rockwell medical technologies update

27
Innovative Bio-pharmaceutical Company July 2009

Upload: pro-active-capital-resources-group

Post on 19-Jun-2015

448 views

Category:

Investor Relations


3 download

DESCRIPTION

Rockwell Medical is a BioPharmaceutical Company with increasing year to year revenues. Rockwell Medical has an established operating business and develop unique, proprietary renal drugs. They have 27% of the market share with regard to renal business.

TRANSCRIPT

Page 1: RockWell Medical Technologies Update

Innovative Bio-pharmaceuticalCompany

July 2009

Page 2: RockWell Medical Technologies Update

2

Safe Harbor StatementThis presentation contains forward-looking statements. All statements, other than statements of historical facts, including, among others, statements regarding the Company’s future financial position, business strategy, projected levels of growth, projected costs and projected financing needs, are forward-looking statements. Those statements include statements regarding the intent, belief or current expectations of Rockwell Medical Technologies, Inc. and members of the Company’s management team, as well as the assumptions on which such statements are based, and generally are identified by the use of words such as “may,” “will,“ “seeks,” “anticipates,” “believes,” “estimates,” “expects,” “plans,” “intends,” “should” or similar expressions. Forward looking statements are not guarantees of future performance and involve risks and uncertainties that actual results may differ materially from those contemplated by such forward-looking statements.

The company believes these forward-looking statements are reasonable; however, undue reliance should not be placed on any forward-looking statements, which are based on current expectations. All written and oral forward-looking statements attributable to the Company or persons acting on its behalf are qualified in their entirety by these cautionary statements. Further, forward-looking statements speak only as of the date they are made, and the Company undertakes no obligation to update or revise forward-looking statements to reflect changed assumptions, the occurrence of unanticipated events or changes to future operating results over time unless required by law.

Page 3: RockWell Medical Technologies Update

Agenda

3

Corporate Overview

Lead Drug Candidate

Financial Overview

Investment Opportunity &

Upcoming Milestones

Page 4: RockWell Medical Technologies Update

Corporate Overview

4

Page 5: RockWell Medical Technologies Update

5

Corporate Summary

Bio-pharmaceutical drug company with established operating business

Development of unique, proprietary renal drugs Lead drug iron delivery via dialysate – new standard of care Expect superior patient outcomes vs. current therapies Women’s health, oncology and parenteral nutrition

Strong, growing core operating renal business Broad distribution channel and manufacturing base 3 manufacturing plants; 27% U.S. market share Dialysate; removes toxins and replaces nutrients in blood Products needed to maintain human life Provided 16.6 million+ treatments in 2008

Page 6: RockWell Medical Technologies Update

6

Kidney Disease Market Chronic kidney disease (CKD) 19.2 million

Progressive loss of renal function; 5-Stages End Stage Renal Disease (ESRD) – irreversible loss of

kidney function need dialysis to live Stage 5 = 395,000 US patients; 2 million worldwide Stages 3 and 4 = 8.1 million US patients

Steady, growing market unaffected by economies

Causes Diabetes and obesity, CVD, hypertension, aging

Current treatment options Dialysis: life-saving blood filtering treatment Kidney transplant

Page 7: RockWell Medical Technologies Update

Lead Drug CandidateSFP

Soluble Ferric Pyrophosphate

7

Page 8: RockWell Medical Technologies Update

SFP – Iron via Dialysate

8

Replaces kidney function Removes waste and excess fluids Replenishes nutrients 3-4 hour treatment

Dialysis

Dialyzer

Blood flows through Dialyzer membrane

Dialysate flows outside of membrane in opposite direction

Nutrients (Ca, K, Mg, Na) delivered into blood while toxins and waste are removed

SFP (iron) now delivered into blood via dialysate just like nutrients

Page 9: RockWell Medical Technologies Update

9

SFP – Iron via Dialysate

Bio-available iron delivered directly to blood stream

Replaces iron in blood lost during each dialysis treatment

Rapid uptake of iron by transferrin Transferrin takes iron to bone

marrow Maintains iron balance within

target hemoglobin range

Inside Dialyzer Filter

Page 10: RockWell Medical Technologies Update

10

SFP Advantages Physiological iron maintenance therapy

Slow infusion (10-15 µg/dL iron dose) via dialysate during scheduled dialysis session; 3X per week; works like dietary iron

