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10-Q – Quarterly report [Sections 13 or 15(d)]

(Filing, 10-Q, Epizyme, NOV 9, 2015, View Source [SID:1234508108])

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8-K – Current report

On November 9, 2015 Calithera Biosciences, Inc. (Nasdaq: CALA), a clinical-stage pharmaceutical company focused on discovering and developing novel small molecule drugs directed against tumor metabolism and tumor immunology targets for the treatment of cancer, reported its financial results for the third quarter ended September 30, 2015. As of September 30, 2015, cash, cash equivalents and investments totaled $81.9 million (Filing, 8-K, Calithera Biosciences, NOV 9, 2015, View Source [SID:1234508336]).

"In the third quarter, we continued to move clinical programs forward with presentations of data at key medical meetings," said Susan Molineaux, PhD, President and Chief Executive Officer of Calithera. "We presented dose expansion monotherapy cohorts for our lead product CB-839 at the AACR (Free AACR Whitepaper)-EORTC-NCI meeting, and we are currently opening enrollment across six combination cohorts. For our lead immuno-oncology program, we presented initial preclinical data for CB-1158, an oral, small molecule arginase inhibitor with the potential to treat cancer by blocking the immunosuppression of the tumor micro-environment. We believe that we have multiple opportunities to positively impact clinical outcomes for cancer patients by building a pipeline of novel therapeutic product candidates."

Third Quarter 2015 and Recent Highlights

• CB-839 Solid Tumor Phase 1 data presented at the AACR (Free AACR Whitepaper)-NCI-EORTC meeting. On November 8, 2015, solid tumor phase 1 data were presented at the American Association of Cancer Research (AACR) (Free AACR Whitepaper)-National Cancer Institute (NCI)-European Organization for Research and Treatment of Cancer (EORTC) meeting that demonstrated the clinical activity, tolerability and unique mechanism of action of CB-839 as a single agent in patients with solid tumors. Among efficacy-evaluable patients across a range of tumor types treated on the twice daily schedule of CB-839 administered with food, 22 of 50 patients (44%) had stable disease (SD) or better lasting at least 3 cycles (63 days). One renal cell carcinoma (RCC) patient has an ongoing partial response (PR; on study > 5 months); this patient showed a 32% reduction in target lesions by RECIST with generalized shrinkage of lymph node metastases. Among the 15 evaluable patients with RCC, 9 (60%) had SD or PR, with four patients remaining on study.

• Arginase inhibitor CB-1158 preclinical data presented at the AACR (Free AACR Whitepaper)-NCI-EORTC Meeting. Calithera selected CB-1158 as the clinical candidate for Calithera’s first immuno-oncology program targeting inhibition of arginase, a critical T-Cell immunosuppressive enzyme produced by myeloid-derived suppressor cells in tumors. On November 6, 2015, Calithera presented data at the AACR (Free AACR Whitepaper)-NCI-EORTC meeting showing that CB-1158 is a highly selective, orally bioavailable, small molecule inhibitor of human arginase with nanomolar potency. Administration of CB-1158 to mice results in a dose-dependent increase in tumor arginine levels, consistent with target inhibition. Evaluation of anti-tumor efficacy in immunocompetent syngeneic mouse models has shown that oral administration of CB-1158 results in significant inhibition of tumor growth, and it combines well with checkpoint inhibitors with no evidence of additional toxicity.

• Augmented Board of Directors. In September 2015, Calithera appointed Sunil Agarwal, M.D., Senior Vice President and Chief Medical Officer at Ultragenyx, to the company’s Board of Directors.
Anticipated Upcoming Milestones

• Hematologic Phase 1 clinical trial data by year end 2015

• Initiation of six CB-839 combination expansion cohorts by year end 2015

• CB-839 combination therapy data in mid-2016

• Filing of investigational new drug application (IND) for arginase inhibitor CB-1158 in the first half of 2016

Selected Third Quarter 2015 Financial Results

Research and development expenses were $6.8 million for the three months ended September 30, 2015, compared with $3.9 million for the same period in the prior year. The increase of $2.9 million was primarily attributed to higher expenses associated with the continued advancement of CB-839 and investments in the Company’s arginase inhibitor program.
General and administrative expenses were $2.2 million for the three months ended September 30, 2015, compared with $1.3 million for the same period in the prior year. The increase of $0.9 million was primarily due to higher employment-related expenses, including stock-based compensation expense and professional service fees associated with operating as a publicly-traded company.
Net loss for the three months ended September 30, 2015 was $8.9 million.

