Stem Cell Clinic

Dr. Peggy Goodell elected to National Academy of Medicine – Baylor College of Medicine News

Dr. Margaret Peggy Goodell, chair of the Department of Molecular and Cellular Biology at Baylor College of Medicine, has been elected to the National Academy of Medicine. She is among 100 new members, including 10 international members, announced today at the Academys annual meeting in Washington, D.C.

Dr. Goodells long career in regenerative medicine proves she is well-deserving of this distinction, said Dr. Paul Klotman, president, CEO and executive dean of Baylor. She is a renowned scientist who has made important contributions to the field of stem cell biology, particularly genetic and epigenetic regulation of self-renewal and differentiation of hematopoietic stem cells.

Goodell is known for discovering a novel method to isolate adult stem cells. At Baylor, she serves as a professor in several departments, including pediatrics hematology-oncology and molecular and human genetics and in the Center for Cell and Gene Therapy, as well as programs in integrative molecular and biomedical sciences, development biology and translational biology and molecular medicine. She is the director of the Stem Cells and Regenerative Medicine Center at Baylor and holds the Vivian L. Smith Chair in Regenerative Medicine. She is co-leader of the Cancer Cell and Gene Therapy Program in the Dan L Duncan Comprehensive Cancer Center at Baylor.

I am extremely honored by election to the National Academy of Medicine. This distinction represents recognition of my work, as well as the contributions of the many members of my laboratory who have worked with me over the past 20 years, Goodell said. I am looking forward to the opportunity to contribute to the NAM on relevant national science and medicine issues.

Election to the National Academy of Medicine is considered one of the highest honors in the fields of health and medicine and recognizes individuals who have demonstrated outstanding professional achievement and commitment to service. New members are elected by current members through a process that recognizes individuals who have made major contributions to the advancement of the medical sciences, health care and public health.

Goodell joins the following group of 13 distinguished scientists from Baylor College of Medicine in the National Academy of Medicine:

Dr. Arthur L. BeaudetDr. Dennis M. BierDr. Malcolm K. BrennerDr. William R. BrinkleyDr. C. Thomas CaskeyDr. Mary K. EstesDr. Richard A. GibbsDr. Peter J. HotezDr. Brendan LeeDr. James R. LupskiDr. Bert W. OMalleyDr. Cheryl Lyn WalkerDr. Huda Y. Zoghbi

Originally posted here:
Dr. Peggy Goodell elected to National Academy of Medicine - Baylor College of Medicine News

New Organelle That Helps Prevent Cancer Discovered Inside Our Cells – University of Virginia

Scientists at theUniversity of Virginia School of Medicinehave discovered a strange neworganelle inside our cells that helps to prevent cancer by ensuring that genetic material is sorted correctly as cells divide.

The researchers have connected problems with the organelle (a subcellular structure) to a subset ofbreast cancer tumorsthat make lots of mistakes when segregating chromosomes. Excitingly, they found their analysis offered a new way for doctors to sort patient tumors as they choose therapies. Theyhope these insights will allow doctors to better personalize treatments to best benefit patients sparing up to 40% of breast cancer patients, for example, a taxing treatment that wont be effective.

Some percentage of women get chemotherapy drugs for breast cancer that are not very effective. They are poisoned, in pain and their hair falls out, so if it isnt curing their disease, then thats tragic, said researcher P. Todd Stukenberg of UVAs Department of Biochemistry and Molecular Genetics and theUVA Cancer Center. One of our goals is to develop new tests to determine whether a patient will respond to a chemotherapeutic treatment, so they can find an effective treatment right away.

The organelle Stukenberg and his team have discovered is essential, but ephemeral. It forms only when needed to ensure chromosomes are sorted correctly and disappears when its work is done. Thats one reason scientists havent discovered it before now.

Another reason is its mind-bending nature: Stukenberg likens it to a droplet of liquid that condenses within other liquid. That was the big wow moment, when I saw that on the microscope, he said.

These droplets act as mixing bowls, concentrating certain cellular ingredients to allow biochemical reactions to occur in a specific location. Whats exciting is that cells have this new organelle and certain things will be recruited into it and other things will be excluded, Stukenberg said. The cells enrich things inside the droplet and, all of a sudden, new biochemical reactions appear only in that location. Its amazing.

Its tempting to think of the droplet like oil in water, but its really the opposite of that. Oil is hydrophobic it repels water. This new organelle, however, is more sophisticated.

Its more of a gel, where cellular components can still go in and out, but it contains binding sites that concentrate a small set of the cells contents, Stukenberg explained. Our data suggests this concentration of proteins is really important. I can get complex biochemical reactions to occur inside a droplet that Ive been failing to reconstitute in a test tube for years. This is the secret sauce Ive been missing.

While its been known for about eight years that cells make such droplets for other processes, it was unknown that they make them on chromosomes during cell division. Stukenberg believes these droplets are very common and more important than previously realized.