Small dose replaces 5-7mg avg blood loss per session Maintains iron balance efficiently matches EPO and hemoglobin

Superior safety benefits for patient + lowers provider cost Safely travels direct to bloodstream bypasses liver and avoids toxicity ~2000 Clinical Doses to Date No Adverse Reactions Significantly lowers IV iron administration costs eliminates needles,

syringes and RN time Bundled reimbursement provides superior patient-outcome at lowest

cost; significant EPO savings expected

Market potential U.S. IV iron CKD $500 million ($430M+ ESRD) Global IV iron CKD $850 million U.S. dialysis concentrates $180 million

Page 11: RockWell Medical Technologies Update

Maximizes Erythropoiesis

11

By maintaining constant iron balance SFP maximizes creation of red blood cells, generation of hemoglobin and optimization of EPO treatment response

Page 12: RockWell Medical Technologies Update

SFP vs IV IronHemodialysis Dependent Chronic kidney Disease (HDD-CKD)

SFP Venofer Ferrlecit Feraheme

Dose Size (iron) 10-15 μg/dL 100mg 125mg 500mg

Complexed with carbohydrate (sugar)backbone

No Yes Yes Yes

Molecular Weight(Da)*

< 1,000 34,000 – 60,000

289,000 –440,000

730,000

Est’d Iron Use (grams per year)

~1.5-50% less-

3 3 3

Iron Delivery Method

Slow Infusion during Dialysis

Injection/Infusion

Injection/Infusion

Injection

Approx. # of Admin./Yr

156 30 24 6

Rate of Iron Injection

2-3 mg/hr 20 mg/min 12.5mg/min > 510 mg/min

Nurse Time Per Dose

Not Applicable 2-5 min 5-10 min < 1 min

12* high molecular weight sugar molecules have been linked to anaphylactic reactions; not present in SFP

Page 13: RockWell Medical Technologies Update

SFP Development Program

Past

•Pre-clinical safety, pharmacology & toxicity studies completed•Exception

al safety profile

•Phase IIa clinical trial completed •Proof of

concept study:12 mcg iron/dL dose

Present

•Phase IIb clinical trial – dose ranging•6 months/

25-30 sites/ 100-120 patients

•Data expected Q4 2009 / Q1 2010

•NIH funded clinical safety study•9 months/

multi sites/ 30 patients

•Data expected 2010

Future

•Q2 2010: Phase III initiation

•2011: Anticipated NDA/ANDA filing

•2011/2012: Estimated product launch

13

Page 14: RockWell Medical Technologies Update

SFP Phase IIb Study

Primary Endpoint: Drop in hemoglobin of 1 gm/dL or more and safety.

Secondary Endpoints: Time taken to drop hemoglobin by 1 g/dL or more, increase in hemoglobin to more than 12.5 g/dL, reticulocyte hemoglobin, infection, iron transferred into the patient.

Enrollment completed April 2009 Two positive Data Safety Monitoring Board (DSMB)

reviews to date; validates SFP superior safety profile Expect data Q4 2009 / Q1 2010

14

Page 15: RockWell Medical Technologies Update

SFP Commercial Launch: 2012

2009

• 27% US dialysate share • $51.6M sales • 16.6M+ treatments

2012 Goals*

• 35% US dialysate share • $88M sales• 23M treatments• $200M captive US iron

market; $600M total• International partnerships

and royalty stream - additive

15

Strategy: Leverage captive client relationships and distribution channels for fast SFP penetration

* Forecast

Page 16: RockWell Medical Technologies Update

Intellectual Property

Rockwell owns exclusive worldwide license Patents issued in U.S, Europe and Japan three

largest ESRD markets in the world Iron delivery via dialysate in hemodialysis (HD) and

peritoneal dialysis (PD) Composition of matter and method of delivery Patent expires 2021 (including 5 year Hatch-Waxman)

