April 2018

The Pulse

Patient Centricity: A positive shift for the patient experience

April 30, 2018

Sponsors’ heightened awareness of the importance of patient engagement in the design and execution of research studies has led to the discovery of crucial information about participation pain points. The information often reveals huge disconnects in what patients prefer and what they experience. Resolving these disconnects can have a significantly positive impact on patient recruitment.


Novartis Announces Real-Time, Self-Reported Data-Based App for Ophthalmic Trials

April 30, 2018

Novartis has launched an ophthalmic digital research app that will allow researchers to monitor disease progression through self-reported patient data. The app, FocalView, collects real-time, voluntarily disclosed data from consenting patients, which will allow Sponsors to adapt clinical trial design to patients’ routines. The goal is to eliminate some of the most common obstacles to trial participation and increase practical understanding of ophthalmic diseases. The company is planning to test the app in a prospective, non-interventional study to determine its use for evaluating visual function such as acuity and contrast sensitivity. “Because patients with eye diseases are often not as mobile, FocalView has the potential to offer tremendous benefit for the ophthalmic community and for researchers looking to develop better treatments for these patients,” said Mark Bullimore, dean of the Southern California College of Optometry, Marshall B. Ketchum University, who served as medical advisor for the creation of the app.


Pipeline new from Pfizer, Sage, Polyphor, Mithra, Catalyst and QIAGEN

April 30, 2018

Company Drug/Device Medical Condition Status
AOBiome Therapeutics Ammonia Oxidizing Bacteria (AOB) intranasal spray episodic migraine Phase II trial initiated enrolling 303 subjects
Mithra Donesta Vasomotor Symptoms (VMS) in post-menopausal women Phase II top-line results announced evaluating 257 subjects
Concert Pharmaceuticals CTP-543 moderate-to-severe alopecia areata Phase IIa trial initiated evaluating 90 subjects
Aldeyra Therapeutics, Inc. topical ocular reproxalap allergic conjunctivitis Phase III trial initiated enrolling 300 subjects
Catalyst Pharmaceuticals, Inc. Firdapse (amifampridine phosphate) MuSK antibody positive Myasthenia Gravis (MuSK-MG) Phase III trial initiated enrolling 60 subjects in the U.S. and Italy
Pfizer 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 by the FDA
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 by the FDA
Varian Calypso Anchored Beacon transponder tumor detection 510(k) clearance granted by the FDA
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 filed with the FDA
Sage Therapeutics intravenous formulation of brexanolone (SAGE-547) postpartum depression (PPD) NDA filed with the FDA
Veloxis Pharmaceuticals A/S de novo indication of ENVARSUS XR (tacrolimus extended-release tablets) Prophylaxis of organ rejection in kidney transplant patients sNDA filed with the FDA
Abeona Therapeutics Inc. ABO-102 AAV-mediated gene therapy for the treatment of Sanfilippo syndrome Type A (MPS IIIA) RMAT Designation granted by the FDA
MeiraGTx Limited AAV-RPGR X-linked retinitis pigmentosa (SLRP) due to defects in the retinitis pigmentosa GTPase regulator (RPGR) gene Fast Track Designation granted by the FDA
Polyphor balixafortide (POL6326) in combination with eribulin patients with HER2-negative metastatic breast cancer who previously received at least two chemotherapeutic regiments in the metastatic setting Fast Track Designation granted by the FDA
QIAGEN PartoSure preterm birth FDA approved
Trial Results

Alnylam Reports Results from the APOLLO Phase III Study

April 30, 2018

Alnylam Pharmaceuticals announced new results from the APOLLO Phase III study of patisiran, an investigational RNAi therapeutic for the treatment of hereditary ATTR (hATTR) amyloidosis. The APOLLO Phase III trial was a randomized, double-blind, placebo-controlled, global study designed to evaluate the efficacy and safety of patisiran in hATTR amyloidosis patients with polyneuropathy. The primary endpoint of the study was the change from baseline in modified Neurologic Impairment Score +7 (mNIS+7) relative to placebo at 18 months. The trial enrolled 225 hATTR amyloidosis patients from 19 countries with 39 genotypes who were randomized 2:1, patisiran:placebo, with patisiran administered at 0.3 mg/kg once every three weeks for 18 months. There were 13 deaths in the APOLLO study; none were considered related to study drug and the frequency of deaths was lower in the patisiran group (4.7 percent) as compared with placebo (7.8 percent). While treatment benefit is observed across all stages of disease, these results support the rationale for early treatment with patisiran to potentially halt or improve neuropathy progression or impairment, respectively.

