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RepliGen




            RepliGen
            NASDAQ: RGEN
           Corporate Overview
             October 2010
RepliGen                         Company Profile
Repligen acquires product candidates, advances them to a key inflection
       point and captures value by commercializing or partnering

• Late-stage pipeline with significant market opportunity
   – RG1068 to diagnose pancreatitis; >$100M U.S./E.U. market (Phase 3)
   – RG2417 for bipolar depression; ~$2B market opportunity (Phase 2b)
   – Two potentially transformative drugs for orphan CNS diseases; >$500M market
     opportunity each (Pre-clinical)
• Financial sustainability
   – Strong cash position (~$59M); low cash burn and no debt
   – Profits from Bioprocessing business and royalties fund therapeutics pipeline
• Proven strategy
   – Acquired patent from MIT; developed Erbitux® cell line; licensed to ImClone for $65M
   – Acquired patent from Univ. of Michigan; developed Orencia® application; licensed to
     Bristol-Myers for ~$65M(e) in royalties through 2013

Our goal for 2011 is to file an NDA in the U.S. and an MAA in the E.U. for RG1068,
         secure partner for RG2417; assuming positive data in Q1 2011
RepliGen
                           Advancing Pipeline
                      Fueled by Commercial Assets
                          Pre-Clinical      Phase 1      Phase 2       Phase 3
                                                                                   Market
                            Animal       First Human     Proof of      Proof of
   Therapeutic Product      Models            Trial      Concept       Efficacy
        Pipeline

      RG1068                                                                      Complete
  Pancreatic Imaging                                                              Phase 3

      RG2417                                                         Complete
   Bipolar Disorder                                                  Phase 2b

       RG2833                               Initiate
   Friedreich’s Ataxia                      Phase 1

       RG3039                            Qualify clinical
Spinal Muscular Atrophy                  candidate
                                                                                  FY2011(e)
    Commercial Assets
                                                                                  ~$13M in
   Bioprocessing                                                                  revenue
      Business
 Biologics Purification                                                           ~$10M in
                                                                                  revenue
 Orencia® Royalties
 Rheumatoid Arthritis
                                                       = FY2011 Goal (03/31/11)
Pancreatic Imaging
     Pre-RG1068                                                    Post-RG1068
                                            Biology                       Liver
             Liver
                                   RG1068 stimulates the
                                                              Bladder
Bladder                            release of fluid by the
                                   pancreas; filling the                            Pancreas
                       Pancreas
                       Pancreas                                                     Pancreas
                                   pancreatic ducts, which
                                   makes them larger

           Intestine                                                    Intestine


          Pre-RG1068                                              Post-RG1068
                                      Clinical Application
                                   4-6 minutes post-RG1068
                                   enhanced MRI leads to an
                                   improved image of ducts;
                                   diagnosis of pancreas
                                   divisum which means
                                   patient is a candidate
                                   for surgery
RG1068 Phase 3 Highlights
• ERCP is an invasive, risky endoscopic procedure for diagnosis
  of pancreatic duct abnormalities; associated with high morbidity
  and measurable mortality
• Patients in the Phase 3 study were evaluated for detection of 10
  pre-specified structural pancreatic abnormalities. MRI images
  pre- and post-RG1068 were analyzed by three independent
  radiologists; ERCP used as “truth standard”
   – Radiologists evaluation of data was flawed due to significant deviations
     from the protocol
   – FDA and EMA agreed to a “re-read” of images using three          new
     radiologists
   – Study showed that RG1068 aids in avoiding unnecessary ERCP; for
     every patient on day of hospitalization from ERCP was saved
• Expect to complete Phase 3 re-read in Q1 2011
• If successful, file New Drug Application (NDA) in Q2/Q3 2011
Phase 3 Results
                                         78%
                                                                              74%

                                                      66%
                                                                  60%

                                 52%
                                                            46%


                                                34%                     35%




                                  p<0.001        p<0.001    p<0.001     p<0.001

       Enhanced image quality and duct visualization results in improved
        sensitivity to detect abnormalities and confidence in diagnosis
*Sensitivity data is for Reader 1 only
Market Opportunity
               Clinical Application                           U.S.           E.U.
 Abdominal MRI
                                                             200,000       150,000
 Enhance existing MRI images
 Endoscopy
 Expand market opportunity; triage to appropriate            100,000         TBD
 therapy and convert diagnostic endoscopy to MRI


