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see Real-World Trial on page 4 »

July 29, 2019

Industry Briefs…2

Up and Coming…3

Drug & Device Pipeline News…7Twenty-seven drugs and devices have entered a new trial phase this week.

CenterWatchWeekly

By Leslie Ramsey

When the FDA shows up with inspection notice in hand, will you be able to produce the docu-

ments they request quickly and efficiently? Knowing where all your records are stored and what condition they are in is vital to inspection success, says one former FDA district director.

Even though “clinical trial monitoring records are sort of like ‘mini audits’,” says quality consultant and 23-year FDA veteran David Chesney, sites still are subject to FDA inspection.

Chesney offers several tips for making sure records the FDA wants to see are la-beled, stored, archived and easily accessible during an inspection.

Start with a consistent naming conven-tion that is unique but succinct. File names in electronic systems often are cut short, so document titles should be identifiable by only a few words or numeric designation. And whatever convention you use, train the staff that will retrieve documents to recog-nize them quickly.

Archived documents should be held in easily accessed locations or designated drives, in the case of electronic records, which also should have controls over read-ing, copying and printing.

Inventory documents by owner, Chesney advises, and periodically reconcile that inventory to make sure documents are pres-ent. And develop both a written retrieval

Easy Document Access Key to Inspection Success, Expert Says

By Sony Salzman

Trying to make a real-world data study as easy as possible for practitioners and participants requires a great deal

of work behind the scenes, experts say, but careful planning pays off.

Representatives of GlaxoSmith Kline’s Salford Lung Study (SLS) say there are unique challenges in conducting a trial that brings together both clinical research and real-world data.

The benefit of such hybrid studies is their ability to answer questions about a therapy’s safety and efficacy while simultaneously collecting information about the best way to implement that therapy in a real-world setting.

The SLS, the first major drug trial con-ducted under “real world” conditions,

evaluated GSK’s Relvar among 4,233 patients with asthma and 2,802 patients with chronic obstructive pulmonary disease (COPD). The studies were designed as open-label, Phase 3, randomized trials.

The goal of the trial was to show the benefit of a once-daily administered drug, says Elaine Irving, senior director and head of real-world study delivery at GSK, which can’t be done in a blinded scenario. GSK focused the trial on practical endpoints that physicians use in day-to-day practice, Irving says, and tried to keep the patients as close to routine care as possible. Active random-ization helped maintain scientific rigor.

The first lesson learned, Irving points out, was how many moving parts such a

Simple on the Surface, Complex Underneath: Lessons from a Real-World Trial

PipelineNews

FDA Actions

Company name Drug name Indication FDA action

Lumendi, LLC DiLumen C2 second-generation endoscopic accessory indicated to ensure complete positioning of an endoscope in the large intestine and assist with optical visualization, diagnosis and endoscopic treatment

510(k) clearance granted

Varian Calypso Anchored Beacon transponder

tumor detection 510(k) clearance granted

Conavi TM Medical Inc. Novasight Hybrid System simultaneous imaging of coronary arteries with both intravascular ultrasound (VUS) and Optical Coherence Tomography (OCT)

510(k) clearance granted

Prisyna, the oral care division of Synedgen

Moisyn product line xerostomia 510(k) clearance granted

C4 Imaging LLC HDR MRI Marker use prior to high dose rate (HDR) brachytherapy to accurately locate the position of the applicators that guide the placement of radioactive sources for the treatment of multiple cancers

510(k) clearance granted

Amerigen Pharmaceuticals Limited and Dipharma S.A.

