On August 8, 2016 Vericel Corporation (NASDAQ:VCEL), a leading developer of expanded autologous cell therapies for the treatment of severe diseases and conditions, reported financial results for the second quarter ended June 30, 2016 (Press release, Vericel, AUG 8, 2016, View Source [SID:1234514365]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Total net revenues for the quarter ended June 30, 2016 were approximately $12.8 million and included approximately $9.0 million of Carticel net revenues and approximately $3.8 million of Epicel net revenues. Previously announced downtime for the Carticel and Epicel cleanrooms to replace a rooftop air handler unit resulted in a two-week, or approximately 16%, reduction in product shipment dates for both products during the second quarter. As a result, total Carticel and Epicel net revenues decreased 3.9% compared to the second quarter of 2015, with Carticel net revenues decreasing less than $0.1 million and Epicel net revenues decreasing approximately $0.4 million, respectively, compared to the second quarter of 2015. For the first half of 2016, total net revenues were $26.9 million and included $17.8 million of Carticel net revenues and $9.1 million of Epicel net revenues. Total Carticel and Epicel net revenues for the first half of 2016 increased 12% compared to the first half of 2015, with Carticel revenues increasing 10% and Epicel revenues increasing 15%, respectively, compared to the same period in 2015.

Gross profit for the quarter ended June 30, 2016 was $5.5 million, or 43% of net product revenues, compared to $6.7 million, or 49% of net product revenues, for the second quarter of 2015. The reduction in gross profit was primarily due to the reduced volume resulting from the cleanroom downtime. Gross profit for the first half of 2016 was $13.1 million, or 49% of net product revenues, compared to $12.0 million, or 49% of net product revenues, for the first half of 2015.

Research and development expenses for the quarter ended June 30, 2016 were $4.1 million compared to $3.4 million in the second quarter of 2015. The increase in second-quarter research and development expenses is primarily due to an increase in expenses associated with the completion of the ixCELL-DCM clinical trial and preparing to treat patients in the open-label crossover extension portion of the study, as well as for research, development, and regulatory consulting expenses for MACI (Autologous Cultured Chondrocytes on Porcine Collagen Membrane). MACI is Vericel’s investigational third-generation autologous cultured chondrocyte implant intended for the treatment of symptomatic full-thickness cartilage defects of the knee.

Selling, general and administrative expenses for the quarter ended June 30, 2016 were $6.4 million compared to $5.6 million for the same period in 2015. The increase in selling, general and administrative expenses is primarily due to costs associated with the start-up of the Dohmen collaboration for patient support and reimbursement services for Carticel and MACI, if approved, professional services related to preparing for the potential launch of MACI, as well as legal fees, shared facility fees and an increase in personnel costs.

Loss from operations for the quarter ended June 30, 2016 was $5.0 million, compared to $2.3 million for the second quarter of 2015. Material non-cash items impacting the operating loss for the quarter included $0.8 million of stock-based compensation expense and $0.5 million in depreciation and amortization expense.

Other income for the quarter ended June 30, 2016 was $1.9 million compared to $0.1 million for the same period in 2015. The change in other income for the quarter is primarily due to the change in the fair value of warrants in the second quarter of 2016 compared to the same period in 2015.

Vericel’s reported GAAP net loss for the quarter ended June 30, 2016 was $3.0 million, or $0.22 per share, compared to a net loss of $2.2 million, or $0.16 per share, for the same period in 2015. Vericel reported an adjusted net loss for the quarter ended June 30, 2016 of $5.0 million dollars, or $0.21 per share, compared to an adjusted net loss of $2.3 million, or $0.10 per share, for the same period in 2015. The adjusted net loss excludes the non-cash change in the fair value of warrants and the non-cash accumulated dividend on the Series B convertible preferred stock. The adjusted net loss per share includes common shares reserved as treasury shares received in exchange for the Series A non-voting convertible preferred stock.

As of June 30, 2016, the company had $9.8 million in cash compared to $14.6 million in cash at December 31, 2015.

