Sonoma State University Will Host The Academy of Wine Business Research Conference July 26 – 27

The Wine Business Institute (WBI) at Sonoma State University (SSU) will host the 10th annual Academy of Wine Business Research (AWBR) conference July 26 and 27, including paper presentations, breakout sessions, and panel discussions on current issues, innovations, and challenges faced by the global wine industry. Confirmed attendees represent wine businesses and academic institutions in Austria, Australia, Brazil, Chile, China, France, Germany, Italy, New Zealand, Spain, and the United States.

From Ray Johnson, Executive Director of the Wine Business Institute:

“It’s a pleasure to offer this opportunity for the industry’s influencers and academics to convene and connect on pressing issues. It highlights California’s importance to the global wine industry, and the Wine Business Institute’s leadership in education and research. The wine industry is changing, and this conference offers a deep dive for exchanges, discoveries, and takeaways on the role of research and education in advancing the industry’s competitiveness and sustainability in a global environment.”

WHO:          

Wine Business Institute at Sonoma State University

The Academy of Wine Business Research

WHAT:         

10th annual Academy of Wine Business Research Conference, including research presentations under the headings:

  • Online Purchases of Wine
  • Managing Uncertainty in the Wine Industry
  • Measuring Market Performance
  • Understanding Wine Purchasing Behavior
  • Supply Chain and Exporting Issues
  • Industry Perspectives on Sustainability
  • Managerial Approaches
  • Sustainability and its Link with Customers
  • Wine Ratings and Raters
  • Wine Industry Trends
  • The Role of Tradition in the Wine Industry
  • Case Study Workshop
  • A Better Understanding of Wine Consumers
  • Marketing of Wine Terroir and Territories
  • Wine Marketing in a Digital Age
  • Wine Tourism
  • Marketing and the Wine Purchase Decision
  • Management Strategy
  • Wine Sales and Trends
  • Wine Marketing in Eastern and Southern Europe
  • Wine Industry Development and Growth

WHERE:       

Sonoma State University Student Center

1801 East Cotati Avenue

Rohnert Park, CA 94928

WHEN:         

Wednesday, July 26 at 8:30 a.m.

Thursday, July 27 at 8:30 a.m.

MEDIA:        

Media is encouraged to attend the conference sessions. Please RSVP by clicking below                       or contact Dan Virkstis at (707) 664-2831 or virkstis@sonoma.edu.

Kaiser Permanente Honored With 17 Environmental Excellence Awards

In recognition of its groundbreaking achievement and innovation in health care sustainability, Kaiser Permanente, the nation’s largest integrated health system, has garnered 17 Practice Greenhealth Environmental Excellence Awards, including the designation of the Vacaville Medical Center as one of the top 25 hospitals in the country for environmental excellence.

Practice Greenhealth is the nation’s leading organization dedicated to environmental sustainability in health care. The Environmental Excellence Awards are given each year to honor environmental achievement in the health care sector. Kaiser Permanente has been recognized by Practice Greenhealth for its sustainability work every year since the awards program began in 2002.

Kaiser Permanente’s awards recognize the commitment, embodied in its 2025 Environmental Stewardship Goals, to buying safer products, conserving energy, using solar and wind power, and managing waste that puts the organization among the nation’s leaders in environmental responsibility.

“This recognition underscores the many ways environmental stewardship has become embedded throughout our organization as we work to deliver on our commitment to improve the total health of our employees, members and the communities we serve,” said Kathy Gerwig, Kaiser Permanente’s vice president for employee safety, health and wellness and its environmental stewardship officer. “We are thrilled to be part of the momentum that is building around the benefits of green practices within the health care industry and beyond.”

