Genome Editing a Progressing Trend

The Rush for Genome Editing

Frank Magliochetti Report

The Rush for Genome Editing

Gene-editing technology has sparked a frenzy of competitors fueled to develop the first of what may be thousands of treatments for genetic disorders.

From science fiction of movies like Jurassic Park to the reality of GMO foods, the concept of customizing plants, animals and even human genomes has mesmerized our imagination and culture for decades. This obsession is not surprising considering the remarkable impact of genetic engineering on our lives, and the promise it may hold for our future.crispr-genome-editing-franlmagliochetti-report_northandoverma

Modifying the genes of an organism is no easy task. It’s not as simple as removing the parts you do not like with an eraser. It involves making changes at a molecular level that are both complex and challenging.

One concern among scientists is the modifications made in human germline cells – i.e. the sperm or the egg. These are genetic changes that would actually be passed on through generations and the fear is that there could be unexpected side effects. Couple that with the ethical concerns of people misusing this technology to intentionally modify the genome to make “designer babies” with enhanced characteristics. These are some real concerns that are causing scientists to pause and weigh the pros and cons of use of this technology.

More and more companies are racing to stay ahead of the trend and become the leaders in the field.

In Cambridge MA, Intellia Therapeutics Inc. has a 65,000 square foot build out underway, however, it is still planning to functioning while under construction.

Intellia is one of a trio of startups in the Cambridge area working locally in the biotech niche known as CRISPR-Cas9. The gene-editing technology was frank-magliochetti-report_intelliadiscovered just four years ago.

Drug development historically has been a slow process – it often takes more than a decade to bring a medicine from lab to marketplace. But that’s changing, thanks to new machines that can screen drug targets faster and an entrepreneurial environment that’s more akin to high-tech than Big Pharma.frankmagliochettireport_genome_editing

Intellia has high-profile collaboration deals with Swiss drug giant Novartis AG and Regeneron Pharmaceuticals Inc. of Tarrytown, N.Y. It raised $108 million in an initial public offering in May. Their scientists are working on gene-editing treatments for everything including the hepatitis B virus to a rare form of blindness. If all goes well, it could move its earliest drug candidates into clinical trials within 18 to 24 months.

Competitors for Intellia include Editas Medicine and Crispr Therapeutics Inc. both completed their own IPOs this year. There are more to come: all with the heightened interest of investors. These companies are in a race to develop the first treatment based on gene editing.

Genome editing is going to be a hot trend to watch in 2017 and for many years to come.

Frank Magliochetti is Managing Partner for Parcae Capital.

  • North Andover, Massachusetts

This column of posts is directed at the Healthcare Industry.  Frank Magliochetti Report is a new site dedicated to the industry.  He currently assists companies who are building, restructuring, transforming and resurrecting there business’s. An example of his client base are, Xenetic Biosciences , IPC Medical Corp, Just Fellowship Corp, Environmental Services Inc., Parsons Post House LLC, ClickStream Corporation as well as having a business talk radio show; The Business Architect on the URBN network.

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Personal Healthcare Spending Trends

New Study Sheds Light on Personal Health Care Spending Trends in the U.S.

The amount of dollars the Unites Spends on healthcare surpasses every other nation on the planet; all in all accounting for 17% of the economy in the U.S.

Frank Magliochetti

People spend more for health care in the United States than in any other nation on earth and, according to results of a new study published in JAMA, they spend more on diabetes and ischemic heart disease than on any other health condition.

Health care spending continues to rise in the U.S., now accounting for 17 percent of the nation’s economy. health-spending-Frank-MAgliochetti-ReportDespite this spending, there is very little information on how spending varies by condition, age and through time. Joseph L. Dieleman, PhD, from the Institute for Health Metrics and Evaluation, University of Washington and a team of researchers hoped to estimate national spending on personal health care by various factors.

