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Catalyst Biosciences
Exceptional Science. Essential Medicines.
Company Overview December 2015
Forward Looking Statements
includes forward-looking statements relating to the Catalyst Biosciences, Inc. (the Company ). Forward-looking statements include statements about the potential markets for the Company s product candidates, the potential advantages
of the Company s product candidates, product development plans and timelines, potential safety and efficacy of the Company s product candidates, potential sales of product candidates, if approved, the Company s intellectual property
and any statement of belief or assumptions underlying any of the foregoing. These statements reflect the current views of the Company s senior management with respect to future events. Forward-looking statements address matters that involve
risks and uncertainties, such as the timing of, costs associated with and outcomes of development, clinical and regulatory activities, risks associated with third-party arrangements, including the risk that Catalyst must negotiate with Pfizer about
obtaining manufacturing technology and know-how related to CB 813d, potential adverse effects arising from the testing or use of the Company s drug candidates, risks related to the Company s ability to develop, manufacture and
commercialize product candidates, to obtain regulatory approval of product candidates and to obtain marketplace acceptance of product candidates, to avoid infringing patents held by other parties and to secure and defend patents of the Company, and
to manage and obtain capital, including through any future financing or the conversion of outstanding convertible promissory notes. Further information regarding these and other risks is included in the Company s Form 10-Q for the quarter
ending September 30, 2015 filed with the Securities and Exchange Commission on November 5, 2015, under the heading Risk Factors.
Catalyst Biosciences : CBIO
Exceptional Science.
Essential Medicines.
~$3.3 billion/year in sales
, Catalyst Next Generation products have potential for multibillion/year in sales from growth in prophylaxis, new markets & new indications
Next generation protease therapeutics Billion dollar market opportunities
Anti-C3 IRI and Eye & anti-FB
anti-complement drug (Soliris Alexion) generates
~$2 billion/year in sales
, Catalyst Anti-Complement products have critical advantages in multiple new indications
Catalyst Biosciences Pipeline
Hemostasis Programs Research Preclinical Phase 1 Phase 2/3 Commercial Rights
Hemophilia A&B w Inhibitors, Surgical Bleeding
FIX: CB 2679d/ISU 304
Universal Pro-coagulant
Anti-Complement Programs
Renal Delayed Graft Function, IRI cardiovascular
Proteases A Unique Mechanism Of Action
The Catalyst Advantage
Proteases Are Safe & Effective Therapeutics
Currently Approved Proteases
(Vitreomacular adhesion) Botulinum Toxin (Blepharospasm, wrinkles) Factor VIIa (Bleeding disorders) Factor IX (Hemophilia B) t-PA (Myocardial infarction, stroke) Thrombin (Bleeding disorders) FEIBA (FXa Bleeding disorders) u-PA (Catheter
clearing, PAO) Zenpep (digestive aid)
Catalyst Biosciences
, Hereditary, chronic condition orphan disease with a growing population
, Two primary forms: hemophilia A (FVIII) and hemophilia B (FIX), combined ~400,000 patients WW*
, Patients have complete or severe deficiency of a clotting factor (receive FVIII or FIX) needed to form stable blood
clots or have antibodies (inhibitors) against their replacement factor (receive FVIIa or FEIBA)
bleeding in joints causes substantial pain, inflammation, joint damage, and loss of mobility
*Bolton-Maggs & Pasi, The Lancet 2003, v361 p1831
Market Characteristics
, Recombinant replacement factors, FVIII or FIX, or FVIIa/FEIBA are the dominant modes of treatment
, Drugs administered intravenously by patients or caregivers
, P1 trials are in hemophilia patients with PD efficacy endpoints
, Recent registration trials have been single P2/3s
, Small sales force requirement
, Products that enable
prophylactic treatment, prevent internal joint damage
, Faster-acting and more efficacious products for bleeds
, One product that does both
FVIIa Program: CB 813d
, Current FVIIa market
, Leading next-generation FVIIa in the clinic
, Significant improvements (6-9 fold) in potency, duration of effect and an improved therapeutic index vs NovoSeven in
multiple pre-clinical animal models
, Phase I in severe hemophilia patients
( inhibitors) demonstrated Proof-of-Mechanism (PoM) with excellent safety and tolerability
, Safe and well tolerated, no serious TEAEs
, Improved correction of PT and aPTT (vs NovoSeven) for up to 48 h
Activated blood coagulation Factor VII
CB 813d Clinical Trial
dose dependent correction of PT & aPTT at all doses
CB 813d Clinical Trial
, Single doses up to 30
g/kg were very well tolerated when administered to 25 hemophilia A and B patients in the non-bleeding state
, There were no instances of thrombosis or bleeding
, Evidence of pharmacologic activity was observed with dose-dependent changes of PT, aPTT, PF1+2, and TGA for up to 48
, The terminal half-life of CB 813d was approximately 3.5 hours and was similar across all dose groups
, The results for safety and pharmacologic activity support further clinical development of CB 813d for
treatment of individuals with hemophilia and inhibitors to FVIII or FIX
, Phase 2/3 trial anticipated to begin
Factor VIIa: CB 813d Advantages & Competition
Overall Avg. Doses Prophylactic
Control of to Control Dosing
vs Product Indication(s) Sales Bleeding Bleed Frequency Safety
Catalyst Commercial Products Inhibitors ~$1.5 billion* Good 2-3 Daily Good
CB 813d N/A Excellent 1 2-3x/week
(Company) Mechanism Stage Notes
/ Competitive Attributes
(Baxter) rhFVIIa Phase 3 Biosimilar; will not be differentiated from NovoSeven
(rEVO Biologics) rhFVIIa Phase 3
Biosimilar; only difference from NovoSeven is manufacturing cost
Products rFVIIa-albumin fusion for longer PK;
half-life is 6-9 fold greater than
(CSL Behring) Albumin fusion Phase 2/3 NovoSeven in rats but has significantly reduced activity; Phase 2/3
Pipeline initiated in August 2015
ACE910 Bi-specific antibody Unlikely to treat breakthrough bleeding; multiple ADA s (including
(Roche/Chugai) to FIXa and FX
neutralizing) observed in early trials
ALN-AT3 RNAi anti-thrombin Very unlikely to treat breakthrough
bleeding. Likely to have narrow
(Alnylam) inhibitor therapeutic window and may compromise safety for rescue options
*NovoNordisk 2014 Annual Report Gene therapy NOT applicable to FVIIa
Factor IX: CB 2679d (ISU 304)
~$1B (BeneFIX) Designed as best-in-class long-acting recombinant FIX product Significantly longer acting and more potent than BeneFIX , Alprolix (FIX-Fc) & FIX-GP
Development Alliance with ISU Abxis, a Korean biopharmaceutical company (Cerezyme & ReoPro) Preclinical
IND-enabling development initiated Phase 1 in 2016