Year: 2016

Autolus Limited secures £40 million funding

On March 3, 2016 UCLB spinout, Autolus Limited, a biopharmaceutical company focused on the development and commercialisation of next-generation engineered T-cell therapies for haematological and solid tumours, reported that it has raised £40 million of new capital in a Series B financing round (Press release, UCLB, MAR 3, 2016, View Source [SID:1234509353]).

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Woodford Investment Management LLP ("Woodford") and Perceptive Bioscience Investments Ltd ("Perceptive Bioscience") participated in the new investment, which augments the previous £30 million Seed and Series A investment from founding investor Syncona LLP ("Syncona").

The funds will enable Autolus to develop its proprietary pipeline of engineered T-cell products, and to further implement its industry-leading platform of T-cell programming technologies. In parallel with the financing, the Company’s technology platform was enhanced by a licence to additional technologies from UCLB, UCL’s technology transfer company.

In association with the financing, Dr Joe Anderson, Chief Executive Officer of Perceptive Bioscience, has joined the board of directors of Autolus.

Dr Christian Itin, Chairman of Autolus, said:
"We are pleased to welcome investors of the calibre of Woodford Investment Management and Perceptive Bioscience, which support our goal of building Autolus into a leading engineered T-cell company. The quality of Autolus’ technology and pipeline has allowed the company to raise £70m since its foundation in September 2014, and positions us to take multiple programmes into the clinic. We have also expanded the scope of our licence with UCLB to bring additional inventions from founder Dr Martin Pule’s group into the company adding to the suite of Autolus’ T-cell programming technologies."

Dr Joe Anderson, CEO of Perceptive Bioscience, added:
"We are delighted that Perceptive Bioscience’s first investment is in a company with the potential to transform cancer therapy. Autolus is at the cutting-edge of T-cell engineering to create a new generation of programmed T-cells acting as agents to kill tumour cells. Our investment in Autolus underlines our commitment to supporting emerging, innovative companies. Our flexible investment platform enables us to assist companies with finance and practical support whatever their stage of development."

Dr Martin Murphy, CEO of Syncona, said:
"We are excited that Woodford and Perceptive have invested in Autolus. The company is now funded by a group of investors that share our vision of creating sustainable standalone businesses that will develop products designed to deliver exceptional benefit to patients in areas of high unmet need."

Cengiz Tarhan, Managing Director of UCLB, commented:
"We are delighted to support this significant milestone in Autolus’ development. The licence and the commitment from Syncona, Woodford and Perceptive Bioscience provide a solid foundation to translate the licensed UCL technologies into healthcare benefits for patients."

Autolus is founded upon the work of Dr Martin Pule, an academic clinical haematologist at the UCL Cancer Institute and NIHR University College London Hospitals Biomedical Research Centre and a thought-leader in T-cell engineering. It is a next-generation engineered T-cell company, developing a series of T-cell products based on its proprietary targets, constructs and technologies.

Agenus Reports Fourth Quarter and Full Year 2015 Financial and Operational Results and Recent Highlights

On March 3, 2016 Agenus Inc. (NASDAQ:AGEN), an immuno-oncology company developing checkpoint modulator antibodies and cancer vaccines, reported a corporate update and reported financial results for the fourth quarter and year ended December 31, 2015 (Press release, Agenus, MAR 3, 2016, View Source [SID:1234509351]).

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"During the course of 2015, we broadened our capabilities and advanced our portfolio of checkpoint antibodies and diversified vaccine platforms," commented Garo H. Armen, Ph.D., Chairman and CEO of Agenus. "We made a series of strategic acquisitions, in our efforts to speed discovery and development, as well as to improve the quality of our leads and programs. We also validated our portfolio and discovery capabilities through our strategic alliances with Incyte and Merck. Our greatest asset, however, is our team of scientists with their breadth of expertise.

We believe a multidisciplinary approach is critical because the future of cancer therapy will be based on the ability to identify subpopulations of patients that are likely to best respond to specific immuno-oncology (I/O) agents or combinations. This will require tailored combinations of I/O modalities, including antibodies, vaccines and adjuvants, and cell therapies. At Agenus, we believe that we are uniquely positioned in the I/O landscape by having assembled the critical parts of what could be the future I/O ecosystem.

To support all these endeavors, last year we strengthened our cash position through partnerships, a stock offering and a non-dilutive royalty financing, all affording the company a financial runway through the first half of 2017."

