the newsletter - Life Sciences Pennsylvania

Q1 2018

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

2 018 A N N U A L D I N N E R - M A R C H 14 - P H I L A D E L P H I A , PA

2018 ANNUAL DINNER RECAP On March 14, 2018, Life Sciences PA held its Annual Dinner at the Pennsylvania Convention Center in Philadelphia. More than 1,300 life sciences industry professionals – a record-breaking number of attendees – gathered to celebrate the successes of the past year. Across the street at the Pennsylvania Academy of the Fine Arts, Pennsylvania Governor, Tom Wolf, gave remarks at the Board of Directors Reception that precedes the Annual Dinner. At the invitation-only reception, Pennsylvania Senator, Kim Ward, was presented the State Official of the Year Award, and U.S. Congressman, Brendan Boyle, was recognized as the Federal Official of the Year Awardee. The dinner kicked off with a video celebrating Life Sciences PA’s member milestones of 2017. Following the video, Life Sciences PA’s new Board Chairman, Stephen Tullman, gave opening remarks and presented the first honor of the evening – our Patient Impact Award. Over the course of the evening, the remaining seven awards were presented to organizations and individuals who shaped Pennsylvania’s life sciences landscape in 2017. The full list of award winners and finalists can be found on pages 14 and 15.

IN THIS ISSUE Featured Content. . ........... 1 Events............................ 4 Membership . . ................. 6

The Annual Dinner ended with remarks from Caroll Neubauer, Chairman and CEO, B. Braun Medical, and Christopher Molineaux, President and CEO, Life Sciences PA – both of whom extended a community-wide invitation to the MedTech Conference being held in Philadelphia in September of this year. Following the dinner program, guests convened in the foyer for networking over dessert and coffee. Life Sciences PA looks forward to next year’s Annual Dinner, and to celebrate the accomplishments of the life sciences community in Pennsylvania in 2018.

President’s Column.......... 9 Financial Bulletin........... 12 Annual Dinner Awards... 14 Advocacy......................16 Supported Events...........17 New Members Q1......... 18 LSPA Team . . ...................19 1 | T H E L S PA N E W S L E T T E R , Q 1 2 0 1 8

A LOOK AHEAD Pennsylvania will host several international conventions over the next 18 months. The staff at Life Sciences PA has already been hard at work planning for the upcoming MedTech Conference in September 2018 and the return of the BIO International Convention in June 2019. These conventions provide a showcase of Pennsylvania’s vibrant life sciences community on an international stage and deliver on many other fronts – they are economic drivers for Pennsylvania and, when well planned, can greatly benefit attendees by informing them of emerging industry trends, opening extensive, diverse networks and – most importantly – creating new business opportunities. As your trade association, we work to partner with the convention organizers to maximize the effectiveness in attending. As evidenced in 2015, when the BIO International Convention returned to Philadelphia after a 10-year hiatus, 70+ companies utilized the Pennsylvania Pavilion as their home base and meeting hub during the convention. Over the past few years, Life Sciences PA has represented Pennsylvania’s life science industry both here in the Commonwealth and in cities across the country – San Diego, San Francisco, Chicago, Washington, D.C., Minneapolis and San Jose. In January, we attended the J.P. Morgan Healthcare Conference in San Francisco. This conference, the largest healthcare investment conference in the country, brought together life sciences companies and investors of all sizes. Life Sciences PA was pleased to co-host a reception for the Pennsylvanians in attendance and took part in a number of meetings with business leaders from across the country. Looking further into 2018, we are proud to again be hosting the Pennsylvania Pavilion at the BIO International Convention in Boston. BIO has set a goal of 18,000 attendees, and we’re looking forward to another strong showing from Pennsylvania’s life sciences community. Please contact Karen DeLone if you would like more information on the Pavilion, and how your company can expand its network and business opportunities at this international convention.

CONFERENCE FORECAST FOR 2018

J.P. Morgan Healthcare Conference SAN FRANCISCO January 8-11

Life Sciences Week PITTSBURGH April 16-20

CPhl North America Conference PHILADELPHIA April 24-26

BIO International Convention BOSTON June 4-7

The MedTech Conference PHILADELPHIA September 24-26

Life Sciences Future PHILADELPHIA October 29-30

“Attending my industry’s annual conference (The MedTech Conference) is among the most rewarding things I do each year! From inspiring keynotes to thought-provoking programming to endless networking, it truly is a must-go event. If you have anything to do with MedTech, plan to be in Philadelphia this September and, if you’re in biotech, be in Boston this June.” CAROLL NEUBAUER, Chairman & CEO, B. Braun Medical Inc.

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2017 ANNUAL REPORT The 2017 Annual Report covers highlights from the 2017 economic impact report, advocacy successes, program highlights, membership breakdowns, and a timeline of key milestones from the year.

