On August 4, 2015 Soligenix, Inc. (OTCQB: SNGX) (Soligenix or the Company), a late-stage biopharmaceutical company developing products that address unmet medical needs in the areas of inflammation, oncology and biodefense, reported that the European Commission, acting on the positive recommendation from the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP), has granted orphan drug designation to synthetic hypericin (the active pharmaceutical ingredient in SGX301) for the treatment of cutaneous T-cell lymphoma (CTCL), a rare disease and a class of non-Hodgkin’s lymphoma, a type of cancer of the white blood cells that are an integral part of the immune system (Press release, Soligenix, AUG 4, 2015, View Source [SID:1234512917]). SGX301 has previously been granted both orphan drug and fast track designations from the US Food and Drug Administration (FDA) for the first-line treatment of CTCL.

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Soligenix is currently working with leading CTCL centers, as well as with the National Organization for Rare Disorders (NORD) and the Cutaneous Lymphoma Foundation (CLF) to begin a 120 subject pivotal Phase 3 clinical trial with SGX301 in the second half of 2015. SGX301 is a novel, first-in-class, photodynamic therapy utilizing safe, visible light for activation. Synthetic hypericin is a potent photosensitizer which is topically applied to skin lesions and activated by visible fluorescent light. This treatment approach avoids the risk of secondary malignancies (including melanoma) inherent with the frequently employed DNA-damaging chemotherapeutic drugs and other photodynamic therapies that are dependent on ultraviolet exposure. Topical hypericin has demonstrated safety in a Phase 1 clinical study in healthy volunteers. In a Phase 2, double-blind, placebo-controlled clinical study in CTCL patients, the drug was safe and well tolerated, with 58.3% of the CTCL patients responding to SGX301 treatment compared to only 8.3% receiving placebo (p ≤ 0.04).

The European Commission grants orphan designations for medicines that treat a life-threatening or chronically debilitating condition affecting no more than five in 10,000 persons in the EU and where no satisfactory treatment is available. In addition to a 10-year period of marketing exclusivity in the EU after product approval, orphan drug designation provides incentives for companies seeking protocol assistance from the EMA during the product development phase, and direct access to the centralized authorization procedure.

"We are extremely pleased to have received European orphan drug designation for the SGX301 program," stated Christopher J. Schaber, PhD, President & Chief Executive Officer of Soligenix, Inc. "This EU orphan designation, combined with our US orphan and fast track designations, positions SGX301 for an accelerated global regulatory product development pathway to address the significant unmet need that exists for this important indication. We look forward to aggressively advancing our pivotal Phase 3 study of SGX301 in CTCL."

About Cutaneous T-Cell Lymphoma
Cutaneous T-cell lymphoma (CTCL) is a class of non-Hodgkin’s lymphoma (NHL), a type of cancer of the white blood cells that are an integral part of the immune system. Unlike most NHLs which generally involve B-cell lymphocytes (involved in producing antibodies), CTCL is caused by an expansion of malignant T-cell lymphocytes (involved in cell-mediated immunity) normally programmed to migrate to the skin. These malignant cells migrate to the skin, causing various lesions to appear that may change shape as the disease progresses, typically beginning as a rash and eventually forming plaques and tumors. Mortality is related to the stage of CTCL, with median survival generally ranging from about 12 years in the early stages to only 2.5 years when the disease has advanced. There is currently no cure for CTCL.

CTCL constitutes a rare group of NHLs, occurring in about 4% of the approximate 500,000 individuals living with the disease. It is estimated, based upon review of historic published studies and reports and an interpolation of data on the incidence of CTCL that it affects over 20,000 individuals in the US, with approximately 2,800 new cases seen annually.

About SGX301
SGX301 is a novel first-in-class photodynamic therapy utilizing safe visible light for activation. The active ingredient in SGX301 is synthetic hypericin, a potent photosensitizer which is topically applied to skin lesions and then activated by fluorescent light 16 to 24 hours later. Combined with photoactivation, hypericin has demonstrated significant anti-proliferative effects on activated normal human lymphoid cells and inhibited growth of malignant T-cells isolated from CTCL patients. In a published Phase 2 clinical study in CTCL, patients experienced a statistically significant (p ≤ 0.04) improvement with topical hypericin treatment whereas the placebo was ineffective: 58.3% compared to 8.3%, respectively. SGX301 has received orphan drug and fast track designations from the FDA.

