On August 9, 2017 Puma Biotechnology, Inc. (NASDAQ: PBYI), a biopharmaceutical company, reported financial results for the second quarter ended June 30, 2017 (Press release, Puma Biotechnology, AUG 9, 2017, View Source [SID1234520131]).

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Unless otherwise stated, all comparisons are for the second quarter and six months ended June 30, 2017, compared to the second quarter and six months ended June 30, 2016.

Based on accounting principles generally accepted in the United States (GAAP), Puma reported a net loss applicable to common stock of $77.8 million, or $2.10 per share, for the second quarter of 2017, compared to a net loss applicable to common stock of $66.6 million, or $2.05 per share, for the second quarter of 2016. Net loss applicable to common stock for the first six months of 2017 was $150.7 million, or $4.08 per share, compared to $137.6 million, or $4.23 per share, for the first six months of 2016.

Non-GAAP adjusted net loss was $50.9 million, or $1.38 per share, for the second quarter of 2017, compared to non-GAAP adjusted net loss of $37.9 million, or $1.17 per share, for the second quarter of 2016. Non-GAAP adjusted net loss for the first six months of 2017 was $94.0 million, or $2.54 per share, compared to non-GAAP adjusted net loss of $79.3 million, or $2.44 per share, for the first six months of 2016. 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 second quarter of 2017 was $45.9 million. Net cash used in operating activities for the first six months of 2017 was $82.0 million. At June 30, 2017, Puma had cash and cash equivalents of $80.8 million and marketable securities of $70.8 million, compared to cash and cash equivalents of $194.5 million and marketable securities of $35.0 million at December 31, 2016.

"During the second quarter of 2017, we achieved a significant milestone for Puma with the U.S. Food and Drug Administration’s (FDA) Oncologic Drugs Advisory Committee meeting, which led to last month’s FDA approval of NERLYNX (neratinib) for the extended adjuvant treatment of HER2-positive early stage breast cancer. This marked a major milestone for breast cancer patients and for Puma," said Alan H. Auerbach, Chairman, Chief Executive Officer and President of Puma. "Despite advances in early stage HER2-positive breast cancer treatment, there continues to be a need to reduce the risk of disease recurrence. NERLYNX has been demonstrated to significantly reduce that risk and offers physicians and their patients another treatment option. NERLYNX is now commercially available by prescription in the United States. We are also working with the European Medicines Agency (EMA) on their review of our marketing authorization application (MAA) for this indication and we expect the Committee for Medicinal Products for Human Use (CHMP), the scientific committee of the EMA, to issue an opinion regarding the MAA for neratinib in the first quarter of 2018."

Mr. Auerbach added, "Also, during the second quarter, we presented data at the 2017 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting from a Phase II trial of neratinib, which highlighted positive results from the TBCRC 022 trial in patients with HER2-positive metastatic breast cancer with brain metastases. In addition, during the quarter, we also achieved the targeted patient enrollment in our Phase III NALA trial of neratinib in patients with HER2-positive metastatic breast cancer who have failed two or more prior lines of HER2-directed treatments (third-line disease) in the setting of metastatic disease. We anticipate that primary analysis of data related to the NALA trial will be available during the first half of 2018.

"In the second half of this year, we anticipate the following clinical milestones: (i) presentation of the 5-year disease free survival (DFS) data from the ExteNET Phase III trial of NERLYNX as an extended adjuvant treatment in HER2-positive early stage breast cancer in the third quarter of 2017 and (ii) reporting additional data in the fourth quarter of 2017 from the Phase II trial of neratinib as an extended adjuvant treatment in HER2-positive early stage breast cancer using loperamide, budesonide and colestipol antidiarrheal prophylaxis."

Operating Expenses

Operating expenses were $78.2 million for the second quarter of 2017, compared to $66.5 million for the second quarter of 2016. Operating expenses for the first six months of 2017 were $151.4 million, compared to $137.7 million for the first six months of 2016.

Selling, General and Administrative Expenses:

Selling, general and administrative (SG&A) expenses were $24.9 million for the second quarter of 2017, compared to $12.3 million for the second quarter of 2016. SG&A expenses for the first six months of 2017 were $43.3 million, compared to $23.3 million for the first six months of 2016. The approximately $20.0 million increase during the first six months of 2017 compared to the first six months of 2016 resulted primarily from increases of approximately $2.6 million for stock-based compensation, $13.7 million for professional fees and expenses, and $2.1 million for payroll and related costs. These increases reflect overall corporate growth.

