The Newsletter - Life Sciences Pennsylvania

Q4 2017

THE NEWSLETTER L I F E S C I E N C E S P A’ S Q U A R T E R L Y N E W S S O U R C E

C E O D I N N E R - N OV E M B E R 2 8 - A R D M O R E , PA

Q4 HIGHLIGHT: Nearly 100 people, including 23 emerging companies and 10 life sciences industry “all stars,” gathered November 14 at LSPA’s office in Wayne, PA, for the first-ever StartUp Stadium – Advice from the All Stars. The Start-Up Stadium was designed to help new entrepreneurs learn the strategies and techniques used by experienced, successful entrepreneurs. This event was an

IN THIS ISSUE F e a t u r e d C o n t e n t.. . . . . . . 1 Events. . . . . . . . . . . . . . . . . . . . 2

educational opportunity for the start-up community to receive direct feedback on their presentations while also showcasing the most promising and innovative emerging companies in the state. The day began with a networking lunch, followed by two discussion groups for attendees to choose from: “How to Make Your Pitch” and “JP Morgan Prep.” “How to Make Your Pitch” was led by Nick Pachuda of Johnson & Johnson, Bob Smith of Pfizer, and Steve Bowman of BizClarity; and moderated by Greg Seltzer of Ballard Spahr. The JP Morgan discussion was led by Erin Brubaker of GlaxoSmithKline, Chris Cashman

“. ..appreciation and thanks for our participation in the Start-Up Stadium. We found this to be a great learning experience for our team and have already included the feedback and learnings into our business development efforts.” – S tephen Manobianco, Abzyme Therapeutics LLC of Marinus Pharmaceuticals, and Bruce Peacock of CARMA Therapeutics; and moderated by LSPA president and CEO, Chris Molineaux. The goal of this discussion was to help companies prepare for the JP Morgan Healthcare

M e m b e r s h i p .. . . . . . . . . . . . 4 President ’s Column...... 7 F i n a n c i a l B u l l e t i n .. . . . . .. 10 A d v o c a c y . . . . . . . . . . . . . . . 12 S u p p o r t e d E v e n t s . . . . . . 13 N e w M e m b e r s Q 4.. . . . . 14 S TA R T - U P S TA D I U M R E C A P C O N T I N U E D O N PAG E 11

L S PA Te a m . . . . . . . . . . . . . . 15

1 | T H E L S PA N E W S L E T T E R , Q 4 2 017

EVENTS L I F E S C I E N C E S PA H O S T S C H I E F E X E C U T I V E O F F I C E R S F O R D I N N E R AT M E R I O N G O L F C L U B F E AT U R I N G M A D E L I N E B E L L , P R E S I D E N T & C E O O F C H I L D R E N ’ S H O S P I TA L O F PHIL ADELPHIA (CHOP)

Life Sciences Pennsylvania held its 2017 Chief Executive Officer Dinner on November 28 at the Merion Golf Club in Ardmore, PA. The relaxing evening provided an opportunity to meet with new colleagues and network with other members. The event featured a keynote discussion by Madeline Bell, president & CEO of Children’s Hospital of Philadelphia (CHOP). She spoke about upcoming technologies at CHOP, approaches to disseminating their science, the importance of strategic partnerships to CHOP’s future, and the ecosystem at CHOP that allows them to grow in the life sciences market.

LSPA SUMMIT SERIES IN Q4 Supply Chain Summit October 24, 2017 The Desmond Hotel Malvern Malvern, PA Start-Up Stadium – Advice from the All Stars November 14, 2017 Life Sciences Pennsylvania UPCOMING EVENTS To learn about upcoming events in 2018, visit http://www.lifesciencespa. org/connect/events/ 2 | W W W. L I F E S C I E N C E S PA . O R G

Wayne, PA CRO/CMO Summit November 17, 2017 West Pharmaceuticals Services, Inc. Exton, PA

EVENTS

L I F E S C I E N C E S PA H O S T S C H I E F FINANCIAL OFFICERS FOR D I N N E R AT S T. DAV I D S FEATURING GUEST SPEAKER DR. KETAN PATEL FROM F -PRIME CAPITAL PARTNERS

St. Davids Golf Club in Wayne, PA was the setting for the 2017 Chief Financial Officer Dinner hosted by Life Sciences Pennsylvania on November 7. This exclusive event, offered to those serving in the capacity of chief financial officer, chief executive officer, and/or VP of finance, featured a night of networking, meeting new colleagues, and a discussion with guest speaker, Ketan Patel, M.D., Partner, F-Prime Capital Partners. Dr. Patel discussed recent investment and growth within the life sciences industry, the investment climate in today’s industry, and the nuances in investment culture between the Boston, San Francisco and Philadelphia regions.

