On May 05, 2016 Celldex Therapeutics, Inc. (NASDAQ:CLDX) reported business and financial highlights for the first quarter ended March 31, 2016 (Press release, Celldex Therapeutics, MAY 5, 2016, View Source [SID:1234511977]).

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"While the recent setback of the RINTEGA program was certainly a disappointment, the Company is focused on executing across the breadth and depth of our pipeline, including six ongoing company-led clinical trials—the pivotal METRIC study in triple negative breast cancer, two Phase 2 studies across a broad range of indications and multiple Phase 1/2 studies that are actively enrolling patients," said Anthony Marucci, Co-founder, President and Chief Executive Officer of Celldex Therapeutics.

"To this end, Celldex and our collaborators presented seven posters at the recent AACR (Free AACR Whitepaper) meeting across multiple compounds in our pipeline and introduced a new preclinical program that has identified promising agonist antibodies targeting the CD40 receptor. Most importantly, we reported favorable safety and immune monitoring data from the Phase 1 study of varlilumab and Opdivo. The Phase 2 study enrolled its first patients last month and will continue to add to a wealth of data slated for presentation later in 2016 and throughout 2017, including the presentation of Phase 2 data from the glembatumumab vedotin study in metastatic melanoma later this year. With the recent realignment of our pipeline, we believe our current cash position will carry us through the first half of 2018, allowing us to read out all current ongoing studies and to initiate several new studies, as well," concluded Marucci.

Program Updates:

RINTEGA ("rindopepimut"; "rindo"; CDX-110), an EGFRvIII(v3)-specific therapeutic vaccine for glioblastoma (GBM)

In March, Celldex announced that the independent Data Safety and Monitoring Board (DSMB) determined, based on a preplanned interim analysis, that continuation of the Phase 3 ACT IV study of RINTEGA in patients with newly diagnosed EGFRvIII-positive glioblastoma would not reach statistical significance for overall survival in patients with minimal residual disease, the primary endpoint of the study, as both the RINTEGA arm and the control arm were performing on par with each other. In the ACT IV study, RINTEGA performed consistently with prior Phase 2 studies, but the control arm significantly outperformed expectations. Based on this recommendation, Celldex discontinued the study and does not anticipate incurring substantial additional costs related to RINTEGA at this time. The Company is conducting an analysis of the data and plans to present the study at a future scientific/medical meeting or in a peer-reviewed publication. All patients on the RINTEGA arm of the ACT IV study, prior Phase 2 studies and existing compassionate use recipients have been offered ongoing access to RINTEGA on a compassionate use basis.
Glembatumumab vedotin ("glemba"; CDX-011), an antibody-drug conjugate (ADC) targeting gpNMB in multiple cancers

Enrollment continues in the Company’s Phase 2b randomized study (METRIC) of glembatumumab vedotin in patients with metastatic triple negative breast cancers that overexpress gpNMB, a molecule associated with poor outcomes for triple negative breast cancer patients and the target of glembatumumab vedotin. Enrollment is open across the United States, Canada, and Australia and recently opened in the European Union, with the goal of completing enrollment by year-end 2016.
Patient enrollment is complete in the Phase 2 single-agent study of glembatumumab vedotin in metastatic melanoma. The Company is currently amending the protocol to add a second cohort of patients to a glembatumumab vedotin and varlilumab combination arm to assess the potential clinical benefit of the combination and to explore varlilumab’s potential biologic and immunologic effect when combined with an ADC. The Company expects to present data from the single-agent cohort at an appropriate medical meeting in the second half of 2016.
Celldex is also evaluating glembatumumab vedotin in other cancers in which gpNMB is expressed.
Celldex has entered into a collaborative relationship with PrECOG, LLC, which represents a research network established by the Eastern Cooperative Oncology Group (ECOG), and PrECOG, LLC is conducting a Phase 1/2 study in squamous cell lung cancer. This study opened to enrollment in April 2016.
Celldex and the National Cancer Institute (NCI) have entered into a Cooperative Research and Development Agreement (CRADA) under which the NCI is sponsoring two studies of glembatumumab vedotin—one in uveal melanoma and one in pediatric osteosarcoma. Both studies are currently open to enrollment.
Data enhancing the understanding of glembatumumab vedotin’s mechanism of action and further validation of the overexpression of its target, gpNMB, in a wide range of tumor types were presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2016 in April. Using a validated immunohistochemistry (IHC) assay to detect the expression of gpNMB, the Company examined tissues from multiple types of solid tumors and normal tissue. Overexpression of gpNMB in samples of tumor tissue versus normal tissue was found in squamous cell carcinoma of the lung (85%), osteosarcoma (62%), pancreatic cancer (55%), lung adenocarcinoma (45%) and squamous cell carcinoma of the head and neck (40%). These results support the potential broad applicability of gpNMB as a therapeutic target across a wide range of tumor types. In addition, in a preclinical study investigating resistance mechanisms in melanoma, glembatumumab vedotin demonstrated synergies with therapies for BRAF mutated melanoma and overcame phenotypes associated with resistance, suggesting use of glembatumumab vedotin may be particularly effective as a single-agent or in combination in this refractory patient population.
Varlilumab ("varli"; CDX-1127), a fully human monoclonal agonist antibody that binds and activates CD27, a critical co-stimulatory molecule in the immune activation cascade