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PROGRAMS

M E M B E R C O M PA N I E S

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135

SPEAKERS

NEW MEMBERS IN 2017

1,121

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C O M PA N I E S

4,150

MEMBERS CONNECTED V I A L S PA P R I VAT E LINKEDIN GROUP

AT T E N D E E S

MEMBERSHIP

PROGRAMS GROUP PURCHASING

HIGHLIGHTS

$8.4M

S AV E D B Y M E M B E R S T H R O U G H T H E L S PA S . A . V. E . P R O G R A M

NEW MEMBER BENEFIT

@LSPA - THE LIFE SCIENCES CENTER OPENED ITS DOORS TO MEMBERS

ADVOCACY SUCCESSES

LIFE SCIENCES PA CONTINUED TO MAKE PROGRESS TOWARD FULL REPEAL OF THE MEDICAL DEVICE TAX

E C O N O M I C I M PA C T

LIFE SCIENCES PA AND KPMG RELEASED THE 2017 PENNSYLVANIA LIFE SCIENCES IMPACT REPORT To view the full 2017 Annual Report, visit the Advance tab at www.lifesciencespa.org. 3 | T H E L S PA N E W S L E T T E R , Q 1 2 0 1 8

EVENTS PA’ S L I F E S C I E N C E S C O M M U N I T Y AT T E N D S J . P. M O R GA N H E A LT H C A R E C O N F E R E N C E C E L L I C O N VA L L E Y A D S G R E E T M E M B E R S I N S A N F R A N C I S C O

On the heels of the Q4 Start-Up Stadium – Advice from the All Stars event, many Life Sciences PA members traveled to San Francisco for the investor-focused 36th Annual J.P. Morgan Healthcare Conference held January 8-11. Upon arrival, attendees were greeted by a series of electronic billboards featuring newly launched branding for “Cellicon Valley,” the nickname for the Philadelphia region’s scientific and business prowess in cell and gene therapy. The series of ads were featured in the San Francisco International Airport, local taxis, and in 37 office buildings and hotels near the conference venue. Ads highlighted Philadelphia as a leader in academic research, manufacturing, and medical devices and diagnostics. Colleagues from Penn hosted a program titled Innovation in Action, where the Cellicon Valley branding was also prominently displayed. In a recent Philadelphia Business Journal article by Jeff Blumenthal, a number of Life Sciences PA members shared their own thoughts on why Philadelphia should be recognized as Cellicon Valley. LSPA_CelliconValley_banner_4x14_v2.pdf

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“I think the answer is yes,” Azam said. “All the pieces are there for Philadelphia to be seen as the center for precision medicine.” DR. USMAN AZAM on Philadelphia’s potential to be the life sciences epicenter

“I remember being at an event and I was sitting next to Carl June [a pioneer in the field of CAR-T Cell therapy and director of translational research at the Abramson Cancer Center at Penn],” Skolnik said. “I texted my wife and said, ‘You’ll never guess who’s sitting next to me.’ She said, `Mick Jagger?’ And I said, ‘No, Carl June.’ For people in our world, he is bigger.” JEFFREY SKOLNIK, Inovio Pharmaceuticals Vice President for Clinical Development, on Philadelphia’s A-list life sciences celebrities

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WHERE THE NEXT GENERATION OF C

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MEDICAL BREAKTHROUGHS ARE HAPPENING

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HOME OF THE

first FDA-approved cell and gene therapies

LSPA_CelliconValley_banner_4x14_v2.pdf

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PHILADELPHIA | PENNSYLVANIA

www.celliconvalley.com

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WHERE THE NEXT GENERATION OF C

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MEDICAL BREAKTHROUGHS ARE HAPPENING

WHERE MORE THAN $450 million HAS ALREADY BEEN INVESTED IN

advanced cell and gene therapy manufacturing


PHILADELPHIA | PENNSYLVANIA

www.celliconvalley.com

EVENTS

CELEBRATING DR. PAUL KORNBLITH’S BIRTHDAY LONG-TIME LIFE SCIENCES COLLEAGUE AND MENTOR TURNS 80

On January 18, colleagues and friends of Dr. Paul Kornblith gathered at Hofbräuhaus Pittsburgh to celebrate his 80th birthday, which occurred just days before on January 15. A world-renowned neurosurgeon and clinical cancer researcher, Paul Kornblith, M.D., currently serves as the Western Pennsylvania Director for Life Sciences PA; Medical Advisor to Helomics, the Pittsburgh Life Sciences Greenhouse, and the University of Pittsburgh Innovation Institute; and Adjunct Professor in the School of Health and Rehabilitation at the University of Pittsburgh.

M A K I N G T H E M O S T O F YO U R M E M B E R S H I P SOLD-OUT SESSIONS BAC K BY POPUL AR DEMAND

Life Sciences PA hosted two sold-out Making the Most of Your Membership events, on February 13 and March 22. The interactive seminar is designed for members who are looking for ways to build and improve their life sciences network. Participants heard from longtime members Charlie Huntington, Senior Vice PresidentInvestment Officer of Huntington Kraus Financial Group, and John Maslowski, Chief Executive Officer of Fibrocell Science. Topics included how to position a company as a valuable resource within the life sciences community, what to know about the unique industry, and techniques to optimize Life Sciences PA membership.

2 018 A N N UA L D I N N E R B U S R I D E FROM PITTSBURGH ABSORPTION SYSTEMS A HIT WITH PITTSBURGH RIDERS

Following the 2018 Annual Dinner, a group of attendees from Pittsburgh had the opportunity to tour Absorption Systems in Exton, PA.

“The tour of Absorption Systems was a highlight for the bus riders from Pittsburgh. Absorption Systems is a linchpin for Philadelphia’s Cellicon Valley, as one of the leaders in potency assay development and testing. Nearly every new immunotherapy or gene therapy drug on the market has utilized their assays to gain FDA approval and continued batch releases. Their expertise is another reason that Philadelphia is leading this charge into cellular therapies.” CHRISTIAN MANDERS, Chief Operating Officer, Promethean LifeSciences, Inc.