• Market opportunity in U.S./E.U. for MRI enhancement is greater than
  $100M, assuming a price of $350/vial
• Evaluating other clinical applications and market opportunity outside
  of U.S./E.U.
    – Surgery: improve confidence in diagnosis and pre-surgical planning
    – New potential indication: pancreatic cancer detection/staging
    – Japan is a third promising market; high utilization of MRI and potential for
      favorable pricing
RG2417 for Bipolar Depression

• Bipolar disorder symptoms include episodes of severe
  depression and mania; depressive episodes account for
  majority of impairment
• Current therapies for bipolar depression are ineffective in
  most patients and have significant side effects
• Bipolar disorder is linked to abnormal activity in the
  mitochondria, the power plant of a cell
• RG2417, an oral formulation of uridine, may affect
  mitochondrial activity and improve symptoms of depression
  in bipolar disorder with minimal to no side effects
RG2417 Phase 2a Highlights
• Conducted a Phase 2a study resulting in statistically
  significant improvement in depression on MADRS scale
   – RG2417 well tolerated by patients; the adverse event profile for
     RG2417 was equivalent to placebo
• Completed enrollment of Phase 2b study to confirm
  Phase 2a results
• Expecting results from the Phase 2b study in the first
  quarter of 2011
• Objective: license to a pharmaceutical partner in 2011
Phase 2a Results
                   All Patients            Significant History of Symptoms
                                                         15 depressive episodes (n=33)




                                           MADRS Score
MADRS Score




                                                          P= .03 .09 .01 .08 .22


              P=0.01 (repeated measures)                  P<0.001 (repeated measures)
Market Opportunity
• Patients primarily seek treatment for the depressive symptoms
   – Physicians report that 77% of the time patients seek treatement for
     depressive symptoms as opposed to mania symptoms
• Psychiatrists have a high level of dissatisfaction with current
  bipolar depression therapies because they are ineffective in
  most patients and may have significant side effects
• Total costs of bipolar disorder ~$45 billion per year (NIH est.)
   – Accounts for more than 16 million visits to a physician each year and
     approximately 150,000 hospitalizations
• Worldwide prevalence >5 million
• Approximately $2B market opportunity (10% market
  penetration)
RG2833 for Friedreich’s Ataxia
                                                    • Friedreich’s ataxia is an orphan
                                                      disease characterized by progressive
                                                      loss of muscle function which leads to
Frataxin Protein Level




                                                      incapacitation and loss of life
                                                    • Caused by a defective gene, which
                                                      results in low levels of Frataxin protein
                                                    • RG2833, an HDAC* inhibitor, is the
                                                      first compound to target activation of
                                                      the defective gene to increase
                                Patients Healthy      frataxin production
                                         Carriers
                                                    • >$500M market opportunity
                          A 2.5 fold increase in
                         Frataxin levels may be
                           sufficient to arrest          *Histone deacetylase inhibitors (HDAC inhibitors) are a
                                                         class of compounds that interfere with the function of
                            disease progress             histone deacetylase, which is related to gene expression
RG2833 Next Steps

• Initiate Phase 1 study following FDA approval
• Evaluate HDAC inhibitors in animal models of
 Huntington’s disease and cognition
• Prosecute worldwide patent protection; composition
 of matter patents; priority date May 2008
RG3039 for Spinal Muscular
                            Atrophy
• Spinal Muscular Atrophy (SMA) is an orphan disease characterized by
  progressive loss of muscle function and early death in most patients
• Caused by a defective gene SMN2, which results in low levels SMN protein
• RG3039, is the first compound to target activation of the defective gene;
  increasing the production of SMN and potentially arresting the progress of
  the disease
• >$500M market opportunity