L I F E S C I E N C E S PA 2 017 H O L I DAY M I X E R A FESTIVE GATHERING TO MEET THE BOARD OF DIRECTORS & NEW MEMBERS

In December, Life Sciences Pennsylvania hosted its annual Holiday Mixer at its headquarters in Wayne, PA. The mixer was an opportunity to bring the community together one last time before the holidays. The informal reception gave LSPA members a chance to connect over cocktails and hors d’oeuvres, and meet the Board of Directors and LSPA’s newest members.


MEMBERSHIP B R A N DY W I N E A N D D R E X E L B R E A K G R O U N D O N S C H U Y L K I L L YA R D S B R A N DY W I N E R E A LT Y T R U S T A N D D R E X E L U N I V E R S I T Y L AY F O U N DAT I O N F O R $ 3 . 5 B I L L I O N P R O J E C T

When two Life Sciences Pennsylvania members team up, anything is possible. Brandywine Realty Trust, in partnership with Drexel University, broke ground on phase one construction of what’s officially called the Schuylkill Yards – a $3.5 billion, 14-acre plan in University City, Philadelphia. The multi-year project will enhance the West Philadelphia community, with developments including Drexel Square, a 1.3-acre community park designed by SHoP Architects and West 8 Landscape Architects. The park will be developed across the street from Philadelphia travel hub, 30th Street Station. John Fry, president of Drexel University, praised Drexel Square as “the long-awaited sixth square of our great city,” a nod to other iconic

full-block parks such as Rittenhouse Square and Washington Square.

on November 8, attended by local politicians including Mayor Jim Kenney.

The partnership between Brandywine and Drexel will help create a forwardthinking, sustainable Philadelphia neighborhood. Additional projects in the masterplan, expected to be executed within the next 15 to 20 years, call for 6.5 acres of green space and better-quality streetscapes, 7 million square feet of developed space, and the execution of a fully mixed-use project to include professional offices/labs, as well as residential properties and retailers.

Brandywine Realty Trust is also investing $500,000 to seed the Grow Philadelphia Capital Initiative, a project aiming to help fund small businesses in the city.

“Schuylkill Yards will be a defining development that becomes the model for urban America,” said Fry at the groundbreaking ceremony

“As Brandywine continues to expand West Philadelphia, we do so with a forward-thinking, inclusive approach to the future. We embrace the changing habits of how people are living, creating, working and spending their time. We are proud that our first project in Schuylkill Yards will deliver a green public gathering space where the community can connect, interact and share experiences,” said Jerry Sweeney, president and CEO of Brandywine Realty Trust.

PITTSBURGH LIFE SCIENCES GREENHOUSE AWA R D E D G R A N T F O R H E A LT H I N F O R M AT I O N T E C H N O LO G Y P R O G R A M S T H E $ 5 0 0 K G R A N T W I L L S U P P O R T M A J O R D E V E LO P M E N T S I N P I T T S B U R G H L I F E S C I E N C E S H E A LT H I N F O R M AT I O N T E C H N O LO GY P R O G R A M S

Health information technology (HIT) has become a productive, high-grossing area that offers the opportunity for new jobs. Many healthcare providers are turning to HIT as a way to provide better health outcomes, at lower costs and with greater efficiencies.

Challenge is hosted by the U.S. Economic Development Administration (EDA) through their Regional Innovation Strategies (RIS) program, which supports innovation and entrepreneurship that can be translated into products, services, and jobs.

Pittsburgh Life Sciences Greenhouse (PLSG) earned a $500,000 “i6 Challenge” grant from the U.S. Department of Commerce to create and expand their own HIT programs from proof-of-concept through commercialization, paving the way for expansive job creation. The i6

The grant will help fund PLSG’s analysis of where health information technology is of primary need, as well as entrepreneur training and education, assessment of new technological opportunities, and workforce development. The funding will further allow PLSG to gain a

4 | W W W. L I F E S C I E N C E S PA . O R G

better understanding of the region’s strengths and weaknesses, allowing for new strategies for development in a growing life sciences field. “We see this as the first step in making the region a hotbed for HIT,” said Jim Jordan, president and CEO of PLSG. “Maximizing our knowledge regarding HIT promises to create jobs, increase commercialization of products and services, and further the development of Pittsburgh, and the region, as a national leader in the life sciences.”

