Selecta Biosciences has filed a 10-K – Annual report [Section 13 and 15(d), not S-K Item 405] with the U.S. Securities and Exchange Commission (Filing, 10-K, Selecta Biosciences, 2018, MAR 15, 2018, View Source [SID1234524797]).

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On March 15, 2017 Infinity Pharmaceuticals, Inc. (NASDAQ: INFI) reported its fourth quarter 2017 financial results and provided an update on the company, including its progress with IPI-549, a first-in-class oral immuno-oncology product candidate that selectively inhibits phosphoinositide-3-kinase-gamma (PI3K-gamma) and targets immune-suppressive tumor microphages (Press release, Infinity Pharmaceuticals, MAR 15, 2018, View Source [SID1234524810]). Infinity is evaluating IPI-549 as a monotherapy and in combination with Opdivo (nivolumab), a PD-1 immune checkpoint inhibitor, in a Phase 1/1b study in approximately 200 patients with advanced solid tumors.

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"We made significant progress with IPI-549 over the past year, and 2018 will be an important year for us as we report data from the monotherapy expansion and combination dose escalation components of our study and from seven combination expansion cohorts, which will help define our development and regulatory strategy for this first-in-class product candidate. Based on our initial clinical data, IPI-549 has a favorable tolerability profile and has demonstrated clinical and biological activity, both as a monotherapy and in combination with nivolumab. We believe that IPI-549 has the potential to increase the number of patients who respond to immunotherapies as well as to increase the duration of those responses," said Adelene Perkins, Chief Executive Officer and Chair of Infinity Pharmaceuticals. "In addition, we’ve continued to position Infinity for success by expanding our board and clinical leadership team and establishing a scientific advisory board with thought leaders in the field of immuno-oncology."

Recent developments include the following:

IPI-549

Advanced and expanded clinical development of IPI-549: The Phase 1/1b monotherapy and combination dose escalation components of the study have been completed, and the monotherapy expansion component has been fully enrolled. In addition, six disease-specific combination expansion cohorts are enrolling at the recommended Phase 2 dose of 40 mg once daily of IPI-549 plus Opdivo (nivolumab) at 240 mg every two weeks in patients with non-small cell lung cancer, melanoma, head and neck cancer, triple-negative breast cancer, mesothelioma, and adrenocortical carcinoma. An additional combination expansion cohort of patients pre-selected for having high baseline blood levels of myeloid derived suppressor cells (MDSCs) is expected to open for enrollment in the next few weeks. Studies have shown that poor response to checkpoint inhibitor therapy is correlated with the presence of high baseline blood levels of MDSCs in cancer patients.1,2,3 Preliminary translational data from the study demonstrated an association between high baseline blood levels of MDSCs and clinical responses. Preselecting patients with high blood MDSCs could lead to improved clinical activity for patients treated with the combination of IPI-549 and anti-PD1.
Presented Phase 1/1b clinical and translational data at SITC (Free SITC Whitepaper): In November, Infinity announced updated data from the monotherapy dose-escalation component of the Phase 1/1b study of IPI-549 in a late-breaking presentation at the SITC (Free SITC Whitepaper) Annual Meeting 2017. These data demonstrated that IPI-549 dosed once daily was well tolerated and clinically active. Among 18 patients evaluable for activity, there was a 44 percent clinical benefit rate, defined as patients who had remained on treatment for at least 16 weeks, including one partial response in a patient with advanced peritoneal mesothelioma. Initial translational data from patient blood samples demonstrated that IPI-549 treatment results in a reduction in immune suppression and increased immune stimulation, with upregulation of interferon-gamma responsive factors and reinvigoration/proliferation of exhausted T cells across multiple tumor types and dose levels. Additionally, those patients who showed a clinical benefit had increased numbers of activated monocytes, suggesting a biologic correlate can be identified in patients who remain on treatment longer.
Corporate

