Biogen price target raised to $400 from $300 at Jefferies. Jefferies analyst Michael Yee raised his price target for Biogen to $400 following the positive Phase II Alzheimer’s data for BAN-2401. After Friday’s rally, the question is whether there is significant incoming moneyflow above and beyond peers as well and what’s risk/reward into the presentation of BAN-2401 data now, Yee tells investors in a research note. The analyst keeps a Hold rating on Biogen shares after analyzing the bull/bear setup for 2018.
Monday, July 9, 2018
Sunday, July 8, 2018
IT Solutions for Easier EHRs Save Physicians Time, Burnout
Yale Medicine is effectively targeting electronic health record (EHR) use and functionality as a way to improve physician job satisfaction and reduce burnout, according to an article published in the American Medical Association’s AMA Wire.
EHR inefficiencies were making physicians unhappy and adding extra work hours, including bringing home work at night and on weekends. A series of discussions with leadership led to implementation of information technology (IT) solutions that would address physician burnout and improve the user experience with EHRs.
Three IT solutions are enabling Yale Medicine physicians to save hours of time. First, easier login identification, using badges to tap in and out of the EHR system throughout a shift, eliminates repetitive typing of the physician’s username and password. Physicians now just do a one-time login at the start of their shift, which saves physicians between six and 20 minutes daily by eliminating 20 to 140 logins per physician per day. Additionally, Yale Medicine implemented speech recognition for physicians. Voice-recognition software that connects directly to EHRs has reduced the time it takes physicians to complete and close encounters by 50 percent. The average time to close an encounter is down by eight hours a week. The third major IT initiative is an ongoing pilot using virtual scribes.
“One of the things we realized is that there is still a limitation to the keyboard-and-mouse user interface,” Allen Hsiao, M.D., the chief medical information officer at Yale, said in the article. “Anything we can do to tackle that can make a big difference and help take clicks away for physicians to complete their work.”
850M worldwide have kidney disease
Kidney disease is a “hidden epidemic” affecting more than 850 million people worldwide, renal experts say.
That’s twice the number of diabetics (422 million) and more than 20 times the number of people with cancer (42 million) or HIV/AIDS (36.7 million).
But most people don’t realize that kidney disease is a major health issue.
“It is high time to put the global spread of kidney diseases into focus,” said David Harris and Adeera Levin of the International Society of Nephrology. Harris is the group’s president and Levin is past president.
They noted that kidney diseases often cause no early symptoms. And many people aren’t aware of their increased risk for heart problems, infections, hospitalization and kidney failure.
Chronic kidney diseases (ones lasting more than three months) affect 10 percent of men and nearly 12 percent of women around the world. Up to 10.5 million people need dialysis or a kidney transplant, but many don’t receive these lifesaving treatments due to cost or lack of resources.
In addition, more than 13 million people suffer acute kidney injury. Some will go on to develop chronic kidney disease or kidney failure.
“Using all these sources of data, and existing estimates of acute and chronic kidney diseases, we estimate approximately 850 million kidney patients — a number which surely signifies an ‘epidemic’ worldwide,” Levin said.
Kidneys remove waste products and help balance the volume of fluids and minerals in the body. They also produce a hormone that tells the body to make red blood cells, the researchers explained.
Even if many patients with damaged kidney function don’t feel ill, they’re at high risk for other health problems, said Carmine Zoccali, president of the European Renal Association — European Dialysis and Transplant Association.
Heart disease deaths due to chronic kidney disease are high — 1.2 million cardiovascular deaths were attributed to kidney disease in 2013.
“The number of people with kidney diseases is alarmingly high, but the public is not aware of this reality. These patients have outcomes and kidney diseases impose a heavy financial burden on health care budgets,” said Mark Okusa, president of the American Society of Nephrology.
The annual per-patient cost of dialysis is $88,195 in the United States, he said in a ASN news release.
More information
The National Kidney Foundation has more on kidney disease.
Prana preclinical data set for Parkinson’s conference
Prana Biotechnology Ltd (ASX PBT: NASDAQ PRAN) has today announced it will be presenting further pre-clinical evidence for PBT434 at the International Congress of Parkinson’s Disease and Movement Disorders® to be held in Hong Kong from October 5-9, 2018.
The data to be presented will include new in-vivo evidence of the efficacy of PBT434 to prevent neuron loss and improve function in an animal model of Multiple system atrophy (MSA), an important cause of atypical Parkinsonism. MSA is a rapidly progressive and devastating neurological disease with no established treatments and is one of the target indications for PBT434.
