UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM 8-K
CURRENT REPORT
Pursuant to Section 13 or 15(d)
of the Securities Exchange Act of 1934
Date of Report (Date of earliest event reported): June 18, 2020
CATALYST BIOSCIENCES, INC.
(Exact name of registrant as specified in its charter)
| Delaware | 000-51173 | 56-2020050 | ||
| (State or other jurisdiction of incorporation) |
(Commission File Number) |
(IRS Employer Identification No.) |
611 Gateway Blvd, Suite 710, South San Francisco, CA 94080
(Address of principal executive offices)
(650) 871-0761
(Registrant’s telephone number, including area code)
Not Applicable
(Former name or former address, if changed since last report.)
Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:
| ☐ | Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425) |
| ☐ | Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12) |
| ☐ | Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b)) |
| ☐ | Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c)) |
Securities registered pursuant to Section 12(b) of the Act:
| Title of each class |
Trading Symbol(s) |
Name of each exchange on which registered | ||
| Common Stock | CBIO | Nasdaq |
Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§ 230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§ 240.12b-2 of this chapter).
Emerging growth company ☐
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐
| Item 7.01 | Regulation FD Disclosure. |
On June 17, 2020, Catalyst Biosciences, Inc. (the “Company”) posted an update to its corporate presentation (the “Presentation”) on its website, ir.catalystbiosciences.com/presentations-events. The Presentation was also presented by the Company on June 18, 2020 at the Raymond James Health Innovation Conference. A copy of the Presentation is attached hereto as Exhibit 99.1.
The information in this Item 7.01 of this Current Report on Form 8-K (including Exhibit 99.1) is being furnished and shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities of that section. The information in this Current Report shall not be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended, or the Exchange Act, except as expressly set forth by specific reference in such a filing.
| Item 9.01 | Financial Statements and Exhibits. |
(d) Exhibits
| Exhibit |
Description | |
| 99.1 | Presentation slide deck. | |
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
| CATALYST BIOSCIENCES, INC. | ||||||
| Date: June 18, 2020 | /s/ Nassim Usman | |||||
| Nassim Usman, Ph.D. | ||||||
| President and Chief Executive Officer | ||||||
Exhibit 99.1 CATALYST BIOSCIENCES Corporate Overview 18 June 2020 1Exhibit 99.1 CATALYST BIOSCIENCES Corporate Overview 18 June 2020 1
Nasdaq: CBIO Forward looking statements Various important factors could cause actual results or events to differ This presentation includes forward-looking statements that involve materially, including, but not limited to, the risk that the risk that trials substantial risks and uncertainties. All statements included in this and studies may be delayed as a result of the COVID-19 virus and presentation, other than statement of historical facts, are forward- other factors, that trials may not have satisfactory outcomes, that looking statements. Examples of such statements include, but are potential adverse effects may arise from the testing or use of MarzAA not limited to, potential markets for MarzAA, DalcA and CB 2782- or DalcA, including the generation of antibodies, which has been PEG, potential benefits of subcutaneous dosing, potential use of observed in patients treated with DalcA, that clinical trials will take MarzAA as a subcutaneous therapy for patients with hemophilia A longer than anticipated to be completed, that costs required to or B with inhibitors, treatment of bleeding, Factor VII deficiency, develop or manufacture the Company’s products will be higher than Glanzmann’s Thrombasthenia and other bleeding disorders, anticipated, that Biogen will discontinue development of CB 2782- potential use of DalcA as a subcutaneous therapy for patients with PEG, competition and other factors that affect our ability to establish hemophilia B, potential benefits of CB 2679d-GT as gene therapy, collaborations on commercially reasonable terms and other risks the use of engineered proteases to treat diseases, including dAMD, described in the “Risk Factors” section of the Company’s annual by mediating the complement cascade, clinical trial results, plans for report on Form 10-K filed with the Securities and Exchange a registrational trial for MarzAA and a Phase 1/2 trial in Factor VII Commission on February 20, 2020, and the Company’s quarterly deficiency, Glanzmann’s Thrombasthenia and treatment of bleeding report on Form 10-Q filed on May 11, 2020, and in other filings with in Hemlibra subjects in Q4 2020, plans to declare development the Securities and Exchange Commission. The Company does not candidates for CB 2679d-GT and in the complement program in Q4 assume any obligation to update any forward-looking statements, 2020, and potential milestone and royalty payments from Biogen. except as required by law. Actual results or events could differ materially from the plans, expectations and projections disclosed in these forward-looking statements. 