On May 4, 2018 Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, is hosting an Investor Day in New York City today. During the event, reported that the company will provide a comprehensive business update and report financial results for the first quarter ended March 31, 2018 (Press release, Agios Pharmaceuticals, MAY 4, 2018, View Source [SID1234526111]). The presentations will highlight how Agios’ drug discovery platform and broad clinical portfolio set Agios on the path to become a sustainable, multi-product biopharmaceutical company. The event will be webcast today starting at 8:00 a.m. ET at investor.agios.com.

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"As we prepare to launch our second Agios-discovered and first wholly owned medicine later this year, we continue to invest in our productive drug discovery engine and advance a robust pipeline of first-in-class medicines," said David Schenkein, M.D., chief executive officer at Agios. "Our first quarter progress against that objective was highlighted by the NDA acceptance of TIBSOVO in IDH1m relapsed or refractory AML and multiple clinical trial initiations, including dosing the first patient with our MAT2A inhibitor AG-270 and the start of our AG-348 pivotal program in PK deficiency."

HIGHLIGHTS FROM INVESTOR DAY PRESENTATIONS

• Communicated a robust research pipeline consisting of nine advanced drug discovery programs against novel targets across oncology, rare genetic diseases and metabolic immuno-oncology with the potential to deliver multiple INDs over the next 24 months.

• Expanded rare genetic disease portfolio:

• The company disclosed active research programs in three rare genetic diseases: phenylketonuria (PKU), erythroid porphyria and Friedreich’s ataxia
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• The most advanced research program is in PKU, where Agios has developed a novel approach to stabilize the mutant phenylalanine hydroxylase (PAH) protein and has demonstrated significantly decreased blood phenylalanine levels in a severe pre-clinical model of the disease. PKU is an autosomal recessive disease caused by mutations in the PAH gene affecting approximately 16,000 patients in the U.S.1

• Updated clinical milestones to advance the development of isocitrate dehydrogenase (IDH) 1 inhibitors in solid tumors:

• Glioma pivotal development strategy expected to be finalized by year-end 2018

• Completion of enrollment of ClarIDHy, a global, registration-enabling randomized Phase 3 study for ivosidenib in IDH1m positive advanced cholangiocarcinoma, accelerated to the first half of 2019

• Announced acceptance of the following presentations at 2018 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting:

• Updated data from the expansion phase of the ongoing Phase 1 study of ivosidenib in IDH1m relapsed or refractory (R/R) acute myeloid leukemia (AML)

• Updated data from the ongoing Phase 1/2 combination trial of enasidenib or ivosidenib with VIDAZA in patients with newly diagnosed AML with an IDH2 or IDH1 mutation ineligible for intensive chemotherapy

• First clinical data from the Phase 1 study of AG-881 in advanced IDHm positive solid tumors, including glioma

• Completed commercial infrastructure build, including the deployment of an expanded sales force, to successfully launch TIBSOVO (ivosidenib) within 48 hours of potential FDA approval.
FIRST QUARTER 2018 HIGHLIGHTS & RECENT PROGRESS

• Initiated ACTIVATE-T, a single-arm pivotal trial for AG-348, in adult pyruvate kinase (PK) deficiency patients who receive regular blood transfusions.

• Initiated PEAK, a global registry, for adult and pediatric patients with PK deficiency.

• Initiated a perioperative ‘window’ trial with ivosidenib and AG-881 in IDHm low-grade glioma to further investigate their effects on brain tumor tissue.

• Initiated a Phase 1 dose-escalation trial for AG-270, a first-in-class methionine adenosyltransferase 2a (MAT2A) inhibitor, in patients with methylthioadenosine phosphorylase (MTAP)-deleted tumors.

• Announced FDA acceptance, priority review and a Prescription Drug User Fee Act (PDUFA) action date of August 21, 2018 for the new drug application (NDA) for TIBSOVO (ivosidenib) for the treatment of patients with R/R AML with an IDH1 mutation.

• Completed an underwritten public offering of 8,152,986 shares of common stock at the offering price of $67.00 per share, resulting in proceeds to the company, net of underwriting discounts and commissions, of approximately $516.2 million.
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UPCOMING 2018 MILESTONES & EXPECTED DATA PRESENTATIONS

The company expects to achieve the following additional milestones in 2018:

Cancer:

Potential approval and commercialization of TIBSOVO (ivosidenib) in the United States for R/R AML with an IDH1 mutation in the third quarter of 2018.

Submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for TIBSOVO (ivosidenib) for the treatment of patients with R/R AML and an IDH1 mutation in the fourth quarter of 2018.

Support, in collaboration with Celgene, the initiation of HO150, an intergroup sponsored, global, registration-enabling Phase 3 trial combining ivosidenib or enasidenib with standard induction and consolidation chemotherapy in frontline AML patients with an IDH1 or IDH2 mutation in the fourth quarter of 2018.

Present updated data from the ongoing Phase 1 combination trial of enasidenib or ivosidenib with standard-of-care intensive chemotherapy in patients with newly diagnosed AML with an IDH2 or IDH1 mutation to the 2018 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition.
Rare Genetic Diseases:

Initiate ACTIVATE, a global, placebo-controlled, pivotal trial for AG-348 in approximately 80 adults with PK deficiency who do not receive regular blood transfusions in the second quarter of 2018.

Initiate a Phase 2 proof of concept trial of AG-348 in thalassemia in the fourth quarter of 2018.
Research:

Submit an investigational new drug (IND) application for our newest development candidate, AG-636, an inhibitor of the metabolic enzyme dihydroorotate dehydrogenase (DHODH) for the treatment of hematologic malignancies in the fourth quarter of 2018.
FIRST QUARTER 2018 FINANCIAL RESULTS & CASH GUIDANCE

Revenue for the quarter ended March 31, 2018 was $8.8 million, which includes $7.4 million of collaboration revenue and $1.4 million of royalty revenue from net sales of IDHIFA. Revenue for the quarter ended March 31, 2017 was $10.5 million and consisted solely of collaboration revenue. The decrease in collaboration revenue recognized for the quarter ended March 31, 2018 compared to the comparable period in 2017 was primarily driven by adoption of the new revenue recognition standard.

Research and development (R&D) expenses were $78.2 million, including $8.6 million of stock-based compensation expense, for the quarter ended March 31, 2018, compared to $62.7 million, including $7.0 million in stock-based compensation expense, for the comparable period in 2017. The increase in R&D expense was primarily attributable to start-up costs for the AG-348 pivotal program in PK deficiency, including the initiation of the ACTIVATE-T trial. R&D expense also increased as a result of the initiation of a Phase 1 dose-escalation study of AG-270, our first-in-class MAT2A inhibitor, and IND enabling activities for AG-636, our DHODH inhibitor.

General and administrative (G&A) expenses were $24.6 million, including $5.9 million of stock-based compensation expense, for the quarter ended March 31, 2018, compared to $14.8 million, including $3.7 million of stock-based compensation expense, for the quarter ended March 31, 2017. The increase in G&A expense was primarily attributable to the growth in our U.S. commercial organization in order to support the expected launch of TIBSOVO (ivosidenib) in the third quarter of 2018.

Net loss for the quarter ended March 31, 2018 was $90.8 million, compared to a net loss of $66.2 million for the quarter ended March 31, 2017.

Cash, cash equivalents and marketable securities as of March 31, 2018 were $994.7 million, compared to $567.8 million as of December 31, 2017. The increase in cash was driven by the net proceeds of $516.2 million from the January follow on offering, $4.4 million of cost reimbursements under our collaboration agreements with Celgene and $12.3 million received from employee stock transactions. This was offset by expenditures to fund operations of $104.8 million during the quarter ended March 31, 2018.

The company expects that its cash, cash equivalents and marketable securities as of March 31, 2018, together with the anticipated product and royalty revenue, anticipated interest income, and anticipated expense reimbursements, but excluding any additional program-specific milestone payments, will enable the company to fund its anticipated operating expenses and capital expenditure requirements through at least the end of 2020.

WEBCAST INFORMATION

The live webcast from today’s event can be accessed under "Events & Presentations" in the Investors section of the company’s website at www.agios.com. The archived webcast will be available on the company’s website after the event.

On May 3, 2018 RhoVac AB ("RhoVac") reported that the company has been awarded the European Commission grant under the EU’s research and innovation programme Horizon 2020, with significant appraisal score of "Very good to Excellent" (Press release, RhoVac, MAY 3, 2018, View Source [SID1234555935]).

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Horizon 2020 EU funding supports Small and Medium-sized Enterprises (SME) with breakthrough innovation projects and a market-creating potential. The highly competitive fund is only offered to SMEs which fall under European Innovation Council’s (EIC) SME definition. The fund is part of the Horizon 2020 work programme 2018-2020, funding high-potential innovations in the EU. The fund offers Europe’s brightest and boldest innovators the funding for breakthrough ideas with the potential to create entirely new markets or revolutionise existing ones. The round of applications in February 2018, has been highly competitive with success rate of 12.6% among 2009 submitted proposals.

