On August 2, 2018 Emergent BioSolutions Inc. (NYSE: EBS) reported financial results for the quarter and six months ended June 30, 2018 (Press release, Emergent BioSolutions, AUG 2, 2018, View Source [SID1234528439]).

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Q2 2018 AND RECENT BUSINESS ACCOMPLISHMENTS

Completed Mutual Recognition Procedure for market authorization of BioThrax (Anthrax Vaccine Adsorbed) in five Concerned Member States within the European Union – Italy, the Netherlands, Poland, the U.K. and France; to date, BioThrax has received market authorization in four of the five countries.

Initiated an investment of up to $50 million over the next three years in the Camden fill/finish facility located in Baltimore, an expansion project that will significantly enhance the capabilities of this key site within the Company’s CDMO Business Unit.

Announced Framework Partnering Agreement under which the Company will provide technical and manufacturing support for the development and manufacture of a vaccine against Nipah virus in collaboration with Profectus BioSciences, Inc. and CEPI (Coalition for Epidemic Preparedness Innovations); under a separate agreement with Profectus, Emergent will retain the exclusive option to license and assume control of development activities for the Nipah virus vaccine from Profectus.

Initiated a Phase 1 clinical study of ZIKV-IG, the Company’s anti-Zika virus immune globulin being developed as a therapeutic intervention against Zika virus disease; the candidate was granted Fast Track designation by the U.S. Food and Drug Administration in December 2017.

2018 FINANCIAL PERFORMANCE

(I) Quarter Ended June 30, 2018 (Unaudited)

Revenues

Total Revenues
For Q2 2018, total revenues were $220.2 million, an increase of 118% over 2017. Total revenues reflect a significant increase in product sales.

Product Sales
For Q2 2018, product sales were $180.1 million, an increase of 183% as compared to 2017. The increase is principally attributable to sales of BioThrax and ACAM2000, (Smallpox (Vaccinia) Vaccine Live) previously expected in the first quarter as well as continued sales of both products in the second quarter.

Contract Manufacturing
For Q2 2018, revenue from the Company’s contract manufacturing operations was $23.6 million, an increase of 46% as compared to 2017. The increase primarily reflects manufacturing services at the Company’s Canton site.

Contracts and Grants
For Q2 2018, revenue from the Company’s development-based contracts and grants was $16.5 million, a decrease of 21% as compared to 2017. The decrease primarily reflects a reduction in R&D activities related to certain ongoing funded development programs.

Operating Expenses

Cost of Product Sales and Contract Manufacturing
For Q2 2018, cost of product sales and contract manufacturing was $89.2 million, an increase of 158% as compared to 2017. The increase was primarily attributable to the increase in product sales and contract manufacturing activities at the Company’s Bayview and Canton facilities.

Research and Development (Gross and Net)
For Q2 2018, gross R&D expenses were $24.7 million, a decrease of 4% as compared to 2017. The decrease primarily reflects lower costs associated with contract development services.

For Q2 2018, net R&D expense (calculated as gross research and development expenses minus contracts and grants revenue) was $8.2 million, an increase of $3.4 million as compared to 2017, reflecting increased investment in development-stage programs not currently funded in whole or in part by third-party partners. These include costs associated with the Raxibacumab (Anthrax Monoclonal Antibody) technology transfer and the SIAN device, an intranasal antidote spray device for the treatment of known or suspected acute cyanide poisoning.

Selling, General and Administrative
For Q2 2018, selling, general and administrative expenses were $39.5 million, an increase of 24% as compared to 2017, attributable primarily to increased professional services and compensation-related costs.

Income Taxes
For Q2 2018, the provision for income tax expense in the amount of $15.7 million includes a discrete benefit of $0.9 million primarily related to stock compensation activity resulting in an effective tax rate of 24%. Excluding the discrete benefit, the Q2 2018 effective tax rate was 25%.

Net Income & Adjusted Net Income
For Q2 2018, the Company recorded net income of $50.1 million, or $0.98 per diluted share, versus net income of $4.6 million, or $0.11 per diluted share, in 2017. (1).

