On May 9, 2018 Protagen AG has reported the start of a collaboration with the University of California, San Francisco (UCSF) to utilize Protagen’s SeroTag technology to investigate the immuno-profiling of prostate cancer patients treated with checkpoint inhibitors and therapeutic vaccines (Press release, Protagen, MAY 9, 2018, View Source [SID1234526386]).

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Cancer immunotherapies can be very powerful and provide novel opportunities for the treatment of cancer. However, they currently work for a limited number of indications and patients. In addition, as these therapies re-activate the immune system to fight the cancer, they sometimes can cause severe immune-related Adverse Events (irAEs). Through the collaboration, Protagen and UCSF intend to provide further insight into utilizing immune system profiling to predict treatment response and monitor prostate cancer patients for irAEs, specifically, a so-called cold tumor that it is difficult to target with immuno-therapies.

Dr. Peter Schulz-Knappe, Protagen’s Chief Scientific Officer, commented: "Our proprietary SeroTag technology has enabled patient stratification and immuno-profiling of patients into homogenous disease subgroups for several autoimmune indications. The strong link between immuno-oncology and autoimmune disease, confirmed by the observed irAEs under immunotherapy, provides us with an opportunity to improve immuno-profiling of cancer patients. We feel honored that Dr. Fong and UCSF share this view and we are excited about our collaboration, especially in an indication like prostate cancer that has shown to be difficult to target with immuno-therapies."

Dr. Lawrence Fong, the UCSF Efim Guzik Distinguished Professor in Cancer Biology and leader of the Cancer Immunotherapy Program in the UCSF Helen Diller Family Comprehensive Cancer Center added: "Although cancer immunotherapies can be effective in many different cancers, success in prostate cancer has been more limited. Nevertheless, we know that a small proportion of prostate cancer patients can respond to monotherapies. Immunologic profiling of these patients could enable approaches to patient selection. This collaboration could provide opportunities to accomplish this goal."

Disclaimer

The information stated above was prepared by Protagen AG and reflects solely the opinion of Protagen AG. Nothing in this statement shall be construed to imply any support or endorsement of Protagen AG, or any of its products, by the Regents of the University of California, its officers, agents and employees.

About UC San Francisco (UCSF)

UCSF is a leading university dedicated to promoting health worldwide through advanced biomedical research, graduate-level education in the life sciences and health professions, and excellence in patient care. It includes top ranked graduate schools of dentistry, medicine, nursing and pharmacy; a graduate division with nationally renowned programs in basic, biomedical, transitional and population sciences; and a preeminent biomedical research enterprise.

It also includes UCSF Health, which comprises three top-ranked hospitals, UCSF Medical Center and UCSF Benioff Children’s Hospitals in San Francisco and Oakland, and other partner and affiliated hospitals and healthcare providers throughout the Bay Area.

Please visit www.ucsf.edu/news.

On May 9, 2018 Portola Pharmaceuticals, Inc. (Nasdaq:PTLA) reported financial results for the three months ended March 31, 2018 and provided a corporate update (Press release, Portola Pharmaceuticals, MAY 9, 2018, View Source;p=RssLanding&cat=news&id=2348250 [SID1234526385]).

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"We achieved several major manufacturing and regulatory milestones this past year, including last week’s U.S. approval of Andexxa. We are now concentrating the Company’s efforts on the successful launch of Andexxa and Bevyxxa, which both have the potential to impact public health and become standards of care in the field of thrombosis," said Bill Lis, chief executive officer of Portola. "We are also encouraged by interim data for our next compound for hematologic cancers, cerdulatinib, which will be presented next month at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. Together, these three compounds comprise a leading thrombosis and hematology portfolio, all with global rights."

Recent Achievements, Upcoming Events and Milestones

Andexxa [coagulation factor Xa (recombinant), inactivated-zhzo] – antidote for the reversal of the Factor Xa inhibitors rivaroxaban and apixaban.

