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Re: changing course of treating DMD?? SRPT??I wish it weren't so - but Sarepta has the appearance of trying to act like something it is not. Hire, hire, hire - create all these separate programs, hire, hire, hire, create lots and lots of departments and job titles, hire, hire, hire, create lots of marketing programs for different audiences, hire, hire, hire, create branding around "urgency to treat rare disease", HIRE, HIRE, HIRE. These are the references to Sarepta's utrophin programs from the 10-K Sarepta filed in March, 2019. "GALGT2. An additional gene therapy program for DMD and other muscular dystrophies, also in collaboration with Nationwide, aims to express the enzyme GALGT2 from an AAV vector. We believe that GALGT2 modifies the dystrophin associated protein complex (DAPC) and up-regulates utrophin (a protein significantly homologous to dystrophin) to protect muscle from damage in the absence of dystrophin. We believe that the micro-dystrophin and GALGT2 technologies have the potential to treat all or nearly all DMD patients regardless of mutation. In the fourth quarter of 2017, an IND application for GALGT2 was cleared by the FDA, and a Phase 1/2a clinical trial testing GALGT2 for the treatment of DMD was initiated." "Summit On October 3, 2016, we entered into an exclusive Collaboration and License Agreement (the “Collaboration Agreement”) with (Oxford) Ltd. (“Summit”), which grants us the exclusive right to commercialize products in Summit’s utrophin modulator pipeline in the EU, Switzerland, Norway, Iceland, Turkey and the Commonwealth of Independent States. On June 27, 2018, Summit announced that it decided to discontinue the development of ezutromid after reviewing the top-line results from its Phase 2 trial." "Under the terms of the Summit Collaboration Agreement, the Company made an up-front payment of $40.0 million to Summit, with additional payments of up to $192.0 million based on achievement of certain development and regulatory milestones for ezutromid, a Summit product candidate in its utrophin modulator pipeline. For each of Summit’s future generation small molecule utrophin modulators, the Company may be required to make up to $290.0 million in development and regulatory milestone payments. Additionally, on a product-by-product basis, the Company may be required to make up to $330.0 million in sales milestone payments. The Summit Collaboration Agreement also grants the Company an option to expand the Licensed Territory (“Option Territory”). If the Company exercises this option, it will be liable for a one-time $10.0 million option fee as well as up to $7.0 million in regulatory milestone payments. For each licensed product in the Option Territory, the Company may be liable for up to $82.5 million in sales milestone payments. Additionally, the Company may be required to make tiered royalty payments ranging from a low to high teens percentage of net sales on a product-by-product basis in the Licensed Territory. Under the Summit Collaboration Agreement, a joint steering committee was established to plan, monitor and coordinate future development activities for ezutromid and future generation small molecule utrophin modulators. Summit was solely responsible for all research and development costs for the licensed products until December 31, 2017. Thereafter, Summit will be responsible for 55.0% of the budgeted research and development costs related to the licensed products in the Licensed Territory, and the Company will be responsible for 45.0% of such costs. Any costs in excess of 110.0% of the budgeted amount are borne by the party that incurred such costs. Summit is also obligated to spend a specified minimum amount on the research and development of certain licensed products prior to the end of 2019. For the year ended December 31, 2018, the Company incurred approximately $8.6 million in collaboration expense for ezutromid, with no similar activity in 2017 and 2016. In June 2018, Summit announced that it is discontinuing the Phase 2 clinical trial on ezutromid. As a result, no development, regulatory or sales milestones were deemed as probable of being achieved as of December 31, 2018. For the years ended December 31, 2017, and 2016, the Company recorded a $22.0 million milestone payment and a $40.0 million up-front payment to Summit, respectively, as research and development expense in its consolidated statement of operations and comprehensive loss." |
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Msg # | Subject | Author | Recs | Date Posted |
13963 | Re: changing course of treating DMD?? SRPT?? | redplate | 0 | 10/15/2019 6:42:38 AM |