Avenue Therapeutics, Inc. May 2017 Forward Looking Statements - - PowerPoint PPT Presentation

Avenue Therapeutics, Inc.

May 2017

Forward Looking Statements

Statements in this presentation that are not descriptions of historical facts are forward‐looking statements within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995. We have attempted to identify forward‐looking statements by terminology including “anticipates,” “believes,” “can,” “continue,” “could,” “estimates,” “expects,” “intends,” “may,” “plans,” “potential,” “predicts,” “should,” or “will” or the negative of these terms or other comparable terminology. Forward‐looking statements are based on management’s current expectations and are subject to risks and uncertainties that could negatively affect our business,

• perating results, financial condition and stock price. Factors that could cause actual results to

differ materially from those currently anticipated are risks relating to: our growth strategy; results

• f research and development activities; uncertainties relating to preclinical and clinical testing; our

dependence on third party suppliers; our ability to obtain, perform under and maintain financing and strategic agreements and relationships; our ability to attract, integrate, and retain key personnel; the early stage of products under development; our need for substantial funds; government regulation; patent and intellectual property matters; competition; as well as other risks described in our SEC filings. We expressly disclaim any obligation or undertaking to update or revise any statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances after the date of this presentation.

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Avenue and its Value Proposition

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What We Do and Why

 We focus on pharmaceuticals for the acute/intensive care hospital setting  U.S. hospital market is concentrated and requires a small sales force  Our lead asset intravenous (IV) tramadol is ready to enter Phase III development  Trend on “multimodal” treatment for pain favors new products that help patients avoid strong narcotics  Tramadol is an opioid without the typical side effects of narcotics  We are a subsidiary of Fortress Biotech that is run by an experienced management team and advisors  Backing by Fortress’ BD effort = New pipeline product candidates

U.S. Post‐Op Pain Market

 IV analgesics sells ~$1 billon (~300 million units)/yr

• IV acetaminophen sells ~$250MM ‐ ~25% of total dollar

market with approximately 3 to 4% of the volume

• Room for Improvement

4 Available Classes Pain Levels Common Limitations & Contraindications IV narcotics Moderate to severe Strong sedation Respiratory depression Constipation Risk of dependence IV NSAIDS Mild to moderately severe Post‐op bleeding risk GI side effects Renal impairment IV acetaminophen Mild to moderate Hepatic impairment

What is Tramadol

Not a typical opioid

• Two distinct mechanisms of action: (1)weak opioid agonist, and (2)inhibitor
• f monoamine neurotransmitter reuptake

Labeled for “moderate to moderately severe pain”

• Analgesic effect similar to other opioid therapies
• Differentiated safety profile compared to other opioids

Oral tramadol widely prescribed in the U.S.

• Schedule IV and one of the most prescribed pain medications
• Prescriptions increased from 23.3 million in 2008 to 43.8 million in 2013
• ~$1.2 billion annual sales despite being generic

IV tramadol widely used outside the U.S.

• Accounts for ~10% of IV analgesic use in the post‐op setting in E.U.

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IV Tramadol

Summary

Indication Moderate to moderately severe pain Regulatory pathway 505b(2) Stage of development Phase III to begin in 2017 Clinical efficacy*

• Parenteral tramadol has been tested in several randomized, double‐

blind, parallel‐group, comparative studies.

• It effectively relieved moderate to severe post‐op pain associated with

several types of surgeries (abdominal, orthopedic, and cardiac surgery) in these studies. Tolerability*

• In general, tramadol was well tolerated in clinical studies. The most

common adverse events were nausea, dizziness, drowsiness, tiredness, sweating, vomiting, and dry mouth.

• Unlike other opioids, it did not cause clinically relevant respiratory

depression.

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*Tramadol: A Review of its Use in Perioperative Pain. Scott LJ, Perry CM. Drugs. 2000 Jul;60(1):139‐76.