Research and Development Expenses:

Research and development (R&D) expenses were $53.3 million for the second quarter of 2017, compared to $54.2 million for the second quarter of 2016. R&D expenses for the first six months of 2017 were $108.1 million, compared to $114.4 million for the first six months of 2016. The approximately $6.3 million decrease during the first six months of 2017, compared to the first six months of 2016, resulted primarily from decreases of approximately $4.1 million for stock-based compensation and $3.6 million for clinical trial expenses, offset by increases of $0.9 million for internal clinical development and $0.7 million for consultants and contractors related expenses.

About Puma Biotechnology

Puma Biotechnology, Inc. is a biopharmaceutical company with a focus on the development and commercialization of innovative products to enhance cancer care. The Company in-licenses the global development and commercialization rights to three drug candidates — PB272 (neratinib (oral)), PB272 (neratinib (intravenous)) and PB357. NERLYNX (neratinib) is approved for commercial use by prescription in the United States as extended adjuvant therapy for early stage HER2-positive breast cancer following adjuvant trastuzumab-based therapy and is marketed as NERLYNX. Nertatinib is a potent irreversible tyrosine kinase inhibitor that blocks signal transduction through the epidermal growth factor receptors, HER1, HER2 and HER4. Currently, the Company is primarily focused on the commercialization of NERLYNX and the continued development of its other advanced drug candidates directed at the treatment of HER2-positive breast cancer. The Company believes that NERLYNX has clinical application in the treatment of several other cancers as well, including non-small cell lung cancer and other tumor types that over-express or have a mutation in HER2. Further information about Puma Biotechnology can be found at www.pumabiotechnology.com.

IMPORTANT SAFETY INFORMATION

NERLYNX (neratinib) tablets, for oral use

INDICATIONS AND USAGE: NERLYNX is a kinase inhibitor indicated for the extended adjuvant treatment of adult patients with early-stage HER2 overexpressed/amplified breast cancer, to follow adjuvant trastuzumab-based therapy.

CONTRAINDICATIONS: None

WARNINGS AND PRECAUTIONS:

• Diarrhea: Aggressively manage diarrhea occurring despite recommended prophylaxis with additional antidiarrheals, fluids, and electrolytes as clinically indicated. Withhold NERLYNX in patients experiencing severe and/or persistent diarrhea. Permanently discontinue NERLYNX in patients experiencing Grade 4 diarrhea or Grade ≥ 2 diarrhea that occurs after maximal dose reduction.

• Hepatotoxicity: Monitor liver function tests monthly for the first 3 months of treatment, then every 3 months while on treatment and as clinically indicated. Withhold NERLYNX in patients experiencing Grade 3 liver abnormalities and permanently discontinue NERLYNX in patients experiencing Grade 4 liver abnormalities.

• Embryo-Fetal Toxicity: NERLYNX can cause fetal harm. Advise patients of potential risk to a fetus and to use effective contraception.

ADVERSE REACTIONS: The most common adverse reactions (≥ 5%) were diarrhea, nausea, abdominal pain, fatigue, vomiting, rash, stomatitis, decreased appetite, muscle spasms, dyspepsia, AST or ALT increase, nail disorder, dry skin, abdominal distention, epistaxis, weight decreased and urinary tract infection.

To report SUSPECTED ADVERSE REACTIONS, contact Puma Biotechnology, Inc. at 1-844-NERLYNX (1-844-637-5969) and www.NERLYNX.com or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch .

DRUG INTERACTIONS:

Gastric acid reducing agents: Avoid concomitant use with proton pump inhibitors (PPI) and H2-receptor antagonists. Separate NERLYNX by 3 hours after antacid dosing.
Strong or moderate CYP3A4 inhibitors: Avoid concomitant use.
Strong or moderate CYP3A4 inducers: Avoid concomitant use.
P-glycoprotein (P-gp) substrates: Monitor for adverse reactions of narrow therapeutic agents that are P-gp substrates when used concomitantly with NERLYNX.
USE IN SPECIFIC POPULATIONS:

• Lactation: Advise women not to breastfeed.

Please see Full Prescribing Information for additional safety information.