2 catalystbiosciences.comNasdaq: CBIO Forward looking statements Various important factors could cause actual results or events to differ This presentation includes forward-looking statements that involve materially, including, but not limited to, the risk that the risk that trials substantial risks and uncertainties. All statements included in this and studies may be delayed as a result of the COVID-19 virus and presentation, other than statement of historical facts, are forward- other factors, that trials may not have satisfactory outcomes, that looking statements. Examples of such statements include, but are potential adverse effects may arise from the testing or use of MarzAA not limited to, potential markets for MarzAA, DalcA and CB 2782- or DalcA, including the generation of antibodies, which has been PEG, potential benefits of subcutaneous dosing, potential use of observed in patients treated with DalcA, that clinical trials will take MarzAA as a subcutaneous therapy for patients with hemophilia A longer than anticipated to be completed, that costs required to or B with inhibitors, treatment of bleeding, Factor VII deficiency, develop or manufacture the Company’s products will be higher than Glanzmann’s Thrombasthenia and other bleeding disorders, anticipated, that Biogen will discontinue development of CB 2782- potential use of DalcA as a subcutaneous therapy for patients with PEG, competition and other factors that affect our ability to establish hemophilia B, potential benefits of CB 2679d-GT as gene therapy, collaborations on commercially reasonable terms and other risks the use of engineered proteases to treat diseases, including dAMD, described in the “Risk Factors” section of the Company’s annual by mediating the complement cascade, clinical trial results, plans for report on Form 10-K filed with the Securities and Exchange a registrational trial for MarzAA and a Phase 1/2 trial in Factor VII Commission on February 20, 2020, and the Company’s quarterly deficiency, Glanzmann’s Thrombasthenia and treatment of bleeding report on Form 10-Q filed on May 11, 2020, and in other filings with in Hemlibra subjects in Q4 2020, plans to declare development the Securities and Exchange Commission. The Company does not candidates for CB 2679d-GT and in the complement program in Q4 assume any obligation to update any forward-looking statements, 2020, and potential milestone and royalty payments from Biogen. except as required by law. Actual results or events could differ materially from the plans, expectations and projections disclosed in these forward-looking statements. 2 catalystbiosciences.com
Nasdaq: CBIO Essential Medicines – Superior Outcomes Late-Stage Asset Hemophilia Complement SQ MarzAA (FVIIa) IVT Anti-C3 Dry AMD SQ Marzeptacog alfa CB 2782-PEG (activated) SQ Dalcinonacog MarzAA (FVIIa) alfa – DalcA (FIX) Factor IX Gene Therapy SQ Systemic Phase 3 in 2020 Complement Factor Xa Inhibitors Protease Engineering Platform 3 catalystbiosciences.comNasdaq: CBIO Essential Medicines – Superior Outcomes Late-Stage Asset Hemophilia Complement SQ MarzAA (FVIIa) IVT Anti-C3 Dry AMD SQ Marzeptacog alfa CB 2782-PEG (activated) SQ Dalcinonacog MarzAA (FVIIa) alfa – DalcA (FIX) Factor IX Gene Therapy SQ Systemic Phase 3 in 2020 Complement Factor Xa Inhibitors Protease Engineering Platform 3 catalystbiosciences.com
Nasdaq: CBIO Pipeline Hemostasis R PC P1/2 P2 P3 SQ Marzeptacog alfa MarzAA” – (rFVIIa) Hemophilia A or B w Inhibitors – ToB FVIID/Glanzmann/Hemlibra – ToB SQ Dalcinonacog alfa “DalcA” Hemophilia B (rFIX) FIX-Gene Therapy Hemophilia B (CB 2679d-GT) Complement IVT CB 2782-PEG anti-C3 protease for Dry AMD SQ Systemic complement inhibitors – CB DC 4 catalystbiosciences.comNasdaq: CBIO Pipeline Hemostasis R PC P1/2 P2 P3 SQ Marzeptacog alfa MarzAA” – (rFVIIa) Hemophilia A or B w Inhibitors – ToB FVIID/Glanzmann/Hemlibra – ToB SQ Dalcinonacog alfa “DalcA” Hemophilia B (rFIX) FIX-Gene Therapy Hemophilia B (CB 2679d-GT) Complement IVT CB 2782-PEG anti-C3 protease for Dry AMD SQ Systemic complement inhibitors – CB DC 4 catalystbiosciences.com