Miglustat 100 mg capsules Adult patients with mild to moderate type 1 Gaucher disease for whom enzyme replacement therapy is not a therapeutic option

aNDA � led

Sage Therapeutics intravenous formulation of brexanolone (SAGE-547)

postpartum depression (PPD) NDA � led

Veloxis Pharmaceuticals A/S

de novo indication of ENVARSUS XR (tacrolimus extended-release tablets)

Prophylaxis of organ rejection in kidney transplant patients

sNDA � led

Jazz Pharmaceuticals Xyrem (sodium oxybate) oral solution

cataplexy and Excessive Daytime Sleepiness (EDS) in pediatric narcolepsy patients

sNDA � led

Abeona Therapeutics Inc. ABO-102 AAV-mediated gene therapy for the treatment of San� lippo syndrome Type A (MPS IIIA)

RMAT Designation granted

Immusoft Corporation Immune System Programming MPS I (Mucopolysaccharidosis type I) Orphan Drug Designation granted

Stealth Biotherapeutics elamipretide Leber’s hereditary optic neuropathy (LHON) Orphan Drug Designation granted

P� zer Inc. TRUMENBA (Meningococcal Group B Vaccine)

Active immunization to prevent invasive disease caused by Neisseria meningitides group B (MenB) in children ages 1 through 9 years

Breakthrough Therapy Designation granted

MeiraGTx Limited AAV-RPGR X-linked retinitis pigmentosa (SLRP) due to defects in the retinitis pigmentosa GTPase regulator (RPGR) gene

Fast Track Designation granted

The following is a sampling of FDA regulatory actions taken during the previous month, compiled from CenterWatch and third-party sources including the FDA and company press releases. For more information on FDA approvals, visit www.centerwatch.com/drug-information/fda-approvals/.For custom drug intelligence reports, email marketresearch@centerwatch.com. Join the LinkedIn Drug Research Updates group!

9:45 AMiPad3

Addressing Barriersto Entry and Retention

By Sony Salzman

B eing a principal investigator (PI) is tough. So tough, in fact, that more than half of new investigators give up er

thei rst FDA-regulated drug trial.Studies by the T s Center for the Study of

Drug Development (CSDD) have revealed a variety of challenges for rst-time investigators.

e rst — and some would say worst — challenge is that the path to success for PIs is murky, said Gerrit Hamre, project manager of CTTI and co-author of the group’s paper on the study. Although there are training programs and resources for investigators, most new investigators are unaware of their existence (outside of GCP training), and un-derestimate the infrastructure, st ng, bud-geting, contracting, and operational skills necessary to succeed.

e second overwhelming obstacle for edgling investigators is administra-

tive burden. Complex protocols, rigorous reporting demands and multiple technol-ogy platforms place a huge strain on inves-tigators and sta .

e third major obstacle new PIs face is the lack of accolades and/or s cient nancial reward to make all of that e ort worthwhile.

With mounting evidence that the prob-lem isn’t getting better, sponsors and CROs are rethinking the site selection process and trying to develop new ways to support inex-perienced investigators.

Sponsors and CROs are doubling-down on their top-performing sites — a trend that has helped fuel industry consolidation, says Ken Getz, director and associate professor of CSDD.

“At the same time that sponsors are look-ing for scaled, experienced sites and site networks, the CROs are buying them,” said Getz.

He predicts that in the short term, the in-dustry will continue to see the largest and most expensive sites and site networks build more share of the market.

Yet sponsors can’t rely solely on vetted academic medical centers and large com-mercial sites for all of their study partici-pants; the burgeoning elds of rare disease and oncology research in particular require them to cast a wider net for patients.

Experts agree that rst time investiga-tors can take some steps to bolster success — including nding a mentor, hiring an ex-perienced research coordinator, learning to budget appropriately and being technologi-cally savvy and platform-agnostic — but most also agree that ultimately, sponsors and CROs need to step up if they want their PIs to thrive.

“I’m sympathetic to investigators because you don’t know what you’re getting into,” says Hamre, adding, “I do think investiga-tors very en bite o more they can chew, but they don’t realize it. In that regard, I think it’s the sponsors and the CROs that have a bit more responsibility.”

Getz added that sponsors must “take ownership for site selection practices that

Industry Tries to Stem Investigator Dropout

© 2018 CenterWatch. Duplication or sharing of this publication is strictly prohibited.