On August 5, 2015 Endocyte Inc., a leader in developing targeted small molecule drug conjugates (SMDCs) and companion imaging agents for personalized therapy, reported financial results for the second quarter ending June 30, 2015, and provided a clinical update (Press release, Purdue Research Foundation, AUG 4, 2015, View Source [SID:1234507029]).

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"We are pleased with our continued success in escalating the dose of our folate and PSMA-targeted tubulysin SMDCs," said Ron Ellis, Endocyte’s president and chief executive officer. "Increasing the activity of our agents through higher doses and the use of a more potent drug payload were two key elements of our second generation SMDC strategy. We also continue to hone our patient selection process through refining our use of imaging agents to define targeted patients. We believe that all of these adjustments will increase the likelihood of success as we expand our trials for EC1456 and EC1169, once we determine the maximum tolerated dose."

"We were also pleased to announce today the appointment of Alison Armour, M.D. to the position of Chief Medical Officer," added Mr. Ellis. "Alison strengthens the team at Endocyte with her tremendous experience in both clinical practice and oncology drug development. Her leadership has been directly responsible for the regulatory approval of oncology therapies in the United States and international markets. We are very excited to engage her in the development of our pipeline of SMDCs and diagnostic imaging agents." Dr. Armour was most recently the VP of Oncology at Novartis and GSK.

The Company also announced that the final overall survival results from the Phase 2 TARGET trial of EC145 in NSCLC have been accepted for oral presentation at the IASLC World Conference on Lung Cancer to be held in Denver from September 6-9, 2015. While development efforts have focused on the second generation SMDCs, results of this trial provide insights into the effectiveness of the SMDC platform, particularly in targeting the folate receptor.

Second Quarter 2015 Financial Results

Endocyte reported a net loss of $10.6 million, or $0.25 per basic and diluted share, for the second quarter of 2015, compared to net income of $22.4 million, or $0.54 per basic share and $0.52 per diluted share, for the same period in 2014. The second quarter of 2014 included the recognition of the balance of deferred revenue related to the former collaboration with Merck.

Research and development expenses were $6.7 million for the second quarter of 2015, compared to $19.0 million for the same period in 2014. The decrease in research and development expense was primarily attributable to a decrease in expenses related to the TARGET trial, which is now completed, and the PROCEED trial, which was terminated in May 2014 and for which a termination charge of $4.7 million was recorded in the second quarter of 2014, along with a decrease in manufacturing expenses for vintafolide and etarfolatide related to pre-commercial activity.

General and administrative expenses were $4.1 million for the second quarter of 2015, compared to $8.0 million for the same period in 2014. The decrease in expenses was primarily attributable to a reduction in headcount and ceasing all commercial activities during the second quarter of 2014 following the withdrawal of the marketing applications in Europe.

Cash, cash equivalents and investments were $188.6 million at June 30, 2015, compared to $219.2 million at June 30, 2014, and $206.8 million at December 31, 2014.

Financial Expectations

The Company reiterated its expectation that its 2015 year-end cash balance will exceed $155 million. Spending is expected to increase from current levels as the trials for EC1456 and EC1169 are expanded once the maximum tolerated doses are determined.

On April 4, 2015 Ligand Pharmaceuticals Incorporated (NASDAQ: LGND) reported financial results for the three and six months ended June 30, 2015, and provided an operating forecast and program updates (Press release, Ligand, AUG 4, 2015, View Source [SID:1234507012]).

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Financial highlights for the second quarter of 2015 include:

Total revenues were $18.4 million and royalty revenues were $6.6 million, an increase of 74% and 26%, respectively, compared with the second quarter of 2014.

Adjusted EPS was $1.81 and GAAP EPS was $1.11.

The company generated operating cash of $17.6 million, a significant increase over $6.6 million of operating cash generated in the second quarter of 2014.

A description of adjusted calculations and reconciliation to comparable GAAP financial measures is provided in the accompanying table titled "Adjusted Financial Measures."

