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Emergent BioSolutions Reports Second Quarter and Six Months 2015 Financial Results and Reaffirms 2015 Guidance

On August 6, 2015 Emergent BioSolutions Inc. (NYSE:EBS) reported financial results for the quarter and six months ended June 30, 2015 (Press release, Emergent BioSolutions, AUG 6, 2015, View Source;p=RssLanding&cat=news&id=2076503 [SID:1234507057]).

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Financial highlights include:
Total revenues: Q2 2015 of $126.1 million, up 14% over prior year; six months 2015 of $189.7 million, up 16% over prior year;
GAAP net income/loss: Q2 2015 net income of $14.1 million, or $0.32 per diluted share; six months 2015 net loss of $7.4 million, or $0.19 per diluted share;

Adjusted net income/loss: Q2 2015 net income of $17.0 million, or $0.36 per diluted share; six months 2015 net loss of $1.8 million, or $0.05 per diluted share;

EBITDA: Q2 2015 of $29.6 million, or $0.62 per diluted share; six months 2015 of $9.6 million, or $0.25 per diluted share; and
Adjusted EBITDA: Q2 2015 of $31.0 million, or $0.65 per diluted share; six months 2015 of $12.2 million, or $0.32 per diluted share.

2015 business accomplishments:

FDA approval of Anthrasil (Anthrax Immune Globulin Intravenous (Human))
Awards to manufacture Ebola monoclonal antibodies under our CIADM arrangement with BARDA
Successful dosing of our first patient in the Phase I trial for MOR209/ES414, our immunotherapeutic treatment for prostate cancer
FDA approval and launch of IXINITY, a recombinant factor IX treatment for Hemophilia B
Continued steady progress on Building 55 sBLA approval

2015 outlook:

Reaffirmation of previous guidance – FY 2015 total revenues of $510-$540 million, net income of $50-$60 million (GAAP) and $60-$70 million (adjusted); and

New guidance – Q3 2015 total revenues of $140 to $155 million.

2015 FINANCIAL PERFORMANCE
(I) Quarter Ended June 30, 2015 (unaudited)

Revenues

Product Sales

For Q2 2015, product sales were $82.0 million, an increase of 5% as compared to 2014. The increase primarily reflects increased sales of BioThrax during the quarter.

Contract Manufacturing

For Q2 2015, revenue from our contract manufacturing operations was $8.9 million, a decrease of 3% as compared to 2014. The decrease was primarily due to the timing of fill/finish facility service to third parties.
Contracts, Grants and Collaborations
For Q2 2015, contracts, grants and collaborations revenue was $35.2 million, an increase of 54% as compared to 2014. The increase was primarily due to development funding for Anthrasil.

Operating Expenses

Cost of Product Sales and Contract Manufacturing

For Q2 2015, cost of product sales and contract manufacturing was $27.3 million, a decrease of 21% as compared to 2014. The decrease was primarily attributable to the decrease in the BioThrax cost per dose sold associated with increased production yield in the period in which the doses were produced.

Research and Development
For Q2 2015, gross research and development (R&D) expenses were $40.9 million, an increase of 9% as compared to 2014. The increase was primarily attributable to additional R&D expenditures associated with product development programs in the Biodefense segment. Net R&D expenses, which are more representative of the company’s actual out-of-pocket investment in product development, are calculated as gross research and development expenses less contracts, grants and collaboration revenues. For Q2 2015, net R&D expenses were $5.7 million, a decrease of 61% as compared to 2014.

Selling, General and Administrative
For Q2 2015, selling, general and administrative expenses were $36.5 million, an increase of 19% as compared to 2014. The increase was primarily attributable to selling, general and administrative costs associated with the launch of IXINITY and professional services to support the company’s strategic growth initiatives.

Net Income
For Q2 2015, GAAP net income per diluted share is computed using the if-converted method. This method requires GAAP net income to be adjusted in the amount of $1.0 million, from $14.1 million to $15.1 million, related to interest expense and amortization of debt issuance cost, both net of tax, associated with the company’s 2.875% Convertible Senior Notes due 2021.
(II) Six Months Ended June 30, 2015 (unaudited)

Revenues

Product Sales
For the six months of 2015, product sales were $100.3 million, a decrease of 12% as compared to 2014. The decrease was primarily attributable to the timing of deliveries of BioThrax to the SNS due to our decision to suspend shipments to the CDC in Q1 2015. Shipments were subsequently resumed in Q2 2015.

