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see Improving Clinical Site Payments on page 5 »

see New Guidance on page 4 »

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

The CenterWatch Monthly provides in-depthand data-rich insights on key trends impactingthe clinical resarch landscape.

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July 23, 2018

Industry Briefs…2-3

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

Trial Results…8CenterWatch reports on results for four drugs.

CWMarketPlace…9 Industry service provider profiles.

P aying sites on time is critical to a trial’s success, sponsors agree, and most believe they’re making the grade. So

why are sites so frustrated with how they’re paid? Are their expectations unreasonable or are sponsors operating in the dark?

A recent survey by Metrics Champion Consortium (MCC) indicates the latter is true. More than 70 percent of trial sponsors who manage in-house payment procedures said they’re pleased with their processes; ditto 65 percent of CROs. But sites were overwhelm-ingly dissatisfied, with 70 percent expressing a negative view.

So what’s the deal? The survey shows sponsors don’t pay at-

tention to the kind of metrics that can help

them solve payment woes. They may know they’re not ponying up promptly but they’re looking for answers in all the wrong places – if at all.

There are many details and data points that can help sponsors figure out how well their payment processes are (or aren’t) working, says Linda Sullivan, MCC’s executive director.

MCC discovered that sites aren’t the only ones unhappy. Sponsors that outsource payment to other entities (like CROs) have less control over the process and often have less insight into how it is working on the contractor’s end.

“Those who are running the process – a sponsor [managing payments] in house or a

New Report: Improving Clinical Site Payment Practices

By William Myers

The FDA has issued new guidelines designed to help researchers navi-gate sometimes conflicting regional

requirements and differences in global clinical trials in an effort to pave the way for better cooperation and faster international drug development.

There currently is no single set of rules governing how multiregional clinical trials (MRCTs) should be crafted. But the FDA is hoping to help make the road less bumpy with this fresh ICH E17 addendum.

Among its key recommendations: Plan ahead to make sure an international study is the best route to take. That is, assess poten-tial challenges and regional differences —

like environment, diet, cultural twists — to make sure they’re not too great to overcome or skew findings.

Consider all potential factors —and ways to account or adjust for them (by doing ge-netic testing, for example), the FDA advises, noting that “even in the case of expected major differences ... it may still be possible to conduct MRCTs by excluding some regions or a defined subgroup within a region.”

Your best bet for smooth sailing if you do opt for international probes?

Design them with seven principles in mind, the FDA suggests:

}} Identify early how different regional fac-tors can affect drug trials.

New Guidance for Multiregional Clinical Trials

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Industry Briefs

FDA Issues New Guides on Use of Electronic Health Information in Clinical Trials The FDA is urging researchers to work with electronic health record keepers to help improve clinical trial accuracy and efficiency. The agency doesn’t typically regulate companies or organizations that maintain electronic medical records (EMRs). But it says it’s weighing in to highlight and clarify ways electronic health record (EHR) systems can help clinical trials run more smoothly and use data from foreign studies conducted outside investigational new drug applica-tions and device exemptions. In guidelines released July 18, the FDA encourages spon-sors and investigators to use approved EHR or electronic capture data capture (EDC) systems to exchange key information, not-ing that doing so can dramatically speed up information sharing and precision.

Other FDA tips:

}} Sponsors and investigators should use EHR systems endorsed by the Office of the National Coordinator for Health Information Technology (ONC). If they lack access to ONC-certified systems, they should make sure security mea-sures are in place to protect study data.

}} Study monitors should have easy access to “all relevant information” de-tailed in consent forms, including how long identifying records will be kept. (Federal law requires investigators to keep records from drug or human biological product trials for at least two years after marketing applications have been approved or trials have been scrapped and the FDA notified. Records relating to medical devices must be retained for at least two years after an investigation wraps up or after it’s determined they are no longer needed to support premarket approval applications or notices of completion.)

Read the FDA’s guidance here: https://www.fdanews.com/07-18-18-HealthRecordData.pdf.

