On March 10, 2020 Constellation Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company using its expertise in epigenetics to discover and develop novel therapeutics, reported its fourth-quarter and full-year 2019 financial results (Press release, Constellation Pharmaceuticals, MAR 10, 2020, View Source [SID1234555363]).

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"The fourth quarter of 2019 was transformational for Constellation," said Jigar Raythatha, president and chief executive officer of Constellation Pharmaceuticals. "The Company is now advancing toward its goal of becoming a fully integrated oncology company, with broad-based capabilities from target identification and compound discovery to translational science, clinical development, and commercialization. We presented encouraging preliminary data at the American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting in December showing signals of activity for CPI-0610 across a broad range of disease parameters in myelofibrosis. These data suggest possible disease-modifying effects, such as bone marrow fibrosis improvements and hemoglobin increases, in addition to spleen volume reductions and symptom score improvements. We also raised capital in December, extending our cash runway into the second half of 2022.

"We are targeting CPI-0610 in combination with ruxolitinib to become the standard of care in first-line treatment of myelofibrosis," Mr. Raythatha continued. "In second-line patients who have previously been treated with ruxolitinib, CPI-0610 could potentially provide meaningful benefits as a monotherapy or as an add-on to ruxolitinib.

"We look forward to discussions on CPI-0610 with regulatory agencies in the coming year. These discussions will help refine our plans for a potential registration path for CPI-0610, including our plan to initiate a global randomized Phase 3 clinical trial of CPI-0610 + ruxolitinib in JAK-inhibitor-naïve MF patients in the third quarter of 2020.

"We are also working to create value for patients in other ways," Mr. Raythatha concluded. "We believe that CPI-0610 may benefit patients with other hematologic and oncology diseases. In addition, our EZH2-inhibitor franchise of CPI-1205 and CPI-0209 provides potential opportunities to treat a wide range of oncology patients. Our robust discovery platform is expected to deliver a steady stream of clinical candidates in coming years."

Program Updates

CPI-0610

Submitted abstracts for the European Hematology Association (EHA) (Free EHA Whitepaper) annual meeting in June 2020
Expect to present 24-week data on 25-30 first-line (1L) and 70-80 second-line (2L) patients at EHA (Free EHA Whitepaper)
Continuing enrollment in previously expanded Arm 3 (1L combo patients) and Cohort 2A (2L combo transfusion-dependent patients)
Expanding Cohort 1A (2L monotherapy transfusion-dependent patients) from 16 to up to 60 patients based on conversions of transfusion-dependent patients to transfusion independence
Received orphan drug designation for CPI-0610 from the FDA and EMA
CPI-1205

Completed Phase 2 enrollment in our ProSTAR trial
Planning for data cut and analysis in mid-2020, with a clear signal required to move program into Phase 3
CPI-0209

Ongoing Phase 1 dose escalation phase of Phase 1/2 clinical trial studying CPI-0209 monotherapy in advanced, relapsed solid tumor patients
Aim to determine recommended Phase 2 dose (RP2D) for monotherapy in second half of 2020
After determining RP2D, intend to initiate Phase 2 expansion arms for monotherapy in selected solid tumor indications as well as dose escalation studies of combinations with other therapies
Employing biomarker strategy that includes assessment of ARID1A mutations
Milestones

The Company anticipates achieving the following milestones during 2020:

CPI-0610 – Provide MANIFEST program update at EHA (Free EHA Whitepaper) in June

CPI-0610 – Initiate Phase 3 clinical trial in the third quarter

CPI-0610 – Provide additional MANIFEST program update by yearend

CPI-1205 – Provide ProSTAR program update mid-year

CPI-0209 – Provide program update, including recommended Phase 2 dose, by end of year

Fourth Quarter 2019 Financial Results

Cash, cash equivalents, and marketable securities as of December 31, 2019, were $383.9 million, an increase of 235.0% compared to December 31, 2018, primarily due to proceeds from the private placement in October 2019 and the public offering in December 2019, offset by operating expenses.
Research and development (R&D) expenses increased 11.8% year over year to $18.6 million in the fourth quarter of 2019 mainly due to increased clinical trial expenses.
General and administrative (G&A) expenses grew 36.6% year over year to $5.5 million in the fourth quarter of 2019, primarily due to building out the organization of the company.
The net loss attributed to common shareholders increased 21.4% year over year to $24.2 million for the fourth quarter of 2019, mainly due to increased R&D and G&A expenses. The net loss per share attributable to common shareholders decreased 10.4% to $0.69 per share due to an increase in weighted average shares outstanding as a result of the private placement in October 2019 and the public offering in December 2019, offset in part by the increased net loss.
Full Year 2019 Financial Results

Research and development (R&D) expenses increased 36.3% year over year to $66.5 million in full-year 2019, mainly due to increased clinical trial expenses.
General and administrative (G&A) expenses grew 57.1% year over year to $19.6 million in full-year 2019, primarily due to building out the organization of the company.
The net loss attributed to common shareholders increased 42.8% year over year to $85.6 million for full-year 2019, mainly due to increased R&D and G&A expenses. The net loss per share attributable to common shareholders decreased 39.2% to $3.04 per share largely due to an increase in weighted average shares outstanding as a result of the initial public offering and related conversion of preferred stock to common stock in July 2018 and the additional shares issued in 2019.
Financial Guidance

Constellation expects that its current cash, cash equivalents, and marketable securities will enable it to fund operations into the second half of 2022.