Contract Manufacturing
For six months of 2015, revenue from our contract manufacturing operations was $21.1 million, an increase of 77% as compared to 2014. The increase was primarily due to the impact of fill/finish services revenues for the entire six month period in 2015.

Contracts, Grants and Collaborations
For six months of 2015, contracts, grants and collaborations revenue was $68.3 million, an increase of 78% as compared to 2014. The increase was primarily due to development funding for Anthrasil.

Operating Expenses
Cost of Product Sales and Contract Manufacturing
For the six months of 2015, cost of product sales and contract manufacturing was $46.0 million, a decrease of 14% as compared 2014. The decrease was primarily attributable to a decrease in product sales and contract manufacturing revenues.

Research and Development
For the six months of 2015, gross R&D expenses were $79.6 million, an increase of 18% as compared to 2014. The increase was primarily attributable to additional R&D expenditures in the Biodefense segment.

Net R&D expenses for the six months of 2015 were $11.3 million, a decrease of 62% as compared to 2014.

Selling, General and Administrative

For the six months of 2015, selling, general and administrative expenses were $70.9 million, an increase of 17% as compared to 2014. The increase was primarily attributable to additional post-acquisition selling, general and administrative costs largely associated with the operations acquired in Q1 2014, including IXINITY launch costs, as well as professional services to support the company’s strategic growth initiatives.

(III) Reconciliation of GAAP Net Income to Adjusted Net Income/(Loss), EBITDA and Adjusted EBITDA

This press release contains three financial measures (Adjusted Net Income/(Loss), EBITDA or earnings before interest, taxes, depreciation and amortization, and adjusted EBITDA) that are considered "non-GAAP" financial measures under applicable Securities & Exchange Commission rules and regulations. These non-GAAP financial measures should be considered supplemental to and not a substitute for financial information prepared in accordance with generally accepted accounting principles. The company’s definition of these non-GAAP measures may differ from similarly titled measures used by others. Adjusted Net Income adjusts for specified items that can be highly variable or difficult to predict, or reflect the non-cash impact of charges resulting from purchase accounting. EBITDA reflects net income excluding the impact of depreciation, amortization, interest expense and provision for income taxes. Adjusted EBITDA also excludes specified items that can be highly variable and the non-cash impact of certain purchase accounting adjustments. The company views these non-GAAP financial measures as a means to facilitate management’s financial and operational decision-making, including evaluation of the company’s historical operating results and comparison to competitors’ operating results. These non-GAAP financial measures reflect an additional way of viewing aspects of the company’s operations that, when viewed with GAAP results and the reconciliations to the corresponding GAAP financial measure, may provide a more complete understanding of factors and trends affecting the company’s business.

The determination of the amounts that are excluded from these non-GAAP financial measures are a matter of management judgment and depend upon, among other factors, the nature of the underlying expense or income amounts. Because non-GAAP financial measures exclude the effect of items that will increase or decrease the company’s reported results of operations, management strongly encourages investors to review the company’s consolidated financial statements and publicly filed reports in their entirety.

Curis Reports Second Quarter 2015 Financial Results

On August 6, 2015 Curis, Inc. (NASDAQ:CRIS), a biotechnology company focused on the development and commercialization of innovative drug candidates for the treatment of human cancers, reported its financial results for the second quarter ended June 30, 2015 (Press release, Curis, AUG 6, 2015, View Source [SID:1234507055]).

"During the second quarter, we presented results of the Phase 1 trial of CUDC-907 at scientific conferences, where we reported clinical activity, including complete responses in heavily pre-treated patients with relapsed or refractory diffuse large B cell lymphoma (DLBCL)," said Ali Fattaey, Ph.D., Curis’ President and Chief Executive Officer. "CUDC-907 appears to be well tolerated with a manageable side effect profile at the recommended dose and schedule. Enrollment of patients with relapsed or refractory DLBCL in the expansion arms of the ongoing Phase 1 study is continuing, where we are administering CUDC-907 either as monotherapy or in combination with rituximab. We are in the process of finalizing the Phase 2 study design for CUDC-907, and we expect to initiate the study later this year."

Dr. Fattaey continued, "Our partner Aurigene has made progress in advancing programs under our collaboration, and we remain on-track to file an IND application for the first immuno-oncology drug candidate later this year. We also expect to file an IND application for the lead IRAK4 inhibitor candidate and initiate its Phase 1 program during the first half of 2016."