"We posted strong second quarter financial results featuring growth in royalty revenues from both of our lead commercial assets, Promacta and Kyprolis, and continued strong operating cash flow. Our second quarter royalty revenue reflects product sales by partners during the first quarter and the resetting of tiered royalty rates that occurs annually on January 1st," said John Higgins, Chief Executive Officer of Ligand. "Progress reported by our partners during the second quarter and subsequent weeks has been tremendous, and includes approval of a pediatric indication for Promacta in the U.S., the start of a new global Phase 3 study with Kyprolis and FDA clearance of Kyprolis for expanded uses. With our recent transaction with Selexis, we now have more than 120 fully-funded programs and more than 70 partners."

Second Quarter 2015 Financial Results

Total revenues for the second quarter of 2015 were $18.4 million, compared with $10.6 million for the same period in 2014. Royalty revenues increased 26% to $6.6 million from $5.2 million for the same period in 2014 primarily due to higher royalties from Promacta and Kyprolis. Material sales were $10.7 million compared with $3.5 million for the same period in 2014 due to an increase in Captisol purchases for use in clinical trials and commercial products. Collaborative and other revenues were $1.1 million compared with $1.9 million for the same period in 2014 due primarily to the timing of milestones and upfront fees earned in the second quarter of 2014.

Cost of goods sold was $2.6 million for the second quarter of 2015 compared with $1.2 million for the same period in 2014, as a result of higher Captisol sales in the second quarter of 2015. Research and development expenses were $4.0 million compared with $2.7 million for the same period of 2014. The increase was primarily due to costs associated with funding internal development programs. General and administrative expenses for the second quarter of 2015 were $7.2 million compared with $5.2 million for the same period in 2014, with the increase primarily due to costs associated with business development activities and non-cash stock-based compensation expense. During the second quarter of 2015 the Company recognized a $28.2 million gain on deconsolidation of Viking Therapeutics primarily related to the equity milestone received upon the close of Viking’s IPO.

Net income for the second quarter of 2015 was $23.6 million, or $1.11 per diluted share, compared with net income for the second quarter of 2014 of $1.6 million, or $0.07 per diluted share. Adjusted net income for the second quarter of 2015 was $38.5 million, or $1.81 per diluted share, compared with adjusted net income for the second quarter of 2014 of $5.2 million, or $0.24 per diluted share.

As of June 30, 2015, Ligand had cash, cash equivalents and short-term and restricted investments of $182.2 million.

Year-to-Date Financial Results

Total revenues for the six months ended June 30, 2015 increased 24% to $33.0 million compared with $26.6 million for the same period in 2014. Royalty revenues increased 29% to $16.9 million from $13.1 million for the same period in 2014 primarily due to higher royalties from Promacta and Kyprolis. Material sales increased to $14.4 million from $9.2 million for the same period of 2014 due to an increase in customer purchases of Captisol for use in clinical trials and commercial products. Collaborative research and development and other revenues were $1.7 million compared with $4.3 million for the same period in 2014 primarily due to timing of upfront fees and milestones earned.

Cost of goods sold was $3.7 million for the first six months of 2015 compared with $3.6 million for the same period in 2014. While material sales were higher in the first six months of 2015 versus last year, related cost of goods sold were flat due to the mix of Captisol sales and slightly lower cost of goods sold overall. Research and development expenses for the first six months of 2015 were $8.0 million compared with $5.8 million for the same period in 2014. The increase was primarily due to costs associated with funding internal development programs and non-cash stock-based compensation expense. General and administrative expenses for the first six months of 2015 were $13.2 million compared with $10.3 million for the same period of 2014. The increase is primarily due to costs associated with business development activities and non-cash stock-based compensation expense.

Net income for the first six months of 2015 was $24.3 million, or $1.16 per diluted share, compared with net income of $3.7 million, or $0.17 per diluted share, for the same period in 2014. Adjusted net income for the first six months of 2015 was $45.4 million, or $2.16 per diluted share, compared with adjusted net income of $12.5 million, or $0.57 per diluted share, in the same period of 2014.

Full-Year and Third Quarter 2015 Financial Forecast

The Company affirms expectations for full-year 2015 total revenues to be between $81.0 million and $83.0 million, and adjusted earnings per diluted share to be between $3.45 and $3.50.

For the second half of 2015, Ligand expects total revenues to be between $48.0 million and $50.0 million, and adjusted earnings per diluted share to be between $1.29 and $1.34. Approximately one-third of this second half revenue and earnings outlook is projected for the third quarter. Adjusted earnings per diluted share guidance excludes changes in contingent liabilities, mark-to-market adjustment for amounts owed to licensors, non-cash stock-based compensation expense, non-cash debt-related costs and pro-rata non-cash net losses of Viking Therapeutics.