Nasdaq: CBIO Investment highlights Anti-C3 Dry AMD with Biogen Novel subcutaneous factors with orphan drug designation, FVIIa SQ systemic complement MarzAA & DalcA – P2 efficacy FIX regulator research program in prophylaxis studies complete Multibillion-dollar market Experienced team opportunities 177 worldwide patents Strong balance sheet, CBIO retains full ownership $104.5 M cash – Q1 5 of all compounds 5 catalystbiosciences.comNasdaq: CBIO Investment highlights Anti-C3 Dry AMD with Biogen Novel subcutaneous factors with orphan drug designation, FVIIa SQ systemic complement MarzAA & DalcA – P2 efficacy FIX regulator research program in prophylaxis studies complete Multibillion-dollar market Experienced team opportunities 177 worldwide patents Strong balance sheet, CBIO retains full ownership $104.5 M cash – Q1 5 of all compounds 5 catalystbiosciences.com
Nasdaq: CBIO Addressing unmet needs in orphan bleeding disorders SQ treatment of episodic bleeding and prophylaxis – $4B+ market Hemophilia A MarzAA & DalcA Prophylaxis w/ Inhibitors Hemophilia B Hemophilia B Hemophilia A w/ Inhibitors Acquired Factor VII Hemophilia Deficiency Glanzmann Thrombasthenia 6 catalystbiosciences.comNasdaq: CBIO Addressing unmet needs in orphan bleeding disorders SQ treatment of episodic bleeding and prophylaxis – $4B+ market Hemophilia A MarzAA & DalcA Prophylaxis w/ Inhibitors Hemophilia B Hemophilia B Hemophilia A w/ Inhibitors Acquired Factor VII Hemophilia Deficiency Glanzmann Thrombasthenia 6 catalystbiosciences.com
Nasdaq: CBIO The Catalyst Biosciences subcutaneous solution Our highly potent candidates + Quick & simple self-administered SQ injection + SQ dosing is the future in hemophilia, other rare hematology indications & complement mediated diseases + Significantly increases half-life + Much higher & more stable factor levels for prophylaxis + Enables SQ treatment of bleeds + Ideal for children and adolescents 7 catalystbiosciences.comNasdaq: CBIO The Catalyst Biosciences subcutaneous solution Our highly potent candidates + Quick & simple self-administered SQ injection + SQ dosing is the future in hemophilia, other rare hematology indications & complement mediated diseases + Significantly increases half-life + Much higher & more stable factor levels for prophylaxis + Enables SQ treatment of bleeds + Ideal for children and adolescents 7 catalystbiosciences.com
Nasdaq: CBIO Marzeptacog alfa (activated): MarzAA rFVIIa Addresses a clear unmet need in hemophilia & other bleeding disorders Four amino acid substitutions T128N P129A + Multiple advantages over NovoSeven RT + 9-fold higher activity vs NovoSeven RT + Potency allows for SQ dosing M298Q Only SQ bypass agent for on demand treatment Q286R + Simple, small volume SQ administration + Improved bioavailability & prolonged half-life Increased procoagulant activity Orphan Drug Designation in US and EU 8 catalystbiosciences.comNasdaq: CBIO Marzeptacog alfa (activated): MarzAA rFVIIa Addresses a clear unmet need in hemophilia & other bleeding disorders Four amino acid substitutions T128N P129A + Multiple advantages over NovoSeven RT + 9-fold higher activity vs NovoSeven RT + Potency allows for SQ dosing M298Q Only SQ bypass agent for on demand treatment Q286R + Simple, small volume SQ administration + Improved bioavailability & prolonged half-life Increased procoagulant activity Orphan Drug Designation in US and EU 8 catalystbiosciences.com
Nasdaq: CBIO MarzAA Phase 2 demonstrates efficacy in prophylaxis Greater than 90% reduction in all bleeding – Median ABR = 0 7 of 9 subjects had no bleeding at final dose level Safe & well tolerated, ~1% ISR (6/517 doses) & no ADA 12.3% 19.8 Annualized Proportion of Days Bleed Rate with Bleeding n = 9 n = 9 1.6, p = 0.009 0.8%, p = 0.009 6 m pre-treatment On treatment 6 m pre-treatment On treatment Mahlangu et al. EAHAD 2020 9 catalystbiosciences.comNasdaq: CBIO MarzAA Phase 2 demonstrates efficacy in prophylaxis Greater than 90% reduction in all bleeding – Median ABR = 0 7 of 9 subjects had no bleeding at final dose level Safe & well tolerated, ~1% ISR (6/517 doses) & no ADA 12.3% 19.8 Annualized Proportion of Days Bleed Rate with Bleeding n = 9 n = 9 1.6, p = 0.009 0.8%, p = 0.009 6 m pre-treatment On treatment 6 m pre-treatment On treatment Mahlangu et al. EAHAD 2020 9 catalystbiosciences.com