All of the 13 Kaiser Permanente hospitals honored are in California. The full list of Kaiser Permanente’s Environmental Excellence awards includes:

  • Circle of Excellence for Environmentally Preferable Purchasing: Kaiser Permanente
  • Top 25 Environmental Excellence Award: Kaiser Permanente Vacaville Medical Center
  • Partners for Change Emerald:
    • Kaiser Permanente Riverside Medical Center
    • Kaiser Foundation Health Plan of Colorado
    • Kaiser Permanente Anaheim Medical Center
    • Kaiser Permanente Moreno Valley Medical Center
    • Kaiser Permanente San Diego Medical Center
    • Kaiser Permanente San Jose Medical Center
    • Kaiser Permanente San Rafael Medical Center
  • Partner for Change:
    • Kaiser Foundation Health Plan of Georgia, Inc.
    • Kaiser Foundation Health Plan of the Mid-Atlantic States
    • Kaiser Permanente Fontana Medical Center
    • Kaiser Permanente Oakland Medical Center
    • Kaiser Permanente Richmond Medical Center
    • Kaiser Permanente Santa Clara Medical Center
  • Partner Recognition:
    • Kaiser Permanente Irvine Medical Center
    • Kaiser Permanente Ontario Medical Center

The awards will be presented at the Practice Greenhealth Environmental Excellence Awards Gala, set for May 18 in Minneapolis, at the conclusion of the CleanMed Conference & Exhibition.

About Kaiser Permanente
Kaiser Permanente is committed to helping shape the future of health care. We are recognized as one of America’s leading health care providers and not-for-profit health plans. Founded in 1945, Kaiser Permanente has a mission to provide high-quality, affordable health care services and to improve the health of our members and the communities we serve. We currently serve more than 11.3 million members in eight states and the District of Columbia. Care for members and patients is focused on their total health and guided by their personal physicians, specialists and team of caregivers. Our expert and caring medical teams are empowered and supported by industry-leading technology advances and tools for health promotion, disease prevention, state-of-the-art care delivery and world-class chronic disease management. Kaiser Permanente is dedicated to care innovations, clinical research, health education and the support of community health. For more information, go to: kp.org/share.

BioMarin’s 20th Anniversary

2017 marks BioMarin’s 20th anniversary. This means that not only are they celebrating 20 years as a company, they are also celebrating 20 years in Marin County. For the past two decades, they have been at the forefront of the development of treatments for rare genetic diseases that affect mostly children. During that time, they have brought six products to market, and are gearing up for a potential seventh later this year. In the past 10 years alone, BioMarin has experienced tremendous growth, and now boast over 1,500 employees in Marin County, and 2,500 worldwide.

Their success as a company is due in no small part to their ability to focus on the unmet therapeutic needs of their patients. BioMarin excels at charting new courses to bring a therapy from the lab bench to the patient’s bedside when none existed previously. With this in mind, Forbes has ranked BioMarin among the top 10 most innovative companies in the world for three years in a row.

It’s also excited to share that we have launched a new YouTube channel, which will house videos featuring patient stories and information on BioMarin products. A new short documentary about the history of BioMarin, Finding Tomorrows, is also up on the site. The film provides an inside look at how BioMarin persevered to become one of the world leaders in developing therapies for rare genetic diseases.

Finally, one more piece of exciting news – their public conference space is now open! They have a number of beautiful conference rooms of all sizes available at 750 Lindaro St. in San Rafael. Please let them know if you are interested in hosting a meeting there!

BioMarin wants to give a tremendous thank you to the County of Marin, and specifically the cities of Novato and San Rafael, for their hospitality and collaboration over the past two decades which has enabled so much of their success.

Sonoma Raceway NASCAR Weekend in Sonoma Raises Record $271,000 for Sonoma County Youth Groups

Charitable programs combined to raise record amounts for local youth groups during the Toyota/Save Mart 350 Monster Energy NASCAR Cup Series weekend at Sonoma Raceway, June 23-25.

More than $271,000 was raised during the race weekend, the majority of which will benefit Sonoma County youth organizations through the Sonoma chapter of Speedway Children’s Charities (SCC). The record amount for the NASCAR weekend marks over $55,000 more than 2016. SCC has distributed more than $5.6 million to youth-serving non-profit groups since 2001.