Second Quarter and Recent Business Highlights

Partnered Program Progress

Promacta/Revolade

Novartis announced FDA approval of Promacta (eltrombopag) for the treatment of children six years and older with chronic immune thrombocytopenia (cITP) who have had an insufficient response to corticosteroids, immunoglobulins or splenectomy.
Novartis announced that Promacta is now approved in more than 100 countries worldwide for the treatment of adults with cITP.
Novartis announced that the CHMP adopted a positive opinion for Revolade (eltrombopag) for the treatment of adults with acquired severe aplastic anemia (SAA) who were either refractory to prior immunosuppressive therapy or heavily pretreated and are unsuitable for hematopoietic stem cell transplantation. The European Commission will review the CHMP recommendation and is expected to deliver its final decision within three months.
Kyprolis

Amgen announced FDA approval of the sNDA for Kyprolis for injection in combination with Revlimid and dexamethasone for the treatment of patients with multiple myeloma who have received one to three prior lines of therapy. The FDA approved the expanded indication for Kyprolis based on data from the ASPIRE study, which showed that patients treated in the Kyprolis arm lived 50% longer (8.7 months longer) without their disease worsening compared with patients treated with Revlimid and low-dose dexamethasone alone.

Amgen announced initiation of the ARROW trial, a global Phase 3 study evaluating the benefit of Kyprolis administered once-weekly with dexamethasone versus the current FDA approved twice-weekly administration schedule in patients with relapsed and refractory multiple myeloma who have received prior treatment with bortezomib and an immunomodulatory agent.
Amgen announced the submission of the sNDA for Kyprolis in patients with relapsed multiple myeloma who have received at least one prior therapy. The filing was based on data from the Phase 3 head-to-head ENDEAVOR study showing relapsed multiple myeloma patients treated with Kyprolis lived twice as long as those treated with Velcade (18.7 vs. 9.4 months) without the disease worsening.

Amgen announced completion of enrollment in the Phase 3 CLARION study of Kyprolis versus Velcade in newly diagnosed multiple myeloma patients.

Additional Pipeline and Partner Developments

SAGE Therapeutics announced initiation of a Phase 3 open-label expanded access protocol (study 302), designed to evaluate the safety of SAGE-547 in patients with super-refractory status epilepticus (SRSE). In conjunction with this event, Ligand earned a $500,000 milestone payment.

SAGE Therapeutics announced that SAGE-547 demonstrated robust activity, with a 77% response rate in evaluable patients with SRSE, in a successfully completed Phase 1/2 clinical trial.

Spectrum Pharmaceuticals presented data at the 2015 meeting of the American Pharmacists Association showing superior solution stability of EVOMELA over Alkeran (melphalan containing propylene glycol as a co-solvent). EVOMELA was shown to be stable for four hours at room temperature and for up to 48 hours in refrigerated conditions after preparation of infusion solution.
TG Therapeutics announced the first presentation of preclinical data demonstrating single-agent and combination activity with two of Ligand’s IRAK4 inhibitor compounds under development. The data demonstrated that:

Both IRAK4 inhibitor compounds not only inhibited cell proliferation but also induced apoptosis as single agents in various B-cell lymphoma cell lines, potentially elucidating the mechanism of action of and clinical rationale for IRAK4 inhibition.

Both compounds demonstrated marked synergy when combined with the novel targeted kinase inhibitors TGR-1202, TG’s proprietary PI3K delta inhibitor, and with the BTK inhibitor ibrutinib.

Merrimack Pharmaceuticals announced final clinical results for the Phase 1 study of MM-302 in HER2-positive metastatic breast cancer. Results from the trial showed that the group of patients treated with 30 mg/m2 or more of MM-302 alone, in combination with trastuzumab or with trastuzumab and cyclophosphamide, had a median progression free survival (mPFS) of 7.6 months (95% CI: 3.6-10.9 months) and an overall response rate (ORR) of 11%. Of note, 25 patients who had not been previously treated with anthracyclines had an mPFS of 11 months (95% CI: 1.8-13.1 months) and an ORR of 24%.