Recent Business Highlights

During and since the second quarter of 2016, the company:

Limited the impact of the manufacturing downtime for the Carticel and Epicel cleanrooms; total Carticel and Epicel net revenues increased 12% for the first half of 2016 compared to the same period in 2015;
Initiated the collaboration with Dohmen Life Science Services, LLC for patient support services, as well as payer contracting and product reimbursement services for Carticel and MACI, if approved, which is expected to increase operating profit margin for these products by retaining margin previously captured by a distributor;
Increased MACI launch preparation and operational activities in anticipation of the January 3, 2017, MACI PDUFA goal date;
Announced results from the Phase 2b ixCELL-DCM clinical trial of ixmyelocel-T in patients with advanced heart failure due to ischemic dilated cardiomyopathy, which were presented at the American College of Cardiology’s 65th Annual Scientific Session and published in The Lancet; and
Initiated activities to explore potential expedited development and review pathways and partnering discussions for ixymyelocel-T in the U.S., Japan and Europe in light of meeting the primary endpoint in the ixCELL-DCM clinical trial.
"We are pleased with the commercial performance of the business in light of the manufacturing downtime as we have generated strong growth in our core commercial business during the first half of the year," said Nick Colangelo, president and CEO of Vericel. "We believe that we are building a strong foundation for our cartilage repair franchise, and we look forward to continuing to work productively with the FDA during the ongoing MACI BLA review process as we prepare for the potential launch of MACI, if approved, in the first quarter of 2017."

On August 8, 2016 FibroGen, Inc. (NASDAQ: FGEN) ("FibroGen"), a research-based biopharmaceutical company, reported financial results for the quarter ended June 30, 2016 (Filing, Q2, FibroGen, 2016, AUG 8, 2016, View Source [SID:1234514414]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We continue to develop our multiple key programs across focused therapeutic areas," said Thomas B. Neff, chief executive officer of FibroGen. "Working jointly with our partners, Astellas and AstraZeneca, we have advanced global clinical development for roxadustat for treatment of anemia in chronic kidney disease patients to Phase 3 in four independent regulatory pathways − the U.S., Europe, China, and Japan − and remain on track to initiate new drug application submissions in 2016 in China, and in 2018 in the U.S. We have completed enrollment in the placebo-controlled portion of our Phase 2 study of FG-3019 (now known as pamrevlumab) for treatment of idiopathic pulmonary fibrosis, and expect to report data from this study in the middle of next year, and our Phase 2 studies in pancreatic cancer and Duchenne muscular dystrophy continue to progress."
Program Updates
Anemia of Chronic Kidney Disease (CKD): roxadustat (FG-4592)

·
China Phase 3 enrollment on track: Completed enrollment in a 300-patient dialysis study, one of two Phase 3 pivotal trials in China; expect to complete enrollment in the second study, a 150-patient non-dialysis study now over 70% enrolled, in Q3 of this year. We expect first reportable data in each of the Phase 3 studies by the end of the year.

·
The independent data safety monitoring board overseeing roxadustat U.S and Europe Phase 3 studies met in July 2016 to review the roxadustat safety data, and confirmed that the trials should proceed with current Phase 3 protocols without modification.

·
Continue to expect to initiate new drug application submissions for roxadustat in 2016 for China and in 2018 for the U.S.
Fibrosis and Other Fibroproliferative Diseases: pamrevlumab (FG-3019)

·
Completed enrollment in the 48-week main study portion of a placebo-controlled Phase 2 trial for treatment of IPF. We plan to report topline data for the entire study in mid-2017, including the six month combination therapy sub-study in which patients will receive pamrevlumab in combination with pirfenidone or nintedanib, which will continue to enroll until the end of the year.

·
Continue to advance our Phase 2 trial in patients with unresectable, locally advanced pancreatic cancer, and anticipate that we will present available findings early next year.

·
Enrollment continues in our open-label Phase 2 study of pamrevlumab in non-ambulatory Duchenne muscular dystrophy (DMD) patients.
Financial Highlights

·
Net income per basic share for the quarter ended June 30, 2016, was $0.39, and $0.35 on a per diluted share basis.

·
At June 30, 2016, FibroGen had $368.6 million of cash, cash equivalents, investments, receivables, and restricted cash.

·
During the quarter ended June 30, 2016, we received a $62.0 million upfront payment under the AstraZeneca Agreement. We also recognized $10.0 million milestone revenue under the Astellas Agreement, which payment was received in early July 2016.