UPCOMING EVENTS To learn about upcoming events in 2018, visit http://www.lifesciencespa.org/connect/events/

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MEMBERSHIP SORRENTO THERAPEUTICS & SUBSIDIARY, SCILEX PHARMACEUTICALS, GAIN FDA APPROVAL FOR ZTLIDO Z T L I D O I S A P P R OV E D F O R T H E R E L I E F O F PA I N A S S O C I AT E D W I T H P O S T - S H I N G L E S PA I N

Sorrento Therapeutics and its majority-owned subsidiary, Scilex Pharmaceuticals, received U.S. Food and Drug Administration (FDA) approval for ZTlido on February 28. ZTlido, a topical lidocaine, is indicated for the relief of pain associated with post-herpetic neuralgia (PHN), also referred to as post-shingles pain. The wearable patch is a major advancement in pain relief as an alternative to opioids. PHN patients have been turning to opioids, none of which are approved as first-line treatment. The new patch is a safe non-opioid alternative. Its adhesion

technology demonstrates 12 hours of effective lidocaine delivery, even during exercise. Scilex will be working toward a commercial launch of ZTlido in the U.S. sometime this year. “ZTlido was designed to solve a problem that is commonly reported with transdermal/topical patches: they don’t stay on. Based on the adhesion study results with ZTlido, we believe the ZTlido product will be welcomed by healthcare providers, patients and payers who are looking for an effective and efficient, local pain treatment,” said Dr. Henry Ji, Chairman and Chief Executive of Sorrento and Scilex.

PHASEBIO PHARMACEUTICALS AWARDED GRANT TO SUPPORT CLINICAL DEVELOPMENT IN PULMONARY ARTERIAL HYPERTENSION T H E $ 2 . 8 M I L L I O N G R A N T W I L L S U P P O R T T H E D E V E LO P M E N T O F I N N OVAT I V E T H E R A P I E S F O R O R P H A N D I S E A S E S

As an emerging player in the development of innovative therapies for orphan diseases, PhaseBio Pharmaceuticals, Inc., based in Malvern, received a $2.8 million Fast Track Small Business Innovation Research (SBIR) grant from the National Institutes of Health for its new drug candidate – PB1046 – targeting pulmonary arterial hypertension (PAH). “Recent advancements in the treatment of PAH have improved patient symptoms and exercise capacity but are not curative, and long-term disease prognosis remains poor,

leaving the door open for innovative new therapeutic options like PB1046,” said John Lee, PhaseBio’s Chief Medical Officer. The SBIR grant will support clinical trials for PB1046. The ongoing clinical trial is enrolling adult PAH patients with a permanently implanted hemodynamic monitor. The monitor tracks how the heart is functioning by measuring blood flow and how much oxygen is in the blood. PhaseBio expects to start the Phase II clinical trial by mid-2018.

NEURO KINETICS, INC. RECEIVES FDA CLEARANCE FOR I-PORTAL RESEARCH DEVICE A B R E A K T H R O U G H S YS T E M I N T E G R AT I N G C L I N I C A L E Y E T R AC K I N G W I T H A D I G I TA L D I S P L AY

As innovators in clinical eye-tracking technology, Pittsburghbased Neuro Kinetics, Inc. received the U.S. Food and Drug Administration’s (FDA) approval of the I-Portal® Portable Assessment System, or I-PAS™, on March 7, adding to its portfolio of cleared medical devices and tests. The head-mounted, multi-modal system is used as a nystagmograph (vestibular and neurotologic diagnoses). I-PAS slips on like a virtual reality headset, and is fully portable. It integrates clinical eye tracking with a digital display to run non-invasive oculomotor and vestibular tests, while capturing eye movement objectively and precisely. Tests using I-PAS


can support research on concussions, drug effectiveness, Parkinson’s disease and more. “I-PAS’s portability, affordability, and ease of use is transformational for healthcare professionals who evaluate and treat concussion and vestibular patients, whether the setting is a physical therapy clinic, physician’s office, or sports field,” said Howison Schroeder, President and Chief Executive Officer of Neuro Kinetics. I-PAS shipments begin in April, and the product is also being used to support research studies involving OVRT-C, to assess the effectiveness of drugs to treat concussion.

MEMBERSHIP

LIBERTY PROPERTY TRUST MOVES HEADQUARTERS TO WAYNE C O M PA N Y L AU N C H E S A $ 12 M I L L I O N R E D E V E LO P M E N T OF THE PROPERT Y

After more than four decades in Great Valley, Liberty Property Trust is embarking on a new venture, relocating to more modern headquarters in Wayne. This will mark the completion of the company’s transformation into an industrial real estate investment trust with some urban holdings. Over the years, Liberty Property Trust has evolved by taking on urban development, doubling down on industrial space, and moving on from owning suburban office space; it’s now a $9 billion real estate company with space across the U.S. and UK. The company launched a $12 million redevelopment of the new property, and plans to occupy the 4th floor of the building, a 25,000-square-foot space. “This company has always been moving forward and thinking

about the next place we should be whether it’s a new market, the right balance of product mix...this is a natural logical extension of that process,” said Bill Hankowsky, Chief Executive Officer of Liberty Property Trust. “The location has that Wayne-King of Prussia, live-work-play characteristic that is analogous to what we have done at the Navy Yard and we decided to take that skill set and apply that to a building we own,” Hankowsky said. According to Brian Cohen, Vice President at Liberty, the two main goals to Liberty’s approach are to stand out in the market and differentiate it from the competition. The company is also looking for the building to act as a developer, bringing in something new to the market, while attempting to elevate and increase the value.