I think this is a general paradigm, he said. Cells are using these non-membranous organelles to regulate much of their work.

In addition to helping us understand mitosis how cells divide Stukenbergs new discovery also sheds light on cancer and how it occurs. The organelles main function is to fix mistakes in tiny microtubules that pull apart chromosomes when cells are dividing. That ensures each cell winds up with the correct genetic material. In cancer, though, this repair process is defective, which can drive cancer cells to become more aggressive.

Stukenberg has also developed tests to measure the amount of chromosome mis-segregation in tumors, and he hopes that this might allow doctors to pick the proper treatment to give cancer patients. We have a way to identify the tumors where the cells are mis-segregating chromosomes at a higher rate, he said. My hope is to identify the patients where treatments such as [chemotherapy medication] paclitaxel are going to be the most effective.

Having looked at breast cancer already, he next plans to examine the strange organelles role in colorectal cancer.

Stukenberg and his colleagues described their latest findings in the scientific journal Nature Cell Biology. The research team consisted of Prasad Trivedi, Francesco Palomba, Ewa Niedzialkowska, Michelle A. Digman, Enrico Gratton and Stukenberg.

The research was supported by the National Institutes of Health, grants R01GM124042, R24OD023697 and P41-GM103540; and the National Science Foundation, grant MCB-1615701.

To keep up with the latest medical research news from UVA, subscribe to theMaking of Medicineblog.

Visit link:
New Organelle That Helps Prevent Cancer Discovered Inside Our Cells - University of Virginia

Capricor Therapeutics to Host Key Opinion Leader Call on the Role of CAP-1002 for the Treatment of Duchenne Muscular Dystrophy (DMD) – BioSpace

LOS ANGELES, Oct. 21, 2019 (GLOBE NEWSWIRE) -- Capricor Therapeutics (Nasdaq: CAPR), a clinical-stage biotechnology company focused on the development of first-in-class biological therapeutics for the treatment of Duchenne muscular dystrophy (DMD) and other rare disorders, announced today that it will host a Key Opinion Leader (KOL) call on the role of CAP-1002 in the of treatment of Duchenne Muscular Dystrophy (DMD) on Thursday, October 24th at 10:30am Eastern Time.

The call will feature a presentation by KOL Craig M. McDonald, MD, UC Davis Health, and principal investigator of Capricors HOPE-2 trial who will provide an overview of Duchenne Muscular Dystrophy (DMD), its progression, current treatment options, and new treatments in development for the disease. Dr. McDonald will be available to answer questions at the conclusion of the call.

Capricors management team will also provide an overview of the Companys lead candidate, CAP-1002, a cell therapy that is currently in clinical development for the treatment of Duchenne muscular dystrophy (DMD). CAP-1002 consists of allogeneic cardiosphere-derived cells, or CDCs, a unique population of cells that contains cardiac progenitor cells. CAP-1002 has been shown to exert potent immunomodulatory activity and alter the immune systems activity to encourage cellular regeneration. Data from the pre-specified interim analysis demonstrated that teens and young men in the advanced stages of DMD saw improvements in skeletal, pulmonary, and cardiac measurements after receiving multiple doses of CAP-1002. Specifically, patients showed improvements in the Performance of the Upper Limb (PUL), a tool specifically designed for assessing high (shoulder), mid (elbow) and distal (wrist & hand) function, with a conceptual framework reflecting the progression of weakness in ambulant and non-ambulant patients.

The FDA has granted Capricors CAP-1002 (Regenerative Medicine Advanced Therapy Designation) RMAT and Orphan Drug Designation, for which the FDA has also granted a Rare Pediatric Disease Designation.

Craig M. McDonald, MD is currently a Professor of Medicine and the Chair of the Department of Physical Medicine & Rehabilitation at UC Davis Health. He received his M.D. and M.R.M. from the University of Washington School of Medicine as well as his A.B. in Human Biology from Stanford University. Dr. McDonald is also board-certified in neuromuscular medicine and pediatric rehabilitation medicine. He is an internationally recognized expert in clinical management, rehabilitation, and precision therapeutics for children and adults with neuromuscular diseases. Dr. McDonald has been a pioneer in the development of novel outcome measures for clinical trials focused on disabled populations. He is widely known for his expertise in the treatment and evaluation of children and young adults with Duchenne muscular dystrophy and other neuromuscular diseases. Dr. McDonald serves as director and principal investigator of UC Davis' successfully renewed NINDS-funded site in the NeuroNEXT Neurosciences Clinical Trials National Consortium (one of two NeuroNEXT sites on the West Coast). Dr. McDonald is also the director of rehabilitation services at Shriners Hospital for Children - Northern California.

About HOPE-2HOPE-2 is a randomized, double-blind, placebo-controlled, Phase II clinical trial of the companys lead investigational therapy, CAP-1002 in steroid-treated boys and young men who are in advanced stages of DMD. The study protocol called for treatment via intravenous delivery with either CAP-1002 (150 million cells per infusion) or placebo every 3 months.

