Market Overview

Acceleron Reports Second Quarter 2019 Operating and Financial Results

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- BLA and MAA filings of luspatercept for beta-thalassemia- and myelodysplastic syndromes-associated anemia accepted by U.S. Food and Drug Administration and European Medicines Agency, respectively -

- Acceleron receives $25 million milestone payment from Celgene for luspatercept's BLA acceptance and MAA validation -

- The ACE-083 Phase 2 trial in Charcot-Marie-Tooth disease (CMT) has completed enrollment with topline results anticipated in Q1 2020, and results from the facioscapulohumeral muscular dystrophy (FSHD) trial are expected in 2H19 -

- PULSAR Phase 2 trial of sotatercept in pulmonary arterial hypertension (PAH) is fully enrolled, with topline results expected in Q1 2020 -

Acceleron Pharma Inc. (NASDAQ:XLRN), a leading biopharmaceutical company in the discovery and development of TGF-beta superfamily therapeutics to treat serious and rare diseases, today provided a corporate update and reported financial results for the second quarter ended June 30, 2019.

"The significant progress across our entire pipeline over the last few years has us well positioned to execute during one of the most exciting times in Acceleron's 16-year history. With the luspatercept U.S. and European regulatory approval filings under review, we are now one step closer to the first-ever potential approval of an Acceleron-discovered medicine," said Habib Dable, President and Chief Executive Officer of Acceleron. "Alongside our global collaboration partner, Celgene, we are focused on preparing for luspatercept's potential commercial launch, and we continue to execute on our ongoing clinical trials in first-line lower-risk MDS-, non-transfusion-dependent beta-thalassemia- and myelofibrosis-associated anemia."

Added Mr. Dable: "In parallel, we have advanced our two Acceleron-led clinical programs in neuromuscular and pulmonary disease as we work to establish key proof-of-concept results in three placebo-controlled Phase 2 trials over the next nine months. Following robust enrollment in our PULSAR Phase 2 trial in patients with PAH, we now expect topline results in the first quarter of 2020. For ACE-083, we anticipate topline results in patients with FSHD and CMT in the second half of this year and early 2020, respectively."

Development Program Highlights

Hematology

Luspatercept: Myelodysplastic Syndromes (MDS), Beta-Thalassemia, and Myelofibrosis (MF)

Luspatercept is an investigational first-in-class erythroid maturation agent designed to address a late-stage erythroid maturation defect that results in chronic anemia and the need for regular red blood cell transfusions in adults with serious hematologic diseases. Luspatercept is part of the global collaboration between Acceleron and Celgene.

  • The U.S. Food and Drug Administration (FDA) accepted the Biologics License Application (BLA) for luspatercept for the treatment of adult patients with very low- to intermediate-risk MDS-associated anemia who have ring sideroblasts and require red blood cell (RBC) transfusions, and for the treatment of adult patients with beta-thalassemia-associated anemia who require RBC transfusions.
  • The FDA granted priority review for the beta-thalassemia indication and set a target action date of December 4, 2019, and set a target action date of April 4, 2020, for the MDS indication.
  • The Marketing Authorization Application (MAA) for luspatercept in adult patients with MDS- or beta-thalassemia-associated anemia has been validated by the European Medicines Agency (EMA). The EMA decision on the MAA is expected in the second half of 2020.
  • Results from the Phase 2 trial of luspatercept in patients with MF are expected later this year.
  • Enrollment is ongoing in the COMMANDS Phase 3 trial in patients with treatment-naïve lower-risk MDS and the BEYOND Phase 2 trial in patients with non-transfusion-dependent beta-thalassemia. Topline results from the BEYOND trial are expected by year-end 2020.

Neuromuscular Disease

ACE-083: Facioscapulohumeral Muscular Dystrophy (FSHD) and Charcot-Marie-Tooth Disease (CMT)

ACE-083 is an investigational locally-acting therapeutic designed to have a concentrated effect on muscle mass and strength in target muscles for diseases that cause focal muscle weakness. ACE-083 utilizes the "Myostatin+" approach to inhibit multiple TGF-beta superfamily ligands involved in muscle formation.

  • Topline results from Part 2 of the Phase 2 trial in patients with FSHD are expected in the second half of 2019.
  • Enrollment was recently completed in Part 2 of the Phase 2 trial in patients with CMT, with topline results expected in the first quarter of 2020.
  • The Phase 2 extension trial is open for patients who participated in the FSHD and CMT Phase 2 trials of ACE-083.