THE UNIVERSITY OF PITTSBURGH PARTNERS WITH UPMC TO BUILD $200 MILLION IMMUNOLOGY INNOVATION FACILITY A PA R T N E R S H I P TO A DVA N C E I M M U N O LO GY S C I E N C E

The University of Pittsburgh and UPMC are once again partnering to create a new center for immunology, cancer treatment and aging. The $200 million UPMC Immune Transplant and Therapy Center will be part of a redesigned eight-story, 200,000-square-foot building located at 5000 Baum Boulevard. The redesigned building will be utilized as a world-class space for labs, offices, start-up companies, and industry partners. With a focus on immunology and other life sciences, the center will bring together cutting-edge research from the

University and UPMC, most of it geared toward clinical use at UPMC facilities. The center’s work will initially focus on three major immunotherapy areas: organ transplant acceptance, cancer treatment and aging. “Beyond the UPMC facility, the site on Baum will also be used to build up Pittsburgh’s life sciences innovation and commercialization of technologies and treatments that are being developed by Pitt researchers. The goal is to create an Innovation District that is centered around 5000 Baum,” said Pitt Chancellor Patrick Gallagher.

INOVIO PHARMACEUTICALS RECEIVES $4.6 MILLION GRANT TO ADVANCE TUBERCULOSIS & MALARIA VACCINES G R A N T TO A S S I S T W I T H R E S E A R C H I N PA R T N E R S H I P W I T H T H E W I S TA R I N S T I T U T E

Inovio Pharmaceuticals, Inc., received $4.6 million in grant funding from the Bill & Melinda Gates Foundation and the National Institutes of Health (NIH) to advance work on vaccine programs targeting tuberculosis and malaria. Inovio is conducting its research as part of a partnership with the Wistar Institute. Both programs involve the use of Inovio’s SynCon synthetic vaccine technology that helps a person’s immune system recognize cells with antigens associated with a virus or disease. The Gates Foundation grant will be used for malaria research, while the grant from the NIH’s National Institute of Allergy & Infectious Diseases will fund the tuberculosis research. The pharma company will collaborate with researchers in

the laboratory of the Vaccine & Immunotherapy Center at The Wistar Institute; Inovio co-founder Dr. David B. Weiner currently serves as the director. “What we learn from developing vaccines against these infectious disease programs we can also apply to advance our cancer-focused therapies,” said Dr. Laurent Humeau, Senior Vice President for Research and Development at Inovio. “This platform is well suited to address global health outbreaks, with its demonstrated potency and long safety track record in the clinic as well as its rapid design, ease of manufacturing and transportation/storage. Based on the quality of preclinical data, Inovio and its collaborators plan to obtain additional funding to advance the vaccines into clinical studies.” 7 | T H E L S PA N E W S L E T T E R , Q 1 2 0 1 8

MEMBERSHIP

UNIVERSITY OF PENNSYLVANIA ONE OF SEVEN CHOSEN IN $7 MILLION STAND UP TO CANCER CONVERGENCE RESEARCH PROGRAM USING AI C O N V E R G E N C E 2 . 0 R E S E A R C H I N I T I AT I V E U T I L I Z E S A I TO I N V E S T I GAT E I M M U N E S YS T E M R E S P O N S E S TO C A N C E R S

Several University of Pennsylvania scientists were among the seven multi-disciplinary research teams awarded $11 million from Stand Up To Cancer’s (SU2C) “Convergence 2.0” research initiative, who will have access to Microsoft Research’s experts in machine learning and artificial intelligence to investigate immune system response to cancers. The seven awardees were announced at SU2C’s Scientific Summit in January. The Penn team is comprised of experts in life sciences, physical sciences, mathematics and engineering, and will have the opportunity to work collaboratively with Microsoft’s machine learning experts to uncover key characteristics of the interaction between cancer and the immune system, potentially leading to the development of new treatments. The project chosen by the Convergence 2.0 Selection Committee and SU2C Scientific Advisory Committee was the Single-Cell Functional Multi-Omics to Characterize and Monitor CAR-T Therapy. The team is led by Rong Fan, Ph.D., Yale University, and team members Stephanie Halene, M.D., Ph.D., Yale School of Medicine and Yale University;

and Carl H. June, M.D., Pablo G. Cámara, Ph.D., and J. Joseph (“Jos”) Melenhorst, all from the University of Pennsylvania Perelman School of Medicine. In addition to the support from Microsoft, the Lustgarten Foundation for Pancreatic Cancer Research committed $1.76 million in funding, and the Society for Immunotherapy of Cancer (SITC) provided $1 million for early-career scientists to work with leading researchers. Over the course of three years, the SU2C Research Teams will explore a host of factors that may contribute to a patient’s response to a specific routine through data derived from multiple data sets, including information on a patient’s genome, imaging studies, and medical and medication records, among other data. The Penn team will assess the mechanisms of efficacy and/or immune toxicity in using CAR-T therapy, utilizing the machine learnings that look at the data. If successful, Penn will have created a “tool” that clinicians can use to alleviate patient risk associated with CAR-T therapies.

FDA APPROVES EXPANDED USE OF ASTRAZENECA PHARMACEUTICAL’S CANCER DRUG LYNPARZA ASTRAZENECA TAKES STEP FORWARD IN EXPANDING BEYOND OVARIAN CANCER TREATMENT

In a key win for cancer patients, the U.S. Food and Drug Administration (FDA) approved expanded use of AstraZeneca Pharmaceuticals’ cancer drug, Lynparza, to include patients with metastatic breast cancer associated with BRCA gene mutation. This is the first time a PARP inhibitor has been approved to treat breast cancer and the first time any drug has been approved to treat certain patients with metastatic breast cancer associated with the same genetic mutation. Phase III data showcased during last summer’s American Society of Clinical Oncology further enhanced the FDA’s focus on drugs that can target cancers triggered by the same genetic causes. The drug was linked with tumor shrinkage in 60 percent of patients with germline BRCA1 or BRCA2 mutations taking the drug, compared to 29 percent in the chemo group. “This approval demonstrates the current paradigm of developing drugs that target the underlying genetic causes of a cancer, often across cancer types,” said Pascal Soriot, Chief Executive Officer at AstraZeneca. “Between 5-10 percent of patients with breast cancer have a BRCA mutation.”