The Phase 2 portion of the varlilumab and nivolumab (Opdivo) study opened to enrollment in April 2016. The study includes cohorts in advanced non-small cell lung cancer (n=35), colorectal cancer (n=18), ovarian cancer (n=18), head and neck squamous cell carcinoma (n=18), renal cell carcinoma (n=25) and glioblastoma (n=20). The study is being conducted by Celldex under a clinical trial collaboration with Bristol-Myers Squibb Company. The companies are sharing development costs.
Data were presented from the Phase 1 portion of the varlilumab and nivolumab study in a poster at the AACR (Free AACR Whitepaper) Annual Meeting in April 2016. The Phase 1 portion of the study, conducted in patients with solid tumors, has completed enrollment (n=36) and primarily enrolled patients with colorectal and ovarian cancer. The primary objective of the Phase 1 portion of the study was to evaluate the safety and tolerability of the combination.
The combination showed acceptable tolerability and safety across all dose levels without any evidence of increased autoimmunity or inappropriate immune activation.
Marked changes in the tumor microenvironment including increased infiltrating CD8+ T cells and increased PD-L1 expression, which have been shown to correlate with a greater magnitude of treatment effect from checkpoint inhibitors in other clinical studies, were observed.
Additional favorable immune biomarkers, such as increase in inflammatory chemokines and decrease in T regulatory cells, were also noted.
In a subset of patients (n=17) on study who had both pre- and post-tumor biopsies available, preliminary evidence suggest a correlation between biomarker data and stable disease or better in seven of these patients (4 ovarian cancer, 2 colorectal cancer, 1 squamous cell carcinoma of the head and neck).
The Phase 1/2 study of varlilumab and atezolizumab (anti-PDL1) is currently enrolling patients with multiple solid tumors in the dose escalation Phase 1 portion of the study. The Phase 2 portion of the study will be conducted in renal cell carcinoma. This study is being conducted by Celldex under a clinical trial collaboration with Roche. Roche is providing study drug, and Celldex is responsible for conducting and funding the study.
Additional combination studies of varlilumab continue to enroll patients including:
A Phase 1/2 safety and tolerability study examining the combination of varlilumab and sunitinib (Sutent) in patients with metastatic clear cell renal cell carcinoma (CC-RCC).
A Phase 1/2 safety and tolerability study examining the combination of varlilumab and ipilimumab (Yervoy) in patients with stage III or IV metastatic melanoma. In the Phase 2 portion of the study, patients with tumors that express NY-ESO-1 will also receive Celldex’s CDX-1401, an NY-ESO-1-antibody fusion protein for immunotherapy.
CDX-1401, an NY-ESO-1-antibody fusion protein for immunotherapy

