On March 6, 2017 Mast Therapeutics, Inc. (NYSE MKT: MSTX), a clinical-stage biopharmaceutical company, reported financial results for the fourth quarter and year ended December 31, 2016 (Filing, Q4/Annual, Mast Therapeutics, 2016, MAR 6, 2017, View Source [SID1234518024]).

“We are pleased with the progress made with AIR001 during the year and anticipate the closing of the merger with Savara to take place in the second quarter of this year,” stated Brian M. Culley, Chief Executive Officer. “We believe this merger offers our stockholders a diversified, late-stage product development pipeline with important forthcoming milestones.”

Fourth Quarter 2016 Operating Results

The Company’s net loss for the fourth quarter of 2016 was $6.0 million, or $0.02 per share (basic and diluted), compared to a net loss of $10.2 million, or $0.06 per share (basic and diluted), for the same period in 2015.

The Company recognized $83,000 of revenue for the fourth quarter of 2016, representing reimbursement of costs related to the nonclinical study of vepoloxamer that is being funded by a Small Business Innovation Research (SBIR) grant. The Company recognized no revenue for the same period in 2015.

Research and development (R&D) expenses for the fourth quarter of 2016 were $78,000, a decrease of approximately $7.1 million, or 99%, compared to $7.2 million for the same period in 2015. This reduction in R&D expense was due principally to the Company’s decision to discontinue clinical development of vepoloxamer in September 2016. External clinical study fees and expenses decreased by $3.9 million, external nonclinical study fees and expenses decreased by $2.9 million and R&D personnel costs decreased by $0.3 million for the fourth quarter of 2016 compared to the same period in 2015.

Selling, general and administrative (SG&A) expenses for the fourth quarter of 2016 were $1.9 million, a decrease of $0.6 million, or 23%, compared to $2.5 million for the same period in 2015. The decrease was primarily due to reduced fees for consulting and legal services and personnel costs compared to the 2015 period.

Interest expense was $0.2 million for the fourth quarter of 2016, compared to $0.5 million for the same period in 2015. The decrease in interest expense was primarily due to the Company’s prepayment of $10.0 million of the principal balance of its debt facility in October 2016, which lowered the overall principal balance of the debt significantly. The principal balance was $3.3 million at December 31, 2016 compared to $15.0 million at December 31, 2015.

Fiscal Year 2016 Financial Results

The Company’s net loss for the year ended December 31, 2016 was $36.1 million, or $0.17 per share (basic and diluted), compared to a net loss of $39.8 million, or $0.25 per share (basic and diluted), for the same period in 2015.

The Company recognized $128,000 of revenue for the year ended December 31, 2016, representing reimbursement of costs related to the nonclinical study of vepoloxamer that is being funded by a SBIR grant. The Company recognized no revenue for the same period in 2015.

R&D expenses for the year ended December 31, 2016 were $20.8 million, a decrease of $7.5 million, or 26%, compared to $28.3 million for the same period in 2015. The decrease was due primarily to a $4.1 million decrease in external nonclinical study fees and expenses, a $3.3 million decrease in external clinical study fees and expenses, and a $0.3 million decrease in personnel costs, offset by a $0.3 million increase in share-based compensation expense.

The decrease in external nonclinical study fees and expenses resulted primarily from decreases in research-related manufacturing costs for vepoloxamer ($4.7 million) and nonclinical studies of vepoloxamer ($1.5 million), offset by

increased costs related to preparing a new drug application for vepoloxamer ($1.9 million), which project was discontinued in September 2016, and research-related manufacturing costs for AIR001 ($0.2 million). The decrease in external clinical study fees and expenses was due primarily to decreases in costs for the Phase 3 study of vepoloxamer in sickle cell disease ($5.0 million) and the Phase 2 study of vepoloxamer in ALI that was discontinued in the third quarter of 2015 ($0.5 million), offset by increased costs related to the Phase 2 study of vepoloxamer in heart failure ($1.3 million) and the investigator-sponsored Phase 2 studies of AIR001 in HFpEF ($0.9 million). The $0.3 million decrease in personnel costs was due primarily to reductions in the Company’s workforce that occurred in the fourth quarter of 2016.

