•

Achieved key milestones with CB 2679d/ISU304, the Company’s next-generation coagulation Factor IX, including:

o

Announced top-line multi-dose results from the subcutaneous Factor IX CB 2679d Phase 1/2 proof-of-concept clinical trial in individuals with severe hemophilia B; and

o

Signed a commercial-scale manufacturing agreement with AGC Biologics for subcutaneous Factor IX CB 2679d.

•

Advanced the development of marzeptacog alfa (activated), the Company’s next-generation Factor VIIa, including the following accomplishments:

o

Initiated the Factor VIIa marzeptacog alfa (activated) Phase 2 part of a Phase 2/3 subcutaneous efficacy clinical trial in individuals with hemophilia A or B with an inhibitor.

•

Successfully raised ~$125 million through two underwritten public equity offerings.  During December 2017 raised $10.5 million and during February raised $115 million, including the full exercise of the underwriters’ over-allotment option to purchase additional shares.

•

Announce interim data from an open-label Phase 2 part of the Phase 2/3 program of marzeptacog alfa (activated), subcutaneous efficacy trial in individuals with hemophilia A or B with inhibitors to evaluate the ability of MarzAA to minimize, spontaneous bleeding episodes in July 2018.

•

Announce additional data from the Factor IX CB2679d Phase 1/2 proof-of-concept clinical trial in individuals with severe hemophilia B in the second quarter of 2018. Initiate a Phase 2b trial of CB 2679d in individuals with severe hemophilia B in the third quarter of 2018.

•

Cash, cash equivalents and short-term investments, as of Mar. 1, 2018 were in excess of $135 million, due primarily to the $115 million in gross financing in February 2018 and the proceeds from the exercise of warrants. Cash, cash equivalents and short-term investments, as of Dec. 31, 2017 and 2016 were $32.4 and $17.1 million, respectively.  

•

In the fourth quarter the Company completed a follow-on financing of $10.5 million in gross proceeds.

•

Research and development expense for the three months ended Dec. 31, 2017 was $3.6 million, compared with $3.1 million for the prior year period. The increase was due primarily to manufacturing expenses for marzeptacog alfa (activated). Research and development expense for the year ended Dec. 31, 2017 was $12.8 million, compared with $11.6 million for the prior year respectively, an increase of $1.3 million, due primarily to manufacturing expenses for marzeptacog alfa (activated).

•

General and administrative expense for the three months ended Dec. 31, 2017 was $2.6 million compared with $2.2 million for the prior year period.  The increase was due primarily to increased headcount. General and administrative expenses for the years ended Dec. 31, 2017 and 2016 were $10.0 million and $9.3 million, respectively, an increase of $0.7 million, due primarily to increased headcount and financing expenses.

•

Interest and other income for the three months ended Dec. 31, 2017 was $0.1 million, compared with $1.5 million for the prior year period.  The decrease was due primarily to the 2016 gain related to the sale of noncore NNR assets. Interest and other income for the years ended Dec. 31, 2017 and 2016, were $0.3 million and $3.5 million, respectively, a decrease of $3.2 million.

•

Net loss attributable to common stockholders for the year ended Dec. 31, 2017 was $25.5 million, or ($7.45) per basic and diluted share, compared with $16.9 million, or ($21.75) per basic and diluted share, for the prior year.  

•

On February 19, 2018, the last $5 million of redeemable convertible notes matured and were repaid in full with $5 million from the restricted cash indenture. The Company has no outstanding Notes or debt.

•

As of February 27, 2018, the Company had 10,968,644 shares of common stock outstanding.

December 31, 2017

December 31, 2016

Assets

Current assets:

Cash and cash equivalents

$

14,472

$

10,264

Short-term investments

17,971

6,800

Restricted cash

5,333

19,468

Prepaid and other current assets

1,309

958

Accounts receivable

24

31

Total current assets

39,109

37,521

Restricted cash, noncurrent

—

125

Deposits, noncurrent

128

—

Property and equipment, net

276

444

Total assets

$

39,513

$

38,090

Liabilities and stockholders’ equity

Current liabilities:

Accounts payable

$

747

$

837

Accrued compensation

1,366

596

Other accrued liabilities

1,322

805

Deferred revenue, current portion

212

283

Deferred rent, current portion

7

41

Redeemable convertible notes

5,085

19,403

Total current liabilities

8,739

21,965

Deferred revenue, noncurrent portion

—

47

Deferred rent, noncurrent portion

—

7

Total liabilities

8,739

22,019

Stockholders’ equity:

Preferred stock, $0.001 par value, 5,000,000 shares authorized; 3,680 and 0 shares

   issued and outstanding at December 31, 2017 and 2016, respectively

—

—

Common stock, $0.001 par value, 100,000,000 shares authorized; 6,081,230 and

   801,756 shares issued and outstanding at December 31, 2017 and 2016,

   respectively

6

1

Additional paid-in capital

204,262

164,053

Accumulated other comprehensive income (loss)

—

(1

)

Accumulated deficit

(173,494

)

(147,982

)

Total stockholders’ equity

30,774

16,071

Total liabilities and stockholders’ equity

$

39,513

$

38,090

Year Ended December 31,

2017

2016

Contract revenue

$

1,018

$

399

Operating expenses:

Research and development

12,847

11,555

General and administrative

9,993

9,262

Total operating expenses

22,840

20,817

Loss from operations

(21,822

)

(20,418

)

Interest and other income, net

261

3,473

Net loss

(21,561

)

(16,945

)

Deemed dividend for convertible preferred stock beneficial conversion feature

(3,951

)

—

Net loss attributable to common stockholders

$

(25,512

)

$

(16,945

)

Net loss per share attributable to common stockholders, basic and

   diluted

$

(7.45

)

$

(21.75

)

Shares used to compute net loss per share attributable to common

   stockholders, basic and diluted

3,423,901

779,166