Second Quarter and First Half 2015 Financial Results

Curis reported a net loss of $8.1 million, or ($0.06) per share on both a basic and fully diluted basis for the second quarter of 2015, as compared to a net loss of $1.9 million or ($0.02) per share on both a basic and fully diluted basis for the same period in 2014. Curis reported a net loss of $40 million, or ($0.34) per share on both a basic and fully diluted basis for the six months ended June 30, 2015, as compared to a net loss of $7.5 million or ($0.09) per share on both a basic and fully diluted basis for the same period in 2014. The net loss for the first half of 2015 includes an in-process research and development charge of $24.3 million related to Curis’ license agreement with Aurigene.

Revenues for the second quarter of 2015 were $2.1 million, as compared to $4.8 million for the same period in 2014. The decrease in revenues was primarily due to a decrease in license fee revenues due to a $3 million milestone payment that Curis earned from Genentech/Roche upon achievement by Genentech/Roche of certain development objectives during the second quarter of 2014. Offsetting these decreases, royalty revenues recorded on Genentech/Roche’s net sales of Erivedge increased to $2.0 million in the second quarter of 2015 as compared to $1.8 million during the same period in 2014.

Revenues for the six months ended June 30, 2015 were $3.7 million, as compared to $6.1 million for the same period in 2014.

Operating expenses for the second quarter of 2015 were $9.5 million, as compared to $6.3 million for the same period in 2014. Operating expenses for the six months ended June 30, 2015 were $42.1 million, as compared to $12.4 million for the same period in 2014 and were comprised of the following:

Costs of royalty revenues. Costs of royalty revenues, which are comprised of amounts due to third-party university patent licensors in connection with Genentech/Roche’s Erivedge net sales, were $103,000 and $92,000 during the second quarters of 2015 and 2014, respectively. Costs of royalty revenues for the six months ended June 30, 2015 were $187,000, as compared to $157,000 for the same period in 2014.

In-process research and development expenses. The Company recorded a one-time charge for in-process research and development expense of $24.3 million during the first half of 2015 associated with the issuance of 17,120,131 shares of Curis common stock to Aurigene as partial consideration for the rights granted under the terms of the parties’ January 2015 collaboration agreement.

Research and development expenses. Research and development expenses were $5.9 million for the second quarter of 2015 as compared to $3.3 million for the same period in 2014. The increase in research and development expense was primarily due to increased spending on CUDC-907 and preclinical programs under the Company’s collaboration with Aurigene. The Company incurred expenses of $2.7 million and $1.4 million on CUDC-907 for the quarters ended June 30, 2015 and 2014, respectively, related to its ongoing Phase 1 studies. Spending of $2.5 million on the Company’s preclinical research programs for the three months ended June 30, 2015 includes a $2 million milestone payment to Aurigene for selection of a third program under that collaboration and also includes costs to support planned development activities, primarily consisting of personnel costs, compared to costs of $81,000 in the prior year quarter. Offsetting these increases, spending on CUDC-427 decreased by $900,000 during the three months ended June 30, 2015 as compared to the prior year period. Research and development expenses were $10.7 million for the six months ended June 30, 2015 as compared to $6.5 million for the same period in 2014.

General and administrative expenses. General and administrative expenses were $3.4 million for the second quarter of 2015 as compared to $2.9 million for the second quarter of 2014. Increased legal costs and stock-based compensation were partially offset by decreased professional and consulting costs. General and administrative expenses were $6.9 million for the six months ended June 30, 2015 as compared to $5.8 million for the same period in 2014.

Other expense was $759,000 for the second quarter of 2015, as compared to $351,000 for the same period in 2014. Other expense primarily consisted of $843,000 and $950,000 in interest expense for the quarters ended June 30, 2015 and 2014, respectively, related to the loan made by BioPharma-II to Curis Royalty, a wholly-owned subsidiary of Curis. The Company also recorded other income of $557,000 and $649,000 during the three and six month periods ended June 30 2014, respectively, associated with the change in fair value of a warrant liability. Other expense was $1.6 million and $1.2 million for the six month periods ended June 30, 2015 and 2014, respectively.

As of June 30, 2015, Curis’ cash, cash equivalents, marketable securities and investments totaled $99.2 million, and there were approximately 128.4 million shares of common stock outstanding.