As discussed above, a Phase 1/2 study examining the combination of varlilumab and ipilimumab continues to enroll patients with stage III or IV metastatic melanoma. In the Phase 2 portion of the study, patients with tumors that express NY-ESO-1 will also receive CDX-1401.
Celldex continues to support several external collaborations, including an NCI sponsored Phase 2 study of CDX-1401 and CDX-301 for patients with metastatic melanoma, which has completed enrollment. Based on results to date, plans for additional studies are being considered by NCI. Additionally, Roswell Park Cancer Center is conducting an investigator sponsored study evaluating CDX-1401, poly-ICLC (Hiltonol) and the IDO1 inhibitor epacadostat (INCB24360) in patients in remission with ovarian, fallopian tube or primary peritoneal cancer. Patients’ tumors must have expressed NY-ESO-1 or the LAGE-1 antigen to be eligible for the study. Celldex is providing CDX-1401 and poly-ICLC in support of this study.
CDX-301 (recombinant human Flt3L), a potent hematopoietic cytokine that uniquely expands dendritic cells and hematopoietic stem cells

The Company presented early data from the pilot study of CDX-301 alone and in combination with plerixafor (Mozobil) in hematopoietic stem cell transplantation (HSCT) in February at the annual meeting of the American Society for Blood and Marrow Transplantation (ASBMT). Data on three donor/patient pairs from the non-plerixafor treated arm showed that CDX-301 given as a single agent was well tolerated and effective at mobilizing hematopoietic stem cells in healthy donors. The stem cell graft contained notable increases in naïve lymphocytes and plasmacytoid dendritic cells consistent with preclinical data suggesting a possible better outcome. Recipients experienced successful engraftment in an expected time frame. Given that hematopoietic stem cell transplantation is outside of Celldex’s core focus, in an effort to prioritize human and capital resources, the Company has decided not to advance CDX-301 in this particular indication at this time and instead to focus near-term efforts on its potential role in combination immunotherapy.
CDX-301’s potential activity is being explored in a Phase 1/2 study of CDX-301 and poly-ICLC in combination with low-dose radiotherapy in patients with low-grade B-cell lymphomas conducted by the Icahn School of Medicine at Mount Sinai.
First Quarter 2016 Financial Highlights and Updated 2016 Guidance

Cash position: Cash, cash equivalents and marketable securities as of March 31, 2016 were $254.0 million compared to $289.9 million as of December 31, 2015. The decrease was primarily driven by our first quarter cash used in operating activities of approximately $34.9 million. As of March 31, 2016 Celldex had 98.7 million shares outstanding.

Revenues: Total revenue was $1.3 million in the first quarter of 2016, compared to $0.5 million for the comparable period in 2015. The increase in revenue was primarily due to our clinical trial collaboration with Bristol-Myers Squibb, our research and development agreement with Rockefeller University and an increase in grant revenue.

R&D Expenses: Research and development (R&D) expenses were $27.4 million in the first quarter of 2016, compared to $25.1 million for the comparable period in 2015. The increase in R&D expenses was primarily attributable to increased headcount.

G&A Expenses: General and administrative (G&A) expenses were $9.3 million in the first quarter of 2016, compared to $6.1 million for the comparable period in 2015. The increase in G&A expenses was primarily due to higher stock-based compensation of $1.1 million, increased headcount, and RINTEGA and glembatumumab vedotin commercial planning costs.

Net loss: Net loss was $34.7 million, or ($0.35) per share, for the first quarter of 2016, compared to a net loss of $30.2 million, or ($0.33) per share, for the comparable periods in 2015.

Financial guidance: Celldex expects that its cash, cash equivalents and marketable securities will be sufficient to fund our operating expenses and capital expenditure requirements through the first half of 2018.

RINTEGA is a registered trademark of Celldex Therapeutics. Opdivo and Yervoy are registered trademarks of Bristol-Myers Squibb. Sutent is a registered trademark of Pfizer. Mozobil is a registered trademark of sanofi-aventis U.S. LLC. Hiltonol is a registered trademark of Oncovir.

On May 05, 2016 BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) reported financial results for the first quarter ended March 31, 2016 (Press release, BioCryst Pharmaceuticalsa, MAY 5, 2016, View Source [SID:1234511974]).

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"We are currently working through the start-up activities for the APeX-1 trial of BCX7353 for prevention of angioedema attacks in HAE patients and are targeting the end of the year to report results," said Jon P. Stonehouse, President & Chief Executive Officer. "In addition, we are conducting a Phase 1 clinical pharmacology study in healthy volunteers to determine if we are able to meaningfully increase exposure and get to a twice-daily oral dosage form of avoralstat. We expect to report results from this study this summer."

