Amgen Inc. (NASDAQ:AMGN) said erenumab (AMG 334) met the primary endpoint in the Phase III ARISE trial to prevent episodic migraine. From baseline to 12 weeks, a 70 mg dose of the compound significantly reduced monthly migraine days by 2.9 vs. 1.8 for placebo.
Erenumab is a human mAb inhibiting the receptor for calcitonin gene-related peptide (CGRP). The ARISE data are the first Phase III results for a mAb targeting the peptide or its receptor. This year, Amgen expects to complete another Phase III study, STRIVE, evaluating 70 mg and 140 mg erenumab in episodic migraine patients.
The 1.1 day difference in ARISE is identical to the difference reported for the 70 mg dose group in a Phase II study of erenumab. Three other mAbs that target the CGRP ligand have reported reducing monthly migraine days by about 1-3 days vs. placebo in episodic migraine patients. Earlier this month, Amgen also reported top-line data from a Phase IIb study in chronic migraine showing that 70 mg and 140 mg erenumab each reduced monthly migraine frequency by 2.4 days (p<0.001) (see BioCentury, Sept. 19, 2016).
ARISE tested once-monthly subcutaneous dosing of placebo or erenumab in 577 patients with a baseline of 4-14 migraine days per month. Amgen said erenumab's safety profile was "similar to placebo and consistent with previously reported studies."
VP of Neuroscience Global Development Robert Lenz told BioCentury earlier this month that Amgen believes ARISE and STRIVE plus the Phase IIb data could be sufficient to support approval in both episodic and chronic migraine. Spokesperson Kristen Davis declined to give a regulatory timeline.
Novartis AG (NYSE:NVS; SIX:NOVN) has global co-development rights and commercialization rights to erenumab outside the U.S., Canada and Japan (see BioCentury, Sept 14, 2015).
Amgen lost $1.92 to $169.71 on Wednesday. It announced the news after market close, and gained $0.61 to $170.32 in early after-hours trading.
Cigna Corp. (NYSE:CI) will cover Exondys 51 eteplirsen from Sarepta Therapeutics Inc. (NASDAQ:SRPT) to treat Duchenne muscular dystrophy (DMD) "in patients who have a confirmed mutation of a specific section of the DMD gene," Cigna spokesperson Karen Eldred told BioCentury.
FDA granted accelerated approval to Exondys 51 this month to treat DMD that is amendable to exon 51 skipping. The drug is a phosphorodiamidate morpholino oligomer (PMO) that induces skipping of exon 51 in dystrophin mRNA (see BioCentury, Sept. 26).
Exondys 51's net annual cost is $300,000 for a 25 kg boy, taking into account changes in weight-based dosing over time and expected compliance rates. The approved dose is 30 mg/kg once weekly.
Eldred said Cigna's coverage is not restricted to DMD patients that have characteristics similar to patients evaluated in clinical studies of the drug.
In an email to BioCentury earlier this month, Sarepta said it expected wide reimbursement of Exondys 51 based on discussions it had been having with payers, which did not include risk-sharing or pay-for-performance deals.
Sarepta rose $3.14 to $62.24 on Wednesday.
Boehringer Ingelheim GmbH (Ingelheim, Germany) partnered with oncolytic virus play ViraTherapeutics GmbH (Innsbruck, Austria) to jointly develop ViraTherapeutics' oncolytic virus therapy platform and its lead candidate, VSV-GP.
Boehringer also gained an option to acquire ViraTherapeutics after Phase I testing of VSV-GP is complete. The companies said the deal has a potential value of EUR 210 million ($235.8 million), but did not provide details and did not respond to inquiries.
ViraTherapeutics is aiming to develop a chimeric oncolytic virus that is non-immunogenic by pairing a potent oncolytic virus -- vesicular stomatitis virus (VSV) -- with a different viral envelope (see BioCentury, Sept. 14, 2015).
ViraTherapeutics will be responsible for preclinical and Phase I testing of VSV-GP, which carries the surface protein from the lymphocytic choriomeningitis virus (LCMV).
Boehringer said the deal will complement its growing immuno-oncology pipeline, which also includes next-generation checkpoint inhibitors and mRNA-based cancer vaccine BI 1361849, which the company licensed from CureVac AG (Tuebingen, Germany).
