On February 27, 2019 Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) reported financial results for the fourth quarter and full year 2018 and reviewed highlights of its successful year (Press release, Ionis Pharmaceuticals, FEB 27, 2019, View Source [SID1234533727]).

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"We begin 2019 in the strongest position in our 30-year history. Building on this foundation, we believe we are positioned for continued growth," said Stanley T. Crooke, M.D., Ph.D., chairman of the board and chief executive officer of Ionis. "In 2018, we launched TEGSEDI globally through our affiliate, Akcea, adding revenue from TEGSEDI sales to our substantial commercial revenue from SPINRAZA. We also achieved many important milestones in our pipeline, particularly among the medicines within our late-stage pipeline. This week, Novartis exercised its option to license AKCEA-APO(a)-LRx for which we earned $150 million. Novartis plans to initiate a Phase 3 cardiovascular outcomes study and initiation activities are already underway. We and Akcea are finalizing Phase 3 study designs for the AKCEA-TTR-LRx pivotal program that we plan to initiate in the second half of this year. In addition, our late-stage neurological disease programs recently achieved important milestones. Roche is now enrolling patients in the Phase 3 study of IONIS-HTTRx for Huntington’s disease and Biogen is planning to add an additional cohort to the ongoing study of IONIS-SOD1Rx for patients with SOD1-related ALS that has the potential to support marketing approval. We achieved these successes while growing revenues, investing in the commercialization of TEGSEDI, advancing our broad and diverse pipeline, and consistently leading our industry in innovation – demonstrating the success of our business model and robust technology platform."

2018 Financial Results and Highlights

Revenues increased by 17 percent compared to 2017

Total revenue was $600 million compared to $514 million in 2017.

Commercial revenue from SPINRAZA for 2018 was $238 million, more than double compared to 2017.

TEGSEDI sales were $2.2 million in the fourth quarter of 2018, with commercial sales commencing in the EU in October and in the U.S. in December.

Commercial revenue was over 40 percent of total revenue in 2018 compared to less than 25 percent in 2017, reflecting Ionis’ transition to a commercial-stage company.

Achieved third consecutive year of non-GAAP operating profitability

GAAP operating income was $11 million for the fourth quarter and an operating loss of $61 million for the full year 2018, compared to an operating loss of $6 million and operating income of $31 million for the same periods in 2017.

Non-GAAP operating income was $45 million for the fourth quarter and $70 million for the full year 2018, compared to operating income of $16 million and $117 million for the same periods in 2017

Operating expenses increased in 2018 primarily due to investment in the commercialization of TEGSEDI.

Strong financial results trigger the recognition of a significant tax benefit

In 2018, Ionis reported GAAP net income attributable to Ionis common stockholders of $274 million, primarily driven by a $291 million one-time non-cash income tax benefit Ionis recorded in 2018 related to its income tax assets.

Because of Ionis’ strong financial performance over the past few years and its outlook regarding the continued growth of its business, the Company believes it is more likely than not that it will be able to use the significant amount of income tax assets it has accumulated to offset future taxable income.

Substantial cash position of over $2 billion enables continued investment in commercial products and pipeline

2019 Financial Guidance

"Building upon our success in 2018, we expect to continue our momentum this year and beyond. We anticipate both commercial and R&D revenues to contribute to our overall earnings growth this year, potentially making 2019 our fourth consecutive year of non-GAAP operating profitability. And for the first time, due to our strong financial performance in recent years and strong confidence in our future, we are projecting to be profitable on the bottom line on a non-GAAP basis in 2019. We believe our ability to be profitable while investing in commercial activities, fully exploiting our pipeline and advancing our technology, clearly sets us apart from our peers," said Elizabeth L. Hougen, chief financial officer of Ionis.

All non-GAAP amounts referred to in this press release exclude non-cash compensation expense related to equity awards. Please refer to the reconciliation of non-GAAP and GAAP measures, which is provided later in this release. In prior financial results releases, Ionis referred to amounts that excluded non-cash compensation expense related to equity awards as "pro forma". Additionally, Ionis has labeled its prior period financial statements "as revised" to reflect the revenue recognition accounting standard the Company adopted on January 1, 2018.