Scynexis has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 10-Q, Scynexis, 2017, AUG 8, 2016, View Source [SID1234521725]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

On August 8, 2016 Cascadian Therapeutics (NASDAQ:CASC), a clinical-stage biopharmaceutical company, reported a corporate update and reported financial results for the quarter ended June 30, 2016 (Press release, Cascadian Therapeutics, AUG 8, 2016, View Source [SID:1234514367]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"During the second quarter, we continued to accelerate the clinical development of our lead product candidate ONT-380, a promising small molecule for the treatment of advanced, metastatic HER2+ breast cancer," said Scott Myers, President and CEO of Cascadian Therapeutics. "Early clinical results with ONT-380 when combined with T-DM1 or capecitabine have suggested a favorable safety and tolerability profile, systemic activity in advanced disease and the potential to improve outcomes in patients with and without brain metastases. We’re looking forward to reporting new data from the ongoing Phase 1b Triplet study expected later this year. In addition, we were granted Fast Track designation from the FDA for ONT-380 in HER2+ metastatic breast cancer."

"Our continued progress and the refocusing of resources on development of ONT-380 and our Chk1 inhibitor, supported by a recent financing in June, leave us well-positioned to deliver on our business and clinical development plans," said Mr. Myers.

SECOND QUARTER AND RECENT HIGHLIGHTS

Clinical Development

Presented positive results from the Phase 1b "Triplet" study of ONT-380 in combination with trastuzumab and capecitabine at the Company’s R&D Day in June. The study is evaluating ONT-380 in patients with locally advanced or metastatic breast cancer with and without brain metastases who have previously received treatment with multiple HER2 agents, including T-DM1. Topline results showed a progression free survival (PFS) of 6.3 months and an overall response rate (ORR) of 58%. A majority of adverse events were Grade 1, with most patients being able to continue on the full dose of ONT-380. Additionally, Grade 3 diarrhea was infrequent, without the need for a prophylactic anti-diarrheal medicine. These data suggest ONT-380’s promising systemic activity, favorable safety profile and activity against brain metastases as well as support the ongoing Phase 2 trial in this combination, known as HER2CLIMB, which the Company initiated in February 2016. The Company expects to present more mature data from the Triplet study before the end of the year.

Presented updated safety and activity data from the Phase 1b combination trial of ONT-380 with T-DM1 at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting in June. This study includes patients with advanced disease who were previously treated with trastuzumab and a taxane. Results showed a PFS of 8.2 months, with an ORR of 47%. Many of the patients with brain metastases in the study had long-term control of both brain metastases and systemic disease. PFS in the 30 patients with brain metastases was similar to patients without brain metastases, and there were no patients without brain metastases at baseline who developed new clinically apparent brain metastases while on the study.

Phase 2 ONT-380 combination trial, HER2CLIMB, continues to enroll patients with late-stage HER2+ metastatic breast cancer. This randomized, double-blind, placebo-controlled Phase 2 study is evaluating ONT-380 versus placebo in combination with capecitabine and trastuzumab in late stage HER2+ breast cancer patients, with and without brain metastases, who have previously been treated with a taxane, trastuzumab, pertuzumab and T-DM1. The Phase 2 trial is expected to enroll approximately 180 patients at sites in the U.S., Canada and select countries in Western Europe. Building on encouraging Phase 1b Triplet results, the primary and secondary endpoint objectives are designed to measure ONT-380’s contribution to treating systemic disease in patients with and without brain metastases. For more information, visit www.clinicaltrials.gov: NCT02614794.

Received Fast Track Designation for ONT-380 from FDA for the treatment of advanced HER2+ metastatic breast cancer. The Company continues to evaluate additional opportunities for ONT-380 and establish the most efficient regulatory and commercialization path forward.

Developing novel Chk1 cell cycle inhibitors with plans to move the program forward through IND-enabling studies in 2017. Topline preclinical results presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2016 showed that select Cascadian Chk1 inhibitors display cellular potency against Chk1, are active against a diverse range of cancer cell lines, and demonstrate synergistic activity in combination with certain chemotherapeutic drugs.
Corporate Update

Raised gross proceeds of $46 million in a concurrent, but separate, underwritten public offering and a registered direct offering to support ongoing program development.

