1 | THE LSPA NEWSLETTER , Q3 2017

T H E N E W S L E T T E RL I F E S C I E N C E S P A’ S Q U A R T E R LY N E W S S O U R C E

Q 3 2 0 1 7

I N T H I S I S S U E

Featured Conten t . . . . . . . 1

Membersh ip News . . . . . 3

F inanc ia l Bu l le t in . . . . . . 8

Advocacy . . . . . . . . . . . . . . 10

Event Recap . . . . . . . . . . . 12

Suppor ted Events . . . . . 12

New Members Q3 . . . . . 14

LSPA Team . . . . . . . . . . . . . 15

L I FE SCIENCES FUTURE 2017

The Philadelphia Marriott Downtown was the venue September 18 and 19 for Life Sciences Future - Life Sciences Pennsylvania’s annual two-day conference. Leading executives traveled from all corners of the U.S. and abroad to attend the event - bringing together the academic, industry, funding, and strategic service provider communities. Life Sciences Future highlights Life Sciences Pennsylvania’s diverse membership and the

broader community of life sciences stakeholders in the Mid-Atlantic region, focusing on the newest scientific advancements while looking into the future of our industry.

CONNECTING THE MID- ATLANTIC’S L IFE SCIENCES COMMUNIT Y

L I F E S C I E N C E S F U T U R E - S E P T E M B E R 18 - 19 - P H I L A D E L P H I A

“Life Sciences Future is one of the only fully-inclusive events for the life sciences community in the Mid-Atlantic region,” said Lori Reiner, Partner-In-Charge, Philadelphia, EisnerAmper LLP and Chair of Life Sciences Future. “This year’s conference was designed to highlight Pennsylvania as home to all facets of the life sciences industry, but also to showcase the commonwealth as an epicenter for leading cell and gene therapy programs. Life Sciences Future is the premier opportunity to bring together - and facilitate partnerships - between academic research, entrepreneurs, industry, investors, government and, ultimately, patients.”

September 18, the opening day of Life Sciences Future, featured the Partnerships in Science symposium, created to put research institutions, early to mid-stage companies, and business development teams in the same room to forge relationships that could lead to business opportunities. The day kicked off with a panel featuring chief medical executives from Egalet, HVH Precision Analytics, and Geistlich Pharma; followed by an opening keynote address from Dr. Jonathan A. Epstein of the Perelman School of Medicine. Directly following Dr. Epstein’s remarks was a fireside chat with The Wistar Institute’s President and CEO, Dr. Dario Altieri.

The action in the afternoon took place on the Presentation Stage, where global pharma and medical device company teams from Bristol-Myers Squibb, Johnson & Johnson, Merck, B. Braun, and AstraZeneca, described their business development philosophies and their areas of therapeutic or technical interest and introduced the key contacts and members of their Business Development teams. On the same stage, emerging companies - including SFA Therapeutics, CARMA Therapeutics, Sonavex, Oncoceutics, Yiviva, Quantitative Radiology Solutions, Sen-Jam Pharmaceuticals,

LIFE SCIENCES FUTURE RECAP CONTINUED ON PAGE 9

2 | WWW.L I FESC IENCESPA .ORG

On September 19, 2017, Life Sciences Pennsylvania released a new economic impact report on the life sciences in Pennsylvania. The research, conducted by KPMG, presents a new and fresh focus on the life sciences community.

The findings of this study reflect our community and its evolution from reliance on large anchor pharmaceutical companies to an ecosystem that includes hundreds of start-up biotech, pharma, medical device and diagnostic companies, as well as academic research, CROs, digital health start-ups, contract manufacturers and distributors, just to name a few. The executive summary of this report is featured below and the full report is available online at www.lifesciencespa.org.

PENNSYLVANIA’S L IFE SCIENCES INDUSTRY ECONOMIC IMPACT REPORT - 2017

Executive Summary

Amid rapid change brought on by new forms of competition, new regulations, and innovation, the life sciences industry in Pennsylvania is in the process of reinventing itself. The life sciences industry has historically been a strong contributor to Pennsylvania’s economy. Following the financial crisis, the pace of growth has slowed in the face of increased costs, heightened uncertainty, changes in regulations and risk factors, and greater demands for efficiency. These forces are reshaping the industry, forcing companies to adopt new technologies, integrate data analytics into their management and operational practices, and reassess the business models used to develop new products and deliver goods and services to the market. Employment in this industry increased on net by fewer than 1,000 jobs during the five years between 2011 and 2016, but stronger net hiring during 2015 and 2016 more than offset all of the job losses that occurred between 2011 and 2013.