Business Highlights

·
SPINRAZA – the worldwide standard-of-care for the treatment of all people with spinal muscular atrophy

o
In 2018, SPINRAZA sales nearly doubled to $1.7 billion compared to 2017, driven by growth in the U.S. and outside the U.S., as reported by Biogen.

o
As of the fourth quarter of 2018, more than 6,600 SMA patients from over 40 countries were on SPINRAZA, including commercial patients and patients in the expanded access program and clinical trials.

o
In the fourth quarter of 2018, the number of adult patients on therapy in the U.S. grew by over 20 percent compared to the third quarter, accounting for more than 50 percent of new patient starts in the U.S.

·
TEGSEDI (inotersen) – launch underway in multiple markets for the treatment of polyneuropathy of hereditary transthyretin amyloidosis (hATTR) in adult patients

o
TEGSEDI generated $2.2 million in sales from the U.S. and EU in its first quarter of launch.

o
PTC Therapeutics, Ionis and Akcea’s commercialization partner in Latin America, filed for marketing authorization for TEGSEDI in Brazil and was granted priority review.

Key Upcoming Events

·
Roche plans to present data from the OLE study of IONIS-HTTRx in patients with Huntington’s disease in 2019.
·
Biogen plans to present data from the completed portions of the Phase 1/2 study of IONIS-SOD1Rx in 2019.
·
Ongoing regulatory discussions on WAYLIVRA in the EU, and if approved, launch.
·
Ionis and its partners plan to report data from numerous Phase 2 studies including IONIS-FXIRx, IONIS-HBVRx and IONIS-GHR-LRx.

Revenue

Ionis’ revenue in the three months and year ended December 31, 2018 was $192 million and $600 million, respectively, compared to $168 million and $514 million for the same periods in 2017 and was comprised of the following (amounts in millions):

The increase in revenue in 2018 compared to 2017 was primarily due to increasing commercial revenue from SPINRAZA royalties, which more than doubled. Additionally, Ionis earned more than $2 million from TEGSEDI product sales in the fourth quarter of 2018.

Ionis’ R&D revenue demonstrates the Company’s ability to generate sustainable revenue from its numerous partnerships. R&D revenue from the amortization of upfront payments increased over $25 million in 2018 compared to 2017. The increase in amortization was primarily due to Ionis’ 2018 strategic neurology collaboration with Biogen. Also, in 2018, Ionis added amortization revenue from its new collaboration with Roche to develop IONIS-FB-LRx. Ionis’ R&D revenue from milestone payments, license fees and other services for 2018 continued to make a significant contribution to Ionis’ financial results.

Already in the first quarter of 2019, Ionis has earned $185 million. The Company earned $150 million from Novartis when it licensed AKCEA-APO(a)-LRx and $35 million from Roche when it enrolled the first patient in the Phase 3 study of IONIS-HTTRx in patients with Huntington’s disease.

Operating Expenses

Operating expenses for the three months and year ended December 31, 2018 on a GAAP basis were $181 million and $661 million, respectively, and on a non-GAAP basis were $147 million and $530 million, respectively. These amounts compare to GAAP operating expenses for the three months and year ended December 31, 2017 of $174 million and $483 million, respectively, and non-GAAP operating expenses of $152 million and $397 million, respectively. The full year increase in operating expenses in 2018 compared to 2017 was principally due to Ionis’ investments in the global launch of TEGSEDI. The Company’s SG&A expenses also increased due to an increase in fees the Company owed under its in-licensing agreements related to SPINRAZA, due to increased SPINRAZA product sales.

Income Tax Benefit

Ionis reported an income tax benefit of $292 million and $291 million for the three months and year ended December 31, 2018, respectively, compared to $7 million and $6 million for the same periods in 2017. Ionis’ tax benefit increased significantly in 2018 primarily due to a one-time non-cash tax benefit related to its deferred income tax assets. In the fourth quarter of 2018, the Company released a large portion of the valuation allowance associated with its deferred tax assets. Because of Ionis’ strong financial performance over the past few years and its outlook regarding the continued growth of its business, the Company determined that it is more likely than not that it will be able to utilize most of the deferred income tax assets it has accumulated to offset future taxable income.

