we will discuss their MOA in treating alopecia. Finally, we will discuss Vanda’s pruritus compound, tradipitant, which is expected to enter Ph.3 studies in atopic dermatitis in 2018 and which JMP models peak sales of >$500M.

Unmet Needs and Novel Approaches to Treat CNS Disorders

1:00 PM

Moderator:
Jason Butler, Senior Analyst, Biotechnology

Panelists:
Lucy Lu, Chief Executive Officer, Avenue Therapeutics, Inc. (AXTI)
Michael Cola, Chief Executive Officer, Aevi Genomic Medicine, Inc. (GNMX)
Jonathan Javitt, Executive Chairman and Chief Executive Officer, NeuroRX, Inc. (Private)
Yaron Werber, Chief Business and Financial Officer, Ovid Therapeutics Inc. (OVID)

Description:
This panel will focus on unmet need and therapeutics in development for a wide range of CNS indications of varying severity, unmet need, and prevalence. Each of the companies has early to mid-stage clinical programs ongoing for their novel approaches to treating CNS disorders. Diseases affecting the CNS remain one of the largest burdens in healthcare and effective treatments can be transformational for patients and caregivers, thus driving considerable value to the system and investors.

Bioelectronic Medicine: The Accelerating Therapeutic Opportunity Straddling the Drug-Device Divide

2:30 PM

Moderator:
David Turkaly, Senior Analyst, Medical Devices & Supplies

Panelists:
Ray Cohen, Chief Executive Officer, Axonics (Private)
Milton Morris, Ph.D., President and Chief Executive Officer, NeuSpera Medical (Private)
Scott Drees, Chief Executive Officer, Nuvectra Corporation (NVTR)

Description:
Featuring CEOs of three companies in the arena, the bioelectronic medicine panel will focus on novel urologic treatments (for issues like OAB and Incontinence), regulatory pathways and timelines, reimbursement, and other potential opportunities. Participants will include Scott Drees from Nuvectra, Ray Cohen from Axonics Modulation, and Milton Morris, Ph.D. from NeuSpera. We believe that one of the most interesting new therapeutic evolutions in health care is occurring in the emerging field of bioelectronics. Delivering medical therapies in an electronic fashion allows for precise disease state targeting without the systemic effects of pharmaceuticals. These technologies are straddling the device-drug continuum, allowing the potential for a pharma model housed in a medtech delivery mechanism (think electronic dosing and prescriptions). Given the potential for what we see as disruptive launches and outsized returns ahead, this new subsector has already garnered significant attention of late from the well-established medtech players in addition to many pharmaceutical and technological powerhouses. Featured technologies are aimed at a wide range of unmet / undermet clinical needs including: overactive bladder, incontinence, migraines, cluster headaches, GERD, sleep apnea, back pain, epilepsy, chronic pain, post-surgical pain, post-amputation pain, depression, and inflammatory diseases such as rheumatoid arthritis and Crohn’s Disease. Looking forward, there are potential applications in a range of other disease states from diabetes and obesity to hypertension and pulmonary diseases, to name a few.

Combination Strategies in Immuno-Oncology

3:30 PM

Moderator:
Konstantinos Aprilakis, Senior Analyst, Biotechnology

Panelists:
Phil Serlin, Chief Executive Officer, BioLineRx Ltd. (BLRX)
Keith Orford, Senior Vice President of Clinical Development, Calithera Biosciences, Inc. (CALA)
Henry Adewoye, Chief Medical Officer, Compugen Ltd. (CGEN)
Michael Metzger, President and Chief Operating Officer, Syndax Pharmaceuticals, Inc. (SNDX)

Description:
Recent clinical and commercial success with immune checkpoint blockade and cell-based therapy approaches has resurrected intense scientific interest in cancer immunotherapy. Our panel includes companies in our coverage universe that we believe have the potential to make an impact on this remarkably exciting field. With approaches ranging from immune cell mobilization to epigenetic modulation and metabolic conditioning, our panel participants are slated to discuss the opportunities afforded by the field as well as the challenges posed by it, and how their individual development strategies have been designed to maximize the chances of ultimate success in immuno-oncology. Key topics slated for discussion include next-generation immuno-oncology therapeutics, novel targets (e.g., inhibitory checkpoints, co-stimulatory molecules, etc.), biomarker strategies, the future of combination therapies, and challenges associated with drug cost and coverage.