AbbVie Blood Cancer Drug Fails Phase III Trial AbbVie has announced that ibrutinib doesn’t appear to enhance benefits for blood cancer patients being treated with the R-CHOP che-motherapy cocktail. Researchers at AbbVie subsidiary Pharmacyclics and Janssen Biotech had hoped that adding ibrutinib to R-CHOP — a mix of rituximab, cyclophosphamide, doxorubicin, vincristine and prednisone — would improve outcomes for patients with untreated diffuse large B-cell lymphoma. But a Phase III study found it was no more effec-tive than a placebo at improving event-free survival. Ibrutinib has been available in the U.S. since 2013. It’s approved for use in five different B-cell blood cancers and chronic graft-versus-host-disease.

NCI, VA Boost Veteran Access to Clinical Trials The National Cancer Institute (NCI) and De-partment of Veterans Affairs are teaming up to make it easier for vets with cancer to get into clinical trials testing novel cancer treat-ments. The NCI and VA Interagency Group to Accelerate Trials Enrollment (NAVIGATE) is rolling out in a dozen VA centers around the country, boosting the ability of veterans to participate in NCI-sponsored trials and re-ceive promising new treatments locally. “VA centers often face challenges initiating and completing externally-funded trials because of the need for partners to navigate the sys-tem,” the agencies said. “This program aims

to overcome these challenges with dedi-cated staffing and a sustainable infrastruc-ture, and to address existing barriers to trial enrollment that veterans, including minority patients, often experience.” NAVIGATE is be-ing launched in VA centers in Atlanta; New York; Charleston, South Carolina; Denver; Durham, North Carolina; Hines, Illinois; Long Beach, California; Minneapolis; Palo Alto, California; Portland; San Antonio and West Haven, Connecticut.

Finnish Company Opens Phase III Trial of ALS Drug Finnish drug company Orion has recruited its first patients for a Phase III trial to see if orally administered levosimendan can help ease ALS symptoms. Some 450 patients with the neurodegenerative disease Amyotropic Lateral Sclerosis are set to participate in placebo-controlled trials at clinical sites in Europe, North America and Australia, the company announced last week. Orion said it plans to spend about 60 million euros (nearly $70 million) over the next three years to support its levosimendan research. ALS, also known as Lou Gehrig’s Disease after the New York Yankees’ beloved slugger who died from the illness, causes rapidly progressive muscle weakness. It specifically affects nerve cells (motor neurons) that control muscles that help us move, speak, breathe and swallow. There’s no cure for ALS but Orion researchers hope levosimendan — which helps strength-en respiratory muscle function — may help slow disease progression and keep patients breathing longer on their own.

see Industry Briefs on page 3 »

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WCG | CWWeekly July 23, 2018 3 of 9

Industry Briefs (continued from page 2)

FDA Hopeful New Extended-Release Opioids Will Help Curb Addiction The FDA has released more than two dozen new product-specific draft guidances for generic drug trial designs and revised 17 others in a move aimed at getting less expensive drugs to market faster. The proposed guidelines cover a range of drugs from antibiotics such as ciprofloxacin to the antiretroviral drug ritonavir — and shed light on the FDA’s view of three different opioids (two of them extended release and a third that combines morphine with the opioid antagonist naltrexone) using so-called abuse-deterrent formulations. “We recognize that the science of abuse deter-rence is relatively new and we need to continue to study and confirm the potential role of ADFs in reducing the rate of misuse and abuse via different routes when used in a population,” FDA Commissioner Scott Gottlieb said in announcing the new proposals. “Both the formulation tech-nologies and the methods for evaluating those technologies are rapidly evolving. We believe that transitioning from the current market, dominated by conventional opioid analgesics, to one where most opioids have abuse-deterrent properties may have the potential to further reduce misuse and abuse.” Two of the revised opioid propos-als deal with extended-release hydrocodone bitartrate (the generic name for Vicodin, among others) and extended-release oxycodone HCL tablets. Both encourage researchers to conduct two sets of studies (involving participants who either fast or eat before taking pills) to determine if the meds can help patients resist addiction: one in which participants chew pills and another in which they absorb the drugs through a nasal

spray. The guidance urges researchers not to use naltrexone or other opioid antagonists in the tri-als. The third revised draft focuses on an extend-ed-release capsule containing morphine sulfate and naltrexone hydrochloride. It urges research-ers to conduct three bioequivalence studies: one in which participants fast before doses, another in which they eat first and a third in which fasting subjects take the drug sprinkled into applesauce. It also encourages investigators to conduct a pair of risk-abuse trials. Gottlieb said he’s optimistic the opioid tests will yield medicines that “can’t be easily abused” but cautioned it’s important to remember that no opioid is “abuse-proof.”