MEMBERSHIP

A R B U T U S B I O P H A R M A C O R P O R AT I O N & G R I T S T O N E O N C O LO G Y A N N O U N C E C O L L A B O R AT I O N I N IMMUN OTHERAPY COMPANIES SIGN LICENSING DEAL FOR PERSONALIZED CANCER IMMUNOTHERAPIES

Arbutus BioPharma Corporation, a Bucks County-based company developing hepatitis B treatments, entered into a collaborative licensing agreement with Gritstone Oncology, a California-based biopharmaceutical company leading personalized cancer immunotherapies. The licensing deal provides Gritstone Oncology with access to Arbutus’s lipid nanoparticle products and associated intellectual property. The lipid nanoparticle platform has the potential to make a big impact on therapy development for the treatment of hepatitis B infection and for immunotherapies to enhance the body’s ability to recognize and destroy tumors.

“This agreement represents validation of our industry-leading LNP technology. We are excited to be working with Gritstone to advance their innovative personalized immunotherapies for the treatment of cancer,” said Dr. Mark J. Murray, Arbutus’s president and chief executive officer. “This transaction is part of realizing the value of our LNP platform, which has broad potential to deliver nucleic acid-based products including mRNA and gene-editing therapeutics, and enable Arbutus to continue to focus on development of curative therapies for HBV.”

On October 18th, Arbutus held a ribbon cutting for the grand opening of its new research facility in Warminster, Bucks County. This new, 35,000 squarefoot facility will house more than 40 employees.

Gritstone will pay Arbutus for achievements in development, as well as milestones and fees. Additionally, Gritstone will reimburse Arbutus for development and providing manufacturing support for their product candidates.

I N OV I O P H A R M AC E U T I C A L S , W I S TA R I N S T I T U T E , THE UNIVERSIT Y OF PENN, AND GENEONE LIFE SCIENCES TA K E O N Z I K A P R E V E N T I O N F I R S T H U M A N T R I A L S H OW S P R O G R E S S TOWA R D P R E V E N T I N G Z I K A I N F E C T I O N S I N A D U LT S

A blockbuster partnership is giving new hope to an infectious virus. A new generation DNA-based Zika vaccine candidate, called GLS-5700, is the first to demonstrate both safety and the ability to elicit an immune response against Zika in humans, according to new research published in the New England Journal of Medicine in October 2017. The vaccine was developed by the Wistar Institute, and their early-stage study was conducted in partnership with the Perelman School of Medicine at the University of Pennsylvania, Inovio Pharmaceuticals, Inc., and GeneOne Life Science, Inc.

“Synthetic DNA vaccines are an ideal approach for emerging infectious diseases like Zika,” said David B. Weiner, director of Wistar’s Vaccine and Immunology Center. “This new generation of DNA vaccines can be designed and manufactured rapidly, they appear to be highly predictable for the generation of immunity in humans, have significant conceptual safety advantages, and they are more stable than most traditional vaccines, making them exceptionally practical to distribute during outbreaks – especially in regions where resources are limited and we need to be able to quickly respond to curb an emerging epidemic.”

S H A R E YO U R U P DAT E S Want to be featured here? Forward news articles, event recaps, or announcements to [email protected] to be considered for our next issue.

“This new generation of DNA vaccines can be designed and manufactured rapidly, they appear to be highly predictable for the generation of immunity in humans, have significant conceptual safety advantages, and they are more stable than most traditional vaccines, making them exceptionally practical to distribute during outbreaks.” – David B. Weiner, Wistar Inovio is attempting to commercialize the vaccine as a treatment to help fight against multiple strands of the Zika virus. The early study shows great promise for synthetic DNA vaccines in the future of vaccine development.


MEMBERSHIP

C I C PA R T N E R S W I T H U N I V E R S I T Y C I T Y S C I E N C E CENTER & VENTURE CAFÉ IN PHIL ADELPHIA I N N OVAT I O N C E N T E R UCIT Y SQUARE WILL HOUSE THE SCIENCE CENTER’S HEADQUARTERS AND THE FIRST PHIL ADELPHIA LOCATION FOR CIC

Update your directories – in 2018, uCity Square, or 3675 Market Street, will become the new location for the Science Center headquarters, expanded laboratories from Quorum and FirstHand, and the Philadelphia location for CIC, which will manage the building in partnership with the University City Science Center and Venture Café Global Institute. In a reception on November 29, the partners announced more details about their collaborative efforts to catalyze and accelerate innovation and entrepreneurship in the Greater Philadelphia area.

The University City Science Center will join the Venture Café Network, a global network founded by CIC, expanding its Quorum program through partnerships in the U.S. and the Netherlands. The Science Center and Venture Café will also offer programming beginning in Fall 2018, when the building is expected to open.

“We’ve been working toward establishing CIC Philly for several years and, thanks to the partnership and guidance of Steve Tang and the Science Center, it’s a thrill to see it all coming together,” says Dougan Sherwood, CIC managing director. Adds Science Center president & CEO Dr. Stephen S. Tang, “We’re taking business incubation to the next level as we shift our focus away from operating physical spaces for startups and reinvest our efforts into the people and ideas that make up those companies – the innovators and innovations who are changing our collective future.”