Net Loss Attributable to Noncontrolling Interest in Akcea

At December 31, 2018, Ionis owned approximately 75 percent of Akcea. The shares of Akcea third parties own represent an interest in Akcea’s equity that Ionis does not control. However, because Ionis continues to maintain overall control of Akcea through its voting interest, Ionis reflects the assets, liabilities and results of operations of Akcea in Ionis’ consolidated financial statements. Ionis reflects the noncontrolling interest attributable to other owners of Akcea’s common stock in a separate line called "Net loss attributable to noncontrolling interest in Akcea" on Ionis’ statement of operations. Ionis’ net loss attributable to noncontrolling interest in Akcea for the three months and year ended December 31, 2018, was $17 million and $59 million, respectively. Ionis’ net loss attributable to noncontrolling interest in Akcea for the three months and year ended December 31, 2017, was $6 million and $11 million, respectively.

Net Income (Loss) Attributable to Ionis Common Stockholders

Ionis reported net income attributable to Ionis’ common stockholders of $320 million and $274 million for the three months and year ended December 31, 2018, respectively, compared to a net loss of $3 million and net income of $0.3 million for the same periods in 2017, all on a GAAP basis. On a non-GAAP basis, Ionis reported net income attributable to Ionis’ common stockholders of $351 million and $394 million for the three months and year ended December 31, 2018, respectively, compared to $18 million and $85 million for the same periods in 2017. The increase in 2018 net income attributable to Ionis’ common stockholders was primarily due to increases in revenue and the income tax benefit Ionis recognized in the fourth quarter of 2018.

For the three months ended December 31, 2018, basic and diluted net income per share were $2.32 and $2.21, respectively. For the year ended December 31, 2018, basic and diluted net income per share were $2.09 and $2.07, respectively. For the three months ended December 31, 2017, basic and diluted net loss per share were each $0.03. For the year ended December 31, 2017, basic and diluted net income per share were each $0.15. All amounts are on a GAAP basis.

Balance Sheet

As of December 31, 2018, Ionis had cash, cash equivalents and short-term investments of $2.1 billion compared to $1.0 billion at December 31, 2017. The increase in Ionis’ cash, cash equivalents and short-term investments was primarily due to the $1 billion Ionis received from Biogen for the 2018 strategic neurology collaboration.

Webcast and Conference Call

Today, at 11:30 a.m. Eastern Time, Ionis will conduct a live webcast conference call to discuss this earnings release and related activities. Interested parties may listen to the call by dialing 877-443-5662 or access the webcast at www.ionispharma.com. A webcast replay will be available for a limited time.

On February 27, 2019 Molecular Templates, Inc., (Nasdaq: MTEM) a clinical stage biopharmaceutical company focused on the discovery and development of Engineered Toxin Bodies (ETBs), a new class of targeted biologic therapies that possess unique mechanisms of action in oncology, reported that new data on its pipeline programs and technology platform will be presented in four posters at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2019, to be held March 29 – Apr 3, 2019 at the Georgia World Congress Center in Atlanta, Georgia (Press release, Molecular Templates, FEB 27, 2019, View Source [SID1234533763]).

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Preclinical data on TAK-169, the company’s CD38-targeted ETB (partnered with Takeda Pharmaceutical Company Limited), will be presented for the first time at the AACR (Free AACR Whitepaper) meeting. This molecule is the most potent ETB that Molecular Templates has developed against any target to date. Importantly, in preclinical models, TAK-169 is active in the presence of daratumumab and active against daratumumab resistant cells.

"We are excited to be presenting new data on multiple pipeline programs as well as potential new applications of our ETB technology platform," said Eric Poma, Ph.D., CEO and CSO of Molecular Templates. "We believe that TAK-169 could be an important new therapy for multiple myeloma patients and these new preclinical data support our rationale for the partnership we announced with Takeda in September 2018. We look forward to the start of clinical development for TAK-169 this year. We believe that data on our CD20 and PD-L1 ETBs support our clinical development plans for those programs. Furthermore, the data on bispecific ETBs highlight the breadth of potential application of our technology, which we expect to drive continued pipeline expansion and partnership opportunities."