Feds to IRBs: Drop Grant Application Reviews It’s not necessary for IRBs to review grant pro-posals or applications to ensure patient safety, the HHS says in a new draft guidance. “Experi-ence suggests that review and approval of the application or proposal is not a productive use of IRB time,” say the proposals issued by HHS’s Office for Human Research Protection. Federal officials say the change won’t increase patient risks because IRBs will still have to review research protocols for any studies that seek federal funding. Read the draft guidance here: www.fdanews.com/07-23-18-IRB.pdf.

FDA Oks Breast Cancer Drug after Successful Trials Pilot The FDA has greenlighted anti-breast cancer drug Kisqali, making it the first drug approved under a new pilot program designed to help bring cancer drugs to the market more quickly. The agency OK’d the use of Kisqali in combination

with aromatase inhibitors to treat women with hormone receptor-positive, human epidermal growth factor receptor 2-negative advanced or metastatic cancer. It also approved using Kisqali with fulvestrant for postmenopausal women with the same cancers. The go-ahead gives Kisquali “the broadest first-line indications” of any drug in its class, drug maker Novartis said in a statement. The move comes less than a month after the company submitted its application for the FDA’s approval. The FDA credited the fast turnaround to two pilot programs it launched earlier this year — one in which regulators are given “real-time” review of data from oncology trials and another in which applicants work from a single template.

HHS Mulls Waiving Continuing IRB Review Rules Federal officials are considering waiving rules that require continuing IRB review of a small sample of expedited and late-stage clinical trials. New regulations easing IRB review requirement are scheduled to take effect in January. But HHS’s Office for Human Research Protection last week issued a draft guidance to address studies pending the change. If the FDA adopts the recommendations, IRBs won’t have to continue monitoring trials in transition that are eligible for expedited federal review or in which data analysis is complete and participants are already in follow-up treatment. Nothing in the pending rules or the draft guidance exempts researchers from reporting problems or patient risks uncovered during studies. Read the draft guidance here: www.fdanews.com/07-23-18-ContinuingReview.pdf.

The SOP for Good Clinical Practice by Sponsors of Clinical Trials ensures safe, effective and successful clinical trials and is a comprehensive customizable and easy-to-use SOP.

SOP Highlights inlcude:n FDA guidance documentsn Risk-based monitoring templatesn CAPA plan information and formsn Site selection and feasibility proceduresn Site initiation visit and training guidelines

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Compliance for Sponsors

New Guidance (continued from page 1)

WCG | CWWeekly July 23, 2018 4 of 9

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Your best bet for smooth sailing — design global trials with seven

key principles in mind.

}} Clear communication between spon-sors, sites and regulators is a must.

}} Make sure study designs meet all relevant international quality rules for trial planning, investigator training and monitoring.

}} Stay focused on a single analysis so trials pass regulatory muster across regions.

}} Make sure treatment is consistent across the aboard.

}} Pre-plan pooling regions or subpopula-tions to provide flexibility in sample sizes.

}} Strategically use trials to up drug devel-opment efficiency.

Multiregional trials have historically been used as a quick way to recruit participants with rare diseases or in special populations (like children or the elderly) or for large-scale

studies (such as vaccine safety and effective-ness). But sponsors increasingly have found they’re an efficient way to get more drugs to more people — and they’re fast becoming the preferred choice for investigating new meds in today’s global market.

The difficulty is that disease definitions, diagnostic methods, medical practices, diet and other factors may vary from region to region, complicating global trials. Given all the potential differences at play, it’s vital to create an ongoing system of quality checks.