About Duchenne Muscular DystrophyDuchenne muscular dystrophy is a devastating genetic disorder that causes muscle degeneration and leads to death, generally before the age of 30, most commonly from heart failure. It occurs in one in every 3,600 live male births across all races, cultures and countries. Duchenne muscular dystrophy afflicts approximately 200,000 boys and young men around the world. Treatment options are limited, and there is no cure.

About CAP-1002CAP-1002 consists of allogeneic cardiosphere-derived cells, or CDCs, a proprietary population of cells that contains cardiac progenitor cells that has been shown in pre-clinical and clinical studies to exert potent immunomodulatory activity, and is being investigated for its potential to modify the immune systems activity to encourage cellular regeneration. CDCs have been the subject of over 100 peer-reviewed scientific publications and have been administered to approximately 150 human subjects across several clinical trials.

About Capricor TherapeuticsCapricor Therapeutics, Inc. (CAPR) is a clinical-stage biotechnology company focused on the discovery, development and commercialization of first-in-class biological therapeutics for the treatment of rare disorders. Capricors lead candidate, CAP-1002, is an allogeneic cell therapy that is currently in clinical development for the treatment of Duchenne muscular dystrophy. Capricor is also exploring the potential of CAP-2003, a cell-free, exosome-based candidate, to treat a variety of disorders. HOPE-Duchenne, Capricors Phase I/II trial was funded in part by the California Institute for Regenerative Medicine. For more information, visit http://www.capricor.com.Keep up with Capricor on social media: http://www.facebook.com/capricortherapeutics, http://www.instagram.com/capricortherapeutics/ and https://twitter.com/capricor

Cautionary Note Regarding Forward-Looking StatementsStatements in this press release regarding the efficacy, safety, and intended utilization of Capricor's product candidates; the initiation, conduct, size, timing and results of discovery efforts and clinical trials; the pace of enrollment of clinical trials; plans regarding regulatory filings, future research and clinical trials; regulatory developments involving products, including the ability to obtain regulatory approvals or otherwise bring products to market; plans regarding current and future collaborative activities and the ownership of commercial rights; scope, duration, validity and enforceability of intellectual property rights; future royalty streams, revenue projections; expectations with respect to the expected use of proceeds from the recently completed offerings and the anticipated effects of the offerings, and any other statements about Capricor's management team's future expectations, beliefs, goals, plans or prospects constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not statements of historical fact (including statements containing the words "believes," "plans," "could," "anticipates," "expects," "estimates," "should," "target," "will," "would" and similar expressions) should also be considered to be forward-looking statements. There are a number of important factors that could cause actual results or events to differ materially from those indicated by such forward-looking statements. More information about these and other risks that may impact Capricor's business is set forth in Capricor's Annual Report on Form 10-K for the year ended December 31, 2018 as filed with the Securities and Exchange Commission on March 29, 2019, and as amended by its Amendment No. 1 to Annual Report on Form 10-K/A filed with the Securities and Exchange Commission on April 1, 2019, in its Quarterly Report on Form 10-Q for the quarterly period ended June 30, 2019, as filed with the Securities and Exchange Commission on August 8, 2019, and in its Registration Statement on Form S-3 as filed with the Securities and Exchange Commission on October 24, 2018, and as amended by its Amendment No. 1 to Form S-3 filed with the Securities and Exchange Commission on July 17, 2019, together with prospectus supplements thereto. All forward-looking statements in this press release are based on information available to Capricor as of the date hereof, and Capricor assumes no obligation to update these forward-looking statements.

CAP-1002 is an Investigational New Drug and is not approved for any indications. CAP-2003 has not yet been approved for clinical investigation.

For more information, please contact:

AJ Bergmann, Chief Financial Officer+1-310-358-3200abergmann@capricor.com

View post:
Capricor Therapeutics to Host Key Opinion Leader Call on the Role of CAP-1002 for the Treatment of Duchenne Muscular Dystrophy (DMD) - BioSpace

Two University of Chicago Medicine physicians elected to National Academy of Medicine for 2019 – Newswise

MEDIA CONTACT

Available for logged-in reporters only

Newswise Two University of Chicago faculty members,Vineet Arora, MD, MAPP, andErnst Lengyel, MD, PhD, have been elected tomembership in the National Academy of Medicine.

Election to the NAM is considered one of the highest honors in the fields of health and medicine, and acknowledges individuals who have demonstrated outstanding scientific and professional achievement in biology, medicine and related fields, saidKenneth S. Polonsky, MD, the Richard T. Crane Distinguished Service Professor, Dean of the Division of the Biological Sciences and the Pritzker School of Medicine, and Executive Vice President for Medical Affairs. With the results of the current election, there are now 19 current or emeritus University of Chicago faculty who are members of the NAM.