On August 9, 2017 Intrexon Corporation (NYSE: XON), a leader in the engineering and industrialization of biology to improve the quality of life and health of the planet, reported its second quarter and first half financial results for 2017 (Press release, Intrexon, AUG 9, 2017, View Source [SID1234520130]).

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Business Highlights and Recent Developments:

Accelerated plans to move the healthcare business into Precigen, Inc., a wholly owned subsidiary of Intrexon transitioning into a fully-integrated biotherapeutics company and leading player in gene and cell therapy. The consolidation of health-related assets is ongoing and the Company is on track to complete this project by year-end 2017;
After attaining commercially relevant yields on two high-value industrial molecules, isbobutyraldehyde and 2,3 butanediol (2,3 BDO), retained Moelis & Company to advise on strategic and financial options, later converting the assignment to a transactional objective;
Announced EnviroFlight, LLC, Intrexon’s joint venture with Darling Ingredients Inc. (NYSE: DAR), plans to build the largest commercial-scale black soldier (BSF) larvae production facility in the United States, significantly expanding the production of advanced ingredients for sustainable feed and nutrition derived from BSF with initial capacity expected in the first quarter of 2018;
Entered into an exclusive collaboration with Johnson Matthey (LSE: JMAT), a global leader in science that enables cleaner air, improved health and more efficient use of natural resources, focused on the development of microbial strains for fermentative production of peptide-based active pharmaceutical ingredients;
Entered into a research collaboration agreement with Huvepharma EOOD, a global pharmaceutical company, for the utilization and first commercial application of Intrexon’s proprietary fungal expression platform to produce a novel animal feed enzyme;
AquaBounty Technologies, Inc. (NASDAQ: AQB), a majority-owned subsidiary of Intrexon, entered into an agreement to purchase certain assets of Bell Fish Company, including its land-based farming facility in Albany, Indiana, and recently achieved a major milestone with the first sales of eco-friendly AquAdvantage salmon in Canada;
Completed the acquisition of GenVec, Inc. and announced plans to integrate GenVec’s industry-leading gene delivery system with Intrexon’s synthetic biology technologies to develop a next generation adenoviral platform with a significantly higher payload capacity that exceeds 30kb as compared to current viral delivery methods ranging from 4.5kb – 9kb;
Announced development update on next-generation chimeric antigen receptor T cell (CAR-T) therapy for cancer in strategic collaboration with ZIOPHARM Oncology, Inc. (NASDAQ: ZIOP) and the biopharmaceutical division of Merck KGaA, Darmstadt, Germany, using Sleeping Beauty non-viral gene integration and the proprietary RheoSwitch Therapeutic System (RTS) platform to regulate expression of membrane-bound interleukin-15 co-expressed with CAR targets;
Collaborator ZIOPHARM announced the initiation of enrollment in the stereotactic arm of its Phase 1 multicenter study of Ad-RTS-hIL-12 + veledimex in patients with recurrent glioblastoma (rGBM), which will serve as a lead-in to their next phase of development for this controlled IL-12 gene therapy in brain tumors, including planned anti‑PD‑1 combination therapy and pediatric studies;
Collaborator ZIOPHARM announced updated positive Phase 1 results at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting for its lead gene therapy product candidate, Ad-RTS-hIL-12 + veledimex, for the treatment of rGBM with median overall survival in the expanded 20 mg cohort of 12.5 months, with a mean follow-up time of 9.2 months;
Collaborator ZIOPHARM announced acceptance by the U.S. FDA of investigator-initiated Investigational New Drug application for a Phase 1 trial of CD33-specific CAR-T cell therapy targeting relapsed or refractory acute myeloid leukemia and expects the first patient to begin treatment in the third quarter of 2017;
For second product candidate developed under the Exclusive Channel Collaboration with Intrexon, collaborator Fibrocell Science, Inc. (NASDAQ: FCSC) announced that the FDA has granted Rare Pediatric Disease Designation of FCX-013, which utilizes the RTS platform, for the treatment of moderate to severe localized scleroderma, and highlighted pre-clinical data for FCX-013 at the 20th Annual Meeting of the American Society of Gene & Cell Therapy;
Oxitec, a wholly owned subsidiary of Intrexon, announced a multi-year contract to launch the Friendly Aedes Project in Juiz de Fora, Brazil, the second Brazilian municipality and the first city in the state of Minas Gerais to deploy innovative Friendly Aedes in the fight against dangerous Aedes aegypti mosquitoes – the primary vector of dengue, Zika, chikungunya and yellow fever. The project will initially be implemented covering an area of 10,000 residents chosen because of the high prevalence of dengue. Subsequently, deployment will be expanded to cover 50,000 residents;
Oxitec’s Friendly Aedes received a positive technical evaluation based on standards from the European Food Safety Authority in relation to human health and the environment. The National Institute of Public Health and the Environment in the Netherlands concluded negligible risks releasing Friendly Aedes on the island of Saba. France’s High Council for Biotechnology also published a supportive position;
Oxitec and the Municipality of Santiago de Cali, Colombia announced a memorandum of understanding to deploy Friendly Aedes in the Comuna 16 region, which covers over 104,000 residents, to suppress populations of Aedes aegypti mosquitoes;
Announced the addition of Vinita Gupta to Intrexon’s Board of Directors, as well as several management appointments including Helen Sabzevari, Ph.D. as Head of Research and Development of Precigen, Inc. and as Senior Vice President of Intrexon Human Therapeutics, Dr. Mark Carnegie-Brown as CEO of Oxitec, Jorge Espanha as General Manager of Oxitec’s Brazilian subsidiary, Oxitec do Brasil, and Hadyn Parry as Vice President, Corporate Development Europe, the Middle East, and Africa for Intrexon.
Second Quarter 2017 Financial Highlights:

Total revenues of $54.4 million, an increase of 4% over the second quarter of 2016;
Net loss of $18.7 million attributable to Intrexon, or $(0.16) per basic share, including non-cash charges of $17.4 million;
Adjusted EBITDA of $(2.0) million, or $(0.02) per basic share;
The net change in deferred revenue related to upfront and milestone payments, which represents the cash and stock received from collaborators less the amount of revenue recognized during the period, was a decrease of $9.4 million compared to a net increase of $116.1 million in the second quarter of 2016;
Cash consideration received for reimbursement of research and development services covered 46% of cash operating expenses (exclusive of operating expenses of consolidated subsidiaries);
Total consideration received for technology access fees, reimbursement of research and development services and products and services revenues covered 65% of consolidated cash operating expenses; and
Cash, cash equivalents, and short-term investments totaled $157.2 million, the value of investments in preferred shares totaled $144.7 million, and the value of common equity securities totaled $23.9 million at June 30, 2017.
First Half 2017 Financial Highlights:

Total revenues of $107.9 million, an increase of 13% over the first half of 2016;
Net loss of $50.1 million attributable to Intrexon, or $(0.42) per basic share, including non-cash charges of $42.0 million;
Adjusted EBITDA of $(9.1) million, or $(0.08) per basic share;
The net change in deferred revenue related to upfront and milestone payments, which represents the cash and stock received from collaborators less the amount of revenue recognized during the period, was a decrease of $19.6 million compared to a net increase of $129.6 million in the first half of 2016;
Cash consideration received for reimbursement of research and development services covered 50% of cash operating expenses (exclusive of operating expenses of consolidated subsidiaries); and
Total consideration received for technology access fees, reimbursement of research and development services and products and services revenues covered 65% of consolidated cash operating expenses.
"Through the first half of 2017, the Company has furthered its leadership position in engineered biology, and its efforts have translated into a stable and persistent machine that draws from multiple, world-leading technology platforms to generate high-value solutions addressing significant needs across multiple sectors. That so much may be accomplished on a modest expenditure of our shareholder capital is a testament to our unique business and organizational models and to the quality and dedication of our team," commented Randal J. Kirk, Chairman and Chief Executive Officer of Intrexon.

"Looking to the remainder of this year, we expect to see accelerated rollout of our Friendly Aedes solution, the commercial launch of Arctic apples, the initiation of additional innovative gene and cell therapy trials along with data from existing trials, and progress of several types in our methane bioconversion platform, in crop protection, in AquAdvantage Salmon production as well as in numerous other areas in which our team is engaged. Moreover, while executing on our existing programs always is our top priority, we are very pleased by the resurgence of interest in our space and the shifting away from the incrementalism that has until recently featured so strongly among many commercial-stage companies that operate in the sectors in which we are active," concluded Mr. Kirk.