The Children’s Champions Grand Marshal Banquet, held on Friday, June 23, at Cline Cellars in Sonoma, featured comedian and actress Cristela Alonzo, the voice of the “Cars 3” elite trainer Cruz Ramirez. Alonzo served as the Grand Marshal of the Toyota/Save Mart 350.

The sold-out sit-down dinner also featured an exclusive and sentimental history of the “Cars” franchise by Sonoma resident John Lasseter, chief creative officer of Pixar and Walt Disney Animation Studios. Voice-over characters from “Cars 3,” including NASCAR’s Ray Evernham, “The King” Richard Petty, and NASCAR on FOX broadcaster Mike Joy, took part in a Q&A session during the banquet.

A live auction at the banquet featured exclusive items, including a NASCAR Race-Day Experience, which included a meet and greet with the NASCAR on FOX Broadcast team and a behind- the-scenes tour of the iconic Hollywood Hotel. In addition, a special Pixar Studios package included a “Cars”-themed gift basket and a private tour of the Emeryville headquarters, as well as a Ride of a Lifetime with Dale Earnhardt Jr. were auctioned. Overall, the banquet and live auction generated a record-setting $252,000.

In addition, more than $19,000 was raised through various donations during the weekend, including a $5,000 donation on behalf of the Transporter Drivers of Motorsports Association (TDMA), which participated in the 7th annual NASCAR Hauler Parade in Sacramento on June 22. Great Clips raised over $4,000 in donations throughout the weekend at its mobile hair salon in the main paddock and the online charity auction raised nearly $8,000.

For more information about the Sonoma Chapter of Speedway Children’s Charities or to make a donation, visit SonomaRaceway.com/SCC or contact Cheri Plattner at (707) 933-3950 or cplattner@SonomaRaceway.com. If your non-profit group is interested in working with Levy Restaurants at an upcoming event, contact Kim Conte at kconte@Levyrestaurants.com or 707-939-1454.

Kaiser Permanente’s New Residency Program = More Physicians

Kaiser Permanente is furthering its mission to improve community health by
increasing residency programs for primary care physicians—including one at
Santa Rosa Medical Center!

The newly accredited Santa Rosa Family Medicine Residency Program expects to
welcome its first group of six residents into the three-year program in July 2018.

“Training Family Medicine physicians in the Kaiser Permanente integrated model makes
sense,” said Residency Director Patricia Hiserote, MD, a Santa Rosa Family Medicine
physician since 2012. “Our residents will receive an excellent education across the
broad scope of Family Medicine while caring for our community.”

Nationally, 50 percent of graduating residents choose to stay where they train; for
Kaiser Permanente Northern California, it’s closer to 60 percent. Along with training in
the Kaiser Permanente system, Santa Rosa residents will also train at Petaluma Health
Center to learn the community model of medicine.

“We’re investing our time and energy to develop full-spectrum, effective and efficient
Family Medicine physicians who will succeed wherever they choose to practice,” Dr.
Hiserote said. “What’s important is that we increase the number of primary care
physicians in our community. Research shows that the health of an individual, and the
community, improves with the number of primary care physicians. It’s a win-win.”

Santa Rosa Medical Center is already home to Podiatry, Pharmacy, and Optometry
residents, as well as students from medical schools across the country. In addition, we
will continue partnering with Sutter Health’s Santa Rosa Family Medicine Residency
program (which graduates 12 residents per year), while joining the ranks of Kaiser
Permanente Northern California’s 13 other residency programs.

“We are a center of medical education, and our new Family Medicine Residency
Program will continue to make us a major teaching facility,” said Kirk Pappas, MD,
physician-in-chief, Santa Rosa Medical Center.

Judy Coffey, RN, senior vice president and Marin-Sonoma area manager, echoes that
sentiment. “Expanding educational opportunities for physicians and caregivers, and
increasing the number of primary-care physicians is good for everyone in our
community,” she said.

Kaiser Permanente Makes the Marin County Fair Healthy and Fun-for-All

Going to the fair can be a lot of fun. But can it also be healthy?