2015 HIGHLIGHTS AND CURRENT UPDATES

January: Leveraged in-house resources through a global license, development and commercialization agreement with Incyte Corporation. The alliance initially focused on the development of four CPM antibodies targeting GITR, OX40, LAG-3 and TIM-3, and was expanded in November 2015 to include three additional undisclosed targets. Agenus and Incyte share costs and profits equally for the GITR and OX40 programs, as well as two of the undisclosed programs. Agenus received a $60 million upfront payment, which included a $35 million equity investment. Agenus is also eligible to receive up to $350 million in development, regulatory and commercial milestones across the initial four lead programs.

April / November: Optimized our discovery of antibodies through acquisition of the Celexion, LLC, SECANT yeast display platform and an agreement with IONTAS for an exclusive license to a proprietary phage display library.

May: Raised approximately $75 million through a public offering of common stock.

June: Strengthened management team with the appointment of C. Evan Ballantyne as the Company’s Chief Financial Officer.
July: Acquired rights to antibodies targeting Carcinoembryonic Antigen Cell Adhesion Molecule 1 (CEACAM1), a glycoprotein expressed on T-cell and NK-cell lymphocytes from Diatheva s.r.l., an Italian biotech company.

September: Raised net proceeds of approximately $78 million in a non-dilutive royalty transaction pursuant to a Note Purchase Agreement with an investor group led by Oberland Capital Management, LLC. The investors received our rights to worldwide royalties on future sales of GlaxoSmithKline’s (GSK) shingles (HZ/su) and malaria (RTS,S) prophylactic vaccine products that contain Agenus’ QS-21 adjuvant to repay principal and interest. At its option, Agenus has the right to buy back the loan at any time under pre-specified terms. Agenus also has the right to receive an additional $15 million in cash from the investors after approval of HZ/su by the FDA, provided such approval occurs by June 30, 2018. This financing also allows Agenus to retain any upside from vaccine royalties remaining after the loan terms are satisfied.

December: Secured our own antibody manufacturing capability through the acquisition of XOMA Corporation’s antibody manufacturing pilot plant and an agreement granting Agenus access to Selexis’ cell line development technologies.

December: Expanded our cancer vaccine program by acquiring privately-held PhosImmune Inc., a company with a portfolio of cancer neoantigens based on aberrant phosphorylation of proteins. The acquisition provides Agenus the ability to potentially accelerate the development of novel off-the-shelf and personalized cancer vaccines and is synergistic with Agenus’ AutoSynVax (ASV) vaccine program for targeting patient-specific tumor neoantigens.

December: Advanced first two checkpoint modulator antibodies toward the clinic by submitting Investigational New Drug (IND) applications to the U.S. Food and Drug Administration (FDA) for AGEN1884 (a CPM antibody that binds to CTLA-4) and INCAGN01876 (a CPM antibody that targets GITR and is partnered with Incyte). In January 2016, the FDA cleared both IND applications.

Upcoming 2016 Program Milestones and Events:

Initiation of Phase 1 clinical trials for anti-CTLA-4 antibody candidate (AGEN1884; wholly owned by Agenus), and for anti-GITR antibody candidate (INCAGN01876, partnered with Incyte) in the first half of 2016.

Initiation of Phase 1 clinical trials for one or more additional CPM antibody candidates in the second half of 2016.

Initiation of a well-controlled randomized Prophage clinical vaccine trial in newly diagnosed glioblastoma patients in the second half of 2016.

Initiation of Phase 1 clinical trial with our first ASV vaccine product candidate in the second half of 2016. Agenus’ ASV program targets cancer neoantigens with an autologous synthetic vaccine approach.

Partner (GSK) filing for regulatory approval of its shingles vaccine candidate containing Agenus’ proprietary QS-21 Stimulon adjuvant in the second half of 2016.

Initiation of combination trials with Agenus vaccines and CPM antibody candidates in the second half of 2016.
Continually evaluate additional strategic alliances and partnerships.

Fourth Quarter and 2015 Financial Results

Cash, cash equivalents and short-term investments were $171.7 million as of December 31, 2015.

For the fourth quarter, Agenus reported a net loss attributable to common stockholders of $15.7 million, or $0.18 per share, basic and diluted, compared with a net loss attributable to common stockholders for the fourth quarter of 2014 of $26.0 million, or $0.41 per share, basic and diluted.

The decreased net loss attributable to common stockholders for the quarter ended December 31, 2015, compared to the net loss attributable to common stockholders for the same period in 2014, was due primarily to a $14.8 million decrease in the non-cash charges related to our contingent obligations as well as increased revenue of $6.0 million related to our Incyte collaboration partially offset by increased expenses related to our CPM programs. We recorded non-cash expenses for the quarter ended December 31, 2014 of $6.5 million, due to the fair value adjustment of the contingent purchase price consideration, and $7.7 million related to the fair value adjustment of our contingent royalty obligation. This compares to non-cash income of $623,000 related to the contingent purchase price consideration for the quarter ended December 31, 2015.

