Investors Update: Abuse-Deterrent d-amphetamine Immediate Release (ADAIR) for ADHD

Transcription

1 Investors Update: Abuse-Deterrent d-amphetamine Immediate Release (ADAIR) for ADHD February 7,

2 Forward-Looking Statements This presentation contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995 and other Federal securities laws. Because such statements deal with future events and are based on Alcobra's current expectations, they are subject to various risks and uncertainties and actual results, performance or achievements of Alcobra could differ materially from those described in or implied by the statements in this press release. For example, forwardlooking statements include statements regarding the timing of filing an NDA for ADAIR, if filed at all, the proposed development path to approve ADAIR, the sufficiency of Alcobra s financial resources for its future plans and the potential benefits of ADAIR. In addition, historic results of scientific research do not guarantee that the conclusions of future research would suggest similar conclusions or that historic results referred to in this press release would be interpreted similarly in light of additional research or otherwise. The forward-looking statements contained or implied in this presentation are subject to other risks and uncertainties, including those discussed under the heading "Risk Factors" in Alcobra's Annual Report on Form 20-F for the fiscal year ended December 31, 2015, filed with the Securities and Exchange Commission (SEC) and in subsequent filings with the SEC. Except as otherwise required by law, Alcobra disclaims any intention or obligation to update or revise any forward-looking statements, which speak only as of the date they were made, whether as a result of new information, future events or circumstances or otherwise. 2

3 Alcobra is Developing ADAIR, an Abuse-Deterrent, Immediate-Release Amphetamine ADAIR is a proprietary oral formulation of immediate-release (IR) dextro-amphetamine, designed to deter abuse by snorting or injecting Developed in collaboration with Capsugel, a global leader in delivering high-quality, innovative dosage forms and solutions Recent Pre-IND meeting with FDA defined a 505(b)(2) development path, targeting an NDA submission in the second half of 2018 Alcobra expects the ADAIR development path to be completely supported by existing cash balance There are currently no FDA-approved abuse-deterrent immediate release stimulants 3

4 FDA Commentary, Sep 18, 2014 The Agency agrees that the abuse and misuse of controlled substances, including CNS stimulant drugs, present a serious public health concern, and we support efforts by drug manufacturers to modify formulations to reduce the risk of abuse. Janet Woodcock, MD, Director Center for Drug Evaluation & Research, FDA 4

5 Stimulants are Scheduled, Controlled Substances with High Potential for Abuse and Addiction According to FDA and DEA: Prescribing information, Adderall Nearly two million people misuse or abuse prescription stimulants annually Source: National Survey on Drug Use & Health, 2015; Monitoring the Future Study,

6 Stimulants are Extensively Diverted % Approached to Divert 1 Medications in Lifetime (Adolescents) % Actual Diversion of Prescribed Stimulant (College Students) Sleeping Medication (n=108) Anti-anxiety Medication (n=104) Stimulant Medication (n=141) Opioid Medication (n=704) Source: McCabe et al. Drug Alc Depend 2011 Rabiner et al., 2009 (n=115), previous 6 months Sepulveda et al., 2011 (n=50), previous 12 months 1 Drug diversion = Transfer of legally prescribed controlled substance to another person by selling, trading, or giving away Garnier et al., 2010 (n=81), in a lifetime 6

7 IR Stimulants are Abused More Frequently Illicit Use Survey in ADHD Clinic 78 of 545 (14%) reported stimulant abuse Internet Survey of 10,000 Adults years old (% of respondents) TYPE OF STIMULANT ABUSED Long acting 17% Both 3% Short acting 80% Bright,G. Medscape Jrnl. of Medicine 2008; 10 (5): 111 Cassidy, et al, Jrnl. Attention Disorders, (7)

8 Snorting is a Frequent Route of Abuse 60% 50% Method of Stimulant Abuse by College Students (n=1,025) 55% 80% 70% 60% 50% 74% Method of Stimulant Abuse by Adults Admitted to Abuse Treatment Centers (n=1,905) 40% 30% 40% 40% 30% 38% 20% 20% 10% 0% 4% Swallow Snort Other 10% 0% 10% 3% Swallow Snort Inject Smoke White et al, Stimulant Medication Use, Misuse, and Abuse in an Undergraduate and Graduate Student Sample, JRN of Am College Health, 2006; Random sample: anonymous surveys at the University of New Hampshire administered via and paper, 1,025 received out of 5,000 distributed, 6.6% diagnosed with ADHD, over 16% of students abuse stimulants Cassidy, et al, Nonmedical Use of Prescription ADHD Stimulant Medications Among Adults in a Substance Abuse Treatment Population: Early Findings From the NAVIPPRO Surveillance System, Journal of Attention Disorders,