Last year, ViraTherapeutics raised EUR 3.6 million ($4 million) in the first close of a series A round co-led by Boehringer Ingelheim Venture Fund and EMBL Ventures.
Santhera Pharmaceuticals Holding AG (SIX:SANN) said it enrolled the first patient in its Phase III SIDEROS study of Raxone idebenone to treat Duchenne muscular dystrophy. The company hopes to include SIDEROS data, which are due in 2019, in a planned NDA.
In July, Santhera said FDA would not support the company's plan to submit an NDA for the candidate without data from SIDEROS (see BioCentury Extra, July 14).
The placebo-controlled trial is to evaluate about 260 DMD patients receiving concomitant glucocorticoids. SIDEROS's primary endpoint is change from baseline at week 78 in forced vital capacity percentage predicted.
The candidate is under review in the EU to treat DMD patients in whom respiratory function has started to decline and who are not taking concomitant glucocorticoids.
Last week, FDA approved Exondys 51 eteplirsen from Sarepta Therapeutics Inc. (NASDAQ:SRPT) to treat DMD amenable to exon 51 skipping. The approval was FDA's first for a DMD therapy (see BioCentury Extra, Sept. 19).
Santhera shed CHF0.55 to CHF53.10 on Wednesday.
FDA approved the MiniMed 670G hybrid closed loop system from Medtronic plc (NYSE:MDT) to adjust insulin levels in Type I diabetics aged 14 and older with minimal input from the patient.
The system includes a sensor that measures glucose levels under the skin every five minutes, an insulin pump, and an infusion patch. It uses Medtronic's SmartGuard HCL algorithm to provide appropriate basal insulin doses based on a patient's glucose levels. Patients manually enter mealtime carbohydrates and request insulin doses to counter meal consumption.
Medtronic said it will begin selling the MiniMed 670G system in the U.S. next spring.
Intra-Cellular Therapies Inc. (NASDAQ:ITCI) fell $28.60 (68%) to $13.75 in early after-hours trading after it said neither of two doses of ITI-007 "separated from placebo" on the primary endpoint of a Phase III trial to treat schizophrenia. The company said an usually high placebo response contributed to the miss.
From baseline to six weeks, 60 mg daily ITI-007 lowered Positive and Negative Syndrome Scale (PANSS) total scores by 15.0 points, while a 20 mg dose lowered scores by 14.6 points. Scores fell by 15.1 points in the placebo group, and by 20.5 points in an active control cohort receiving 4 mg risperidone daily. The separation was significant for risperidone vs. placebo.
The company said ITI-007 was significantly better than risperidone "on key safety and tolerability parameters," including weight gain, cholesterol and triglycerides, and had a safety profile similar to placebo. The study included 696 schizophrenia patients with acute psychotic symptoms.
On a conference call Wednesday, SVP of Clinical Development Kimberly Vanover said that in a post hoc analysis that eliminated study sites with "outlier" placebo responses, ITI-007's effect was similar to that seen in earlier studies.
In another Phase III trial last year, 60 mg ITI-007 met the PANSS total score endpoint after four weeks, while a 40 mg dose missed the endpoint (see BioCentury Extra, Sept. 16, 2015).
Intra-Cellular said it plans to meet with FDA to discuss the study results and is "committed and adequately resourced" to continue ITI-007's development.
The company released the results after market close. Intra-Cellular gained $0.83 to $42.35 on Wednesday.
Exelixis Inc. (NASDAQ:EXEL) fell $2.13 (14%) to $13.35 on Wednesday after new data showed a combination of the company's Cabometyx cabozantinib plus PD-1 inhibitor Opdivo nivolumab led to an overall response rate (ORR) in genitourinary cancer that was lower than ORRs produced by other combinations of immuno-oncology agents and targeted therapies. The data were in abstracts released ahead of next month's European Society for Medical Oncology (ESMO) meeting in Copenhagen.
In a Phase I study, Cabometyx plus Opdivo led to an ORR of 33% in 18 evaluable patients with metastatic genitourinary tumors. There was one complete response in a patient with bladder squamous cell carcinoma, and five partial responses in patients with bladder, kidney, urethral or prostate cancers.
