On June 11, 2018 AOP Orphan´s reported that submission for marketing authorization of Ropeginterferon alfa-2b for treatment of Polycythemia Vera (PV) in the European Union (EU) has resumed after clock-stop (Press release, AOP Orphan Pharmaceuticals, JUN 11, 2018, View Source [SID1234527280]). The conclusion of this centralized procedure is expected for Q4/2018.

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Interferons (IFNs) have been successfully applied in various hematological malignancies including PV, Chronic Myeloid Leukemia (CML) and other myeloproliferative neoplasms (MPNs) for about three decades, however no IFN has obtained regulatory approval in these indications yet.

Ropeginterferon alfa-2b is the first monopegylated IFN with an improved application of only once every two weeks (and only once per month in long-term maintenance treatment). The innovative pegylation technology and the molecule have been invented by PharmaEssentia Corporation (Taiwan Stock Exchange: 6446), a long-term partner of AOP Orphan.

In 2009, AOP Orphan has in-licensed from PharmaEssentia Corporation the exclusive rights to develop and commercialize Ropeginterferon alfa-2b in PV, CML and other MPNs for European, Commonwealth of Independent States (CIS), and Middle Eastern markets.

Since 2009, AOP Orphan´s Development Program in PV including the phase I/II trial PEGINVERA and the phase III trials PROUD-PV, PEN-PV and CONTINUATION-PV, have established high efficacy and favorable safety and tolerability of Ropeginterferon alfa-2b in PV. PharmaEssentia Corporation has invested in preparations for commercialization of the drug in its territory and in the capability for commercial manufacturing of the drug. The company´s world-class cGMP biologics facility in Taichung is certified by the Taiwan Food and Drug Administration (TFDA) and since 2018 also by EMA , and it is designed and operated to be compliant with all U.S. FDA requirements.

Commercial availability of Ropeginterferon alfa-2b is expected to fulfill an increasing need of patients and physicians for the long-term management of a yet incurable class of diseases.

On June 11, 2018 BridgeBio Pharma reported that it has entered into an agreement with Alexion Pharmaceuticals, Inc. to acquire cyclic pyranopterin monophosphate (cPMP; ALXN1101), a synthetic enzyme co-factor therapy for patients with the ultra-rare disease caused by molybdenum cofactor deficiency (MoCD) Type A (Press release, BridgeBio, JUN 11, 2018, View Source [SID1234576280]). In addition, BridgeBio announced that it was launching a new subsidiary, Origin Biosciences, with sufficient capital to support clinical development of ALXN1101 through potential regulatory approval and commercialization.

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MoCD is an ultra-rare autosomal recessive inborn error of metabolism. The disease is caused by a mutation in the MOCS1 gene and leads to defective production of cPMP. Clinical signs of MoCD present shortly after birth and progress rapidly. Newborns with MoCD experience difficulty feeding and intractable seizures yet have no approved available therapies. Patients have a median survival of three years, and those who survive often have severe and irreversible injury to their central nervous system.

"Historically, replacing missing or defective proteins has proven highly efficacious for treating loss of function monogenic conditions – in the case of MoCD type A, we are replacing the missing or defective cPMP, providing children with much needed MoCD activity," said Michael Henderson, M.D., senior vice president of asset acquisition at BridgeBio. "BridgeBio’s team is committed to continuing the development of ALXN1101 for infants born with MoCD Type A deficiency, their families and caregivers."

ALXN1101 is a synthetic version of cPMP, the missing cofactor causing MoCD Type A. In previous work with a recombinant form of cPMP, 11 patients with MoCD Type A had normalization of biomarkers within two days, eight patients showed some suppression of seizures, and three patients had near-normal development. ALXN1101 has received Breakthrough Therapy designation from the US FDA.

"Patients born with MoCD face a bleak future, and we will do all we can to pursue the development of this exciting compound, which has the potential to replace the missing enzyme," said Neil Kumar, Ph.D., CEO of BridgeBio. "BridgeBio aims to sustainably pursue even the rarest of diseases, such as MoCD, especially where we can support drug programs that target well described genetic diseases at their source."

While specific terms of the deal have not been disclosed, BridgeBio has committed sufficient resources to Origin Biosciences to enable clinical development, regulatory approval and to support commercialization of ALXN1101. Alexion will receive additional payments upon the realization of development and sales milestones.

On JUN 11, 2018 Targovax ASA ("Targovax" or "the Company"; OSE: TRVX), a clinical stage company focused on developing and commercializing immune activating oncology therapies to target, primarily, treatment resistant solid tumors, reported an update to its clinical development strategy (Press release, Targovax, JUN 11, 2018, View Source [SID1234527412]).

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Targovax has previously reported encouraging proof-of-concept data from clinical trials with both of its immune activator platforms technologies; ONCOS, which uses genetically armed oncolytic adenoviruses, and TG, a neo-antigen vaccine that targets mutant RAS cancers. However, based on recent external clinical data and market dynamics, Targovax has decided to prioritize and strengthen the development focus on the ONCOS program.

