• Clinical data presented at 2019 American Society of Hematology (ASH) annual meeting suggested that CPI-0610 could offer meaningful benefits beyond standard of care in myelofibrosis
• In addition to previously expanded MANIFEST Cohorts 3 and 2A, now expanding Cohort 1A
• Planning update for MANIFEST and discussions with regulatory agencies about CPI-0610 around mid-year, additional MANIFEST update before yearend
• Conference call to discuss Constellation’s clinical programs and financial results scheduled for 5:00 PM EDT today

CAMBRIDGE, Mass., March 10, 2020 (GLOBE NEWSWIRE) -- Constellation Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company using its expertise in epigenetics to discover and develop novel therapeutics, today announced its fourth-quarter and full-year 2019 financial results.

“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 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) 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
• 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 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.

Results of Operations

		Year ended December 31,								Three months ended December 31,
(In thousands, except share and per-share amounts)			2019				2018				2019				2018
Operating expenses:
Research and development		$	66,459			$	48,769			$	18,586			$	16,626
General and administrative			19,596				12,475				5,471				4,006
Total operating expenses			86,055				61,244				24,057				20,632
Loss from operations			(86,055	)			(61,244	)			(24,057	)			(20,632	)
Other income (expense):
Interest income			2,644				1,547				730				688
Interest expense			(2,115	)			(228	)			(857	)			—
Total other income (expense), net			529				1,319				(127	)			688
Loss before income taxes			(85,526	)			(59,925	)			(24,184	)			(19,944	)
Income tax expense			24				—				24				—
Net loss		$	(85,550	)		$	(59,925	)		$	(24,208	)		$	(19,944	)
Other comprehensive loss
Unrealized loss on marketable securities			(6	)			—				(7	)			—
Total other comprehensive loss			(6	)			—				(7	)			—
Net loss attributable to common stockholders		$	(85,556	)		$	(59,925	)		$	(24,215	)		$	(19,944	)
Net loss per share attributable to common stockholders, basic and diluted		$	(3.04	)		$	(5.00	)		$	(0.69	)		$	(0.77	)
Weighted average common shares outstanding, basic and diluted			28,151,763				11,984,293				35,086,817				25,789,305

Consolidated Balance Sheets

		December 31,
(In $ thousands)			2019			2018
Cash and cash equivalents		$	334,332		$	114,592
Marketable securities			49,602			—
Total current assets			386,989			117,303
Total assets			399,130			118,938
Current liabilities			22,755			14,660
Long-term debt, net of current portion and discount			29,642			—
Total liabilities			61,546			14,780
Total stockholders’ equity		$	337,584		$	104,158

Conference Call

Constellation will host a conference call at 5:00 PM EDT on March 10, 2020, to discuss its clinical programs and financial results. The event will be webcast live and can be accessed on the Investor Relations section of Constellation’s website at http://ir.constellationpharma.com/events-and-presentations/events. To participate in the live question-and-answer session, please dial (877) 473-2077 (domestic) or (661) 378-9662 (international) and refer to conference ID 8669443.

About Constellation Pharmaceuticals

Constellation Pharmaceuticals is a clinical-stage biopharmaceutical company developing novel therapeutics that selectively modulate gene expression to address serious unmet medical needs in patients with cancer.  The Company has a deep understanding of how epigenetic and chromatin modifications in cancer cells and in the tumor and immune microenvironment play a fundamental role in driving disease progression and drug resistance. Constellation is driving development of the BET inhibitor CPI-0610 for the treatment of myelofibrosis as well as the EZH2 inhibitors CPI-1205 and CPI-0209 for the treatment of metastatic castration-resistant prostate cancer and other cancers. The Company is also applying its broad research and development capabilities to explore other novel targets that directly and indirectly impact gene expression to fuel a sustainable pipeline of innovative small-molecule product candidates. 

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include, but are not limited to, the Company’s plans, strategies and prospects for its business and statements regarding the development status of the Company’s product candidates, the timing of availability of clinical trial data and the Company’s ability to fund its operations until the second half of 2022. All statements, other than statements of historical facts, contained in this press release, including statements regarding the implications of preliminary or interim clinical data, the development status of the Company’s product candidates, and the Company’s plans for future data presentations, the Company’s strategy, future operations, future financial position, prospects, plans and objectives of management, are forward-looking statements. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with the Company’s ability to: obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in clinical trials; whether preliminary or interim data from a clinical trial will be predictive of the final results of the trial; replicate in later clinical trials positive results found in preclinical studies and early-stage clinical trials of CPI-0610, CPI-1205 and CPI-0209; advance the development of its product candidates under the timelines it anticipates, or at all, in current and future clinical trials; obtain, maintain, or protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. CPI-0610, CPI-1205 and CPI-0209 are investigational therapies and have not been approved by the FDA (or any other regulatory authority). For a discussion of other risks and uncertainties, any of which could cause the Company’s actual results to differ from those contained in the forward-looking statements, see the “Risk Factors” section, as well as discussions of potential risks, uncertainties and other important factors, in the Company’s most recent filings with the Securities and Exchange Commission, including the Company’s Annual Report on Form 10-K for the full year ended December 31, 2019. In addition, the forward-looking statements included in this press release represent the Company’s views as of the date hereof and should not be relied upon as representing the Company’s views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company’s views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

Contacts

Ronald Aldridge
Senior Director, Investor Relations
Constellation Pharmaceuticals
+1 617-714-0539
ron.aldridge@constellationpharma.com

Lauren Arnold
Media Relations
MacDougall Biomedical Communications
+1 781-235-3060
larnold@macbiocom.com