Completed corporate name change to Cascadian Therapeutics from Oncothyreon, reflecting the shift in the Company’s focus from therapeutic vaccines to advancing targeted treatments for cancer.

Prioritized the Company’s pipeline to focus on ONT-380 and the Chk1 cell cycle inhibitor program and ceased development of the protocell research program.
SECOND QUARTER 2016 FINANCIAL HIGHLIGHTS

Cash, cash equivalents and investments totaled $80.9 million as of June 30, 2016, compared to $56.4 million at December 31, 2015, an increase of $24.5 million, or 43.4%. The increase was the result of net proceeds of $43.2 million from the Company’s June 2016 financing offset by cash used to fund operations of $18.7 million.

Net loss attributable to common stockholders for the three months ended June 30, 2016 was $25.1 million, or $0.26 per basic and diluted share, compared with a net loss attributable to common stockholders of $10.9 million, or $0.11 per basic and diluted share, for the comparable period in 2015. The increase in net loss attributable to common stockholders for the quarter was primarily due to the intangible asset impairment charge of $19.7 million during the three months ended June 30, 2016, which was the result of the mutual termination of the STC.UNM agreement and the Company’s intent to no longer develop, license or commercialize the protocell technology. In addition, the increase in net loss attributable to common stockholders was due to increases in general and administrative expenses of $1.7 million primarily due to higher professional fees associated with legal and regulatory compliance and expenses related to the Company’s Retention Payment Plan, and increases in research and development expenses of $0.7 million primarily due to greater activity related to the development of the Company’s product candidates. The Company also recognized a non-cash $1.6 million deemed dividend as a result of the beneficial conversion feature on the Series D convertible preferred stock. The increase in the net loss attributable to common stockholders was partially offset by a $6.9 million tax benefit that resulted from the reversal of the deferred tax liability that was associated with the impaired intangible asset during the three months ended June 30, 2016 and lower non-cash expense from the change in the fair value of the Company’s warrant liability, which was zero for the three months ended June 30, 2016 compared to $2.6 million for the three months ended June 30, 2015. The change in the fair value of warrant liability was due to the expiration of September 2010 warrants, which expired in October 2015.

Net loss attributable to common stockholders for the six month ended June 30, 2016 was $38.0 million, or $0.40 per basic and diluted share, compared with a net loss attributable to common stockholders of $18.8 million, or $0.19 per basic and diluted share, for the comparable period in 2015. The increase in net loss attributable to common stockholders for the six months ended June 30, 2016 was primarily due to the intangible asset impairment charge of $19.7 million during the six months ended June 30, 2016, increases in research and development expenses of $1.2 million primarily due to greater activity related to the development of the Company’s product candidates, and increases in general and administrative expenses of $6.0 million primarily related to the retirement and separation agreement that Cascadian entered into with its former Chief Executive Officer in January 2016, higher professional fees associated with legal and regulatory compliance and expenses related to the Company’s Retention Payment Plan. The Company also recognized a non-cash $1.6 million deemed dividend as a result of the beneficial conversion feature on the Series D convertible preferred stock. The increase in the net loss attributable to common stockholders was partially offset by a $6.9 million tax benefit during the six months ended June 30, 2016 and lower non-cash expense from the change in the fair value of the Company’s warrant liability, which was zero for the six months ended June 30, 2016 compared to $2.5 million for the six months ended June 30, 2015. The change in the fair value of warrant liability was due to the expiration of the September 2010 warrants, which expired in October 2015.
Financial Guidance
Cascadian Therapeutics believes the following financial guidance to be correct as of the date provided. Cascadian Therapeutics is providing this guidance as a convenience to investors and assumes no obligation to update it.

Cascadian Therapeutics currently expects cash used in operations in 2016 to be approximately $38.0 million to $40.0 million. With cash, cash equivalents and investments of $80.9 million as of June 30, 2016, Cascadian Therapeutics estimates that its cash, cash-equivalents and investments will be sufficient to fund operations for at least the next 12 months.

Akebia has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 10-Q, Akebia, 2017, AUG 8, 2016, View Source [SID1234521563]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!