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The Future of Diabetes Control

WEDBUSH 2013 Life Sciences Management Access Conference August 13, 2013 Access Conference W EDBUSH 2013 Life Sciences Management August 13, 2013

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Forward Looking Statements This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements about future activities related to the clinical development plans for the company's drug candidates, including the potential timing, design and outcomes of clinical trials; and the company's ability to develop and commercialize product candidates. Forward-looking statements represent our management's judgment regarding future events. All statements, other than statements of historical facts, including statements regarding our strategy, future operations, future clinical trial results, future financial position, future revenues, projected costs, prospects, plans and objectives of management are forward-looking statements. The words "anticipates," "believes," "could," "estimates," "expects," "intends," "may," "plans," "potential," "predicts," "projects," "should," "will," "would" and similar expressions are intended to identify forwardlooking statements, although not all forward-looking statements contain these identifying words. The company's forward-looking statements are subject to a number of known and unknown risks and uncertainties that could cause actual results, performance or achievements to differ materially from those described or implied in the forward-looking statements, including, but not limited to, the success of our product candidates, particularly our proprietary formulations of injectable insulin that are designed to be absorbed more rapidly than the "rapid-acting" mealtime insulin analogs presently used to treat patients with Type 1 and Type 2 diabetes and our glucagon presentation that is intended to treat patients experiencing severe hypoglycemia; our ability to successfully complete a Phase 2 clinical trial of a proprietary insulin formulation in a timely manner, and the outcome of that trial; our ability to conduct pivotal clinical trials, other tests or analyses required by the U.S. Food and Drug Administration, or FDA, to secure approval to commercialize a proprietary formulation of injectable insulin or a stable glucagon presentation; the success of our formulation development work with insulin analog-based formulations of a proprietary injectable insulin and a stable glucagon presentation; our ability to secure approval from the FDA for our product candidates under Section 505(b)(2) of the Federal Food, Drug, and Cosmetic Act; the progress, timing or success of our research, development and clinical programs, including any resulting data analyses; our ability to develop and commercialize a proprietary formulation of injectable insulin that may be associated with less injection site discomfort than Linjeta™ (formerly referred to as VIAject®), which is the subject of a complete response letter we received from the FDA; our ability to enter into collaboration arrangements for the commercialization of our product candidates and the success or failure of any such collaborations into which we enter, or our ability to commercialize our product candidates ourselves; our ability to protect our intellectual property and operate our business without infringing upon the intellectual property rights of others; the degree of clinical utility of our product candidates; the ability of our major suppliers to produce our products in our final dosage form; our commercialization, marketing and manufacturing capabilities and strategies; our ability to accurately estimate anticipated operating losses, future revenues, capital requirements and our needs for additional financing; and other factors identified in our most recent report on Form 10-Q for the quarter ended March 31, 2013. The company disclaims any obligation to update any forward-looking statements as a result of events occurring after the date of this presentation

W EDBUSH 2013 Life Sciences Management Access Conference August 13, 2013

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Company Overview – Diabetes Focus Strategy and Technology

■ Optimize PK and stability of FDA-approved therapeutics ■ Rapid development through 505(b)(2) regulatory process ■ Focus on multi-billion dollar insulin and glucagon diabetes markets ■ Ultra-Rapid-Acting Prandial Insulins RHI-based ⇒ BIOD-123 Phase 2 trial completed; top line data 3Q13  Analog-based ⇒ Positive Phase 1 top line data reported  Concentrated insulin ⇒ BIOD-531 Phase 1 trial 4Q13; top line data 1Q14 Glucagon  Rescue product: Portable, easy to use, room temperature presentation ⇒ Pivotal trial 2H14; Projected NDA filing 2015  Pump use liquid formulations under development 

Pipeline

Resources

■

■ ~40 employees based in Danbury, CT ■ $42.4 MM in cash and equivalents as of June 30, 2013 ■ 23.0 MM shares outstanding assuming conversion of preferred shares W EDBUSH 2013 Life Sciences Management Access Conference August 13, 2013

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Company Overview – Leadership Management

Board of Directors

Errol B. De Souza, Ph.D. President and CEO

Ira W. Lieberman, Ph.D. Chairman

Alan S. Krasner, M.D. Chief Medical Officer

Julia R. Brown Errol B. De Souza, Ph.D.