2015 Financial Guidance

The Company has revised its 2015 financial guidance for research and development expenses for 2015. The Company currently expects that these expenses will be in the range of $30 to $35 million for 2015. The Company had previously estimated that these expenses would range from $37 to $42 million. As a result, the Company currently expects to end 2015 with cash, cash equivalents and investments of $72 to $77 million versus its previous estimate of $65 to $70 million.

The Company’s decrease in research and development expenses is primarily the result of a decrease in estimated expenses across its development programs, including a recent re-evaluation of its clinical development plans for CUDC-427, an orally-available, small molecule antagonist of IAP proteins, as well as for its HSP90 inhibitor CUDC-305. The Company’s management determined that it would preserve Curis’ current resources for the continued development of CUDC-907 and drug candidates under the Company’s collaboration with Aurigene.

Recent Operational Highlights

CUDC-907:

In May 2015, Curis reported data from the dose escalation (completed) and expansion (ongoing) stages of the Phase 1 study of CUDC-907 at the Annual Meeting of American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) that was held in Chicago, IL. CUDC-907 demonstrated evidence of clinical activity with objective responses reported in patients with relapsed/refractory DLBCL and Hodgkin’s lymphoma. Among 10 response evaluable patients with DLBCL across various cohorts, two complete responses and four partial responses were reported. One patient with Hodgkin’s lymphoma experienced partial response out of a total of 12 response evaluable patients with Hodgkin’s lymphoma. In addition, stable disease was observed in 25 of 44 response evaluable patients across various lymphomas and multiple myeloma.
In June 2015, Curis presented data from the Phase 1 study of CUDC-907 at the 20th Congress of the European Hematology Association (EHA) (Free EHA Whitepaper), in Vienna, Austria, and the 13th International Congress on Malignant Lymphoma (ICML) in Lugano, Switzerland.
Aurigene Collaboration:

In April 2015, Aurigene presented a poster entitled "Novel IRAK4 inhibitors exhibit highly potent anti-proliferative activity in DLBCL cell lines with activating MYD88 L265P mutation" at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2015 Annual Meeting. This poster included data from multiple orally bioavailable molecules that showed potent inhibition of IRAK4 kinase activity in biochemical assays and proliferation in DLBCL cancer cell lines with MYD88 mutation. Some of these compounds were further tested in in vivo models and demonstrated significant anti-tumor activity in a DLBCL xenograft model with MYD88 mutation as well as disease reduction in a rat collagen-induced arthritis model, which is a model for inflammatory conditions.
Upcoming Activities

Curis expects to present at the following investor conferences through October 2015:

Robert W. Baird & Co. 2015 Health Care Conference, September 9-10, 2015 in New York City
FBR 2nd Annual Healthcare Conference, September 9, 2015 in Boston, MA
14th Annual BIO Investor Forum, October 20-21, 2015 in San Francisco

BIND Therapeutics Reports Second Quarter 2015 Financial Results and Provides Corporate Update

On August 6, 2015 BIND Therapeutics, Inc. (NASDAQ: BIND), a clinical-stage nanomedicine company developing targeted and programmable therapeutics called AccurinsTM, reported financial results and business highlights for the second quarter ended June 30, 2015 (Press release, BIND Therapeutics, AUG 6, 2015, View Source [SID:1234507054]).

"In the second quarter we made significant progress with our clinical programs and continued executing on our vision to develop new categories of Accurins that have a significant impact in multiple therapeutic areas," said Andrew Hirsch, president and chief executive officer of BIND Therapeutics. "We believe BIND-014, which is on track for an initial phase 2 data readout from the iNSITE 1 trial later this year, has the potential to have a meaningful impact on patients with multiple solid tumors types. We have also initiated programs that leverage the broad potential of our platform to develop Accurins that target new cell types and compartments with highly efficacious payloads and limited exposure to healthy tissue. We believe our innovative approach to develop new categories of Accurins can fundamentally change how diseases are treated."

During the quarter, BIND advanced its product development pipeline with continued strong enrollment of the KRAS mutant and squamous histology cohorts in the iNSITE 1 non-small cell lung cancer trial with BIND-014. The Company also initiated site activation in the iNSITE 2 trial in patients with four orphan tumor histologies: cholangiocarcinoma, advanced cervical cancer, advanced bladder cancer, and advanced squamous cancer of the head and neck. First patient dosing in the iNSITE 2 trial is anticipated in the third quarter of 2015.