CRA/CRC Shortages Slow Clinical Trial Pace

June 2018 A CenterWatch Publication Volume 25, Issue 06

see CRA/CRC on page 8

Industry DevelopingStandardized TrainingBy Daphne Butas

As clinical trials continue to change, the evolving roles of CRAs and CRCs are exacerbating the problems sponsors and

CRO nding well-trained candidates.“What’s going to really get interesting is how

much these roles are going to evolve as more new initiatives come forward, like eConsent,

ePRO and risk-based monitoring,” said Jim Kremidas, executive director of the Association of Clinical Research Professionals (ACRP). As risk-based monitoring has taken hold and more monitors are analyzing data from a central loca-tion rather than traveling to the sites, sponsors and CROs are looking for CRCs with more of a robust math background, he said. And as the sponsors’ and CROs’ relationships with sites becomes ever more important, sponsors and CROs are looking for CRAs who excel at forg-ing friendly, supportive connections with sites.

“I think we’re going to see a morphing of these role into subspecialties — a data analyst and site-relationship manager type of CRA, along with the traditional role that the CRA has always had,” said Kremidas. “With CRCs, I think we’ll begin to see a quality assurance focus, someone who oversees the input of the data into the eCRFs. I think we’ll see a tech-nology type of specialist for patients to call in order to get help logging on. And I think the traditional role will be there, too.”

see Investigator Dropout on page 6

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Industry Briefs

Sharpless Says Innovative Trial Designs Are FDA PriorityInnovative trial designs are a high priority in FDA plans, acting FDA Commissioner Ned Sharpless said in an agency-wide internal email sent Tuesday.

Sharpless told staffers the agency will continue to push for the use of platform trials, basket studies, adaptive trials and pragmatic randomized controlled trials that will save money and time on research and development. It also plans to promote the use of real-world evidence (RWE) in both pre- and postmarket adverse event detection and efficacy analyses in clinical situations that may not be able to use randomized trials.

“Enhanced use of RWE for regulatory decisionmaking will improve the generalizability of evidence to patient populations that may be excluded from traditional clinical trials,” he added.

The agency will continue to use novel ap-proval pathways for drugs, biologics and devices — including RMAT, Accelerated Approval and Breakthrough — as they can make product development more efficient, Sharpless said.

Big Pharma Scores Well Overall on Data TransparencyRoche/Genentech and Novo Nordisk tied for top honors for data transparency on Bioethics International’s latest Good Pharma Scorecard.

The two companies both scored 100 percent on the nonprofit’s annual evaluation of big pharma’s track record on trial registration, results reporting, publication and data-sharing practices. Novartis was close behind at 99 percent.

The median score of the 12 companies evaluated – based on their sharing of data on 2015 FDA-approved drugs – was a respectable 92 percent, according to the study, but three companies fell below the 70 percent mark: Valeant at 63 percent, Amgen at 56 percent and Allergan, soon to be absorbed by AbbVie, at the bottom of the list with a poor transparency score of 46 percent.

Read the full report here: https://bit.ly/2OjcaXc.

EMA Studies Patient Registries’ Use in Rare Disease Trials and Postmarket SurveillancePatient registries could provide valuable data for regulatory decisions, a new EMA drug safety study indicates.

Registries can be used when randomized controlled trials are not feasible, such as in the case of some rare diseases, or to meet postmar-keting commitments.

The study further explains how registries are currently underused and explained how the ab-sence or incompleteness of ten different factors impeded registry use. Factors include the use of common core data sets, common data coding terminologies, data access and sharing and data linkage capacity, among others.

“From a regulatory perspective, the ultimate requirement of patient registries is that they permit the conduct of high quality studies that evaluate the safety and effectiveness of medi-cines,” EMA says.

The agency plans to prepare a preliminary discussion paper and solicit public comments before issuing methodological and operational guidance on handling registry data in postmar-ket studies.

Industry, Academia Form Consortium to Develop Neurobehavioral Clinical ToolsSeveral big pharma companies have joined with European universities and an AI technology company to advance the development of clini-cal tools that measure neurobehavioral tasks.