Viking Therapeutics closed its IPO in May 2015. Ligand is partnered with Viking on five therapeutic programs including SARM (VK5211) and TR-beta (VK2809).

Viking announced the submission of an IND for development of VK5211 in patients with acute hip fracture. Initial studies will evaluate the safety profile of VK5211 in healthy elderly subjects. The trial will enroll approximately 120 subjects and will evaluate the effects of VK5211 on lean body mass after 12 weeks of treatment.

Viking announced plans to conduct a Phase 2 trial of VK2809 in patients with hypercholesterolemia and fatty liver disease in the fourth quarter of 2015. The trial will evaluate the efficacy, safety and tolerability of VK2809 in approximately 100 patients with elevated LDL cholesterol and fatty liver disease.

New Licensing Deals

Ligand entered into a worldwide agreement with Sanofi for the development and commercialization of SAR-125844, a Captisol-enabled program. Ligand will receive undisclosed milestones, tiered royalties and revenue from Captisol material sales. Sanofi is responsible for all development costs relating to the program.
Ligand entered into a new clinical-stage agreement with AiCuris GmbH & Co for an undisclosed anti-infective Captisol-enabled program.

Recent Acquisition

Ligand acquired financial rights to potential future milestones and royalties for more than 15 development programs from Selexis SA for $4 million in cash. The acquired programs include a mix of novel biologics and biosimilars. The programs are in various stages of development ranging from preclinical through Phase 3. Each acquired program is fully funded by a development partner. The acquisition expands Ligand’s portfolio to more than 120 fully-funded assets and increases the number of partners to more than 70. Ligand previously acquired a portfolio of biologic development programs from Selexis in April 2013.

Internal GRA Program Progress

Ligand announced results from a Phase 1b clinical trial with LGD-6972 that demonstrated favorable safety, tolerability and pharmacokinetics in normal healthy volunteers and in subjects with type 2 diabetes mellitus. The trial results also demonstrated a robust, dose-dependent reduction of fasting plasma glucose.
The molecule is believed to be best-in-class in the GRA (glucagon receptor antagonist) field of diabetes research.

Recent Intellectual Property Expansion

Ligand added to the Captisol patent portfolio with another patent grant in Europe. Ligand also had patents recently allowed or granted in the U.S. and China for Captisol-enabled clopidogrel and in Mexico for Captisol-enabled budesonide.
Ligand also had recent allowances in the U.S. for its GRA, SARM and EPO receptor agonist portfolios.

Adjusted Financial Measures

The adjusted financial measures discussed above and in the tables below for the three and six months ended June 30, 2015 and 2014 exclude changes in contingent liabilities, mark-to-market adjustment for amounts owed to licensors, non-cash stock-based compensation expense, non-cash debt-related costs and pro-rata non-cash net losses of Viking Therapeutics.

Management has presented net income, net income per share, income from continuing operations and income from continuing operations per share in accordance with GAAP and on an adjusted basis. Ligand believes that the presentation of adjusted financial measures provides useful supplementary information to investors and reflects amounts that are more closely aligned with the cash profits for the period as the items that are excluded from adjusted net income are all non-cash items. Ligand uses these adjusted financial measures in connection with its own budgeting and financial planning. These adjusted financial measures are in addition to, and not a substitute for, or superior to, measures of financial performance prepared in conformity with GAAP.

On August 4, 2015 Progenics Pharmaceuticals, Inc. (Nasdaq: PGNX) reported that it has entered into an exclusive worldwide licensing agreement with Johns Hopkins University for [18F]DCFPyL ("PyL"), a clinical-stage prostate specific membrane antigen (PSMA)-targeted imaging agent for prostate cancer (Filing, 8-K, Progenics Pharmaceuticals, AUG 4, 2015, View Source [SID:1234507008]). PyL, when used in conjunction with high-resolution PET imaging, has shown potential for use in identifying prostate cancer and sites of relapse. Financial terms of the transaction were not disclosed.

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PyL was developed by a team led by Martin G. Pomper, M.D., Ph.D. at the Johns Hopkins University School of Medicine. An early stage clinical trial of PyL with PET imaging in men with prostate cancer demonstrated uptake of PyL in sites of putative metastatic disease and primary tumors not seen with currently approved imaging techniques, suggesting the potential for high sensitivity and specificity in detecting prostate cancer.