  Disease Classification         SMN2 Gene Copy             Life Expectancy

          SMA I             73% of patients have 2 copies      ~2 Years

         SMA II             82% of patients have 3 copies      ~20 Years

                            50% of patients have 3 copies
         SMA III                                                50 Years
                               and 45% have 4 copies
  A 1.5 to 2 fold increase in SMN levels has the potential to profoundly
                      affect the course of the disease
RG3039 Next Steps
• Qualify RG3039 as a clinical candidate
   – Complete GLP toxicology study to determine
     appropriateness for human clinical trials

• Initiate Phase 1 study
• Seek funding from non-profits or NIH
• Prosecute composition of matter patent
Bioprocessing Overview
• For over 20 years, Repligen has been a leading supplier
  of Protein A for production of monoclonal antibodies;
  important therapies for cancer, autoimmune diseases
  and osteoporosis
  – OEM supply to GE Healthcare and Millipore
  – FY2011 estimated revenue = ~$13M; 60% gross margin
• Repligen’s objective is to expand bioprocessing market
  opportunity by broadening its product offering, and
  through direct sales to end users
  – Protein A Resins
  – Pre-packed Columns
  – Analytical Tools
Expanding Market Opportunity
                              Legacy Business

                             OEM suppliers       End-users
  Protein A                   • GE Healthcare     • Monoclonals
                              • Millipore
Market Size $25M
                             Emerging Business

   Protein A                     Protein A
                   Protein A Resins
                                  Resins
                                                  End-users
  Plug & Play                   Pre-Packed        •Monoclonals
Chromatography                   Columns          •Biologics
                                                  •Vaccines
                               Membranes
                              Analytical Tools


                                                 Market Size $500M
Bioprocessing Business




         Large Scale Fermentation       Large Scale Purification




 rProtein A
                         “Plug and Play” Opus™         Analytical Tools
Affinity Resin
                                Columns                  ELISA Kits
Projected Orencia® Royalties
Licensed patent to Bristol-Myers for US sales of Orencia®, a novel
                 therapy for rheumatoid arthritis
            $13.3
                                                    $12.8
                     Upfront payment
             $6.3   for back-royalties      $11.5
                                    $10.3                   $10.5

                       $9.0




             $7.0




                    FY2011-FY2014 ~ $45M (e)
RepliGen
                            Corporate Assets
                          Pre-Clinical      Phase 1      Phase 2       Phase 3
                                                                                   Market
                            Animal       First Human     Proof of      Proof of
   Therapeutic Product      Models            Trial      Concept       Efficacy
        Pipeline

      RG1068                                                                      Complete
  Pancreatic Imaging                                                              Phase 3

      RG2417                                                         Complete
   Bipolar Disorder                                                  Phase 2b

       RG2833                               Initiate
   Friedreich’s Ataxia                      Phase 1

       RG3039                            Qualify clinical
Spinal Muscular Atrophy                  candidate
                                                                                  FY2011(e)
    Commercial Assets
                                                                                  ~$13M in
   Bioprocessing                                                                  revenue
      Business
 Biologics Purification                                                           ~$10M in
                                                                                  revenue
 Orencia® Royalties
 Rheumatoid Arthritis
                                                       = FY2011 Goal (03/31/11)
RepliGen                          Financials
                                 FY2010(a)          FY2011(e)
          Ending:                 3/31/2010           3/31/2011
          Revenue                     $21M             $24-26M
          Net Loss                  ($4.1M)             (<$3M)
          EOY Cash*                 $59.1M             $57-58M

                 Shares Outstanding (03/31/10)          30.8M
                 Shares & Vested Options (03/31/10)      32.2M
                 Market Cap (10/04/10)                  $104M
                 90 Day Average Volume (10/04/10)       45,000