LSPA 2017 QUIC K STATS

32

events hosted by LSPA

755

total members

135

new members

Both totals indicate a record for LSPA – and the highest number of Diamond Members we’ve ever had!


S PA R K T H E R A P E U T I C S , I N C . R E C E I V E S L A N D M A R K F DA A P P R OVA L F O R N OV E L G E N E T H E R A P Y LU X T U R N A I S A P P R OV E D F O R R A R E F O R M O F I N H E R I T E D V I S I O N LO S S

LSPA extends our congratulations to Spark Therapeutics! On December 19, 2017, the Food and Drug Administration (FDA) approved the first product from the Philadelphia-based biotech company. Luxturna marks the third gene therapy approved by the FDA, but is the first directly-administered gene therapy approved in the U.S. that targets a disease caused by mutations in a specific gene. It treats children and adult patients with Leber congenital amaurosis, a rare inherited form of retinal disease that may result in blindness. Luxturna is approved for patients with a mutated RPE65 gene – the one responsible for developing an enzyme needed for normal, healthy vision. The therapy uses a modified virus to deliver a healthy copy of the gene, administered directly via surgery to the patient’s retinal cells. “Today’s approval marks another first in the field of gene therapy — both in how the therapy works and in expanding the use of gene therapy beyond the treatment of cancer to the treatment of vision loss — and this milestone reinforces the potential of this breakthrough approach in treating a wide range of challenging diseases,” said FDA Commissioner Scott Gottlieb, M.D. “The culmination of decades of research has resulted in three gene therapy approvals this year for patients with serious and rare diseases. I believe gene therapy will become a mainstay in treating, and maybe curing, many of our most devastating and intractable illnesses.”

PRESIDENT’S COLUMN C H R I S T O P H E R P. MOLINEAUX

than ever before on facilitating strategic connections between and among entrepreneurs and the resources needed to enable life sciences growth and success.

PRESIDENT & CEO

ADVOCACY REMAINS CRUCIAL

L I F E S C I E N C E S P E N N S Y LV A N I A

As the life sciences industry continues to change and we, as your trade association, evolve to match the ever-shifting landscape and our membership, we keep our eyes on the legislative “ball.”

While collecting my thoughts for this President’s Letter, I was reminded of the evolution of the life sciences presence across Pennsylvania, the dynamism of our membership, and the unprecedented opportunity and awesome responsibility that face the science and business leaders of our community.

2017 ushered in new players at the federal and state levels, creating new opportunities to advance the life sciences industry policy agenda.

We released a report in September that showed the life sciences industry in Pennsylvania is in the process of reinventing itself. The industry has historically been a great source of jobs and a strong driver of Pennsylvania’s economy, but we all have witnessed the painful downsizing and contraction of large pharma over the last decade. What many have missed, however, is a quiet but steady re-birth of the life sciences and the formation of hundreds of new companies. During the period between 2011 and 2016, Pennsylvania saw modest growth in life sciences employment – a net increase of approximately 1,000 jobs – and a 4.4% increase in the number of life sciences establishments founded, in many cases, by former academics and pharma and device company executives. This speaks to the entrepreneurial bent of our community…and a strong desire by our scientists and business leaders to stay in Pennsylvania. This growth is counterintuitive to conventional wisdom about the life sciences in Pennsylvania and is certainly encouraging for the future. However, this growth comes with a caution: more than half of the 2,800 life sciences establishments in Pennsylvania today employ fewer than 10 employees. It is a fast-growing, optimistic, entrepreneurial community which is always thrilling. But it is also a fragile community that requires nurturing.

2017 ushered in new players at the federal and state levels, creating new opportunities to advance the life sciences industry policy agenda. We are fortunate to have a supportive Congressional delegation in Washington, DC and at year-end saw tax reform that dramatically lowered the corporate tax to a more globally competitive 21%. However, this good news was accompanied by a reduction in the Orphan Drug Tax Credit that is so vital to companies pursuing treatments and cures for patients with rare diseases. Moreover, efforts to repeal the Medical Device Tax remained stymied despite multiple standalone bills with broad bi-partisan support. Also of note: four of Pennsylvania’s 18 members of Congress have announced they will not be seeking re-election this year. Accordingly, we must remain vigilant with our advocacy activity.