Arora is an academic hospitalist and medical educator who specializes in improving the learning environment for medical trainees and the quality, safety and experience of care delivered to hospitalized adults. She is a Professor in the Department of Medicine and serves as Associate Chief Medical Officer for the Clinical Learning Environment at the University of Chicago Medicine and Assistant Dean for Scholarship and Discovery at the Pritzker School of Medicine. She joined the UChicago faculty in 2004.

Aroras work bridges clinical medicine and medical education to transform the learning environment and the quality of care delivered in teaching hospitals. She is an internationally recognized expert on patient handoffs in health care and also has broad expertise in using technology, such as social media, to improve medical education. She has served as principal investigator of numerous federal and foundation grants that explore novel scientific approaches to improving patient care while also enhancing workplace learning in teaching hospitals.

Arora is an elected member to the American Society of Clinical Investigation, which recognizes physician-scientists for an outstanding record of scholarly achievement in biomedical research, and serves on the American Board of Internal Medicine Board of Directors. As an advocate for gender equity across health care, she has leadership roles in several organizations dedicated to advancing women leaders and combating sexual harassment in health care, including Women of Impact, TIME'S UP Healthcare and the National Academies of Science, Engineering, and Medicines Action Collaborative on Sexual Harassment in Higher Education.

Lengyel is a gynecologic oncologist and expert in the diagnosis and treatment of ovarian cancer. He is the Arthur L. and Lee G. Herbst Professor of Obstetrics and Gynecology and is Chair of the Department of Obstetrics and Gynecology at the University of Chicago. He joined the UChicago faculty in 2004.

Lengyel is a clinically active surgeon. His research is focused on understanding the biological basis of metastasis and in characterizing the metabolic changes that occur in ovarian cancer cells. His laboratory employs high throughput screening using sophisticated organotypic 3D models to evaluate the efficacy and mechanisms of action of new compounds in preclinical models to prepare for their translation to the clinic. His research team also applies advanced -omics techniques to characterize differences between primary and metastatic ovarian cancer in human tumor tissue to gain broad insights into ovarian cancer biology and develop a rationale for ovarian cancer treatments.

Ina 2019 study published inNature, Lengyel and his colleagues described a new treatment target that could prevent the rapid spread of high-grade serous carcinoma, the most common and deadly form of ovarian cancer, which originates in the fallopian tubes or ovaries and spreads throughout the abdominal cavity. This project is being further developed toward clinical testing by the NCI Experimental Therapeutics (NExT) program. His research team is also part of the Human Cell Atlas,a project supported by the Chan Zuckerberg Initiativeto map all of the cells in the human body. The team will characterize the different cell types in the female reproductive tract to understand the origins of gynecological diseases like fibroids, endometriosis or cancer.

TheNational Academy of Medicine, established in 1970 as the Institute of Medicine, is an independent organization of eminent professionals from diverse fields including health, biology, medicine and related fields. It serves alongside theNational Academy of Sciencesand theNational Academy of Engineeringas a scientific and policy adviser to the nation and the international community.

Read more here:
Two University of Chicago Medicine physicians elected to National Academy of Medicine for 2019 - Newswise

Vertex Announces Reimbursement of Cystic Fibrosis Medicines SYMDEKO (tezacaftor/ivacaftor and ivacaftor) for Eligible Patients Ages 12 and Older, and…

LONDON--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX) today announced that SYMDEKO (tezacaftor/ivacaftor and ivacaftor) is reimbursed in Australia for people with cystic fibrosis (CF) ages 12 years and older who are homozygous for the F508del mutation or who have one copy of the F508del mutation and another responsive residual function (RF) mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. People with CF who have one copy of the F508del mutation and another responsive RF mutation in the CFTR gene will have access to a medicine for the cause of their CF for the first time. In addition, ORKAMBI (lumacaftor/ivacaftor) is now also reimbursed for the treatment of children with CF ages 2 to 5 who have two copies of the F508del mutation in the CFTR gene. Patients over the age of 6 have already been able to access ORKAMBI in Australia since October 2018.

Following previously received positive recommendations from the Pharmaceutical Benefits Advisory Committee (PBAC), eligible patients in Australia will be able to access both medicines immediately, and the medicines will be listed on the Pharmaceutical Benefits Scheme (PBS) from December 1st.

We are pleased that SYMDEKO and ORKAMBI will be made available immediately to eligible cystic fibrosis patients in Australia. We appreciate that the PBAC has recognized the value of these medicines to patients and thank the Department of Health and the Minister for Health in Australia for their strong engagement and collaboration to finalize the agreement, said Ludovic Fenaux, Senior Vice President, Vertex International.

Vertexs CF medicines are reimbursed in 17 countries around the world including Austria, Denmark, Germany, the Republic of Ireland, Italy, the Netherlands, Sweden and the U.S.

About CFCystic Fibrosis (CF) is a rare, life-shortening genetic disease affecting approximately 75,000 people worldwide. CF is a progressive, multi-system disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes one from each parent to have CF. While there are many different types of CFTR mutations that can cause the disease, the vast majority of all people with CF have at least one F508del mutation. These mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working and/or too few CFTR proteins at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the early 30s.