SG&A expenses for the year ended December 31, 2016 were $9.3 million, a decrease of $1.7 million, or 15%, compared to $11.0 million for the same period in 2015. This decrease was due primarily to a $1.0 million decrease in personnel costs and a $0.5 million decrease in professional and consulting fees.

Interest expense was $2.1 million for the year ended December 31, 2016, an increase of $1.5 million compared to $0.6 million for the same period in 2015. The increase in interest expense was primarily due to interest expense on a $15 million principal balance under the Company’s debt facility for nine months in 2016 versus approximately four months of interest expense on the debt facility in 2015, as well as increased amortization of debt issuance costs as a result of a change in the amortization schedule of such costs due to prepayment of $10 million of the principal balance in October 2016.

On March 6, 2017 Seattle Genetics, Inc. (Nasdaq: SGEN), a global biotechnology company, reported that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold announced on December 27, 2016 on phase 1 trials of vadastuximab talirine (SGN-CD33A; 33A) in acute myeloid leukemia (AML) (Press release, Seattle Genetics, MAR 6, 2017, View Source [SID1234518019]).

“The clinical hold on our early-stage vadastuximab talirine clinical trials has been resolved through a comprehensive analysis of the clinical data from over 300 patients treated to date, evaluation by an independent committee of clinical experts, collaborative interactions with the FDA, and protocol amendments designed to further enhance patient safety,” said Jonathan Drachman, M.D., Chief Medical Officer and Executive Vice President, Research and Development at Seattle Genetics. “We will resume two phase 1 trials in AML and plan to initiate a randomized phase 2 trial during 2017 evaluating vadastuximab talirine in combination with standard of care chemotherapy in frontline, younger AML patients. In addition, we are continuing to enroll our ongoing phase 3 randomized CASCADE trial in frontline older AML patients and our phase 1/2 trial in frontline high-risk myelodysplastic syndrome (MDS).”

Seattle Genetics will resume two phase 1 trials of vadastuximab talirine. The first is combination treatment with standard of care, or 7+3, chemotherapy in newly diagnosed younger AML patients and the second is monotherapy and combination treatment with hypomethylating agents in both newly diagnosed and relapsed AML patients. Seattle Genetics will not resume the phase 1/2 trial of vadastuximab talirine monotherapy in pre- and post-allogeneic transplant AML patients given the challenges of developing therapies in this specific setting. The company’s randomized global phase 3 CASCADE trial in frontline older AML and phase 1/2 trial in frontline MDS were not placed on clinical hold and have continued to enroll patients. Planned studies include a randomized phase 2 trial of vadastuximab talirine in combination with 7+3 chemotherapy in frontline younger AML patients. Going forward, additional risk mitigation measures will be implemented in all vadastuximab talirine studies, including revised eligibility criteria and stopping rules for veno-occlusive disease (VOD).

About Vadastuximab Talirine
Vadastuximab talirine (SGN-CD33A; 33A) is a novel investigational ADC targeted to CD33 utilizing Seattle Genetics’ proprietary ADC technology. CD33 is expressed on most AML and MDS blast cells. The CD33 engineered cysteine antibody is stably linked to a highly potent DNA binding agent called a pyrrolobenzodiazepine (PBD) dimer via site-specific conjugation technology (EC-mAb). PBD dimers are significantly more potent than systemic chemotherapeutic drugs and the EC-mAb technology allows uniform drug-loading onto an ADC. The ADC is designed to be stable in the bloodstream and to release its potent cell-killing PBD agent upon internalization into CD33-expressing cells.
Vadastuximab talirine was granted Orphan Drug Designation by both the U.S. Food and Drug Administration (FDA) and the European Commission for the treatment of AML. FDA orphan drug designation is intended to encourage companies to develop therapies for the treatment of diseases that affect fewer than 200,000 individuals in the United States.