RhoVac is pleased to announce that company’s RV001 cancer vaccine project, currently in clinical phase I/II focusing on patients with diagnosed prostate cancer, has been awarded the phase I grant. RhoVac has received a significant total score of 13.80 out of 15, which indicates "Very good to Excellent" in the appraisal scale. Company’s application has received excellent scores for major sub-criterions, such as (but not limited to):

Impact: substantial patient’s need for the innovation, realistic analysis of market, scalability and large market size, potential to create new markets generated by the new innovation, knowledge protection, overall strategy and business model.
Excellence: game-changing and breakthrough innovation and clear development outline
Quality and efficiency of implementation: team commitment with deep technical/management experience and realistic timeframe
The SME instrument will boost fast company growth and market-creating innovation thanks to staged funding and ramped up business acceleration services. Following completion of this phase I, RhoVac is preparing to apply for phase II of the Horizon 2020 grant to be submitted in Q4 2018.

Comments from RhoVac´s CEO, Anders Ljungqvist
-We are delighted to receive this €50,000 grant from the European Commission, but what is most important and rewarding is to see the rating that we are acknowledged for: our innovative approach, our understanding of patient’s need and the high quality in RhoVac’s project management. This rating is highly motivating for us in our continuing effort to further develop the RV001 project. This grant will be funded to prepare a feasibility report, as a requirement by European Commission, which will then be the basis for the upcoming phase II application within the program framework.

On May 3, 2018 Ferring Pharmaceuticals reported the signing of an agreement giving the company the option to secure global commercialisation rights to nadofaragene firadenovec/Syn3 (rAd-IFN/Syn3), a novel gene therapy being developed by FKD Therapies Oy (FKD) as a treatment for patients with high-grade non-muscle invasive bladder cancer (NMIBC), who are unresponsive to Bacillus Calmette-Guérin (BCG) therapy (Press release, Ferring Pharmaceuticals, MAY 3, 2018, View Source [SID1234551851]). This option is exercisable on marketing approval from the US FDA. Ferring will create a new US oncology division with the specialist knowledge and presence to introduce novel advanced therapies to the market.

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rAd-IFN/Syn3 is currently undergoing Phase 3 development in the US under the sponsorship of Finnish gene therapy specialists FKD. The results of the earlier Phase 2 trial, published in the Journal of Clinical Oncology, reported 35% of BCG unresponsive NMIBC bladder cancer patients given one dose of rAd-IFN/Syn3 every three months, were free of high-grade disease at one year1. The ongoing Phase 3 study is designed to establish the efficacy and safety of the product. rAd-IFN/Syn3 has been awarded Fast Track and Breakthrough Therapy designations by the FDA.

"We are excited about the potential to commercialise rAd-IFN/Syn3, a novel gene therapy for bladder cancer patients," said Michel Pettigrew, President of the Executive Board and Chief Operating Officer, Ferring Pharmaceuticals. "The gene therapy sector is growing rapidly and building a presence in this specialised area is a very positive opportunity for Ferring."

Bladder cancer is one of the most frequently occurring cancers with an estimated 430,000 new cases being reported worldwide each year2. It is the fourth most common cancer in men in the US3 and is the most expensive cancer to treat on a life-time basis, with a high burden on patients, their relatives and healthcare systems4. In high-grade NMIBC patients, BCG is the gold standard treatment and although effective, over 60% of cases eventually re-occur5, 6. The outcome for such patients is poor, with total cystectomy (complete removal of the bladder) to prevent the cancer spreading to other organs generally being the next treatment option. As such, the BCG unresponsive population is one of high unmet clinical need.

"Today, bladder cancer patients have very limited medical options and new treatments that delay or prevent total removal of the bladder and improve clinical outcomes are urgently needed for patients," said Professor Klaus Dugi, Chief Medical Officer, Ferring Pharmaceuticals. "Phase 2 clinical results for rAd-IFN/Syn3 were very encouraging and we look forward to the Phase 3 data."

Gene therapy is one of a new class of therapeutic treatments known as advanced therapy medicinal products. rAd-IFN/Syn3 is built on adenoviral vector technology, a non-integrating vector, and results in enhanced expression of the therapeutic protein interferon alfa 2b. To date, it has completed three clinical trials in the US.