Nasdaq: CBIO In a world of SQ prophylaxis Patients & KOLs want SQ treatment of a bleed MarzAA has optimal properties ® Individuals on Hemlibra have breakthrough bleeds ü Fast & easy to administer ü Achieves therapeutic levels rapidly ® NovoSeven is safe but is ü Stops bleeding in multiple administered IV preclinical models ü Can be combined with Hemlibra FEIBA should not be used with in vitro without increased Hemlibra and is given IV thrombogenicity 10 catalystbiosciences.comNasdaq: CBIO In a world of SQ prophylaxis Patients & KOLs want SQ treatment of a bleed MarzAA has optimal properties ® Individuals on Hemlibra have breakthrough bleeds ü Fast & easy to administer ü Achieves therapeutic levels rapidly ® NovoSeven is safe but is ü Stops bleeding in multiple administered IV preclinical models ü Can be combined with Hemlibra FEIBA should not be used with in vitro without increased Hemlibra and is given IV thrombogenicity 10 catalystbiosciences.com
Nasdaq: CBIO MarzAA P3: On demand treatment of episodic bleeding CRIMSON-1 Registration Study – A Global Clinical Trial Opportunity in multiple Phase 1 & 2 trials Open label trial evaluating bleeding disorders demonstrated the clinical the safety & efficacy of SQ impact of SQ MarzAA MarzAA in episodic bleeding • MAA-102 rapidly achieved • Primary endpoint: Hemostatic✓ Hemophilia A or B with target activity levels efficacy using a standard inhibitors 4-point assessment scale • MAA-201 demonstrated ✓ Hemlibra breakthrough efficacy in prophylaxis, safe & • ~230 bleeding episodes to be bleeds well tolerated with no ADA treated in ~75 HA/HB ✓ Factor VII deficiency individuals with inhibitors • Clinically support P3 SQ ✓ Glanzmann thrombasthenia MarzAA treatment of episodic • Anticipate first patient enrolled • Acquired hemophilia bleeding by end of 2020 11 catalystbiosciences.comNasdaq: CBIO MarzAA P3: On demand treatment of episodic bleeding CRIMSON-1 Registration Study – A Global Clinical Trial Opportunity in multiple Phase 1 & 2 trials Open label trial evaluating bleeding disorders demonstrated the clinical the safety & efficacy of SQ impact of SQ MarzAA MarzAA in episodic bleeding • MAA-102 rapidly achieved • Primary endpoint: Hemostatic✓ Hemophilia A or B with target activity levels efficacy using a standard inhibitors 4-point assessment scale • MAA-201 demonstrated ✓ Hemlibra breakthrough efficacy in prophylaxis, safe & • ~230 bleeding episodes to be bleeds well tolerated with no ADA treated in ~75 HA/HB ✓ Factor VII deficiency individuals with inhibitors • Clinically support P3 SQ ✓ Glanzmann thrombasthenia MarzAA treatment of episodic • Anticipate first patient enrolled • Acquired hemophilia bleeding by end of 2020 11 catalystbiosciences.com
Nasdaq: CBIO MarzAA development plan in 2020 Phase 3 HA/HB w Inhibitors – ToB Phase 1/2 study in FVIID, Glanzmann & Hemlibra ToB Large commercial opportunity across multiple rare bleeding disorders Phase 1 PK/PD data support on demand as well as prophylactic treatment of bleeding Phase 2 demonstrated clinical efficacy & tolerability for prophylaxis indications Efficacy demonstrated for SQ on demand treatment of bleeding in pre-clinical models MarzAA can be safely combined with Hemlibra in human plasma in vitro 12 catalystbiosciences.comNasdaq: CBIO MarzAA development plan in 2020 Phase 3 HA/HB w Inhibitors – ToB Phase 1/2 study in FVIID, Glanzmann & Hemlibra ToB Large commercial opportunity across multiple rare bleeding disorders Phase 1 PK/PD data support on demand as well as prophylactic treatment of bleeding Phase 2 demonstrated clinical efficacy & tolerability for prophylaxis indications Efficacy demonstrated for SQ on demand treatment of bleeding in pre-clinical models MarzAA can be safely combined with Hemlibra in human plasma in vitro 12 catalystbiosciences.com
Nasdaq: CBIO Dalcinonacog alfa: novel FIX replacement for SQ delivery Three amino acid substitutions + Increased catalytic activity + Higher affinity for FVIIIa + Resistance to antithrombin inhibition + 22-fold increased potency vs BeneFIX Differentiated from marketed IV FIXs + Simple, small volume SQ administration R318Y + Enhanced pharmacokinetics with prolonged half-life + Excellent extravascular distribution Resistance to antithrombin T343R + Potential to maintain continuous protective levels R338E Increased FVIIIa affinity Orphan Drug Designation in US & EU & procoagulant activity 13 catalystbiosciences.comNasdaq: CBIO Dalcinonacog alfa: novel FIX replacement for SQ delivery Three amino acid substitutions + Increased catalytic activity + Higher affinity for FVIIIa + Resistance to antithrombin inhibition + 22-fold increased potency vs BeneFIX Differentiated from marketed IV FIXs + Simple, small volume SQ administration R318Y + Enhanced pharmacokinetics with prolonged half-life + Excellent extravascular distribution Resistance to antithrombin T343R + Potential to maintain continuous protective levels R338E Increased FVIIIa affinity Orphan Drug Designation in US & EU & procoagulant activity 13 catalystbiosciences.com
