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.

On August 9, 2017 Pieris Pharmaceuticals, Inc. (NASDAQ: PIRS), a clinical-stage biotechnology company advancing novel biotherapeutics through its proprietary Anticalin technology platform for cancer, respiratory and other diseases, reported financial results for the second quarter of 2017 and provided an update on the Company’s recent developments, including:

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Strategic alliance in respiratory diseases with AstraZeneca: Announcement of a global and transformative alliance in one of the Company’s core therapeutic areas, respiratory diseases, with AstraZeneca, anchored around lead respiratory program PRS-060 (Press release, Pieris Pharmaceuticals, AUG 9, 2017, View Source [SID1234520121]). Pieris plans to dose healthy subjects in the fourth quarter of 2017 in a single-ascending dose trial, followed by a multi-ascending dose trial, under a clinical trial notification to the Therapeutic Goods Administration in Australia. The dosing of the first subject will trigger a milestone payment of $12.5 million by AstraZeneca to Pieris.

IND approval for PRS-343: Notification by FDA of acceptance of the Company’s IND filing for its lead proprietary immuno-oncology (IO) program, PRS-343. The Company is diligently engaged with its clinical trial sites toward initiation of patient dosing in a Phase I study in patients with HER2-positive solid tumors. In April, at the Annual Meeting of the American Association for Cancer Research (AACR) (Free AACR Whitepaper), Pieris presented preclinical data for PRS-343 that further validated the understanding of its differentiated bispecific mechanism of action in the treatment of HER2-positive tumors.

Positive clinical data and progression of PRS-080: Advancement of the Company’s anemia program, PRS-080, including the filing of separate clinical trial applications with the German and Czech Republic regulatory authorities to conduct a Phase IIa trial in functional iron deficient anemia patients with the intention, pending timely regulatory approval, to initiate enrollment of patients during this quarter. During the second quarter, the Company completed a Phase Ib single ascending dose study in anemic chronic kidney disease patients on hemodialysis and presented positive data at the 54th European Renal Association & European Dialysis and Transplant Association Congress in Spain.

Advancing and broadening IO pipeline with Servier: Advancement of the Company’s IO partnership with Servier, including progression of our lead program PRS-332, through preclinical studies, while initiating activities for two of the collaboration programs beyond PRS-332.
"During the second quarter, we continued to build on the momentum with which we began the year, by announcing a global transformative alliance with AstraZeneca in one of our core therapeutic areas, respiratory diseases. We also advanced our strategic collaboration with Servier in immuno-oncology, while strengthening our relationship with ASKA Pharmaceutical, who holds an exclusive development and commercialization option in Japan for our anemia program, PRS-080. Our partnerships have generated nearly $80 million in cash flow in 2017, and together could result in more than $4.4 billion in potential milestone payments plus royalties from future product sales, not to mention opportunities for direct commercial sales for several products in the United States. This year’s transactions are a significant step towards achieving our goal of becoming a fully integrated, immunology-focused, commercial-stage biopharmaceutical company," said Stephen Yoder, President and CEO of Pieris. "We recently received FDA acceptance of our IND filing for our lead and wholly owned IO program, PRS-343, and soon expect to dose our first patient in HER2-positive cancers. We remain on track to advance our lead respiratory program, PRS-060, into a first-in-human trial in the second half of this year in collaboration with AstraZeneca, while advancing PRS-080 into a Phase IIa study in anemia patients. Finally, our balance sheet remains strong, with a financial runway that extends through several critical, clinical-stage value inflection points."

Second Quarter Financial Update:

Cash Position – Cash and cash equivalents totaled $50.3 million as of June 30, 2017, compared to $29.4 million as of December 31, 2016. The increase in cash was driven primarily by a EUR30.0 million (approximately $32.0 million) upfront payment received from Servier and a $2.8 million option payment received from ASKA, offset by $15.2 million of operating cash expenditures during the first half of the year. In addition, in July 2017 the Company received $45.0 million of upfront payments from AstraZeneca.

R&D Expense – Research and development expenses were $5.4 million and $10.8 million for the three and six-month periods ended June 30, 2017, respectively, as compared to $4.5 million and $8.2 million for the three and six-month periods ended June 30, 2016. The Company’s increases in research and development expenses reflect advancement across its pipeline of programs.