PBT434 is the first of a new generation of small molecules designed to inhibit the aggregation of alpha(α)-synuclein and tau, vital intracellular proteins that are implicated in neurodegenerative diseases such as Parkinson’s disease and atypical Parkinsonism. PBT434 has been shown to reduce the abnormal accumulation of these proteins in animal models of disease by restoring normal iron balance in the brain. Prior non-clinical characterization of PBT434, including animal models of Parkinson’s disease, was published last year in Acta Neuropathologica Communications and may be found at https://doi.org/10.1186/s40478-017-0456-2.
The experimental data to be presented demonstrate that in an animal model of MSA, PBT434 prevents α-synuclein accumulation, preserves neurons, and decreases the number of glial cell inclusions in the brains of treated animals. Glial cell inclusions are the key pathological finding in MSA and contain abundant aggregated α-synuclein that is associated with neurodegeneration. Importantly, these benefits led to improved motor function in treated animals. Alpha-synuclein is of great interest because aggregated forms of the protein are considered a pathological hallmark of Parkinsonian conditions and are a recognised therapeutic target by basic and clinical neuroscientists.
‘Multiple system atrophy, or MSA, is a devastating orphan disease with limited treatment options. These animal data are robust and indicate that PBT434 has excellent potential to help individuals with MSA. Having recently started our first human study of PBT434, these data represent an important step as we pursue new treatments for Parkinsonian diseases’, said David Stamler, Chief Medical Officer and Senior Vice President of Clinical Development.
The initial human study of PBT434 commenced in June 2018.
The International Congress of Parkinson’s Disease and Movement Disorders® is the preeminent scientific meeting for sharing ideas and stimulating interest in the care and research of movement disorders, and is organized annually by the International Parkinson and Movement Disorder Society.
Treatment Centers Authorized to Administer CAR T-Cell Therapy
The following medical facilities are certified to administer chimeric antigen receptor (CAR) T-cell therapy to eligible patients (as of May 16, 2018). However, not all of the centers administer both tisagenlecleucel (Kymriah) and axicabtagene ciloleucel (Yescarta). To identify the centers administering each product, a “T” or “A” has been placed after the center’s name. And, because new treatment centers may have been certified since publication, this may not be a complete listing of all treatment centers now authorized to perform CAR T-cell procedures.
Updated information for treatment centers certified to administer tisagenlecleucel is located at http://www.us.-kymriah.com/acute-lymphoblastic-leukemia-children/about-treatment/where-to-get-treatment. For updated information on treatment centers certified to administer axicabtagene ciloleucel, visit www.yescarta.com/-infusion.html.
MIDWEST
Illinois
Ann & Robert H. Lurie Children’s Hospital of Chicago, Chicago; www.luriechildrens.org; T
Northwestern Medicine Robert H. Lurie Comprehensive Cancer Center of Northwestern, Chicago; http://cancer.northwestern.edu; A
University of Chicago Medicine, Chicago; www.uchospitals.edu/index.shtml; T/A
Kansas
University of Kansas Cancer Center, Westwood; www.kucancercenter.org; T
Michigan
Michigan Medicine, University of Michigan Medical Center, Ann Arbor; www.uofmhealth.org; T
Barbara Ann Karmanos Cancer Institute, Detroit; www.karmanos.org/home; A
University of Michigan Comprehensive Cancer Center, Ann Arbor; www.karmanos.org; A
Minnesota
University of Minnesota Masonic Children’s Hospital, Minneapolis; www.mhealth.org/locations/buildings/university-of-minnesota-masonic-childrens-hospital; T
Missouri
Children’s Mercy Hospital, Kansas City; www.childrensmercy.org; T
The University of Kansas Cancer Center, Kansas City; www.kucancercenter.org; A
Siteman Cancer Center at Barnes-Jewish Hospital at Washington University Medical Center, St. Louis; www.barnesjewish.org/Medical-Services/Cancer-Center; A
Washington University School of Medicine Siteman Kids at St. Louis Children’s Hospital, St. Louis; siteman.wustl.edu/visiting/kids/; T
Nebraska
Nebraska Medicine, Omaha; www.nebraskamed.com; A
University of Nebraska Medical Center, Omaha; www.unmc.edu; A
Ohio