Second Quarter 2017 Financial Results Compared to Prior Year Period

Total revenues increased $1.9 million, or 4%, over the quarter ended June 30, 2016. Collaboration and licensing revenues increased $0.7 million from the quarter ended June 30, 2016 due to the recognition of deferred revenue associated with the payment received in June 2016 from ZIOPHARM to amend the collaborations between the parties which was partially offset by a decrease in research and development services as the Company temporarily redeployed certain resources towards supporting prospective new platforms and additional collaborations. Product revenues decreased $0.9 million, or 8%, primarily due to a decrease in the quantities of pregnant cows and live calves sold due to lower customer demand for these products. Gross margin on products improved in the current period primarily due to a decline in the average cost of cows. Service revenues increased $2.0 million, or 14%, due to an increase in the number of bovine in vitro fertilization cycles performed due to higher customer demand. Gross margin on services decreased slightly in the current period primarily due to an increase in royalties and commissions due to vendors.

Research and development expenses increased $5.6 million, or 20%, due primarily to increases in (i) salaries, benefits and other personnel costs for research and development employees, (ii) lab supplies and consulting expenses, and (iii) depreciation and amortization. Salaries, benefits and other personnel costs increased $2.2 million due to an increase in research and development headcount to support new, expanded and prospective collaborations, and to support additional platform technology development. Lab supplies and consulting expenses increased $1.7 million as a result of (i) the progression of certain programs into the preclinical and clinical phases with certain of Intrexon’s collaborators, and (ii) the expansion or improvement of certain of the Company’s platform technologies. Depreciation and amortization increased $1.0 million primarily as a result of the amortization of developed technology acquired from Oxitec. Selling, general and administrative (SG&A) expenses increased $8.6 million, or 28%. Salaries, benefits and other personnel costs increased $6.4 million primarily due to (i) increased headcount to support the Company’s expanding operations and (ii) the reversal of previously recognized stock-based compensation expense for departed employees in the quarter ended June 30, 2016. Legal and professional fees increased $1.7 million primarily due to (i) increased legal fees to defend ongoing litigation and (ii) the Company’s acquisition of GenVec that was completed in June 2017.

Total other income (expense), net, increased $40.5 million, or 181%. This increase was primarily attributable to (i) increases in fair market value of the Company’s equity securities portfolio, investments in preferred stock and other convertible instruments and (ii) dividend income from the Company’s investments in preferred stock.

First Half 2017 Financial Results Compared to Prior Year Period

Total revenues increased $12.0 million, or 13%, over the six months ended June 30, 2016. Collaboration and licensing revenues increased $9.7 million from the six months ended June 30, 2016, due to the recognition of deferred revenue associated with the payment received in June 2016 from ZIOPHARM to amend the collaborations between the parties. Product revenues decreased $1.3 million, or 7%, primarily due to a decrease in the quantities of pregnant cows and live calves sold due to lower customer demand for these products. Gross margin on products improved in the current period primarily due to a decline in the average cost of cows. Service revenues increased $3.3 million, or 14%, due to an increase in the number of bovine in vitro fertilization cycles performed due to higher customer demand. Gross margin on services decreased slightly in the current period primarily due to an increase in royalties and commissions due to vendors.

Research and development expenses increased $14.0 million, or 26%, due primarily to increases in (i) salaries, benefits and other personnel costs for research and development employees, (ii) lab supplies and consulting expenses, and (iii) depreciation and amortization. Salaries, benefits and other personnel costs increased $4.7 million due to an increase in research and development headcount to support new, expanded, and prospective collaborations, and to support additional platform technology development. Lab supplies and consulting expenses increased $5.2 million as a result of (i) the progression of certain programs into the preclinical and clinical phases with certain of Intrexon’s collaborators, and (ii) the expansion or improvement of certain of the Company’s platform technologies. Depreciation and amortization increased $2.3 million primarily as a result of the amortization of developed technology acquired from Oxitec. SG&A expenses increased $0.8 million, or 1%. Salaries, benefits and other personnel costs increased $1.5 million primarily due to increased headcount to support the Company’s expanding operations. Legal and professional fees increased $3.9 million primarily due to (i) increased legal fees to defend ongoing litigation and (ii) the Company’s acquisition of GenVec that was completed in June 2017. These increases were offset by $4.2 million in litigation expenses recorded in the prior period arising from the entrance of a court order in Trans Ova Genetics, L.C.’s trial with XY, LLC.

