Achillion Reports Fourth Quarter and Full Year 2018 Financial Results
- Phase 2 trial enrollment on schedule in PNH combo, interim data expected in Q2:2019 -
- Phase 2 trial enrollment on schedule in C3G, 24 sites recruiting -
- Next-generation factor D inhibitor ACH-5228 US IND anticipated in Q4:2019 -
- Reduced 2018 net cash spend to
- Well funded with cash and securities of
“We made significant progress advancing our portfolio of oral factor D inhibitors in the fourth quarter of 2018,” said
Mr. Truitt continued, “We took several important steps to strengthen our leadership team and expand the Company’s capabilities from a discovery engine to a clinical-stage organization in 2018. Additionally, we were pleased to announce that the Board of Directors recently elected
Key 2019 Planned Milestones
- ACH-4471, Complement Factor D Inhibitor for PNH and C3G
- The Company expects to report updated interim results for its Phase 2 open-label combination trial of ACH-4471 for PNH, with C5 inhibitor (eculizumab), in the second quarter of 2019 and is targeting an
FDA meeting in the fourth quarter of 2019. - The Company plans to present data from its Phase 2 clinical trials of ACH-4471 for C3G and immune complex membranoproliferative glomerulonephritis (IC-MPGN) to the
FDA in the fourth quarter of 2019.
- The Company expects to report updated interim results for its Phase 2 open-label combination trial of ACH-4471 for PNH, with C5 inhibitor (eculizumab), in the second quarter of 2019 and is targeting an
- ACH-5228, Next Generation Complement Factor D Inhibitor
- The Company recently initiated a Phase 1 MAD study of ACH-5228 and expects to complete the study and submit a US IND Application in the fourth quarter of 2019.
Fourth Quarter 2018 Financial Results
For the three months ended
Research and development expenses were
For the three months ended
Year-end 2018 Financial Results
For the year ended
For the year ended
General and administrative expenses were
Cash, cash equivalents, and marketable securities at
Financial Guidance
The company expects net cash usage for 2019 will be approximately
About ACH-4471, Complement Factor D Inhibitor for PNH and C3G
Achillion’s first-generation oral complement factor D inhibitor, ACH-4471, is being evaluated for safety and efficacy with Phase 2 clinical programs in both PNH and C3G and has demonstrated preliminary proof-of-concept in both indications. The PNH program consists of a Phase 2 clinical trial evaluating ACH-4471 in patients who are inadequately controlled or sub-optimally responding to eculizumab. Additionally, the Company continues to dose patients in its PNH monotherapy extension trial. The C3G program consists of two Phase 2 clinical trials which are currently recruiting, a six-month blinded, placebo-controlled study and a 12-month open-label study. More information is available at http://www.achillion.com/patients-and-clinicians/.
About ACH-5228 and ACH-5548, Next Generation Complement Factor D Inhibitors
ACH-5228 and ACH-5548 are the Company’s next-generation oral factor D inhibitors currently in Phase 1 clinical trials. These compounds demonstrated enhanced potency as well as improved pharmacokinetic properties that should allow for higher alternative pathway inhibition along with a reduced dosing frequency.