For Q2 2018, the Company recorded adjusted net income of $54.7 million, or $1.07 per diluted share, versus adjusted net income of $6.6 million, or $0.13 per diluted share, in 2017. (1) (2)

(I) Six Months Ended June 30, 2018 (Unaudited)

Revenues

Total Revenues
For the six months of 2018, total revenues were $338.0 million, an increase of 55% over 2017. Total revenues reflect a significant increase in product sales.

Product Sales
For the six months of 2018, product sales were $255.8 million, an increase of 76% as compared to 2017. The increase is principally attributable to sales of ACAM2000 and Raxibacumab, both of which were acquired in Q4 2017.

Contract Manufacturing
For the six months of 2018, revenue from the Company’s contract manufacturing operations was $49.8 million, an increase of 47% as compared to 2017. The increase primarily reflects the completion of a milestone related to the expansion of certain contract manufacturing capabilities at the Company’s Lansing site and manufacturing services at the Company’s Canton site.

Contracts and Grants
For the six months of 2018, revenue from the Company’s development-based contracts and grants was $32.4 million, a decrease of 15% as compared to 2017. The decrease primarily reflects a reduction in revenue associated with the successful completion of multiple U.S. government development contracts, as well as reduced R&D activities related to certain ongoing funded development programs.

Operating Expenses

Cost of Product Sales and Contract Manufacturing
For the six months of 2018, cost of product sales and contract manufacturing was $147.2 million, an increase of 82% as compared to 2017. The increase was primarily attributable to the increase in product sales and contract manufacturing activities at the Company’s Bayview and Canton facilities.

Research and Development (Gross and Net)
For the six months of 2018, gross R&D expenses were $53.8 million, an increase of 16% as compared to 2017. The increase primarily reflects costs associated with contract development services, including the cost associated with the technology transfer of the Raxibacumab manufacturing process to the Company’s Bayview manufacturing site in Baltimore.

For the six months of 2018, net R&D expense was $21.4 million, an increase of $13.5 million as compared to 2017, reflecting increased investment in countermeasure development programs not currently funded in whole or in part by third-party partners, notably costs associated with the Raxibacumab technology transfer and the SIAN device, an intranasal antidote spray device for the treatment of known or suspected acute cyanide poisoning.

Selling, General and Administrative
For the six months of 2018, selling, general and administrative expenses were $79.7 million, an increase of 19% as compared to 2017, attributable primarily to increased professional services and compensation-related costs.

Income Taxes
For the six months of 2018, the provision for income tax expense in the amount of $11.2 million includes a discrete benefit of $3.2 million primarily related to stock compensation activity resulting in an effective tax rate of 20%. Excluding the discrete benefit, the six months of 2018 effective tax rate was 25%.

Net Income & Adjusted Net Income
For the six months of 2018, the Company recorded net income of $45.2 million, or $0.89 per diluted share, versus net income of $15.1 million, or $0.35 per diluted share, in 2017. (1)

For the six months of 2018, the Company recorded adjusted net income of $53.1 million, or $1.04 per diluted share, versus adjusted net income of $20.8 million, or $0.42 per diluted share, in 2017. (1) (2)

2018 FINANCIAL FORECAST & OPERATIONAL GOALS
The Company is reaffirming its full year 2018 financial performance forecast:
· Total Revenue
$715 million to $755 million
· Pre-Tax Income
$120 million to $140 million
· Net Income (3)
$95 million to $110 million
· Adjusted Net Income (2) (3)
$110 million to $125 million
· EBITDA (2) (3)
$175 million to $190 million

The Company is also reaffirming its full year 2018 operational goals:

Advance NuThrax development to enable Emergency Use Authorization filing with the FDA in 2018

Complete ACAM2000 deliveries; establish a multi-year follow-on contract with the U.S. government

Deliver Raxibacumab doses under current contract; advance technology transfer to the Company’s Bayview facility in Baltimore, Maryland

Progress pipeline to have at least four product candidates in advanced development

Complete an acquisition that generates revenue within 12 months of closing

Q3 2018 FINANCIAL FORECAST
The Company forecast for Q3 2018 total revenue is $165 million to $190 million.

FOOTNOTES
(1)
See "Calculation of Diluted Earnings Per Share."
(2)
See "Reconciliation of Net Income to Adjusted Net Income and EBITDA" for a definition of terms and a reconciliation table.
(3)
Reflects an estimated tax rate that includes the expected effects of the United States Tax Cuts and Jobs Act of 2017 on the Company’s 2018 income tax provision.