For the year ended December 31, 2015, the company incurred a net loss attributable to common stockholders of $88.1 million, or $1.13 per share, basic and diluted, compared with a net loss attributable to common stockholders of $42.7 million, or $0.71 per share, basic and diluted, compared to the same period in 2014.

The increase in net loss attributable to common stockholders for the year ended December 31, 2015, compared to the net loss attributable to common stockholders for the same period in 2014, was primarily due to the advancement of our check point modulator programs, partially offset by the increased revenues of $17.8 million primarily related to our Incyte collaboration; a $13.2 million charge for the acquisition of the SECANT yeast display platform and other license and technology transfer arrangements. We also recorded a total of $13.6 million in non-cash expense for fair value adjustments to our contingent obligations.

During the same period of 2014, the company recorded non-cash expense of $6.7 million due to the fair value adjustment of the contingent purchase price consideration and non-cash income of $2.1 million related primarily to various GlaxoSmithKline vaccine trial results containing QS-21 Stimulon.

Yondelis® approved for sale in 6 additional countries

On March 3, 2016 Janssen Products, LP reported that it has informed PharmaMar(MSE:PHM) that regulatory authorities in 6 countries have granted 10 new authorisations to sell Yondelis: five for treating relapsed platinum-sensitive ovarian cancer (ROC), in combination with Caelyx (pegylated liposomal doxorubicin), and 6 as monotherapy for treating soft tissue sarcoma (STS) (Press release, PharmaMar, MAR 3, 2016, View Source [SID:1234509347]).

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The six countries that have authorised Yondelis for ROC and/or STS are SaudiArabia, Moldavia, Bangladesh, Brunei, Costa Rica and Kuwait.

As a result, Yondelis is now approved in nearly 80 countries, 31 of which are in the European Economic Area (EEA). The European Commission approved Yondelis for soft tissue sarcoma in 2007, and at the end of 2009 they approved the sale of this
drug in combination with pegylated liposomal doxorubicin for relapsed platinumsensitive ovarian cancer.

In 2015, the U.S. Food and Drug Administration (FDA) gave Janssen Products, LP, the marketing approval for YONDELIS for the treatment of patients with unresectable or metastatic liposarcoma (LPS) or leiomyosarcoma (LMS); and the drug was also approved by the Japanese Minister of Health, Labour and Welfare to Taiho Pharmaceutical Co., Ltd. for the treatment of patients with soft tissue sarcoma.

Yondelis has orphan drug status for soft tissue sarcoma and ovarian cancer in the European Union, the United States, and Switzerland, and for soft tissue sarcoma in Japan and South Korea.

According to the licensing agreement between PharmaMar and Janssen Products, LP, PharmaMar has the rights to sell Yondelis in Europe (including Eastern Europe), while Janssen Products, LP has the rights to sell the drug everywhere else except Japan, where PharmaMar has granted a license to Taiho Pharmaceutical Co., Ltd. for the development and sale of Yondelis.

About YONDELIS (trabectedin)
YONDELIS (trabectedin) is a novel, multimodal, synthetically produced antitumor agent, originally derived from the sea squirt, Ecteinascidia turbinata. The drug exerts its activity by targeting the transcriptional machinery and impairing DNA repair. It is approved in nearly 80 countries in North America, Europe, South America and Asia for the treatment of advanced soft tissue sarcomas as a single-agent and for relapsed ovarian cancer in combination with DOXIL/CAELYX (doxorubicin HCl
liposome injection). Under a licensing agreement with PharmaMar, Janssen Products, L.P. has the rights to develop and sell YONDELIS globally except in Europe, where PharmaMar holds the rights, and in Japan, where PharmaMar has granted a license to Taiho Pharmaceuticals.



8-K – Current report

On February 25, 2016 La Jolla Pharmaceutical Company (NASDAQ: LJPC) (the Company or La Jolla), a leader in the development of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases, reported fourth quarter and full year 2015 financial results and highlighted 2015 corporate progress (Filing, Q4/Annual, La Jolla Pharmaceutical, 2015, MAR 2, 2016, View Source [SID:1234509952]).