Additionally, BIND’s collaborator AstraZeneca filed an investigational new drug application (IND) and received clearance to begin a phase 1 trial with AZD-2811, a novel Aurora B kinase inhibitor that will be the second Accurin to enter the clinic. BIND and AstraZeneca expect the patient enrollment in this trial to begin in the fourth quarter of 2015. Under the terms of its agreement with AstraZeneca, BIND will earn a $4 million milestone payment upon first dosing a patient in a phase 1 clinical trial with AZD-2811.

"We are making great progress with our BIND-014 clinical program and enrollment for both cohorts of iNSITE 1 is proceeding as planned and fully meeting our expectations," said Hagop Youssoufian, M.Sc., M.D., chief medical officer at BIND. "We are also excited to enroll the first patient in the iNSITE 2 trial with BIND-014, an important milestone considering the signals of clinical efficacy we saw in our phase 1 trial in patients with cholagio, cervical and head and neck cancers. BIND-014 has the potential to become an effective new therapy that both improves the treatment experience and offers greater efficacy for patients with multiple tumor types who currently have limited options."

BIND previously announced efforts to develop new categories of Accurins that can be applied to multiple therapeutic areas. During the second quarter of 2015, the Company initiated early formulation work to engineer new Accurin product concepts containing anti-infectives and oligonucleotides. These programs leverage the ability of the Accurin platform to target promising payloads to diseased cells and tissues types that have been challenging to treat with conventional therapies.

BIND also entered into a research collaboration with Macrophage Therapeutics to combine their Manocept platform, which targets the CD206 receptor on disease-associated macrophages, with BIND’s Accurin platform. Activated macrophages play an important role in the tumor microenvironment and other diseases and the goal of the collaboration is to target Accurins to CD206 disease-associated macrophages in the tumor microenvironment.

"Our collaboration with Navidea’s Macrophage Therapeutics is a result of our efforts to develop Accurins that incorporate novel APIs and ligands that target important pathways in additional therapeutic areas," continued Hirsch. "This collaboration could result in Accurins that are able to selectively bind to CD206 positive disease-associated macrophages, which help create an immunosuppressive tumor environment for many types of cancers. In addition to a potentially innovative approach in oncology, this collaboration could result in Accurins that provide new treatments across a broad array of diseases that trigger the activation of disease-associated macrophages, including infectious disease, autoimmune and CNS diseases."

Anticipated upcoming milestones include:

Initial data readouts in the fourth quarter of 2015 from the iNSITE1 trial in KRAS mutant and squamous histology non-small cell lung cancer, which we expect to provide the evidence needed to determine the most appropriate regulatory path to market.
Dose first patient in the iNSITE 2 clinical trial in multiple solid tumors in the third quarter of 2015.
Report overall survival data from the completed phase 2 non-small cell lung cancer study with BIND-014 in the broader patient population following occurrence of all survival events.

Report final results from the phase 2 prostate cancer study with BIND-014 following occurrence of 75 percent of the survival events.

Dose first patient in phase 1 clinical trial with AZD-2811 (with collaborator AstraZeneca) in the fourth quarter of 2015.
Potential selection of Accurin candidate in BIND’s collaboration with Pfizer in September 2015.
Report the results of our proof-of-concept work with Macrophage Therapeutics before December 31, 2015.
Continue advancing BIND-510 through important preclinical studies to position it for an IND filing in 2016.

Second Quarter 2015 Financial Results

Revenue for the second quarter of 2015 increased three percent to $2.5 million compared to the second quarter of 2014. Revenue primarily represents BIND’s reimbursable research and development expenses and collaboration up-front license fees and milestones for which revenue recognition was initially deferred and is being recognized over the performance period from BIND’s collaborations with AstraZeneca and Pfizer.

The increase in revenue from the 2014 period to the 2015 period was primarily due to total revenue recognized under the Pfizer collaboration reflecting completion of the majority of BIND’s activities before Pfizer’s first option decision in September 2015, as well as higher revenue under the AstraZeneca collaboration for increased manufacturing activities in support of the IND filing and the first-in-human clinical trials. AstraZeneca anticipates enrolling the first patient in a AZD-2811 phase 1 clinical trial in the fourth quarter of 2015, which will trigger BIND earning a $4 million milestone under its collaboration agreement with AstraZeneca. Revenue during the second quarter of 2014 included recognition of the remaining up-front license fee from the Amgen collaboration as a result of its completion.