U.S. Spending on Health Care Trends

The researchers included 183 sources of data in the study. Data sources included insurance claims, government budgets, household surveys, facility surveys, and official U.S. records from 1996 to 2013. The scientists grouped ICD-9 codes to form 155 conditions, such as diabetes and ischemic heart disease, for consideration in the study.

One of the more interesting findings of the study was that many of the top 20 conditions of health care spending were chronic conditions with a relatively high prevalence and health burden – many of them were also preventable. This group of conditions included diabetes, ischemic heart disease, chronic obstructive pulmonary disease (COPD), and cerebrovascular disease, all of which are attributable to modifiable risk factors.

Total costs of care

Americans spent $30.1 trillion on personal health care during the years included in the study. The researchers looked at how Americans spent that money, estimating the costs of treating 155 conditions. They found that, at an estimated $101 billion, Americans spent the most on treating diabetes. Spending for ischemic heart disease came in second at $88 billion; spending for low back and neck pain was a close third at $87 billion.

Increases in costs of care

Spending for personal health care increased for 143 of the conditions investigated over the course of the study. Spending on low back and neck pain increased $57 billion during those 18 years, and spending on diabetes US-healthcare-costs-FrankMagliochettiincreased $64 billion during that period.

Of all the conditions included in the study, 57 percent of spending went towards the top 20 conditions. Spending on emergency care and retail pharmaceuticals rose the fastest, at 6.4 percent and 5.6 percent annual growth rate, respectively. When it came to spending on diabetes, 57.6 percent went to pharmaceuticals while 23.5 percent was for ambulatory care.

The study was important in that it was the first to provide modeled estimates of U.S. personal health care spending. The results were revealing in that they showed that diabetes, ischemic heart disease, and low back and neck pain presented the highest costs to American consumers. The study was limited in that it used population-weighted data to represent total national spending, which excludes incarcerated persons and those receiving care from a Veterans Affairs (VA) facility. The University of Washington institutional review board reviewed and approved the project.

The information presented in the study may be useful to health care policy makers and health care providers working towards making health care spending more cost effective for the conditions that most commonly affect people living in the United States.

Source

http://jamanetwork.com/journals/jama/fullarticle/2594716

Frank Magliochetti is Managing Partner for Parcae Capital

  • North Andover, Massachusetts

This column of posts is directed at the Healthcare Industry.  Frank plans to release new sites dedicated to the industry. Frank currently assists companies who are building, restructuring, transforming and resurrecting there business’s. An example of his client base are, Xenetic Biosciences , IPC Medical Corp, Just Fellowship Corp, Environmental Services Inc., Parsons Post House LLC, ClickStream Corporation as well as having a business talk radio show; The Business Architect on the URBN network.

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2017: Pharma Trends- Biosimilars