Marin County Fair has proven that answer to be a resounding YES! The annual event,
slated this year for June 30 –July 4, also proves that big changes can occur in small (yet
significant) steps.

In 2004, “Play Fair Marin” replaced beer as the fair’s title sponsor, evolving into a policy
that eliminates alcohol advertising altogether. And the healthy changes didn’t stop there!

• 2007: The event became the first smoke-free fair in the nation.
• 2008: Food vendors were required to add healthier options to their menus, with
new items becoming so popular, some vendors have rolled them out to other
festivals.
• 2011: A 1k fun run was introduced.

The Play Fair program isn’t just about the health of fair-goers. Environmental well-being
is equally important, with efforts focused on recycling, conservation, valet bike parking,
and even compostable containers.

A Fun, Green, Healthy, Happy, and Award-Winning Event

Marin’s “Greenest Fair on Earth” features attractions such as the first solar-powered
carousel at a county fair, interactive exhibits that showcase sustainable themes, and a
concert stage powered by 99 percent vegetable oil biodiesel.

Even rides at the fair are going green. The Giant Ferris Wheel has been retrofitted with
the latest in LED, as have the Ring of Fire and Super Shot Drop Tower. As a result,
these rides are using 90 percent less power!

“Giving fair-goers healthy choices, eliminating smoking, being careful with advertising
and the sale of alcohol, offering a welcoming baby sanctuary for nursing moms, and
using totally compostable materials all make the fair more family-friendly, and healthier
for our community and the environment,” said Patricia Kendall, medical group
administrator at San Rafael Medical Center. “It makes the fair a gift to all who attend.”

Play Fair Marin now includes more than a dozen community partners and has drawn
accolades and national attention. Working collaboratively, Marin County Fair, Play Fair
Marin, and Healthy Marin Partnership* have received numerous awards from the
Western Fairs Association, including the prestigious Merrill Award. In addition, festivals
across California and in neighboring states have looked to the Marin model for making
their own events healthier.

Kaiser Permanente, Sutter Health, St. Joseph Health and the John Jordan Foundation Work together to create ‘On the Verge’

What does the world need from me?

That’s a question Javier Rivera-Rosales asks himself constantly—and it’s a key reason
why the LGBTQI nonprofit director is participating in Sonoma County’s first On the
Verge cohort.

On the Verge is an intensive year-long leadership program that focuses on emerging
leaders’ personal, interpersonal, and professional skills. Participants receive group and
individual coaching, attend monthly meetings that put developing skills into practice, and
culminate the program with a community-based project.

On the Verge is an offspring of On the Move, an organization that partners with
communities and mobilizes emerging leaders to act in pursuit of social equity.

“Our founders were looking out, looking forward, and asking, ‘What is going to happen
when all the Baby Boomers leave their leadership positions?” explains etsuko kubo, On
the Verge program director. “Will we have people who are trained and who have
personal and professional networks built that will help to serve the social good? “

Last fall, with 18 cohorts already established in California, On the Move brought On the
Verge to Sonoma County. The local cohort includes 15 emerging leaders, primarily from
the mental health field, who are experienced in public and nonprofit sectors. The
dynamic group sparked the idea of combating stigma, access, culture, and language
issues by creating a Latino community center that’s focused on increasing awareness
about, and access to, mental health services.

“Our mental health focus relates to shifting paradigms, because in the Latino culture,
there are layers upon layers of stigma,” Javier says. “Mental health practices are not
one size fits all; we need to be able to change with the times and the needs of the
community – breaking down walls and stigmas.”

Javier and his On the Verge teammates are brimming with energy and enthusiasm for
their vision of opening a vibrant and welcoming community center that combines
Western and traditional Latino healing practices. A second cohort, which begins this fall,
will carry out the inaugural vision, with support from the founding participants.

In Sonoma County, On the Verge is supported by Community Foundation Sonoma
County in collaboration with the John Jordan Foundation, Kaiser Permanente, St.
Joseph Health, and Sutter Health.