Date: Monday, April 1
Time: 1:00pm – 5:00pm Eastern Time
Session: Immunology: Therapeutic Antibodies 3
Abstract #: 2384
Location: Georgia World Congress Center, Exhibit Hall B, Poster Section 25, Poster Board Number 8
Poster Title: TAK-169, an Exceptionally Potent CD38 Targeted Engineered Toxin Body, as a Novel Direct Cell Kill Approach for the Treatment of Multiple Myeloma
Authors: Erin K. Willert1, Garrett L. Robinson1, Jack P. Higgins1, Jensing Liu1, Janice Lee2, Sakeena Syed2, Yuhong Zhang2, Dan Tavares2, Anya Lublinsky2, Nibedita Chattopadhyay2, Haiqing Wang2, Laura Packer2, Pu Shi2, Carole Harbison2, Sanjay Patel2, John Newcomb2
1Molecular Templates Inc., Austin, TX; 2Takeda, Cambridge, MA
Molecular Templates will present on its CD38-targeted ETB, TAK-169. Although CD38 is a poorly internalizing receptor, TAK-169 is able to efficiently internalize and directly kill CD38-expressing cells. This novel mechanism of action may be relevant in patients who have progressed after or are unlikely to respond to CD38-targeted antibody therapy. TAK-169 has demonstrated potent cytotoxicity across a range of myeloma cell lines with a range of CD38 expression in vitro as well as in patient-derived samples including those with previous exposure to daratumumab. Furthermore, TAK-169 retains activity in the presence of excess approved, CD38 targeted therapeutic daratumumab. In xenograft models, complete regressions were observed using both a once-weekly and bi-weekly schedule of TAK-169. Tolerability studies in non-human primates demonstrate that repeat administration is tolerated at doses that are expected to be efficacious. TAK-169 is expected to enter the clinic in 2019.

Date: Monday, April 1
Time: 1:00pm – 5:00pm Eastern Time
Session: Experimental and Molecular Therapeutics: Apoptosis, Necrosis, and Cancer Cell Survival
Abstract #: 2060
Location: Georgia World Congress Center, Exhibit Hall B, Poster Section 9, Poster Board Number 12
Poster Title: Combination of CD20 targeted Engineered Toxin Body, MT-3724, with chemotherapy or IMiDs for the treatment of Non Hodgkin’s Lymphoma
Authors: Jack P. Higgins, Aimee Iberg, Caleigh Howard & Erin K. Willert
MT-3724 is a CD20-targeted ETB that has demonstrated single agent anti-tumor activity in heavily pre-treated relapsed/refractory (R/R) Non-Hodgkin’s lymphoma (NHL) patients in a Phase I clinical study. The combination of MT-3724 with chemotherapeutic agents (doxorubicin, gemcitabine, bendamustine, and vincristine) or an immunomodulatory (IMiD) agent (lenalidomide) all demonstrated additive or synergistic cytotoxicity of NHL cell lines. Additional clinical studies to evaluate MT-3724 as single agent and in combination with gemcitabine and oxaliplatin (GEMOX) or lenalidomide are underway and expected to generate data in 2019.

Date: Tuesday, April 2
Time: 1:00pm – 5:00pm Eastern Time
Session: Experimental and Molecular Therapeutics: Pharmacokinetics and Pharmacodynamics / Preclinical Toxicology
Abstract #: 3900
Location: Georgia World Congress Center, Exhibit Hall B, Poster Section 13, Poster Board Number 20
Poster Title: The Safety and efficacy Profile of a PD-L1 Directed, Engineered Toxin Body, as a Novel Targeted Direct-Cell Kill Approach for the Treatment of PD-L1 Expressing Cancers
Authors: Hilario J. Ramos, Asis K. Sarkar, Sara Le Mar, Brigitte Brieschke, Joseph D. Dekker, Veronica R. Partridge, Pablo A. Maceda, Michaela M. Sousares, Garrett L. Robinson, Aimee Iberg, Shaoyou Chu, Jensing Liu, Jack P. Higgins, Erin K. Willert.
Molecular Templates has developed PD-L1-targeting ETBs as an approach to directly target tumor cells and overcome resistance mechanisms against PD-1 and PD-L1 antibodies. The cytotoxicity delivered by PD-L1-specific ETBs is engineered to be independent of a requirement for tumor infiltrating lymphocytes, high tumor mutational burden, or modulatory effects of the tumor microenvironment. Further, the activity is not dependent on blockade of the PD-1/PD-L1 checkpoint axis. Thus, PD-L1-targeting ETBs represent a distinct class of therapeutics with direct cell-kill mechanism of action and ability for activity in patients who have progressed on current standard of care or checkpoint therapy. In this presentation, we highlight the efficacy and safety profile of MT-6020, a human and cynomolgus cross-reactive, PD-L1 targeted, ETB. MT-6020 binds to cell lines expressing non-human primate PD-L1 and elicits cytotoxic responses comparable to those observed on human tumor target cells. Molecular Templates’ PD-L1 ETB, MT-6035, is built upon the MT-6020 scaffold and can also deliver a viral peptide for cell surface presentation and targeting by a specific antiviral CTL population for a second and complementary mechanism for tumor cell destruction, referred to as antigen seeding. MT-6020 and MT-6035 represent a novel approach to targeting and destroying tumors expressing PD-L1 that is unlikely to be inhibited by resistance mechanisms to current checkpoint inhibitors, is well tolerated in relevant toxicity models, and has the capacity for activity in indications where standard of care has failed. Molecular Templates expects to initiate clinical development of the PD-L1-targeted-ETB (with antigen seeding) in 2H19.