2017: Pharma Trends- Biosimilars

Pharma Trends 2017:  Biosimilars

Frank Magliochetti Report

The FDA approved 56 new drugs in 2015 and, of these, 33 were specialty drugs and 23 were traditional. One was a biosimilar. Development, approval and acceptance of biosimilars may become an important trend in 2017.
The FDA is approving more specialty drugs than ever before and specialty medicines will likely face more competition in 2017. Increased FDA approval and frankmagliochetti-report-pharma-trends-biosimilarsdiscoveries of medications to treat orphan conditions and cancer will fuel competition. Biosimilars could overcome development hurdles and increase this competition even more.
Acceptance of biosimilars has been slow in the past. One of the reasons for the reluctance is that the development of biosimilars is different from that of generic drugs. Generic drugs for small molecules have a relative simple chemical structure that makes it easy to reproduce an identical chemical. It is more difficult to make a protein or peptide identical to the original biologic agent because of differences in the sugar residues attached to the protein, for example, or differences in the protein folding that make it unpredictable. This means that, even though a biosimilar has an identical peptide chain, it may not have an identical effect. These factors complicate the process of making biosimilars and increase the risk for problems.
Physician acceptance has been slow too but, as the cost of other medications rise, biosimilars will seem more attractive.
Approval of Biosimilars in 2015 and 2016
Biosimilars, which are products the FDA will approve with evidence that the medication up for approval is highly similar to an already-approved reference product, will probably continue to gain traction in 2017. The FDA has approved four biosimilars as of October 2016.
Zarxio is similar to Neupogen, according to an article published by Managed Healthcare Executive, except the biosimilar has a 15 percent lower list price. Both drugs treat low white blood cell counts associated with cancer and its treatment. The FDA approvedZarxio, made by Sandoz, in March 2015.
Inflectra is similar to Remicade to treat Crohn’s disease and ulcerative colitis. The FDA approved Inflectra in April 2016 but, due to litigation, the launch date is uncertain.
Erelzi, also made by Sandoz, is similar to Enbrel to treat arthritis. The FDA announced approval of Erelzi in August of 2016 and the expected launch date of biosimilar-frankmagliochetti-pharma-trends-2017February 2017 may be delayed.
Amjevita, by Amgen is similar to Humira and is for the treatment multiple inflammatory diseases. Amjevita became the fourth biosimilar drug to gain approval in September 2016. Launch date is set for March 2017 but may experience delays.
There are several biosimilars awaiting approval, including:
• Filgrastim by Grastofil, Apotex
• Pegfilgrastim by Apotex
• SB2 infliximab by Merck/Samsung Bioepis
• CHS‐1701 by Coherus Biosciences
• Epoetin alfa by Retacrit, Pfizer
The need for biosimilars will grow as the need for other drugs, especially cancer drugs and orphan drugs, increases. Doctors diagnose about 1.7 million cases of cancer each year, yet the death rate has fallen 23 percent since the early 1990s, partially due to new medications and treatments. The introduction of biosimilars could reduce cancer death rates even further.
Biosimilars may become one of the strongest trends in the pharmaceutical industry in upcoming years, as biosimilars have the potential to lower the cost for safe and effective treatments for a wider number of patients. Increased development of biosimilar cancer drugs is possible as some biopharmaceuticals lose patent protection.

Source
https://www.uspharmacist.com/article/record-number-of-fda-new-drug-approvals-in-2015
http://www.zarxio.com/index.jsp
http://managedhealthcareexecutive.modernmedicine.com/managed-healthcare-executive/news/five-specialty-pharmaceutical-trends-watch
http://www.fda.gov/newsevents/newsroom/pressannouncements/ucm436648.htm
http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm494227.htm
http://www.erelzi.com/
http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm518639.htm
http://pi.amgen.com/united_states/AMJEVITA/AMJEVITA_mg.pdf
http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm522243.htm

Frank Magliochetti is Managing Partner for Parcae Capital.

  • North Andover, Massachusetts

This column of posts is directed at the Healthcare Industry.  Frank plans to release a new site dedicated to the industry.  He currently assists companies who are building, restructuring, transforming and resurrecting there business’s. An example of his client base are, Xenetic Biosciences , IPC Medical Corp, Just Fellowship Corp, Environmental Services Inc., Parsons Post House LLC, ClickStream Corporation as well as having a business talk radio show; The Business Architect on the URBN network.

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CDC Recommends Not Using Nasal Spray Flu Vaccine