At Life Sciences PA, we recognize the nature of our new constituency: small, hungry, high-risk enterprises, and we continue to refine our services to meet the needs of this new model of membership. At Life Sciences PA, we recognize the nature of our new constituency: small, hungry, high-risk enterprises, and we continue to refine our services to meet the needs of this new model of membership. In June, we opened the new Life Sciences Center, @LSPA, to provide open desks, huddle rooms, event space and program services unlike any other trade association. We created this “clubhouse” specifically for our start-up and emerging company constituents – it’s a place where anyone in our membership should feel at home and, hopefully, connect with the resources and contacts needed to grow your businesses. In 2018 – at the direction of our Board of Directors – our programs, events and services will have a tighter focus

In the Statehouse, the discussion over drug pricing continues with insurance industry-backed politicians calling for unprecedented disclosure of proprietary business strategies by biopharmaceutical companies. These appear to be thinlyveiled attempts to shift attention away from insurance industry rate hikes which have reached 35-40% in some instances… and we and our allies see right through them. We know pharmaceutical costs are approximately 10% of the healthcare dollar – a percentage that has not changed since 1960 – and with the involvement of our membership, we have been successful the last two years in keeping such poorly-conceived policy proposals from gaining traction, but the pressure continues to build and our educational efforts must endure. Of course, any conversation about state policy, politics and the biopharma industry must include mention of the opioid abuse epidemic, a human tragedy whose effect has been felt by thousands of Pennsylvanians. We continue to educate policy makers and those in law enforcement on efforts by industry to address this tragedy and encourage a science-based and researchdriven discussion about solutions – all the while protecting patients’ appropriate access to these important products. OUR INTERNATIONAL PROFILE CONTINUES TO GROW In the past year, we signed two more Memorandum of Understanding agreements with international organizations. In January, I was pleased to participate in a U.S. delegation P R E S I D E N T ’ S C O LU M N C O N T I N U E D O N PAG E 8


P R E S I D E N T ’ S C O LU M N C O N T I N U E D F RO M PAG E 7

to India. While there, I agreed to a new relationship with the Indo-U.S. Science & Technology Forum. In early December, I met with a delegation from the Lithuanian Biotechnology Association in Philadelphia where a similar agreement was signed. We now have six agreements spanning the globe and we can better assist our members with a warm handoff to our international partners as we work to promote Pennsylvania as a destination for companies as well. LOOKING FORWARD In the face of these challenges, we have reason to remain optimistic about the future of life sciences ingenuity in Pennsylvania and the promise it holds for patient care.

Ours is an industry that has begun to revolutionize the treatment of patients – a community of scientific and business prowess that is delivering potential cures. Ours is an industry that has begun to revolutionize the treatment of patients – a community of scientific and business prowess that is delivering potential cures. 2017 brought the first two FDA-approved cell and gene therapies…from scientists in Pennsylvania. As described in the Wall Street

FACEBOOK.COM/ LIFESCIENCESPENNSYLVANIA

Journal, “Researchers have been pursuing gene therapy for decades in hopes it can yield treatments and cures for genetic conditions that have no other treatments…” This revolution in treatment began 30 years ago in Pennsylvania and, according to Dr. Kathy High of Spark Therapeutics, “It really provides a way forward for a whole range of rare conditions that have not been previously treated.” Indeed, our community continues to persevere and will prevail…with patience, for patients. And we at Life Sciences PA are honored and humbled to support your efforts. To conclude, I offer thanks to our robust and growing membership – 755 organizations as of year-end 2017, spanning pharma, biotechnology, medical device and diagnostic companies, digital health companies, the academic research community, patient advocacy groups, investors, and the entire service community – the industry around the industry – without whom our community could not lead the world. I also extend special thanks to our outstanding Board of Directors for their continued support and guidance. On behalf of the entire team at Life Sciences Pennsylvania, we wish you all continued success in 2018 as, together, we pursue our goal to make Pennsylvania the most attractive place to open and operate a life sciences company.

@LIFESCIENCESPA

@LIFESCIENCESPA

LIFE SCIENCES PENNSYLVANIA

Where Ideas Go To Work

MANIFESTING INNOVATION The community of innovators at the University of Pennsylvania is bound together by a common goal: serving society through the advancement of knowledge. Pennovation Works is the place where researchers and students, entrepreneurs, and the private sector thrive together with the benefit of diverse, flexible, and affordable spaces. This creates a dynamic environment where ideas go to work.


ANCHORING THE WORKS: PENNOVATION CENTER Marked by a striking façade, the Pennovation Center is an icon for the Works as well as for the university and the city. Within the walls of this business and technology incubator, innovators can connect and collide, facilitating productivity and translating exciting new discoveries into products, ventures, and services for real world application.

Penn is now leasing offices, labs and land on this 23 acre site adjacent to its campus and medical district. To learn more, visit: www.pennovation.upenn.edu.

Pennovation Works 3401 Grays Ferry Avenue Philadelphia, PA 19146

Where Ideas Converge.