CONFERENCE CALL AND WEBCAST INFORMATION
Company management will host a conference call at 5:00 pm (Eastern Time) today, August 2, 2018, to discuss these financial results. This conference call can be accessed live by telephone or through Emergent’s website:

Live Teleconference Information:
Dial in: [US] (855) 766-6521; [International] (262) 912-6157
Conference ID: 93342423

Live Webcast Information:
Visit View Source for the live webcast feed.

A replay of the call can be accessed at www.emergentbiosolutions.com under "Investors."

On August 2, 2018 Tetraphase Pharmaceuticals, Inc. (NASDAQ:TTPH), a biopharmaceutical company focused on developing and commercializing novel antibiotics to treat life-threatening multidrug-resistant (MDR) infections, reported financial results for the second quarter ended June 30, 2018 (Press release, Tetraphase, AUG 2, 2018, View Source [SID1234528425]).

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"In the second quarter of 2018, we moved closer to achieving our goal to bring XeravaTM (eravacycline) to market on a global level as an important new antibiotic for patients with serious, often life-threating, complicated intra-abdominal infections (cIAI)," said Guy Macdonald, President and Chief Executive Officer of Tetraphase. "We recently announced a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) for Xerava in cIAI, and we expect a final decision on marketing authorization from the European Commission (EC) later this year. In the U.S., we are eagerly looking forward to a decision on our PDUFA (Prescription Drug User Fee Act) date of August 28, 2018 and we are actively preparing for the potential commercial launch of Xerava in the fourth quarter."

Mr. Macdonald added, "We also have continued to make progress on our eravacycline development plans in China, through Everest Medicines, our licensee in Asia. Everest recently submitted an Investigational New Drug (IND) application for eravacycline in cIAI to the China Food and Drug Administration (CFDA) for which we received a $2.5 million milestone payment. We are excited to be moving forward in this collaboration to bring eravacycline to patients in this part of the world. Along with the U.S. and Europe, we believe there is a significant market opportunity for eravacycline in China and other Asian territories for patients with serious infections caused by Gram-negative bacteria, particularly as resistance rates continue to rise."

Key Upcoming Milestones

Potential approval of Xerava in cIAI in U.S. – Q3 2018

Potential approval of Xerava in cIAI in Europe – 2H 2018

Potential commercial launch of Xerava in cIAI in the U.S. – Q4 2018

Complete phase 1 multiple ascending dose studies for TP-271 and TP-6076 – 2H 2018

Second Quarter and Recent Highlights

Announced that the CHMP of the EMA has recommended Xerava for approval as a treatment for adult patients with cIAI. The CHMP’s opinion will be reviewed by the EC which is expected to make a final decision within three months. If approved by the EC, marketing authorization for Xerava will be granted in all 28 countries of the European Union, Norway, Iceland and Liechtenstein.
Presented data at ASM Microbe 2018, including a poster presentation that shared a pooled analysis of IGNITE1 and IGNITE4, the Company’s phase 3 studies to evaluate the efficacy and safety of Xerava versus ertapenem and meropenem, respectively, in patients with cIAI. This is the first post-hoc analysis of IGNITE1 and IGNITE4 to compare the clinical and microbiological responses at the test-of-cure visit for patients in the two treatment groups, with an emphasis on the response of MDR pathogens to Xerava. The Company also presented data highlighting the in vitro activity of Xerava and comparators against Gram-negative isolates from a large-scale global surveillance study, as well as the activity of TP-6076 against carbapenem-resistant Acinetobacter baumannii isolates.
Announced Everest Medicines’ submission of an IND application for eravacycline in cIAI to the CFDA. Everest has the exclusive license to develop and commercialize eravacycline for the treatment of cIAI and other indications in China, Taiwan, Hong Kong, Macau, South Korea and Singapore. Tetraphase received a milestone payment of $2.5 million following Everest’s IND application submission with the CFDA in June 2018.
Second Quarter 2018 Financial Results
As of June 30, 2018, Tetraphase had cash and cash equivalents of $111.2 million and 52.9 million shares outstanding. The Company expects that its cash and cash equivalents, as well as expected revenue from its U.S. government awards, will be sufficient to fund operations through the third quarter of 2019.