Sonoma State University to Host Academy of Wine Business Research Conference and Education Summit

The Wine Business Institute (WBI) at Sonoma State University (SSU) today announced that it will host the 10th annual Academy of Wine Business Research (AWBR) conference from July 25 – 28, with program activities taking place on its Rohnert Park campus and throughout Sonoma and Napa counties. AWBR is an international society of academics and scholars with research focus on economic, social, and management issues related to the global wine industry. Confirmed attendees represent academic institutions in Austria, Australia, Brazil, Chile, China, France, Germany, Italy, New Zealand, Spain, and the United States. WBI has been involved with the annual conference since its founding in 2003, and previously hosted the gathering in 2005.

The 2017 Wine Business Education Summit, designed as a prelude to this year’s conference, will assess industry needs related to education, hiring, and employee retention. The half-day Summit features executive directors from California wine and grower associations, human resources executives from the wine industry, and executives from local staffing companies.

A full conference schedule may be viewed here: http://web.sonoma.edu/sbe/awbr-conference-2017.html

“We’re pleased that the international Academy wants to bring its annual conference back to the North Coast and to Sonoma State University. It’s a statement of appreciation for California’s importance to the global wine industry, and recognition of the Wine Business Institute’s leadership role in research and education,” Ray Johnson, Executive Director of the Wine Business Institute said. “The wine industry continues to change here and abroad, and we expect both the education summit and conference sessions will offer valuable exchanges, discoveries, and powerful takeaways for industry professionals and academics alike.” 

“We look forward to welcoming our wine business academic colleagues from around the world, to share and discuss recent research on issues of importance to the global wine industry, including how to build profitable and responsible wine businesses,” Dr. Liz Thach, MW, Distinguished Professor of Wine said.

Conference sessions include paper presentations under the headings: “Online Purchases of Wine”; “Managing Uncertainty in the Wine Industry”; “Measuring Market Performance”; “Understanding Wine Purchasing Behavior”; “Supply Chain and Exporting Issues”; “Industry Perspectives on Sustainability”; “Managerial Approaches”; “Sustainability and its Link with Customers”; “Wine Ratings and Raters”; “Wine Industry Trends”; “The Role of Tradition in the Wine Industry”; “Case Study Workshop”; “A Better Understanding of Wine Consumers”; “Marketing of Wine Terroir and Territories”; “Wine Marketing in a Digital Age”; “Wine Tourism”; “Marketing and the Wine Purchase Decision”; “Management Strategy”; “Wine Sales and Trends”; “Wine Marketing in Eastern and Southern Europe”; and “Wine Industry Development and Growth.”

Round table discussions include, “Wine and Intellectual Property, Implications for Innovation, Legislation, Marketing and Branding”; “Defining and Measuring Performance in the Wine Sector, The Sustainability Factor”; “Wine Tourism Experience, the Role of Heritage and Terroir, and the Differences Between “Old World” and “New World” Wine Regions”; and “How California’s Universities and Colleges are Providing the Optimal Workforce to the Local Industry.”

The 2017 Wine Business Education Summit will begin with a morning session facilitated by Ray Johnson, WBI Executive Director, including small and large group discussions to inform workplace issues and future wine business programming at the Wine Spectator Learning Center and globally. The program also includes a Wine Industry Colloquium featuring a panel discussion on the topic, “Wine Business Education: Where Do We Go from Here?”. Moderator Anisya Fritz, proprietor of Lynmar Estate, will facilitate an exploration with panelists Amelia Ceja, President at Ceja Vineyards; Luke Jencks, President at NakedWines.com; Michael Moone, past President at Beringer Wine Estates; and John Williams, President at Frog’s Leap Winery.

For more information regarding the 10th annual Academy of Wine Business Research conference, please contact the Conference Administrative Coordinator, Christine MacMillan, at cmcmillandestinations@gmail.com. For information regarding the 2017 Wine Business Education Summit, please contact Ray Johnson, WBI Executive Director, at ray.johnson@sonoma.edu or (707) 664-3071. For information regarding wine business research, programs, and degrees in the School of Business and Economics at SSU, please call (707) 664-3235, email winebiz@sonoma.edu, or visit www.sonoma.edu/sbe.