Innovation in Pennsylvania’s life sciences industry has also been advancing at a rapid rate. Through increased federal funding, patent applications, and entrepreneurship, Pennsylvania maintains a lead over much of the country in terms of research and development (R&D) in Life Sciences.

Pennsylvania’s life sciences industry faces several hurdles. One of those hurdles is the attraction and retention of entrepreneurial capital. Although it is difficult to measure entrepreneurial capacity directly, anecdotal evidence suggests that a dearth of Pennsylvania-based entrepreneurs with experience in life sciences ventures is a limiting factor for the industry’s rate of expansion in the Commonwealth. The second challenge, closely related to the first, lies in the attraction of venture capital and angel investors. Compared with states such as California and Massachusetts, Pennsylvania ranks relatively lower when it comes to venture capital investment, as discussed later in the report. Although Pennsylvania boasts a favorable relative share of federal health and science research grants and production of doctoral recipients in life sciences disciplines, the Commonwealth lags below its peers in attracting venture capital investment, securing a disproportionately lower share of capital than that flowing into companies in California and Massachusetts. Similarly, the number of patents issued for life sciences discoveries lags well behind Massachusetts and California.

The purpose of this report is to summarize the contribution of the Life Sciences industry to the Pennsylvania economy during 2016. Included in the sidebar are some key findings from this study.

L IFE SCIENCES IN PENNSYLVANIA

More than half of the life sciences industry establishments operating in Pennsylvania in 2016 are comprised of fewer than 10 employees.

Between 2011 and 2016, Pennsylvania experienced modest life sciences employment growth in comparison to peer states. This is primarily due to a decline in pharmaceutical manufacturing in the Commonwealth and concurrent gains in research, testing and medical laboratories.

RESEARCH EXCELLENCE

Pennsylvania had the highest 2016 National Science Foundation (NSF) funding rate in comparison to peer states and was awarded the second highest NSF & National Institute of Health (NIH) funding per capita in comparison to peer states.

Pennsylvania was a top contributor to innovation, boasting recipients of 4,317 patents over the prior 5 years, among the top 5 states nationally.

ACADEMIC CONTRIBUTION

The Commonwealth was home to 7 of the top 100 universities ranked in 2016 according to the Shanghai Index, including 2 universities in the top 100 Biological Sciences Program.

Pennsylvania universities conferred a doctorate degree to 578 recipients, ranking 4th highest in the nation during 2015.

ATTRACTING CAPITAL

During 2016, Pennsylvania secured $147 million in venture capital (VC) funding, ranking 13th nationally.

ECONOMIC CONTRIBUTION

The life sciences industry in Pennsylvania directly employed 112,000 people during 2016 and was responsible indirectly for an additional 230,000 jobs through business purchases and household expenditures.

The industry generated a total (direct and indirect) state economic output of $88.5 billion, comprised of a direct economic impact of $48.8 billion and indirect economic impact of $39.6 billion.

COMMONWEALTH SUPPORT

Pennsylvania offers several supportive incentive programs across various industries which are often utilized by companies in the life sciences industry

3 | THE LSPA NEWSLETTER , Q3 2017

M E M B E R S H I P

Successful partnerships in Pennsylvania’s life sciences community are what help propel the industry forward. In The Newsletter - Q2 - Life Sciences Pennsylvania profiled CSL’s CEO and Managing Director Paul Perreault, who discussed the importance of building a strong community and partnerships to help advance the industry. In mid-September, CSL did just that - by way of a $4.92 million gift to Penn State University to create the multidisciplinary Center for Excellence in Biotechnology, as well as to revitalize the Shared Fermentation Facility, an engine for collaboration and innovation in biological training and research on the University Park campus.

According to a news release issued by Penn State University, the Center of Excellence in Industrial Biotechnology will serve as a unique hub of multidisciplinary teaching and research. The Center will leverage expertise from the Eberly College of Science and the Colleges of Engineering and Agricultural

Sciences at Penn State, while supporting students, faculty and their research. Preparing and educating students to enter the dynamic, rapidly growing field of biotechnology is the center’s primary objective and most important function.

Globally known for its leading edge R&D and production of therapeutic proteins, CSL Behring’s gift will advance Penn State and the Commonwealth of Pennsylvania as transformative leaders in biotechnology education.

“These are exciting times in biotech, and Penn State’s Center of Excellence will help meet the growing need for world-class teaching and research centers,” said CSL CEO and Managing Director Paul Perreault. “At CSL we’re driven by our promise to protect the health of people with rare and serious diseases around the world. Supporting Penn State’s new biotech venture is a natural outgrowth of our promise and of our willingness to consistently foster innovation.”