Cincinnati Children’s Hospital Medical Center, Cincinnati; www.cincinnatichildrens.org; T
Cleveland Clinic Cancer Center, Cleveland; my.clevelandclinic.org/departments/cancer; A
The Ohio State University Comprehensive Cancer Center; Columbus; cancer.osu.edu; T/A
Wisconsin
Froedtert & the Medical College of Wisconsin Cancer Network, Milwaukee; www.froedtert.com/cancer/network; A
UWHealth/American Family Children’s Hospital, Madison; www.uwhealthkids.org; T
NORTHEAST
Maryland
Johns Hopkins Children’s Center, The Charlotte R. Bloomberg Children’s Center, Baltimore; www.hopkinsmedicine.org/johns-hopkins-childrens-center/patients-and-families/bloomberg-childrens-center/index.html; T
University of Maryland Marlene and Stewart Greenbaum Comprehensive Cancer Center, Baltimore; www.umms.org/umgccc; A
Massachusetts
Dana-Farber Boston Children’s Cancer & Blood Disorders Center, Boston; www.danafarberbostonchildrens.org; T
Dana-Farber/Brigham and Women’s Cancer Center, Boston; www.brighamandwomens.org/cancer-center; A
Massachusetts General Hospital Cancer Center, Boston; www.massgeneral.org/cancer/; A
New Jersey
Hackensack University Medical Center–John Theurer Cancer Center, Hackensack; www.hackensackumc.org/services/cancer-care/; A
Joseph M. Sanzari Children’s Hospital at Hackensack Meridian Health, Hackensack; www.hackensackumc.org/locations/joseph-m-sanzari-childrens-hospital/; T
New York
Memorial Sloan Kettering Cancer Center, New York; www.mskcc.org; T/A
Roswell Park Comprehensive Cancer Center, Buffalo; www.roswellpark.org; A
UR Medicine Wilmot Cancer Institute, Rochester; www.urmc.rochester.edu/cancer-institute.aspx; A
Pennsylvania
The Children’s Hospital of Philadelphia, Philadelphia; www.chop.edu; T
Penn Medicine Abramson Cancer Center, Philadelphia; www.pennmedicine.org/cancer; T
UPMC Hillman Cancer Center, Pittsburgh; hillman.upmc.com/find/locations/hillman-cancer-center-pittsburgh-pa; A
SOUTHEAST
Florida
Moffitt Cancer Center, Tampa; moffitt.org; T/A
Sylvester Comprehensive Cancer Center, Miami; sylvester.org; A
Georgia
Aflac Cancer and Blood Disorders Center, Children’s Healthcare of Atlanta, Atlanta; www.choa.org/medical-services/cancer-and-blood-disorders; T
Winship Cancer Institute of Emory University, Atlanta; winshipcancer.emory.edu; A
Tennessee
Vanderbilt University Medical Center, Nashville; ww2.mc.vanderbilt.edu; T/A
SOUTHWEST
Arizona
Banner Health, Gilbert; www.bannerhealth.com; A
Phoenix Children’s Hospital, Phoenix; www.phoenixchildrens.org; T
Texas
Baylor Charles A. Sammons Cancer Center at Dallas–Texas Oncology, Dallas, Texas; www.texasoncology.com/-cancer-centers/dallas/baylor-charles-a-sammons/medical-oncology; A
Children’s Medical Center Dallas, Pauline Allen Gill Center for Cancer and Blood Disorders, Dallas; www.childrens.com; T
Houston Methodist Hospital, Houston; www.houstonmethodist.org; T
Texas Children’s Hospital, Houston; www.texaschildrens.org; T
Texas Transplant Institute–Methodist Healthcare, San Antonio; sahealth.com/service/transplant-services; A
The University of Texas MD Anderson Cancer Center, Houston; www.mdanderson.org; T/A
WEST
California
Children’s Hospital Los Angeles, Los Angeles; www.chla.org; T
City of Hope, Duarte; www.cityofhope.org/homepage; T/A
Lucile Packard Children’s Hospital Stanford, Palo Alto; www.stanfordchildrens.org; T
Stanford Health Care, Palo Alto; stanfordhealthcare.org; A
UCLA Health, Los Angeles; www.uclahealth.org; A
UCSF Benioff Children’s Hospital, San Francisco; www.ucsfbenioffchildrens.org; T
UCSF Helen Diller Family Comprehensive Cancer Center, San Francisco; cancer.ucsf.edu; A
Colorado
Children’s Hospital Colorado, Aurora; www.childrenscolorado.org; T
Colorado Blood Cancer Institute, Denver; bloodcancerinstitute.com; A
Oregon
Oregon Health & Science University, Portland; bloodcancerinstitute.com; T
OHSU Knight Cancer Institute, Portland; www.ohsu.edu/health/cancer/index.html; A
Utah
Huntsman Cancer Hospital Institute at the University of Utah, Salt Lake City, Utah; healthcare.utah.edu/huntsmancancerinstitute/; T/A
Primary Children’s Hospital, Salt Lake City; intermountainhealthcare.org/locations/primary-childrens-hospital/; T
Washington
Seattle Cancer Care Alliance, Seattle; www.seattlecca.org; A
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Weighing the Cost and Value of CAR T-Cell Therapy
How It Works. The above image illustrates the process of making CAR T cells for each individual patient from collecting the patient’s T cells from their blood, shipping the cells to the laboratory for modification and manufacturing, to infusing the engineered CAR-containing T cells into the…