9 IR Amphetamines are the Fastest Growing Prescription Class of Stimulants in the US TRx Growth By Class vs. Previous Year Short Acting AMPH Long Acting AMPH Short Acting MPH 20.7% 19.5% 12.1% 9.5% 14.6% 12.7% 12.3% 16.8% 14.9% 14.0% 3.8% 5.8% 5.0% 7.3% 6.1% 8.4% 9.4% 11.8% 1.5% 0.0% 1.1% Long Acting MPH 1.2% 3.1% 2.0% -3.3% 2.5% 1.3% -0.1% Non-Stimulants -5.9% 28.5% 22.2% 10.9% 4.4% -0.6% 1.3% 24M IR Stimulants TRx during 2015 in the US IMS, NPA,

10 Abuse Deterrent d-amphetamine Immediate Release (ADAIR) 10 10

11 Alcobra s ADAIR: Designed to Deter Intranasal & IV Abuse An oral, semi-solid, gelatin capsule Developed under a services agreement with Capsugel Patent-pending, potentially first in class product Formulation resists manipulation/preparation for insufflation ( snorting ) Meaningful barriers for abuse via injection 11

12 Resists Preparations for Insufflation ADAIR d- AMPH Placed in electric grinder Ground Transferred for particle size analysis Each sieve is weighed <1% of ADAIR mass is under 500µm in size (vs % of reference drug) 12

13 Includes Meaningful Barriers When Manipulated for Injection Purposes Viscosity Comparison of Water, d-amph and ADAIR utilizing Texture analysis ADAIR Texture analysis profiles of water (blue), filtered manipulated d-amph (red) and ADAIR (green) for depressing a plunger of a Leur-Lok 5 ml syringe by 9 mm, whilst expelling the material under test through a 26 G needle (Internal Data) 13

14 ADAIR Commercial Potential is Strong Multiple rounds of market research with high prescribing ADHD physicians demonstrate strong interest in prescribing ADAIR Intention to prescribe for >50% of patients who otherwise would receive an IR amphetamine Market research with Managed Care payers representing >100 million covered lives suggests acceptability of the product concept without intention to block access Consumer research with mothers of teenagers with ADHD and with young adult patients demonstrates strong interest in ADAIR Willing to ask MD about ADAIR Willing to pay a higher insurance co-pay for ADAIR A modest uptake in the IR amphetamine market segment alone (1 of 10 prescriptions) and a price set to the lower end of branded products projects peak annual sales >$300 MM 14

15 Recent FDA meeting charts a rapid and cost efficient development path for ADAIR A 505(b)(2) pathway <2 years development timeline 2 PK studies in small groups of healthy volunteers (BE, relative BA) Studies are expected later this year Alcobra to potentially conduct 1-2 additional studies for Abuse Deterrence labeling and support of commercialization efforts Abbreviated non-clinical work (bridging animal study to IR d-amph) and standard stability before NDA filing Current plan targets NDA filing in 2H

16 ADAIR Budget Plan Financial metric Reported financials / estimate 2016YE cash balance $50M Clinical studies $6M CMC $5M Pre-clinical and regulatory $2M Total expected cost $13M* * Does not include potential post-approval studies & ongoing fixed costs Existing financial resources adequate to support the development program of ADAIR to NDA filing 16

17 Summary Abuse of ADHD Stimulants, and IR Stimulants in particular, is a large and growing public health concern Alcobra has developed ADAIR, an immediate release dextroamphetamine formulation, with substantial abuse deterrent potential Alcobra s recent Pre-IND meeting with FDA defined a clear and rapid development path to an NDA filing in 2H 2018 Market research with physicians, managed care payers, and consumers suggests a large commercial potential for ADAIR Alcobra is well funded to quickly & efficiently develop ADAIR 17

18 Alcobra Ltd. (NASDAQ:ADHD) 18