Revenues during the second quarter of 2018 were $11.6 million compared to $1.6 million for the same period in 2017. The $11.3 million in total revenue consisted of a $7.0 million upfront license fee and the $2.5 million Chinese IND filing milestone, both earned under the Company’s license agreement with Everest Medicines Limited, as well as $2.1 million in contract and grant revenue under the Company’s U.S. government awards.

Research and development (R&D) expenses for the second quarter of 2018 were $14.4 million compared to $28.5 million for the same period in 2017. The decrease in R&D expenses was primarily due to the completion of our IGNITE phase 3 clinical studies for Xerava.

General and administrative expenses for the second quarter of 2018 were $7.2 million compared to $5.1 million for the same period in 2017. This increase was primarily due to pre-commercialization expenses.

For the second quarter of 2018, Tetraphase reported a net loss of $9.5 million, or ($0.18) per share, compared to a net loss of $31.8 million, or ($0.83) per share, for the same period in 2017.

About Xerava
Xerava is a novel, fully-synthetic fluorocycline antibiotic being developed for the treatment of cIAI and other serious infections, including those caused by MDR pathogens that have been highlighted as urgent public health threats by both the World Health Organization (WHO) and the Centers for Disease Control and Prevention (CDC). Xerava has demonstrated potent in vitro activity against MDR pathogens, including carbapenem-resistant Enterobacteriaceae, Acinetobacter baumannii, and colistin-resistant bacteria carrying the mcr-1 gene.

Xerava was investigated for the treatment of cIAI as part of the Company’s Investigating Gram-negative Infections Treated with Eravacycline (IGNITE) phase 3 program. In IGNITE1, a pivotal phase 3 trial in patients with cIAI, twice-daily intravenous (IV) Xerava met the primary endpoint by demonstrating statistical non-inferiority of clinical response compared to ertapenem, was well-tolerated and achieved high cure rates in patients with Gram-negative pathogens, including resistant isolates. In IGNITE4, a second phase 3 clinical trial in patients with cIAI, twice-daily IV Xerava met the primary endpoint by demonstrating statistical non-inferiority of clinical response compared to meropenem, was well-tolerated and achieved high cure rates. Xerava has not been approved for commercial use.

On August 2, 2018 Epizyme, Inc. (NASDAQ: EPZM), a clinical-stage company developing novel epigenetic therapies, reported financial results for the second quarter of 2018 and provided key business updates (Press release, Epizyme, AUG 2, 2018, View Source [SID1234528418]).

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"In the second quarter, we presented encouraging new clinical data regarding tazemetostat’s anti-tumor activity and tolerability in follicular lymphoma and mesothelioma," said Robert Bazemore, president and chief executive officer of Epizyme. "As we enter the second half of 2018, we have focused the organization on several strategic priorities. First and foremost, we are working diligently to resolve the partial clinical hold and resume enrollment in tazemetostat clinical studies. In addition, we are progressing tazemetostat toward a first NDA for the treatment of epithelioid sarcoma, continuing to advance its development in follicular lymphoma based on the strength of our clinical data, and advancing our novel inhibitor of G9a, EZM8266, toward the clinic. We believe the actions we have taken will allow us to capitalize on our near-term tazemetostat opportunities while also extending our cash runway."

Partial Clinical Hold Update

A partial clinical hold pausing the enrollment of new patients into tazemetostat clinical trials was implemented in the second quarter of 2018 in the United States, France and Germany following a safety report of a single pediatric patient who developed a secondary T-cell lymphoblastic lymphoma (T-LBL). Epizyme has reconsented all patients in its clinical trials and updated its informed consent form based on this safety report. The company also reviewed the single T-LBL case in detail, recently completed a comprehensive assessment of tazemetostat safety data and clinical activity observed to date across clinical trials, and convened a panel of external experts to review and validate the assessment. This information will be included in a formal response to regulatory authorities.

Epizyme plans to continue its engagement with the U.S. Food and Drug Administration (FDA) in the weeks ahead and then finalize its response to regulatory authorities, including changes that may be proposed to study protocols. Once the company has gained alignment with regulators in the U.S., France and Germany, it is anticipated that the partial clinical hold would be lifted and that enrollment activities would be allowed to proceed in those countries.

