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.

On May 05, 2016 Geron Corporation (Nasdaq:GERN) reported financial results for the first quarter ended March 31, 2016 and recent events (Press release, Geron, MAY 5, 2016, View Source;p=RssLanding&cat=news&id=2165745 [SID:1234512001]).

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First Quarter 2016 Results

For the first quarter of 2016, the company reported operating revenues of $749,000 and operating expenses of $9.8 million compared to $537,000 and $10.0 million, respectively, for the comparable 2015 period. Revenues for the first quarter of 2016 and 2015 included royalty and license fee revenues under various non-imetelstat agreements. Operating expenses in the first quarter of 2015 included restructuring charges of $406,000 in connection with the company’s organizational resizing announced in March 2015. Net loss for the first quarter of 2016 was $8.8 million, or $0.06 per share, compared to $9.3 million, or $0.06 per share, for the comparable 2015 period. The company ended the first quarter of 2016 with $141.9 million in cash and investments.

Research and development expenses for the first quarter of 2016 and 2015 were $5.0 million for each period. Research and development expenses for 2016 primarily reflect the net result of higher costs for the company’s proportionate share of clinical development expenses under the imetelstat collaboration with Janssen Biotech, Inc. (Janssen), partially offset by lower personnel related expenses as a result of the 2015 organizational resizing. The company expects research and development expenses to increase during the remainder of the year as the clinical development of imetelstat continues in collaboration with Janssen.

General and administrative expenses for the first quarter of 2016 were $4.8 million compared to $4.6 million for the comparable 2015 period. The increase in general and administrative expenses primarily reflects the net result of higher non-cash stock-based compensation expense, partially offset by lower personnel related costs due to the 2015 organizational resizing.

Interest and other income for the first quarter of 2016 was $256,000 compared to $149,000 for the comparable 2015 period. The increase in interest and other income in 2016 compared to 2015 primarily reflects higher yields on the company’s marketable securities portfolio.

Recent Company Events

Clinical Development by Janssen

In January 2016, the first patient was dosed in a Phase 2/3 clinical trial to evaluate imetelstat in patients with myelodysplastic syndromes (MDS). The trial, referred to as IMergeTM, will evaluate imetelstat in transfusion dependent patients with International Prognostic Scoring System (IPSS) Low or Intermediate-1 risk MDS who have relapsed after or are refractory to prior treatment with an erythropoiesis stimulating agent (ESA).

This is the second clinical study to be initiated under the terms of the exclusive worldwide imetelstat license and collaboration agreement between Geron and Janssen. Further information about the trial can be found at View Source

Presentations and Publications

Clinical data on imetelstat safety and efficacy from patients with a form of MDS known as refractory anemia with ring sideroblasts (MDS-RARS) enrolled as part of the Mayo Clinic Pilot Study were published online in the Blood Cancer Journal in March. The full text paper is available online at View Source

The data, previously presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting in December 2015, included nine patients enrolled in the study cohort, classified as having either IPSS intermediate-1 or intermediate-2 risk disease. Six of nine (66.7%) patients had prior treatment with ESAs. Three of the eight (37.5%) patients who were dependent on red blood cell transfusions at study entry became transfusion independent, defined as not requiring transfusions for at least eight weeks. The median duration of transfusion independence was 28 weeks (range: nine weeks to 37 weeks).

Two poster presentations by Janssen and academic collaborators describing non-clinical data on imetelstat were made at the 2016 annual meeting of the American Association for Cancer Research (AACR) (Free AACR Whitepaper) in April:

Impact of hypomethylating agents on hTERT expression and synergistic effect in combination with imetelstat, a telomerase inhibitor, in AML cell lines (Abstract #2731)

Data presented described non-clinical results that treating acute myeloid leukemia (AML) cell lines with imetelstat enhanced the effects of agents currently used for the treatment of AML. These data extend the rationale from prior non-clinical studies for the potential use of imetelstat in hematologic myeloid malignancies, such as AML, including in combination with standard therapies.

Myelosuppression in patients treated with the telomerase inhibitor imetelstat is not mediated through activation of toll-like receptors (Abstract #2732)

Data presented described results from non-clinical studies that provide further evidence for potential on-target mechanisms of telomerase inhibition by imetelstat underlying the reduction in platelets observed in previously conducted imetelstat clinical trials.

On May 05, 2016 OncoMed Pharmaceuticals, Inc. (Nasdaq:OMED), a clinical-stage company developing novel anti-cancer stem cell (CSC) and immuno-oncology therapeutics, reported first quarter financial results (Press release, OncoMed, MAY 5, 2016, View Source [SID:1234512033]). The company also highlighted data presentations related to six oncology drug candidates, including robust preclinical anti-tumor activity data presented at the AACR (Free AACR Whitepaper) Annual Meeting for its wholly owned GITRL-Fc candidate and upcoming presentations at the ASCO (Free ASCO Whitepaper) Annual Meeting from clinical trials of vantictumab, ipafricept, demcizumab and tarextumab. As of March 31, 2016, cash, cash equivalents and short-term investments totaled $193.5 million.