CSL BEHRING TO HELP TRANSFORM BIOTECH EDUCATION AND RESEARCH PRESENTS $4.92 MILL ION GIFT TO PENN STATE UNIVERSIT Y

The city of Philadelphia boasts many firsts since its founding in 1682 by William Penn, including the nation’s first established hospital in 1751, first medical school in 1765, first college of pharmacy in 1821, and the first children’s hospital in 1855 – among many others. As an industry, 2017 marks another first for Philadelphia, and Pennsylvania broadly – the first FDA-approved T-cell cancer treatment for pediatric leukemia – pioneered at the University of Pennsylvania and the Children’s Hospital of Philadelphia and commercialized by Novartis.

In August, the U.S. Food and Drug Administration approved Kymriah(TM), the first chimeric antigen receptor T-cell (CAR-T) therapy, for the treatment of patients up to 25 yeras of age with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse. According to a press release issued by Novartis, Kymriah is a novel immunocelluar therapy and a one-time treatment that uses a patient’s own T-cells to fight cancer.

“At Novartis, we have a long history of

being at the forefront of transformative cancer treatment,” said Joseph Jimenez, CEO of Novartis. “Five years ago, we began collaborating with the University of Pennsylvania and invested in further developing and bringing what we believed would be a paradign-changing immunocellular therapy to cancer patients in dire need. With the approval of Kymriah, we are once again delivering on our commitment to change the course of cancer care.”

“This therapy is a significant step forward in individualized cancer treatment that may have a tremendous impact on patients’ lives,” said Carl June, MD, the Richard W. Vague Professor of Immunotherapy, Director of the Center for Cellular Immunotherapies in Penn’s Perelman School of Medicine, who is a pioneer of this new treatment. “Through our collaboration with Novartis, we are creating the next wave of immunocellular cancer treatments, and are eager to progress CAR-T therapy in a host of hematologic and other cancer types.”

“Tisagenlecleucel is the first CAR-T therapy to demonstrate early, deep and

durable remission in children and young adults with relapsed or refractory B-cell ALL,” said Stephan Grupp, MD, PhD, the Yetta Deitch Novotny Professor of Pediatrics at the Perelman School of Medicine at Penn, and Director of the Cancer Immunotherapy Frontier Program at Children’s Hospital of Philadelphia (CHOP). “We’ve never seen anything like this before and I believe this therapy may become the new standard of care for this patient population.”

Novartis is committed to ensuring eligible patients have access to Kymriah, and is working to ensure payers understand the value of Kymriah and provide coverage for patients.

Novartis plans additional filings for Kymriah in the US and EU later this year, including applications with the FDA and European Medicines Agency (EMA), for the treatment of adult patients with r/r diffuse large B-cell lymphoma (DLBCL). Additional filings beyond the US and EU are anticipated in 2018.

FDA APPROVES F IRST -EVER T -CELL CANCER TREATMENT KYMRIAH™ P IONEERED AT CHOP AND PENN - COMMERCIALIZED BY NOVARTIS

4 | WWW.L I FESC IENCESPA .ORG

Temple University, located in Philadelphia, and the University of Pittsburgh, have been selected as two of only 11 clinical hubs for a new National Institutes of Health (NIH) emergency medicine clinical trials network. The NIH established the Strategies to Innovate Emergency Care Clinical Trials Network, or SIREN, to support the rapid implementation of high quality, large-scale clinical trials in emergency medicine. SIREN will support clinical trials aimed at improving patient outcomes for neurologic, heart, lung, blood and traumatic emergencies. The SIREN clinical hubs will coordinate the efforts of a large group of academic medical centers and emergency medical systems throughout the metro Philadelphia area and across Pennsylvania and New Jersey.

“Temple University Hospital is a leading site for emergency care clinical trials and is proud to be selected by NIH for this innovative clinical trial network, which has the potential to change and improve practices nationwide for emergency medicine physicians treating patients with a wide variety of emergent conditions,” says Nina Gentile, MD, Professor of Emergency Medicine at the Lewis Katz School of Medicine at Temple University, and lead physician for Temple’s participation in the network. “Temple’s emergency department is a tertiary care, academic, level-1 trauma center, and averages more than 127,000 emergency visits per year when you combine visits to its main campus and Episcopal campus. Being one of only eleven clinical sites selected for SIREN is further confirmation of Temple’s national reputation for emergency medicine expertise.”