Paratek Pharmaceuticals Presents Data for Skin Infections Trial
Paratek Pharmaceuticals announced that an analysis of microbiology data from OASIS-2, its second Phase III study of omadacycline in acute skin infections, found that once-daily monotherapy with oral omadacycline is effective in treating frequently isolated pathogens associated with skin infections. The OASIS-2 study was a randomized, double-blind, multi-center study that enrolled 735 adult subjects with moderate to severe ABSSSI at 33 centers in the U.S. Patients received either once-daily, oral omadacycline or twice-daily, oral linezolid for 7 to 14 days. To analyze clinical success per infection type in OASIS-2, the modified intent-to-treat (mITT) population included randomized subjects without a sole Gram-negative pathogen(s) at baseline (n=720). Infection type broke down as follows: 59 percent wound infection; 24 percent cellulitis/erysipelas and 18 percent major abscess. In the pivotal Phase III OASIS-2 study, omadacycline met the FDA-specified primary endpoint of statistical non-inferiority (NI) in the modified intent-to-treat (mITT) population (10 percent NI margin, 95 percent confidence interval) compared to linezolid at the early clinical response (ECR), 48 to 72 hours after the first dose of study drug.

Tarveda Therapeutics Doses First Patient in Phase I/IIa Study
Tarveda Therapeutics announced that it has dosed the first patient in a Phase I/IIa study evaluating PEN-866 in patients with advanced solid tumors. PEN-866 is a miniature drug conjugate that selectively binds to the intracellular target Heat Shock Protein 90 (HSP90) and is linked to SN-38, a known and potent anti-cancer payload. The multi-center, dose escalation and expansion study will assess safety and efficacy across a range of tumor types including those previously shown to be sensitive to topoisomerase 1 inhibitors. This includes but is not limited to small cell lung, pancreatic, triple negative breast, colon and ovarian cancers and sarcomas. The Phase IIa portion of the trial has now been initiated and enrollment has begun in patients with small cell lung cancer as well as GI-midgut and pancreatic neuroendocrine tumors that express SSTR2. PEN-866 is a miniature conjugate that comprises a small molecule, HSP90-targeting ligand linked to SN-38, the active metabolite of irinotecan. Checkmate Pharmaceuticals Announces Start of Phase Ib Trial
Checkmate Pharmaceuticals announced that it had initiated treatment with CMP-001 combined with atezolizumab (TECENTRIQ) in a Phase Ib clinical trial of patients with advanced non-small cell lung cancer (NSCLC) and disease progression on prior anti-PD-1/PD-L1 therapy. CMP-001 is designed to activate innate immunity to convert “uninflamed” tumors, which generally do not respond to anti-PD-1/L1 therapy, into “inflamed” tumors, which are responsive to PD-1 inhibition. The trial is designed as a multi-center, open label, two-part Phase 1b study of CMP-001 administered in combination with atezolizumab with and without low-level radiation therapy. Part one of the study will evaluate CMP-001 (5 mg dose) administered subcutaneously (SC) weekly for two weeks and then intratumorally (IT) weekly for three weeks, followed by either SC or IT injection every three weeks. In the second part of the trial, the combination of CMP-001 and atezolizumab therapy will be preceded by low-level radiation therapy to the target lesion.

Research Center Profiles

April 30, 2018’s Top 10 Research Center Profiles

April 30, 2018

Research Center Spotlight is a monthly selection of clinical research centers who have Research Center 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 expertise and services in conducting and managing clinical studies. To learn more about becoming a Research Center Profile page subscriber, contact Sales at (617) 948-5100.


FDA to Launch Pilot Program on Model-Informed Drug Development

April 23, 2018

The FDA announced a new pilot program in which drug sponsors can meet with agency officials to discuss strategies for model-informed drug development (MIDD) to make their clinical trials more efficient and increase the chances of regulatory approval. Under the pilot, each applicant whose proposal is approved will receive two meetings with the relevant agency center to discuss approaches to applying MIDD, which uses quantitative analysis to assess the risk-benefit profile of in-development drug products. “The goal of the early meeting discussions granted under this pilot program is to provide advice on how specific, proposed MIDD approaches can be used in a specific drug development,” the FDA said. The agency plans to accept two to four meeting requests per fiscal quarter from the fourth quarter of 2018 to the fourth quarter of 2022. Because of the limited number of meeting slots, the agency will prioritize requests that focus on dose selection/estimation, clinical trial simulation and predictive or mechanistic safety evaluation. Applicants should be drug or biologic manufacturers with a relevant IND or pre-IND number. The meeting requests should include the product name, application number, chemical name/structure and proposed indications or context of product development. Applicants should also include a brief overview of the purpose and objectives of the potential meeting, proposed MIDD approaches for the product and a list of issues for discussion with the agency. The submission package should also address drug development issues such as dosing, safety predictions or clinical trial design and the proposed MIDD approach. The pilot will be administered jointly by CDER’s Office of Clinical Pharmacology and CBER’s Office of Biostatistics and Epidemiology. Read the Federal Register notice here: www.fdanews.com/04-16-18-Pilot.pdf.