CDC: Avoid Using Nasal Spray Flu Vaccine for 2016/2017 Season

Frank Magliochetti Report

The Centers for Disease Control and Prevention (CDC) has issued its 2016-2017 influenza vaccination recommendations, which includes the use of inactivated influenza vaccine (IIV) and the recombinant influenza vaccine (RIV). One of the most notable changes to the yearly recommendations is that the CDC is not recommending the use of nasal spray flu vaccinations using live attenuated frankmagliochetti-report_flu-shot-vaccineinfluenza vaccine (LAIV) during 2016-2017, as studies show it is not effective against preventing the flu.
The studies show LAIV had no protective benefit from children ages 2 to 17. In contrast, children who received an injection were 63 percent less likely to catch influenza than are those who received no vaccination at all.
While researchers have not yet determined the underlying cause of the loss of efficacy, but it could be due to changes in the dominant viral strains circulating in the nation during flu season. LAIV worked just as well as the shot at protecting children from H3N3 and the influenza B strain in recent flu seasons, according to a study published in the journal Pediatrics, but youngsters who received LAIV were three times more likely to suffer infection with H1N1 than were children who got the shot.
H1N1 has been the dominant strain for the past few years, according to a February 2016 CDC report, which means LAIVs can leave patients unprotected from the strains most likely to cause illness this flu season.

About Vaccinations, LAIVs, and Flu Season

Influenza vaccinations protect millions of people in the United States from developing influenza each year. Flu shots also reduce severity of influenza symptoms when it does develop, and prevents cdc-frankmagliochettireporthospitalizations relating to the flu. CDC recommends yearly flu shots for everyone 6 months and older. Antibodies typically take about two weeks to develop.
Many patients, and parents of pediatric patients, prefer LAIVs because of the perceived pain or trauma of an injection. LAIVs have been under development in the United States since the 1960s, according to the CDC, and the intranasal, trivalent, cold-adapted, live, LAIV received approval in 2003 as an option for among health patients aged 5 to 49 years. Because the nasal spray contains live, weakened influenza viruses, LAIV theoretically stimulates a stronger immune response than the shots, which contain killed viruses.
Influenza activity often begins to increase in October and peak sometime in late winter or early spring. The peak week of flu activity for the 2015-2016 season occurred on the last week of March 2016,

according to the CDC, which was one of the later season peaks on record. The CDC’s adjusted overall vaccine effectiveness (VE) against influenza A and B viruses for all ages was 47 percent, while the overall VE against A(H1N1)pdm09 was 41 percent and the overall VE against influenza B was 55 percent.

Vaccine manufacturers will provide an estimated 157 to 168 million doses of injectable influenza vaccine for the U.S. market. This is on par with last year’s vaccine distribution.

Source
https://www.cdc.gov/mmwr/volumes/65/rr/rr6505a1.htm?s_cid=rr6505a1_w

http://pediatrics.aappublications.org/content/early/2016/01/04/peds.2015-3279?sso=1&sso_redirect_count=1&nfstatus=401&nftoken=00000000-0000-0000-0000000000000000&nfstatusdescription=ERROR%3a+No+local+token

https://emergency.cdc.gov/han/han00387.asp

https://www.cdc.gov/mmwr/preview/mmwrhtml/rr5213a1.htm

http://www.cdc.gov/flu/about/season/flu-season-2015-2016.htm

https://www.cdc.gov/flu/professionals/vaccination/vaccinesupply.htm

Frank Magliochetti is Managing Partner for Parcae Capital.

  • North Andover, Massachusetts

This column of posts is directed at the Healthcare Industry.  Frank plans to release a new site dedicated to the industry.  He currently assists companies who are building, restructuring, transforming and resurrecting there business’s. An example of his client base are, Xenetic Biosciences , IPC Medical Corp, Just Fellowship Corp, Environmental Services Inc., Parsons Post House LLC, ClickStream Corporation as well as having a business talk radio show; The Business Architect on the URBN network.

frankmagliochetti_ParcaeCapital

Pharma: Innovative Developments in Medicine- Vaccines

Preventative Vaccines:  Bexsero & Trumenba

Frank Magliochetti Report

One of the biggest public health epidemics occurred back in 2014; Ebola and Meningococcal B were responsible. These two diseases took 10,000 people in Sierra Leone, Guinea and Liberia. These three countries have weak health systems, which is the reason why they were unable to treat their people.