Benitec Biopharma in Global Licensing Agreement with Axovant
Benitec Announces Global Licensing Agreement for BB-301 for Treatment of Oculopharyngeal Muscular Dystrophy and Broad Platform Collaboration with Axovant
- • Benitec to receive upfront cash payment of US$10 million with additional cash payments totalling US$17.5 million (a total of US$27.5M) upon completion of near-term milestones for BB-301, now named AXO-AAV-OPMD
- • Benitec is potentially eligible for US$187.5 million in total payments upon the achievement of development, regulatory and commercial milestones on AXO-AAV-OPMD
- • Benitec will retain 30% of the net profits on the worldwide sales of AXO-AAV-OPMD
- • Benitec and Axovant to partner on the development of five additional gene therapy programs; Benitec to receive full research funding and be eligible for US$93.5 million in development, regulatory and commercial milestones for each program
Sydney, Australia, 9 July 2018: Benitec Biopharma Limited (“Benitec” or the “Company”) (ASX:BLT; NASDAQ: BNTC; NASDAQ: BNTCW) today announced that it has licensed to Axovant Sciences (“Axovant”) the exclusive global rights for BB-301 (now named AXO-AAV-OPMD) intended for the treatment of oculopharyngeal muscular dystrophy (OPMD), and has also entered into a fully funded research collaboration for the development of five additional gene therapy products in neurological disorders.
Under the terms of the agreement, Benitec will receive an upfront cash payment of US$10 million and additional cash payments totaling US$17.5 million upon completion of four specific near-term manufacturing, regulatory and clinical milestones. Axovant has been granted worldwide rights to AXO-AAV-OPMD and will assume all future development costs. The total potential value of all of the development, regulatory and commercial milestones achievable by Benitec, of which there are eight milestones including the four near-term milestones, is US$187.5 million. Benitec, working in partnership with Axovant over the next few years, hopes to achieve all eight milestones and thus realize the maximum amount of US$187.5 million. There can be no assurance as to the total amount of payments that the Company will actually receive or when they will be received. Importantly, upon commercialization, Benitec will retain 30% of the net profits on worldwide sales of AXO-AAV-OPMD.
Jerel Banks, MD PhD, Executive Chairman, Benitec Biopharma commented on today’s news, “Today marks a milestone for Benitec as we believe this transaction to be transformative for our company. In addition to bolstering our opportunity to drive broad-based, clinically meaningful patient benefit across several areas of clinical medicine with true unmet need, this partnership significantly enhances the financial, intellectual, and clinical development resources available to facilitate our efforts to build Benitec into a diversified biopharmaceutical company. The non-dilutive capital expected over the near term will allow Benitec to continue to invest in proprietary R&D programs across a range of indications.”
Dr. Banks continued, “Our management team is focused exclusively on expanding the research, development, and commercial opportunities for the core ‘silence-and-replace’platform with the dual goals of enhancing patient benefit and generating shareholder value. We believe Axovant is the idealpartner to advance our OPMD program, and we look forward to working closely with them to develop AXO-AAV-OPMD as we quickly progress towards clinical trials in 2019.”
OPMD is a rare progressive, and often fatal, muscle-wasting disease caused by mutation in the poly(A)-binding protein nuclear 1 (PABPN1) gene, that is characterized by eyelid drooping, swallowing difficulties, and proximal limb weakness. AXO-AAV-OPMD is a single vector, gene therapy construct system that uses a unique “Silence-and-Replace” methodology that employs DNA directed RNA interference (ddRNAi) to silence expression of the mutant gene associated with OPMD, while simultaneously expressing a copy of the normal, healthy version of the same gene to restore the function of that gene. Axovant plans to initiate a placebo-controlled clinical study in 2019 in which a one-time intramuscular administration AXO-AAV-OPMD will be given to patients to treat the dysphagia associated with OPMD.