Over the past 12 months, there has been clinical data released from multiple external studies corroborating the potential of oncolytic viruses as an important class of immune activating agents that can boost the effect of other treatments, such as checkpoint inhibitors. This notion is further strengthened by increased partnering and M&A activity by major global pharmaceutical companies, underscored by the acquisitions earlier this year of Viralytics and Benevir by Merck and Johnson & Johnson, respectively.

Furthermore, data presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting on June 1-5 has fundamentally changed the development preconditions for the TG program. Data from independent trials testing the chemotherapy cocktail Folfirinox in resected pancreas cancer, the lead indication for TG01, has demonstrated an improvement in median overall survival of up to 2 years compared to the current standard of care (gemcitabine and capecitabine). These results are great news for patients suffering from this difficult-to-treat cancer. It is expected that the Folfirinox treatment regimen will be quickly adopted as a new standard of care in resected pancreatic cancer, and it is already clear that that the design of Targovax’s planned randomized phase II trial of TG01 in combination with gemcitabine and capecitabine is inadequate and that the trial will not start. Although we are confident that TG01 will be active in combination with any standard of care therapy, the new Folfirinox median survival benchmark of close to 5 years means that such a combination trial is not practically feasible for Targovax.

Targovax strongly believes in the potential of the TG platform to treat mutant RAS cancers, and is encouraged by the signal of efficacy seen in the recent phase I/II trial in resected pancreas cancer. In addition, the company already has a phase I trial underway in colorectal cancer with TG02, the second-generation product from the TG program, combining with pembrolizumab. This trial is expected to read out in 2019. In light of the new Folfirinox data, the Company will together with its clinical advisors reevaluate and reshape the development plans for TG, and devise a strategy for how to best create value for both patients and shareholders. A revised development strategy for the TG program will be presented during the autumn.

The resources freed up by this decision will be allocated to strengthen and speed up ONCOS development. In particular, Targovax is currently looking into options to expand the ongoing trial in mesothelioma, the target launch indication for ONCOS-102.

Øystein Soug, CEO of Targovax said "It is fortunate for us that the emerging Folfirinox data in resected pancreatic cancer was presented at ASCO (Free ASCO Whitepaper) already this year, as it gave us the opportunity to reassess our trial design before committing to an inadequate combination treatment. We are confident that our TG vaccine has potential to benefit patients with mutant RAS cancers, and will now reassess the TG development plan. ONCOS continues to be our lead program, and we will further sharpen our focus to drive ONCOS-102 forward with full force, and remain in the forefront of oncolytic virus development"

On June 11, 2018 Bristol-Myers Squibb Company (NYSE: BMY) reported that it will take part in Goldman Sachs 39th Annual Global Healthcare Conference on Wednesday, June 13, 2018, in Rancho Palos Verdes, CA (Press release, Bristol-Myers Squibb, JUN 11, 2018, View Source [SID1234527263]). Johanna Mercier, Head of U.S. Commercial, and Fouad Namouni, Head of Oncology Development, will answer questions about the company at 6:20 p.m. ET (3:20 p.m. PT).

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Investors and the general public are invited to listen to a live webcast of the session at View Source An archived edition of the session will be available later that day.

On June 11, 2018 Nkarta Therapeutics, a privately-held cell therapy company developing Natural Killer (NK) immune cells to fight cancer, reported that it has entered into a worldwide exclusive license agreement for proprietary Natural Killer cell engineering technology jointly owned by the National University of Singapore (NUS) and St. Jude Children’s Research Hospital (Press release, Nkarta, JUN 11, 2018, View Source [SID1234530934]). The license, negotiated by the Industry Liaison Office (ILO) of NUS and the St. Jude Office of Technology Licensing (OTL), includes several issued patents and patent applications related to methods to generate large numbers of fully functional NK cells as well as compositions of chimeric receptors for targeting NK cells to tumors and extending their life-span. The licensed technologies are all based on discoveries by Professor Dario Campana, M.D., Ph.D., of NUS and formerly of St. Jude, who is credited with major advances in chimeric antigen receptor (CAR-T) cell therapy in addition to Natural Killer cell therapy.

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"Through this agreement, we have solidified our position as leaders in Natural Killer cell technology," said Paul Hastings, president and chief executive officer of Nkarta. "Natural Killer cells have a unique, innate ability to target and destroy cancer cells, but the amount generated by the body is not sufficient to overcome the disease. With this license we have gained access to exclusive expansion and targeting technologies that will generate an abundant supply of our proprietary engineered and enhanced NK cells that can selectively kill tumor cells."

Dr. Campana added, "As a scientific founder of Nkarta, I am pleased to see the company advance its technology, which enhances the potential of NK cells as a next-generation cell therapy. The company has expertise in key aspects of NK cell engineering, including ways to improve recognition of tumor targets, to support persistence and sustained activity, and to produce genetically-modified NK cells in sufficient quantity to meet clinical requirements."