Marzeptacog alfa (activated) – MarzAA, a SQ administered next-generation engineered coagulation Factor VIIa (FVIIa): The Company presented two posters at the International Society for Thrombosis and Haemostasis (ISTH) Virtual Congress in July. The MAA-102 PK results and population PK simulations confirm that Catalyst has optimized dosing for its Phase 3 MarzAA registration trial. The open-label trial will evaluate the efficacy of SQ MarzAA compared with standard of care to treat episodic bleeding in individuals with hemophilia A or B with inhibitors.

Dalcinonacog alfa – DalcA, a subcutaneously administered next-generation engineered coagulation Factor IX (FIX) being developed for Hemophilia B: Presented final efficacy and safety data from its Phase 2b trial of DalcA at the World Federation of Hemophilia in June. Data from the trial showed that 28 days of daily SQ dosing of DalcA achieved protective target FIX levels of >12% in all participants, with FIX levels of up to 27% and a half-life of 2.5 to 5.1 days with no bleeds, demonstrating effective prophylaxis and the potential for lower or less frequent dosing.

Factor IX gene therapy construct – CB 2679-GT, The Company’s proprietary FIX gene therapy construct CB 2679d-GT is being developed for the treatment of hemophilia B and has demonstrated superiority compared with the Padua variant in preclinical models. Catalyst presented data from preclinical studies of its hemophilia B gene therapy CB 2679d-GT at the World Federation of Hemophilia Virtual Summit. The preclinical data from the Company’s constructs demonstrated a strong dose response and improved reduction in bleeding compared with the Padua variant. Catalyst believes that the enhanced FIX activity and reduced viral dose may offer advantages over current AAV-based gene therapies in clinical development.

Corporate: Appointed Clinton J. Musil as a chief financial officer and Charles Democko as a senior vice president, regulatory affairs. Completed an underwritten public offering of 4,615,384 shares of its common stock, offered at a price of $6.50 per share to the public. The gross proceeds to Catalyst from this offering were approximately $30.0 million, before deducting underwriting discounts and commissions and other estimated offering expenses payable by Catalyst.

MarzAA: Enroll the first patient in a Phase 3 open-label trial in late 2020, evaluating the efficacy of SQ MarzAA to treat episodic bleeding in individuals with hemophilia A or B with inhibitors. Initiate a Phase 1/2 trial in FVII Deficiency, Glanzmann Thrombastenia, and patients using Hemlibra, in late 2020. Timelines for both trials are subject to the readiness of trial sites and potential effects of COVID-19 on clinical trial execution.

CB 2679d-GT (FIX Gene Therapy): Announce a development candidate in late 2020.

Systemic complement inhibitor: Announce a development candidate in late 2020.

Cash, cash equivalents and short-term investments, as of June 30, were $117.4 million.

Research and development expenses were $12.9 million and $11.1 million during the three months ended June 30, 2020 and 2019, respectively, an increase of $1.8 million, or 16%. The increase was due primarily to an increase of $2.7 million in preclinical spending, an increase of $0.6 million in personnel and facilities costs offset by a decrease of $1.6 million in clinical and manufacturing costs.

General and administrative expenses were $4.4 million and $3.3 million during the three months ended June 30, 2020 and 2019, respectively, an increase of $1.1 million. The increase was primarily due to an increase in professional services.

Interest and other income, net was $0.1 million and $0.6 million during the three months ended June 30, 2020 and 2019, respectively, a decrease of $0.5 million. The decrease was primarily due a decrease in interest income on investments.

Net loss attributable to common stockholders for the three-months ended June 30, 2020 was $17.2 million, or ($0.96) per basic and diluted share, compared with $13.8 million, or ($1.15) per basic and diluted share, for the prior year period.

As of June 30, 2020, the Company had 22,050,745 shares of common stock outstanding.

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Preferred stock, $0.001 par value, 5,000,000 shares authorized; zero shares issued and outstanding

Common stock, $0.001 par value, 100,000,000 shares authorized; 22,050,745 and 12,040,835 shares issued and outstanding at June 30, 2020 and December 31, 2019, respectively

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