Date: Tuesday, April 2
Time: 8:00am – 12:00pm Eastern Time
Session: Experimental and Molecular Therapeutics: Diagnostics, Biomarkers, and the Tumor Microenvironment
Abstract #: 2984
Location: Georgia World Congress Center, Exhibit Hall B, Poster Section 11, Poster Board Number 6
Poster Title: Design and Characterization of Bispecific Engineered Toxin Bodies for Targeted Cancer Therapy
Authors: Aimee Iberg, Garrett L. Cornelison, Caleigh Howard, Paul Amador, Steven Rivera, Michael Jamaleddine, Garrett L. Robinson, Erin K. Willert
To further expand the therapeutic benefit of its ETB platform, Molecular Templates is characterizing ETBs that are targeted through multiple binding domains. Bispecific ETBs that target two epitopes on the same receptor, or two distinct cell surface molecules both expressed on cancer cells, may allow for enhanced activity profiles. These possibilities include: (i) activity in the presence of a competitive binding protein (ii) sustained activity when one target molecule is shed or downregulated, (iii) synergistic binding events to increase overall potency, and (iv) increased specificity towards cancer over normal tissue. Bispecific ETBs have been generated to engage a variety of target combinations, relevant to both solid and hematologic cancer treatment. MTEM is exploring therapeutically relevant target combinations to facilitate the development of a bispecific clinical lead. By pairing the biology and potency of ETB-mediated killing with the expanded targeting possibilities afforded by bispecific molecules, Molecular Templates aims to develop a new class of therapeutics to benefit cancer patients.

On February 27, 2019 Oncopeptides AB (Nasdaq Stockholm: ONCO) reported that data from its Phase II HORIZON study and Phase I/II ANCHOR study evaluating the company’s novel candidate melflufen, a peptide-conjugated alkylator belonging to the novel class of peptidase enhanced compounds, have been selected as poster presentations at the 2019 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting on 29 March – 3 April 2019 in Atlanta, Georgia, USA (Press release, Oncopeptides, FEB 27, 2019, View Source [SID1234533791]).

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– Data demonstrate tolerability and high level of activity associated with melflufen in relapsed/refractory multiple myeloma (RRMM) patients –

– Early signs of high efficacy are encouraging; studies are ongoing –

"Our rigorous clinical development program is initially focused on our first-in-class candidate melflufen, which is currently being studied in global Phase II studies and the Phase III OCEAN study," said Jakob Lindberg, CEO of Oncopeptides. "We are extremely pleased that these data have been selected for poster presentation at AACR (Free AACR Whitepaper), following their initial presentation at the ASH (Free ASH Whitepaper) 2018 Annual Meeting, and believe that this further validates the promise that melflufen holds for the treatment of patients with advanced multiple myeloma whose treatment options are limited."

Melflufen’s novel mechanism of action works by eliciting increased cytotoxicity in target multiple myeloma (MM) cells and substantially reduced off-target cell toxicity. Melflufen is in a pivotal Phase III study, OCEAN, for its initial indication in RRMM under a Special Protocol Assessment from the US Food and Drug Administration (FDA). Based on ongoing preclinical studies, Oncopeptides is also exploring the potential for the use of melflufen, or other peptide-conjugated drugs, to treat various cancers beyond MM.

The full AACR (Free AACR Whitepaper) 2019 Annual Meeting abstract book can be viewed at www.aacr.org.