Gerard J. Michel Chief Financial Officer Paul Bavier General Counsel Erik Steiner VP Operations

Barry H. Ginsberg, M.D., Ph.D. Daniel L. Lorber, M.D. Brian J. G. Pereira, M.D. Davey S. Scoon


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Normal Insulin and Glucagon Physiologic Response is Required to Keep Glucose in the Normal Range 1st phase insulin response to a meal signals liver to suspend glucose production Rapid-acting analog insulin segment: NovoLog®, Humalog® and Apidra®

Plasma Insulin (µU/mL)

100 80

N O R M A L G LU C O S E R A N G E

60 40 20

-140 -80

Blood Glucose (mg/dL)

120

Basal insulin manages glucose produced by the liver between meals Long acting insulin segment: Lantus® and Levemir®

0 Meal

Meal

Time

Meal

Glucagon signals liver to produce glucose when glucose is low Glucagon currently used in emergency only — under investigation for pump use


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Current and Potential Market and Pipeline 16 14 Glucagon

Ultra-Rapid-Acting Prandial Insulin

12

Billions

Analog based Orphan indications

 RHI based

10

 Prototype formulation showed better post prandial control

Concentrated Insulin

8 Mixed Insulin

6 4

Prandial Insulin

2 0 2012

2020 Forecasted

Ultra-RapidActing Conc. Insulin*

Stable Glucagon Ultra-Rapid-Acting  Improved rescue Prandial Insulin formulations  Rapid On  Bi-hormonal pump  Rapid Off

Proprietary Formulation Technology

 RHI based  Faster onset than currently marketed U-500 and mixes  Basal coverage  Proof of concept demonstrated in swine

Glucose Responsive Glucagon Insulin  Improved rescue  Glucose modulates the formulations release of insulin from the  Orphan indications formulation  Bi-hormonal  Proof of concept pump demonstrated in swine

* will compete in both concentrated and mix segments

Based on IMS U.S. and Europe Ex Factory data and internal Biodel projections


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Ultra-Rapid-Acting Prandial Insulin for the Treatment of Diabetes

Market Opportunity

■ ~$8B in US and EU; projected to grow to ~$10B by 2020 ■ Key Products: Humalog®, NovoLog® and Apidra® ■ Existing players need to extend franchises with improved products due to near term patent expirations

■ PK profiles of RHI or rapid acting analogs do not match 1st phase insulin response since insulin hexamer is poorly absorbed Unmet Medical Need

■ Mismatch in PK profiles results in glycemic swings after meals ■ Poor glycemic control in people with diabetes leads to morbidities (such as hypoglycemia, weight gain, blindness, kidney disease, nerve damage, amputation, etc.)


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Ultra-Rapid-Acting Prandial Insulin for the Treatment of Diabetes ■ Remove zinc via EDTA to destabilize insulin hexamer; citric acid masks surface charges, preventing re-aggregation Biodel Solution

■ Mimic 1st phase insulin response ■ Applicable to both RHI and rapid acting analogs ■ Intellectual Property Protection: EU ⇒ 2025; US ⇒ 2026 ■ RHI-based BIOD-123

■

Milestones

■

  

Trial completed 3Q13: Phase 2 top line data 2014: Potential for initiation of pivotal studies Analog-based BIOD-238 and BIOD-250  1Q13: Phase 1 top line data reported  Working to replicate BIOD-250 PK and tolerability profiles utilizing lispro (Humalog® API), aspart (NovoLog ® API), and achieving commercial stability RHI-based concentrated insulin  2Q13: BIOD-531 candidate selected for Phase 1 development  Engineering batch completed  IND submitted to FDA  Two clinical supply batches scheduled for 3Q13  Initiate Phase 1 clinical trial in 4Q13  Top line data anticipated in 1Q14


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Pharmacokinetic Profiles of Analog Insulins Do Not Match Profile in Healthy Individuals Glycemic Variability

■ Oxidative stress ■ Diabetic vascular

First 15 to 30 minutes critical for glucose control

complications

■ Quality of life PK Metrics

Normal Insulin Levels Following Meal1

Regular Human Insulin SC Injection2

Humalog® SC Injection2

 Increase dose to minimize post prandial hyperglycemia OR

Early ½ Tmax (minutes)