7M SF

of Opportunity

1M SF Lab

2.8M SF Office

SchuylkillYards.com Philadelphia, PA Accelerate your life science venture here.

987K SF

Innovation Space

215.397.1589 www.brandywinerealty.com

20171019_SY ad.indd 1

10/20/2017 12:33:34 PM


FINANCIAL BULLETIN Q4 We think it is important to highlight both the scientific innovation found in our member companies and also their successes on the financial front. Below you will find a list of the IPOs and funding rounds completed in the 4th Quarter of 2017. • Harmony Biosciences, LLC announced it has raised $270 million and acquired exclusive U.S. rights to develop and market pitolisant, a narcolepsy drug from French company Bioproject. – October 5, 2017 • Strongbridge BioPharma PLC raised $23.4 million from a public stock sale, selling four million shares of common stock at $6.24 per share. Proceeds will support development and expansion of several experimental and approved treatments. – October 6, 2017 • Aevi Genomic Medicine, Inc. announced a $28 million private stock placement led by Children’s Hospital of Philadelphia, selling 22.2 million shares of common stock. The CHOP Foundation announced that it will provide an additional $5 million in equity financing through June 2018. – October 17, 2017 • Optinose, Inc. closed its $138 million stock offering, selling more than 8.6 million shares of its common stock at $16 per share. Money raised will support launch and development efforts for Xhance for treatment of nasal polyps in adults. – October 17, 2017 • Genisphere LLC raised $7 million to advance its 3DNA nanotechnology platform designed to improve targeted drug delivery. – November 1, 2017 • Onconova Therapeutics, Inc. sold about 920,000 shares of common stock at a price of $1.50 per share, raising $1.4 million to focus on late-stage clinical testing of lead drug candidate, IV Rigosertib, for myelodysplastic syndromes. – November 14, 2017 • Recro Pharma, Inc. announced a $100 million financing deal managed by Athyrium Capital Management. Part of the funds will support a milestone for IV meloxicam for moderate-to-severe pain. – November 20, 2017 • Madrigal Pharmaceuticals, Inc. announced it was selling 1.5 million shares of common stock at $83 per share, and seeking to raise $125 million to fund corporate projects such as research and development, clinical trials, manufacturing and supply, and new technologies. – December 18, 2017

T E L L U S W H AT YO U T H I N K Do you like reading the quarterly newsletters? Anything we can do to improve? Share your feedback with us by emailing [email protected]. Thank you in advance for your input. 10 | W W W. L I F E S C I E N C E S PA . O R G

S TA R T - U P S TA D I U M R E C A P C O N T I N U E D F RO M PAG E 1

Conference, navigate the event while there, and determine if and when they should participate in the future. After the discussions, attendees moved into private meetings, company presentations, and networking. In private “Expert Studios,” 16 companies received legal, financial, and general entrepreneurial advice from experienced advisors at Akin Gump, CFGI, Eisner Amper, and Pepper Hamilton. At the main event, Advice from the All Stars, nine specially selected companies presented to some of the most successful and

WHERE THE NEXT GENERATION OF MEDICAL BREAKTHROUGHS ARE HAPPENING

influential life sciences executives in Pennsylvania history: Chris Cashman, Steve Tullman, Vin Milano, Bruce Peacock, Bob Radie, Jim Walker, Julie McHugh, Bob Smith, Erin Brubaker, and Denise McGinn. Those nine companies received customized, focused feedback to help them make successful funding pitches and advance their companies in 2018 and beyond. The day concluded with cocktails, hors d’oeuvres, networking, and celebrating the success of a new event for LSPA members and the start-up community.

HOME OF the

first FDA-approved CAR-T cell therapy AND the first FDA-approved gene therapy 1 out of every 6 doctors IN

THE UNITED STATES SPENT TIME HERE DURING THEIR MEDICAL CAREERS MORE THAN $450 million HAS ALREADY BEEN INVESTED IN advanced

cell & gene therapy manufacturing

PHILADELPHIA | PENNSYLVANIA www.celliconvalley.com


ADVOCACY TA X R E F O R M On December 22, President Trump signed the Tax Cuts and Jobs Act into law. This bill included a number of positive provisions for our small and large member companies, and for our academic research institutions. Those provisions included lowering the overall corporate tax rate to 21%, maintaining the Research and Development Tax Credit, and not repealing the income exclusion for graduate tuition waivers. Unfortunately, the bill significantly weakened the Orphan Drug Tax Credit by cutting it in half. With over 450 orphan therapies developed since its enactment in 1983 (compared to only 10 therapies in the preceding decade), this tax credit clearly incentivized private industry to develop new treatments and cures for rare disease patients and their families. In fact, Life Sciences Pennsylvania sent letters to members who sit on the tax-writing committees in the House and the Senate, and spoke to legislators directly about the importance of this credit. As implementation of the law moves forward, we will continue to work with our partners to advocate for alternative mechanisms to incentivize this critical research and development.