ES Program Update

At the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress in October 2018, Epizyme plans to present updated Phase 2 data from patients with epithelioid sarcoma (ES) who are receiving tazemetostat as a monotherapy. Enrollment in this trial was completed in July 2017. A recent assessment of interim data from the full 62-patient ES cohort in this study has shown that the objective response rate has remained consistent with what was observed in the initial 31 enrolled patients. In addition, durability data from the cohort continue to mature.

Epizyme is continuing to prepare its first New Drug Application (NDA) submission for tazemetostat for the treatment of patients with ES. In order to include more mature durability data

in its submission and based on the potential impact of the partial clinical hold on the timing of the company’s pre-NDA meeting with the FDA, Epizyme now plans to submit its NDA in the first half of 2019.

DLBCL Program Update

Epizyme has been conducting a Phase 2 trial that is assessing tazemetostat activity in cohorts of patients with relapsed and/or refractory diffuse large B-cell lymphoma (DLBCL). These cohorts include DLBCL patients with EZH2 activating mutations and with wild-type EZH2 who are receiving tazemetostat as monotherapy. The trial also includes a cohort of DLBCL patients who are receiving tazemetostat in combination with prednisolone. Epizyme has conducted an interim assessment of data from this trial and concluded that the clinical activity seen in these cohorts is not sufficient to warrant further development of tazemetostat in DLBCL as a monotherapy or in combination with prednisolone. Epizyme plans to present clinical data from each of these study cohorts at a medical meeting in the second half of 2018. The company has two additional combination studies in DLBCL ongoing and plans to evaluate other potential combinations in this aggressive and difficult-to-treat cancer longer term.

Recent Progress

New Chief Medical Officer: Epizyme recently appointed Dr. Shefali Agarwal as chief medical officer. In this role, Dr. Agarwal will oversee all of the company’s activities related to the global strategic development of tazemetostat and additional pipeline candidates. A trained medical oncologist, Dr. Agarwal brings to Epizyme nearly two decades of clinical research and regulatory expertise as well as leadership experience in clinical development, clinical operations and medical affairs.

Positive Data in Follicular Lymhoma (FL): At the 23rd Congress of the European Hematology Association (EHA) (Free EHA Whitepaper) in Stockholm, positive interim data were reported from the follicular lymphoma cohorts in Epizyme’s ongoing Phase 2 study of tazemetostat in non-Hodgkin lymphoma. The data as of May 1, 2018 showed that tazemetostat continued to demonstrate meaningful clinical activity as a monotherapy and was generally well tolerated in adult patients with relapsed and/or refractory FL. An objective response rate (ORR) of 71 percent was observed in the cohort of FL patients with EZH2 activating mutations (n=28), with an interim median duration of response (DOR) of approximately 32 weeks. An ORR of 33 percent was observed in the fully-enrolled cohort of FL patients with wild-type EZH2 (n=54), with an interim median DOR of approximately 76 weeks. The median DOR figures in these cohorts continue to mature, with more than half of the responders still on therapy. After resolving the partial clinical hold, Epizyme plans to re-engage with the FDA to refine the company’s registration plans for tazemetostat in relapsed and/or refractory FL.

Clinical Activity in Mesothelioma: At the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in Chicago, clinical data were reported from Epizyme’s Phase 2 study of tazemetostat as a monotherapy in relapsed and/or refractory malignant mesothelioma patients with BRCA1-associated protein 1 (BAP1) loss-of-function. The primary endpoint in this trial was met with 51 percent of patients (31/61) having achieved disease control at 12 weeks, exceeding the pre-specified disease control rate threshold of ³35 percent. Tazemetostat was generally well tolerated in this study.

Second Quarter 2018 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $215.6 million as of June 30, 2018, which compares to $247.9 million as of March 31, 2018.

Revenue: Revenue for the second quarter of 2018 was $12.0 million, which compares with $10.0 million in revenue for the second quarter of 2017. The increase is due to greater milestone-related revenue from the company’s collaboration and license agreement with GlaxoSmithKline.