G&A Expense – General and administrative expenses were $4.3 million and $8.3 million for the three and six-month periods ended June 30, 2017, respectively, as compared to $2.4 million and $4.3 million for the three and six-month periods ended June 30, 2016. The increase in the 2017 periods as compared to the corresponding periods in 2016 is largely attributable to $1.8 million in transaction fees for the successful close of our license and collaboration agreement with AstraZeneca. Of a more recurring nature, recruiting and personnel related costs are increasing as we continue to build the organization and we increasingly require outside professional services, including for intellectual property and corporate legal work, auditing, finance, communications and in other facets of the business.

Net Loss – Net loss was $10.1 million or ($0.23) per share for the three-month period ended June 30, 2017, compared to a net loss $5.9 million or ($0.14) per share for the three-month period ended June 30, 2016. Net loss was $18.1 million or ($0.42) per share for the six-month period ended June 30, 2017, compared to a net loss $10.0 million or ($0.25) per share for the six-month period ended June 30, 2016.

Upcoming Milestones:

PRS-343: dose first patient in a Phase I multi-ascending dose study involving a range of HER2-positive solid cancers representing unmet medical needs, such as gastrointestinal, bladder and breast cancers.

PRS-080: dose first patient in a Phase IIa trial enrolling FID anemia patients in Germany and the Czech Republic.

PRS-060: complete first-in-human trial activities and dose first subject in a Phase I study, which will be funded by Pieris’ partner, AstraZeneca.

On August 9, 2017 Moleculin Biotech, Inc., (NASDAQ: MBRX) ("Moleculin" or the "Company"), a preclinical pharmaceutical company focused on the development of anti-cancer drug candidates, some of which are based on license agreements with The University of Texas System on behalf of the MD Anderson Cancer Center, reported on several recent FDA approvals for new drugs for the treatment of acute myeloid leukemia (AML) (Press release, Moleculin, AUG 9, 2017, View Source [SID1234520119]).

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Walter Klemp, CEO of Moleculin commented: "the recent approvals of three new drugs (Rydapt, Vyxeos and Idhifa) for the treatment of AML are exciting, since they provide additional options for treatments in defined subpopulations, and because they help underscore the magnitude of the potential opportunity for Annamycin, which we will be studying for relapsed or refractory AML. With regard to AML, Rydapt is approved only for patients with a specific gene mutation, and for use in combination with the standard of care chemotherapy. Vyxeos is approved as an option to the standard of care, but only for specific AML patients, namely those with newly-diagnosed therapy-related acute myeloid leukemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC). Jazz Pharmaceuticals purchased this drug in their $1.5 billion acquisition of Celator Pharmaceuticals.

Mr. Klemp continued: "although FDA approval of both of those drugs was based on overall survival comparisons with a standard of care, Idhifa was approved based on an accelerated clinical trial design that showed a 19% response rate in patients with relapsed or refractory AML and IDH2 mutation. What’s interesting is that Idhifa was approved with a single Phase 1/2 clinical trial based on response rate, not overall survival, and a relatively low response rate at that. Also, the patient population for which it is approved represents only 13% of all AML patients. We look forward to working with FDA on a similar approach for Annamycin — reliance on response rate in an accelerated path — but for a larger population of AML patients."

"While these new drugs make valuable incremental improvements in AML therapy," concluded Mr. Klemp, "most AML patients will still fail to respond to (or relapse from) initial therapy; therefore, our initial clinical development plan will attempt to address the significant unmet need of patients who relapse from, or are refractory to, initial therapy. We also believe that, if Annamycin can demonstrate superior efficacy and safety to the current standard of care, the drug may be able to fill major areas for first-line AML treatment. In the meantime, these transactions serve to remind us of the opportunity for our company if Annamycin shows significant activity in our planned clinical trials."

On August 9, 2017 Heron Therapeutics, Inc. (Nasdaq: HRTX) (the Company or Heron), a commercial-stage biotechnology company focused on developing novel, best-in-class treatments to address some of the most important unmet patient needs, reported financial results for the three and six months ended June 30, 2017 and highlighted recent corporate progress (Press release, Heron Therapeutics, AUG 9, 2017, View Source [SID1234520118]).