About
Cautionary Note Regarding Forward-Looking Statements
This press release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that are subject to risks, uncertainties and other important factors that could cause actual results to differ materially from those indicated by such forward-looking statements. Achillion may use words such as “expect,” “anticipate,” “project,” “target,” “intend,” “plan,” “aim,” “believe,” “seek,” “estimate,” “can,” “could” “focus,” “will,” “look forward,” “continue,” “goal,” “strategy,” “objective,” “may,” “potential,” and similar expressions to identify such forward-looking statements. These forward-looking statements also include statements about: the potential benefits of factor D inhibition as a treatment for complement-mediated diseases; the potential benefits of, and indications for, Achillion’s compounds that inhibit factor D, including ACH-4471, ACH-5228 and ACH-5548; Achillion’s belief that its portfolio of compounds could expand factor D portfolio opportunities, provide strategic optionality or create significant value; the status of enrollment in Achillion’s ongoing clinical trials; Achillion’s expectations regarding the advancement of, and timeline for reporting results from, clinical trials of its product candidates as well as its ability to advance additional compounds; Achillion’s expectations regarding the timing of regulatory interactions and filings; Achillion’s anticipated cash expenditures for 2019 and the sufficiency of its existing cash resources; and other statements concerning Achillion’s strategic goals, efforts, plans, and prospects. Among the important factors that could cause actual results to differ materially from those indicated by such forward-looking statements are risks relating to, among other things, Achillion’s ability to: demonstrate in any current and future clinical trials the requisite safety, efficacy and combinability of its product candidates; advance the preclinical and clinical development of its complement factor D inhibitors under the timelines it projects in current and future preclinical studies and clinical trials; enroll patients in its clinical trials on its projected timelines; obtain and maintain patent protection for its product candidates and the freedom to operate under third party intellectual property; obtain and maintain necessary regulatory approvals, and the granting of orphan designation does not alter the standard regulatory requirements and process for obtaining such approval; establish commercial manufacturing arrangements; identify, enter into and maintain collaboration and other commercial agreements with third-parties; compete successfully in the markets in which it seeks to develop and commercialize its product candidates and future products; manage expenses; manage litigation; raise the substantial additional capital needed to achieve its business objectives; and successfully execute on its business strategies. These and other risks are described in the reports filed by Achillion with the
In addition, any forward-looking statement in this press release represents Achillion's views only as of the date of this press release and should not be relied upon as representing its views as of any subsequent date. Achillion disclaims any duty to update any forward-looking statement, except as required by applicable law.
Investors:
Senior VP, Chief Financial Officer
Tel. 215-709-3032
bdidonato@achillion.com
Media:
Senior VP, Corporate Communications
Tel. 215-709-3055
sheinzinger@achillion.com
ACHILLION PHARMACEUTICALS INC. (ACHN) | |||||||||||||||
Statements of Operations | |||||||||||||||
(in thousands, except per share amounts) | |||||||||||||||
Three Months Ended | Year Ended | ||||||||||||||
December 31, | December 31, | ||||||||||||||
2018 | 2017 | 2018 | 2017 | ||||||||||||
Revenue | $ | - | $ | - | $ | - | $ | - | |||||||
Operating expenses: | |||||||||||||||
Research and development | 12,202 | 15,411 | 50,118 | 63,607 | |||||||||||
General and administrative | 5,972 | 8,944 | 23,896 | 25,969 | |||||||||||
Restructuring charges | - | 1,900 | - | ||||||||||||
Total operating expenses | 18,174 | 24,355 | 75,914 | 89,576 | |||||||||||
Loss from operations | (18,174 | ) | (24,355 | ) | (75,914 | ) | (89,576 | ) | |||||||
Other income (expense): | |||||||||||||||
Interest income | 1,585 | 1,165 | 5,678 | 4,390 | |||||||||||
Interest expense | (12 | ) | (13 | ) | (36 | ) | (50 | ) | |||||||
Net loss | $ | (16,601 | ) | $ | (23,203 | ) | $ | (70,272 | ) | $ | (85,236 | ) | |||
Net loss per share - basic and diluted | $ | (0.12 | ) | $ | (0.17 | ) | $ | (0.51 | ) | $ | (0.62 | ) | |||
Wtd avg shares outstanding - basic and diluted | 138,638 | 137,870 | 138,418 | 137,180 | |||||||||||
Balance Sheets | |||||||||||||||
(Unaudited, in thousands) | |||||||||||||||
December 31, | December 31, | ||||||||||||||
2018 | 2017 | ||||||||||||||
Cash, cash equivalents, and marketable securities | $ | 270,977 | $ | 330,585 | |||||||||||
Working capital | 263,551 | 291,054 | |||||||||||||
Total assets | 277,858 | 337,613 | |||||||||||||
Long-term liabilities | 17 | 214 | |||||||||||||
Total liabilities | 11,846 | 13,098 | |||||||||||||
Total stockholders' equity | 266,012 | 324,515 | |||||||||||||
Source: Achillion Pharmaceuticals, Inc.