Andexxa received Accelerated Approval from the FDA on May 3, 2018 for patients treated with rivaroxaban and apixaban, when reversal of anticoagulation is needed due to life-threatening or uncontrolled bleeding.
Earned an additional $100 million milestone payment from the Company’s royalty-based financing with Health Care Royalty Partners based on the FDA approval of Andexxa in May 2018.
Early Supply Program to launch in June with broader commercial launch anticipated in early 2019, upon FDA approval of the Generation 2 product.
Presented interim data from the ongoing Phase 3b/4 ANNEXA-4 study in a late-breaking clinical trial presentation at the American College of Cardiology’s 67th Annual Scientific Session & Expo (ACC.18). Enrollment on track for completion this summer.
Received a positive CHMP trend vote and working with regulatory authorities to address their accompanying request for additional data.
Built significant Generation 2 product inventory to meet broad demand upon regulatory approval in the U.S. and Europe.
Bevyxxa (betrixaban) – oral, once-daily Factor Xa inhibitor approved for extended prophylaxis of venous thromboembolism (VTE) in acute medically ill patients with risk factors for VTE.

Initiated U.S. commercial launch and continued to expand the field force and market access teams.
Additional results from the APEX trial published in the American Heart Journal and the American Journal of Medicine continue to highlight betrixaban’s effect on symptomatic VTE and VTE-related deaths.
Eight abstracts accepted at the upcoming International Society on Thrombosis and Haemostasis (ISTH) and European Society of Cardiology (ESC) meetings.
Cerdulatinib – an oral, dual-spleen tyrosine kinase (SYK) and janus kinase (JAK) inhibitor in development for the treatment of relapsed/refractory B-cell and other T-cell malignancies in patients who have failed multiple therapies.

Completed enrollment in two of four cohorts of the ongoing Phase 2a study evaluating the safety and efficacy of cerdulatinib in patients with relapsed/refractory B-cell and T-cell malignancies who have failed multiple therapies.
Interim results from the ongoing Phase 2a study accepted for presentation at the 2018 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting.
Received initial feedback from the FDA on the potential regulatory pathway.
First Quarter 2018 Financial Results
Total revenue for the first quarter of 2018 was $6.6 million, compared with $5.1 million for the first quarter of 2017. This includes $6.0 million in collaboration and license revenue earned under Portola’s collaboration and license agreements with Bristol-Myers Squibb Company, Pfizer, Bayer Pharma, Janssen Pharmaceuticals and Daiichi Sankyo, as well as $0.6 million in product revenue from initial sales of Bevyxxa, which was launched in the U.S. in January 2018.

Total operating expenses for the first quarter of 2018 were $91.9 million, compared with $45.7 million for the same period in 2017. Total operating expenses for the first quarter of 2018 included $11.0 million in stock-based compensation expense, compared with $9.0 million for the same period in 2017.

Research and development expenses were $60.1 million for the first quarter of 2018, compared with $30.6 million for the first quarter of 2017. The increase is due to the second Generation 2 commercial manufacturing campaign. Selling, general and administrative expenses for the first quarter of 2018 were $31.5 million, compared with $15.0 million for the same period in 2017. The increase is due to the build-out of the field force and marketing spend for the Bevyxxa launch.

For the first quarter of 2018, Portola reported a net loss of $84.2 million, or $1.28 net loss per share, compared with a net loss of $41.7 million, or $0.74 net loss per share, for the same period in 2017. Shares used to compute net loss per share attributable to common stockholders were 65.5 million for the first quarter of 2018 compared with 56.7 million for the same period in 2017.

Cash, cash equivalents and investments at March 31, 2018 totaled $451.1 million, compared with cash, cash equivalents and investments of $534.2 million as of December 31, 2017.

Based on the FDA approval of Andexxa in May 2018, the Company earned an additional $100 million milestone payment from its royalty-based financing with Health Care Royalty Partners.

Conference Call Details
Portola will host a conference call today, Wednesday, May 9, 2018, at 4:30 p.m. ET, during which time management will provide first quarter 2018 financial results, updates on Andexxa, the U.S. launch of Bevyxxa and other matters. The live call can be accessed by phone by dialing (844) 452-6828 from the U.S. and Canada or 1 (765) 507-2588 internationally and using the passcode 7068059. The webcast can be accessed live on the Investor Relations section of the Company’s website at View Source It will be archived for 30 days following the call.

On May 9, 2018 PharmaCyte Biotech, Inc. (OTCQB: PMCB), a clinical stage biotechnology company focused on developing targeted cellular therapies for cancer and diabetes using its signature live-cell encapsulation technology, Cell-in-a-Box, reported that it has successfully completed the "pore size studies" on its Cell-in-a Box capsules that are required by the U.S. Food and Drug Administration (FDA) (Press release, PharmaCyte Biotech, MAY 9, 2018, View Source [SID1234526384]).