Pulmonary Disease

Sotatercept: Pulmonary Arterial Hypertension (PAH)

Sotatercept is an investigational agent designed to be a selective ligand trap for members of the TGF-beta superfamily to rebalance BMPR2 signaling, which is a key molecular driver of PAH. In preclinical studies of PAH, sotatercept reversed pulmonary vessel muscularization and improved indicators of right heart failure.

  • The PULSAR Phase 2 trial in patients with PAH has completed enrollment, with topline results expected in Q1 2020.
  • Enrollment is ongoing in the exploratory SPECTRA trial in patients with PAH, with preliminary results expected in 2020.

Financial Results

  • Cash Position – Cash, cash equivalents and investments as of June 30, 2019 were $500.9 million. This cash balance includes the receipt of a $25.0 million gross milestone payment for the acceptance of the luspatercept BLA and MAA filings. As of December 31, 2018, the Company had cash, cash equivalents and investments of $291.3 million. Based on the Company's current operating plan and projections, it believes that current cash, cash equivalents and investments will be sufficient to fund projected operating requirements until such time as it expects to receive significant royalty revenue from luspatercept sales.
  • Revenue – Second quarter revenue was $27.7 million. The revenue is all from the Company's collaboration partnership with Celgene, and is largely related to the milestone payment received, as well as expenses incurred by the Company in support of luspatercept.
  • Costs and Expenses – Total costs and expenses for the second quarter were $48.8 million. This includes R&D expenses of $34.8 million and G&A expenses of $14.0 million.
  • Net Loss – The Company's net loss for the second quarter ended June 30, 2019 was $17.9 million.

Conference Call and Webcast

The Company will host a webcast and conference call to discuss its second quarter 2019 financial results and provide an update on recent corporate activities on August 5, 2019, at 5:00 p.m. EDT.

The webcast will be accessible under "Events & Presentations" in the Investors/Media page of the Company's website at www.acceleronpharma.com. Individuals can participate in the conference call by dialing 877-312-5848 (domestic) or 253-237-1155 (international) and referring to the "Acceleron Second Quarter 2019 Earnings Call."

The archived webcast will be available for replay on the Acceleron website approximately two hours after the event.

About Acceleron

Acceleron is a clinical-stage biopharmaceutical company dedicated to the discovery, development, and commercialization of therapeutics to treat serious and rare diseases. The Company's leadership in the understanding of TGF-beta superfamily biology and protein engineering generates innovative compounds that engage the body's ability to regulate cellular growth and repair.

Acceleron focuses its research and development efforts in hematologic, neuromuscular, and pulmonary diseases. In hematology, the Company and its global collaboration partner, Celgene, are developing luspatercept for the treatment of chronic anemia in myelodysplastic syndromes, beta-thalassemia, and myelofibrosis. Acceleron is also advancing its neuromuscular program with ACE-083, a locally-acting Myostatin+ agent in Phase 2 development in facioscapulohumeral muscular dystrophy and Charcot-Marie-Tooth disease and is conducting a Phase 2 pulmonary program with sotatercept in pulmonary arterial hypertension.

For more information, please visit www.acceleronpharma.com. Follow Acceleron on Social Media: @AcceleronPharma and LinkedIn.

ACCELERON PHARMA INC.

CONDENSED CONSOLIDATED BALANCE SHEET

(Amounts in thousands)

(unaudited)

 

June 30, 2019

 

December 31, 2018

Cash and cash equivalents

$

163,609

 

 

$

144,052

 

Short and long-term investments

337,307

 

 

147,260

 

Operating lease assets

26,549

 

 

 

Other assets

23,403

 

 

23,509

 

Total assets

$

550,868

 

 

$

314,821

 

 

 

 

 

Short-term and long-term operating lease liabilities

$

29,153

 

 

$

 

Warrants to purchase common stock

1,389

 

 

1,491

 

Other liabilities

20,429

 

 

21,292

 

Total liabilities

50,971

 

 

22,784

 

Total stockholders' equity

499,897

 

 

292,037

 

Total liabilities and stockholders' equity

$

550,868

 

 

$

314,821

 

ACCELERON PHARMA INC.

CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS

(Amounts in thousands except per share data)

(unaudited)

 

Three Months Ended June 30,

 

Six Months Ended June 30,

 

2019

 

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