Financial Guidance for 2015

Calithera is updating its guidance of its cash, cash equivalents and investments to be at least $70 million at the end of 2015, exclusive of any license milestone payments or funds arising from any additional new collaborations or partnerships, equity financings or other new sources.

8-K – Current report

On November 9, 2015 Puma Biotechnology, Inc. (NYSE: PBYI), a biopharmaceutical company, reported financial results for the third quarter ended September 30, 2015 (Filing, 8-K, Puma Biotechnology, NOV 9, 2015, View Source [SID:1234508176]).

Unless otherwise stated, all comparisons are for the third quarter and nine months ended September 30, 2015, compared to the third quarter and nine months ended September 30, 2014.

Based on accounting principles generally accepted in the United States (GAAP), Puma reported a net loss of $60.4 million, or $1.87 per share, for the third quarter of 2015, compared to a net loss of $35.9 million, or $1.19 per share, for the third quarter of 2014. Net loss for the nine months ended September 30, 2015 was $177.6 million, or $5.55 per share, compared to $94.5 million, or $3.16 per share, for the nine months ended September 30, 2014.

Non-GAAP adjusted net loss was $35.5 million, or $1.10 per share, for the third quarter of 2015, compared to $25.4 million, or $0.84 per share, for the third quarter of 2014. Non-GAAP adjusted net loss for the nine months ended September 30, 2015 was $104.3 million, or $3.26 per share, compared to $71.7 million, or $2.40 per share, for the nine months ended September 30, 2014. Non-GAAP adjusted net loss excludes stock-based compensation expense, which represents a significant portion of overall expense and has no impact on the cash position of the Company. For a reconciliation of GAAP net loss to non-GAAP adjusted net loss and GAAP net loss per share to non-GAAP adjusted net loss per share, please see the financial tables at the end of this news release.

Net cash used in operating activities for the third quarter of 2015 was $36.8 million. Net cash used in operating activities for the nine months ended September 30, 2015 was $121.4 million. At September 30, 2015, Puma had cash and cash equivalents of $23.6 million and marketable securities of $224.2 million, compared to cash and cash equivalents of $38.5 million and marketable securities of $102.8 million at December 31, 2014. Puma’s current level of cash and cash equivalents and marketable securities includes net proceeds of approximately $205.1 million from a public offering of the Company’s common stock, which was completed in January 2015.

"We continued to make significant progress with the neratinib clinical program in the third quarter," said Alan H. Auerbach, Chairman, Chief Executive Officer and President of Puma. "Puma achieved a number of milestones, including the presentation of additional data on patients with centrally confirmed HER2-positive disease from our Phase III ExteNET Trial at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2015 Breast Cancer Symposium and the publication in the Journal of the National Comprehensive Cancer Network of a patient with HER2 non-amplified (HER2-negative) metastatic breast cancer who also had a HER2 activating mutation and was successfully treated with PB272. We continue to anticipate filing for regulatory approval of PB272 for the extended adjuvant treatment of HER2-positive breast cancer in the first quarter of 2016.

"We anticipate a number of additional milestones through the end of 2015 and beyond. These include (i) presentations of additional data from the Phase III ExteNET Trial in the extended adjuvant treatment of early stage HER2-positive breast cancer (anticipated in the fourth quarter of 2015); (ii) publication of Phase III ExteNET Trial results (anticipated in the fourth quarter of 2015); (iii) presentation of the Phase II FB-7 trial of PB272 as a neoadjuvant treatment for patients with HER2-positive breast cancer (anticipated in the fourth quarter of 2015); (iv) presentation of data from our Phase II trial of PB272 in HER2 non-amplified breast cancer that has a HER2 mutation (anticipated in the fourth quarter of 2015); (v) reporting initial data from the Phase II trial of neratinib in extended adjuvant HER2-positive early stage breast cancer using loperamide prophylaxis (anticipated in the fourth quarter of 2015); (vi) publication of results to date on the use of prophylactic loperamide to reduce the neratinib-related diarrhea (anticipated in the fourth quarter of 2015); (vii) completing the ongoing Phase II trial of PB272 in patients with HER2-positive metastatic breast cancer that has metastasized to the brain (anticipated in the first half of 2016); and (viii) expanding additional cohorts in our Phase II basket trial of PB272 in patients with solid tumors with activating HER2 mutations (anticipated in the fourth quarter of 2015)."