David Beier appointed to Board of Directors: David Beier, J.D., is a Managing Director of Bay City Capital and brings a wealth of experience to the Infinity Board. David serves as an advisor to the Parker Institute for Cancer Immunotherapy, as a Senior Fellow at the USC Schaeffer Center for Health Policy & Economics and as a member of the Board of Directors of Arcus Biosciences, UCSF Benioff Children’s Hospitals and the California Life Sciences Association. He also serves as an appointee of Governor Brown on the California State Government Organization and the Economy Commission. Having spent two decades as part of the senior management teams for Amgen and Genentech, he brings invaluable perspective regarding strategy for entrepreneurial biotechnology firms and the industry in general as a globally recognized leader in health care policy, regulatory affairs, healthcare economics, and pricing. Mr. Beier also previously served in the White House during the Clinton Administration as a Chief Domestic Policy Advisor to the Vice President.
Clinical leadership team expanded: Marie-Louise Fjällskog M.D., Ph.D., has been appointed as Vice President of Clinical Development and will play an integral role in the expanded clinical development of IPI-549. Dr. Fjällskog is an Associate Professor of Oncology at Uppsala University, Sweden, and has over twenty-five years of experience in clinical oncology, translational research, and drug development. She joins Infinity from the Novartis Institute for Biomedical Research, where she served as a Clinical Program Leader, Translational Clinical Oncology, and as the global lead for several immuno-oncology programs, including those targeting CSF-1 and PD-1.

In addition, Suresh Mahabhashyam, M.D., M.P.H., was promoted to Vice President of Safety and Risk Management. Dr. Mahabhashyam has over 20 years of experience in medical practice and epidemiology with the last decade focused on drug development at Infinity, Alexion and Allergan.
Scientific Advisory Board established with four thought leaders in immuno-oncology:
Dmitry Gabrilovich, M.D., PhD., a leader in myeloid cell biology and the Christopher M. Davis Professor in Cancer Research and Program Leader, Immunology, Microenvironment, and Metastasis at the Wistar Institute in Philadelphia and Wistar Professor at the Department of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania;
Roy Herbst, M.D., Ph.D., a leader in lung cancer treatment and research and the Ensign Professor of Medicine (Medical Oncology) and Professor of Pharmacology; Chief of Medical Oncology, Yale Cancer Center and Smilow Cancer Hospital;
Stephen Hodi, M.D., a leader in developing immune therapy and melanoma therapeutics and the Director of the Melanoma Center and the Center for Immuno-Oncology at Dana-Farber/Brigham and Women’s Cancer Center, the Sharon Crowley Martin Chair in Melanoma at Dana-Farber Cancer Institute and Professor of Medicine at Harvard Medical School; and
David H. Munn, M.D., a pioneer in T cell activation and indoleamine 2,3-dioxygenase (IDO) research and a Professor of Pediatric Hematology-Oncology at the Medical College of Georgia, Augusta University.
2018 Program Goals for IPI-549

Report data from the monotherapy expansion component of the study in the second quarter of 2018
Report data from the combination dose-escalation component of the study in the second quarter of 2018
Report initial data from six disease-specific combination expansion cohorts in the second quarter of 2018
Report more mature clinical and translational data (including insights from paired tumor biopsies) from the six disease-specific cohorts and initial data from the cohort of patients pre-selected for having high baseline blood levels of myeloid derived suppressor cells (MDSCs) in the combination expansion component of the study in the second half of 2018
Full Year 2017 Financial Results

At December 31, 2017, Infinity had total cash, cash equivalents and available-for-sale securities of $57.6 million, compared to $92.1 million at December 31, 2016.
Revenue during 2017 was $6.0 million, all of which related to the amount received from Verastem for the DUO study meeting the pre-specified criteria at completion. Revenue during 2016 was $18.7 million related to Infinity’s previous collaboration agreement with AbbVie Inc.
R&D expense for 2017 was $20.8 million, compared to $119.6 million for 2016. The decrease in R&D expense was primarily related to the company’s 2016 restructuring activities and out-licensing of duvelisib to Verastem.
General and administrative expense was $21.6 million for 2017, compared to $42.2 million for 2016. The decrease in G&A expense was primarily due to the company’s 2016 restructuring activities.
Net loss for 2017 was $41.8 million, or a basic and diluted loss per common share of $0.83, compared to a net loss of $30.1 million, or a basic and diluted loss per common share of $0.61 for 2016.
Financial Outlook
Infinity’s updated 2018 financial guidance is as follows:

Net Loss: Infinity expects net loss for 2018 to range from $35 million to $45 million.
Cash and Investments: Infinity expects to end 2018 with a year-end cash, cash equivalents and available-for-sale securities balance ranging from $15 million to $25 million.
Cash Runway: Based on its current operational plans, Infinity expects that its existing cash, cash equivalents and available-for-sale securities will be adequate to satisfy the company’s capital needs into the third quarter of 2019. Infinity’s financial guidance excludes additional funding or business development activities and does not include the potential $22 million payment from Verastem upon the first regulatory approval of duvelisib, or a potential $2 million milestone payment from PellePharm, a private company, upon initiation of a Phase 3 study for the hedgehog inhibitor program, which Infinity licensed to PellePharm in 2013. Verastem announced that it submitted a New Drug Application for duvelisib to the U.S. Food and Drug Administration on February 7, 2018. With the potential Verastem milestone payment, Infinity expects to extend its cash runway into 2020.
Conference Call Information
Infinity will host a conference call today, March 15, 2018, at 4:30pm EDT to discuss these financial results and company updates. A live webcast of the conference call can be accessed in the "Investors/Media" section of Infinity’s website at www.infi.com. To participate in the conference call, please dial 1-877-316-5293 (domestic) and 1-631-291-4526 (international) five minutes prior to start time. The conference ID number is 5558799. An archived version of the webcast will be available on Infinity’s website for 30 days.

About IPI-549 and the Ongoing Phase 1b Study
IPI-549 is an investigational first-in-class, oral, immuno-oncology product candidate targeting tumor-associated myeloid cells through selective phosphoinositide-3-kinase-gamma (PI3K-gamma) inhibition, thereby reducing pro-tumor macrophage function and increasing anti-tumor macrophage function. In preclinical studies, IPI-549 reprograms macrophages from a pro-tumor (M2), immune suppressive function, to an anti-tumor (M1) immune activating function and can enhance the activity of, and overcome resistance to, checkpoint inhibitors. 4,5 As such, IPI-549 may have the potential to treat a broad range of solid tumors and represents a potentially additive or synergistic approach to restoring anti-tumor immunity in combination with other immunotherapies such as checkpoint inhibitors.

The ongoing Phase 1b study being conducted by Infinity is designed to evaluate the safety, tolerability, activity, pharmacokinetics and pharmacodynamics of IPI-549 as a monotherapy and in combination with Opdivo in approximately 200 patients with advanced solid tumors.6 The study includes monotherapy and combination dose-escalation components, in addition to monotherapy expansion and combination expansion components. The monotherapy dose-escalation component is complete, and the monotherapy expansion component has been fully enrolled. The combination dose-escalation component is also complete, and combination expansion cohorts are enrolling.

The combination expansion component of the study includes multiple cohorts designed to evaluate IPI-549 in patients with specific types of cancer, including patients with non-small cell lung cancer (NSCLC), melanoma and head and neck squamous cell carcinoma (HNSCC) whose tumors show initial resistance or initially respond to but subsequently develop resistance to immune checkpoint blockade therapy. The combination expansion component also includes a cohort of patients with triple negative breast cancer (TNBC) who have not been previously treated with immune checkpoint blockade therapy, a cohort of patients with mesothelioma, a cohort of patients with adrenocortical carcinoma and a cohort of patients with high baseline blood levels of MDSCs.

IPI-549 is an investigational compound and its safety and efficacy has not been evaluated by the U.S. Food and Drug Administration or any other health authority.

On March 15, 2018 Novelion Therapeutics Inc. (NASDAQ: NVLN), a biopharmaceutical company dedicated to developing and commercializing therapies for individuals living with rare diseases ("Novelion" or the "Company"), reported financial results for the fourth quarter and full year ended December 31, 2017 and provided an overview of recent business activities (Press release, QLT, MAR 15, 2018, View Source [SID1234524839]).

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Chief Operating Officer Jeff Hackman said, "We are focused on executing near-term plans that we believe will position our company for sustainable future growth. These priorities include cost control and expense management, reviewing our holding and capital structure with a view toward optimizing our assets for shareholders, advancing the metreleptin development program, and continuing to provide important therapies to our patients."

Business Update

•
JUXTAPID: Novelion reported net revenues of JUXTAPID of $20.1 million in the fourth quarter of 2017, $14.2 million, or 71%, of which were from prescriptions written in the U.S.

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MYALEPT: Novelion reported net revenues of MYALEPT of $18.8 million in the fourth quarter of 2017, $13.3 million, or 71%, of which were from prescriptions written in the U.S.

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Novelion reported total consolidated net revenues of $138.4 million for the year ended December 31, 2017.

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Novelion ended 2017 with $55.4 million in unrestricted cash, compared with $70.5 million at the end of the third quarter of 2017.

•
As announced separately today, subsidiary Aegerion Pharmaceuticals entered into a new secured financing facility with affiliates of Sarissa Capital Management and Broadfin Capital LLC providing for a $20 million term loan to Aegerion, strengthening Aegerion’s balance sheet and liquidity, and positioning the Company for ongoing capital structure review.

•
In January 2018, Novelion undertook significant cost reduction plans as it continues to manage its limited cash resources.