Nasdaq: CBIO Dalcinonacog alfa phase 2b SQ clinical trial Trial completed Initial 35 min Daily SQ Dosing to Day 28 Wash-out Post-IV Daily FIX activity levels DalcA IV DalcA SQ DalcA SQ measured for 5 days 50 IU/kg 100 IU/kg 100 IU/kg endpoint achieved with steady state FIX activity levels > 12% + Primary endpoint: Steady state FIX + 6 severe Hemophilia B subjects dosed activity level above 12% with daily dosing + Rare propeptide mutation excluded + HLA profile associated with nAb risk was + Secondary endpoints: safety including excluded weekly ADA testing, pharmacokinetics, pharmacodynamics, bleeding events 14 catalystbiosciences.comNasdaq: CBIO Dalcinonacog alfa phase 2b SQ clinical trial Trial completed Initial 35 min Daily SQ Dosing to Day 28 Wash-out Post-IV Daily FIX activity levels DalcA IV DalcA SQ DalcA SQ measured for 5 days 50 IU/kg 100 IU/kg 100 IU/kg endpoint achieved with steady state FIX activity levels > 12% + Primary endpoint: Steady state FIX + 6 severe Hemophilia B subjects dosed activity level above 12% with daily dosing + Rare propeptide mutation excluded + HLA profile associated with nAb risk was + Secondary endpoints: safety including excluded weekly ADA testing, pharmacokinetics, pharmacodynamics, bleeding events 14 catalystbiosciences.com
Nasdaq: CBIO DalcA P2b efficacy & safety demonstrated Target levels >12% achieved with 100 IU/kg dosing for 28 Days + Dosed 6 severe HB subjects § 102 withdrew on Day 7 + Steady state FIX levels up to 27% achieved after 14 days + No breakthrough bleeds + No neutralizing ADAs Target + Consistent PK profiles Level + Terminal half-life is 2.5 - 5.1 days Wash-Out 15 catalystbiosciences.comNasdaq: CBIO DalcA P2b efficacy & safety demonstrated Target levels >12% achieved with 100 IU/kg dosing for 28 Days + Dosed 6 severe HB subjects § 102 withdrew on Day 7 + Steady state FIX levels up to 27% achieved after 14 days + No breakthrough bleeds + No neutralizing ADAs Target + Consistent PK profiles Level + Terminal half-life is 2.5 - 5.1 days Wash-Out 15 catalystbiosciences.com
Nasdaq: CBIO Dalcinonacog alfa Potential to provide effective SQ prophylaxis for individuals with Hemophilia B Phase 2b trial complete Excellent protective therapeutic FIX activity levels achieved No bleeding events during treatment demonstrates effective prophylaxis No SAEs, systemic hypersensitivity, nAb to DalcA or wild-type FIX Mild to moderate ISR primarily with initial injections – transient & self-limiting Long half-life – demonstrates potential to lower dose / reduce dosing frequency 16 catalystbiosciences.comNasdaq: CBIO Dalcinonacog alfa Potential to provide effective SQ prophylaxis for individuals with Hemophilia B Phase 2b trial complete Excellent protective therapeutic FIX activity levels achieved No bleeding events during treatment demonstrates effective prophylaxis No SAEs, systemic hypersensitivity, nAb to DalcA or wild-type FIX Mild to moderate ISR primarily with initial injections – transient & self-limiting Long half-life – demonstrates potential to lower dose / reduce dosing frequency 16 catalystbiosciences.com
Nasdaq: CBIO FIX gene therapy: CB 2679d-GT for hemophilia B CB 2679d-GT in combination with a novel chimeric AAV capsid provides significant improvements + Stable high activity levels in a mouse hemophilia B model – no nAb + Vector dose reduced 10-fold compared to current constructs FIX AAV Study Dose FIX Activity Transgene Capsid (vg/kg) (U/mL) + Potential for an improved efficacy & safety profile Novel 10 CB 2679d-GT 8.0x10 20 Chimeric + AAV license and sponsored research agreement with Stanford University School of Medicine * 11 Padua TAK-748 7.4x10 20 Superior preclinical efficacy of * 10 Padua TAK-748 7.4x10 1 CB 2679d-GT vs Padua + 4 to 5-fold reduction in bleeding time *Weiller et al. (2019) Blood Vol. 134, Supplement S1 P4633 + Activity levels elevated throughout the study - no nAb Wholly-owned & issued patents covering gene therapy Blouse et al. EAHAD 2019 & 2020 17 catalystbiosciences.comNasdaq: CBIO FIX gene therapy: CB 2679d-GT for hemophilia B CB 2679d-GT in combination with a novel chimeric AAV capsid provides significant improvements + Stable high activity levels in a mouse hemophilia B model – no nAb + Vector dose reduced 10-fold compared to current constructs FIX AAV Study Dose FIX Activity Transgene Capsid (vg/kg) (U/mL) + Potential for an improved efficacy & safety profile Novel 10 CB 2679d-GT 8.0x10 20 Chimeric + AAV license and sponsored research agreement with Stanford University School of Medicine * 11 Padua TAK-748 7.4x10 20 Superior preclinical efficacy of * 10 Padua TAK-748 7.4x10 1 CB 2679d-GT vs Padua + 4 to 5-fold reduction in bleeding time *Weiller et al. (2019) Blood Vol. 134, Supplement S1 P4633 + Activity levels elevated throughout the study - no nAb Wholly-owned & issued patents covering gene therapy Blouse et al. EAHAD 2019 & 2020 17 catalystbiosciences.com