Total other income (expense), net, increased $65.3 million, or 149%. This increase was primarily attributable to (i) increases in fair market value of the Company’s equity securities portfolio, investments in preferred stock and other convertible instruments and (ii) dividend income from the Company’s investments in preferred stock.

On August 9, 2017 Aduro Biotech, Inc. (Nasdaq:ADRO), a biopharmaceutical company with three distinct immunotherapy technologies, reported that the United States Patent and Trademark Office has issued two patents to Aduro related to the activation of the STING (Stimulator of Interferon Genes) Pathway (Press release, Aduro Biotech, AUG 9, 2017, View Source [SID1234520129]). The first, U.S. Patent 9,724,408, jointly owned with the University of California and licensed to Aduro Biotech, covers ADU-S100 (MIW815), an investigational STING Pathway Activator being developed as a cancer therapy, and certain methods for its use. The second patent, U.S. Patent 9,695,212, claims methods of using certain additional cyclic dinucleotides (CDNs) to induce STING-dependent immune responses.

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"As leaders in cutting edge research and development of STING technologies, it is imperative that we protect our discoveries and expertise related to the modulation of the STING pathway," said Stephen T. Isaacs, chairman, president and CEO of Aduro Biotech. "These issued patents strengthen and broaden the intellectual property coverage for fundamental aspects of our clinical CDN, ADU-S100, in addition to protecting our innovative research recognizing the importance of STING as a central mediator in triggering the development of tumor-specific immunity. We have demonstrated preclinically that activating STING promotes an inflamed tumor phenotype, which led to a profound anti-cancer immune response. ADU-S100 is the first STING Pathway Activator to enter into the clinic and is in an ongoing Phase 1 dose escalation study in patients with cutaneously accessible tumors."

The issued patents bolster our patent portfolio for STING, where Aduro owns and licenses families of patents and patent applications for compounds that target the STING receptor, which would expire, or if issued would expire, between 2025 and 2038. These include both U.S. and international patent applications.

About STING Pathway Activator Platform
The Aduro-proprietary STING pathway activator product candidates, including ADU-S100, are synthetic small molecule immune modulators that are designed to target and activate human STING. STING is generally expressed at high levels in immune cells, including dendritic cells. Once activated, the STING receptor initiates a profound innate immune response through multiple pathways, inducing the expression of a broad profile of cytokines, including interferons and chemokines. This subsequently leads to the development of a systemic tumor antigen-specific T cell adaptive immune response.

Data from preclinical studies using multiple models demonstrated that ADU-S100 activates the STING Pathway and induced a durable local and systemic anti-tumor immune response as evidenced by induction of type I interferons (IFNs) and a CD8+ T-cell response. Additionally, preclinical data showed the combination of STING activation in the tumor microenvironment and PD-1 blockade enhanced antitumor efficacy.

ADU-S100 is currently being evaluated in a Phase 1 dose escalation study in patients with cutaneously accessible tumors. An Investigational New Drug application for the combination of ADU-S100 with an anti-PD-1 immune checkpoint antibody was recently cleared by the U.S. Food and Drug Administration, and the Phase 1b clinical study is expected to begin later in 2017.

On August 9, 2017 Progenics Pharmaceuticals, Inc. (Nasdaq:PGNX) reported financial results and provided a business update for the second quarter of 2017 (Press release, Progenics Pharmaceuticals, AUG 9, 2017, View Source [SID1234520124]).

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"We are nearing completion of the NDA for AZEDRA and expect to complete the rolling submission to the FDA this month," said Mark Baker, Chief Executive Officer of Progenics. "The application is based on our Phase 2b data, which suggest that AZEDRA could deliver meaningful benefits to malignant and/or recurrent and/or unresectable pheochromocytoma and paraganglioma patients, who do not currently have approved therapies in the U.S. We announced topline data on March 30th and look forward to presenting the results from this trial at the International Symposium of Pheochromocytoma and Paraganglioma meeting on September 1st. Our PSMA-targeted pipeline, including our imaging agents 1404 and PyL and our therapeutic agent, 1095, continues to progress with the strategic goal to Find Fight and Follow — detect, treat and monitor — prostate cancer. I am especially pleased to see the momentum building in Oral RELISTOR prescriptions, which we believe underscores the long-term potential for that franchise," Mr. Baker added.