•
With respect to its European application to register metreleptin for marketing authorization, after taking into account the results of an oral hearing of the European Medicines Agency’s Committee for Medicinal Products for Human Use ("CHMP"), which occurred in February

2018, the Company expects the opinion of the CHMP in the second quarter of 2018 and the European Commission’s approval decision in mid-2018.

Fourth Quarter 2017 Financial Results

On November 29, 2016, the Company completed its acquisition of Aegerion Pharmaceuticals, Inc. ("Aegerion"). The acquisition has been accounted for as a business combination in which Novelion was considered the acquirer of Aegerion. As such, under U.S. Generally Accepted Accounting Principles ("GAAP"), the financial statements of Novelion are treated as the historical financial statements of the consolidated companies, with the results of Aegerion being included from November 29, 2016. This release also includes pro forma adjusted non-GAAP financial information showing pro forma results of operations of Novelion as if the acquisition had occurred on January 1, 2016. Reconciliation of the financial results on a GAAP versus non-GAAP basis are provided below the financial information that follows.

GAAP total net revenues for the fourth quarter of 2017 were $38.9 million compared to the prior year’s fourth quarter net revenues of $13.6 million. GAAP net revenues for JUXTAPID in the fourth quarter of 2017 were $20.1 million compared to $8.6 million in the prior year. GAAP net revenues for MYALEPT in the fourth quarter of 2017 were $18.8 million compared to $5.0 million for the same period in 2016.

GAAP total operating expenses for the fourth quarter of 2017 were $35.9 million compared to total operating expenses of $22.0 million for the same period in 2016. GAAP SG&A expenses were $24.1 million in the fourth quarter of 2017 compared to $16.0 million for the same period in 2016. GAAP R&D expenses were $11.8 million in the fourth quarter of 2017 compared to $6.0 million for the same period in 2016.

On a pro forma basis, during the fourth quarter of 2017, SG&A expenses were $22.5 million compared to $55.9 million for the same period in 2016. The decrease in pro forma SG&A expenses in the fourth quarter of 2017 compared with the same period in 2016 was primarily related to a reduction in headcount and legal and consulting fees.

On a pro forma basis, during the fourth quarter of 2017, R&D expenses were $11.6 million compared to $14.2 million for the same period in 2016. The decrease in pro forma R&D expenses in the fourth quarter of 2017 compared with the same period in 2016 was primarily related to a reduction in headcount and the timing of vendor related activities.

GAAP net loss in the fourth quarter of 2017 was $24.6 million compared to GAAP net loss of $19.9 million during the same period in 2016.

On a pro forma basis, net loss in the fourth quarter of 2017 was $3.3 million, compared to $20.1 million for the same period in 2016.

Full Year 2017 Financial Results

GAAP total net revenues for the year ended December 31, 2017 were $138.4 million compared to $13.6 million in 2016. GAAP net revenues for JUXTAPID for the year ended December 31, 2017 were $72.1 million compared to $8.6 million in 2016. GAAP net revenues for MYALEPT for the year ended December 31, 2017 were $66.3 million compared to $5.0 million in 2016.

GAAP total operating expenses for the year ended December 31, 2017 were $148.0 million compared to total operating expenses of $44.3 million in 2016. GAAP SG&A expenses were $96.5 million for the year ended December 31, 2017 compared to $29.5 million in 2016. GAAP R&D expenses were $49.0 million for the year ended December 31, 2017 compared to $14.8 million in 2016.

Cost of product sales were $77.2 million in the year ended December 31, 2017. Cost of product sales in the current year includes $18.8 million reserves recorded for excess and obsolete inventory, which are derived from projected sales activities, respective product shelf-life and their respective fair value. Additionally, cost of product sales was also comprised of the cost of inventory sold, amortization of acquired product rights, which resulted from the acquisition of Aegerion, and estimated royalties payable related to the sales of lomitapide and metreleptin. We expect cost of product sales for metreleptin to increase in 2018 and for the next several years, due primarily to an increasing time-based royalty rate on net sales of metreleptin in the U.S.

On a pro forma basis, for the year ended December 31, 2017, SG&A expenses were $90.7 million compared to $200.1 million in 2016. For the year ended December 31, 2017, R&D expenses on a pro forma basis were $48.2 million compared to $52.1 million in 2016.

GAAP net loss for the year ended December 31, 2017 was $126.7 million compared to GAAP net loss of $52.9 million in 2016.

On a pro forma basis, net loss for the year ended December 31, 2017 was $30.0 million, compared to $136.0 million in 2016.