“Centralized and risk-based monitor-ing may be particularly useful for MRCTs to

monitor and mitigate the impact of emerging regional difference in, for example, trial subject retention or adverse event reporting,” the FDA says. “Timely and accurate flow of information should occur between the sponsor, the trial management team and the participating sites.”

If investigators take the recommended steps and still notice different regional effects, the FDA suggests conducting “a structured exploration” to try to pin down potential culprits.

During their probe, researchers should consider the usual suspects first – things like disease severity and participants’ race, weight and lifestyle (smoker/nonsmoker, for instance). If that fails to turn up answers, the FDA says, they may need to dig deeper —and perhaps cull data from other clinical trials and sources.

Read the FDA’s guidance here: www.fdanews.com/07-18-18-ClinicalTrials.pdf.

I centerwatch.com

Gain the competitive edgewith unique industry insightAccess to independent, insightful market intelligence and analysis is critical in order to make more informed, strategic business decisions. CenterWatch’s Market Research Intelligence Services assists organizations with developing, evaluating and executing business concepts to maximize performance and address operating challenges.

Commission a custom report today: • Study performance and relationship feedback surveys

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Improving Clinical Site Payments (continued from page 1)

WCG | CWWeekly July 23, 2018 5 of 9

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CRO – think the process is going a lot better than those who are outside of the process,” Sullivan says.

One reason CROs like the process may be the fact that they use more metrics – nearly twice as many as sponsors – to evaluate it.

Understanding how well a process is working requires knowing all aspects of its operations. Tracking a simple metric of how many payments were made “on time” provides no information on why the other payments were not.

Fewer than 50 percent of survey respon-dents said they use the kind of metrics that provide insight into the root cause(s) of pay-ment problems, such as the number of:

}} Invoices submitted by sites with errors;}} Payments that are correct the first time;}} Outstanding invoices with queries;}} Days it takes to reconcile invoice discrep-

ancies; and}} Site complaints about payment problems.

This kind of information allows payers to pinpoint areas that need improvement. Sponsors that manage their own payment processes should begin using such metrics; those that outsource payments should make sure their CROs do, too, and require them to share the information.

Sullivan says to truly grasp and fix pay-ment problems, sponsors must shift their focus from overall performance to “quality metrics.” For example, a large number of invoices outstanding may signal glitches in the invoice reconciliation phase. Track-ing the number and nature of payment complaints from sites paints a picture of the depth of the problem.

The survey recommends replacing out-moded clunky manual payment systems with

speedy automated ones to eliminate time-consuming and error-prone procedures.

Another tip: Parties involved need to communicate more effectively to align expectations, improve transparency and develop solutions.

For more information on the MCC report, Improving Site Payment Practices, go to http://store.centerwatch.com/p-563-im-proving-clinical-site-payment-practices.aspx.

Satisfaction with the Current Payment Model, by Organization Type

Source: Metrics Champion Consortium

DissatisfiedNeither Satisfiednor Dissatisfied

Satisfied

72%

29%

65%

20%7% 7% 7% 10%

21%

65%

28%

70%

Sponsors that manage the payment process internally (N=14)

Sites (N=10)

Sponsors that outsource the payment process to a CRA (N=14)CROs (N=14)

Clinical study leads and business opportunities

Research Center Profile9:45 AMiPad3

Clinical Research of WestFloriday, Inc.

Therapeutic AreasTherapeutic Areas

Cardiology/Vascular DiseasesDermatologyDevicesEndocrinologyGastroenterologyHealthy VolunteersImmunologyInfections and Infectious DiseasesInternal MedicineMusculoskeletalNeurologyObstetrics/Gynecology (Women’s Health)OphthalmologyOrthopedics/Orthopedic SurgeryPharmacology/ToxicologyPsychiatry/PsychologyPulmonary/Respiratory DiseasesRheumatologyUrology

Aydin KeskinerPresidentClinical Research of West Florida, Inc.2147 NE Coachman RoadClearwater, FL 33765-2616USA727-466-0078727-461-7793 (fax)research@crwf.comwww.crwf.comView Map

Contact Center

Currently Enrolling Trials

• Are you a current or former smoker? • Are you one of 5 million living with Lupus? • Crohn's Study • Cutaneous Lupus Study[Show More]

Clinical Research of WestFloriday, Inc.