First Quarter Financial Results
For the three months ended March 31, 2016, revenues decreased to $4.8 million from $6.8 million in the first quarter of 2015. The decrease was primarily due to lower collaborative revenue associated with BCX4430 development under the advanced development contract with the Biomedical Advanced Research and Development Authority (BARDA/HHS) awarded in March 2015. In the first quarter of 2015, the Company recorded $537,000 in RAPIVAB revenue, representing drug sold under the sell-through revenue recognition methodology. No RAPIVAB revenue was received in the first quarter 2016 as RAPIVAB commercialization is now being handled by Seqirus UK Limited (Seqirus).

Research and Development (R&D) expenses for the first quarter of 2016 increased to $20.6 million from $17.1 million in the first quarter of 2015. The R&D expense increase in 2016 resulted primarily from higher development costs associated with the Company’s hereditary angioedema (HAE) programs, as well as ongoing post approval clinical trials of RAPIVAB in both pediatric and elderly/high risk influenza patient populations.

General and administrative (G&A) expenses for the first quarter of 2016 decreased to $3.2 million compared to $4 .1 million for the first quarter of 2015. G&A expenses decreased in 2016 due to a significant reduction in unrestricted grants, as well as the elimination of marketing and commercial consulting expense in 2016, as RAPIVAB is now being commercialized by Seqirus.

Interest expense, which is primarily related to non-recourse notes, was $1.5 million in the first quarter of 2016 and $1.3 million in the first quarter of 2015. Also, a $2.8 million mark-to-market loss on the Company’s foreign currency hedge was recognized in the first quarter of 2016, as compared to a $464,000 mark-to-market gain in the first quarter of 2015. The change in the dollar/yen exchange rate between the quarters resulted in a $3.2 million increase to the Company’s net loss for the first quarter of 2016 as compared to 2015. These gains and losses result from periodic changes in the U.S. dollar/Japanese yen exchange rate and the related mark-to-market valuation of our underlying hedge arrangement.

The net loss for the first quarter of 2016 was $22.8 million, or $0.31 per share, compared to a net loss of $15.2 million, or $0.21 per share, for the first quarter 2015.

Cash, cash equivalents and investments totaled $78.9 million at March 31, 2016. Net operating cash use for the first quarter of 2016 was $22.4 million, as compared to $3.8 million for the first quarter of 2015. The first quarter of 2016 is expected to be the largest cash consumption quarter of the four quarters in 2016, and BioCryst expects to remain within previously issued cash use guidance.

Clinical Development Update & Outlook
BioCryst expects to report results of the APeX-1 clinical trial of the once-daily, second generation HAE compound, BCX7353, for prevention of angioedema attacks in HAE patients by year end.

A Phase 1 clinical pharmacology trial testing multiple avoralstat formulations is ongoing in healthy subjects with the aim to develop a twice-daily dosage for HAE patients. Pharmacokinetic results are expected this summer.

BioCryst has completed the Phase 1 clinical trial of single and multiple ascending doses of its broad spectrum antiviral BCX4430 administered by intramuscular (i.m.) injection in healthy volunteers. BCX4430 by i.m. injection was generally safe and well tolerated through doses up to 10mg/kg daily for seven days, the maximum dose planned in the trial.

Nonclinical experiments are continuing with BCX4430 in a model of Zika virus infection in interferon-receptor-deficient mice. Studies conducted to date have shown improved survival with dosing delayed up to 7 days after virus challenge, reduction in viral titer in the blood, and development of protective immunity in surviving animals. This research has been conducted in collaboration with Utah State University and NIAID.

In January, BioCryst submitted a New Drug Submission (NDS) for RAPIVAB in Canada, seeking approval for the treatment of acute uncomplicated influenza in adult patients.

Financial Outlook for 2016
Based upon development plans and our awarded government contracts, BioCryst expects its 2016 net operating cash use to be in the range of $55 to $75 million, and its 2016 operating expenses to be in the range of $78 to $98 million. Our operating expense range excludes equity-based compensation expense due to the difficulty in reliably projecting this expense, as it is impacted by the volatility and price of the Company’s stock, as well as by the vesting of the Company’s outstanding performance-based stock options.