  * Net of acquisitions
RepliGen      Upcoming News Stream

 • September 2010: End of enrollment for RG2417
   Phase 2b study
 • November 2010: Presenting at Society for
   Neurosciences on new indications for HDAC inhibitors
 • November 2010: Q2FY11 results and quarterly update
 • Q1 of 2011: Release RG1068 Phase 3 results
 • Q1 of 2011: Release RG2417 Phase 2b study results
 • H1 of 2011: Initiate phase 1 study of RG2833
 • H1 of 2011: File IND for RG3039
RepliGen      Why Invest in Repligen?
• Expecting data on a phase III development compound as
  MRI contrast agent for the diagnosis of pancreatic duct
  abnormalities in Q1, 2011
• Expecting data on a Phase II development compound for
  bipolar disorder Q1, 2011
• ~$3 stock with $1.50/share in cash. Cash burn is less
  than $0.10/share for 2011. No debt.
• Knowledgeable of monoclonal antibody production
  process; holds 50% market share of protein A market
RepliGen           Business Drivers


 Advance Pipeline                   Accelerate
                                  Bioprocessing


                    Shareholder
                       Value

                                      Protect
   Low Cash Burn
                                    Innovation
RepliGen




           Potential Back Up Slides
RepliGen                   Strategic Track Record
         Acquired                      Developed                  Capture Value Value
                                                                       Captured

                                              Past
Compounds for cancer            Erbitux® cell line                Licensed to ImClone;$65M
treatment from MIT              for National Cancer Institute               settlement
Compounds for RA               Orencia® cell line                 Licensed to Bristol-Myers;
From Univ. of Michigan         Conducted early clinical trials    ~$65M in royalties through 2013

                                    Present and Future
Secretin for CNS diseases      Completed Phase 3 study           Commercialize (US) / Partner (EU);
from Univ. of Maryland         for pancreatic imaging            following FDA approval

Uridine as treatment for       Completed Phase 2a study          Following positive results, qualify
Bipolar Disorder               Conducting a Phase 2b study       potential partners

Compounds for Friedreich’s     Filed New Drug Application        Continue developing to capture
Ataxia from Scripps Research   for RG2833                        value as appropriate

Compounds for SMA from         Identified clinical candidate     Continue developing to capture
Families for SMA               RG3039                            value as appropriate
Phase 3 Trial and Results
                      • 258 patients were evaluated with MRI and ERCP for detection of 10
                        pre-specified structural abnormalities
Trial Protocol            – ERCP used as “truth standard” for abnormalities
                          – MRIs analyzed by three independent radiologists

                      • Statistically significant improvements in the detection of structural
Primary Endpoints       abnormalities using RG1068 enhanced MRI vs. MRI alone, with no
                        false positives

                      • Improvements in image quality, visualization of pancreatic ducts and
Secondary Endpoints     confidence in diagnosis of abnormalities

                      • One out of the three radiologists achieved primary endpoints
                          – 256 additional days of hospitalization with ERCP
Results
                      • RG1068 well tolerated and safe vs. ERCP
                      • All three radiologists achieved secondary endpoints
                      • Radiologists evaluation of data was flawed due to significant
                        deviations from the protocol
Status                • Food and Drug Administration (FDA) and European Medicines Agency
                        (EMA) agreed to a “re-read” of images using three new radiologists
Phase 2a Clinical Trial

                    A study with 83 patients, placebo-controlled, daily dosing
Protocol            and weekly assessment for 6 weeks

                    • Statistically significant decreases in the symptom of
                      depression as measured by the Montgomery Asberg
                      Depression Rating Scale (MADRS) with minimal
Primary Endpoint      side effects
                    • MADRS has been previously accepted by the FDA for
                      drug approvals in bipolar depression