Therapeutic AreasAydin KeskinerPresidentClinical Research of West Florida, Inc.2147 NE Coachman RoadClearwater, FL 33765-2616USA727-466-0078727-461-7793 (fax)research@crwf.comwww.crwf.comView Map

Contact Center

Clinical Research of West Florida, Inc.

Center Information

Aydin KeskinerPresidentClinical Research of West Florida, Inc.2147 NE Coachman RoadClearwater, FL 33765-2616USA727-466-0078727-461-7793 (fax)research@crwf.comwww.crwf.comView Map

Contact Center

Cardiology/Vascular DiseasesDermatologyDevicesEndocrinologyGastroenterologyHealthy VolunteersImmunologyInfections and Infectious DiseasesInternal MedicineMusculoskeletalNeurologyObstetrics/Gynecology (Women’s Health)OphthalmologyOrthopedics/Orthopedic SurgeryPharmacology/ToxicologyPsychiatry/PsychologyPulmonary/Respiratory DiseasesRheumatologyUrology

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WCG | CWWeekly July 23, 2018 7 of 9

Drug & Device Pipeline News

Company Drug/Device Medical Condition Status Sponsor ContactBlade Therapeutics BLD-2660 debilitating fibrotic diseases Phase I trial initiated blademed.comSOM Biotech SOM3355 chorea movements associated

with Huntington’s diseasePhase II trial initiated enrolling 30 subjects

sombiotech.com

Concentric Analgesics, Inc.

CA-008 post-surgical pain Phase II trial initiated concentricanalgesics.com

Ironwood Pharmaceuticals, Inc.

linaclotide irritable bowel syndrome with constipation (IBS-C)

Phase IIIb trial initiated enrolling 600 adult IBS-C subjects in the U.S.

ironwoodpharma.com

RDD Pharma RDD-0315 fecal incontinence in subjects with spinal cord injury

IND clearance granted by the FDA

rddpharma.com

MaxCyte MCY-M11 (chimeric antigen receptor (CAR) therapeutic candidate)

solid tumors IND clearance granted by the FDA

maxcyte.com

Stryker Corporation Surpass Streamline Flow Diverter

unruptured large and giant wide neck intracranial aneurysms

PMA granted by the FDA stryker.com

Astellas Pharma Inc. and Pfizer Inc.

XTANDI (enzalutamide)

castration-resistant prostate cancer, including men with non-metastatic CRPC

sNDA approval granted by the FDA

astellas.uspfizer.com

Adaptiiv 3D Bolus Software cancer 510(k) clearance granted by the FDA

adaptiiv.com

Zavation Normandy VBR System (Cervical and Thoracolumbar Expandable Corpectomy Cage)

cervical spine (C2-C7) and thoracolumbar spine (T1-L5) for partial or total replacement of a diseased, collapsed, damaged or unstable vertebral body

510(k) clearance granted by the FDA

zavation.com

Quidel Corporation Solana Bordetella Complete Molecular Diagnostic Assay

detection of Pertussis (whooping cough) Parapertussis Infections

510(k) clearance granted by the FDA

quidel.com

Genentech TECENTRIQ (atezolizumab) in combination with Avastin (bevacizumab)

chronic lymphocytic leukemia Breakthrough Therapy Designation granted by the FDA

gene.com

Gamida Cell NiCord hematopoietic stem cell transplantation (HSCT)

Orphan Drug Designation granted by the FDA

gamida-cell.com

Inozyme Pharma INZ-701 ENPP1 Deficiency (GACI in infants and ARHR2 post-infancy)

Orphan Drug Designation granted by the FDA

inozyme.com

Vallum Corporation polyetheretherketone (PEEK) spinal interbody fusion device

spinal fusion Clearance granted by the FDA vallumcorp.com

SIGA Technologies Inc. and U.S. Department of Health and Human Services’ BARDA

TPOXX (tecovirimat) smallpox Approval granted by the FDA siga.com

Janssen Pharmaceutical Companies of Johnson & Johnson

SYMTUZA darunavir-based single-tablet regimen (STR) for the treatment of human immunodeficiency virus type 1 (HIV-1) in treatment-naïve and certain virologically suppressed adults