Boehringer Ingelheim, Janssen, Lundbeck and Roche are participating in the Reward Task

Optimization Consortium (RTOC), along with central nervous system experts from medical and mental health institutions in the Nether-lands, Germany, Spain and Greece. BlackThorn Therapeutics, a clinical-stage neurobehavioral health company, will provide next-generation AI solutions to help build predictive models.

RTOC, which will be led by central nervous system research company P1vital, will develop three computerized tools that measure brain ac-tivity in trial participants and then test the tools in trials for schizophrenia and major depression therapies. RTOC expects to start recruiting par-ticipants in Europe in the third quarter of 2019.

“Invisible” Device Uses Radio Signals to Analyze Movement and Collect Patient Data Novartis and MIT are collaborating on develop-ment of a wireless device that can measure physical activity in patients without requiring them to use wearable sensors or monitors.

The Emerald device analyzes surrounding ra-dio signals, using machine learning algorithms to detect the pose and movement of a person, even through walls and in low light. It extracts various physiological metrics related to breath-ing, sleep stages, sleep apnea, mobility and gait, among others

The “invisible” device provides several benefits over traditional patient data collec-tion methods, including improved biomarker development and the ability to detect changes in behavior. And it allows patients to go about their normal routines without any added burden, which provides a unique opportunity to support at-home clinical trials.

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Up and Coming

This feature highlights changes in clinical research organizations’ personnel.

VertexVertex Pharmaceuticals has announced Reshma Kewalramani will assume the role of chief executive officer, effective April 1, 2020, when current CEO Jeffry Leiden transitions to the role of executive chairman of the board. Kewalramani currently serves as chief medical officer, a position she has held since 2017. In addition to CMO, she also serves as executive vice president of global medicines devel-opment and medical affairs. Kewalramani will become the first woman to run a large biotechnology firm.

AstellasChelsea Glinski has been appointed vice president, oncology sales at Astellas U.S. Glinski previously served as executive director of Astellas’ market access and reimbursement strategy.

HealxHealx has named Neil Thompson chief sci-entific officer. Thompson previously served as senior vice president at Astex Pharmaceuticals.

Noven PharmaceuticalsNoven Pharmaceuticals has announced the appointment of Andrew Purcell as head of marketing and sales. Purcell was previously vice president and head of the U.S. diabetes business unit for Sanofi.

Ablative SolutionsKate Rumrill has been named president and chief executive officer at Ablative Solutions. Rumrill most recently served as president and CEO of NeoSync.

Athira PharmaAthira Pharma has named Mark Litton as chief operating officer. Litton most recently served as chief business officer at Alder Bio-pharmaceuticals.

Titan MedicalChad Zaring has been named chief commer-cial officer at Titan Medical. Zaring was most recently vice president of robotics, imaging and navigation at Medtronic.

GlobalMedGlobalMed has named Robert Rennie as vice president, Europe, Middle East and Africa (EMEA). Rennie most recently served as vice president of EMEA for Atomiton.

Gritstone OncologyGritstone Oncology has named Vijay Yaban-navar executive vice president of manufactur-ing and technical operations. Yabannavar was previously vice president of global technical operations at Merck.

SelectaMassachusetts-based Selecta Biosciences has announced the appointment of Alison Schecter as chief medical officer. Schecter was formerly the global acting head of rare diseases and was responsible for leading the Niemann-Pick Disease (ASMD) project at Sanofi.

Navitor PharmaceuticalsRandy Owen has been named chief medical officer at Navitor Pharmaceuticals. Owen most recently served as CMO at Acadia Phar-maceuticals.

SelvitaKrakow, Poland-based Selvita, S.A. has named Setareh Shamsili chief medical officer. Sham-sili was previously interim CMO at AxImmune.

Aurinia PharmaceuticalsMax Donley has been promoted to execu-tive vice president of internal operations and strategy, and Glenn Schulman to senior vice president of corporate communications and investor relations at Aurinia Pharmaceuticals. Donley most recently led human resources, information technology and facilities at

Senseonics. Schulman previously led corpo-rate communications and investor relations at Achillion Pharmaceuticals.