Nasdaq: CBIO Complement cascade is regulated by proteases Lectin Pathway Classical Pathway Alternative Pathway Autoimmune Diseases Inflammatory Diseases Multiple Rare Diseases C3 Convertase Ocular Disorders C3 IVT CB 2782-PEG Amplification Loop Targeting C3 blocks the downstream complement cascade Antigen uptake C3b Cell removal Lysis C3a Cell death Secretion C5 Convertase Proliferation Inflammation C5a C5b C5 MAC Complex 18 catalystbiosciences.comNasdaq: CBIO Complement cascade is regulated by proteases Lectin Pathway Classical Pathway Alternative Pathway Autoimmune Diseases Inflammatory Diseases Multiple Rare Diseases C3 Convertase Ocular Disorders C3 IVT CB 2782-PEG Amplification Loop Targeting C3 blocks the downstream complement cascade Antigen uptake C3b Cell removal Lysis C3a Cell death Secretion C5 Convertase Proliferation Inflammation C5a C5b C5 MAC Complex 18 catalystbiosciences.com
Nasdaq: CBIO CB 2782-PEG: Complement factor 3 (C3) cleaving protease Geographic Atrophy in Dry AMD can result in blindness + Geographic atrophy is an advanced stage of dry age-related macular degeneration (dAMD) + Dry AMD affects ~1M people in the US and over 5M worldwide + Global market estimated at >$5B + C3 is the only clinically (randomized P2) validated target for the treatment of dAMD + No currently approved drugs Sources: National Eye Institute. Facts About Age-Related Macular Degeneration, Tufail 2015, The Eye Diseases Prevalence Research Group 2004, GlobalData 19 catalystbiosciences.comNasdaq: CBIO CB 2782-PEG: Complement factor 3 (C3) cleaving protease Geographic Atrophy in Dry AMD can result in blindness + Geographic atrophy is an advanced stage of dry age-related macular degeneration (dAMD) + Dry AMD affects ~1M people in the US and over 5M worldwide + Global market estimated at >$5B + C3 is the only clinically (randomized P2) validated target for the treatment of dAMD + No currently approved drugs Sources: National Eye Institute. Facts About Age-Related Macular Degeneration, Tufail 2015, The Eye Diseases Prevalence Research Group 2004, GlobalData 19 catalystbiosciences.com
Nasdaq: CBIO CB 2782-PEG long acting anti-C3 protease Best-in-class anti-C3 profile for dry AMD Biogen Collaboration + Generated from Catalyst’s proprietary + Announced December 19, 2019 protease engineering platform + $15M upfront, up to $340M in milestones + Potent, selective and long acting anti-C3 and tiered royalties up to low double digits protease that degrades C3 into inactive + Catalyst to perform fully funded pre-clinical fragments and manufacturing activities + Preclinical NHP PK & PD data* predict + Biogen responsible for IND-enabling best-in-class human intravitreal dosing activities, worldwide clinical development & three or four times a year commercialization *Furfine et al. ARVO 2019 20 catalystbiosciences.comNasdaq: CBIO CB 2782-PEG long acting anti-C3 protease Best-in-class anti-C3 profile for dry AMD Biogen Collaboration + Generated from Catalyst’s proprietary + Announced December 19, 2019 protease engineering platform + $15M upfront, up to $340M in milestones + Potent, selective and long acting anti-C3 and tiered royalties up to low double digits protease that degrades C3 into inactive + Catalyst to perform fully funded pre-clinical fragments and manufacturing activities + Preclinical NHP PK & PD data* predict + Biogen responsible for IND-enabling best-in-class human intravitreal dosing activities, worldwide clinical development & three or four times a year commercialization *Furfine et al. ARVO 2019 20 catalystbiosciences.com
Nasdaq: CBIO CB 2782-PEG long acting anti-C3 protease Best-in-class anti-C3 profile for dry AMD with dosing every 3 to 4 months Non-Human Primates Human Modeling Predicted >90% elimination of C3 at 4 months 21 catalystbiosciences.comNasdaq: CBIO CB 2782-PEG long acting anti-C3 protease Best-in-class anti-C3 profile for dry AMD with dosing every 3 to 4 months Non-Human Primates Human Modeling Predicted >90% elimination of C3 at 4 months 21 catalystbiosciences.com