As of December 31, 2017, the Company’s consolidated unrestricted cash balance was $55.4 million, compared to $70.5 million at September 30, 2017 and $108.9 million at December 31, 2016. As of December 31, 2017, there were 18.7 million shares outstanding. At December 31, 2017, total debt principal was $325 million, reflecting the principal amount of convertible debt, before discount, issued by Aegerion and consolidated as a result of the acquisition.

On March 16, 2018 Shares of Adaptimmune Ltd. surged late Thursday after the company reported it saw three partial responses in three of the four myxoid/ round cell liposarcoma (MRCLS) patients the company dosed with its NY-ESO SPEAR (Specific Peptide Enhanced Affinity Receptor) T-cells (Press release, BioSpace, MAR 15, 2018, View Source [SID1234524855]).

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The news sent company stock up more than 20 percent to $12.41 per share. Since that spike, shares have fallen back some to $10.74 as of 10:51 a.m.

Those positive results are also good news for GlaxoSmithKline, which licensed the NY-ESO SPEAR T-cell therapy program last fall. The two companies initiated their collaboration in 2014 and last year the pharma giant exercised its option to exclusively license Adaptimmune’s NY-ESO SPEAR T-cell therapy program. The transition has not yet been completed.

MRCLS is a type of liposarcoma that is characterized by the proliferation of adipocyte (fat cell) precursors called lipoblasts that have undergone differentiation arrest. MRCLS represents about 30 to 35 percent of liposarcomas and 5 to 10 percent of all adult soft tissue sarcomas. It is estimated that there are approximately 2000 patients in the United States and Europe with MRCLS each year.

Rafael Amado, Adaptimmune’s chief medical officer, said the company is encouraged by the initial responses in the first patients dosed. He said the news validates the potential for its platform to treat a broad range of tumors, including those known to be unresponsive to current immunotherapy treatments.

"Although MRCLS is a soft tissue sarcoma which commonly expresses NY-ESO, there are fundamental differences in its clinical course, natural history, molecular signature, and responsiveness to standard treatments that make it distinct from synovial sarcoma. As we expect data from our other trials with our wholly owned assets throughout 2018, these results in a second solid tumor strengthen our conviction that our pipeline of unique TCRs will be capable of addressing multiple solid tumors," Amado said in a statement.

So far Adaptimmune has dosed four patients with its treatment. Of the three partial responses the company said two have been confirmed and one has yet to be confirmed. The other patient was classified as having stable disease. Adaptimmune said the doses were well-tolerated. However, the company said it did see cases of cytokine release syndrome (CRS), a systemic inflammatory response that has been a persistent concern in other CAR-T and cell therapy trials. The CRS cases Adaptimmune patients encountered were managed following standard treatment guidelines, the company said.

Two years ago the U.S. Food and Drug Administration placed a partial clinical hold on the NY-ESO SPEAR T-cell study for MRCLS. The hold was lifted after Adaptimmune revised the trial protocol.

In addition to its NY-ESO SPEAR T-cell study, Adaptimmune is using SPEAR T-cells in two clinical trials targeting MAGE-A10, one in non-small cell lung cancer (NSCLC), and a triple tumor study in bladder, melanoma, and head & neck cancers. Both studies are dose escalation trials that evaluate three doses of transduced SPEAR T-cells, administered after a lymphodepleting chemotherapy regimen. In addition to the MAGE-A10 trial, Adaptimmune is also targeting MAGE-A4. The newly manufactured SPEAR T-cell at the Philadelphia site will be used in a multiple tumor study in bladder, melanoma, head & neck, ovarian, non-small cell lung, esophageal, and gastric cancers.

On March 14, 2018 Kiadis Pharma N.V. ("Kiadis Pharma" or the "Company") (Euronext Amsterdam and Brussels: KDS), a clinical stage biopharmaceutical company developing a T-cell immunotherapy product designed to reduce Graft versus Host Disease (GVHD) in hematopoietic stem cell transplantations (HSCT), reported that Arthur Lahr, Chief Executive Officer, will present at the Cowen 38th Annual Health Care Conference on Wednesday, March 14, 2018 at 10:00am EDT in Boston, MA (Press release, Kiadis, MAR 15, 2018, View Source [SID1234525132]).

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For more information, please contact:

Kiadis Pharma:
Karl Hård, Head of IR & Communications
Tel. +31 611 096 298
[email protected]

Optimum Strategic Communications:
Mary Clark, Supriya Mathur, Hollie Vile
Tel: +44 (0) 203 714 1787
[email protected]