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"Recent highlights include the presentation of new and compelling data at the AACR (Free AACR Whitepaper) Annual Meeting for our proprietary GITRL-Fc immuno-oncology therapeutic. We observed highly differentiated preclinical data including robust single-agent activity for GITRL-Fc compared to an alternate GITR agonist antibody strategy. We look forward to filing INDs on two distinct IO agents, GITRL-Fc and an undisclosed agent, termed ‘IO#2′, with the first of these IND filings by year-end." said Paul J. Hastings, Chairman and Chief Executive Officer. "Looking ahead, we have a steady stream of data presentations planned, starting with several at the ASCO (Free ASCO Whitepaper) Annual Meeting for ongoing clinical trials, including new data from two of our Phase 1b combination studies for Wnt inhibitors vantictumab and ipafricept, as well as updated Phase 1b survival data for demcizumab in NSCLC."

Corporate Update and Recent Highlights

Initiated a Phase 1b clinical trial of demcizumab (anti-DLL4, OMP-21M18) plus anti-PD-1 (pembrolizumab) in solid tumor patients

Presented five posters at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting:
Preclinical data comparing efficacy and immune system activation of OncoMed’s wholly owned GITRL-Fc to GITR agonist antibodies and evaluating GITRL-Fc alone and in combination with anti-PDL1 and anti-PD1
Predictive biomarker assays for anti-RSPO3 (OMP-131R10) in solid tumors and vantictumab (anti-Fzd7, OMP-18R5) in pancreatic cancer
Preclinical data detailing anti-DLL4’s activity in non-small cell lung cancer, including immunomodulatory mechanism

Announced acceptance of several clinical abstracts for presentation at the upcoming American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2016 Annual Meeting being held June 3-7, 2016 in Chicago, IL. Presentations include:
The first clinical data from Phase 1b trials of the company’s Wnt pathway inhibitors, vantictumab and ipafricept (FZD8-Fc, OMP-54F28), in combination therapy in breast cancer and ovarian cancer, respectively
Updated survival data from OncoMed’s Phase 1b clinical trials of demcizumab (OMP-21M18, anti-DLL4) in non-small cell lung cancer (NSCLC) and of tarextumab (anti-Notch2/3, OMP-59R5) in small cell lung cancer

First Quarter 2016 Financial Results
Cash, cash equivalents and short-term investments totaled $193.5 million as of March 31, 2016, compared to $157.3 million as of December 31, 2015. During the quarter, OncoMed received a $70 million payment from Celgene related to the demcizumab safety milestone achieved in the fourth quarter of 2015. This milestone was recorded as deferred revenue and will be amortized over the performance period.

Revenues for the first quarter 2016 totaled $6.4 million, as compared to $9.7 million in the first quarter of 2015. The decrease in revenue over the same period in 2015 was primarily due to achievement in 2015 of a $5.0 million milestone from GlaxoSmithKline related to brontictuzumab, partially offset by increased amortization of revenue from Celgene related to the demcizumab safety milestone.

Research and development (R&D) expenses for the first quarter 2016 were $28.4 million compared with $19.4 million for the same period in 2015. Higher R&D expenditures during the first quarter 2016 compared to the first quarter of 2015 were attributable to increased Phase 2 costs for the demcizumab and tarextumab programs as well as IND-enabling manufacturing and toxicology costs for the GITRL-Fc and IO#2 programs.

General and administrative (G&A) expenses for the quarter ended March 31, 2016 were $5.2 million, compared to $4.8 million for the same period in 2015. Increased costs during the first quarter 2016 were due to higher employee-related costs including stock-based compensation expenses.

Net loss for the first quarter 2016 was $27.2 million ($0.90 per share), compared to $14.5 million ($0.49 per share) for the same period of 2015. The change in net loss from the prior year quarter was due to an increase in operational expenses, primarily research and development costs, and lower collaboration revenues.

2016 Financial Guidance
OncoMed reiterated financial guidance for the full year 2016. Based on its current plans and expectations, OncoMed anticipates:

Cash expenses in the range of approximately $110-$120 million
2016 year-end cash balance of more than $100 million, without taking into account potential future milestones or payments from partners
Existing cash is anticipated to fund company operations through the first quarter of 2018, without taking into account potential future milestones or payments from partners

Potential milestone and opt-in payments from partners GSK, Bayer and Celgene over the course of 2016, 2017, and 2018 total over $270 million.

On May 5, 2016 Novocure (NASDAQ: NVCR) reported the availability of a new online resource to raise awareness of glioblastoma (GBM) and share stories of inspiration with the GBM community (Press release, NovoCure, MAY 5, 2016, View Source [SID:1234512063]). An interactive exhibit featuring patients, caregivers, doctors, nurses and advocates will also be displayed within the coming months.

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Approximately 12,500 GBM tumors, or tumors that may transform to GBM, are diagnosed in the U.S. each year. GBM can spread quickly and is one of the deadliest forms of cancer.

The website, GBMcommunity.com, features a mosaic of videos and images from the GBM community offering messages of hope and inspiration. The website also provides an overview of GBM and links to resources and advocacy groups.

"Novocure actively engaged members of the community to determine the resources they felt they needed most," said Pritesh Shah, Senior Vice President, Americas. "This new website and exhibit were born from the GBM community, and created with assets provided by the community. No one can speak to the experiences, hopes and needs of those affected by GBM better than they can. We are proud to bring this important resource to the GBM community, and thank all who have provided their words of support and images to this project, and encourage others to do the same. Only by making our collective voices heard can we raise awareness of this disease and deliver increased support."
About Glioblastoma Multiforme

Glioblastoma, also called glioblastoma multiforme, or GBM, is a type of primary brain cancer. This means that GBM tumors begin in the brain, rather than traveling to the brain from other parts of the body, such as the lungs or breasts. GBM is the most common type of primary brain cancer in adults. It is more likely to appear in adults than children and to affect men than women.

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.