Nasdaq: CBIO CB 4332 SQ long-acting systemic complement regulator Non-human primate PK supports weekly SQ dosing in humans Expanding the complement portfolio CB 4332 + Leverages Catalyst’s proprietary protease engineering platform + Designed for SQ administration & improved bioavailability Parent Functional Molecule Improvements + Simple & efficient production process 22 catalystbiosciences.comNasdaq: CBIO CB 4332 SQ long-acting systemic complement regulator Non-human primate PK supports weekly SQ dosing in humans Expanding the complement portfolio CB 4332 + Leverages Catalyst’s proprietary protease engineering platform + Designed for SQ administration & improved bioavailability Parent Functional Molecule Improvements + Simple & efficient production process 22 catalystbiosciences.com
Nasdaq: CBIO Milestones – 2020 Q1 Q2 Q3 Q4 • Initiate pivotal P3 EoP2 ToB PK/PD • MAA-102 data MarzAA • Initiate P1/2 in FVII (FVIIa) • Population PK Deficiency, Glanzmann Thrombasthenia, and Hemlibra patients Interim P2b Final P2b DalcA (FIX) • Development NextGen Vector • NHP Efficacy CB 2679d-GT Candidate (FIX Gene Therapy) CB 2782-PEG (dAMD) • Development CB DC Candidate (Systemic complement) 23 catalystbiosciences.comNasdaq: CBIO Milestones – 2020 Q1 Q2 Q3 Q4 • Initiate pivotal P3 EoP2 ToB PK/PD • MAA-102 data MarzAA • Initiate P1/2 in FVII (FVIIa) • Population PK Deficiency, Glanzmann Thrombasthenia, and Hemlibra patients Interim P2b Final P2b DalcA (FIX) • Development NextGen Vector • NHP Efficacy CB 2679d-GT Candidate (FIX Gene Therapy) CB 2782-PEG (dAMD) • Development CB DC Candidate (Systemic complement) 23 catalystbiosciences.com
Nasdaq: CBIO Team President & CEO Chief Medical Officer Nassim Usman, Ph.D. Howard Levy, M.B.B.Ch., Ph.D., M.M.M. 18 years 26 years in hematology in biotech Chief Financial Officer SVP , Translational Research Clinton Musil, M.B.A Grant Blouse, Ph.D. 16 years in biotech & 13 years i-banking in biotech SVP, Business Development SVP, Technical Operations Jeffrey Landau, M.B.A. Andrew Hetherington, M.B.A. 18 years 20 years in biotech in biotech 24 catalystbiosciences.comNasdaq: CBIO Team President & CEO Chief Medical Officer Nassim Usman, Ph.D. Howard Levy, M.B.B.Ch., Ph.D., M.M.M. 18 years 26 years in hematology in biotech Chief Financial Officer SVP , Translational Research Clinton Musil, M.B.A Grant Blouse, Ph.D. 16 years in biotech & 13 years i-banking in biotech SVP, Business Development SVP, Technical Operations Jeffrey Landau, M.B.A. Andrew Hetherington, M.B.A. 18 years 20 years in biotech in biotech 24 catalystbiosciences.com
Nasdaq: CBIO Summary Disruptive approach to billion-dollar markets – protease engineering platform FVIIa: SQ MarzAA ~$2.2B market Anti-C3 dAMD: IVT CB 2782-PEG >$5B market + P1 PK/PD & preclinical data supports ToB + Biogen collaboration + P2 efficacy & safety demonstrated + $15M upfront, up to $340M in milestones, up to low double digits tiered royalties + P3 patient enrollment in Q4 2020 FIX: SQ DalcA >$1.8B market SQ systemic complement inhibitor program + Large $B+ rare disease opportunity + Phase 2b efficacy & safety demonstrated + Multiple indications & applications + Potential for less frequent dosing st + 1 Development Candidate in Q4 2020 FIX Gene Therapy: CB 2679d-GT Well capitalized + Proprietary preclinical gene therapy + Cash runway into 2022 asset with superior activity vs current clinical constructs 25 catalystbiosciences.comNasdaq: CBIO Summary Disruptive approach to billion-dollar markets – protease engineering platform FVIIa: SQ MarzAA ~$2.2B market Anti-C3 dAMD: IVT CB 2782-PEG >$5B market + P1 PK/PD & preclinical data supports ToB + Biogen collaboration + P2 efficacy & safety demonstrated + $15M upfront, up to $340M in milestones, up to low double digits tiered royalties + P3 patient enrollment in Q4 2020 FIX: SQ DalcA >$1.8B market SQ systemic complement inhibitor program + Large $B+ rare disease opportunity + Phase 2b efficacy & safety demonstrated + Multiple indications & applications + Potential for less frequent dosing st + 1 Development Candidate in Q4 2020 FIX Gene Therapy: CB 2679d-GT Well capitalized + Proprietary preclinical gene therapy + Cash runway into 2022 asset with superior activity vs current clinical constructs 25 catalystbiosciences.com
THANK YOU Nasdaq: CBIO catalystbiosciences.comTHANK YOU Nasdaq: CBIO catalystbiosciences.com