OncoCell MDxMichigan-based OncoCell has named Kirk Wojno as chief medical officer. Wojno was formerly the chief of pathology and laboratory services and director of clinical research at Comprehensive Urology.

Karus TherapeuticsHilary McElwaine-John has been appointed chief medical officer at U.K.-based Karus Thera-peutics. McElwaine-John was previously CMO at cancer gene therapy company PsiOxus.

ImmusoftRobert Hayes has been named chief scientific officer at Immusoft. Hayes, the former head of biologics at Amgen, was most recently CSO at Phylogica.

GlaxoSmithKlineGlaxoSmithKline has announced the appoint-ment of Kim Branson as senior vice president, global head of AI and machine learning. Bran-son was previously head of AI at Genentech.

Pandion TherapeuticsPandion Therapeutics has announced that Rahul Kakkar has been named chief execu-tive officer. Kakkar most recently served as the founding executive of Corvidia Therapeutics.

Escient PharmaceuticalsEscient Pharmaceuticals has named Kristin Taylor as vice president and head of clinical development. Taylor most recently held the same role and helped lead efforts on therapies for Type 2 diabetes and obesity at Zafgen.

AgilVaxJoseph Patti has been named president and chief executive officer at AgilVax. Patti was most recently president of JP Biotech Advisors.

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Features

Real-World Trial continued from page 1

“These types of studies can’t be done unless you have a partnership with all the different parties.”

—Elaine Irving, senior director and head of real-world study delivery, GlaxoSmithKline

seemingly simple study can have. “The simpler we make the concept,” she says, “the more complicated it is behind the scenes and the longer it takes to get one of these studies actually started.”

“I think this study took four years of dis-cussion and planning before we hit the first subject to be recruited.” The sheer numbers of stakeholders in the asthma study alone — 74 physician practices, 132 community pharmacies, 165 trainers and facilitators and 4,233 patients — meant determining everyone’s needs was heavy lifting.

But buy-in is essential, Irving says. “These types of studies can’t be done unless you have a partnership with all the different par-ties.” For the SLS, this meant reaching into all corners of the community in Salford and South Manchester, England, to gather local hospitals, primary care doctors, academic experts and health informatics specialists. In addition, SLS coordinators kept national regulators up to date.

And keeping eligibility standards broad resulted in a more representative study population. Instead of employing tradition-ally rigid clinical trial inclusion criteria, the SLS tried to reach out to everyone in the community with asthma and COPD, even smokers and people with other comorbid conditions.

In the COPD study, 11,720 patients reg-istered to participate at their local doctor’s office, 5,658 were found to be eligible, and 2,802 — 50 percent — were enrolled, a far cry from the 841 patients who would have qualified under more traditional clinical trial standards.

Support for the physicians involved also was a key factor. Trial designers wanted to place as little burden on them as possible. “If we had the design right,” Irving says, “we were asking [them] to do their jobs. We weren’t asking much else.” The SLS provided nurse support teams to take on extra tasks

related to the trial, such as identifying patients, conducting study visits and data entry.

Training also was a necessary compo-nent. These were research-naïve medical professionals, Irving points out, who needed education in good clinical practices and trial procedures. Local pharmacies also needed

training in handling investigational drugs and good manufacturing practices.

It was also important to make the study team as diverse as possible, Irving says, com-bining clinical statisticians used to working only with eCRF data with epidemiologists who specialize in real-world practice. “That involves bringing two completely different worlds together,” she says.

Using real-world data also requires a change in mindset, Irving stresses. Data was collected from patients only when it didn’t in-terfere with their regular care, and then only when the data added value to the study.

Outcomes and safety data were col-lected from the electronic record systems at general practices and pharmacies. That presented a challenge all its own, according to Martin Gibson, University of Manchester professor and head of the team that created the technology behind the study.

When it comes to electronic data records, “standards are like toothbrushes — every-one’s got one, but nobody wants to share,” Gibson says. And varied data sources in an RWD study make standardizing difficult. They may have different coding systems, data may be duplicated, sources may be difficult to query.