Operating Expenses

Based on GAAP, operating expenses were $60.7 million for the third quarter of 2015, compared to $36.0 million for the third quarter of 2014. Operating expenses for the nine months ended September 30, 2015 were $178.2 million, compared to $94.7 million for the nine months ended September 30, 2014. The increase in operating expenses for the third quarter of 2015 compared to the third quarter of 2014 was primarily driven by an increase in stock-based compensation expense of $14.4 million, an increase in clinical trial expense of $5.1 million and an increase in internal research and development expense of $3.0 million. The increase in operating expenses for the nine months ended September 30, 2015 compared to the same period in 2014 was primarily driven by an increase in stock-based compensation of $50.5 million, an increase in clinical trial expense of $19.4 million and an increase in internal research and development expense of $8.2 million.

General and Administrative Expenses:

Based on GAAP, general and administrative expenses were $8.8 million for the third quarter of 2015, compared to $3.9 million for the third quarter of 2014. General and administrative expenses for the nine months ended September 30, 2015 were $22.2 million compared to $11.3 million for the nine months ended September 30, 2014.

Research and Development Expenses:

Based on GAAP, research and development expenses were $51.9 million for the third quarter of 2015, compared to $32.1 million for the third quarter of 2014. Research and development expenses for the nine months ended September 30, 2015 were $156.0 million, compared to $83.4 million for the nine months ended September 30, 2014.

Portola Pharmaceuticals Reports Third Quarter 2015 Financial Results and Provides Corporate Update

On November 09, 2015 Portola Pharmaceuticals (Nasdaq:PTLA) reported a corporate update and reported its financial results for the third quarter ended September 30, 2015 (Press release, Portola Pharmaceuticals, NOV 9, 2015, View Source;p=RssLanding&cat=news&id=2110682 [SID:1234508172]).

"We achieved a number of significant clinical, regulatory and manufacturing milestones over the past quarter," said William Lis, chief executive officer of Portola. "We completed enrollment in the APEX study of betrixaban, our Fast Track-designated product. For andexanet alfa, our breakthrough Factor Xa reversal agent, we initiated our rolling submission of our Biologics License Application, or BLA, to the U.S. Food and Drug Administration, or FDA. That comes on the heels of two important andexanet manufacturing milestones: we completed the commercial manufacturing process validation in support of our submission and commercial launch, and successfully scaled-up our first Generation 2 process, which will allow us to increase our production capacity at lower costs. These achievements keep us on track for our planned launch of andexanet alfa in 2016 followed by betrixaban in 2017, assuming clinical and regulatory success."

Other Recent Achievements, Upcoming Events and Milestones

Betrixaban

Completed APEX (Acute Medically Ill VTE Prevention with Extended Duration Betrixaban) study enrollment.
Successfully completed a sixth and final review of the APEX study Data and Safety Monitoring Committee, to correspond with an increase in sample size from 6,850 to 7,500 patients.
Received Fast Track designation from the FDA for prevention of VTE in acute medically ill patients.
APEX Study metrics, including pooled blinded aggregate event rates, remain on target.
Plan to report topline APEX study data late in the first quarter of 2016.

Plan to submit a New Drug Application to the FDA in 2016, subject to positive data.

Andexanet Alfa

Initiated rolling submission of BLA; we expect the application to be complete by the end of 2015 under Accelerated Approval pathway.

Completed commercial manufacturing process validation at CMC Biologics to support the BLA and commercial launch at 2,500 liter scale.

Successfully scaled up our first Generation 2, 10,000 liter batch product at Lonza.