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2015 Corporate Progress

• The ATHOS (Angiotensin II for the Treatment of High-Output Shock) 3 trial, La Jolla’s multicenter, randomized, double-blind, placebo-controlled, Phase 3 clinical trial of LJPC-501, La Jolla’s proprietary formulation of angiotensin II, in catecholamine-resistant hypotension (CRH) was initiated in March 2015. The initiation of the ATHOS 3 trial followed the reaching of an agreement with the U.S. Food and Drug Administration (FDA) on a Special Protocol Assessment (SPA), in which the agreed-upon primary efficacy endpoint in ATHOS 3 is increase in blood pressure. ATHOS 3 is enrolling as planned, and results are expected by the end of 2016.

• A multicenter, open-label, dose-escalation Phase 1 clinical trial of LJPC-401, the Company’s novel formulation of hepcidin, in patients at risk for iron overload due to conditions such as hereditary hemochromatosis, beta thalassemia, sickle cell disease and myelodysplastic syndrome was initiated in October 2015. Interim results, reported in January 2016, suggested a dose-dependent reduction in serum iron following a single dose of LJPC-401. Additionally, the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) designated LJPC-401 an orphan medicinal product for the treatment of beta thalassemia intermedia and major.

• La Jolla entered into exclusive worldwide license agreements with the Indiana University Research and Technology Corporation and the University of Alabama at Birmingham to acquire intellectual property rights covering LJPC-30Sa and LJPC-30Sb in May 2015. LJPC-30Sa and LJPC-30Sb are La Jolla’s next-generation gentamicin derivatives for the potential treatment of serious bacterial infections and rare genetic disorders, such as cystic fibrosis and Duchenne muscular dystrophy.

• La Jolla completed a public offering of common stock in September 2015, whereby La Jolla received approximately $104.6 million, net of issuance costs. La Jolla finished 2015 with $126.5 million in cash and cash equivalents and believes this is sufficient to fund operations into 2018.

"2015 was a very exciting year for La Jolla, highlighted by the initiation and continued progress of our Phase 3 clinical trial of LJPC-501 and encouraging interim data from our recently initiated Phase 1 clinical trial of LJPC-401," said George Tidmarsh, M.D., Ph.D., La Jolla’s President and Chief Executive Officer. "We look forward to a productive 2016, with the continued advancement of our exciting product candidates and results from our LJPC-501 Phase 3 clinical trial expected by the end of the year."

Results of Operations

As of December 31, 2015, La Jolla had $126.5 million in cash and cash equivalents, compared to $48.6 million as of December 31, 2014. The increase in cash and cash equivalents was primarily due to cash provided by our common stock offering that was completed in September 2015, which was partially offset by net cash used for operating activities. Based on current operating plans and projections, La Jolla believes that its current cash and cash equivalents are sufficient to fund operations into 2018.

La Jolla’s net cash used for operating activities for the three and twelve months ended December 31, 2015 was $8.5 million and $25.2 million, respectively, compared to net cash used for operating activities of $5.4 million and $12.9 million, respectively, for the same periods in 2014. La Jolla’s net loss for the three and twelve months ended December 31, 2015 was $11.8 million and $41.9 million, or $0.69 per share and $2.68 per share, respectively, compared to a net loss of $6.8 million and $21.3 million, or $0.45 per share and $2.00 per share, respectively, for the same periods in 2014. During the three and twelve months ended December 31, 2015, La Jolla recognized contract revenue of approximately $0.4 million and $1.1 million, respectively, which was pursuant to a services agreement initiated in 2015 under which La Jolla provides research and development services to a related party. The net loss includes non-cash, share-based compensation expense of $2.8 million and $13.1 million for the three and twelve months ended December 31, 2015, respectively, compared to $2.5 million and $9.1 million of noncash, share-based compensation expense, respectively, for the same periods in 2014.

The increases in net cash used for operating activities and net loss in 2015 as compared to 2014 were primarily due to increased development costs associated with the Phase 3 clinical trial of LJPC-501 in catecholamine-resistant hypotension and the costs associated with the initiation of the Phase 1 clinical trial of LJPC-401 in iron overload. In addition, there were increases in personnel and related costs, which were mainly due to the hiring of additional personnel and increased facility costs to support the increased development activities.

Endocyte Reports Fourth Quarter and Year End 2015 Financial Results and Provides Clinical and Pipeline Update

On March 02, 2016 Endocyte, Inc. (NASDAQ:ECYT), a leader in developing targeted small molecule drug conjugates (SMDCs) and companion imaging agents for personalized therapy, reported financial results for the fourth quarter ended December 31, 2015, and provided a clinical update (Press release, Endocyte, MAR 2, 2016, View Source [SID:1234509345]).

