With the passage of the FDA Safety and Innovation Act of 2012 (FDASIA), the Breakthrough Therapy Designation (BTD) was introduced as the newest mechanism to facilitate expedited development and review. Here we provide an overview of the BTD pathway and its evolution over the past three years.
Highlights More specific, next-generation FLT3 inhibitors may not have an anti-leukemic benefit over agents with broader TKI activity (such as sorafenib, AMGN), but they may not have as severe myelosuppressive effects. Although the kinase inhibitor rigosertib (ONTX) had poor Phase III results in MDS and AML, the subgroup of patients with primary HMA failure may […]
Key Highlights BIIB-037 Amyloid beta PET data show positive Abeta reduction; cognitive results are not interpretable with such small numbers. (BIIB) BIIB-037 Phase Ib result interpretation may be effected by the ApoE4 dropouts in the treatment arm. (BIIB) MK-8931 and BIIB-037 show sufficient target engagement, but there is no evidence to date that amyloid clearance […]
Highlights Current manufacturing turnaround times for CAR-T products (2-3 weeks) for ALL are not a major limitation for pediatric leukemia patients. CAR-T treatments may be more acutely toxic than transplant immediately after infusion, but patients may have fewer medium- and long-term complications. Agents that target the cell cycle (CDK inhibitors, Aurora kinase inhibitors) will likely […]
Overview We performed a 5-question survey of 25 neurologists in the US to gauge current familiarity with Alzheimer’s Disease (AD) biomarkers and drugs in clinical development for disease modification. Drugs covered include: BIIB037/BART (BIIB), Solanezumab (LLY), Pioglitazone (Takeda), BAN2401 (ESALY/BIIB), MK-8931 (MRK).
Indications: Huntington’s Disease, Alzheimer’s Disease Physician Information Specialty: Neurology Location: London, United Kingdom
Neuromodulation KOL Interview
Following our live coverage of ASH, we interviewed a KOL who specializes in treating lymphoid malignancies to provide further insight on current and future paradigms for the treatment of CLL and NHLs.
We performed a 5-question survey of 40 hematology-oncology specialists in the US to gauge current prescribing practices and physician experience with reimbursement for ibrutinib and idelalisib for the treatment of patients with CLL.
We performed a 5-question survey of 20 infectious disease (ID) specialists in the U.S. to gauge current prescribing practices for community-acquired pneumonia (CAP) and gauge interest in new drugs/new drug candidates.
Over 95% SVR for easy-to-treat patients, and it’s one pill a day for 8-12 weeks for most. In that context, the only way you can improve up on that is to improve care for all populations, not just for easy-to-treat but the difficult-to-treat: decompensated cirrhotics, the post-transplant, and HIV co-infected patients.
Ebola KOL Interview #3
So the history in Africa has been very extensive since the 1970s and outbreaks in those countries havehappened sporadically over the last 40 years. Here in US, right, this is our first case of anyone being sick with Ebola, and there are a number of people who have actually come into the country.
BioMedTracker – Dermatology KOL Interview In recent years, patients have become more comfortable with aesthetic dermatology procedures as they have become more widespread.
We performed a 5-question survey of 21 dermatologists to gauge usage of drugs and devices for the treatment of common dermatological conditions and for aesthetic procedures.
Multiple Sclerosis KOL Insight Interview
We performed a 5-question survey of 20 ophthalmologists who treat retinal diseases to gauge current usage and projected future usage of agents for the treatment of wet and dry age-related macular degeneration (AMD) and diabetic macular edema (DME).
We conducted a survey of 20 infectious diseases specialists to evaluate current drug usage for the treatment of selected severe/inpatient infections and gauge physician interest in new drugs.
This survey of US primary care physicians (n=10) and endocrinologists (n=10) was undertaken to evaluate the impact of GLP-1 agonists if they were to be introduced in the Diabetes market.
We surveyed primary care physicians (n=10) and endocrinologists (n=10) to see how SGLT-2 inhibitors and the combo pills will be used and what the potential impact of usage with GLP-1 agonists may have.
Crenezumab data was negative in a Phase II study in mild to moderate AD. The signal in not pre-specified isn’t convincing. These data likely will have ethical implications for the asymptomatic, genetic AD predisposition study in the Columbian trial.
Discusses percent not meeting goal and projected usage of PCSK9 and CETP inhibitors in various scenarios based on outcomes data.
Discusses usage of PCSK9 and CETP inhibitors in various scenarios based on outcomes data
Zontivity was approved (May 8, 2014) for use with clopidogrel/aspirin, its label was relatively benign, not reporting bleeding in the overall trial, which included stroke/TIA patients who had excessive bleeding with the drug, or in ACS trial, where there was also excessive bleeding.
Physicians are starting to use Perjeta (Roche) as an add-on to Herceptin in the adjuvant setting due to a recent change in NCCN (National Comprehensive Cancer Network, an alliance of major cancer centers) guidelines.
Diagnostics/diagnostic techniques discussed:
etarfolatide/folic acid, BRCA mutation testing
The European Association for the Study of the Liver (EASL) held the 2014 International Liver Meeting in London from April 9-13. The main focus of this year’s meeting was the clinical use of currently approved and soon-to-be approved, direct-acting antivirals for Hepatitis C.
Drugs and drug candidates discussed:
palbociclib, neratinib, Herceptin, Perjeta, Kadcyla, Tykerb, Afinitor
The study was just closed early, as LCZ696 increased time to first occurrence of either
cardiovascular death or heart failure hospitalization, though details have not been released.
We surveyed 10 US Cardiologists to get a preview of how they would use LCZ696 under different
assumptions about what the study found.
These pathways can be targeted either at the cell surface receptor
level or their downstream signaling cascades. Currently, everolimus in combination with
exemestane represents a new standard of care for patients progressing on non-steroidal AIs.
HDAC inhibitors have also shown promising results For innate resistance, the combination of
fulvestrant and AI in the front line setting represents a new treatment option, particularly for
patients who present with de novo metastatic disease.
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