Other ESMO abstracts describing Phase I studies of combinations showed ORRs of 54-83% in genitourinary and solid tumors. In a note issued Wednesday, Stifel analyst Stephen Willey cautioned that while other combinations "appear to be yielding numerically higher ORRs" that baseline differences in the patient populations and tumor types "preclude any cross-trial comparisons of efficacy."
The Exelixis data came from the first portion of a Phase I study that evaluated four dose combinations of once-daily oral Cabometyx plus bi-weekly Opdivo. The study's second part is evaluating a combination of Cabometyx, Opdivo and CTLA-4 inhibitor Yervoy ipilimumab.
Cabometyx is a spectrum-selective kinase inhibitor of VEGF receptor 2 (VEGFR-2; KDR/Flk-1) and c-Met receptor tyrosine kinase (c-MET; MET; HGRF; c-Met proto-oncogene). It is approved in the U.S. and EU for second-line treatment of renal cell carcinoma (RCC) and for thyroid cancer.
Bristol-Myers Squibb Co. (NYSE:BMY) markets Opdivo and Yervoy.
Alcobra Ltd. (NASDAQ:ADHD) said FDA placed a clinical hold on the company's MDX metadoxine ER to treat ADHD and fragile X syndrome, including the Phase III MEASURE study in ADHD. According to Alcobra, FDA said the hold resulted from preclinical studies that showed adverse neurological findings.
The extended-release product is a selective antagonist of the serotonin (5-HT2B) receptor. Alcobra announced the hold after market close. The stock gained a penny to $4.62 on Wednesday.
Array BioPharma Inc. (NASDAQ:ARRY) raised $115 million through the sale of 18.4 million shares at $6.25 in a follow-on underwritten by JPMorgan, Cowen, Stifel, Wells Fargo Securities and SunTrust.
Array proposed to raise $100 million in the offering after market close on Tuesday, when it closed at $6.59. The biotech dipped $0.32 to $6.28 on Wednesday.
On Monday, Array shares rose 81% to $6.61 after it and partner Laboratoires Pierre Fabre S.A. (Castres, France) said encorafenib (LGX818) plus binimetinib (MEK162) met the primary endpoint in the first part of the Phase III COLUMBUS study to treat BRAF-mutant melanoma (see BioCentury Extra, Sept. 26).
MyoKardia Inc. (NASDAQ:MYOK) raised $57 million on Wednesday through the sale of 3.8 million shares at $15 in a follow-on underwritten by Credit Suisse, Cowen, BMO Capital Markets and Wedbush PacGrow.
The price represents a 6% discount to MyoKardia's closing price Monday of $15.88, when the company announced the offering after market hours. It is a 28% discount to MyoKardia's closing price of $20.88 on Sept. 16, when it filed for the offering.
The company expects top-line results in 2H17 from a Phase II study of lead candidate MYK-461 to treat obstructive hypertrophic cardiomyopathy. The compound is a small molecular allosteric modulator of cardiac myosin.
Foamix Pharmaceuticals Ltd. (NASDAQ:FOMX) raised $54.2 million through the sale of 5.7 million shares at $9.50 in a follow-on underwritten by Barclays, Credit Suisse, Cowen and Guggenheim. Foamix shareholders raised $2.9 million through the sale of 300,000 shares in a concurrent offering.
The company proposed the offering after market close on Tuesday, when it closed at $9.79. Foamix lost $0.29 to $9.50 on Wednesday.
Next year, Foamix expects to complete two Phase III studies of FMX101, a topical 4% minocycline foam, to treat moderate to severe acne. The candidate is a semi-synthetic derivative of tetracycline.
The U.S. Senate voted 72-26 on Wednesday to pass a temporary budget bill that includes $1.1 billion in funding to support a response to the Zika virus, including developing vaccines and diagnostics.
The bill, H.R. 5325, would fund the federal government at current levels through Dec. 9 while Congress works to hammer out a FY17 budget.
The bill now goes to the House. If a continuing resolution or full budget is not passed by Sept. 30, then the federal government will be shut down.