On May 05, 2016 Athersys, Inc. (Nasdaq:ATHX) reported its financial results for the three months ended March 31, 2016 (Press release, Athersys, MAY 5, 2016, View Source [SID:1234511972]).

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Highlights of the first quarter of 2016 and recent events include:

Announced positive one-year follow-up results from the Phase 2 study of MultiStem cell therapy to treat ischemic stroke – demonstrating progressive improvements and significantly higher rate of excellent outcomes at one year for MultiStem-treated patients – with greater benefits for patients receiving MultiStem treatment within 36 hours of the stroke;
Established collaboration with HEALIOS K.K. ("Healios") to develop MultiStem cell therapy for stroke in Japan, which included an upfront payment of $15 million, additional potential milestone payments aggregating up to $225 million, double-digit royalties on product sales and an option for Healios to expand the collaboration to include acute respiratory distress syndrome ("ARDS") and another indication with a $10 million expansion payment and additional associated milestone payments and royalties;
Advanced preparations for next stroke trials, including working with Healios to reach agreement with the Japanese Pharmaceuticals and Medical Devices Agency ("PMDA") on study design to potentially clear the way for a Japanese investigational new drug ("J-IND") filing and subsequent trial launch, and engaging with other regulators about requirements for the Company’s planned international stroke study;
Continued enrollment of Phase 2a study evaluating administration of MultiStem therapy to ARDS patients, and Phase 2 acute myocardial infarction ("AMI") study, evaluating and taking measures to accelerate enrollment;
Recorded revenues of $15.5 million for quarter ended March 31, 2016, reflecting the recognition of the $15.0 million license fee payment from Healios, and net income of $4.8 million; and
Ended the quarter with $30.4 million in cash and cash equivalents.
"We remain very excited about our ischemic stroke program," stated Dr. Gil Van Bokkelen, Chairman & CEO at Athersys. "As we have presented at several conferences, we have seen that MultiStem treatment has the potential to help ischemic stroke victims, especially those who can be treated within 36 hours following the stroke. We were especially pleased with the one-year follow-up results, which showed continued and significant functional improvement with MultiStem treatment. As a result, we are moving forward diligently with clinical development in Japan with our partner Healios, and with planning for a corresponding study in the United States and Europe, focused on MultiStem treatment within 36 hours of the stroke.

"Our partnership with Healios is off to a strong start," continued Dr. Van Bokkelen. "Building from our previous engagement with the PMDA and with our support, Healios has reached general agreement with the PMDA about trial design and requirements. Healios is preparing its J-IND filing and planning for the subsequent launch of its planned study, and we are engaged in manufacturing to support their clinical product requirements. Based on discussions with the PMDA, we believe that a successful trial in Japan could make contingent, or even full, approval possible, utilizing Japan’s progressive regulations for the development and approval of regenerative medicine products.

"We are also fully engaged in planning and preparing for an international ischemic stroke study focused in the United States and several European countries. Upcoming discussions with the U.S. Food and Drug Administration and other regulators will help us refine and finalize the trial design and complete trial preparations," noted Dr. Van Bokkelen.

"We are also enrolling our two grant-supported Phase 2 trials, in AMI and ARDS, although progress has been slower than we have anticipated," commented Dr. Van Bokkelen. "We have undertaken a number of actions to accelerate enrollment, including adding clinical sites. We believe that MultiStem cell therapy is well-suited to treat these acute conditions based on our preclinical and clinical experience to date, and we are motivated to move these studies forward expeditiously.

"We continue to focus on other important areas, including actively exploring partnering opportunities around multiple programs. Finally, we have a substantial effort underway in manufacturing and process development focused, first, on supplying our planned clinical studies, and second, on advancing our manufacturing platform and related capabilities to support high-volume, low-cost production important to commercialization," concluded Dr. Van Bokkelen.

First Quarter Results

For the three months ended March 31, 2016, total revenues were $15.5 million compared to $0.7 million in the same period in 2015, reflecting the recognition of $15.0 million in contract revenue from our Healios collaboration in the first quarter of 2016. Grant revenue was $0.3 million less in the first quarter of 2016 compared to the same period in 2015, and grant revenues may fluctuate from period to period based on the timing of grant-related activities and the award and expiration of new grants.