Second Quarter and Recent Key Business Highlights

AZEDRA, Ultra-orphan radiotherapeutic candidate

AZEDRA NDA Expected to be Submitted August 2017
Progenics expects to complete the rolling submission of the NDA for AZEDRA in patients with malignant and/or recurrent pheochromocytoma to the FDA in August 2017. The filing will be supported by data from a pivotal Phase 2b trial, which met the primary endpoint evaluating the proportion of patients who achieved a 50% or greater reduction of all antihypertensive medications for at least six months. In addition, favorable data was reported for a key secondary endpoint, the proportion of patients with overall tumor response as measured by Response Evaluation Criteria in Solid Tumors (RECIST) criteria. AZEDRA was also shown to be safe and generally well tolerated.

AZEDRA holds Breakthrough Therapy and Orphan Drug statuses, as well as a Fast Track designation in the U.S. under a Special Protocol Assessment agreement with the FDA.
Additional Data from Phase 2b Trial of AZEDRA to be Presented at ISP 2017
In September 2017, Progenics will present further results from its Phase 2b study evaluating AZEDRA, for which topline data were announced in March 2017.
PSMA-Targeted Prostate Cancer Pipeline

Data Demonstrating Potential Use of Automated Bone Scan Index Presented at ASCO (Free ASCO Whitepaper) 2017 Meeting
In June 2017, two presentations highlighting the utility of aBSI were presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper)’s Annual Meeting.
• In an oral presentation, investigators demonstrated the potential of aBSI to serve as quantitative prognostic biomarker for survival in patients with bone-metastatic castration resistant prostate cancer (CPRC). The large scale Phase 3 study demonstrated that in these patients, aBSI at baseline was prognostic for overall and disease specific survival (p <0.0001), progression free survival (p =0.0024), radiographic progression-free survival (rPFS) (p =0.0061), and symptomatic skeletal related events (SSEs) (p =0.0068).
• An additional poster showed that aBSI could be used to quantitatively assess total tumor burden during the course of disease progression, as called for by Prostate Cancer Working Group criteria. The data presented in the poster was also published in the Journal of Nuclear Medicine, and the Progenics researcher, Dr. Aseem Anand, was selected for the Alavi—Mandell Award for that publication.
Advancing Phase 3 Study of 1404
Enrollment in the Phase 3 study of 1404, a PSMA-targeted imaging agent, is ongoing. The trial is designed to evaluate the specificity of 1404 in identifying patients without clinically significant prostate cancer, as well as its sensitivity to identify patients with clinically significant disease.
Advancing Phase 2/3 Study of PyL
Enrollment is also continuing in the Phase 2/3 study evaluating the diagnostic accuracy of PyL PET/CT imaging in patients with recurrent and/or metastatic prostate cancer. The trial is being conducted in the U.S. and Canada.
Advancing Phase 1 Trial of 1095
Dosing and enrollment are ongoing in the Phase 1 open-label dose escalation study of 1095 in patients with metastatic CRPC who have demonstrated tumor avidity to 1095. The study is being conducted at Memorial Sloan Kettering.
RELISTOR, treatment for opioid-induced constipation (partnered with Valeant Pharmaceuticals International, Inc.)

Second Quarter 2017 RELISTOR Net Sales of $17.3 Million
The second quarter 2017 sales, as reported to Progenics by its partner Valeant, translated to $2.6 million in royalty revenue for Progenics for the quarter. Oral RELISTOR prescriptions increased 68% over the preceding quarter.
Second Quarter 2017 Financial Results

Second quarter revenue totaled $2.8 million, down from $8.5 million in the second quarter of 2016. RELISTOR royalty income was $2.6 million during the second quarter compared to $2.4 million in the corresponding period of 2016. The prior year period included upfront and milestone revenue of $5.0 million under the Bayer license agreement.

Second quarter research and development expenses increased by $3.3 million compared to the corresponding prior year period, resulting primarily from higher clinical trial expenses for PyL and 1404, and costs associated with the preparation of the NDA for AZEDRA. Second quarter general and administrative expenses increased by $0.7 million compared to the corresponding prior year period, primarily attributable to higher costs associated with building our commercial capabilities in preparation of the AZEDRA launch and higher stock-based compensation expense, partially offset by lower depreciation expense. For the three months ended June 30, 2017, Progenics recognized interest expense (including amortization of the debt discount) of $1.1 million related to the RELISTOR royalty-backed loan.