                    • Statistically significant improvement in depression on
                      MADRS scale
                    • Well tolerated by patients; the adverse event profile for
Results
                      RG2417 was equivalent to placebo
                    • Clinical benefit demonstrated primarily in patients with
                      moderate-severe disease history
RepliGen                                       Safe Harbor
 This presentation contains forward-looking statements which are made pursuant to the safe harbor
 provisions of Section 21E of the Securities Exchange Act of 1934. The forward-looking statements in this
 presentation do not constitute guarantees of future performance. Investors are cautioned that statements in
 this presentation which are not strictly historical statements, including, without limitation, statements
 regarding current or future financial performance, management's strategy, plans and objectives for future
 operations, clinical trials and results and product development and manufacturing plans and performance
 such as the anticipated growth in the monoclonal antibody market and projected growth in product sales,
 constitute forward-looking statements. Such forward-looking statements are subject to a number of risks and
 uncertainties that could cause actual results to differ materially from those anticipated, including, without
 limitation, risks associated with: the success of current and future collaborative relationships, the market
 acceptance of our products, our ability to compete with larger, better financed pharmaceutical and
 biotechnology companies, new approaches to the treatment of our targeted diseases, our expectation of
 incurring continued losses, our uncertainty of product revenues and profits, our ability to generate future
 revenues, our ability to raise additional capital to continue our drug development programs, the success of
 our clinical trials, our ability to develop and commercialize products, our ability to obtain required regulatory
 approvals, our compliance with all Food and Drug Administration regulations, our ability to obtain, maintain
 and protect intellectual property rights for our products, the risk of litigation regarding our intellectual
 property rights, our limited sales and manufacturing capabilities, our dependence on third-party
 manufacturers and value added resellers, our ability to hire and retain skilled personnel, our volatile stock
 price, and other risks detailed in Repligen's filings with the Securities and Exchange Commission. Repligen
 assumes no obligation to update any forward-looking information contained in this presentation or with
 respect to the announcements described herein.

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RepliGen Advances Late-Stage Therapeutics Pipeline Fueled by Commercial Assets