Research and development expenses increased to $6.7 million in the 2016 first quarter from $5.7 million in the 2015 first quarter, primarily due to increased clinical and preclinical development costs, including process development activities to support manufacturing. General and administrative expenses were relatively consistent at $2.0 million and $1.9 million for the three months ended March 31, 2016 and 2015, respectively.

We recognized net income for the three months ended March 31, 2016 of $4.8 million compared to net loss of $12.5 million for the same period in 2015. The $17.2 million net variance includes the impact of the $15.0 million Healios license revenue, the $0.3 million decrease in grant revenues, the $1.1 million increase in combined R&D and G&A expenses, a $3.4 million decrease in non-cash expense from the change in the fair value of our warrant liabilities, and a $0.2 million increase in net other income. Cash provided in operating activities was $7.3 million during the 2016 first quarter (reflecting $14.8 million of cash received from Healios), compared to $1.1 million in the 2015 first quarter (including $8.0 million of cash received from our former collaborator, Chugai Pharmaceutical Co., Ltd). As of March 31, 2016, we had $30.4 million in cash and cash equivalents, compared to $23.0 million at December 31, 2015.

On May 05, 2016 Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the fields of cancer metabolism and rare genetic metabolic disorders, reported business highlights and financial results for the first quarter ended March 31, 2016 (Press release, Agios Pharmaceuticals, MAY 5, 2016, View Source;p=RssLanding&cat=news&id=2165400 [SID:1234511970]).

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"We have been focused on executing against the important milestones we laid out in January, and I’m proud of the progress across both our cancer and rare genetic disorders programs to date," said David Schenkein, M.D., chief executive officer at Agios. "Notably, enrollment is complete ahead of schedule for the Phase 2 expansion cohort for the Phase 1/2 study of AG-221 in advanced AML. This achievement is crucial to getting this potential new therapy to patients as quickly as possible. Additionally, we are pleased that the first data from our Phase 2 DRIVE PK study of AG-348 will be presented at EHA (Free EHA Whitepaper) this June, along with the first data from the Phase 1 study of AG-519 in healthy volunteers."

FIRST QUARTER 2016 HIGHLIGHTS & RECENT PROGRESS

PKR Activators:

An abstract for the first data from DRIVE PK, a global Phase 2, open-label safety and efficacy trial of AG-348 in adult, transfusion-independent patients with pyruvate kinase (PK) deficiency has been accepted for presentation at the 21st Congress of the European Hematology Association (EHA) (Free EHA Whitepaper) in June 2016. An abstract for preclinical data for AG-348 in beta-thalassemia has also been accepted for presentation at EHA (Free EHA Whitepaper).
Three abstracts on AG-519, including from the Phase 1 study in healthy volunteers and preclinical findings on the molecule, have been accepted for presentation at EHA (Free EHA Whitepaper).
Agios provided the following updates on its clinical development programs in collaboration with Celgene:

IDH Mutant Inhibitors in Hematologic Malignancies:

Completed enrollment of the Phase 2 expansion cohort for the Phase 1/2 study of AG-221 in patients with R/R AML in May 2016
Initiated a Phase 1/2 frontline combination study of AG-221 or AG-120 with VIDAZA (azacitidine) in newly diagnosed AML patients not eligible for intensive chemotherapy in March 2016
Received EMA Orphan Drug Designation for AG-221 for the treatment of AML in April 2016
Cancer Metabolism Research:

In April, Agios published preclinical findings from its program focused on MTAP (methylthioadenosine phosphorylase) deleted cancers in the peer-reviewed journal Cell Reports
2016 EXPECTED MILESTONES IN CANCER METABOLISM PROGRAMS

IDH Mutant Inhibitors in Hematologic Malignancies:

Complete enrollment of the 125-patient expansion cohort for the Phase 1 study of AG-120 in patients with R/R AML in the second half of 2016
Initiate a global, registration-enabling Phase 3 study of AG-120 in frontline AML patients with an IDH1 mutation in the second half of 2016
Initiate an expansion arm in high-risk myelodysplastic syndrome patients for AG-221 in 2016
Continue to enroll patients in the following ongoing clinical trials:
Phase 3 IDHENTIFY study of AG-221 vs. standard of care chemotherapy in R/R AML
Phase 1b frontline combination study of AG-221 or AG-120 with standard-of-care intensive chemotherapy in AML
Phase 1/2 frontline combination study of AG-221 or AG-120 with VIDAZA in AML
Phase 1 dose-escalation and expansion study of AG-881 in IDH mutant positive hematologic malignancies
IDH Mutant Inhibitors in Solid Tumors:

Present data from the expansion phase of the ongoing Phase 1 study of AG-120 in advanced IDH1 mutant positive low-grade glioma in the second half of 2016
Initiate a randomized Phase 2 study of AG-120 in IDH1 mutant positive cholangiocarcinoma in the second half of 2016
Continue to enroll patients in the following ongoing clinical trials:
Expansion phase of the ongoing Phase 1 study of AG-120 in advanced IDH1 mutant positive solid tumors
Phase 1 dose-escalation and expansion study of AG-881 in IDH mutant positive solid tumors
Cancer Metabolism Research:

Initiate preclinical development activities for the first molecule in the MTAP program in 2016
2016 EXPECTED MILESTONES IN RARE GENETIC METABOLIC DISORDERS PROGRAMS

Present new findings from the Natural History Study of PK deficiency being conducted with Boston Children’s Hospital in the second half of 2016
Outline the clinical development plans for Agios’ PKR activators in beta-thalassemia in the second half of 2016
FIRST QUARTER 2016 FINANCIAL RESULTS

Cash, cash equivalents and marketable securities as of March 31, 2016 were $355.8 million, compared to $375.9 million as of December 31, 2015. The decrease was driven by cash expenditures to fund operating activities of $54.1 million, which was offset by funding of $35.1 million from Celgene during the quarter ended March 31, 2016 related to our collaboration agreements.

Collaboration revenue was $31.3 million for the quarter ended March 31, 2016, compared to $34.2 million for the comparable period in 2015. In the first quarter of 2016, the Company received and recognized as revenue $25.0 million related to a substantive clinical development milestone for the AG-221 program.

Research and development (R&D) expense was $44.0 million, including $5.5 million of stock-based compensation expense, for the quarter ended March 31, 2016, compared to $32.4 million, including $2.6 million in stock-based compensation expense, for the quarter ended March 31, 2015. 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. Celgene is responsible for all development costs for AG-221 and certain development costs for AG-120 and AG-881 and reimburses the company for development costs incurred for these investigational medicines.

General and administrative (G&A) expense was $10.8 million, including $3.6 million of stock-based compensation expense, for the quarter ended March 31, 2016, compared to $7.0 million, including $2.4 million of stock-based compensation expense, for the quarter ended March 31, 2015. The increase in G&A expense was largely due to increased headcount and other professional expenses to support growing operations.

Net loss for the quarter ended March 31, 2016 was $23.2 million, compared to a net loss of $5.0 million for the quarter ended March 31, 2015.

On May 5, 2016 Acceleron Pharma Inc. (NASDAQ:XLRN), a clinical stage biopharmaceutical company focused on the discovery and development of novel therapeutic candidates that engage the body’s ability to rebuild and repair its own cells and tissues, reported a corporate update and reported financial results for the first quarter ended March 31, 2016 (Press release, Acceleron Pharma, MAY 5, 2016, View Source [SID:1234511968]).

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"In the first quarter, we continued to build momentum across our entire clinical pipeline, including the start of patient recruitment into our Phase 3 trials of luspatercept in MDS and beta-thalassemia with our partner Celgene," said John Knopf, Ph.D., Chief Executive Officer of Acceleron. "Receipt of the $15 million luspatercept milestone payment and the $140.3 million in net proceeds from our equity follow-on offering in January secured our ability to accelerate the pace of development of our wholly owned programs. To that end, we made significant progress toward the planned Phase 2 trial initiation of ACE-083 in facioscapulohumeral muscular dystrophy in the second half of 2016 and advancing our preclinical work in the area of fibrosis as well as the generation of new product candidates with our IntelliTrap platform."