Midstate Construction Completes Canyon Ridge at Napa Junction

Canyon Ridge Apartments at Napa Junction

Midstate Construction Corporation recently constructed a new luxury apartment complex in American Canyon, CA.

This project included new construction of 148 apartments in seven, three-story, wood framed buildings with an eighth building that houses the community center.

Project highlights:

  • Community Center
  • Saltwater Pool
  • Hot Tub
  • Tot Lot
  • Fitness Room
  • Individual Garages
  • Bocce Ball Court
  • Indoor Dog Grooming Station
  • Water Reclamation System for Toilet and Site Irrigation

Canyon Ridge received North Bay Business Journal’s 2016 Top Project Award

BioMarin Announces The Approval and Launch of Brineura™

BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced that the U.S. Food and Drug Administration (FDA) approved Brineura™ (cerliponase alfa) to slow the loss of ambulation in symptomatic pediatric patients 3 years of age and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency. Brineura is the first treatment approved to treat children with CLN2 disease, a form of Batten disease.

CLN2 disease is an ultra-rare, rapidly progressive fatal brain condition, which affects less than one in one million U.S. residents, many of whom are undiagnosed.  Every year approximately 20 children are born in the U.S. with CLN2 disease.  These affected children completely lose the ability to walk and talk around 6 years of age.  During the later stages of the disease, feeding and tending to everyday needs become very difficult with death often occurring between 8 and 12 years of age.

In clinical trials, Brineura, an enzyme replacement therapy, was shown to slow the loss of ambulation in symptomatic pediatric patients 3 years of age and older with CLN2 disease. It is the first enzyme replacement therapy to be directly administered to the brain, treating the underlying cause of the condition by replacing the deficient TPP1 enzyme. Using an established technique most often used in oncology – intraventricular administration– the therapy is delivered directly into fluid surrounding the brain, known as the cerebrospinal fluid.

To support the community of families with children with neurologic disorders, BioMarin in partnership with a commercial lab is offering a no cost genetic testing program called ‘Behind the Seizure’ to support early testing for children who experience seizures.  In addition, BioMarin is investing in tools and resources to educate physicians on CLN2 disease in order to help them diagnose patients with this disease earlier and prevent them from being misdiagnosed during critical years when therapy could help slow the loss of ambulation.

In addition, BioMarin RareConnections™, a resource available to patients and families, provides a variety of personalized support services at no cost to patients, including education on CLN2 disease and Brineura, and coordination of additional services, such as information about financial assistance programs.

“We thank the FDA for recognizing Brineura’s potential to alter the course of CLN2 disease and its urgency in delivering this treatment to children as quickly and safely as possible. Brineura was approved in under four years from starting the first clinical trial to today, a significant achievement for a condition that progresses so rapidly,” said Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin. “Treating children with CLN2 disease requires an extraordinary amount of collaboration between families, hospitals, advocates and physicians. We are grateful for the partnership of all those involved and look forward to continuing to work together to make Brineura accessible to children who may benefit.”

“CNL2 is a devastating diagnosis that robs families of life with their children much too young,” said Emily de Los Reyes, M.D., attending pediatric neurologist at Nationwide Children’s Hospital and principal investigator for Brineura studies. “Today’s announcement gives my patients and their families hope.”

“The approval of Brineura is an extraordinary medical breakthrough for the CLN2 Batten community who have been waiting for this moment for more than a century when the condition was first described,” said Margie Frazier, PhD, LISW-S, Executive Director of Batten Disease Support and Research Association. “We appreciate BioMarin’s commitment and partnership to the CLN2 Batten community and investing the resources needed to bring this pivotal treatment to families.”

With this approval, the FDA also issued a Rare Pediatric Disease Priority Review Voucher, which confers priority review to a subsequent drug application that would not otherwise qualify for priority review. The rare pediatric disease review voucher program is designed to encourage development of new drugs and biologics for the prevention or treatment of rare pediatric diseases.