  • 1. RepliGen RepliGen NASDAQ: RGEN Corporate Overview October 2010
  • 2. RepliGen Company Profile Repligen acquires product candidates, advances them to a key inflection point and captures value by commercializing or partnering • Late-stage pipeline with significant market opportunity – RG1068 to diagnose pancreatitis; >$100M U.S./E.U. market (Phase 3) – RG2417 for bipolar depression; ~$2B market opportunity (Phase 2b) – Two potentially transformative drugs for orphan CNS diseases; >$500M market opportunity each (Pre-clinical) • Financial sustainability – Strong cash position (~$59M); low cash burn and no debt – Profits from Bioprocessing business and royalties fund therapeutics pipeline • Proven strategy – Acquired patent from MIT; developed Erbitux® cell line; licensed to ImClone for $65M – Acquired patent from Univ. of Michigan; developed Orencia® application; licensed to Bristol-Myers for ~$65M(e) in royalties through 2013 Our goal for 2011 is to file an NDA in the U.S. and an MAA in the E.U. for RG1068, secure partner for RG2417; assuming positive data in Q1 2011
  • 3. RepliGen Advancing Pipeline Fueled by Commercial Assets Pre-Clinical Phase 1 Phase 2 Phase 3 Market Animal First Human Proof of Proof of Therapeutic Product Models Trial Concept Efficacy Pipeline RG1068 Complete Pancreatic Imaging Phase 3 RG2417 Complete Bipolar Disorder Phase 2b RG2833 Initiate Friedreich’s Ataxia Phase 1 RG3039 Qualify clinical Spinal Muscular Atrophy candidate FY2011(e) Commercial Assets ~$13M in Bioprocessing revenue Business Biologics Purification ~$10M in revenue Orencia® Royalties Rheumatoid Arthritis = FY2011 Goal (03/31/11)
  • 4. Pancreatic Imaging Pre-RG1068 Post-RG1068 Biology Liver Liver RG1068 stimulates the Bladder Bladder release of fluid by the pancreas; filling the Pancreas Pancreas Pancreas Pancreas pancreatic ducts, which makes them larger Intestine Intestine Pre-RG1068 Post-RG1068 Clinical Application 4-6 minutes post-RG1068 enhanced MRI leads to an improved image of ducts; diagnosis of pancreas divisum which means patient is a candidate for surgery
  • 5. RG1068 Phase 3 Highlights • ERCP is an invasive, risky endoscopic procedure for diagnosis of pancreatic duct abnormalities; associated with high morbidity and measurable mortality • Patients in the Phase 3 study were evaluated for detection of 10 pre-specified structural pancreatic abnormalities. MRI images pre- and post-RG1068 were analyzed by three independent radiologists; ERCP used as “truth standard” – Radiologists evaluation of data was flawed due to significant deviations from the protocol – FDA and EMA agreed to a “re-read” of images using three new radiologists – Study showed that RG1068 aids in avoiding unnecessary ERCP; for every patient on day of hospitalization from ERCP was saved • Expect to complete Phase 3 re-read in Q1 2011 • If successful, file New Drug Application (NDA) in Q2/Q3 2011
  • 6. Phase 3 Results 78% 74% 66% 60% 52% 46% 34% 35% p<0.001 p<0.001 p<0.001 p<0.001 Enhanced image quality and duct visualization results in improved sensitivity to detect abnormalities and confidence in diagnosis *Sensitivity data is for Reader 1 only
  • 7. Market Opportunity Clinical Application U.S. E.U. Abdominal MRI 200,000 150,000 Enhance existing MRI images Endoscopy Expand market opportunity; triage to appropriate 100,000 TBD therapy and convert diagnostic endoscopy to MRI • Market opportunity in U.S./E.U. for MRI enhancement is greater than $100M, assuming a price of $350/vial • Evaluating other clinical applications and market opportunity outside of U.S./E.U. – Surgery: improve confidence in diagnosis and pre-surgical planning – New potential indication: pancreatic cancer detection/staging – Japan is a third promising market; high utilization of MRI and potential for favorable pricing
  • 8. RG2417 for Bipolar Depression • Bipolar disorder symptoms include episodes of severe depression and mania; depressive episodes account for majority of impairment • Current therapies for bipolar depression are ineffective in most patients and have significant side effects • Bipolar disorder is linked to abnormal activity in the mitochondria, the power plant of a cell • RG2417, an oral formulation of uridine, may affect mitochondrial activity and improve symptoms of depression in bipolar disorder with minimal to no side effects
  • 9. RG2417 Phase 2a Highlights • Conducted a Phase 2a study resulting in statistically significant improvement in depression on MADRS scale – RG2417 well tolerated by patients; the adverse event profile for RG2417 was equivalent to placebo • Completed enrollment of Phase 2b study to confirm Phase 2a results • Expecting results from the Phase 2b study in the first quarter of 2011 • Objective: license to a pharmaceutical partner in 2011
  • 10. Phase 2a Results All Patients Significant History of Symptoms 15 depressive episodes (n=33) MADRS Score MADRS Score P= .03 .09 .01 .08 .22 P=0.01 (repeated measures) P<0.001 (repeated measures)