Research and development expenses totaled $8.3 million for the second quarter of 2015, compared to $6.9 million for the second quarter of 2014. The increase was primarily driven by headcount growth to support the advancement of BIND’s internal pipeline and collaborations, as well as higher expenses as the Company scales up its clinical material manufacturing capability under our AstraZeneca collaboration.

General and administrative expenses totaled $4.4 million for the second quarter of 2015, compared to $3.8 million for the second quarter of 2014. The increase was primarily driven by general and administrative headcount growth related to compensation, including stock-based expenses.

Net loss for the second quarter of 2015 was $10.5 million, compared to a net loss of $8.4 million for the second quarter of 2014.

Cash, cash equivalents and short-term investments were approximately $53 million as of June 30, 2015. The Company expects that its cash, cash equivalents and short-term investments as of June 30, 2015 will fund operating expense and capital expenditure requirements into the third quarter of 2016. This expectation is based on the Company’s current operating plans and research and development funding that it expects to receive under its existing collaborations, but excludes any potential milestone payments.

Agios Reports Second Quarter 2015 Financial Results and Provides Updates on Clinical Development Progress

On August 6, 2015 Agios Pharmaceuticals (NASDAQ:AGIO), a leader in the fields of cancer metabolism and rare genetic metabolic disorders, reported business highlights and financial results for the second quarter ended June 30, 2015 (Press release, Agios Pharmaceuticals, AUG 6, 2015, View Source;p=RssLanding&cat=news&id=2076543 [SID:1234507053]).

"In the first half of 2015, we continued to drive rapid and meaningful progress across our development programs, as well as selecting our fourth molecule for development, AG-881, and advancing it into two Phase 1 studies," said David Schenkein, M.D., chief executive officer of Agios. "Additionally, we recently initiated DRIVE PK, a Phase 2 study of AG-348 in PK deficiency patients, and are on track to initiate two global, registration-enabling Phase 3 studies of AG-221 and AG-120 in patients with acute myeloid leukemia in the next six to 12 months. These efforts bring us closer to our goal of being a late-stage biopharmaceutical company capable of changing the treatment paradigm for people with cancer and rare genetic disorders."

KEY UPCOMING MILESTONES

Agios anticipates the following milestones from its IDH clinical development programs in collaboration with Celgene:

AG-221: a first-in-class, oral, selective, potent inhibitor of the mutated IDH2 protein

Initiate a global Phase 3 registration-enabling study in relapsed/refractory acute myeloid leukemia (AML) patients who harbor an IDH2 mutation by the end of 2015.

Initiate combination trials to evaluate AG-221 as a potential frontline treatment for patients with AML who harbor an IDH2 mutation by the end of 2015.

AG-120: a first-in-class, oral, selective, potent inhibitor of the mutated IDH1 protein

Begin combination trials to evaluate AG-120 as a potential frontline treatment for AML patients who harbor an IDH1 mutation by the end of 2015.

Initiate a global registration-enabling Phase 3 study in AML patients who harbor an IDH1 mutation in the first half of 2016.
Present data on the ongoing Phase 1 study of AG-120 in IDH1 mutant-positive advanced solid tumors at a medical meeting in the fourth quarter of 2015.

RECENT DEVELOPMENT UPDATES IN CANCER METABOLISM

Agios has provided the following updates on its clinical development programs in collaboration with Celgene:

AG-221

New data from the dose-escalation phase and expansion cohorts from the ongoing Phase 1 study evaluating single agent AG-221 were presented in June at the 20th Congress of the European Hematology Association (EHA) (Free EHA Whitepaper). Read the full AG-221 data here.
As of July, Agios completed the dose-escalation portion of the Phase 1 study of AG-221 in IDH2 mutant-positive hematologic malignances. The expansion cohorts are on track, including the fifth expansion cohort of 125 patients with IDH2 mutant-positive AML who are in second or later relapse, refractory to second-line induction or re-induction treatment, or have relapsed after allogeneic transplantation.

Dose escalation continues in the Phase 1/2 trial in patients with advanced solid tumors and angioimmunoblastic T-cell lymphoma (AITL) who carry an IDH2 mutation.

AG-120

New data from the dose-escalation phase of the ongoing Phase 1 study evaluating single agent AG-120 in advanced hematologic malignancies were presented in June at EHA (Free EHA Whitepaper). Read the full AG-120 data here.