About SYMDEKO (tezacaftor/ivacaftor) in combination with ivacaftorSome mutations result in CFTR protein that is not processed or folded normally within the cell, and that generally does not reach the cell surface. Tezacaftor is designed to address the trafficking and processing defect of the CFTR protein to enable it to reach the cell surface and ivacaftor is designed to enhance the function of the CFTR protein once it reaches the cell surface.

Mutations in the CFTR gene, responsive to SYMDEKO, that are currently registered in Australia include F508del/f and P67L, R117C, L206W, R352Q, A455E, D579G, 711+3AG, S945L, S977F, R1070W, D1152H, 2789+5GA, 3272-26AG, 3849+10kbCT, E56K, R74W, D110E, D110H, E193K, E831X, F1052V, K1060T, A1067T, F1074L and D1270N.

About ORKAMBI (lumacaftor/ivacaftor) and the F508del mutationIn people with two copies of the F508del mutation, the CFTR protein is not processed and trafficked normally within the cell, resulting in little-to-no CFTR protein at the cell surface. Patients with two copies of the F508del mutation are easily identified by a simple genetic test. Lumacaftor/ivacaftor is a combination of lumacaftor, which is designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the F508del-CFTR protein, and ivacaftor, which is designed to enhance the function of the CFTR protein once it reaches the cell surface.

About VertexVertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has three approved medicines that treat the underlying cause of cystic fibrosis (CF) a rare, life-threatening genetic disease and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational medicines in other serious diseases where it has deep insight into causal human biology, such as sickle cell disease, beta thalassemia, pain, alpha-1 antitrypsin deficiency, Duchenne muscular dystrophy and APOL1-mediated kidney diseases.

Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London, UK. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including nine consecutive years on Science magazine's Top Employers list and top five on the 2019 Best Employers for Diversity list by Forbes.

Special Note Regarding Forward-Looking Statements

This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, the statements in the second and third paragraphs of the press release. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from the company's development programs may not support registration or further development of its compounds due to safety, efficacy or other reasons, and other risks listed under Risk Factors in Vertex's annual report and subsequent quarterly reports filed with the Securities and Exchange Commission and available through the company's website at http://www.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

Original post:
Vertex Announces Reimbursement of Cystic Fibrosis Medicines SYMDEKO (tezacaftor/ivacaftor and ivacaftor) for Eligible Patients Ages 12 and Older, and...

FDA Approves TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to Treat the Underlying Cause of Cystic Fibrosis in People Ages 12 and Older…

Today marks a milestone for CF patients, their families and Vertex. After a 20-year journey together, we have received FDA approval of TRIKAFTA: a single breakthrough medicine with the potential to treat up to 90% of all people with CF in the future. For approximately 6,000 people with CF in the U.S., TRIKAFTA is the first medicine that can treat the underlying cause of their disease, said Jeffrey Leiden, M.D., Ph.D., Vertex's Chairman, President and Chief Executive Officer. I want to personally thank the hundreds of Vertex scientists who have been working on this program for nearly 20 years many of whom have dedicated their entire careers to changing the course of this disease; the CF Foundation which has provided support, encouragement and help throughout the journey; and most importantly the thousands of patients, caregivers, doctors and advocates who have courageously and persistently worked side-by-side with us to get to where we are today.

Todays approval is a historic moment in cystic fibrosis care, with the potential for more people to benefit from CFTR modulator therapy to treat the basic defect of their disease, said Steven Rowe, M.D., Director, Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham. In clinical trials, TRIKAFTA was generally well tolerated and demonstrated improvements in multiple outcome measures in CF, including improvements in FEV1, improvements in respiratory symptoms and, in the 24-week F/MF study, a reduced rate of pulmonary exacerbations and improvements in BMI.

The incredible speed of this approval underscores our shared sense of urgency with the FDA and the CF community for bringing this medicine to eligible people with CF, particularly those without a medicine targeting the underlying cause of their disease, said Reshma Kewalramani, M.D., Executive Vice President, Global Medicines Development and Medical Affairs and Chief Medical Officer at Vertex. We remain committed to relentlessly pursuing the development of transformative therapies for all people living with this disease.

Vertex has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for the elexacaftor/tezacaftor/ivacaftor combination regimen. Vertex is currently evaluating elexacaftor/tezacaftor/ivacaftor in people ages 6 through 11 with F/MF and F/F CF mutations in an ongoing Phase 3 study and is committed to evaluating elexacaftor/tezacaftor/ivacaftor in children <6 years of age as part of planned future studies.

For more information on Vertexs patient assistance program, please visit VertexGPS.com.