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 Liz Murnane at [email protected] to be considered for our next issue.

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LIFE SCIENCES PENNSYLVANIA

PRESIDENT’S COLUMN C H R I S T O P H E R P. MOLINEAUX PRESIDENT & CEO L I F E S C I E N C E S P E N N S Y LV A N I A

The life sciences community in Pennsylvania has historically been a reliable source of jobs and a strong driver of Pennsylvania’s economy. During the last decade, however, the industry and its broader community have witnessed painful downsizing and contraction in the large pharma sector, a major presence in the Commonwealth. What many have missed, however, is a quiet but steady re-birth of the life sciences and the formation of hundreds of new companies, founded, in many cases, by former academicians and biopharma and device company executives. 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 percent increase in the number of life sciences establishments. This speaks to the entrepreneurial orientation 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 should be 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, but it is also a fragile community that requires nurturing. Life Sciences PA recognizes the nature of this growing constituency: small, hungry, high-risk enterprises, and continues to refine its advocacy activity and member services to meet the needs of this new model of membership. In 2018 – and for the foreseeable future – Life Sciences PA’s advocacy activity will be ever more inclusive of small company interests and span a more diverse membership that includes nearly equal numbers of biotech, pharmaceutical, medical device and diagnostic companies, contract research organizations and academic research intuitions. Moreover, the Association’s events and services will have a tighter focus than ever before on facilitating strategic connections between and among entrepreneurs and the resources needed to enable life sciences growth and success. ADVOCACY REMAINS CRUCIAL As the life sciences industry continues to change and Life Sciences PA evolves to match the ever-shifting landscape and membership, engagement in shaping the public policy environment becomes increasingly important. Efforts at the federal government level in 2017 to repeal and replace the Affordable Care Act failed multiple times, although its gradual dissolution seems inevitable. While the financial markets seem buoyed by new federal policies, the broader political climate in Washington continues to pose uncertainty

2018-2021 STRATEGIC PLAN PRIORITIES •E nsure a supportive business and public policy climate in Pennsylvania to support company formation and retention in the Commonwealth •F acilitate partnerships between and among entrepreneurs, large industry, small companies, research institutions and funding organizations to advance company formation, innovation, job creation and success in the industry •P romote and support strategic engagement between the Pennsylvania life sciences industry and key industry stakeholders •S upport efforts to position Pennsylvania as a top location for life sciences business and opportunity through select communication initiatives •P rovide valued services and access to cost-efficient resources for Association members to make informed decisions and grow their business for innovator life sciences companies of every size, both as research-driven organizations and as employers of people. 2017 ushered in new players at the federal and state levels, creating new opportunities – and new challenges – to the life sciences industry policy agenda. Pennsylvania is fortunate to have a supportive Congressional delegation in Washington, D.C. and at year-end 2017 saw Federal tax reform that dramatically lowered the corporate tax to a more globally competitive 21 percent. However, this good news was accompanied by a reduction in the Orphan Drug Tax Credit that is vital to companies pursuing treatments and cures for patients with rare diseases. Moreover, efforts to fully repeal the Medical Device Tax remained stymied despite multiple standalone bills with broad bi-partisan support. Of note: seven of Pennsylvania’s 18 members of Congress announced they will not be seeking reelection in 2018. In the Pennsylvania 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 thinly-veiled attempts to shift attention away from annual insurance industry rate hikes, which have reached 3540 percent in some instances. Life Sciences PA and its allies have been successful in the last two years in keeping such poorly conceived policy proposals from gaining traction. Nevertheless, pressure in the name of “transparency” continues to build across the political and healthcare communities, and the Association’s educational efforts must endure. Any conversation about state policy, politics and the life sciences industry must include mention of the opioid abuse epidemic, a human tragedy whose effect has been felt by thousands of Pennsylvanians. Life Sciences PA must continue to educate policy makers and those in law enforcement (e.g., the office of the Attorney General) on efforts by industry to address this tragedy. Furthermore, the entire life sciences 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 10


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community must encourage a science-based and researchdriven discussion about solutions – while protecting patients’ appropriate access to these important products. INTERNATIONAL PROFILE At the dawn of 2018, Life Sciences PA enjoys six agreements with partners outside the United States. These connections and formal partnerships allow Life Sciences PA to better assist its members with a warm hand-off to its international partners as the Association simultaneously works to promote Pennsylvania as a destination for companies.

diagnostics and pharmaceuticals, in addition to the growing category of Research, Testing and Medical Laboratories. Pennsylvania also boasts the other elements of the life sciences value chain: academic research, investment organizations, manufacturing capability and commercialization expertise. As the industry trade association for the life sciences in Pennsylvania, Life Sciences PA strives to marshal these components through its programs and its unmatched network of contacts to facilitate the new model and usher in a new industry, characterized by more streamlined companies that rely on multi-sourced expertise for much of their discovery and development efforts.