SIREN consists of a Clinical Coordinating Center at the University of Michigan, a Data Coordinating Center at the Medical University of South Carolina, and 11 clinical centers (Hubs) across the country that will coordinate and conduct clinical trials in both emergency department treatment and pre-hospital settings (such as in the ambulance). These trials will cover all areas of emergency research from the most critical, such as heart attack and traumatic brain injury, to chronic conditions such as asthma

NIH SELECTS TEMPLE UNIVERSIT Y AND THE UNIVERSIT Y OF P ITTSBURGH HUBS FOR CL INICAL TR IALS NET WORK WILL SERVE AS T WO OF 11 CLINICAL HUBS NATIONWIDE

MEMBERSHIP

and migraine headache, that frequently lead people to emergency departments.

The institutions announced as part of SIREN are:

Clinical Coordinating Center University of Michigan Ann Arbor, MI Data Coordinating Center Medical University of South Carolina Charleston, SC Clinical Hubs

Temple University Philadelphia, PA

University of Pittsburgh Pittsburgh, PA Emory University Atlanta, GA

Massachusetts General Hospital Boston, MA

Medical College of Wisconsin Milwaukee, WI

Oregon Health Sciences University Portland, OR

University of California Los Angeles, CA

University of Cincinnati Cincinnati, OH

University of Minnesota Minneapolis, MN

University of Washington Seattle, WA

Wayne State University Detroit, MI

5 | THE LSPA NEWSLETTER , Q3 2017

The National Lacrosse League (NLL), the largest men’s professional indoor lacrosse league in North America - founded in 1986 and headquartered in Conshohocken, PA, and Egalet Corporation, a specialty pharmaceutical company and member of Life Sciences Pennsylvania focused on developing innovative treatments for pain and other conditions has forged a strategic partnership focused on physician education around responsible pain management.

The two-year agreement will include a product symposium at the 2017 NLL Medical Summit to educate physicians on how to help treat pain with a nonsteroidal anti-inflammatory drug (NSAID), which is also a non-narcotic pain management

option. Egalet’s product SPRIX® (ketorolac tromethamine) Nasal Spray, is an NSAID approved in the United States for the short term (up to 5 days) management of moderate to moderately severe pain in adults that requires analgesia at the opioid level.

“The health and safety of our players is paramount and we’re proud to partner with Egalet, a company committed to providing innovative pain medications,” said Nick Sakiewicz, Commissioner of the NLL. “Box lacrosse is a fast-paced and physical sport and given NLL’s commitment to the safety and well-being of our players, it is critical that our team physicians are aware of options like SPRIX Nasal Spray.”

Egalet is the first pharmaceutical partner for the NLL. Player health is overseen by an extensive base of private practice healthcare providers including primary care, orthopedic surgeons, neurologists and dentists.

“Egalet is committed to responsible pain management education and increasing awareness of a non-narcotic pain medication as a treatment option for moderate to moderately severe acute pain in adults,” said Pat Shea, chief commercial officer of Egalet. “Together with NLL, we can help educate healthcare providers about a non-narcotic treatment option for acute pain.”

MEMBERSHIP

NAT IONAL L ACROSSE LEAGUE AND EGALET CORPORATION ANNOUNCE EDUCATION PARTNERSHIP PROMOTE NON-NARCOTIC PAIN OPTION AND RESPONSIBLE PAIN MANAGEMENT

IMAGE OF NLL COMMISSIONER NICK SAKIEWICZ V IA JOHN GEORGE, PHIL ADELPHIA BUSINESS JOURNAL

REALTY VILLAGE AD

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

6 | WWW.L I FESC IENCESPA .ORG

In the world of life sciences, CAR-T and T-cell therapies continue to be at the forefront of industry news - and rightfully so - in many cases it means the cure for cancer could be just around the corner. Life Sciences PA members, and leaders in the field of T-cell therapy, Adaptimmune Therapeutics and GlaxoSmithKline have formed a collaboration to research, develop, and advance and commercialize Adaptimmune’s NY-ESO SPEAR T-cell therapy program.

According to a joint press release issued by Adaptimmune and GSK, during this partnership Adaptimmune will receive up to $61 million from GSK which includes development milestones of up to $23 million and the option payment of $38 million which also allows GSK to nominate two additional targets following completion of the transition. Successful continuation of development and subsequent commercialization of NY-ESO would trigger additional payments for development milestones, tiered sales milestones, and mid-single to low

double-digit royalties on worldwide net sales.

“This is a very exciting day for Adaptimmune as GSK has exercised its option over our NY-ESO program, earlier than originally planned,” said James Noble, Chief Executive Officer at Adaptimmune. “The commitment by one of the world’s leading pharmaceutical companies to the NY-ESO SPEAR T-cell program as a new treatment modality is a testament to the strength of our data in synovial sarcoma recently presented at ASCO. From a financial perspective, this option exercise extends our cash runway into 2020. We anticipate the transition of NY-ESO to GSK to be completed over the coming months, after which we will focus our clinical resources on delivery and execution from our wholly-owned assets MAGE-A4, MAGE-A10, and AFP.”