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"2015 laid the groundwork for what we expect to be an important year for Endocyte in 2016. After establishing strong proof of concept data with our first generation SMDC vintafolide, the advancements we’ve made with our second generation agents will be tested this year in patients selected by our companion imaging agents," said Ron Ellis, Endocyte’s president and chief executive officer. "These advancements include enhanced potency, higher dosing, extended drug circulation, and improved patient selection criteria enabled by more advanced imaging equipment now widely available. We look forward to providing updates on the efficacy and safety of these agents in targeted patients before the end of the year, as well as on exciting progress on the earlier-stage pipeline, including two new clinical development candidates."

"Having treated more than 60 patients with EC1456 and EC1169, we have been very pleased with the safety profile for both agents. Considering the potency of the warhead we are delivering, this speaks to the stability of the SMDCs and their specificity in targeting diseased cells," commented Alison Armour M.D., Endocyte’s Chief Medical Officer. "We are of course looking forward to reaching optimal dosing and begin evaluation of these agents in patients selected by our companion imaging agents. In the meantime, the imaging team has done some great work advancing the criteria for selecting patients."

EC1456 (Folate-targeted tubulysin) Development

Endocyte outlined the next phase of development for EC1456.

Dose escalation is underway, currently evaluating 2 schedules
Once maximum tolerated dose is determined, these schedules will be evaluated in up to 40 second-line non-small cell lung cancer (NSCLC) patients selected with EC20 imaging with all FR-positive disease
Response rates will be assessed after 15 patients enroll on each schedule to trigger the potential expansion of each cohort to 40
As the optimal schedule becomes evident, cohorts with new indications and combinations with other drugs may be initiated in preparation for randomized trials
In addition to NSCLC, triple-negative breast and ovarian cancers are priority target indications based on sensitivity to the drug payload and prominence of the FR
In NSCLC, the combination of EC1456 with approved PD1 inhibitors is among the most promising paths forward as pre-clinical models demonstrate the most compelling synergy observed by the company to date
EC1169 (PSMA-targeted tubulysin) Development

Endocyte also outlined the next phase of development for EC1169, which is currently in dose escalation with a once a week schedule. Once the maximum tolerated dose is determined, the drug will be evaluated as a single agent therapy in advanced prostate cancer patients previously treated with hormone therapy. The companion imaging agent, EC0652, has provided superior images of prostate-specific membrane antigen (PSMA) positive disease during dose escalation and will provide a valuable tool for patient selection.

Pipeline Programs Leverage SMDC Capability to Create Multiple New Opportunities

"We have been prudent to ensure our two lead programs have remained the priority for the company and that we have fully invested in the clinical activity required to optimize their success," commented Mike Sherman, Endocyte’s chief operating officer and chief financial officer. "With those programs well underway, we also want to make sure we take advantage of our unique SMDC capability and flexibility to extend this platform to include new mechanisms of action and new indications."

Upcoming Expected Milestones

Phase 1 dose escalation updates at ASCO (Free ASCO Whitepaper) (American Society of Clinical Oncology) Annual meeting
EC1456 single agent efficacy data (tumor response) in NSCLC before year-end 2016
EC1169 single agent efficacy data in prostate cancer in late 2016 or 2017
Updates on plans for earlier stage programs
Fourth Quarter 2015 Financial Results

Endocyte reported a net loss of $9.8 million, or $0.23 per basic and diluted share, for the fourth quarter of 2015, compared to a net loss of $8.1 million, or $0.19 per basic and diluted share, for the same period in 2014.

Research and development expenses were $6.4 million for the fourth quarter of 2015, compared to $4.0 million for the same period in 2014. In the fourth quarter of 2014 there was a reduction in the PROCEED termination accrual. With no comparable adjustment in the fourth quarter of 2015, this led to an increase in expense. The increase was also driven by an increase in the expenses related to the EC1456 and EC1169 dose escalation trials which now have more active patients.

General and administrative expenses were $3.5 million for the fourth quarter of 2015, compared to $4.2 million for the same period in 2014. The decrease in expenses was primarily attributable to a reduction in legal and professional fees in the fourth quarter of 2015 as compared to the same period in 2014.

Cash, cash equivalents and investments were $173.6 million at December 31, 2015, compared to $180.3 million at September 30, 2015, and $206.8 million at December 31, 2014.

Financial Expectations

The Company expects that its cash balance at the end of 2016 will be between $125 and $130 million. Spending is expected to increase from the first half of 2016 to the second half as the trials for EC1456 and EC1169 are expanded once the maximum tolerated doses are determined. The advancement of the pre-clinical programs is not expected to materially impact current levels of spending and are reflected in the 2016 cash guidance.