About rAd-IFN/Syn3

rAd-IFN/Syn3 (nadofaragene firadenovec/Syn3) is an investigational gene therapy consisting of an adenovirus containing the gene interferon alfa-2b. It is administered by catheter into the bladder, where the virus enters the cells of the bladder wall. Inside the cells, the virus breaks down leaving the active gene to do its work. The internal gene/DNA machinery of the cells picks up the gene and translates its DNA sequence, resulting in the cells secreting high quantities of interferon alfa-2b protein, a naturally occurring protein the body uses to fight cancer. This novel gene therapy approach turns the patient’s own bladder wall cells into multiple interferon microfactories, enhancing the body’s natural defences against the cancer. The Phase 3 trial for rAd-IFN/Syn3 opened in 2016 with up to 150 patients to be enrolled across 35 centers in the US.

On May 3, 2019 Alligator Bioscience (Nasdaq Stockholm: ATORX), a biotechnology company developing antibody-based pharmaceuticals for tumor-directed immunotherapy, reported that it will present pre-clinical data for the drug candidate ATOR-1017 at the Protein Engineering Summit (PEGS) 14th Annual Meeting in Boston, US (Press release, Alligator Bioscience, MAY 3, 2018, View Source [SID1234538678]). ATOR-1017 is a monoclonal antibody that activates the costimulatory receptor 4-1BB expressed on T cells in the tumor area and is being developed for the treatment of metastasizing cancer.

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The data support a best-in-class profile for ATOR-1017, with a strong potential for high efficacy and tumor-directed immune activation.

ATOR-1017 is dependent on cross-linking with Fc-gamma receptors. This means that it has to bind both 4-1BB and Fc receptors for full activity. Both 4-1BB and Fc-gamma receptors are highly expressed in certain tumors and these receptors could be used as predictive efficacy biomarkers. Patients that overexpress these receptors should demonstrate a higher efficacy response to ATOR-1017, resulting in an increased immune activation in the tumors compared to the rest of the body. This supports that ATOR-1017 has the potential for a superior benefit/risk profile.

"I am very encouraged by these pre-clinical data. ATOR-1017 has the ideal profile to become a best-in-class 4-1BB antibody, with strong efficacy and minimal side-effects. This is perfectly in line with Alligator’s strategy to build a pipeline of tumor-directed immunotherapies" said Christina Furebring, SVP Research at Alligator Bioscience.

Today at 4:20 p.m. local EDT (10:20 pm CEST) Dr Peter Ellmark, VP Discovery at Alligator, will give an oral presentation with the title: "Tumor-directed targeting of Effector T cells and Regulatory T cells". Dr Ellmark is chairing the session on "Agonist Immunotherapy Targets" at PEGS.

For further information, please contact:
Cecilia Hofvander, Director Investor Relations & Communications
Phone +46 46 286 44 95
E-mail: [email protected]

The information was submitted for publication, through the agency of the contact person set out above, at 3:00 p.m. CEST on 3 May 2018.

About ATOR-1017
ATOR-1017 is an immunostimulatory antibody (IgG4) that binds to the costimulatory receptor 4-1BB (also known as CD137) expressed on tumor-specific T cells and NK cells. 4-1BB has the capacity to support the immune cells involved in tumor control, making 4-1BB a particularly attractive target for cancer immunotherapy.

ATOR-1017 is differentiated from other 4-1BB antibodies, partly because of its unique binding profile, but also because its immunostimulatory function is dependent on cross-linking to Fc-gamma receptors on immune cells. The aim is to achieve effective tumor-targeted immune stimulation with minimum side effects.

On May 3, 2018 Coherus BioSciences, Inc. (NASDAQ:CHRS), reported the re-submission of its biologics license application (BLA) for CHS-1701, a pegfilgrastim (Neulasta) biosimilar candidate, to the U.S. FDA under the 351(k) pathway (Press release, Coherus Biosciences, MAY 3, 2018, View Source [SID1234531702]).

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The BLA is supported by similarity data from analytical, pharmacokinetic, pharmacodynamics, and immunogenicity studies comparing CHS-1701 and Neulasta and integrates new immunogenicity data obtained from using a more revised immunogenicity assay.

"The CHS-1701 BLA re-submission marks a significant milestone in our ongoing transition to a commercial company as we tightly focus on execution of our strategic plan," said Denny Lanfear, President and CEO of Coherus BioSciences. "Pegfilgrastim is the largest selling oncology product in the U.S., and CHS-1701 is the cornerstone of our oncology franchise. We believe we have a strong competitive position with this product, exemplified by our comprehensive clinical immunogenicity data as well as our excellent analytical biosimilarity data."