Brineura is expected to be available in the United States by early June, and BioMarin will begin promotion of Brineura immediately.

Last week, the Committee for Medicinal Products for Human Use (CHMP), the scientific committee of the European Medicines Agency (EMA), adopted a positive opinion for the company’s Marketing Authorization Application (MAA) for Brineura to treat children with Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) disease, a form of Batten disease, which is also known as tripeptidyl peptidase 1 (TPP1) deficiency.  The CHMP’s recommendation is now referred to the European Commission (EC), which is expected to render its final decision by the second quarter of 2017.

Clinical Trial Results

The approval was supported by safety and efficacy data assessed over 96 weeks in a non-randomized, single-arm dose escalation clinical study of patients with CLN2 disease. Brineura treated patients were compared to untreated patients from a natural history cohort.

Patients were assessed for decline in the motor domain of the CLN2 Clinical Rating Scale. The scale measures performance of mobility with normal function being a score of 3 and no function being a score of 0.  Decline was defined as having a sustained 2-point decline or an unreversed score of 0 in the motor domain of the CLN2 Clinical Rating Scale.

Twenty-four patients aged 3-8 years were enrolled in the clinical study. One patient withdrew after week 1 due to inability to continue with study procedures; 23 patients were treated with Brineura every other week for 48 weeks and continued treatment during the extension.

Results from the 96-week analysis demonstrated the odds of Brineura-treated patients not having a decline were 13 times the odds of natural history cohort patients not having a decline (Odds Ratio (95% Confidence Interval): 13.1 (1.2, 146.9)).

Of the 22 patients treated with Brineura and evaluated for efficacy at week 96, 21 (95%) did not decline, and only the patient who terminated early was deemed to have a decline in the motor domain of the CLN2 Clinical Rating Scale. In comparison, 50% of patients in an independent natural history cohort demonstrated progressive decline in motor function.  Two Brineura treated patients with a maximum score were excluded from the analyses; they maintained that score throughout the study period.

In the clinical study, intraventricular access device-related infections were observed in two patients. In each case, antibiotics were administered, the intraventricular access device was replaced and the patient continued on Brineura treatment.  Hypotension was reported in 2 (8%) patients, which occurred during or up to 8 hours after Brineura infusion. Patients did not require alteration in treatment and reactions resolved spontaneously or after IV fluid administration.

Hypersensitivity reactions have been reported in 11 (46%) Brineura treated patients during the clinical studies.  The most common adverse reactions (≥8%) are pyrexia, ECG abnormalities, decreased cerebrospinal fluid (CSF) protein, vomiting, seizures, hypersensitivity, increased CSF protein, hematoma, headache, irritability, pleocytosis, device-related infection, bradycardia, feeling jittery, and hypotension.

About CLN2 Disease

Children with CLN2 disease typically begin experiencing seizures between the ages of 2 and 4 years old, preceded in the majority of cases by language development delay.  The disease progresses rapidly with most affected children losing the ability to walk and talk by approximately 6 years of age.  Initial symptoms are followed by movement disorders, motor deterioration, dementia, blindness, and death usually occurring between the ages of 8 and 12 years of age.  During the later stages of the disease, feeding and tending to everyday needs become very difficult. BioMarin estimates the incidence of CLN2 disease is approximately one in 200,000 with up to 1,200 to 1,600 children in the regions of the world where BioMarin operates, many of whom are undiagnosed.

The neuronal ceroid lipofuscinoses (NCLs) are a heterogeneous group of lysosomal storage disorders that includes the autosomal recessive neurodegenerative disorder CLN2 disease. CLN2 disease is caused by mutations in the TPP1 gene resulting in deficient activity of the enzyme tripeptidyl peptidase 1 (TPP1). In the absence of TPP1, lysosomal storage materials normally metabolized by this enzyme accumulate in many organs, particularly in the brain and retina. Buildup of these storage materials in the cells of the nervous system contribute to the progressive and relentless neurodegeneration which manifests as loss of cognitive, motor, and visual functions.