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Recent Corporate Progress
Pain Franchise
Initiation of Phase 3 Program for HTX-011 in Postoperative Pain Following Successful End-of-Phase 2 Meeting with FDA. Heron reached a general agreement with the U.S. Food and Drug Administration (FDA) on the design and key elements for HTX-011’s Phase 3 program that will be required to support a New Drug Application (NDA). The program includes two pivotal Phase 3 efficacy studies in bunionectomy and hernia repair and an approximately 200-patient Phase 3 safety and pharmacokinetics study to meet the target patient numbers established by the FDA and to provide further evidence of the broad utility of HTX-011 across multiple surgical models. Importantly, the FDA noted that, beyond the agreed-upon Phase 3 studies, no additional clinical work is needed to meet the "Combination Rule" for fixed-dose combination products. The Phase 3 program is designed to achieve a broad indication for the reduction in postoperative pain for 72 hours and reduce the need for opioid analgesic medications following surgery. Heron recently initiated patient enrollment in its Phase 3 program and anticipates completing the Phase 3 program in the first half of 2018. Heron expects to file an NDA in 2018.

Hired Key Talent. Anita Gupta, D.O., Pharm.D. joined Heron as Senior Vice President, Medical Strategy and Government Affairs and will be a key team member providing medical and regulatory input, and working with governmental agencies to support the use of opioid alternatives for postoperative pain. Dr. Gupta has led influential research, advocacy, community and healthcare policy efforts in pain medicine, anesthesiology and opioid prevention. Dr. Gupta is a board-certified anesthesiologist and internationally recognized pain specialist. She recently served as a member of the FDA’s Anesthetic and Analgesic Drug Products Advisory Committee and is a fellow at Princeton University at the Woodrow Wilson School.

CINV Franchise
SUSTOL Sales Increase. Net product sales of SUSTOL (granisetron) extended-release injection for the three months ended June 30, 2017 were $8.5 million, compared to $3.6 million for the three months ended March 31, 2017.

SUSTOL Guidance Increased. Based on results for the first half of 2017, Heron has increased its full year 2017 net product sales of SUSTOL guidance to a range of $25 to $30 million.

CINVANTI (HTX-019) FDA Action Date in Q4 2017. The FDA set a Prescription Drug User Fee Act (PDUFA) goal date of November 12, 2017 for a decision on the Company’s NDA for CINVANTI.

"The first half of 2017 has been very exciting for Heron with the submission of the CINVANTI NDA, a successful End-of-Phase 2 meeting for HTX-011, and the excellent growth in net product sales of SUSTOL," said Barry D. Quart, Pharm.D., Chief Executive Officer of Heron. "We have made important progress in the launch of SUSTOL and plan to build upon our success with the anticipated approval of CINVANTI by year-end 2017, which would add a second, complementary commercial product."

Financial Results
Net product sales of SUSTOL for the three months ended June 30, 2017 were $8.5 million, which represents 134% sequential quarter-over-quarter growth, compared to the $3.6 million of net product sales of SUSTOL for the three months ended March 31, 2017. Net product sales of SUSTOL for the six months ended June 30, 2017 were $12.1 million.

Heron’s net loss for the three and six months ended June 30, 2017 was $42.8 million and $93.1 million, or $0.80 per share and $1.79 per share, respectively, compared to a net loss of $43.2 million and $76.7 million, or $1.17 per share and $2.09 per share, respectively, for the same periods in 2016. Net loss for the three and six months ended June 30, 2017, included non-cash, stock-based compensation expense of $8.2 million and $16.2 million, respectively, compared to $5.8 million and $11.2 million, respectively, for the same periods in 2016.

Heron’s net cash used for operating activities for the three months ended June 30, 2017 was $32.0 million, compared to $50.6 million for the three months ended March 31, 2017, a decrease of $18.6 million quarter-over-quarter. Net cash used in operating activities for the six months ended June 30, 2017 was $82.6 million, compared to $59.5 million for the same period in 2016. During the three months ended June 30, 2017, Heron repaid $25.0 million in principal on its outstanding secured promissory note payable.

Heron believes that its cash, cash equivalents and short-term investments of $109.3 million and accounts receivable of $18.6 million at June 30, 2017, along with collections from SUSTOL sales after June 30, 2017, provides the Company with funding sufficient to complete the HTX-011 pivotal Phase 3 efficacy studies in the first half of 2018.