Also announced in June, the U.S. Food and Drug Administration (FDA) granted Agios orphan drug designation for AG-120 for the treatment of patients with IDH1 mutant-positive AML.

In May, Agios announced that the FDA granted Fast Track designation to AG-120 for the treatment of patients with IDH1 mutant-positive AML.

As of July, Agios completed the dose-escalation portion of the Phase 1 study of AG-120 in IDH1 mutant-positive hematologic malignances. The three expansion cohorts to evaluate AG-120 in 175 patients with IDH1-mutated advanced hematologic malignancies, including one cohort with 125 patients with relapsed and/or refractory AML, are on track.
AG-881: a brain-penetrant, first-in-class, oral, potent pan-inhibitor of the mutated IDH1 and IDH2 proteins

In June, Agios announced that the first patient was dosed in a Phase 1, open-label, dose-escalation and expansion study of single agent AG-881 in patients with IDH mutant-positive advanced solid tumors, including gliomas.
In August, the first patient was dosed in a second dose-escalating and expansion trial for patients with IDH mutant-positive advanced hematologic malignancies, whose cancer has progressed on a prior IDH inhibitor therapy.

RECENT DEVELOPMENT UPDATES IN RARE GENETIC METABOLIC DISORDERS

AG-348: a novel, first-in-class, oral activator of pyruvate kinase-R (PKR) for the treatment of pyruvate kinase (PK) deficiency

In June, final data from the Phase 1 multiple-ascending dose (MAD) study in healthy volunteers were presented at EHA (Free EHA Whitepaper), establishing clear proof-of-mechanism for AG-348. Read the full AG-348 data, as well as data from Boston Children’s natural history study, here.

Also in June, Agios initiated DRIVE PK, a global Phase 2, open-label safety and efficacy trial in adult, transfusion-independent patients with PK deficiency. The first patient was dosed in July.

CORPORATE UPDATE

Agios plans to host and webcast an investor event in October 2015 in an effort to provide background and education on the novel programs it is targeting and the diseases the company aims to treat. Additional details will be provided in the coming weeks.

SECOND QUARTER 2015 FINANCIAL RESULTS

Cash, cash equivalents and marketable securities as of June 30, 2015 were $434.0 million, compared to $467.4 million as of December 31, 2014. The decrease was driven by cash expenditures for operating activities of approximately $64.9 million, which was offset by cash received from Celgene of approximately $43.3 million during the six months ended June 30, 2015 related to our collaboration agreements.

Collaboration revenue was $13.2 million for the second quarter of 2015, compared to $8.4 million for the comparable period in 2014. The increase reflects revenues recognized under the company’s collaboration agreements with Celgene, including $8.8 million related to the delivery of the U.S. and ex-U.S. licenses for AG-881.

Research and development (R&D) expense was $36.4 million, including $4.6 million of stock- based compensation expense in the second quarter of 2015, compared to $22.6 million, including $1.4 million in stock-based compensation expense for the comparable period in 2014. The increase in R&D expense was primarily due to increased costs to support advancement of the company’s lead investigational medicines toward later-stage development.

General and administrative (G&A) expense was $8.9 million, including $3.6 million of stock-based compensation expense, in the second quarter of 2015, compared to $4.2 million, including $1.0 million of stock-based compensation expense, for the comparable period in 2014. The increase in G&A expense was largely due to increased headcount and other professional expenses to support growing operations.

Net loss for the second quarter of 2015 was $31.9 million, compared to net loss of $18.3 million for the comparable period in 2014.

ADJUSTED FINANCIAL GUIDANCE FOR THE FULL YEAR 2015

Today Agios raised its previous year end cash guidance and now expects to end 2015 with more than $350.0 million of cash, cash equivalents and marketable securities. The anticipated year end 2015 cash position does not include any additional program-specific milestone payments. Agios expects that its cash, cash equivalents and marketable securities would be sufficient to fund its operating expenses and capital expenditure requirements until late 2017.

Acceleron Pharma Reports Second Quarter 2015 Financial and Operational Results

On August 6, 2015 Acceleron Pharma Inc. (NASDAQ:XLRN), a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of novel therapeutic candidates that regulate cellular growth and repair, provided a corporate update and reported financial results for the second quarter ended June 30, 2015 (Press release, Acceleron Pharma, AUG 6, 2015, View Source [SID:1234507052]).