Commenting on the agreement, Pavan Cheruvu, MD, Chief Executive Officer of Axovant said, “The ‘silence-and-replace’ platform is a targeted approach which directly addresses the underlying genetic cause of diseases arising from expression of dysfunctional proteins, including those caused by nucleotide repeat expansion. I am excited about the potential of this platform for patients suffering from OPMD, many of whom have limited treatment options today.”
In addition to AXO-AAV-OPMD, Axovant and Benitec will collaborate on a total of five additional investigational gene therapy products for neurological disorders, with Axovant fully funding each of the research programs. Axovant will have exclusive global rights to products developed under these programs. The first additional investigational gene therapy product will focus on developing a single vector “Silence-and-Replace” gene therapy product targeting the c9orf72 gene, which is associated with amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). In addition to receiving funding for development of the new research programs, each new research program target is eligible for development, regulatory and commercial milestones totaling US$93.5 million and tiered royalties on global sales. There can be no assurance as to the total amount of payments that the Company will actually receive or when they will be received.
Dr. Banks concluded, “We are extremely excited about Axovant’s collaborative andfinancial commitments to these five additional research programs as it plants the seeds for a long and robust partnership between our organizations. This partnership provides Benitec with an extraordinarily rare opportunity to unambiguously demonstrate the exceptional breadth of the scientific, clinical, and commercial applications of the ‘silence-and-replace’ platform. Additionally, the non-dilutive capital expected by Benitec over the near term will be used to fund operations as we will continue to innovate and strengthen our platform. I look forward to making future announcements on our joint progress with Axovant as well as on other material developments.”
Biotech week ahead, May 9
Biotech stocks held their ground in a holiday-shortened week amid a drought of market-moving catalysts.
Looking ahead, the following are catalytic events for biotech investors to watch.
Conferences
- Fourth International Conference on Neurological Disorders & Stroke: July 9-10 in Sydney, Australia.
- 15th International Conference on Digestive Disorders and Gastroenterology: July 11-12 in Sydney, Australia.
- 13th International Conference on Tissue Engineering & Regenerative Medicine: July 12-13 in Paris, France.
- Hematologists Global Summit 2018: July 13-14 in Sydney, Australia.
- Third International Conference on Ophthalmology: July 10-11 in Bangkok, Thailand.
- 12th International Conference on Pediatric, Perinatal and Diagnostic Pathology: July 13-14 in Toronto, Canada.
- International Conference on Pediatric Pharmacology and Therapeutics: July 13-14 in Toronto, Canada.
- Annual Congress on Mental Health: July 9-11 in Paris, France.
PDUFA Dates
Bristol-Myers Squibb Co BMY 1.05%‘s sBLA for its Opdivo-Yervoy combination for treating adults with microsatellite instability-high or mismatch repair-deficient metastatic colorectal cancer that has progressed following treatment with a fluoropyrimidine, oxaliplatin and irinotecan will come up before the FDA, with the agency set to rule July 10 on the application.
Adcom Meeting Schedule
FDA’s Antimicrobial Drugs Advisory Committee is set to discuss GlaxoSmithKline plc (ADR) GSK 0.72%‘s NDA for the tafenoquine tablet in a 150-milligram dosage for prevention of relapse of Plasmodium vivax malaria July 12.
Clinical Trials
Zynerba Pharmaceuticals Inc ZYNE 6% is set to present Phase 2 data for ZYN002 in Fragile X syndrome. The data released last September showed that the study met the primary endpoint.
Early Q3 Release
Altimmune Inc ALT 3.74% is set to release Phase 1 data for its anthrax therapy Nasoshield.
Mid-2018 Releases
GlaxoSmithKline is likely to release Phase 2b data for its anti-SAP mAb, chemically dezamizumab, which is being tested for amyloidosis.
Bellerophon Therapeutics Inc BLPH will release interim analysis of Phase 3 data for its INOpulse delivery device meant to treat pulmonary arterial hypertension.
BIOLINERX Ltd/S ADR BLRX 0.98% is set to release Phase 3 results from the GENESIS clinical trial, which evaluates its BL-8040 for the mobilization of hematopoietic stem cells used for autologous transplantation in multiple myeloma patients.
Celgene Corporation CELG 2.55% and Acceleron Pharma Inc XLRN 1.02% are likely to release Phase 3 data for the b-thalassemia treatment Luspatercept, based on the BELIEVE trial.
IPO Quiet Period Expirations
Verrica Pharmaceuticals Inc VRCA 0.25%, which debuted on Nasdaq June 15 following a 5-million-share IPO at $15 per share, will see its IPO quiet period expire.
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