Net loss attributable to Progenics for the quarter was $16.6 million or $0.24 per diluted share, compared to a net loss of $5.6 million or $0.08 per diluted share in the corresponding 2016 period. Progenics ended the quarter with cash and cash equivalents of $114.0 million, a decrease of $24.9 million compared to cash and cash equivalents as of December 31, 2016.

On August 9, 2017 Portola Pharmaceuticals Inc. (Nasdaq:PTLA) reported financial results and provided a corporate update for the quarter ended June 30, 2017 (Press release, Portola Pharmaceuticals, AUG 9, 2017, View Source [SID1234520122]).

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"FDA approval of our first product, Bevyxxa, in the second quarter marked the ultimate milestone for Portola and the millions of patients who could benefit from this important new medicine," said Bill Lis, chief executive officer of Portola. "We resubmitted our BLA for AndexXa in the U.S. and are committed to working closely with the FDA toward approval, and with the EMA for approval of both products in 2018. Based on robust clinical data, both Bevyxxa and AndexXa are potentially life-saving medicines and are highly anticipated by the medical community."

Recent Achievements, Upcoming Events and Milestones

Bevyxxa (betrixaban) -– an oral, once-daily Factor Xa inhibitor approved by the U.S. Food and Drug Administration (FDA) under Priority Review on June 23, 2017

First and only anticoagulant for hospital and extended duration prophylaxis (35 to 42 days) of venous thromboembolism (VTE) in adult patients hospitalized for an acute medical illness who are at risk for thromboembolic complications due to moderate or severe restricted mobility and other risk factors for VTE
Anticipate U.S. launch between September and November 2017
Expect opinion from the Committee for Medicinal Products for Human Use (CHMP) by late 2017 or early 2018
AndexXa (andexanet alfa) – a Factor Xa inhibitor antidote in development for patients treated with a Factor Xa inhibitor when reversal of anticoagulation is needed due to life-threatening bleeding or when urgent surgery is required; designated a Breakthrough Therapy and an Orphan Drug by the FDA

Resubmitted Biologics License Application (BLA) to the FDA on August 3, 2017
Expect to receive an opinion from the CHMP by early 2018
Cerdulatinib – an oral, dual Syk/JAK inhibitor in development to treat relapsed and refractory hematologic cancers

Presented interim data at the International Congress of Malignant Lymphoma and the European Hematology Association (EHA) (Free EHA Whitepaper) from a Phase 2a study evaluating cerdulatnib in patients with relapsed/refractory B-cell malignancies that demonstrated evidence of clinical activity in patients with relapsed/refractory B-cell malignancies; also presented preliminary data suggesting activity in t-cell lymphoma
Second Quarter 2017 Financial Results
Collaboration and license revenue earned under Portola’s collaboration and license agreements with Bristol-Myers Squibb Company and Pfizer, Bayer Pharma, Janssen Pharmaceuticals and Daiichi Sankyo was $3.8 million for the second quarter of 2017 compared with $4.2 million for the second quarter of 2016.

Total operating expenses for the second quarter of 2017 were $69.6 million, compared with $61.9 million for the same period in 2016. Total operating expenses for the second quarter of 2017 included $13.3 million in stock-based compensation expense, compared with $7.6 million for the same period in 2016.

Research and development expenses were $49.3 million for the second quarter of 2017, compared with $44.8 million for the second quarter of 2016. The increase in R&D expenses was largely attributable to an increase in manufacturing costs to produce betrixaban active pharmaceutical ingredient and other program costs related to cerdulatinib.

Selling, general and administrative expenses for the second quarter of 2017 were $20.3 million, compared with $17.0 million for the same period in 2016.

For the second quarter of 2017, Portola reported a net loss of $69.7 million, or $1.22 net loss per share, compared with a net loss of $57.3 million, or $1.02 net loss per share, for the same period in 2016.

Cash, cash equivalents and investments at June 30, 2017 totaled $269.7 million, compared with cash, cash equivalents and investments of $318.8 million as of December 31, 2016.