"In the second quarter, we presented promising new longer-term treatment data from our luspatercept programs. These results further reinforce our excitement about the potential of luspatercept to help patients with myelodysplastic syndromes and beta-thalassemia. We and our collaboration partner, Celgene, look forward to starting pivotal trials in both of these indications by the end of this year," said John Knopf, Ph.D., Chief Executive Officer of Acceleron. "At Acceleron, we remain focused on bringing innovative therapies to patients, and we are continuing to advance and expand our earlier stage pipeline as well. We have scheduled our first Research and Development Day for investors on October 23, 2015. At that meeting, we will review our clinical and preclinical programs, including a first look at top-line data from the phase 1 trial with our candidate muscle agent, ACE-083."

Recent Highlights and Current Updates

Development Programs

Hematology

Completed enrollment and treatment in the luspatercept phase 2 dose escalation study in myelodysplastic syndromes (MDS) – Enrollment and treatment ongoing in the phase 2 dose escalation beta-thalassemia study.

Long-term phase 2 extension studies ongoing with luspatercept in both MDS and beta-thalassemia – Patients who completed their 3-month treatment in the MDS or beta-thalassemia dose escalation studies were eligible to enroll in the 12-month MDS or beta-thalassemia extension study. Enrollment in the MDS extension study is complete and enrollment in the beta-thalassemia extension study is ongoing.

Presented luspatercept phase 2 data supporting the advancement to phase 3 clinical trials at the European Hematology Association (EHA) (Free EHA Whitepaper)’s annual meeting – In lower risk MDS patients, which represent a large majority of patients affected by the disease, longer-term treatment with luspatercept led to sustained increases in hemoglobin levels and transfusion independence. In both transfusion and non-transfusion dependent beta-thalassemia patients, luspatercept generated durable increases in hemoglobin levels, reductions in transfusion burden and improvements in iron overload. Based on these promising data, Acceleron and Celgene are advancing luspatercept to phase 3 clinical trials in MDS and beta-thalassemia.

Luspatercept granted Fast Track designations for beta-thalassemia – The United States Food and Drug Administration (FDA) has granted Fast Track Designations to luspatercept for two separate indications—the use of luspatercept for the treatment of patients with transfusion dependent beta-thalassemia and the use of luspatercept for the treatment of patients with non-transfusion dependent beta-thalassemia.

Oncology

Presented preliminary renal cell carcinoma (RCC) phase 2 data showing that the combination of dalantercept and axitinib generated encouraging, progression-free survival – In 2nd through 4th line RCC patients, the aggregate median progression-free survival (PFS) rate for the combination of dalantercept and axitinib across all three dose levels of dalantercept tested was 8.3 months. The median PFS has not yet been reached for the 0.9 mg/kg dose group, which has been chosen as the dose level for the randomized Part 2 stage of this study.

Muscle Diseases

Completed enrollment and treatment in ACE-083 phase 1 clinical trial – The phase 1 study of ACE-083, a therapeutic candidate designed to selectively increase muscle mass and strength in the muscle into which it is administered, has completed enrollment and treatment in healthy volunteers.
Advanced and expanded pipeline – Acceleron is advancing several promising preclinical programs, particularly those designed to increase muscle mass and strength. Our goal is to advance a fifth, internally discovered therapeutic candidate into clinical trials by the end of 2016.

Upcoming Milestones and Events

Phase 3 clinical trials with luspatercept in MDS and beta-thalassemia expected to start by year-end – Acceleron and Celgene plan to initiate pivotal programs for luspatercept in beta-thalassemia and MDS by year-end.

Acceleron Research and Development (R&D) Day scheduled for Friday October 23, 2015 – Acceleron will hold its first R&D Day on Friday morning, October 23, 2015 in New York City.

ACE-083 phase 1 clinical data – Preliminary, top-line data from the ACE-083 phase 1 clinical trial will be presented at the Acceleron R&D Day on October 23.

Data from a new preclinical muscle program expected to be presented at the World Muscle Society annual meeting – Acceleron plans to present data from a new preclinical program, ACE-2494, at the World Muscle Society’s 20th International Congress in Brighton, UK, which runs from September 30 to October 4, 2015.

Financial Results

Cash Position – Cash, cash equivalents and investments as of June 30, 2015 were $156.6 million. As of December 31, 2014 the company had cash and cash equivalents of $176.5 million. Acceleron expects that its cash, cash equivalents and investments as of June 30, 2015 will be sufficient to fund the Company’s operations into the second half of 2017.