~15

Tmax (minutes)

~30 — 40

37 ± 7.0

120 ± 22

26 ± 3.2

66 ± 10.8

 Decrease dose to minimize hypoglycemic events

Long Term Glucose Control

■ Blindness ■ Kidney disease ■ Nerve damage, amputation Hypoglycemia

■ Loss of consciousness ■ Coma and death Weight gain

■ Increased morbidity and mortality

1 Journal

of Nutrition. 1996, 126: 2807-2812 Diabetes. 1988, 37: 736-44 IEEE TRANSACTIONS ON BIOMEDICAL ENGINEERING. 2007, 54: 1740-1749 2 Diabetologia. 2008;51(9):1602-1606

■ Increased insulin resistance


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Ultra-Rapid-Acting Insulins Which Replicate “Normal” Insulin Response May Represent a Step Change in Insulin Therapy Ultra-Rapid-Acting Insulins May Address the Market’s Unmet Need 2005

Market Value

Faster Onset Less Glycemic Variability Less Weight Gain

■ Biodel initiated first-in-man Ultra-RapidActing Insulin

studies with Linjeta™, or BIOD-090; the term “ultrarapid-acting insulin” did not exist

Faster Onset

Rapid-Acting Insulin Analogs

2013

■ Novo Nordisk: “NN1218” Phase 3 initiation late 2013

Regular Human Insulin

Mimics Normal Pancreatic Response to Meal

■ Halozyme ■ Sanofi-Aventis ■ Eli Lilly


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Biodel’s Ultra Rapid Insulins’ Unique Mechanism Results in More Rapid Absorption Relative to Marketed Prandial Insulins Biodel Technology: EDTA chelates zinc to destabilize insulin hexamer; citric acid masks insulin monomers’ surface charges, impeding re-aggregation and facilitating absorption

Analog insulins have modified the primary structure using genetic engineering to decrease the tendency to form hexamers


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Biodel Ultra-Rapid-Acting Insulin Intellectual Property – Long Patent Life ■ Broad composition of matter

United States

patents ■ Multiple additional patents filed ■ Protects various insulin formulations:  RHI-based  Analog-based  Subcutaneous delivery  Sub-lingual delivery  Non-hexameric insulin  Multiple combinations of excipients ■ No external royalties

■ Patent: US7279457 ■ Expiration: 2026

European Union

■ Patent: EP1740154 ■ Expiration: 2025


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Summary of Biodel’s EDTA/Citrate Recombinant Human Insulin (RHI) and Analog Formulations ■ BIOD-090 (VIAject™, Linjeta™) and BIOD-100 (Linjeta™) - RHI based 

Multiple Phase 1, 2 and 3 studies



Superior PK/PD (versus RHI and Humalog®) trending towards better postprandial control, less weight gain and lower rates of hypoglycemia (Phase 3 studies vs. RHI)…



… but poor injection site tolerability

■ BIOD-123 - RHI based 

Phase 1 trial demonstrated superior PK (versus Humalog®) with excellent tolerability



Phase 2 trial with top line data anticipated in 3Q2013

■ BIOD-250 – Lispro (Humalog®) based 

Phase 1 trial demonstrated superior PK (versus Humalog®) with excellent tolerability W EDBUSH 2013 Life Sciences Management Access Conference August 13, 2013

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Aside from Tolerability, Trials with Previous BIOD-090 (Linjeta™) Formulation Showed Strong Trends Towards Superior Clinical Profile BIOD-90 Clinical Profile

Humalog® BIOD-100

Early ½ Tmax (min)

23.3 ± 1.0

7.9 ± 0.5

 5 single dose Phase 1 and Phase 2 trials

 4 multi dose Tmax (min)

Tolerability (VAS 0 – 100 mm)

57.1 ± 3.4

5.3 ± 1.0

29.4 ± 4.6

Phase 3 and extension studies with >900 type 1 and type 2 diabetes patients

17.3 ± 2.5

Glycemic Variability  Narrower glycemic excursions compared to Humalog® in standard meal challenge test  Reductions in markers of oxidative stress Long Term Glucose Control  HbA1c adequately controlled  Non-inferiority versus RHI (narrowly missed; study design and other issues identified) Hypoglycemia  Less hypoglycemia versus RHI Weight gain  Less weight gain versus RHI