Axel Hoos, SVP Oncology R&D, GSK said, “the aim of GSK’s R&D is to develop medicines with transformational potential for

patients. We have seen compelling data for the NY-ESO investigational cell therapy in synovial sarcoma and, following this option exercise, we will capitalize on our in-house Cell and Gene Therapy capabilities to support the development program for GSK3377794. We will continue to explore the potential for this novel cell therapy in multiple tumor types, and in combination with other cancer therapies.”

GSK’s Jessica Imrie and Adaptimmune’s Ad Rawcliffe shared the stage - along with WuXi AppTec and Spark Therapeutics - at Life Sciences Future - in mid-September.

GSK AND ADAPT IMMUNE PARTNER TO ADVANCE AND COMMERCIAL IZE T -CELL THERAPY PROGRAM LEADING THE CHARGE IN CELL ICON VALLEY

Life Sciences Pennsylvania member, Cutanea Life Sciences, based in Wayne, PA, has released a companion mobile app to accompany its first dermatology drug, Aktipak. Facing ForwardTM, available on the iTunes and Google App stores, features a selfie camera function to dynamically track acne progress, dosing and refill reminders, a how-to video for using the medication, and other helpful acne information.

While Aktipak is Cutanea’s first product, the company plans a similar product-plus-tech co-launch strategy for each of the products in its 10-drug deep, all-dermatology pipeline.

“The target demographic for us are the same people who are constantly on their phones. Today’s teenagers and young adults are constantly on the go, and they’re into technology,” said Cutanea Brand Manager Bob Bitterman Jr. “The app provides an appealing way to help tech savvy patients use the medication as dosed and as directed by their physician.”

CUTANEA RELEASES NEW MOBILE APP FOR ACNE PAT IENTS BRIDGES THE GAP BET WEEN SELF IES AND MEDICINE

MEMBERSHIP

7 | THE LSPA NEWSLETTER , Q3 2017

SchuylkillYards.comPhiladelphia, PA

Accelerate your life science venture here.

Where Ideas Converge.

1M SFLab

2.8M SFOffice

7M SFof Opportunity

987K SF Innovation Space

215.397.1589 www.brandywinerealty.com

20171019_SY ad.indd 1 10/20/2017 12:33:34 PM

8 | WWW.L I FESC IENCESPA .ORG

F I N A N C I A L B U L L E T I N - Q 3• Marinus Pharmaceuticals, Inc. announced the closing of an underwritten public offering

of 10,733,334 shares of its common stock for a public offering price of $3.75 per share. - September 19, 2017

• Nabriva Therapeutics plc announced the pricing of its underwritten public offering of 9,411,765 ordinary shares at a public offering price of $8.50 per share. - September 19, 2017

• Aclaris Therapeutics, Inc., a dermatologist-led, biopharmaceutical company, announced the pricing of its underwritten public offering of 3,260,000 shares of its common stock at a price to the public of $23.02 per share. - August 10, 2017

• VenatoRx Receives $9.4 million from CARB-X to Develop a New Class of Antibiotic to Combat Multi-Drug Resistant Bacteria - July 27, 2017

• Complexa Raises $62 Million in Series C Financing to Test CXA-10 in Two Orphan Disease Proof-of-Concept Trials - July 26, 2017

9 | THE LSPA NEWSLETTER , Q3 2017

and Renerva - showcased their breakthrough technologies to a packed - standing room only - house.

To further the mission of facilitating connections at Life Science Future, Funding Meet-Ups took place on both Day One and Day Two - offering exclusive networking opportunities with leading funding organizations from throughout the U.S.

To wrap up a successful Day One, science of the future met cocktails of today in an adjacent ballroom where 36 scientific research posters from 21 academic institutions were presented. September 19 began with Breakfast in the C-Suite for CEOs, CFOs, Chief Business Officers and Chief Scientific Officers, with special guest Air Force General Mark Rosenker, Director of the White House Military Office on 9/11 and former Chairman of the National Transportation Safety Board (NTSB) when USAirways pilot “Sully” Sullenberger landed Flight 1549 in the Hudson River.

Following Breakfast in the C-Suite was the release of the findings from the 2017 Life Sciences Economic Impact Report - commissioned by Life Sciences Pennsylvania and compiled by KPMG [see related story on pg. 2].

The first plenary panel discussion of day two, “From the Heart of Cellacon Valley” - where leaders from Adaptimmune, GSK, Spark Therapeutics, and WuXi AppTec shared the stage to discuss research, funding, manufacturing, and commercializing cell and gene therapies - kicked off immediately following the economic impact report overview. The discussion was moderated by 6ABC’s Health and Medical Reporter Ali Gorman, R.N.