The posters we present are found under the title:
HORIZON: Melflufen in patients (pts) with relapsed/refractory multiple myeloma (RRMM) refractory to daratumumab (dara) and/or pomalidomide (pom) (OP-106)

ANCHOR: Melflufen and dexamethasone (dex) plus bortezomib (BTZ) or daratumumab (dara) in patients (pts) with relapsed/refractory multiple myeloma (RRMM) (OP-104)

For more information, please contact:
Jakob Lindberg, CEO of Oncopeptides
Telephone: +46 (0)8 615 20 40
E-mail: [email protected]

Rein Piir, Head of Investor Relations at Oncopeptides
Cell phone: +46 70 853 72 92
E-mail: [email protected]

This information was submitted for publication at 22.30 CET, 27 February 2019

On February 27, 2019 Starpharma (ASX: SPL, OTCQX: SPHRY) reported its interim report and financial results for the half-year ended 31 December 2018 (Press release, Starpharma, FEB 27, 2019, View Source [SID1234533744]).

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Financial Summary

Reported loss of $7.3M (Dec 2017: $6.2M); and
Cash position at 31 December 2018 of $44.4M (June 2018: $51.3M), which excludes the expected $4.0M FY18 R&D tax incentive to be received.
VivaGel

VivaGel BV was licensed to ITF Pharma, Inc., for the US for milestones of up to US$101M in addition to escalating, double-digit royalties;
Starpharma and its partners, Mundipharma and Aspen, undertook extensive preparations for the upcoming launches of VivaGel BV in multiple regions scheduled for the first half of 2019. Extensive marketing activities such as packaging design, sales staff training, promotional material development, and launch meetings occurred in parallel with supply chain activities. Manufactured product has been delivered to Aspen subsequent to half-year end, and manufacturing of product for Europe is well-advanced;
US FDA completed its review of the VivaGel BV NDA and advised it requires confirmatory clinical data prior to approval. In preparation for a meeting with the FDA to discuss the data required, Starpharma has been working closely with expert FDA consultants and is currently awaiting confirmation of the meeting date;
VivaGel condom received final regulatory approval in Japan and Starpharma’s partner, Okamoto, commenced launch activities; and
Positive independent market research was conducted in the US for SPL7013 ophthalmic drops for viral conjunctivitis and a patent was granted for the product.
DEP Drug Delivery

Recruitment activities progressed well for two DEP clinical trials – for DEP docetaxel (phase 2) and DEP cabazitaxel (phase 1 / 2), with new sites opened to support recruitment. Efficacy signals have been observed in a number of patients and both products continue to exhibit a notable lack of bone marrow toxicity and other common side effects including hair-loss, anaphylaxis and oedema;
Final preclinical work for the DEP irinotecan phase 1 / 2 trial was completed, study product was manufactured, and other trial preparations, including CRO/site selection, are well advanced ahead of the planned trial commencement later in FY19;
AstraZeneca’s first patent application on DEP Bcl2/xL inhibitor conjugates was published and included compelling efficacy data on DEP Bcl2/xL inhibitor conjugates, both alone and in combination with market-leading anti-cancer treatments, in various human leukemia models;
DEP irinotecan showed impressive efficacy and safety benefits over standard irinotecan in combination with 5-FU in a human pancreatic cancer model;
DEP docetaxel & DEP cabazitaxel outperformed both gemcitabine & Abraxane in a human pancreatic cancer model; and
A range of DEP radiopharmaceutical and other DEP candidates have been made and are currently undergoing testing in a variety of models.
Starpharma concluded the half-year in a strong financial position with a cash balance of $44.4 million, which does not include the $4.0M FY18 R&D tax incentive expected to be received after 31 December 2018. The net loss after tax for the half-year of $7.3 million (Dec 2017: $6.2 million) reflects investment across the VivaGel and DEP portfolio, including DEP docetaxel, DEP cabazitaxel, and DEP irinotecan. Expenditure includes an increased investment in commercialisation, regulatory and operating costs associated with the US VivaGel BV licence and the preparation for product launch in a number of territories.

Starpharma’s CEO, Dr Jackie Fairley, commented: "We achieved several important milestones in the VivaGel portfolio during the half-year, including signing a US licence for VivaGel BV with ITF Pharma, Inc. We were clearly surprised and disappointed by the FDA’s request for confirmatory data and we are working with expert FDA consultants to expedite this process. In non-US territories, launch activities are in the final stages as we approach the launch of VivaGel BV in Australia and Europe (1HCY19). We also continue to work closely with our partner, Mundipharma, on further registrations to support launches in several other regions."