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BIOD-123 (“Linjeta™” with MgSO4) Replicates Linjeta™ PK Profile in Type 1 Diabetes Patients … 120

100

30J BIOD-100

3-101 BIOD-123

30J Humalog®

3-101 Humalog®

Insulin Concentration (% of Cmax)

120 100 80

80

60 40 60

20 0

0

10

40

20

30 40 Time (min)

50

60

20

0

0

60

120

180

240 Time (min)

300

360

420


480

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...With Better Injection Site Tolerability Visual Analog Scale (mm)

25 Humalog®

BIOD-123

Tolerability (VAS 0 – 100mm)

1.8 ± 1.1

3.6 ± 2.1

0.17 ± 0.11

0.36 ± 0.15

5

Absolute Severity Score (0=none, 1=mild)

0

Relative Severity Score

2.92 ± 0.08

2.91 ± 0.25

20 15 10

BIOD-123

® Humalog®

Visual Analog Scale (mm)

25 20

* Injection Site Discomfort Severity

15 None

Mild

Moderate

Severe

10 (83.3%)

2 (16.7%)

0

0

7 (63.6%)

4 (36.4%)

0

0

10 Humalog® (n=12)

5 0 BIOD-100 Linjeta™

BIOD-123 (n=11)

Humalog®

 Primary endpoint of injection site tolerability was measured on a 100 mm Visual Analog Scale (VAS); 0 = no discomfort;100 = worst possible discomfort; 3 = usual injection  Data represents means ± standard error, *p < 0.05 vs. Humalog®


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BIOD-123 Phase 2 Trial Design Randomized 2 arm study, ~130 Type 1 diabetes patients, ~30 U.S. investigative centers Study duration 18 weeks

12 weeks stable dosing

6 weeks active titration

BIOD-123

Primary Endpoint

■

Secondary Endpoints

vs.

■

Humalog®

■ ■

Basal insulin: Lantus®

Milestones

■

1Q13: Enrollment completed

■

3Q13: Top line data anticipated

HbA1c control

■

Postprandial glucose control Glycemic variability Hypoglycemic event rates Weight changes


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Humalog®, NovoLog® and Apidra® Analogs Formulated with BIOD-100 Excipients Have an Ultra-Rapid-Acting Insulin Pharmacokinetic Profile in Diabetic Swine 160

100

NovoLog® NovoLog® plus EDTA and citric acid

140 Insulin (uU/mL)

120 Insulin (uU/mL)

160

Humalog® Humalog® plus EDTA and citric acid

140

80 60 40 20

120 100 80 60 40 20 0

0 0

40

80

120 160 Time (minutes)

200

0

240

40

80

120

160

200

240

Time (minutes)

160

Apidra® Apidra® plus EDTA and citric acid

Insulin (uU/mL)

140 120 100 80 60 40 20 0 0

40

80

120

160

200

240

Time (minutes) Source: 2010 “EASD 46th Annual Meeting”, Stockholm, Sweden pp. S384

Preclinical study : Pharmacokinetic profiles in diabetic swine


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In Man, Lispro Based* BIOD-238 and BIOD-250 Demonstrated a More Rapid In and Out vs. Humalog BIOD-238 BIOD-250

80

Humalog® Insulin Concentration (μU/mL)

80

60

60

40

40

20

0 0

20

10

20

30

40

50

60

0 0

60

120

180

240 Time (min)

300

360

420

480

* Biodel’s proprietary excipients added to commercial Humalog to create BIOD-238 and BIOD-250


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Decline from peak concentration

Absorption

Improved PK Performance of BIOD-238 and BIOD-250 vs. Humalog  Supported By Multiple Measurements Variable

BIOD-238 N=10

BIOD-250 N=11

Humalog® N=10

P-value BIOD-238 vs. Humalog

P-value BIOD-250 vs. Humalog

Early ½ Tmax (minutes)

13.7 ± 1.8 (13.6)

14.6 ± 1.9 (12.9)

24.9 ± 2.9 (22.6)