On the Presentation Stage, Company Reverse Pitch and Emerging Company presentations were again intertwined to

L I F E S C I E N C E S F U T U R ECONTINUED FROM PAGE 1

create a continuous stream of content throughout the day. Business Development leaders from DSM Biomedical, CSL Behring, Teva, GlaxoSmithKline, Teleflex, Shire, and Pfizer, discussed their in-licensing strategies, while Trovita Health Science, ConverGene, Wellesley Pharmaceuticals, LifeCuff Technologies, Genome Profiling, Recensa Therapeutics and QR Pharma, discussed their technologies and opportunities for partnerships. Short Take Presentations focusing on Genetic Testing; Cell and Gene Therapy Licensing Deals; Value Based Care and Pricing; Companion Diagnostics; and Digital Health; were also taking place on the same stage.

Finally, to wrap up Day Two of Life Sciences Future, Emmy Award-winner, TV personality, multiple sclerosis patient, and advocate, Montel Williams, took to the Main Stage as the Patient Spotlight Keynote. Montel shared his experience with PoNSTM, an experimental medical device being developed by Helius Medical Technologies – a Pennsylvania-based life sciences company – and focused our attention on the true

beneficiaries of the community’s daily efforts: patients. Joining Montel on stage was Jeri Lake - a traumatic brain injury patient who shared her own personal experience with the PoNSTM experimental device - and the positive impact it had on her life.

10 | THE LSPA NEWSLETTER , Q3 2017

A D V O C A C Y

As issues in our nation’s capital remain fluid, Life Sciences Pennsylvania worked with two of its member companies to organize congressional “in-district” visits in July and August.

Congressman Brendan Boyle (D-13th) visited BTG Pharmaceuticals U.S. headquarters in West Conshohocken (PHOTO: Top-R). During the visit the Congressman received an executive briefing on BTG and its work in his district and around the country, after which he gave an insightful update on all of the news coming out of Washington, D.C.

Congressman Mike Doyle (D-14th) visited Helomics (PHOTO: Bottom-R), a diagnostics company in Pittsburgh. While visiting Helomics the Congressman was given an overview of their work by company leadership, followed by a tour of their lab space.

These “in-district” member visits are an important way to amplify our advocacy efforts as they give our elected officials direct access to the life science companies and their employees working to find the next breakthrough cures or treatments for patients.

Life Sciences Pennsylvania was again honored to take part in the annual Rally for Medical Research in Washington, D.C. This event, initiated by the Pennsylvania-based American Association for Cancer Research (AACR), brings together more than 300 national organizations to call on our country’s elected officials to support robust National Institutes of Health funding, and ensure those officials understand the importance of medical research for patients, and for our life science economy. While the President’s budget made significant cuts to the NIH budget, advocacy efforts like this have ensured that most, if not all, of the Pennsylvania congressional delegation opposed those cuts and supported an increase in NIH funding.

RALLY FOR MEDICAL RESEARCH ORGANIZES HILL DAY PUSHES FOR INCREASED NIH FUNDING

L IFE SCIENCES PENNSYLVANIA ORGANIZES SUMMER MEMBER V IS ITS CONGRESSMEN BOYLE AND DOYLE VIS IT BTG AND HELOMICS

Photo Credit: Association of American Medical Colleges

11 | THE LSPA NEWSLETTER , Q3 2017

RESEARCH IN YOUR BACKYARD WITH CONGRESSMAN RYAN COSTELLO AT BIOCLINICA

In August, Life Sciences Pennsylvania partnered with We Work for Health to hold a discussion and press event on PhRMA’s recently updated Research in Your Backyard study. We were honored to be joined by Congressman Ryan Costello (R-6th) at this event, as well as BioClinica’s President and CEO, John Hubbard, Emma Watson from the American Cancer Society and Elizabeth Beil of the Epilepsy Foundation of Eastern Pennsylvania. The updated study reinforced the fact that Pennsylvania is home to one of the most robust clinical trial landscapes anywhere in the country.

Report highlights include:

Since 2004, biopharmaceutical research companies have conducted or are conducting more than 7,800 clinical trials of new medicines in Pennsylvania.

As of July 2017, there were 1,178 clinical trials active in Pennsylvania.

546 of those were devoted to cancer alone, but they seek to address all of the Commonwealth’s healthcare needs including Alzheimer’s, arthritis and cardiovascular disease, among many others.

With a strong biopharmaceutical presence and top-tier healthcare systems, in 2013 alone, an estimated 47,538 Pennsylvania patients were enrolled in a clinical study.