In a real-world study, it’s necessary to either build or buy “a system that brings all those different data standards together,” he says.

The outcome of GSK’s efforts ultimately was positive. The SLS demonstrated not only that Relvar was safe and effective, but also that it improved patient outcomes in a real-world setting, a factor that may strengthen the drug’s position among payers.

Recruiting a ‘typical’ population — keeping it ‘real’

Source: Salford Lung Studies

SLS patients eligible for inclusion

in FF/VI exacerbation studies

COPD patients enrolled in SLS

COPD patients eligible for

inclusion in SLS

COPD patients registered

at SLS practices (Salford and

South Manchester area)

11,720

5,658

2,802

841

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WCG | CWWeekly July 29, 2019 5 of 8

Features

Easy Document Accesscontinued from page 1 “The challenge is to meet

the FDA requests in a timely and complete manner.”

—David Chesney, DL Chesney Consulting

plan and a written strategy for processing inspection requests. It’s helpful, he says, to create a document matrix that shows the assigned document owners for each type of record likely to be requested.

For clues on what documents the FDA is likely to request, consult the FDA’s Compli-ance Program 7348.810, which provides agency personnel with instructions for conducting inspections.

“The challenge,” Chesney says, “is to meet the FDA requests in a timely and complete manner.” To evaluate your ability to do that, he suggests asking the following 10 ques-tions:1. Do your archives contain everything that may be requested during an inspection?2. Are file names clear (ideally self-explana-tory)?

3. Are there written requirements for in-house, remote or contracted employees and third-party vendors to transfer materials into the archives?4. Do the archives contain more than one original and, if so, how are they differentiated?5. Do the archives contain draft documents and, if so, how are they differentiated from final versions?6. If archiving has been contracted to a third party, does the contract allow for prompt access for the records owner?7. Are records produced by other parties for your company readily accessible (for

example, CROs, contractors, consultants)?8. Can documents be retrieved in a prompt and effective manner?9. Have the access security measures associ-ated with the archives been tested?10. Do personnel responsible for the archives have the access and resources needed to produce documents during an inspection?

Chesney recommends testing document retrieval capabilities during mock inspec-tions and internal audits, tracking how long it takes to properly fulfill a document re-quest and working to improve that response time.

Finally, make sure to assign and train personnel who have first-hand knowledge of the documents to explain them during an inspection.

Read Compliance Program 7348.810 here: https://bit.ly/2YqimR5.

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Drug & Device Pipeline News

Company Drug/Device Medical Condition Status Sponsor Contact

Ascentage Pharma APG-2575 hematologic malignancies Phase 1 trial initiated enrolling subjects with acute myelogenous leukemia (AML) and non-Hodgkin's lymphoma (NHL) in China

ascentagepharma.com

Halozyme Therapeutics

efgartigimod (ARGX-113)

severe autoimmune diseases Phase 1 trial initiated halozyme.com

AC Immune SA ACI-3024 neurodegenerative diseases that are characterized by the presence of pathological Tau aggregates

Phase 1 trial initiated enrolling healthy volunteers

acimmune.com

NuCana plc NUC-7738 cancer Phase 1 trial initiated nucana.com

CStone Pharmaceuticals

ivosidenib (TIBSOVO)

relapsed or refractory acute myeloid leukemia (R/R AML) with an IDH1 mutation

Phase 1 trial initiated cstonepharma.com/en/

Prescient Therapeutics Limited

PTX-100 cancer Phase 1b trial initiated enrolling subjects in Australia

ptxtherapeutics.com

Axial Biotherapeutics

AB-2004 gastrointestinal dysfunction and associated behavioral symptoms of Autism Spectrum Disorder (ASD)

Phase 1b/2a trial initiated enrolling 25 male subjects with ASD

axialbiotherapeutics.com

ImaginAb Inc. CD8 tracer 89Zr-Df-IAB22M2C

cancer Phase 2 trial initiated enrolling subjects at Dana-Farber Cancer Institute in Boston

imaginab.com

DelMar Pharmaceuticals, Inc.