LOOKING FORWARD In the face of these challenges and opportunities, 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. 2017 brought the first two FDA-approved cell and gene therapies...from scientists in Pennsylvania. Indeed, researchers have been pursuing gene therapy for more than three decades in hopes it can yield treatments and cures for genetic conditions that have no other treatments. Pennsylvania scientists have now provided a path forward for treatments to a wide range of rare conditions that have not been previously treated. Amidst this change, Pennsylvania is one of the few locations in the world where all disciplines of the emerging life sciences industry model can be found. In Pennsylvania, the life sciences sector includes companies in the fields of biotechnology, medical devices,


As the state-wide trade association for this important economic driver for the Commonwealth, Life Sciences PA is the sole organization that represents their common voice. Moreover, Life Sciences PA’ s affiliations with the national peer groups PhRMA, BIO, AdvaMed, MDMA and ACRO provide it an unmatched state presence with national and international reach. The Strategic Plan for 2018-2021 assumes the transformation of the industry will continue at its current rapid pace. It also assumes this transformation and uncertainty offer unprecedented opportunity for Life Sciences PA to leverage its size and influence to attract, retain and grow the future drivers of the life sciences industry within Pennsylvania and beyond its borders.

REPRINTED WITH PERMISSION FROM THE PHILADELPHIA BUSINESS JOURNAL

Wanna bet? Pa. and Mass. biotech groups wager on Super Bowl outcome By John George – Senior Reporter, Philadelphia Business Journal Jan 26, 2018, 11:35am

Add the state trade associations representing the life sciences industries in Pennsylvania and Massachusetts to the list of organizations placing a friendly wager on the outcome of next week’s Super Bowl. Chris Molineaux, president and CEO of Wayne, Pa.-based Life Sciences Pennsylvania, initiated the bet with his counterpart at the Massachusetts Biotechnology Council. In a letter to Robert K. Coughlin, president and CEO of MassBio, Molineux wrote:

The terms of the bet will be met on June 5, 2018 at the BIO Convention.

“Dear Bob, I hope this finds you well on this sunny January day as you and your fellow Patriots fans brace yourselves for another Super Bowl. We in Pennsylvania certainly admire the track record of Bill Belichick, Tom Brady, Bob Kraft and the entire Pats organization; our Eagles will no doubt face a formidable opponent on February 4 in Minneapolis. However, what the Eagles might lack in Super Bowl championships, Eagles fans more than make up with team spirit, loyalty and a recognition of when we’ve got a real winner headed to the Big Game. “With this in mind, I propose a small wager between my team and yours. And I’m not referring to the Eagles and the Patriots, although these NFL powerhouses will determine who receives the spoils. I’m referring to Life Sciences PA and MassBio. Ironically – or perhaps by serendipity – one of the largest annual conventions of our industry, the BIO International Convention, will be held in Boston this June and in Philadelphia in June of 2019…making the stage for exchanging the “winnings” of this proposed wager even more rich. So what’s at stake? Well, the bet — which MassBio has accepted — has three parts: a jersey swap, a beer swap and a food swap. Life Sciences Pa. wins if the Eagles do. MassBio wins if the Patriots do. For the jersey swap, the loser of the bet will be required to don the opposing team’s jersey either on the exhibit floor of the BIO Convention, or on stage during one of the widely attended plenary events. “If this happens on stage, it would be clear for all to see,” Molineaux wrote. “If this happens on the floor, the loser is required to make the “walk of shame” from one state pavilion to the other while wearing the jersey.” For the beer swap, the loser will present the winner a full case of beer from his home state at the home pavilion of the winner. Yards Brewing Co. in Philadelphia and Harpoon Brewing Co. In Boston have already announced a similar bet. For the food swap, the loser of the wager will present the winner with a food item from their home state or city to be eaten in their respective pavilion. “I would offer you one of the Philadelphia cheesesteaks you enjoyed during your visit to Philly,” Molineaux wrote, “but since the Eagles are going to win the Super Bowl, I will place my order with you now for some clam chowder from the Union Oyster House.”

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 Liz Murnane at [email protected] Thank you in advance for your input. 11 | T H E L S PA N E W S L E T T E R , Q 1 2 0 1 8

FINANCIAL BULLETIN Q1 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 funding news from the 1st Quarter of 2018. • Forest Devices, Inc. announced it has raised $2.3 million to assist in furthering research and development of its portable stroke screening technology tool, AlphaStroke, for use by medical personnel – January 11, 2018 • Spark Therapeutics, Inc. entered a $105 million deal with Novartis Pharmaceuticals to license the rights to its gene therapy treatment, Luxturna, outside the U.S., and the potential to earn an additional $65 million in milestone payments – January 25, 2018 • Inovio Pharmaceuticals, Inc., entered into an amended agreement that calls for ApolloBio Corp. to make an upfront payment of $23 million for the exclusive rights to develop and commercialize Inovio’s experimental DNA immunotherapy product in and around China – February 12, 2018 • 1315 Capital made the first close for its second fund 1315 Capital II, raising more than million to invest in pharmaceutical and medtech companies – February 12, 2018

$300

• Cernostics announced its $2.5 million Series A1 financing, with new lead investor, Illumina Ventures, to grow clinical and marketing development studies for TissueCypher® Barrett’s Esophagus Assay – March 21, 2018 • Scout Bio, a gene therapy start-up company spun out of the University of Pennsylvania, raised $9.29 million from a private stock sale to advance its portfolio of immunotherapies to treat cancer – March 27, 2018

THREE LIFE SCIENCES PA MEMBERS AID IN $500K TO SUPPORT CHRONIC DRY EYE CLINICAL TRIALS LSPA MEMBER FUNDING CLOSES FIRST-ROUND SEED FOR TREATMENT RELATED TO DIABETES