Commenting on the DEP drug delivery portfolio, Dr Fairley said: "We’re pleased to see efficacy signals once again in both our DEP clinical trials as well as a notable lack of bone marrow toxicity and other common side effects. An abundance of positive data from the DEP platform, both preclinical and clinical, continues to build amid partnered program discussions with a number of multinational and US pharmaceutical companies. Our first partnered program with AstraZeneca for AZD0466 is expected to commence clinical trials this year and preparations at AstraZeneca for this activity continue with priority. This partnered program is just one example of the value that can be generated from our unique DEP platform."

Dr Fairley concluded: "We expect to announce a number of key milestones throughout 2019, in addition to the launch of VivaGel BV in Australia and Europe, and regulatory progress across a range of markets, including the US; launch of the VivaGel condom in Japan; regulatory approval of the VivaGel condom in other regions; and progress across internal and partnered DEP programs including commencement of the DEP irinotecan and AZD0466 clinical trials."

On February 27, 2019 Oncolytics Biotech Inc. (NASDAQ: ONCY) (TSX: ONC), currently developing pelareorep, an intravenously delivered immuno-oncolytic virus, reported the publication of an abstract demonstrating a biomarker for predicting clinical response in patients treated with pelareorep (Press release, Oncolytics Biotech, FEB 27, 2019, View Source [SID1234533792]). This analysis was conducted in patient samples from REO 024; a study of pelareorep and Keytruda in combination with chemotherapy in patients with second-line pancreatic cancer. Detailed results will be presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting taking place March 30 through April 3 in Atlanta, Georgia.

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”With a simple blood draw, this biomarker data allows physicians to understand which patients are likely to respond to treatment, allowing for the design of clinical studies that are cheaper, faster and more likely to succeed,” said Dr. Matt Coffey, President and CEO of Oncolytics Biotech. ”Our biomarker data that we will present at AACR (Free AACR Whitepaper) is immediately applicable to future clinical studies. Investigators should now be able to predict which patients will respond to pelareorep in combination with a checkpoint inhibitor. This biomarker will be evaluated in our clinical studies with checkpoint inhibitors, including both of our multiple myeloma studies, the AWARE-1 breast cancer study, and importantly, in the same setting this data was produced, our phase two second-line pancreatic study in combination with Keytruda.”

"Using patient blood samples from our REO 024 study in second line pancreatic cancer, T cell receptor sequencing was performed with Adaptive Biotechnologies’ immunoSEQ Assay to measure the diversity or clonality of the T cell population,” said Dr. Rita Laeufle, CMO of Oncolytics Biotech. ”Results from this analysis demonstrate that higher clonality after one three-week cycle of treatment can identify patients likely to respond to combination treatment of pelareorep and a checkpoint inhibitor.”

The results suggest that those patients with a statistically significant change in their T cell population demonstrate a clinical benefit from pelareorep treatment. High T cell clonality correlates with progression free survival at baseline (HR=0.05, p=0.01). Moreover, high clonality correlates with overall survival at both baseline (HR=0.124, p=0.01) and after one cycle of treatment (HR=0.08, p=0.01). This research highlights the potential utility of measuring T cell clonality as a predictive and prognostic biomarker of pelareorep therapy.

The abstract, authored by Dr. Grey Wilkinson, a translational scientist at Oncolytics Biotech, and his colleagues, in collaboration with Northwestern University, UT Health San Antonio and Adaptive Biotechnologies, can be found online at View Source!/6812/presentation/4866. Full details from the poster presentation will be announced after it is presented.

Poster Board Number: 1
Presentation Number: 2272
Title: Exploratory analysis of T cell repertoire dynamics upon systemic treatment with the oncolytic virus pelareorep in combination with pembrolizumab and chemotherapy in patients with advanced pancreatic adenocarcinoma
Date: Monday, April 1
Lecture Time: 1:00 p.m. ET – 5:00 p.m. ET
Location: Georgia World Congress Center, Exhibit Hall B, Poster Section 20
Speakers: Grey Wilkinson
Session Category: Clinical Research
Session: Current Developments in Non-invasive Biomarkers for Assessment of Cancer 3

About Pelareorep

Pelareorep is a non-pathogenic, proprietary isolate of the unmodified reovirus: a first-in-class intravenously delivered immuno-oncolytic virus for the treatment of solid tumors and hematological malignancies. The compound induces selective tumor lysis and promotes an inflamed tumor phenotype through innate and adaptive immune responses to treat a variety of cancers and has been demonstrated to be able to escape neutralizing antibodies found in patients.