New Restrictions on Opioids

• New guidelines from CDC to physicians attempts

to limit opioid prescribing

• Many states have passed laws restricting opioid

prescriptions

• The U.S. DEA has reduced the amount of almost

every Schedule II opioid medication that may be produced in the U.S. in 2017 by 25 percent or more

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IV Tramadol Opportunity

 IV tramadol may be used in all types of inpatient and

• utpatient surgeries and as part of a multimodal

analgesic plan for major surgery  It may be an improved option for patients

• With poor cardiopulmonary function
• Elderly patients at risk for respiratory depression
• Obese patients with sleep apnea
• Contraindicated for NSAIDS
• Patients who cannot tolerate strong narcotics such as

morphine due to side effects such as sedation, constipation, and history of drug dependence, etc.

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Results from a Survey of 30 U.S. Anesthesiologists*

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*Survey conducted through LEERINK and available upon request

Patients taking Switch to IV tramadol Add IV tramadol IV morphine 40% 41% IV NSAIDS 26% 37% IV acetaminophen 24% 35%

Advantages of IV Tramadol

Documented efficacy and safety Physician familiarity with tramadol Less addictive than widely prescribed narcotics in the hospital Available “step‐down” therapy

• Our IV tramadol dosing regimen provides similar

Cmax and AUC to that of oral Ultram 100 mg Q6h at steady state.

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Phase 3 Program*

11 Study Goals Design Orthopedic model (Bunionectomy)

• 1. To demonstrate safety and

efficacy of IV tramadol (50 mg dose) versus placebo

• 2. To understand dose

response 405 patients, 3‐arm (IV tramadol 50 mg, 25 mg, or placebo), 1:1:1 randomized, and double‐blind study Soft tissue model (Abdominoplasty)

• 1. To demonstrate safety and

efficacy of IV tramadol (50 mg dose) versus placebo

• 2. To understand safety and

tolerability versus standard

• f care

360 patients, 3‐arm (IV tramadol 50 mg, placebo, or morphine), 3:3:2 randomized, and double‐ blind study Safety study

• 1. To achieve total exposure
• f 500 patients on IV

tramadol (50 mg dose)

• 2. To understand safety in

“real‐world” situations 250 patients, open‐label, single arm, all surgical types *Key elements clarified and confirmed at EOP2 Meeting

Executive Team and Key Advisor

Lindsay Rosenwald, MD, Executive Chairman

• Chairman and CEO of Fortress Biotech, Inc.
• Prolific and successful investor in the life sciences industry for over 20 years
• Co‐founder and Co‐Portfolio Manager of Opus Point Partners

Lucy Lu, MD, Interim President & Chief Executive Officer

• EVP and CFO of Fortress Biotech, Inc.
• Former Senior Analyst at Citi Investment Research
• 15 years of experience in life sciences

David Horin, Interim Chief Financial Officer

• Managing Partner, Chord Advisors, LLC.
• Former Chief Financial Officer of Rodman & Renshaw Capital Group, Inc.
• Former Managing Director of Accounting Policy and Financial Reporting at Jefferies Group,

Inc.

Scott Reines, MD‐PhD, Interim Chief Medical Officer

• Former Vice President/Senior Vice President at Merck Research Laboratories and Johnson &

Johnson Pharmaceutical R&D

• Oversaw development and approval of drugs including INVEGA, NUCYNTA, RISPERDAL

CONSTA, TOPAMAX, EMEND, MAXALT, SINEMET, etc.

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Scientific Advisory Board

Robert Dworkin, PhD

• Director of the Analgesic, Anesthetic, and Addiction Clinical

Trial Translations, Innovations, Opportunities, and Networks (ACTTION) Harold Minkowitz, MD

• Assistant Professor of Anesthesiology at the University of

Texas Health Science Center at Houston Neil Singla, MD

• Founder and Chief Scientific Officer, Lotus Clinical Research

Mark Wallace, MD

• Director, Center for Pain Medicine, UC San Diego

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Upcoming Milestones

 Commence Phase 3 program in 2017  Phase 3 Data 2018  NDA filing 2019  ~$30 million to complete Phase 3 Program

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Avenue Therapeutics, Inc.

Contact: Lucy Lu, M.D. 2 Gansevoort St., 9th Floor New York, NY 10014 Tel: 781‐652‐4511 Email: llu@avenuetx.com

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