Patent filed on SFP-GMP grade formulation To cover dialysis, oral OTC/Rx, TPN, Oncology, and other

extensions

16

Page 17: RockWell Medical Technologies Update

Financial Overview

17

Page 18: RockWell Medical Technologies Update

Key Financial Facts

Shares Outstanding** 14.1M

Fully Diluted Shares Outstanding** 14.9M

Share Price (NASDAQ: RMTI)* $8.61

Market Cap* $122M

Revenues (ttm)** $52.1M

SFP Expected Annual Burn Rate*** $3.5-4M

Cash and Cash Equivalents** $3.35M

18

*As of July 24, 2009**As of 1Q09**Excluding cash flow generation from core business

Page 19: RockWell Medical Technologies Update

Annual Sales Growth

0102030405060

2004 2005 2006 2007 2008 2009 - Q1

19

2008Forecast*

($, in Millions)

5-Year CAGR 23.6%

Page 20: RockWell Medical Technologies Update

Investment Opportunity & Upcoming Milestones

20

Page 21: RockWell Medical Technologies Update

21

Investment Opportunity Bio-Pharma transition

Innovative, proprietary disruptive technology with lead-drug candidate SFP Safer, more effective and substantial cost savings Significant market potential $500M U.S. / $850M Global

Ready-made commercial distribution channel for fast market penetration of high-margin renal drugs Solid customer base; $51.6M revenues in 2008 Captive 35% market share to leverage ($88M) in 2011*

Regulatory development progressing Ongoing Phase IIb FDA study / NIH funded study Phase III study anticipated to begin enrollment Q2 2010 FDA approval and commercial launch anticipated 2011/2012

*Forecast

Page 22: RockWell Medical Technologies Update

22

Investment OpportunityBio-Pharma transition

Strong scientific clinical team; focus SFP success; leverage renal drug pipeline Expanded SAB with 3 new anemia/iron deficiency experts Hired VP of Drug Development and Medical Affairs Hired Chief Scientific Officer SFP formulation opportunity – leveraging into new markets Secured other proprietary renal drugs Favorable environment for bio-pharma therapeutics – big pharma

pipelines scarce

Opportunistic market conditions Growing, worldwide renal patient population unaffected by economic

cycles; predictable recurring revenues Critical need for iron therapeutics and renal products CMS bundling 2011 – should prove favorable to SFP and extension

opportunities

Page 23: RockWell Medical Technologies Update

Upcoming Milestones

23

2008: Initial DSMB Phase IIb trial review 2008: Expand Scientific Advisory Board (+3 members)2008: Hire VP Drug Development & Medical AffairsQ2 2009: Second DSMB Phase IIb trial reviewQ2 2009: Complete enrollment for Phase IIbQ2 2009: Hire Chief Scientific Officer2009: Create Steering Committee Q4 2009: Complete Phase IIb trial Q4 2009 / Q1 2010: Release Phase IIb data Q2 2010: Initiate Phase III trial 2010: Release NIH data2011: File NDA/ANDA 2011 / 2012: Launch product

Page 24: RockWell Medical Technologies Update

Innovative Bio-pharmaceuticalCompany

30142 Wixom RoadWixom, MI 48393 USA

(248) 960-9009

[email protected]

Page 25: RockWell Medical Technologies Update

Appendix

Page 26: RockWell Medical Technologies Update

FDA Clinical Studies

FDA Phase IIa FDA Phase IIb

StudyType

Dose Escalating Dose Ranging

Study Goals/Objectives

Determine optimal SFP iron dose

Determine maximum tolerated dose & minimum level benefits

Study Outcomes

Optimal Dose: 12mg/dl

700 human doses

Proven safe & effective. No adverse reactions

Dose range: 0, 5, 10, 12, 15, 20 mg/dl

10,000 human doses

Data expected Q4 2009

Study Duration

6 months 6 months

Number of Patients

24 100-120

Number of Sites

1 25-30

26

Page 27: RockWell Medical Technologies Update

EPO & Hemoglobin Generation

It takes 25 days to create a red blood cell

- Day 16: EPO receptors surround, protect, and incorporate into cell in order for it to mature

- Day 19-20: more transferrin receptors exist around cell looking for iron to incorporate into cell

It is crucial that iron be present between day 19-20 (not effective to add it later)

important to maintain iron balance all the time

- Day 21: If iron exists to incorporate into cell, it becomes a reticulocyte iron used to generate hemoglobin reticulocyte mature red blood cell (RBC) within 4 days

27