VAL-083 MGMT-unmethylated glioblastoma multiforme (GBM)

Phase 2 trial initiated enrolling 24 newly-diagnosed subjects who have undergone surgery and chemoradiation with temozolomide (TMZ) at the University of Texas MD Anderson Cancer Center (MDACC)

delmarpharma.com

EIP Pharma, Inc. neflamapimod cognitive dysfunction associated with dementia with Lewy bodies (DLB)

Phase 2 trial initiated enrolling 80 subjects at several sites in the U.S. and Netherlands

eippharma.com

Oyster Point Pharma, Inc.

OC-01 Nasal Spray dry eye disease Phase 3 trial initiated enrolling 750 subjects at 20 centers in the U.S.

oysterpointrx.com

LifeMax Laboratories, Inc.

LM-030 Netherton Syndrome Rare Pediatric Disease designation granted by the FDA

lifemaxlabs.com

Merck and Eisai KEYTRUDA in combination with LENVIMA

subjects with advanced unresectable hepatocellular carcinoma (HCC) not amenable to locoregional treatment

Breakthrough Therapy designation granted by the FDA

merck.comeisai.com

continues on next page »

For news on trial results, FDA approvals and drugs in development, Join the LinkedIn Drug Research Updates group!

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WCG | CWWeekly July 29, 2019 8 of 8

Drug & Device Pipeline News (continued from page 7)

Company Drug/Device Medical Condition Status Sponsor Contact

LivaNova PLC LifeSPARC Advanced Circulatory Support (ACS) system

cardiac and respiratory failure 510(k) clearance granted by the FDA

livanova.com

Aridis Pharmaceuticals, Inc.

AR-501 cystic fibrosis Orphan Drug designation granted by the FDA

aridispharma.com

Arrowhead Pharmaceuticals Inc.

ARO-ANG3 homozygous familial hypercholesterolemia (HoFH)

Orphan Drug designation granted by the FDA

arrowheadpharma.com

Apic Bio, Inc. APB-102 genetic SOD1 amyotrophic lateral sclerosis (ALS)

Orphan Drug designation granted by the FDA

apic-bio.com

Teva Pharmaceuticals Industries Ltd.

AirDuo Digihaler (fluticasone propionate 113 mcg and salmeterol 14 mcg) Inhalation Powder

asthma Approval granted by the FDA tevapharm.com

Novadoz Pharmaceuticals

Abiraterone Acetate 250mg tablet

prostate cancer when used with a steroid medication (prednisone or methylprednisolone)

Approval granted by the FDA novadozpharma.com

Bayer Gadavist (gadobutrol) injection

coronary artery disease (CAD) Approval granted by the FDA bayer.com

Abbott MitraClip heart valve repair device

mitral regurgitation Approval granted by the FDA abbott.com

Merck RECARBRIO (imipenem, cilastatin and relebactam) for injection (1.25 grams)

adults with complicated urinary tract and complicated intra-abdominal bacterial infections where limited or no alternative treatment options are available

Approval granted by the FDA merck.com

Celgene OTEZLA (apremilast)

adult with oral ulcers associated with Behçet’s Disease

Approval granted by the FDA celgene.com

Baxter Myxredlin ready-to-use insulin for IV infusion Approval granted by the FDA baxter.com

Pfizer, Inc. RUXIENCE (rituximab-pvvr)

adult subjects with non-Hodgkin’s lymphoma (NHL), chronic lymphocytic leukemia (CLL) and granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA)

Approval granted by the FDA pfizer.com

Samsung Bioepis Co., Ltd.

HADLIMA (adalimumab-bwwd)

rheumatoid arthritis, juvenile idiopathic arthritis, psoriatic arthritis, ankylosing spondylitis, adult Crohn’s disease, ulcerative colitis and plaque psoriasis

Approval granted by the FDA samsungbioepis.com

Eli Lilly and Company

Baqsimi (glucagon) nasal powder 3 mg

severe hypoglycemia Approval granted by the FDA lilly.com