As the focus in Washington, D.C. has turned away from healthcare reform, tax reform has become the next target for congressional leadership.

Life Sciences Pennsylvania partnered with Ernst and Young, and host, BioTelemetry, to participate in a tax reform roundtable discussion with Congressman Pat Meehan (R-7), a member of the House Ways and Means Committee – the chief tax-writing committee of the U.S. House. Much of the meeting focused on generally lowering the corporate tax-rate in the United States, with Life Sciences Pennsylvania ensuring the Congressman is aware of the tax reform priorities of our members, including the Research & Development Tax Credit, the Orphan Drug Tax Credit, and full and permanent repeal of the Medical Device Tax - messages that we have also echoed to our U.S. Senators Casey and Toomey, both of whom sit on the Senate’s tax writing committee – the Senate Finance Committee.

EYES TURN FROM HEALTHCARE REFORM TO TAX REFORM IN D.C. TAX REFORM DISCUSSION WITH CONGRESSMAN MEEHAN

12 | WWW.L I FESC IENCESPA .ORG

Life Sciences Pennsylvania held its first MedTech + Digital Health Summit in July at The Wistar Institute in Philadelphia. The half-day program drew senior leaders, regulatory and quality experts, entrepreneurs, and business development executives from the MedTech + Digital Health community in Pennsylvania and beyond.

The day opened with a panel focused on MedTech innovation and funding - featuring New Enterprise Associates, Red and Blue Ventures, Penn Center for Innovation, and DSM Biomedical. The rapid-fire agenda moved quickly into three, 20-minute, back-to-back “Knowledge Bursts” - covering FDA’s Expedited Pathway Process, Digital Health Analytics, and CMS Reimbursement.

The FDA’s Director of the Center for Devices and Radiological Health, Dr. Jeffrey Shuren, and the president and CEO of the Medical Device Manufacturers Association (MDMA), Mark Leahey, shared the stage to discuss the strategic priorities of the FDA, and the potential impact, both positive and negative, of a new administration.

To wrap up the agenda, panelists had an open and honest discussion which took its direction from the previous fireside chat with Shuren and Leahey.

The day concluded with a networking lunch in The Wistar Institute’s atrium. Life Sciences Pennsylvania will be hosting three additional summits in 2017 - Supply Chain (October 24), Start-Up Stadium - Advice from the All-Stars (November 14), and CRO & CMO (November 17).

E V E N T SL IFE SCIENCES PENNSYLVANIA L AUNCHES SPECIAL INTEREST SUMMIT SERIES IN PHIL ADELPHIAMEDTECH + DIGITAL HEALTH SUMMIT

13 | THE LSPA NEWSLETTER , Q3 2017

Life Sciences Pennsylvania held its 2017 Chief Scientific

Officer Dinner in July at St. David’s Golf Course in

Wayne, PA. This exclusive event, offered to those

serving in the capacity of chief executive officer, chief

science officer, chief medical officer, or head of R&D

at a Life Sciences PA Core Member company, featured

cocktails, dinner, networking, and a keynote discussion

by Dr. David H. Ledbetter, executive vice president and

chief scientific officer, Geisinger Health System.

L IFE SCIENCES PA HOSTS CHIEF SCIENTIF IC OFF ICERS AND CEOS FOR DINNER AT ST. DAVID’S

FEATURING DR. DAVID H. LEDBETTER

Amyloidosis Foundation 2nd Annual Pittsburgh Amyloidosis Research Benefit October 27, 2017 Montour Heights Country Club - Coraopolis, PA

Emily Whitehead Foundation Believe Ball October 28, 2017 Valley Forge Casino Resort - King of Prussia

2017 ACS CAN Forum-Research and Emerging Treatments in the Cancer Sphere November 1, 2017 Independence Blue Cross - Philadelphia

Hepatology and Liver Transplantation Conference November 3, 2017 Chemical Heritage Foundation - Philadelphia

Quest for a Cure Gala: Turning Promising Research into Permanent Cures: A Fundraiser for Castleman Disease Research November 10, 2017 The Mutter Museum - Philadelphia

2017 Philly Walk to End Alzheimer ’s November 11, 2017 Citizens Bank Park - Philadelphia

Epilepsy Education Exchange November 11, 2017 Delaware County Intermediate Unit - Morton, PA

PA Breast Cancer Coalition Webinar Clinical Trials: Breast Cancer Research to Improve Treatment presented by Priya Rastogi, MD November 14, 2017

RareVoice Awards November 15, 2017 Arena Stage - Washington, DC

Introduction to the 2017 National Standards for Diabetes Self-Management Education/Support Recognition November 17, 2017 Hilton Philadelphia City Ave - Philadelphia