FIRST QUARTER 2016 HIGHLIGHTS

Development Programs

Hematology

Acceleron received a $15 million milestone payment from collaboration partner Celgene related to the initiation of the luspatercept Phase 3 clinical trials. Luspatercept is being studied in two Phase 3 clinical trials in patients with myelodysplastic syndromes (MDS) or beta-thalassemia, both rare hematologic diseases.
Enrollment is ongoing for both of the Celgene-partnered Phase 3 studies of luspatercept. The MEDALIST trial in MDS is evaluating the efficacy and safety of luspatercept versus placebo in patients with anemia due to very low, low, or intermediate-risk MDS with ring sideroblasts (≥ 15%) who require red blood cell transfusions. The BELIEVE trial in beta-thalassemia is evaluating the efficacy and safety of luspatercept versus placebo in adults who require regular red blood cell transfusions. Luspatercept increases hemoglobin levels and is being developed to help patients reduce or eliminate their need for red blood cell (RBC) transfusions. Luspatercept has been granted Fast Track Designation from the FDA in both indications.
Ongoing Phase 2 trials of luspatercept in MDS and beta-thalassemia continue to generate longer-term safety and efficacy data. The Company expects to report updated results for these trials at the 21st Congress of the European Hematology Association (EHA) (Free EHA Whitepaper) in June 2016.
Enrollment is underway in two additional Phase 2 patient cohorts evaluating luspatercept in low- or intermediate-risk MDS patients who are ring sideroblast negative or are eligible but have not yet received an erythropoietin-stimulating agent (ESA). Initial data from these additional MDS patient cohorts are expected by year-end.
Musculoskeletal Diseases

ACE-083 advancing toward the initiation of a Phase 2 trial in facioscapulohumeral muscular dystrophy (FSHD), a neuromuscular disorder, in the second half of 2016. ACE-083 is a locally acting agent designed to increase muscle mass and strength in the muscles in which it is administered.
We continue to make progress with ACE-2494 toward IND submission. ACE-2494, Acceleron’s first IntelliTrap molecule, is a systemic therapeutic designed to increase muscle mass and strength across a range of musculoskeletal diseases. Preclinical data in mice presented in 2015 showed that after 4 weeks of treatment, ACE-2494 generated substantial dose-dependent mean increases in muscle mass: 41% in rectus femoris, 53% in gastrocnemius, and 87% in pectoralis.
Oncology

Enrollment continues in Part 2 of the Phase 2 DART study, a randomized, double-blind study of dalantercept plus axitinib, compared to placebo plus axitinib in patients with advanced renal cell carcinoma. Dalantercept has been granted FDA Fast Track Designation for this indication.
Nephrology

Acceleron and Celgene assessing the opportunity for the development of sotatercept in the pre-dialysis chronic kidney disease (CKD) setting, and expect to provide an update in the second half of the year.
Corporate Updates

Raised $140.3 million of net proceeds in a follow-on public offering of common stock in January 2016 to advance wholly owned programs.
Financial Results

Cash position – Cash, cash equivalents and investments as of March 31, 2016 were $278.7 million. Net cash provided by operating activities in first quarter 2016 was $0.9 million. As of December 31, 2015 the Company had cash, cash equivalents and investments of $136.0 million. We believe that our existing cash, cash equivalents and investments, including the net proceeds of $140.3 million from our January 2016 common stock offering and the receipt of the $15.0 million Celgene milestone, will be sufficient to fund our projected operating requirements into the second half of 2019.
Revenue – Collaboration revenue for the first quarter was $18.2 million. License and milestone revenue was $15.1 million and includes the $15 million milestone payment received from Celgene related to luspatercept. Cost sharing reimbursement revenue from our Celgene partnership was $3.1 million and is related to expenses incurred by the Company in support of our partnered programs.
Costs and expenses – Total costs and expenses for the first quarter were $22.1 million. This includes R&D expenses of $16.2 million and G&A expenses of $5.9 million.
Other income, net – Other income for the first quarter was $9.0 million and includes an $8.7 million, non-cash, gain on marking our common stock warrant liability to market.
Net income – The Company posted a net income for the first quarter ended March 31, 2016 of $5.1 million.