"PSMA-targeted imaging agents have the potential to change how prostate cancer is diagnosed, monitored and treated and have the potential to lead to better outcomes for patients," said Mark Baker, CEO. "Based on studies conducted by Dr. Pomper’s team, PyL, a PET imaging agent, has demonstrated the potential to detect even minimal levels of prostate cancer which will complement our SPECT/CT imaging agent 1404, currently in development. We are excited about the opportunity to advance the development of PyL."

PyL complements Progenics’ existing portfolio of candidates for the detection and treatment of prostate cancer, including 1404, the Company’s lead PSMA-targeted imaging agent that is used in conjunction with widely-available SPECT-CT imaging. Progenics intends to initiate a Phase 3 program for 1404 for initial diagnosis and early monitoring applications, while initially focusing the development of PyL with high resolution PET imaging to detect and localize recurrent disease in patients who have experienced a biochemical relapse.

About PyL for PET Imaging of Prostate Cancer
PyL (also known as [18F]DCFPyL) is a clinical-stage, fluorinated PSMA-targeted PET imaging agent for prostate cancer that was discovered and developed at the Center for Translational Molecular Imaging at the Johns Hopkins University School of Medicine. A proof-of-concept study published in the April 2015 issue of the Journal of Molecular Imaging and Biology demonstrated that PET imaging with PyL showed high levels of PyL uptake in sites of putative metastatic disease and primary tumors, suggesting the potential for high sensitivity and specificity in detecting prostate cancer.

About 1404, an Imaging Compound Targeting Prostate Specific Membrane Antigen

Progenics’ molecular imaging radiopharmaceutical product candidate 1404 targets the extracellular domain of prostate specific membrane antigen (PSMA), a protein amplified on the surface of >95% of prostate cancer cells and a validated target for the detection of primary and metastatic prostate cancer. 1404 is labeled with technetium-99m, a gamma-emitting isotope that is widely available, is easy to prepare, and is attractive for nuclear medicine imaging applications. The image created provides the opportunity to visualize cancer, potentially allowing for improved detection and staging, more precise biopsies, and a targeted treatment plan including active surveillance as a disease management tool.

About Prostate Cancer

Prostate cancer is the second most common form of cancer affecting men in the United States: an estimated one in seven men will be diagnosed with prostate cancer in his lifetime. The American Cancer Society estimates that approximately 220,800 new cases of prostate cancer will be diagnosed and about 27,540 men will die of the disease and that approximately 2.9 million men in the U.S. currently count themselves among prostate cancer survivors.

On August 4, 2015 Portola Pharmaceuticals (Nasdaq:PTLA) provided a corporate update and reported its financial results for the second quarter ended June 30, 2015 (Press release, Portola Pharmaceuticals, AUG 4, 2015, View Source;p=RssLanding&cat=news&id=2075608 [SID:1234507007]).

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"We continue to make solid progress toward near-term significant milestones that will allow us to launch two of our late-stage thrombosis products that target multi-billion dollar hospital-based markets – andexanet alfa in 2016 and betrixaban in 2017. In preparation, we are building our commercial capabilities to launch our products independently, including bringing on a new head of commercial and business development and a head of sales," said William Lis, chief executive officer of Portola. "We also continue to advance the development of our dual kinase inhibitor cerdulatinib, which has demonstrated initial clinical proof-of-concept and good tolerability in our ongoing Phase 1/2a study in patients with relapsed/refractory hematologic cancers who have failed multiple therapies. We plan to have final data from this study by the end of the year."

Recent Achievements

Betrixaban – Potential first-to-market oral Factor Xa inhibitor anticoagulant for extended prophylaxis of venous thromboembolism (VTE) in acute medically ill patients

The Phase 3 APEX Study is on track for completion by the end of the year. Study metrics, including pooled blinded aggregate event rates, remain on target.