Kickin’ Cushings to the Curb November 17, 2017 Hershey Country Club - Hershey, PA

The Lemon Ball January 13, 2018 Philadelphia Marriott Downtown - Philadelphia

Philadelphia Fight for Air Climb March 24, 2018 Three Logan Square - Philadelphia

National Osteoporosis Foundation’s Jumping Jack Challenge #JumpingJackChallenge @NationalOsteoporsisFoundation

The 7th Ataxia Investigators Meeting (AIM 2018): From Bench to Bedside: Entering a New Era of Ataxia Therapies April 2-5, 2018 Philadelphia Marriott Downtown - Philadelphia

S U P P O R T E D PAT I E N T A DVO C AC Y E V E N T S

EVENTS

14 | WWW.L I FESC IENCESPA .ORG

S P E C I A L W E LC O M E TO O U R Q 3 N E W M E M B E R S

3FX

ACCURA TECHTONIQ LLC

BLINK CONSULTING

CAREER CONCEPTS, INC. DBA CCI CONSULTING

CBM GROUP

CENERO

CENTER CITY FILM & VIDEO

CO-SERVE LLC

FULTON BANK

HELIUS MEDICAL TECHNOLOGIES

INDUSTRY STANDARD RESEARCH

JPG HEALTHCARE LLC

L.E.A.F. PHARMACEUTICALS LLC

LIPELLA PHARMACEUTICALS, INC.

LOCKTON, CO.

LUMIS CORP.

NATIONAL ATAXIA FOUNDATION

NMS LABS

NOBLE LIFE SCIENCES, INC.

PA BREAST CANCER COALITION

QUANTITATIVE RADIOLOGY SOLUTIONS

RECENSA THERAPEUTICS

RECTOR COMMUNICATIONS, INC.

RENERVA, LLC

SFA THERAPEUTICS

SL VERNICK COMMUNICATIONS

SMITHWISE

SPRINGER LEADERSHIP

TALEE BIO

TMUNITY THERAPEUTICS INC.

TUNNELL CONSULTING

WIZSOLUTION LLC

www.interphasesystems.com

BioPharmDevices BioPharmInsight

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

15 | THE LSPA NEWSLETTER , Q3 2017

CHRISTOPHER P. MOLINEAUX President & CEO cmolineaux@lifesciencespa.org 610-947-6800 ext. 203

KAREN DELONE Senior Vice President & Chief Operating Officer kdelone@lifesciencespa.org 610-947-6800 ext. 210

SOPHIE ALMY Senior Director & Chief Administrative Officer salmy@lifesciencespa.org 610-947-6800 ext. 218

ADVOCACY

CRAIG L. TUCKER Vice President, Policy and Public Affairs ctucker@lifesciencespa.org 610-947-6800 ext. 205

KURT IMHOF Senior Director, Policy and Public Affairs kimhof@lifesciencespa.org 610-947-6800 ext. 211

OPERATIONS

BETTY PIKE Accountant bpike@lifesciencespa.org 610-947-6800 ext. 207

LAURA NIKORAWALLA Office Manager lnikorawalla@lifesciencespa.org 610-947-6800 ext. 201

PROGRAMS

MELISSA IRWIN Programs Manager mirwin@lifesciencespa.org 610-947-6800 ext. 208

SHANNON WILSON Programs Coordinator swilson@lifesciencespa.org 610-947-6800 ext. 212

MEMBERSHIP

PATRICK HAYAKAWA Director, Member Relations phayakawa@lifesciencespa.org 610-947-6800 ext. 202

PAUL KORNBLITH Director, Western Pennsylvania pkornblith@lifesciencespa.org 412-770-1637

ANNE HART Manager, @LifeSciencesPA ahart@lifesciencespa.org 610-947-6800 ext. 204

LOIS STEPHENS Membership Services Coordinator lstephens@lifesciencespa.org 610-947-6800 ext. 209

COMMUNICATIONS

LIZ MURNANE Communications Coordinator emurnane@lifesciencespa.org 610-947-6800 ext. 217

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

A Nexus for Collaboration

Powered by Wexford Science + Technology, the University City Science Center, and

Ventas, uCity Square is a 6.5 million-square-foot mixed-use development including office, lab, residential, and retail space.

ucitysquare.com

LIFE SCIENCES PENNSYLVANIA650 EAST SWEDESFORD ROAD | SUITE 190

WAYNE, PA 19087

LIFESCIENCESPA.ORG

PRSRT STDU.S. POSTAGE

PAIDPERMIT #117

SOUTHEASTERN PA19399