About TRIKAFTA TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients ages 12 years and older who have at least one copy of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Patients should talk to their doctor to learn if they have an indicated CF gene mutation. It is not known if TRIKAFTA is safe and effective in children under 12 years of age. TRIKAFTA is designed to increase the quantity and function of the F508del-CFTR protein at the cell surface. The approval of TRIKAFTA was supported by positive results of two global Phase 3 studies in people ages 12 years and older with CF: a 24-week Phase 3 study in 403 people with one F508del mutation and one minimal function mutation (F/MF) and a 4-week Phase 3 study in 107 people with two F508del mutations (F/F).

Helping Patients Access TRIKAFTA The people who work at Vertex understand that medicines can only help people who can get them. The Vertex Guidance & Patient Support (Vertex GPS) program provides a team of Vertex employees dedicated to helping eligible people in the United States who have been prescribed our medicines understand their insurance benefits and the resources that are available to help them.

Vertex also offers a co-pay assistance program for eligible people with commercial insurance coverage and a free medicine program for qualifying people who meet certain income and other eligibility criteria. More information is available by visiting http://www.VertexGPS.com or by calling 1-877-752-5933.

About Cystic Fibrosis Cystic Fibrosis (CF) is a rare, life-shortening genetic disease affecting approximately 75,000 people worldwide. CF is a progressive, multi-system disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes one from each parent to have CF. While there are many different types of CFTR mutations that can cause the disease, the vast majority of all people with CF have at least one F508del mutation. These mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working and/or too few CFTR proteins at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the early 30s.

INDICATIONS AND IMPORTANT SAFETY INFORMATION FOR TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) TABLETS TRIKAFTA is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients aged 12 years and older who have at least one copy of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Patients should talk to their doctor to learn if they have an indicated CF gene mutation. It is not known if TRIKAFTA is safe and effective in children under 12 years of age.

Patients should not take TRIKAFTA if they take certain medicines, such as: antibiotics such as rifampin or rifabutin; seizure medicines such as phenobarbital, carbamazepine, or phenytoin; St. Johns wort.

Before taking TRIKAFTA, patients should tell their doctor about all of their medical conditions, including if they: have kidney problems, have or have had liver problems, are pregnant or plan to become pregnant because it is not known if TRIKAFTA will harm an unborn baby, or are breastfeeding or planning to breastfeed because it is not known if TRIKAFTA passes into breast milk.

TRIKAFTA may affect the way other medicines work, and other medicines may affect how TRIKAFTA works. Therefore, the dose of TRIKAFTA may need to be adjusted when taken with certain medicines. Patients should especially tell their doctor if they take: antifungal medicines including ketoconazole, itraconazole, posaconazole, voriconazole, or fluconazole; antibiotics including telithromycin, clarithromycin, or erythromycin; other medicines including rifampin, rifabutin, phenobarbital, carbamazepine, phenytoin, and St. Johns wort.

TRIKAFTA may cause dizziness in some people who take it. Patients should not drive a car, operate machinery, or do anything that requires alertness until they know how TRIKAFTA affects them.

Patients should avoid food or drink that contains grapefruit while they are taking TRIKAFTA.

TRIKAFTA can cause serious side effects, including:

High liver enzymes in the blood, which is a common side effect in people treated with TRIKAFTA. These can be serious and may be a sign of liver injury. The patients doctor will do blood tests to check their liver before they start TRIKAFTA, every 3 months during the first year of taking TRIKAFTA, and every year while taking TRIKAFTA. Patients should call their doctor right away if they have any of the following symptoms of liver problems: pain or discomfort in the upper right stomach (abdominal) area; yellowing of the skin or the white part of the eyes; loss of appetite; nausea or vomiting; dark, amber-colored urine.

Abnormality of the eye lens (cataract) in some children and adolescents treated with TRIKAFTA. If the patient is a child or adolescent, their doctor should perform eye examinations before and during treatment with TRIKAFTA to look for cataracts.

The most common side effects of TRIKAFTA include headache, diarrhea, upper respiratory tract infection (common cold) including stuffy and runny nose, stomach (abdominal) pain, inflamed sinuses, increase in liver enzymes, increase in a certain blood enzyme called creatine phosphokinase, rash, flu (influenza), and increase in blood bilirubin.

These are not all the possible side effects of TRIKAFTA. Please click here to see the full Prescribing Information for TRIKAFTA.

About Vertex Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has four approved medicines that treat the underlying cause of cystic fibrosis (CF) - a rare, life-threatening genetic disease - and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational medicines in other serious diseases where it has deep insight into causal human biology, such as sickle cell disease, beta thalassemia, pain, alpha-1 antitrypsin deficiency, Duchenne muscular dystrophy and APOL1-mediated kidney diseases.

Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London, UK. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including nine consecutive years on Science magazine's Top Employers list and top five on the 2019 Best Employers for Diversity list by Forbes. For company updates and to learn more about Vertex's history of innovation, visit http://www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

Special Note Regarding Forward-looking Statements This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, the statements by Dr. Leiden in the second paragraph, Dr. Rowe in the third paragraph, and Dr. Kewalramani in the fourth paragraph and statements regarding Vertexs current and future plans to study elexacaftor/tezacaftor/ivacaftor in the fifth paragraph of this press release. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from the company's development programs may not support registration or further development of its compounds due to safety, efficacy or other reasons, obtaining approval and commercializing elexacaftor/tezacaftor/ivacaftor in Europe, developing additional medicines to treat cystic fibrosis, and other risks listed under Risk Factors in Vertex's annual report and subsequent quarterly reports filed with the Securities and Exchange Commission and available through the company's website at http://www.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

View source version on businesswire.com: https://www.businesswire.com/news/home/20191021005792/en/

Read this article:
FDA Approves TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to Treat the Underlying Cause of Cystic Fibrosis in People Ages 12 and Older...

Genentech’s Tecentriq in Combination With Avastin Increased Overall Survival and Progression-free Survival in People With Unresectable Hepatocellular…

Oct. 21, 2019 05:00 UTC

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)-- Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that the Phase III IMbrave150 study, evaluating Tecentriq (atezolizumab) in combination with Avastin (bevacizumab) as a treatment for people with unresectable hepatocellular carcinoma (HCC) who have not received prior systemic therapy, met both of its co-primary endpoints demonstrating statistically significant and clinically meaningful improvements in overall survival (OS) and progression-free survival (PFS) compared with standard-of-care sorafenib.

Safety for the combination of Tecentriq and Avastin was consistent with the known safety profiles of the individual medicines, with no new safety signals identified. Data from the IMbrave150 study will be presented at an upcoming medical meeting.

We are very pleased with the results of our study testing the combination of Tecentriq and Avastin, which marks the first treatment in more than a decade to improve overall survival in people with unresectable hepatocellular carcinoma who have not received prior systemic therapy, said Levi Garraway, M.D., Ph.D., chief medical officer and head of Global Product Development. HCC is a major cause of death globally and particularly in Asia, making this study an important step in our mission of addressing unmet medical needs for patients around the world. We will submit these data to global health authorities as soon as possible. Our hope is to bring a new treatment to people with this aggressive disease who currently have limited options.

In July 2018, the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation (BTD) for Tecentriq in combination with Avastin in HCC based on data from an ongoing Phase Ib trial.

Genentech has an extensive development program for Tecentriq, including multiple ongoing and planned Phase III studies, across lung, genitourinary, skin, breast, gastrointestinal, gynecological and head and neck cancers. This includes studies evaluating Tecentriq both alone and in combination with other medicines.

About the IMbrave150 study

IMbrave150 is a global Phase III, multicenter, open-label study of 501 people with unresectable HCC who have not received prior systemic therapy. People were randomized 2:1 to receive the combination of Tecentriq and Avastin or sorafenib. Tecentriq was administered intravenously, 1200 mg on day 1 of each 21-day cycle, and Avastin was administered intravenously, 15 mg/kg on day 1 of each 21-day cycle. Sorafenib was administered by mouth, 400 mg twice per day, on days 1-21 of each 21-day cycle. People received the combination or the control arm treatment until unacceptable toxicity or loss of clinical benefit as determined by the investigator. Co-primary endpoints were OS and PFS by independent review facility (IRF) per Response Evaluation Criteria in Solid Tumors Version 1.1 (RECIST v1.1). Secondary efficacy endpoints included overall response rate (ORR), time to progression (TTP) and duration of response (DoR), as measured by RECIST v1.1 (investigator-assessed [INV] and IRF) and HCC mRECIST (IRF), as well as patient-reported outcomes (PROs), safety and pharmacokinetics.

About hepatocellular carcinoma

According to the American Cancer Society, it is estimated that more than 42,000 Americans will be diagnosed with liver cancer in 2019. Liver cancer incidence has more than tripled since 1980 and HCC accounts for approximately 75% of all liver cancer cases in the United States. HCC develops predominantly in people with cirrhosis due to chronic hepatitis (B and C) or alcohol consumption, and typically presents at an advanced stage where there are limited treatment options.

About the Tecentriq and Avastin combination

There is a strong scientific rationale to support further investigation of Tecentriq plus Avastin in combination. Avastin, in addition to its anti-angiogenic effects, may further enhance Tecentriqs ability to restore anti-cancer immunity by inhibiting vascular endothelial growth factor (VEGF)-related immunosuppression, promoting T-cell tumor infiltration and enabling priming and activation of T-cell responses against tumor antigens.

About Tecentriq (atezolizumab)

Tecentriq is a monoclonal antibody designed to bind with a protein called PD-L1. Tecentriq is designed to bind to PD-L1 expressed on tumor cells and tumor-infiltrating immune cells, blocking its interactions with both PD-1 and B7.1 receptors. By inhibiting PD-L1, Tecentriq may enable the re-activation of T cells. Tecentriq may also affect normal cells.