  • 11. Market Opportunity • Patients primarily seek treatment for the depressive symptoms – Physicians report that 77% of the time patients seek treatement for depressive symptoms as opposed to mania symptoms • Psychiatrists have a high level of dissatisfaction with current bipolar depression therapies because they are ineffective in most patients and may have significant side effects • Total costs of bipolar disorder ~$45 billion per year (NIH est.) – Accounts for more than 16 million visits to a physician each year and approximately 150,000 hospitalizations • Worldwide prevalence >5 million • Approximately $2B market opportunity (10% market penetration)
  • 12. RG2833 for Friedreich’s Ataxia • Friedreich’s ataxia is an orphan disease characterized by progressive loss of muscle function which leads to Frataxin Protein Level incapacitation and loss of life • Caused by a defective gene, which results in low levels of Frataxin protein • RG2833, an HDAC* inhibitor, is the first compound to target activation of the defective gene to increase Patients Healthy frataxin production Carriers • >$500M market opportunity A 2.5 fold increase in Frataxin levels may be sufficient to arrest *Histone deacetylase inhibitors (HDAC inhibitors) are a class of compounds that interfere with the function of disease progress histone deacetylase, which is related to gene expression
  • 13. RG2833 Next Steps • Initiate Phase 1 study following FDA approval • Evaluate HDAC inhibitors in animal models of Huntington’s disease and cognition • Prosecute worldwide patent protection; composition of matter patents; priority date May 2008
  • 14. RG3039 for Spinal Muscular Atrophy • Spinal Muscular Atrophy (SMA) is an orphan disease characterized by progressive loss of muscle function and early death in most patients • Caused by a defective gene SMN2, which results in low levels SMN protein • RG3039, is the first compound to target activation of the defective gene; increasing the production of SMN and potentially arresting the progress of the disease • >$500M market opportunity Disease Classification SMN2 Gene Copy Life Expectancy SMA I 73% of patients have 2 copies ~2 Years SMA II 82% of patients have 3 copies ~20 Years 50% of patients have 3 copies SMA III 50 Years and 45% have 4 copies A 1.5 to 2 fold increase in SMN levels has the potential to profoundly affect the course of the disease
  • 15. RG3039 Next Steps • Qualify RG3039 as a clinical candidate – Complete GLP toxicology study to determine appropriateness for human clinical trials • Initiate Phase 1 study • Seek funding from non-profits or NIH • Prosecute composition of matter patent
  • 16. Bioprocessing Overview • For over 20 years, Repligen has been a leading supplier of Protein A for production of monoclonal antibodies; important therapies for cancer, autoimmune diseases and osteoporosis – OEM supply to GE Healthcare and Millipore – FY2011 estimated revenue = ~$13M; 60% gross margin • Repligen’s objective is to expand bioprocessing market opportunity by broadening its product offering, and through direct sales to end users – Protein A Resins – Pre-packed Columns – Analytical Tools
  • 17. Expanding Market Opportunity Legacy Business OEM suppliers End-users Protein A • GE Healthcare • Monoclonals • Millipore Market Size $25M Emerging Business Protein A Protein A Protein A Resins Resins End-users Plug & Play Pre-Packed •Monoclonals Chromatography Columns •Biologics •Vaccines Membranes Analytical Tools Market Size $500M
  • 18. Bioprocessing Business Large Scale Fermentation Large Scale Purification rProtein A “Plug and Play” Opus™ Analytical Tools Affinity Resin Columns ELISA Kits
  • 19. Projected Orencia® Royalties Licensed patent to Bristol-Myers for US sales of Orencia®, a novel therapy for rheumatoid arthritis $13.3 $12.8 Upfront payment $6.3 for back-royalties $11.5 $10.3 $10.5 $9.0 $7.0 FY2011-FY2014 ~ $45M (e)
  • 20. RepliGen Corporate Assets Pre-Clinical Phase 1 Phase 2 Phase 3 Market Animal First Human Proof of Proof of Therapeutic Product Models Trial Concept Efficacy Pipeline RG1068 Complete Pancreatic Imaging Phase 3 RG2417 Complete Bipolar Disorder Phase 2b RG2833 Initiate Friedreich’s Ataxia Phase 1 RG3039 Qualify clinical Spinal Muscular Atrophy candidate FY2011(e) Commercial Assets ~$13M in Bioprocessing revenue Business Biologics Purification ~$10M in revenue Orencia® Royalties Rheumatoid Arthritis = FY2011 Goal (03/31/11)
  • 21. RepliGen Financials FY2010(a) FY2011(e) Ending: 3/31/2010 3/31/2011 Revenue $21M $24-26M Net Loss ($4.1M) (<$3M) EOY Cash* $59.1M $57-58M Shares Outstanding (03/31/10) 30.8M Shares & Vested Options (03/31/10) 32.2M Market Cap (10/04/10) $104M 90 Day Average Volume (10/04/10) 45,000 * Net of acquisitions
  • 22. RepliGen Upcoming News Stream • September 2010: End of enrollment for RG2417 Phase 2b study • November 2010: Presenting at Society for Neurosciences on new indications for HDAC inhibitors • November 2010: Q2FY11 results and quarterly update • Q1 of 2011: Release RG1068 Phase 3 results • Q1 of 2011: Release RG2417 Phase 2b study results • H1 of 2011: Initiate phase 1 study of RG2833 • H1 of 2011: File IND for RG3039
  • 23. RepliGen Why Invest in Repligen? • Expecting data on a phase III development compound as MRI contrast agent for the diagnosis of pancreatic duct abnormalities in Q1, 2011 • Expecting data on a Phase II development compound for bipolar disorder Q1, 2011 • ~$3 stock with $1.50/share in cash. Cash burn is less than $0.10/share for 2011. No debt. • Knowledgeable of monoclonal antibody production process; holds 50% market share of protein A market