Penn State University & College of Medicine spin-out biotech start-up, Ocunova, closed a round of $500,000 seed funding in support of clinical trials for treatment associated with chronic dry eye condition as it relates to diabetes. The first-round seed comes from two other Life Sciences PA members — the Life Sciences Greenhouse of Central Pennsylvania and Ben Franklin Technology Partners/Central and Northeastern Pennsylvania. The clinical trials for treatment enlist a drug that’s already approved by the U.S. Food and Drug Administration (FDA) - Naltrexone, which manages alcohol and opioid dependence. Currently, more than three million diabetic patients experience chronic dry eye in the U.S. “Current treatments are limited to either over-the-counter eye drops that provide temporary relief to the eye, or two prescription anti-inflammatory medications, which have delayed onset of action, variable efficacy and side effects, which limit compliance. There are no treatments approved for diabetic dry eye. Ocunova’s drug candidate, OCU-001, is designed to affect the underlying cause of dry eye, which is a novel approach,” said Michael Shine, Chief Executive Officer at Ocunova. There are a few life sciences companies that have also developed dry eye disease treatments; however, Shine believes his company’s treatment will be a game-changer for the disease.


SAVE THE DATE OCTOBER 29-30, 2018

THE AN N U AL C O N F E R E N C E F OR T H E L I F E SC I E N C E S CO M M U N I TY OF T HE M I D - A T L A N T I C R E GI O N

PLANNING COMMITTEES ARE FORMING Visit: www.lifesciencespa.org and save $200 off registration

PENNSYLVANIA CONVENTION CENTER 13 | T H E L S PA N E W S L E T T E R , Q 1 2 0 1 8

2018 ANNUAL DINNER THOUGHT LEADER OF THE YEAR AWA R D W I N N E R Dietrich Stephan, Ph.D. Chief Executive Officer LifeX

H U B E R T J . P. S C H O E M A K E R L E A D E R S H I P AWA R D W I N N E R

Andrew Greenspan, Janssen, and Chris Molineaux, Life Sciences PA present Benson F. Smith the Hubert J.P. Schoemaker Leadership Award

Benson F. Smith Chairman of the Board and former Chief Executive Officer Teleflex, Inc.

FRANK BALDINO BIOSCIENCE CEO O F T H E Y E A R AWA R D W I N N E R Peter Miller Chief Executive Officer Optinose, Inc.

S C I E N T I F I C A C H I E V E M E N T AWA R D W I N N E R Carl H. June, M.D. Richard W. Vague Professor, Immunotherapy Department of Pathology and Laboratory Medicine Perelman School of Medicine, University of Pennsylvania Lisa Waitte, Fisher Scientific and Chris Molineaux present Dr. Carl June the Scientific Achievement Award

PAT I E N T I M PA C T AWA R D H O N O R E E S CSL Behring Spark Therapeutics, Inc. Teleflex, Inc.

D E A L O F T H E Y E A R AWA R D W I N N E R Harmony Biosciences’ $270 million in Equity Financing

D E A L O F T H E Y E A R AWA R D F I N A L I S T S Joseph Reagan, Wexford Science + Technology and Chris Molineaux present the Deal of the Year Award to Harmony Biosciences 14 | W W W. L I F E S C I E N C E S PA . O R G

Complexa, Inc.’s $62 million Series C Financing Teleflex, Inc.’s Acquisition of Neotract

WINNERS + FINALISTS E M E R G I N G C O M PA N Y O F T H E Y E A R AWA R D W I N N E R Optinose, Inc.

E M E R G I N G C O M PA N Y O F T H E Y E A R FINALISTS Neos Therapeutics, Inc. Paratek Pharmaceuticals, Inc.

FEDERAL OFFICIAL OF THE YEAR AWA R D W I N N E R

Steve Abrams, Hogan Lovells and Chris Molineaux present the Emerging Company of the Year Award to Optinose, Inc.

U.S. Congressman Brendan F. Boyle D-Philadelphia/Montgomery

S TAT E O F F I C I A L O F T H E Y E A R AWA R D W I N N E R Pennsylvania State Senator Kim L. Ward Pennsylvania State Senate

P R E S I D E N T ’ S S E R V I C E AWA R D W I N N E R Thomas Hess, CPA Senior Vice President and Chief Financial Officer Genomind

Dr. Todd Johnson, Optum and Chris Molineaux present the Thought Leader of the Year Award to Dr. Dietrich Stephan

CLARKSTON SCHOLARS PROGRAM SCHOLARSHIP RECIPIENT Noah Yann Lee Sophomore, Computational Biology Mellon College of Science and the Carnegie Mellon School of Computer Science

For more information about our award winners, visit the Connect tab at www.lifesciencespa.org

Joseph D’Ambrosio, Clarkston Consulting and Chris Molineaux present Noah Yann Lee with the Clarkston Scholars Program scholarship 15 | T H E L S PA N E W S L E T T E R , Q 1 2 0 1 8

ADVOCACY R O U N D TA B L E W I T H S E N AT O R T O O M E Y As issues continue to swirl around our nation’s capital, Life Sciences PA was pleased to coordinate a roundtable discussion with U.S. Senator Pat Toomey. The discussion was hosted at Genomind in King of Prussia, and we had a strong turnout of many emerging companies. It was a welcome opportunity to thank the Senator for his hard work in passing the Tax Cuts and Jobs Act, and suspending the Medical Device Tax for two years – though he remains committed to a full and permanent repeal of the tax. The Senator was also able to provide the attendees with a general update on what has been happening in Washington, D.C., and was engaging and receptive to feedback on the FDA, and recent policy changes to Medicare that may impact the life sciences community. These discussions are an important way to amplify our advocacy efforts and ensure our elected officials are hearing directly from leaders in the life sciences community who are working to find the next breakthrough cures and treatments.