In order to fight this disease, which made its first appearance in 1976,  WHO (World Health Organization) had been trying to find a cure for many years. On August 8, 2015, WHO declared that Ebola and Meningococcal B is a disease of international concern and thus began the search for its vaccine. By late October, 2015 WHO had finally come up with two vaccines that were guaranteed to prevent Meningococcal B. Sadly; the cure for Ebola has not yet been found however, its vaccines trials are currently being conducted in Sierra Leone.who_frankmagliochettireport

Meningococcal B is caused by bacteria called “Neisseria Meningitidis”. Neisseria Meningitidis has five serogroups: B, C, Y, W and A. The most common serogroups that affect people in the US is B, C and Y.

Meningococcal B disease is not caught through the air. In fact, it is a diseasewhich is transmitted from one person to another in close physical contact. The disease commonly spreads faster in the family due to the exchange of fluids from daily contact. The disease is found at the back of the throat and later causes sepsis. Sepsis is transferred to the blood that infects the blood stream, which causes damage to the brain and spinal column. Meningococcal B often results in liver failure, vision loss, amputation of limbs and death.

bexsero-frankmagliochettireportThe two vaccines Bexsero and Trumenba specifically treat Meningococcal B and were approved by the FDA in October 2015. The trials for Bexsero were conducted in UK, Chile, Australia and Canada amongst 2,600 young adults and adolescents. The results were that the three infectious strains that cause Meningococcal were destroyed.

Another 5,000 participants in the trials conducted in the USA experienced headache, muscle and joint pain, chills, fatigue and diarrhea after receiving the vaccine. Whereas, 15,000 participants experienced less side effects. All in all, the FDA approved the vaccines because the side effects were not viewed aslong term or fatal.

An accelerated approval was used by the FDA to make this vaccine go public claiming that Meningococcal B is a life-threatening disease. The effectiveness of Bexsero was approved two months prior to the trials being conducted.

FDA recommends that the vaccine should be given to infants, children from the ages of ten till twenty three and people who have a weak immune system. It is recommended that more than one dosage should be given to people having serogroup B Meningococcal.

Anna Jaques, a non-profit Hospital in Newburyport, Massachusetts has taken extra measures and brought more doctors and experts on anna_jaques_hospitaltheir team to prepare themselves if a patient with Ebola or Meningococcal B comes to their door.

https://www.ajh.org/

Anna Jaques is a remarkable medical facility housed with some of the top health care professionals North of Boston.

Frank Magliochetti

With 2016 being the year of medical innovations, the vaccine for Meningococcal B has come as a godsend that will save millions of lives in rural areas where this disease is commonly found. The vaccines are set to be released worldwide by the end of the year.

Other News and information on Trumenba and Bexsero

Trumenba Offical Website:

http://www.trumenba.com/

Important Safety Information

  • Trumenba should not be given to anyone with a history of a severe allergic reaction after a previous dose of Trumenba
  • Individuals with weakened immune systems may have a reduced immune response
  • The most common adverse reactions were pain at the injection site, fatigue, headache, muscle pain, and chills
  • Data is not available on the safety and effectiveness of using Trumenba and other meningococcal group B vaccines interchangeably to complete the vaccination series
  • Tell your healthcare provider prior to the vaccination if you are pregnant, or plan to become pregnant
  • Ask your healthcare provider about the risks and benefits of Trumenba. Only a healthcare provider can decide if Trumenba is right for you or your child

 

trumenba-frankmagliochettireportIndication

  • Trumenba is a vaccine indicated for individuals 10 through 25 years of age for active immunization to prevent invasive disease caused by Neisseria meningitidisgroup B
  • Trumenba is approved based upon demonstrated immune response against four group B strains representative of prevalent strains in the US. The effectiveness of Trumenba against diverse group B strains has not been confirmed

Bexsero

Official Site

https://www.gsksource.com/pharma/content/gsk/source/us/en/brands/bexsero/pi/po.html?cc=3282D9F3E686&pid=

Important Safety Information

Indication

BEXSERO is a vaccine indicated for active immunization to prevent invasive disease caused by Neisseria meningitidis serogroup B. BEXSERO is approved for use in individuals 10 through 25 years of age.