  • 24. RepliGen Business Drivers Advance Pipeline Accelerate Bioprocessing Shareholder Value Protect Low Cash Burn Innovation
  • 25. RepliGen Potential Back Up Slides
  • 26. RepliGen Strategic Track Record Acquired Developed Capture Value Value Captured Past Compounds for cancer Erbitux® cell line Licensed to ImClone;$65M treatment from MIT for National Cancer Institute settlement Compounds for RA Orencia® cell line Licensed to Bristol-Myers; From Univ. of Michigan Conducted early clinical trials ~$65M in royalties through 2013 Present and Future Secretin for CNS diseases Completed Phase 3 study Commercialize (US) / Partner (EU); from Univ. of Maryland for pancreatic imaging following FDA approval Uridine as treatment for Completed Phase 2a study Following positive results, qualify Bipolar Disorder Conducting a Phase 2b study potential partners Compounds for Friedreich’s Filed New Drug Application Continue developing to capture Ataxia from Scripps Research for RG2833 value as appropriate Compounds for SMA from Identified clinical candidate Continue developing to capture Families for SMA RG3039 value as appropriate
  • 27. Phase 3 Trial and Results • 258 patients were evaluated with MRI and ERCP for detection of 10 pre-specified structural abnormalities Trial Protocol – ERCP used as “truth standard” for abnormalities – MRIs analyzed by three independent radiologists • Statistically significant improvements in the detection of structural Primary Endpoints abnormalities using RG1068 enhanced MRI vs. MRI alone, with no false positives • Improvements in image quality, visualization of pancreatic ducts and Secondary Endpoints confidence in diagnosis of abnormalities • One out of the three radiologists achieved primary endpoints – 256 additional days of hospitalization with ERCP Results • RG1068 well tolerated and safe vs. ERCP • All three radiologists achieved secondary endpoints • Radiologists evaluation of data was flawed due to significant deviations from the protocol Status • Food and Drug Administration (FDA) and European Medicines Agency (EMA) agreed to a “re-read” of images using three new radiologists
  • 28. Phase 2a Clinical Trial A study with 83 patients, placebo-controlled, daily dosing Protocol and weekly assessment for 6 weeks • Statistically significant decreases in the symptom of depression as measured by the Montgomery Asberg Depression Rating Scale (MADRS) with minimal Primary Endpoint side effects • MADRS has been previously accepted by the FDA for drug approvals in bipolar depression • Statistically significant improvement in depression on MADRS scale • Well tolerated by patients; the adverse event profile for Results RG2417 was equivalent to placebo • Clinical benefit demonstrated primarily in patients with moderate-severe disease history
  • 29. RepliGen Safe Harbor This presentation contains forward-looking statements which are made pursuant to the safe harbor provisions of Section 21E of the Securities Exchange Act of 1934. The forward-looking statements in this presentation do not constitute guarantees of future performance. Investors are cautioned that statements in this presentation which are not strictly historical statements, including, without limitation, statements regarding current or future financial performance, management's strategy, plans and objectives for future operations, clinical trials and results and product development and manufacturing plans and performance such as the anticipated growth in the monoclonal antibody market and projected growth in product sales, constitute forward-looking statements. Such forward-looking statements are subject to a number of risks and uncertainties that could cause actual results to differ materially from those anticipated, including, without limitation, risks associated with: the success of current and future collaborative relationships, the market acceptance of our products, our ability to compete with larger, better financed pharmaceutical and biotechnology companies, new approaches to the treatment of our targeted diseases, our expectation of incurring continued losses, our uncertainty of product revenues and profits, our ability to generate future revenues, our ability to raise additional capital to continue our drug development programs, the success of our clinical trials, our ability to develop and commercialize products, our ability to obtain required regulatory approvals, our compliance with all Food and Drug Administration regulations, our ability to obtain, maintain and protect intellectual property rights for our products, the risk of litigation regarding our intellectual property rights, our limited sales and manufacturing capabilities, our dependence on third-party manufacturers and value added resellers, our ability to hire and retain skilled personnel, our volatile stock price, and other risks detailed in Repligen's filings with the Securities and Exchange Commission. Repligen assumes no obligation to update any forward-looking information contained in this presentation or with respect to the announcements described herein.