Nasdaq: CBIO MarzAA is only bypass agent for both SQ prophylaxis and SQ treatment of bleeds Attractive commercial profile targeting an existing $2.2B bypass agent market SQ MarzAA has a superior profile to IV IV NovoSeven ($1.2B 2019 sales) validates NovoSeven – over 100 clinicians & 175 rFVIIa in multiple rare bleeding disorders patients surveyed + Hemophilia A or B with inhibitors + Physicians & patients overwhelmingly + Severe Factor VII Deficiency prefer SQ MarzAA over IV NovoSeven + Glanzmann Thrombasthenia + SQ MarzAA can create & expand multiple + Acquired Hemophilia A episodic bleed & prophylaxis markets 27 catalystbiosciences.comNasdaq: CBIO MarzAA is only bypass agent for both SQ prophylaxis and SQ treatment of bleeds Attractive commercial profile targeting an existing $2.2B bypass agent market SQ MarzAA has a superior profile to IV IV NovoSeven ($1.2B 2019 sales) validates NovoSeven – over 100 clinicians & 175 rFVIIa in multiple rare bleeding disorders patients surveyed + Hemophilia A or B with inhibitors + Physicians & patients overwhelmingly + Severe Factor VII Deficiency prefer SQ MarzAA over IV NovoSeven + Glanzmann Thrombasthenia + SQ MarzAA can create & expand multiple + Acquired Hemophilia A episodic bleed & prophylaxis markets 27 catalystbiosciences.com
Nasdaq: CBIO MarzAA phase 2/3 SQ clinical trial MAA-201 if bleed occurs, if bleed occurs, if bleed occurs, then dose escalation then dose escalation then dose escalation MarzAA IV 18 µg/kg PK 24 hours 50 days 50 days 50 days 50 days MarzAA SQ 30 µg/kg PK 48 hours Daily Daily Daily Daily MarzAA SQ MarzAA SQ MarzAA SQ MarzAA SQ 30 µg/kg 60 µg/kg 90 µg/kg 120 µg/kg Endpoint achieved if ABR decreases from ≥12 to ≤6 + Patients with documented annual + Primary endpoint: reduction in annualized bleeding rate (ABR) >12 bleed rate at final dose level + Open label SQ study with individual dose + Secondary endpoints: safety and escalation if needed in Hemophilia A or B tolerability, inhibitor formation with inhibitors 28 catalystbiosciences.comNasdaq: CBIO MarzAA phase 2/3 SQ clinical trial MAA-201 if bleed occurs, if bleed occurs, if bleed occurs, then dose escalation then dose escalation then dose escalation MarzAA IV 18 µg/kg PK 24 hours 50 days 50 days 50 days 50 days MarzAA SQ 30 µg/kg PK 48 hours Daily Daily Daily Daily MarzAA SQ MarzAA SQ MarzAA SQ MarzAA SQ 30 µg/kg 60 µg/kg 90 µg/kg 120 µg/kg Endpoint achieved if ABR decreases from ≥12 to ≤6 + Patients with documented annual + Primary endpoint: reduction in annualized bleeding rate (ABR) >12 bleed rate at final dose level + Open label SQ study with individual dose + Secondary endpoints: safety and escalation if needed in Hemophilia A or B tolerability, inhibitor formation with inhibitors 28 catalystbiosciences.com
Nasdaq: CBIO SQ MarzAA reduces bleeding when dosed After the Injury Reduced bleeding After Injury Acute mouse injury model with dosing after the injury Untreated + Hemophilic mice bleed considerably Hemophilia A more than normal mice Bleeding + SQ treatment of MarzAA one min after traumatic bleeding has started 50% reduction significantly reduces blood loss and stops the bleed Normal + The effect is dose dependent Bleeding Response + Reduction in blood loss is comparable with IV NovoSeven MarzAA NovoSeven Subcutaneous IV + 1 min + 1 min 29 catalystbiosciences.comNasdaq: CBIO SQ MarzAA reduces bleeding when dosed After the Injury Reduced bleeding After Injury Acute mouse injury model with dosing after the injury Untreated + Hemophilic mice bleed considerably Hemophilia A more than normal mice Bleeding + SQ treatment of MarzAA one min after traumatic bleeding has started 50% reduction significantly reduces blood loss and stops the bleed Normal + The effect is dose dependent Bleeding Response + Reduction in blood loss is comparable with IV NovoSeven MarzAA NovoSeven Subcutaneous IV + 1 min + 1 min 29 catalystbiosciences.com
Nasdaq: CBIO MAA-102: PK MarzAA levels support SQ treatment of a bleed + Target of 24-120 ng/mL to treat a bleed is based on continuous infusion levels of NovoSeven for surgery + Target levels are rapidly achieved + 25% and 50% of C at 1 and 2 hours, max respectively 24 ng/mL + Dose-proportional increases in C and max AUC + Target levels can be maintained for 18 hours with a single SQ dose of 60 μg/kg + No ADA + Multiple dosing cohorts completed § 60 μg/kg 3-hourly; twice and thrice Neuman, 2020 30 catalystbiosciences.comNasdaq: CBIO MAA-102: PK MarzAA levels support SQ treatment of a bleed + Target of 24-120 ng/mL to treat a bleed is based on continuous infusion levels of NovoSeven for surgery + Target levels are rapidly achieved + 25% and 50% of C at 1 and 2 hours, max respectively 24 ng/mL + Dose-proportional increases in C and max AUC + Target levels can be maintained for 18 hours with a single SQ dose of 60 μg/kg + No ADA + Multiple dosing cohorts completed § 60 μg/kg 3-hourly; twice and thrice Neuman, 2020 30 catalystbiosciences.com