Approval granted by the FDA janssen.com/us/

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Trial Results

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Viking Therapeutics Announces VK5211 Phase II Study Results Viking Therapeutics announced results from the company’s Phase II study of VK5211 in pa-tients recovering from hip fracture. The Phase II clinical trial was a randomized, double-blind, placebo-controlled, parallel group, international study designed to evaluate the efficacy, safety and tolerability of VK5211 in patients recovering from hip fracture surgery. The top-line data from this study showed that the trial successfully achieved its primary efficacy endpoint, demonstrating statistically significant, dose dependent increases in lean body mass and less head, among patients treated with VK5211, as compared to placebo. The study also achieved important secondary endpoints, demonstrating statistically sig-nificant improvements in appendicular lean body mass and total lean body mass com-pared to placebo. Patients receiving VK5211 also demonstrated numerical improvements in certain exploratory assessments of func-tional performance. VK5211 demonstrated encouraging safety and tolerability, with no drug-related serious adverse events reported in this study.

Ironwood Pharmaceuticals Initiates Phase IIIb Study of Linaclotide for Irritable Bowel Syndrome Ironwood Pharmaceuticals announced the initiation of a Phase IIIb clinical trial evaluating the efficacy and safety of linaclotide 290 mcg on multiple abdominal symptoms in addition to pain, including bloating and discomfort, in adult patients with irritable bowel syndrome with constipation (IBS-C). The randomized, double-blind, placebo-controlled, parallel-group study aims to enroll approximately 600 adult IBS-C patients in the United States. Eligible patients will be randomized to pla-cebo or linaclotide 290 mcg once daily for 12 weeks, followed by a four-week randomized withdrawal period. The primary efficacy end-point is a change from baseline in abdominal

score based on daily patient assessments of abdominal bloating, discomfort, and pain at their worst, as reported on an 11-point numerical rating scale. Additional endpoints include change from baseline in spontaneous bowel movement (SBM) frequency, complete spontaneous bowel movement (CSBM) fre-quency, stool consistency and straining.

Pfizer and Lilly Announce Positive Top-Line Results from Phase III Trial Pfizer and Eli Lilly and Company announced that a 16-week Phase III study in patients with osteoarthritis (OA) pain evaluating subcutaneous administration of tanezumab, an investigational humanized monoclonal antibody, met all three co-primary endpoints. The study demonstrated that patients who received two doses of tanezumab separated by eight weeks experienced a statistically significant improvement in pain, physical function and the patients’ overall assessment of their OA, compared to those receiving placebo. Preliminary safety data showed that tanezumab was generally well tolerated, with approximately 1 percent of patients discontinuing treatment due to adverse events. Rapidly progressive osteoarthritis was observed in tanezumab-treated patients at a frequency of less than 1.5 percent, and was not observed in the placebo arm. The Phase III OA study (A4091056) was a 16-week ran-domized, double-blind, placebo-controlled, multicenter, parallel-group trial evaluating the efficacy and safety of subcutaneous ad-ministration of tanezumab compared to pla-cebo in patients with OA of the knee or hip. The trial included a 24-week safety follow-up period. In the study, patients were enrolled with moderate to severe OA pain who had experienced inadequate pain relief with other treatment options for OA pain or were unable to take other pain medications. A total of 698 patients were randomized to three treat-ment groups in a 1:1:1 ratio to receive two injections over the 16-week study, once every

eight weeks. One group received two doses of placebo, the second group received two doses of tanezumab 2.5 mg, and the third group received one dose of tanezumab 2.5 mg followed by one dose of tanezumab 5 mg eight weeks later. The efficacy of tanezumab vs. placebo was measured by changes from baseline at 16 weeks.