Approval of BEXSERO is based on demonstration of immune response, as measured by serum bactericidal activity against three serogroup B strains representative of prevalent strains in the United States. The effectiveness of BEXSERO against diverse serogroup B strains has not been confirmed.Bexsero_logo_4C_temp_norDNA_bigger

  • BEXSERO is contraindicated in cases of hypersensitivity, including severe allergic reaction, to any component of the vaccine, or after a previous dose of BEXSERO
  • Appropriate observation and medical treatment should always be readily available in case of an anaphylactic event following the administration of the vaccine
  • The tip caps of the prefilled syringes contain natural rubber latex, which may cause allergic reactions in latex-sensitive individuals
  • Syncope (fainting) can occur in association with administration of BEXSERO. Ensure procedures are in place to avoid injury from falling associated with syncope
  • The most common solicited adverse reactions observed in clinical trials were pain at the injection site (≥83%), myalgia (≥48%), erythema (≥45%), fatigue (≥35%), headache (≥33%), induration (≥28%), nausea (≥18%), and arthralgia (≥13%)
  • Vaccination with BEXSERO may not provide protection against all meningococcal serogroup B strains
  • Vaccination with BEXSERO may not result in protection in all vaccine recipients

AAP issues meningococcal B vaccine ( Trumenba & Bexsero)  guidelines for adolescents
Trumenba and Bexsero have both been approved for the prevention of serogroup B meningococcal disease in individuals aged 10 through 25 years by the American Academy of Pediatrics Committee on Infectious Diseases.

Please Click the link below to read the entire post:

http://www.healio.com/internal-medicine/vaccination/news/online/%7B9d42e15b-0c08-477d-8b1e-f28a59c545ea%7D/aap-issues-meningococcal-b-vaccine-guidelines-for-adolescents

Pfizer heads back to school with Trumenba vaccination campaign

Please click the link below to read the entire post:

http://www.fiercepharma.com/marketing/pfizer-menb-vaccination-campaign-for-trumenba-gears-up-for-back-to-school

Teaching kids to share is usually a parental goal, but a new ad from Pfizer may have some parents rethinking the parameters.

The first campaign for meningococcal group B vaccine Trumenba highlights the dangers of MenB, which can be spread through habits common among teens–including kissing and sharing drinks and food.

The TV ad shows a mother in the hospital with her stricken son, asking “how did we get here?” The ad then goes back through events earlier in evening showing the son sharing food and drinks with several different friends at a party and kissing a girl.

“The campaign was designed to reach parents of teens and young adults during the summer and back-to-school season, when many parents are thinking about steps they can take to help protect their child’s health, including vaccination,” she said.

Trumenba is one of two vaccinations to protect against MenB, along with GlaxoSmithKline’s Bexsero. But last year, the CDC’s Advisory Committee for Immunization Practices (ACIP) stopped short of granting the pair universal recommendation. The vaccines are recommended for young adults ages 16 to 23, with a preferred age of 16 to 18, which means that doctors decide on an individual basis whether to vaccinate their patients against MenB.

watch the ad on iSpot.tv

https://www.ispot.tv/ad/ARCj/trumenba-meningitis-b

Frank Magliochetti is Managing Partner for Parcae Capital.

  • North Andover, Massachusetts

This column of posts is directed at the Healthcare Industry.  Frank plans to release a new site dedicated to the industry.  He currently assists companies who are building, restructuring, transforming and resurrecting there business’s. An example of his client base are, Xenetic Biosciences , IPC Medical Corp, Just Fellowship Corp, Environmental Services Inc., Parsons Post House LLC, ClickStream Corporation as well as having a business talk radio show; The Business Architect on the URBN network.

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