M E D I C A L D E V I C E TA X After inclusion in every version of healthcare reform legislation that came before the House and the Senate, the repeal of the Medical Device Tax appeared all but certain. Unfortunately, none of those bills made it through the Senate, and we were left searching for another legislative vehicle on which to move this critical repeal. There was a package of bills related to Affordable Care Act taxes that was introduced before the holiday break, which included Rep. Paulsen’s (R-MN) legislation to repeal the tax that ultimately went nowhere. In the two weeks leading up to the holidays, we spent time on Capitol Hill discussing the importance and urgency of repealing the Medical Device Tax and the difficulty of fixing it retroactively. Thankfully, the repeal enjoys broad, bi-partisan support amongst the Pennsylvania delegation. Unfortunately, in the week preceding Christmas, we received some unfortunate feedback that Speaker Paul Ryan was willing to let the Device Tax go back into effect in 2018 only to try and address it in the New Year. This was difficult news to digest given the early momentum for repeal, but we will be working diligently with our national partners to ensure full and permanent repeal of the Medical Device Tax in early 2018.

S E N AT E B I L L 6 37 Our activity on opposing legislation concerning “drug price transparency” saw a setback in 2017. State Senator Don White’s legislation came up for a vote on the final day of 2017. We registered our strong opposition to this bill with renewed outreach to committee members and key staff. We worked closely with our national partners at PhRMA and BIO. This is a nationwide issue that has appeared in numerous states. This bill would seek to expose proprietary information from our member companies as they discover and develop new innovative drugs. With what we understood about the Chairman of this important committee and his background in the insurance industry, we knew the approach that would be taken. The vote of 10 to 4 to support this dangerous legislation out of his committee did not surprise us or our many friends in the industry. We will continue to oppose SB 637 as it proceeds in the legislative process. We will be calling on our membership in 2018 to engage our legislative champions.


A DVO C AC Y

S U P P O R T E D PAT I E N T A D VO C AC Y E V E N T S Takedown Cancer: Clearfield vs Philipsburg Osceola Wrestling Match

Strength & Survival: The Universal Language

February 6, 2018

Kimmel Center – Philadelphia, PA

Clearfield Area Senior High School – Clearfield, PA

This free event is hosted by Susan G. Komen Philadelphia as a breast health empowerment program and musical celebration.

Proceeds from the bake sale, silent auction, and other fundraising activities will benefit the Emily Whitehead Foundation.

March 3, 2018

Simon’s Soirée March 17, 2018

ATTRwt Cardiac Amyloid: Often Overlooked, Not Uncommon and Manageable February 7, 2018 Webinar Register for a Webinar presentation from Mathew Maurer, M.D., Arnold and Arlene Goldstein Professor of Cardiology and Professor of Medicine at Columbia University Medical Center.

Citizen’s Bank Park – Philadelphia, PA Proceeds from the event support Simon’s Fund, aimed at saving children by raising awareness about conditions that lead to sudden cardiac arrest and death. The 7th Ataxia Investigators Meeting (AIM 2018): From Bench to Bedside: Entering a New Era of Ataxia Therapies

You Make a Difference Awards Luncheon

April 2-5, 2018

February 9, 2018

Philadelphia Marriott Downtown – Philadelphia, PA This meeting, sponsored by the National Ataxia Foundation, precedes the 61st NAF Annual Ataxia Conference.

The Grand Hall at the Priory – Pittsburgh, PA The luncheon is hosted by the Community Liver Alliance, to recognize accomplishments in liver health awareness and prevention.

Blue Cross Broad Street Run May 6, 2018

The Western PA Chapter of the National Hemophilia Foundation’s Third Annual Iced Tees Winter Golf Outing and Chili Cook-Off February 17, 2018 Diamond Run Golf Club – Sewickley, PA Registration includes nine holes of golf, a gift bag, t-shirt, drink ticket, and chili cook-off entry.

Broad Street and W. Fisher Avenue – Philadelphia, PA Team up with the AACR Runners for Research at a 10-mile run to raise awareness and funds for cancer research. To register, email: [email protected]. NOF’s Interdisciplinary Symposium on Osteoporosis (ISO18)

Epilepsy Foundation Eastern PA’s Annual Mardi Gras Gala

May 17-19, 2018

February 23, 2018

Posted early bird registration deadline is February 1st.

New Orleans Marriott – New Orleans, LA

Crystal Tea Room – Philadelphia, PA Email [email protected] for information about tickets, sponsorship, or how to get involved.