The APEX Data Safety Monitoring Committee (DSMC) held its final review on May 4, 2015.
Andexanet Alfa – Factor Xa inhibitor antidote with U.S. Food and Drug Administration (FDA) breakthrough therapy designation and orphan drug status on an Accelerated Approval pathway

Announced complete results from the second part of our Phase 3 ANNEXA-A study, which evaluated the safety and efficacy of andexanet alfa administered as an intravenous bolus followed by a continuous two-hour infusion in healthy volunteers given the Factor Xa inhibitor Eliquis (apixaban). The study achieved all primary and pre-specified secondary endpoints with high statistical significance, demonstrating that andexanet alfa produced rapid reversal of the anticoagulant effect of apixaban and sustained it for the duration of the infusion. The data were presented in a Late-Breaking Clinical Trial oral session at the International Society on Thrombosis and Haemostasis (ISTH) 2015 Congress.
Presented new preclinical data demonstrating that andexanet alfa significantly reduced bleeding and reversed the anticoagulant effects of the Factor Xa inhibitor Xarelto (rivaroxaban), as measured by anti-Factor Xa activity, in an animal model of bleeding. In contrast, the four-factor prothrombin complex concentrate (PCC) Kcentra did not impact bleeding or the anti-Factor Xa coagulation biomarker. Results were presented in an oral session at the ISTH 2015 Congress.
Phase 4 ANNEXA confirmatory study is on track to support the submission of a Biologics License Application (BLA).

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

Presented updated safety and efficacy data from the Phase 1 part of the ongoing Phase 1/2a study demonstrating evidence of clinical activity in patients with relapsed/refractory B-cell malignancies who have failed multiple therapies, including partial responses in patients with chronic lymphocytic leukemia (CLL), follicular lymphoma (FL) and transformed FL. Results, presented in a poster discussion session at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2015 Annual Meeting, showed that cerdulatinib was well tolerated with no dose-limiting toxicities identified.
Continued to dose-escalate in the Phase 1 part of the study to determine the maximum tolerated dose (MTD).
Opened patient enrollment in two clinical expansion cohorts in the Phase 2a part of the study – one in patients with CLL/small lymphocytic leukemia and a second in patients with FL.

Corporate

Expanded our executive team with the appointment of Tao Fu as executive vice president, chief commercial and business officer, to lead Portola’s commercial operations and business development, and the appointment of Randy St. Laurent as vice president of sales.

Planned Upcoming Events and Milestones

Betrixaban

Complete patient enrollment in the APEX Study by the end of 2015.
Report topline APEX Study data in early 2016.
Submit a New Drug Application (NDA) to the FDA in 2016.

Andexanet Alfa

Present topline results from Part 2 of the Phase 3 ANNEXA-R study with rivaroxaban in Q3 2015.
Initiate a Phase 2 study with betrixaban in 2015.
Complete commercial manufacturing process validation for BLA and commercial launch.
Scale-up the second-generation commercial manufacturing process at Lonza to supply expected worldwide demand.
Submit a BLA to the FDA under an Accelerated Approval pathway at the end of 2015.
Continue to enroll patients in the Phase 4 ANNEXA confirmatory study.

Cerdulatinib

Continue to dose-escalate in the Phase 1 part of the Phase 1/2a study to establish the MTD.
Begin enrolling patients in two clinical expansion cohorts in the Phase 2a part of the study.
Second Quarter Financial Results

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

Total operating expenses for the second quarter of 2015 were $61.2 million compared with $33.9 million for the same period in 2014. Total operating expenses for the second quarter of 2015 included $4.8 million in stock-based compensation expense compared with $2.4 million for the second quarter of 2014. Research and development expenses were $52.3 million for the second quarter of 2015 compared with $29.0 million for the second quarter of 2014 as the Company continued to support its manufacturing scale-up in preparation for commercial launch and its work on the second-generation manufacturing process of andexanet alfa, its Phase 3 and 4 studies of andexanet alfa, its Phase 3 APEX Study of betrixaban, and its Phase 1/2a clinical study of cerdulatinib. Selling, general and administrative expenses for the second quarter of 2015 were $8.9 million compared with $4.9 million for the same period in 2014 as the Company increased headcount to support its growth, resulting in higher headcount-related costs including stock-based compensation expense and commercial launch activities.

Portola reported a net loss of $58.3 million, or $(1.12) per share, for the second quarter of 2015 compared with a net loss of $31.4 million, or $(0.76) per share, for the second quarter of 2014. Shares used to compute net loss per share attributable to common stockholders were approximately 52.1 million for the second quarter of 2015 compared with approximately 41.2 million for the same period in 2014.

As of June 30, 2015, cash, cash equivalents and investments totaled $407.6 million compared with cash, cash equivalents and investments of $392.3 million as of December 31, 2014.