On March 6, 2017 Pieris Pharmaceuticals, Inc. (NASDAQ: PIRS), a clinical-stage biotechnology company advancing novel biotherapeutics through its proprietary Anticalin technology platform, reported that a set of ex vivo and in vivo data informing the trial design of a first-in-patient clinical trial for PRS-343, a first-in-class 4-1BB/HER2 bispecific, will be presented in a poster session at the 2017 Annual Meeting of the American Association for Cancer Research (AACR) (Free AACR Whitepaper) to be held in Washington D.C., April 1 – 5, 2017 (Press release, Pieris Pharmaceuticals, MAR 6, 2017, View Source [SID1234518017]). PRS-343 binds to 4-1BB (CD137) on T cells and HER2 on tumor cells, producing tumor-targeted immune activation.

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Further details include:

Poster Number : 16

Title : Preclinical toxicology and pharmacology for the 4-1BB/HER2 bispecific PRS-343: A first-in-class costimulatory T cell engager.

Date and Time : Tuesday April 4, 2017, 8:00 AM – 12:00 PM

Location : Walter E. Washington Convention Center, Halls A-C, Poster Section 27
About PRS-343:
PRS-343 is a bispecific monoclonal antibody/Anticalin fusion protein comprised of a HER2 tumor-targeting mAb genetically linked to a potent Anticalin specific for the immune costimulatory TNF family receptor 4-1BB (CD137). PRS-343 is being developed as the first 4-1BB based therapeutic to mediate the activation of tumor-specific T lymphocytes selectively within the tumor microenvironment (TME). 4-1BB is a potent costimulatory immunoreceptor and an established marker for tumor-specific infiltrating T lymphocytes (TILs), and is, therefore, an attractive target for cancer immunotherapy. In in vivo preclinical tumor models, PRS-343 has demonstrated potent T lymphocyte activation localized to the TME of established HER2-positive tumors, indicating the potential for both enhanced safety and efficacy.

On March 6, 2017 Kura Oncology, Inc. (NASDAQ:KURA), a clinical stage biopharmaceutical company focused on the development of precision medicines for oncology, reported updated results from the ongoing, Phase 2 trial of tipifarnib, a selective inhibitor of farnesyl transferase, and additional preclinical data in HRAS mutant squamous cell carcinomas of the head and neck (SCCHN) (Press release, Kura Oncology, MAR 6, 2017, View Source [SID1234518010]). The data were presented today at the 15th International Congress on Targeted Anticancer Therapies (TAT 2017) in Paris, France.

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This update on the patients in the first stage of the second cohort of the Phase 2 clinical trial in HRAS mutant tumors reported that, as of February 28, 2017, the two patients with HRAS mutant SCCHN with objective responses remain on study and are currently at treatment cycle 19 and cycle 12. Among the five patients with HRAS mutant salivary gland cancer, although no objective responses were observed, three patients experienced disease stabilization and were on study for 9, 10 and 14 cycles. In addition, tipifarnib was generally well tolerated with adverse events consistent with its known safety profile.

"The durability of the responses we have observed with tipifarnib is compelling considering the setting of relapsed and/or refractory SCCHN," said Alan L. Ho, M.D., Ph.D., a medical oncologist at Memorial Sloan Kettering Cancer Center and a lead investigator on the study. "These data reinforce the relevance of testing for HRAS mutations in these patients."

"We are very encouraged by the durable responses we have observed in patients with relapsed and/or refractory HRAS mutant SCCHN who are treated with tipifarnib," said Troy Wilson, Ph.D., J.D., President and CEO of Kura Oncology. "The two patients with partial responses have now been on treatment with tipifarnib for more than 16 months and 10 months, respectively. Recruitment of patients with SCCHN has become more challenging in the United States with the approval of immune therapy agents. To facilitate enrollment in the second stage of our trial we are adding additional clinical sites in Western Europe and Asia, and are working with third party laboratories to facilitate HRAS screening. We anticipate that additional results from this ongoing Phase 2 trial of tipifarnib in HRAS mutant SCCHN will be available during the second half of 2017."

Dr. Ho’s slide presentation will be available at View Source