Energy for Life Walk – Pittsburgh June 9, 2018 Heinz Field Riverwalk – Pittsburgh, PA

Party in Purple: Fundraiser for the Alzheimer ’s Chapter

The walkathon supports the United Mitochondrial Disease Foundation (UMDF).

February 24, 2018 The Franklin Institute – Philadelphia, PA Registration is open; Proceeds will benefit the Alzheimer’s Association Delaware Valley Chapter.

American Diabetes Association Camp Freedom Diabetes Camp for Kids June 16-22, 2018 Camp Kweebec – Schwenksville, PA

Rare Disease Week on Capitol Hill February 25 – March 1, 2018

The application period is open for this resident camp program for boys and girls ages 7 to 16 with diabetes.

Washington, DC Registration is open on the Rare Disease Legislative Advocates (RDLA) website. All events are free. 13 | T H E L S PA N E W S L E T T E R , Q 4 2 017

S P E C I A L W E LC O M E T O O U R Q 4 N E W M E M B E R S ADVANCED RPO

FIVE RIVERS RX

ORTHO-TAG

APO TECHNOLOGIES, INC.

FRESH START CAREERS

PULMONESCENCE DIAGNOSTICS

AZER SCIENTIFIC

HARMONY BIOSCIENCES

QUARTESIAN

BAKER TILLY

HUMOTECH

QUEBEC TRADE OFFICE IN PHILADELPHIA

BOTANIX PHARMACEUTICALS LTD.

INTRAVENT MEDICAL PARTNERS, LP

THE CHEMICAL HERITAGE FOUNDATION

CAPGENIC ADVISORS

MARKITECTS, INC.

SICK CELLS

CIVI BIOPHARMA, INC.

MEDNET SOLUTIONS

SMARTCPR

CONSEGNA PHARMA

MOBILION SYSTEMS

SUAZIO CONSULTING

COOK MYOSITE, INC.

NAMSA

SUNOVION PHARMACEUTICALS, INC.

DECHERT LLP

NEOVITA BIOPHARM

THERAPEUTIC ARTICULATIONS LLC

DREAMIT VENTURES

NEURORX, INC.

THERMAQUIL, INC.

ELEVEN BIOTHERAPEUTICS, INC.

ONSPIRA THERAPEUTICS, INC.

VILLANOVA UNIVERSITY

EXCALIVIR, INC.

OPTUM LIFE SCIENCES

VIRPAX PHARMACEUTICALS, INC.

2 017 D I A M O N D M E M B E R S

BioPharm Devices

BioPharm Insight

www.interphasesystems.com


T H E L I F E S C I E N C E S P E N N S Y LVA N I A T E A M CHRISTOPHER P. MOLINEAUX President & CEO

[email protected] 610-947-6800 ext. 203 KAREN DELONE Senior Vice President & Chief Operating Officer

[email protected] 610-947-6800 ext. 210 SOPHIE ALMY Senior Director & Chief Administrative Officer

[email protected] 610-947-6800 ext. 218 ADVOCACY

OPERATIONS BETTY PIKE Accountant

[email protected] 610-947-6800 ext. 207 LAURA NIKORAWALLA Office Manager

[email protected] 610-947-6800 ext. 201 PROGRAMS MELISSA IRWIN Programs Manager

[email protected] 610-947-6800 ext. 208

CRAIG L. TUCKER

MEMBERSHIP

Vice President, Policy and Public Affairs

PATRICK HAYAKAWA

[email protected] 610-947-6800 ext. 205 KURT IMHOF Senior Director, Policy and Public Affairs


ANNE HART Manager, @LifeSciencesPA

[email protected] 610-947-6800 ext. 204 LOIS STEPHENS Membership Services Coordinator

[email protected] 610-947-6800 ext. 209 COMMUNICATIONS LIZ MURNANE Communications Coordinator


Director, Member Relations

[email protected] 610-947-6800 ext. 202 PAUL KORNBLITH Director, Western Pennsylvania

[email protected] 412-770-1637

A Nexus for Collaboration Powered by Wexford Science + Technology, the University City Science Center, and Ventas, uCity Square is a 6.5 million-squarefoot mixed-use development including office, lab, residential, and retail space. ucitysquare.com


L I F E S C I E N C E S P E N N SY LVA N I A 6 5 0 E A S T SW E D E S F OR D ROA D | S U I T E 19 0 WAY N E , PA 19 0 87 L I F E S C I E N C E S PA . O RG

PRSRT STD U.S. POSTAGE PAID PERMIT #117 SOUTHEASTERN PA 19399

W W W. L I F E S C I E N C E S PA . O RG