M E D I C A L D E V I C E TA X On January 22, Life Sciences PA was proud to report that the Medical Device Tax was suspended for two years. The Medical Device Tax was passed into law as part of the Affordable Care Act (“Obamacare”) in 2010. Its full and permanent repeal has been a top priority of Life Sciences PA’s advocacy efforts since that time. While this bill presents only a two-year suspension of this destructive tax, it is an important step in the right direction, as the IRS was set to begin collecting this tax on January 29. We have been in regular contact with our Pennsylvania Congressional Delegation since the tax’s inception, and we are pleased that the repeal of the Medical Device Tax enjoys broad, bi-partisan support. We will continue to work with our federal elected officials on a full and permanent repeal of this tax.

A D VA M E D F LY - I N At the end of February, Life Sciences PA was proud to participate in the annual AdvaMed Fly-In. Held early each year in Washington, D.C., this advocacy opportunity focuses on the public policy priorities that are important to our medical device and diagnostic membership. At the top of the priority list this year was thanking members for their support of the two-year suspension of the Medical Device Tax, urging members to support a bill that will provide temporary transitional Medicare coverage for FDAdesignated breakthrough products, and helping members of congress understand the role medical devices can play in the opioid crisis.


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 Walk to Defeat ALS

Walk for Epilepsy Philadelphia

May 2018

NOF’s Interdisciplinary Symposium on Osteoporosis (ISO18)

Philadelphia, PA

May 17-19, 2018

Philadelphia, PA

Multiple dates; See website

Get Fit for Research Month

Philadelphia, Pittsburgh, & multiple PA locations

June 24, 2018

New Orleans, LA

The Western PA Hemophilia Foundation’s 4th Semi- Annual Cornhole Tournament

Flavors of Philadelphia

April 7, 2018

May 2, 2018

Oakdale, PA

Philadelphia, PA

May 2, 2018

Steel Chef Challenge

Lupus Links Golf Tournament

Philadelphia, PA

May 20, 2018

July 16, 2018

Pittsburgh, PA

Lafayette Hill, PA

Energy for Life Walkathon – Pittsburgh

Simon Says Golf - A Round for Kids’ Hearts

June 9, 2018

July 16, 2018

Pittsburgh, PA

Plymouth Meeting, PA

rideATAXIA Blue Bell, PA

Muldoon’s Saloon

Wine Women & Shoes Alzheimer ’s Association Fundraiser

35th Anniversary Celebration Presenting the Rare Impact Awards

April 15, 2018

May 17, 2018

Greater Philadelphia Tour de Cure

Haverford, PA

Washington, D.C.

June 16, 2018

October 14, 2018

Doylestown, PA

Cocktails for the Cure April 26, 2018 Jamison, PA

For more information about these and other upcoming events, please visit http://www.lifesciencespa.org/connect/events/.

2018-2019

S TAT E BUDGET U P D AT E

The process to determine Pennsylvania’s 2018-2019 budget commenced with the address by Governor Tom Wolf in early February, to a joint meeting of the House and Senate. As this is an election year with the Governor on the ballot, as well as half of the State Senate and all the members of the State House, radical proposals were not anticipated. The Governor’s budget proposal seeks to maintain the funding levels of the Life Sciences Green Houses and the Ben Franklin Technology Partners – both important organizations for the growing life sciences community in Pennsylvania. The Governor also proposes to maintain the $55 million for the Research and Development Tax Credit from which 36 Life Sciences Pennsylvania member companies were awarded over $15 million in tax credits. As a tradable tax credit, these credits can provide needed funds to small and emerging companies. His budget proposal also sought to keep the Keystone Innovation Zones funded at $15 million. In late March, the House and Senate Appropriation hearings concluded. We continue to monitor the negotiations between the Wolf administration and the leadership in the legislature. We will use our Advocacy Day in May to reinforce the importance of the life sciences in Pennsylvania, and the role these funds and tax credits play in supporting a fragile ecosystem.

17 | T 17 H E |L STPA HE N L SEPA W SN L EETW T ESRL,E TQT1E R2, 0Q 11 8 2018

S P E C I A L W E LC O M E T O O U R Q 1 N E W M E M B E R S ALLIANCEBERNSTEIN

KORN FERRY INTERNATIONAL

AVMBIOMED, LLC

LONGFELLOW REAL ESTATE PARTNERS

CHIP DIAGNOSTICS, INC.

LUBERT-ADLER PARTNERS, LP

CLOCKWORK CHANNEL CONSULTING

LYGENESIS, INC.

DRUSOLV THERAPEUTICS, INC.

MASY BIOSERVICES

EDWARD JONES INVESTMENTS

MLP VENTURES

FINGERPAINT MARKETING

MS2 ARRAY, LLC

GLOBAL SYMMETRY

MUTCHLER LYONS, P.C.

INVITAE

OPTOVIBRONEX, LLC

KEEN360 INC.

SKYRAN BIOLOGICS, INC.

KEY BIOPHARMA PARTNERS, LLC

TRIUMVIRATE ENVIRONMENTAL, INC

KNOLL

YETHI MEDICAL SYSTEMS PVT LTD

2 018 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


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