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.

On May 5, 2016 Heron Therapeutics, Inc. (NASDAQ:HRTX), a biotechnology company focused on improving the lives of patients by developing best-in-class medicine that address major unmet medical needs, reported first quarter 2016 financial results and highlighted recent corporate progress (Press release, Heron Therapeutics, MAY 5, 2016, View Source;p=RssLanding&cat=news&id=2165669 [SID:1234512002]).

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Recent Corporate Progress:
On April 18, 2016, Heron announced that the U.S. Food and Drug Administration (FDA) has indicated that there are no substantive deficiencies in the New Drug Application (NDA) for SUSTOL (granisetron) Injection, extended release, Heron’s lead product candidate for the prevention of chemotherapy-induced nausea and vomiting (CINV) in cancer patients, and has begun labeling discussions with the Company.

Heron has continued to implement a broad-based Phase 2 clinical program of HTX-011, its lead product candidate for the prevention of post-operative pain. In February 2016, Heron initiated a Phase 2 clinical trial of HTX-011 in patients undergoing abdominoplasty, and in April 2016, Heron initiated a Phase 2 clinical trial of HTX-011 in patients undergoing bunionectomy. In addition, the Company continues to enroll patients in an ongoing Phase 2 clinical trial in patients undergoing inguinal hernia repair.
"We continue to work closely with the FDA on our NDA for SUSTOL and look forward to bringing this important therapeutic to patients suffering from CINV," commented Barry D. Quart, Pharm.D., Chief Executive Officer of Heron Therapeutics. "We also continue to make important progress in our HTX-011 post-operative pain program, including the recent initiation of our fourth Phase 2 study of HTX-011. We look forward to reporting top-line data from our ongoing studies, beginning with data from our study in inguinal hernia repair, which we expect late in this quarter."

Results of Operations
As of March 31, 2016, Heron had approximately $100.4 million in cash, cash equivalents and short-term investments, compared to $131.2 million as of December 31, 2015. The net decrease in cash, cash equivalents and short-term investments was primarily due to net cash used in operating activities in the first quarter of 2016. Based on current operating plans and projections, Heron believes that its current working capital is sufficient to fund operations through 2016.

Heron’s net cash used for operating activities for the quarter ended March 31, 2016 was $32.4 million compared to net cash used for operating activities of $19.7 million for the same period in 2015.

Heron’s net loss for the quarter ended March 31, 2016 was $33.4 million, or $0.92 per share, compared to a net loss of $20.6 million, or $0.70 per share, for the same period in 2015.

The increase in net cash used for operating activities and net loss in the first quarter of 2016 as compared to the same period in 2015 were primarily due to costs incurred in preparation for the commercial launch of SUSTOL, as well as clinical and manufacturing costs related to our Phase 2 clinical studies for HTX-011 and costs associated with the development of HTX-019.

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 Insmed Incorporated (Nasdaq:INSM), a global biopharmaceutical company focused on the unmet needs of patients with rare diseases, reported financial results for the quarter ended March 31, 2016 (Press release, Insmed, MAY 5, 2016, View Source [SID:1234512005]).

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Business Update

Global Phase 3 CONVERT study advancing. Patient enrollment continues to proceed on track in the company’s global phase 3 study of ARIKAYCE (liposomal amikacin for inhalation or LAI) in nontuberculous mycobacteria (NTM) lung disease caused by Mycobacterium avium complex (MAC) (CONVERT or INS-212 study). The CONVERT study is taking place in 16 countries and at more than 130 sites. The company continues to expect to achieve its enrollment objective in the second half of 2016.
EMA regulatory review of ARIKAYCE progressing. The company remains on track with previous guidance, having submitted its responses to the European Medicine Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) 180-day list of outstanding issues related to the company’s Marketing Authorization Application (MAA) for ARIKAYCE. Insmed expects to participate in an oral explanation meeting in the second quarter of 2016 and the CHMP to render an opinion on its MAA around the middle of 2016.

Data accepted for presentation at ATS 2016. Three ARIKAYCE-related abstracts and one treprostinil prodrug abstract have been accepted for presentation at the American Thoracic Society (ATS) 2016 International Conference taking place May 13-18 in San Francisco. The ATS presentations include (i) one-year follow-up data from the phase 2 112 study, (ii) lung distribution and retention data from a scintigraphy study in patients with NTM lung disease, (iii) a preclinical study of ARIKAYCE, and (iv) a preclinical study of a variety of treprostinil prodrugs.

Phase 1 clinical study of INS1009 submitted for presentation. Insmed has completed a phase 1 study of INS1009 and submitted the results for presentation at a future medical meeting. This first-in-human study of INS1009 was designed to determine the maximum-tolerated dose of a single dose of INS1009 and to characterize the pharmacokinetic profile of free treprostinil and INS1009 in healthy volunteers. INS1009 is one of the company’s nebulized treprostinil prodrugs, which may offer a differentiated product profile with therapeutic potential in rare pulmonary disorders such as pulmonary arterial hypertension (PAH), idiopathic pulmonary fibrosis (IPF), sarcoidosis, and severe refractory asthma.

Canadian patent strengthens global patent portfolio. The Canadian Intellectual Property Office issued patent no. 2,838,111, which covers pharmaceutical formulations that include mixtures of liposomal quinolone antibiotics, together with free, unencapsulated quinolone antibiotics, such as ciprofloxacin. The patent also covers the use of such formulations for the treatment of various pulmonary disorders, for example, in bronchiectasis patients. The Canadian patent complements ARIKAYCE’s global intellectual property estate. Counterpart patent applications to this Canadian patent are pending in other countries.

"2016 is off to a solid start with all of our clinical, regulatory, and commercial-readiness activities remaining on track with our previously stated timelines," said Will Lewis, president and chief executive officer of Insmed. "Our top corporate priority is our global phase 3 CONVERT study and we look forward to achieving our patient enrollment objective later this year. In parallel with our clinical activities, our team is advancing the regulatory process for ARIKAYCE in Europe. For INS1009, we completed the phase 1 study and submitted the results for presentation at an international respiratory congress in the third quarter. Lastly, our talented team of scientists remain focused on advancing a number of preclinical programs and identifying our next candidates for clinical development."

First Quarter Financial Results

For the first quarter of 2016, Insmed posted a net loss of $33.5 million, or $0.54 per share, compared with a net loss of $27.4 million, or $0.55 per share, for the first quarter of 2015.

Research and development expenses were $20.5 million for the first quarter of 2016, compared with $17.2 million for the first quarter of 2015. The increase was primarily due to the advancement of the company’s global phase 3 CONVERT study of ARIKAYCE in NTM lung disease.

General and administrative expenses for the first quarter of 2016 were $12.5 million, compared with $9.5 million for the first quarter of 2015. The increase was primarily related to pre-commercial activities in Europe, namely the buildout of the company’s infrastructure and NTM disease awareness activities, as well as an increase in headcount and related expenses.

Balance Sheet Highlights and Cash Guidance

As of March 31, 2016, Insmed had cash and cash equivalents of $253 million. Excluding depreciation and stock-based compensation expense, the company’s cash operating expenses for the quarter ended March 31, 2016 were $28 million. Insmed ended the first quarter of 2016 with $25 million in debt and working capital of $233 million.

The company is investing in the following activities in 2016: (i) clinical development of ARIKAYCE, (ii) regulatory and pre-commercial initiatives for ARIKAYCE, and (iii) preclinical and clinical activities for its earlier-stage pipeline. Insmed continues to expect its cash-based operating expenses for the first half of 2016 to be in the range of $58 to $68 million.