R&D Expenses: Research and development (R&D) expenses were $31.3 million for the second quarter of 2018, which compares to $27.3 million for the second quarter of 2017. The increase is primarily due to greater tazemetostat manufacturing expense, increased clinical and regulatory activities associated with tazemetostat’s development and preclinical studies related to the company’s G9a inhibitor candidate.

G&A Expenses: General and administrative (G&A) expenses were $10.9 million for the second quarter of 2018, which compares to $11.2 million for the second quarter of 2017. The decline is primarily due to reduced consulting costs.

Net Loss: The company’s net loss was $29.1 million, or $0.42 per share, for the second quarter of 2018, which compares to a net loss of $28.0 million, or $0.48 per share, for the second quarter of 2017.

Financial Guidance

Epizyme has extended its cash runway guidance based on changes that are being made to its planned operating expenditures. The company now expects that its existing cash, cash equivalents and marketable securities will be sufficient to fund its planned operations into the fourth quarter of 2019.

Conference Call Reminder

As previously announced, management plans to host a conference call and webcast at 8:30 a.m. ET today to discuss the company’s second quarter 2018 results and other business updates. To participate, please dial (877) 844-6886 (domestic) or (970) 315-0315 (international) and refer to conference ID 5734199. A live webcast will be available in the investor section of the company’s website at www.epizyme.com. The webcast also will be archived on the website for 60 days.

About the Tazemetostat Clinical Trial Program

Tazemetostat, a first-in-class EZH2 inhibitor, is currently being studied as a monotherapy in ongoing Phase 2 programs in certain molecularly defined solid tumors, including epithelioid sarcoma and other INI1-negative tumors; follicular lymphoma (FL); and combination studies in diffuse large B-cell lymphoma (DLBCL) and non–small cell lung cancer (NSCLC).

On August 2, 2018 Aeterna Zentaris Inc. (NASDAQ:AEZS) (TSX: AEZS) reported that it will announce its second quarter 2018 financial and operating results after market close on Thursday, August 9, 2018. The Company will host a conference call to discuss these results on Friday, August 10, 2018 at 8:30 a.m. Eastern Time.

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Participants may access the conference call by telephone using the following numbers:

Toll-Free: 877-407-8029, Confirmation #13681858
Toll: 201-689-8029, Confirmation #13681858
A replay will also be available on the Company’s website for a period of 30 days.

On August 2, 2018 Acceleron Pharma Inc. (Nasdaq:XLRN), a leading biopharmaceutical company in the discovery and development of TGF-beta therapeutics to treat serious and rare diseases, reported financial results for the second quarter ended June 30, 2018.

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"We have had a very successful start to 2018, and we look forward to carrying this momentum forward throughout the rest of the year and beyond. We are extremely pleased that luspatercept, our lead product candidate and first-in-class erythroid maturation agent, achieved positive and highly statistically significant Phase 3 results in both the MEDALIST and BELIEVE trials, confirming our confidence in its clinical profile in myelodysplastic syndromes and beta-thalassemia," said Habib Dable, President and Chief Executive Officer of Acceleron. "We and our collaboration partner, Celgene, look forward to presenting the Phase 3 data at an upcoming medical congress and are focused on the execution of key regulatory activities, including US and EU submission in the first half of 2019. We believe luspatercept is a potential platform treatment to transform the lives of patients suffering from a range of hematologic diseases associated with anemia.
Added Mr. Dable: "Our neuromuscular and pulmonary programs achieved critical milestones, putting us in a position for important Phase 2 readouts for ACE-083 and sotatercept in 2019 and 2020, respectively."

Development Program Highlights

Hematology

Luspatercept:

Myelodysplastic Syndromes (MDS), Beta-Thalassemia, and Myelofibrosis (MF)
Luspatercept is a first-in-class erythroid maturation agent (EMA) designed to treat the 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 MEDALIST and BELIEVE Phase 3 trials in patients with lower-risk MDS and transfusion-dependent beta-thalassemia, respectively, met all primary and key secondary endpoints.

Data will be submitted to a future medical meeting for presentation in late 2018.

Acceleron and Celgene plan to submit regulatory filings in the United States and Europe in the first half of 2019.

Updated results from the ongoing Phase 2 trials in MDS and beta-thalassemia were presented at the 2018 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting and the 23rd Congress of the European Hematology Association (EHA) (Free EHA Whitepaper) in June 2018.