Phase III Data Show Vedolizumab Meets Primary Endpoint Takeda Pharmaceutical Company Limited announced top-line results from the VISIBLE 1 clinical trial evaluating the efficacy and safety of an investigational subcutaneous (SC) formulation of vedolizumab for maintenance therapy in adult patients with moderately to severely active ulcerative colitis (UC)/ VISIBLE 1 is a pivotal Phase III, randomized, double-dummy, double-blind, placebo-controlled study with a vedolizumab IV reference arm, to evaluate the safety and efficacy of an inves-tigational SC formulation of vedolizumab for adult patients with moderately to severely ac-tive UC who have achieved clinical response at week six following two doses of open-label vedolizumab IV therapy at weeks zero and two.The study enrolled 384 patients, all of whom had inadequate response with, loss of response to, or intolerance to corticosteroids, immunomodulators or tumor necrosis factor-alpha (TNFα)-antagonist therapy prior to being enrolled. Patients who achieved clinical response at week six were randomized into one of three treatment groups, vedolizumab SC 108 mg and placebo IV, vedolizumab IV 300 mg and placebo SC or placebo SC and placebo IV. In the primary endpoint of the trial, a statistically significant proportion of patients receiving vedolizumab SC beginning at week six and every two weeks follow-ing achieved clinical remission at week 52 compared to placebo. The safety data were consistent with the known safety profile of vedolizumab, and no new safety signals were identified.

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WCG | CWWeekly July 23, 2018 9 of 9

CWMarketPlace

CWMarketPlace is a monthly section featuring a range of clinical research service providers who have Industry Provider Profile pages posted on CenterWatch.com. Included in their annual subscriptions, company profiles are randomly selected to appear in this section, providing added exposure for their products and services. To learn more about becoming an Industry Provider Profile page subscriber, contact Sales at (617) 948-5100 or sales@centerwatch.com.

Click on any provider to view the company’s complete online profile or click here to search more profiles.

Q-Pharm Herston , Queensland, Australia+61487166677enquiries@qpharm.com.au

Q-Pharm is a state-of-the-art, early-phase clinical trials company providing a broad range of high-quality services for commercial and academic clients from around the world.

Juno Clinical Research Services LLP Houston, TX(713) 882-6857miguel_posada@junoresearch.us

Founded in 2004, Juno Research is a fully dedicated research management organization offering phase I-IV project management or “a-la-carte” services.

Criterium, Inc. Saratoga Springs, NY (518) 583-0095rkschnel@criteriuminc.com

Founded in 1991, Criterium offers a mix of clinical research services, real-time data acquisition and management and personalized communication processes.

Emericlin Scotch Plains, NJ(908) 603-1316dberk@emericlin.com

Emericlin is a project-based staffing company with over 20 years of experi-ence that strives to match your business needs with the CRA’s that best suit your project, budget requirements, timelines and individual personalities.

Confidence Pharmaceutical Research Russia+1 401 965-3377anna.ravdel@confidenceresearch.com

With a 25 year history conducting clinical research, Confidence is a CRO, a vendor management organization and a concierge service.

Splash Clinical Wauwatosa, WI (414) 443-3280matt@splashclinical.com

An innovative patient recruitment firm that’s pioneered the use of digital & social media to recruit patients for clinical trials. The team works with sponsor’s and CRO’s from across the globe, supporting 5,000+ study sites in 19 countries.

LabConnect LLC Seattle, WA(206) 322-4680info@labconnectllc.com

LabConnect, with more than 4,000 validated tests across their network, has an extensive test menu that includes specialized oncology assays, biomarker analysis, pharmacokinetic analysis and method development services.

Evolution Research Group, LLC & Thievon-Wright Consulting Group, LLC Watchung, NJ info@cnssites.com

ERG represents Thievon-Wright Consulting Group and a 15-member network of owned and affiliate clinical research sites.

WCCT Global Costa Mesa, CA(657) 229-6907mgr@wcct.com

WCCT Global is a CRO offering services including study design, study startup, study management, pharmacovigilance, study rescue, patient recruitment, medical writing, regulatory affairs and biostatistics.

Vascular Imaging Solutions and Clare Diagnostic Echo & Research Surprise, AZ(623) 262-7199eileen.spengler@gmail.com

A source for diagnostic ultrasound services, they provide equipment, trained sonographers and image storage system.

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