Panel Discussions

Thursday, June 21, 2018

NASH: Making Sense of the Pipeline

8:00 AM

Moderator:
Liisa Bayko, Senior Analyst, Biotechnology

Panelists:
Sujal Shah, President and Chief Executive Officer, CymaBay Therapeutics (CBAY)
Jay Luly, President and Chief Executive Officer, Enanta Pharmaceuticals, Inc. (ENTA)
Frederic Cren, Chief Executive Officer, Inventiva SA (EPA: IVA)
Becky Taub, Director, Founder, Chief Medical Officer and Executive Vice President, Research & Development, Madrigal Pharmaceuticals, Inc. (MDGL)
Scott L. Friedman, M.D., Dean for Therapeutic Discovery and Chief of the Division of Liver Diseases, at the Icahn School of Medicine at Mount Sinai

Description:
Today there are dozens of clinical programs pursuing NASH and as of yet, no mechanism has clearly differentiated itself as the favorite. Over the next year, data from key program representing several novel and distinct targets will be revealed which will begin to shape the first wave of therapeutic intervention for this epidemic. Will there be a magic bullet or different mechanism for different disease stages? Will it be mono or combination therapy? Will one mechanism emerge as a backbone? How are patients going to be identified as candidates for therapy and how will clinical response be assessed? Our panel of companies representing different disease classes will discuss and debate these topics.

Novel Treatments in Pain Management

9:30 AM

Moderator:
Jason Butler, Senior Analyst, Biotechnology

Panelists:
Blair Clark-Schoeb, Executive Vice President, Communications, Egalet Corporation (EGLT)
Barry Quart, Chief Executive Officer, Heron Therapeutics, Inc. (HRTX)
Carrie Bourdow Chief Operating Officer, Trevana, Inc. (TRVN)

Description:
Pain control in multiple settings remains an important incompletely-fulfilled need, particularly in light of the continued difficulties with opioid abuse and misuse. The companies on this panel are developing novel approaches to treating pain, from abuse deterrent formulations and delivery methods, to molecules that act through a unique pain pathway. We will discuss the important aspects of the companies’ different approaches and the evolution of the market for pain control. We think investors can benefit from these novel approaches.

Renal Disease: Recent Advancements and Future Prospects

11:00 AM

Moderator:
Jason Butler, Senior Analyst, Biotechnology

Panelists:
John Butler, Chief Executive Officer, Akebia Therapeutics, Inc. (AKBA)
Scott Holmes, Chief Financial Officer, Keryx Biopharmaceuticals, Inc. (KERX)
Timothy Noyes, President and Chief Executive Officer, Proteon Therapeutics, Inc. (PRTO)

Description:
The renal space is complicated from multiple angles, including the biologic bases of disease, therapeutic approaches, and the reimbursement environment. The companies on our panel are developing, or have approved, novel approaches to addressing important un-met needs in the renal space.

Latest Developments in Back-of-the-Eye Therapies

1:00 PM

Moderator:
Don Ellis, Senior Analyst, Specialty Pharmaceuticals

Panelists:
Daniel White, Chief Executive Officer, Clearside Biomedical, Inc. (CLSD)
Dr. Arunan Sivalingam, Co-Director of the Retina Service, WillsEye Hospital

Description:
The panel will discuss innovations in treating back of the eye diseases with the Co-Director of the Retina Service at WillsEye Hospital and Clearside Biomedical. Clearside is developing transformative, elegant, precise solutions to restore and preserve vision. By leveraging exclusive access to and proprietary technology for suprachoroidal injection of drugs to access the retina and choroid of the eye, we believe that clinicians can more effectively treat diseases like uveitis, retinal vein occlusion (RVO) and diabetic macular edema (DME). The panel will discuss the current diagnosis methods, evaluation and treatment of DME & RVO. We will also discuss how CLS-TA may affect the standard of practice in treating macular edema related diseases. Finally, we will discuss the likely responses physicians and managed care will have to a potential FDA approval of CLS-TA.