Announced topline data from the second part of the Phase 3 ANNEXATM-R study, which demonstrated that andexanet alfa administered as an intravenous bolus followed by a continuous two-hour infusion produced rapid reversal of the anticoagulant effect of rivaroxaban and sustained it for the duration of the infusion. The full data set from Part 2 of the study will be presented during a Late-Breaking Clinical Trial session at the American Heart Association’s Scientific Sessions 2015 on November 11 in Orlando.
Enrollment remains on track in ANNEXA-4, a Phase 4 confirmatory study of patients receiving apixaban, rivaroxaban, edoxaban or enoxaparin who present with an acute major bleed.

Initiated a Phase 2 proof-of-concept study in healthy volunteers to evaluate the safety and efficacy of andexanet alfa in reversing the anticoagulant effect of betrixaban, and define the dose of betrixaban required for inclusion in ANNEXA-4.
Plan to present two abstracts at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December in Orlando.

Cerdulatinib – Oral, dual Syk/JAK kinase inhibitor for hematologic cancers

Continued to dose-escalate in the Phase 1 part of the ongoing Phase 1/2a study in patients with relapsed/refractory B-cell malignancies who have failed multiple therapies to determine the maximum tolerated dose. Exploring alternate dosing regiments and formulations to get to a higher exposure. Cerdulatinib continues to show activity and is well tolerated.
Plan to present three abstracts at the ASH (Free ASH Whitepaper) Annual Meeting in December in Orlando.

Corporate

Elected David C. Stump, M.D., former executive vice president, research and development, at Human Genome Sciences, Inc., to Portola’s board of directors.

Third Quarter Financial Results

Collaboration revenue earned under Portola’s collaborations with Bristol-Myers Squibb Company and Pfizer, Bayer Pharma and Janssen Pharmaceuticals, Daiichi Sankyo and Lee’s Pharmaceutical was $2.9 million for the third quarter of 2015 compared with $2.4 million for the third quarter of 2014.

Total operating expenses for the third quarter of 2015 were $58.5 million compared with $38.2 million for the same period in 2014. Total operating expenses for the third quarter of 2015 included $6.1 million in stock-based compensation expense compared with $2.2 million for the third quarter of 2014. Research and development expenses were $48.4 million for the third quarter of 2015 compared with $31.8 million for the third quarter of 2014 as the Company continued to support its manufacturing scale-up of andexanet alfa in preparation for BLA submission and commercial launch and work on its larger scale Generation 2 manufacturing process at Lonza; its Phase 3 and Phase 4 ANNEXA studies of andexanet alfa; completing enrollment in the Phase 3 APEX Study of betrixaban and its Phase 1/2a clinical study of cerdulatinib. Selling, general and administrative expenses for the third quarter of 2015 were $10.1 million compared with $6.4 million for the same period in 2014 as the Company increased headcount to support its growth, resulting in higher headcount-related costs including stock-based compensation expense, and increased pre-commercial launch activities.

Portola reported a net loss of $55.2 million, or $(1.05) net loss per share, for the third quarter of 2015 compared with a net loss of $35.8 million, or $(0.86) net loss per share, for the third quarter of 2014. Shares used to compute net loss per share attributable to common stockholders were approximately 52.6 million for the third quarter of 2015 compared with approximately 41.4 million for the same period in 2014.

As of September 30, 2015, cash, cash equivalents and investments totaled $356.0 million compared with cash, cash equivalents and investments of $392.3 million as of December 31, 2014.

Merrimack Reports Third Quarter 2015 Financial Results

On November 9, 2015 Merrimack Pharmaceuticals, Inc. (Nasdaq: MACK), a biopharmaceutical company that applies systems biology to develop cancer treatments and diagnostics, reported its third quarter 2015 financial results (Press release, Merrimack, NOV 9, 2015, View Source [SID:1234508166]). Merrimack will host a live conference call and webcast today, Monday, November 9 at 4:30 p.m., Eastern time, to provide an update on Merrimack’s progress as well as a summary of these results.

Investors and the general public are invited to listen to the call by dialing (877) 564-1301 (domestic) or (224) 357-2394 (international) five minutes prior to the start of the call and providing the passcode 69525411. A listen-only webcast of the call can be accessed in the Investors section of Merrimack’s website, investors.merrimack.com, and a replay of the call will be archived there for six weeks following the call.