About Avastin (bevacizumab)

Avastin is a prescription-only medicine that is a solution for intravenous infusion. It is a biologic antibody designed to specifically bind to a protein called VEGF that plays an important role throughout the lifecycle of the tumor to develop and maintain blood vessels, a process known as angiogenesis. Avastin is designed to interfere with the tumor blood supply by directly binding to the VEGF protein to prevent interactions with receptors on blood vessel cells. The tumor blood supply is thought to be critical to a tumors ability to grow and spread in the body (metastasize).

Tecentriq U.S. Indications

Tecentriq is a prescription medicine used to treat adults with:

A type of bladder and urinary tract cancer called urothelial carcinoma. Tecentriq may be used when your bladder cancer:

The approval of Tecentriq in these patients is based on a study that measured response rate and duration of response. Continued approval for this use may depend on the results of an ongoing study to confirm benefit.

A type of lung cancer called non-small cell lung cancer (NSCLC).

A type of breast cancer called triple-negative breast cancer (TNBC).

Tecentriq may be used with the medicine paclitaxel protein-bound when your breast cancer:

The approval of Tecentriq in these patients is based on a study that measured the amount of time until patients disease worsened. Continued approval for this use may depend on results of an ongoing study to confirm benefit.

A type of lung cancer called small cell lung cancer (SCLC).

It is not known if Tecentriq is safe and effective in children.

Important Safety Information

What is the most important information about Tecentriq?

Tecentriq can cause the immune system to attack normal organs and tissues and can affect the way they work. These problems can sometimes become serious or life threatening and can lead to death.

Patients should call or see their healthcare provider right away if they get any symptoms of the following problems or these symptoms get worse.

Tecentriq can cause serious side effects, including:

Getting medical treatment right away may help keep these problems from becoming more serious. A healthcare provider may treat patients with corticosteroid or hormone replacement medicines. A healthcare provider may delay or completely stop treatment with Tecentriq if patients have severe side effects.

Before receiving Tecentriq, patients should tell their healthcare provider about all of their medical conditions, including if they:

Patients should tell their healthcare provider about all the medicines they take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

The most common side effects of Tecentriq when used alone include:

The most common side effects of Tecentriq when used in lung cancer with other anti-cancer medicines include:

The most common side effects of Tecentriq when used in triple-negative breast cancer with paclitaxel protein-bound include:

Tecentriq may cause fertility problems in females, which may affect the ability to have children. Patients should talk to their healthcare provider if they have concerns about fertility.

These are not all the possible side effects of Tecentriq. Patients should ask their healthcare provider or pharmacist for more information. Patients should call their doctor for medical advice about side effects.

Report side effects to the FDA at 1-800-FDA-1088 or http://www.fda.gov/medwatch.

Report side effects to Genentech at 1-888-835-2555.

Please visit http://www.Tecentriq.com for the Tecentriq full Prescribing Information for additional Important Safety Information.

Avastin is approved for:

Avastin in combination with paclitaxel, pegylated liposomal doxorubicin or topotecan, is approved to treat platinum-resistant recurrent epithelial ovarian, fallopian tube or primary peritoneal cancer (prOC) in women who received no more than two prior chemotherapy treatments.

Avastin, either in combination with carboplatin and paclitaxel or with carboplatin and gemcitabine, followed by Avastin alone, is approved for the treatment of patients with platinum-sensitive recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer (psOC)

Possible serious side effects

Everyone reacts differently to Avastin therapy. So, its important to know what the side effects are. Although some people may have a life-threatening side effect, most do not. Their doctor will stop treatment if any serious side effects occur. Patients should contact their health care team if there are any signs of these side effects.

Side effects seen most often

In clinical studies across different types of cancer, some patients experienced the following side effects:

Avastin is not for everyone

Patients should talk to their doctor if they are:

Patients should talk with their doctor if they have any questions about their condition or treatment.

Report side effects to the FDA at 1-800-FDA-1088 or http://www.fda.gov/medwatch.

Report side effects to Genentech at 1-888-835-2555.

For full Prescribing Information and Boxed WARNINGS on Avastin please visit http://www.avastin.com.

About Genentech in personalized cancer immunotherapy

For more than 30 years, Genentech has been developing medicines with the goal to redefine treatment in oncology. Today, were investing more than ever to bring personalized cancer immunotherapy (PCI) to people with cancer. The goal of PCI is to provide each person with a treatment tailored to harness his or her own immune system to fight cancer. Genentech is currently studying more than 10 cancer immunotherapy medicines across 70 clinical trials alone or in combination with other medicines. In every study we are evaluating biomarkers to identify which people may be appropriate candidates for our medicines. For more information visit http://www.gene.com/cancer-immunotherapy.

About Genentech

Founded more than 40 years ago, Genentech is a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious and life-threatening medical conditions. The company, a member of the Roche Group, has headquarters in South San Francisco, California. For additional information about the company, please visit http://www.gene.com.

View source version on businesswire.com: https://www.businesswire.com/news/home/20191020005095/en/

See original here:
Genentech's Tecentriq in Combination With Avastin Increased Overall Survival and Progression-free Survival in People With Unresectable Hepatocellular...