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PharmaCyte’s Chief Executive Officer, Kenneth L. Waggoner, explained the significance of the study saying, "PharmaCyte’s treatment for locally advanced, non-metastatic, inoperable pancreatic cancer (LAPC) utilizes genetically engineered live human cells that produce a particularly potent cytochrome P450 enzyme that is able to activate the chemotherapy prodrug ifosfamide. These cells are encapsulated using the Cell-in-a-Box technology, and the capsules are implanted near the cancerous tumor so that a high local concentration of the cancer-killing ifosfamide metabolite is produced near the tumor.

"Therefore, it is essential that the ifosfamide can easily and quickly enter the capsules so that it can be efficiently converted into the ifosfamide tumor-killing metabolite. It is equally important that this metabolite can then exit the capsules and destroy the tumor. The completed studies clearly demonstrate that this is the case and underscore the stability of the capsules over the freezing, transport and storage cycle."

As part of PharmaCyte’s Investigational New Drug Application (IND) for its clinical trial in patients with LAPC, the FDA required PharmaCyte to provide data showing that the size of the pores in the outer shell of the Cell-in-a-Box capsules is appropriate to allow ifosfamide to enter the interior of the capsules where the ifosfamide-activating cells are located. Additionally, PharmaCyte is required to provide data showing that the pores are also of appropriate size to allow the activated form of ifosfamide to leave the capsules.

The FDA also required PharmaCyte to conduct experiments to demonstrate that the pore size was not affected by the freezing and thawing process of the capsules. To provide the information required by the FDA, a series of laboratory experiments were performed with non-frozen and freshly thawed capsules that were previously frozen and that contained labelled particles. Each set of samples studied contained particles of a particular size, and the appropriate size range was covered by the series of experiments.

Both the release of the particles from the capsules over time and the accumulation of the particles outside the capsules over time were evaluated. The experiments clearly demonstrated that molecules of the size of ifosfamide or its activated cancer-killing form could pass through the capsule’s pores virtually instantaneously. Further, there was no difference detected in the release parameters between freshly produced capsules and those that had been frozen and then thawed.

On May 9, 2018 PDL BioPharma, Inc. (PDL or the Company) (NASDAQ: PDLI) reported financial results for the first quarter ended March 31, 2018 including (Press release, PDL BioPharma, MAY 9, 2018, View Source [SID1234526383]):

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Total revenues of $38.5 million for the three months ended March 31, 2018.

GAAP diluted EPS of $0.01 for the three months ended March 31, 2018.

GAAP net income attributable to PDL’s shareholders of $1.6 million for the three months ended March 31, 2018.

Non-GAAP net income attributable to PDL’s shareholders of $13.4 million for the three months ended March 31, 2018. A full reconciliation of all components of the GAAP to non-GAAP financial results can be found in Table 3 at the end of the release.

Revenue Highlights

Total revenues of $38.5 million for the three months ended March 31, 2018 included:
◦
Product revenues of $23.3 million, which consisted of $18.3 million from sales of Tekturna and Tekturna HCT in the United States, Rasilez and Rasilez HCT in the rest of the world (collectively, the Noden Products) and $5.0 million for product sales of the LENSAR Laser System;
◦
Net royalty payments from acquired royalty rights and a change in fair value of the royalty rights assets of $11.1 million, which consisted of the change in estimated fair value of our royalty right assets, primarily related to the Depomed royalty asset;
◦
Royalties from PDL’s licensees to the Queen et al. patents of $2.8 million, which consisted of royalties earned on sales of Tysabri; and

Interest revenue from note receivable investment to CareView Communications of $0.7 million.