The initiation of the COMMANDS Phase 3 trial in patients with lower-risk MDS who are treatment naïve is planned for the third quarter of 2018.

Enrollment and treatment are ongoing in the BEYOND Phase 2 trial in non-transfusion-dependent beta-thalassemia and the Phase 2 trial in MF.

Neuromuscular Disease

ACE-083:

Facioscapulohumeral Muscular Dystrophy (FSHD) and Charcot-Marie-Tooth (CMT) Disease
ACE-083 is a 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 ligands.

Preliminary results from Part 1 of the ACE-083 Phase 2 trial in patients with CMT disease were highlighted in oral and poster presentations at the 2018 Peripheral Nerve Society (PNS) Annual Meeting in July.

Part 2 of the Phase 2 trial in patients with CMT was recently initiated with preliminary results expected by the end of 2019.

The Company plans to present results from all Part 1 dose cohorts in the FSHD Phase 2 trial in October 2018.

Enrollment and treatment are ongoing in Part 2 of the Phase 2 trial in patients with FSHD and preliminary results are expected in the second half of 2019.

ACE-083 received FDA Fast Track status and Orphan Drug designation in FSHD.

ACE-2494:
ACE-2494 is designed to have a systemic effect on muscle mass and strength for diseases that cause muscle weakness throughout the body. ACE-2494 utilizes the "Myostatin+" approach to inhibit multiple TGF-beta ligands.

Enrollment and treatment are ongoing in the Phase 1 healthy volunteer trial with preliminary results expected in the first half of 2019.

Pulmonary Disease
Sotatercept:

Pulmonary Arterial Hypertension (PAH)

Sotatercept acts as a ligand trap for members of the TGF-beta superfamily directly involved in the BMP signaling pathway, which is proven critical for maintaining healthy pulmonary vasculature. In multiple preclinical studies in PAH, sotatercept significantly decreased pulmonary vessel muscularization, improved pulmonary arterial pressures, and decreased indicators of right heart failure.

The Company initiated the PULSAR Phase 2 trial in patients with PAH with preliminary results expected in the first half of 2020.

The Company plans to initiate an exploratory imaging study in Q1 2019 to provide additional understanding of endpoints in anticipation of a potential pivotal trial in the future.

In November 2018, the Company will host a PAH Research and Development Deep Dive event in New York City.

The event will include internal and external expert presentations to discuss disease background, current treatment landscape, key disease pathways including BMP signaling, Acceleron’s clinical development activities, and the latest sotatercept preclinical results.

Corporate Highlights

Robert K. Zeldin, M.D., was appointed Chief Medical Officer (CMO). He brings 20 years of industry experience and joined from Ablynx, where he served as Chief Medical Officer. Prior to Ablynx, Dr. Zeldin served in senior roles at Novartis, Merck, and the U.S. Food & Drug Administration’s Center for Biologics Evaluation and Research.

Janethe Pena, M.D., Ph.D., recently joined us as Vice President of Pulmonary to lead the company’s clinical development efforts in this area. Dr. Pena most recently served as Vice President and Group Head of Pulmonology Clinical Development at Bayer Pharmaceuticals. At Bayer, she was responsible for the pulmonary portfolio, including leading clinical trials with riociguat (Adempas) in different pulmonary hypertension indications and the overall program life cycle management.
Financial Results

Cash position – Cash, cash equivalents and investments as of June 30, 2018 were $332.3 million. As of December 31, 2017, the Company had cash, cash equivalents and investments of $372.9 million. The Company believes that existing cash, cash equivalents and investments will be sufficient to fund projected operating requirements into 2021.

Revenue – Collaboration revenue for the second quarter was $3.7 million. The revenue is all from Acceleron’s partnership with Celgene and is primarily related to expenses incurred by the Company in support of luspatercept.

Costs and expenses – Total costs and expenses for the second quarter were $33.6 million. This includes R&D expenses of $25.9 million and G&A expenses of $7.7 million.

Net loss – The Company’s net loss for the second quarter ended June 30, 2018 was $28.9 million.
Conference Call and Webcast
The Company will host a webcast and conference call to discuss its second quarter financial results for 2018 and provide an update on recent corporate activities on August 2, 2018, 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 2018 Earnings Call."

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