Key Recent Events

Approval of ONIVYDE (irinotecan liposome injection) by the U.S. Food and Drug Administration on October 22, 2015;
Commercial launch of ONIVYDE in the United States on October 26, 2015;

Enrollment of first patient in a Phase 2 clinical study of ONIVYDE in previously untreated front-line metastatic pancreatic cancer; and

Achievement of $66.5 million in net milestone payments from collaborations in 2015, consistent with Merrimack’s previous financial guidance.

Upcoming Milestones

Merrimack anticipates the following clinical milestones:

Initiation of a Phase 3 clinical study of ONIVYDE in front-line HER2-negative gastric cancer in 2016;

Results from the Phase 2 clinical study of MM-121 in patients with heregulin-positive, locally advanced or metastatic non-small cell lung cancer in the second half of 2016;

Results from the Phase 2 clinical study of ONIVYDE in previously untreated front-line metastatic pancreatic cancer in the first half of 2017;

Results from HERMIONE, the Phase 2 clinical study of MM-302 in patients with HER2-positive metastatic breast cancer designed to support a potential Accelerated Approval application to the FDA, in 2017; and

Results from the Phase 2 clinical study of MM-141 in patients with front-line metastatic pancreatic cancer who have high serum levels of free IGF-1 in 2017.

Third Quarter 2015 Financial Results

Revenue for the third quarter of 2015 was $16.4 million compared with revenue of $28.0 million for the third quarter of 2014, a decrease of $11.6 million or 41%. This decrease was primarily due to the termination of Merrimack’s collaboration with Sanofi effective December 17, 2014 and was offset by increased revenue recognized related to Merrimack’s collaboration with Baxalta during the third quarter of 2015.

Research and development expenses for the third quarter of 2015 decreased $5.9 million over the corresponding quarter of the preceding year. This decrease was primarily due to $12.0 million of non-recurring ONIVYDE collaboration milestones that were expensed in the third quarter of 2014. This decrease was offset by increased spending on Merrimack’s remaining pipeline.

General and administrative expenses for the third quarter of 2015 increased $8.9 million over the corresponding quarter of the preceding year. This increase was primarily attributable to increased infrastructure and personnel expenses as Merrimack prepared for the commercialization of ONIVYDE.

Merrimack’s net loss for the third quarter of 2015 was $42.4 million, or basic and diluted net loss per share available to common stockholders of $0.38, as compared to a net loss for the third quarter of 2014 of $28.0 million, or basic and diluted net loss per share available to common stockholders of $0.27.

Financial Outlook

Merrimack expects to be able to fund operations into the second quarter of 2016 based on the following:

– unrestricted cash and cash equivalents and available-for-sale securities of $62.4 million as of September 30, 2015;
– $47.5 million of milestones from Baxalta anticipated in the fourth quarter of 2015 related to the first patient dosed in the Phase 2 clinical study of ONIVYDE in previously untreated front-line metastatic pancreatic cancer; and
– anticipated cost sharing reimbursements from Baxalta.

Additional funding for operations would also come from:

– anticipated receipt of $46.5 million of net milestones related to ONIVYDE in 2016 from Baxalta, after offsetting payments to PharmaEngine;

– sales of ONIVYDE; and

– up to $15.0 million in additional debt that is currently available at Merrimack’s option under its loan agreement with Hercules Technology Growth Capital.

Upcoming Investor Conferences

Merrimack will attend the following investor conferences this fall:

Credit Suisse 24th Annual Healthcare Conference on November 11 in Scottsdale, AZ;
Jefferies Autumn 2015 Global Healthcare Conference on November 18 in London;
Oppenheimer 26th Annual Healthcare Conference on December 8 in New York; and
Guggenheim 3rd Annual Boston Healthcare Conference on December 15 in Boston.
Live webcasts of the presentations at the Credit Suisse 24th Annual Healthcare Conference, Jefferies Autumn 2015 Global Healthcare Conference and Oppenheimer 26th Annual Healthcare Conference can be accessed by visiting the Investors section of Merrimack’s website at investors.merrimack.com. A replay of the webcasts will be archived there for two weeks following each presentation.