Total revenues decreased by 15 percent or $6.9 million for the three months ended March 31, 2018, when compared to the same period in 2017. The evolution of our revenues reflects PDL’s strategic shift to a specialty biopharmaceutical business model and the residual decline in royalty income from our expired Queen et al. patents.
◦
The 85 percent increase in product revenues was derived from the sales of the Noden Products and the LENSAR Laser System, the latter of which PDL did not begin to recognize until May 2017. Product revenues accounted for approximately 61 percent of total revenues compared to approximately 28 percent in the first quarter of 2017. Rasilez and Rasilez HCT revenues were $7.8 million, which was the first full quarter of revenue recognized from the ex-U.S. commercialization by Noden, having assumed commercialization from Novartis in November 2017;

PDL received $18.6 million in net cash royalties from its royalty rights in the first quarter of 2018, compared to $13.5 million for the same period of 2017. The increase in cash royalties is mainly due to royalties from Glumetza sold by Valeant Pharmaceuticals International, Inc., partially offset by the decrease of royalties from ARIAD Pharmaceuticals, Inc. as royalties ceased when the asset was sold in the first quarter of 2017;

Royalties from PDL’s licensees to the Queen et al. patents were 80 percent or $11.4 million lower than in the first quarter of 2017 as product supply of Tysabri manufactured prior to patent expiry in the United States have been extinguished and ex-U.S. product supplies are rapidly being exhausted; and
◦
The decrease in interest revenues was primarily due to the sale of the kaléo, Inc. note receivable in September 2017.

Operating Expense HighlightsOperating expenses were $34.2 million for the three months ended March 31, 2018, compared to $26.9 million for the same period of 2017. The increase in operating expenses for the three months ended March 31, 2018, as compared to the same period in 2017, was primarily a result of Noden Products and LENSAR contributing additional cost of product revenue of $5.6 million and $2.4 million, respectively, which was the result of increased revenue and recognition of costs of goods for ex-U.S. revenue from Noden Products and increased revenue from LENSAR, which PDL did not begin to recognize until May 2017. Sales and marketing expenses at Noden and LENSAR increased an additional $1.4 million and $1.5 million, respectively, and research and development expenses increased an additional $0.6 million due to LENSAR clinical studies. These increases were partially offset by a decrease in the fair value of acquisition-related contingent consideration of $2.0 million, a decrease in research and development costs for the completion of a pediatric trial for Tekturna and a decrease in general and administration asset management and legal expenses related to the Merck litigation.

Recent Developments

Stock Repurchase Program

From April 1, 2018 to May 8, 2018, the Company repurchased approximately 2.8 million shares of its common stock under the share repurchase program at a weighted average price of $3.03 per share for a total of $8.4 million.

Since the inception of the share repurchase program in March 2018, the Company has repurchased approximately 4.2 million shares of its common stock for a total of $12.6 million.

Approximately $12.4 million remains available under the current share repurchase program.

Other Financial Highlights

PDL had cash, cash equivalents, short-term investments and other investments of $405.1 million at March 31, 2018, compared to $532.1 million at December 31, 2017. The change in cash balance for the quarter was primarily a result of PDL retiring the remaining $126.4 million of principal of its 4.0% Convertible Senior Notes due 2018 at their stated maturity by making a payment to the noteholders of $129.0 million, which included $2.6 million of accrued interest.

Conference Call and Webcast Details

PDL will hold a conference call to discuss financial results at 4:30 p.m. Eastern Time today, May 9, 2018.

To access the live conference call via phone, please dial (800) 668-4132 from the United States and Canada or (224) 357-2196 internationally. The conference ID is 1798597. Please dial in approximately 10 minutes prior to the start of the call. A telephone replay will be available beginning approximately one hour after the call through one week following the call, and may be accessed by dialing (855) 859-2056 from the United States and Canada or (404) 537-3406 internationally. The replay passcode is 1798597.

To access the live and subsequently archived webcast of the conference call, go to the Company’s website at View Source and go to the Investor Relations section and select "Events & Presentations." Please connect to the website at least 15 minutes prior to the call to allow for any software download that may be necessary.

On May 9, 2018 Onconova Therapeutics, Inc. (NASDAQ:ONTX), a Phase 3-stage biopharmaceutical company focused on discovering and developing novel products to treat cancer, with a primary focus on myelodysplastic syndromes, reported that the Company will release its first quarter 2018 financial results on May 15, 2018 before the market opens (Press release, Onconova, MAY 9, 2018, View Source [SID1234526382]). The Company will host a conference call on May 15, 2018 at 9:00 a.m. Eastern Time to discuss these results.

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Interested parties may access the call by dialing toll-free (855) 428-5741 from the US or (210) 229-8823 internationally, and using conference ID 2573768.

The call will also be webcast live. Please click here to access the webcast.

A replay will be available at that link until August 31, 2018.