1% Tirbanibulin Ointment for the Treatment of Actinic Keratoses

2021 ◽  
pp. 106002802110313
Author(s):  
Diem-Phuong D. Dao ◽  
Vikram Nath Sahni ◽  
Dev Ram Sahni ◽  
Esther A. Balogh ◽  
Ayman Grada ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Adverse Events ◽  
Safety Profile ◽  
Application Site ◽  
Phase 2 ◽  
Phase 3 ◽  
Local Skin ◽  
Actinic Keratoses ◽  
Favorable Safety Profile ◽  
Favorable Safety

Objective Actinic keratoses (AKs) are cutaneous lesions that arise in sun-damaged skin. AKs may transform into squamous cell carcinoma in situ. Tirbanibulin 1% ointment is a new topical treatment for AKs, recently approved by the Food and Drug Administration. Data Sources The PubMed database was searched for articles published from 1960 to March 31, 2021, using the keywords tirbanibulin and Klisyri. Data Extraction Phase 2 and phase 3 clinical trials were reviewed. Data Synthesis In phase 2 clinical trials, 43% of patients treated with tirbanibulin experienced complete clearance by day 57 (43% [95% CI = 32, 54]). Across two phase 3 clinical trials (pooled data), complete (100%) clearance occurred in 49% of patients in tirbanibulin groups and in only 9% of the vehicle groups (difference, 41% points; 95% CI = 35 to 47; P < 0.001). Although no comparative studies are available, tirbanibulin is applied for a shorter duration (5 days) compared with diclofenac 3% gel, fluorouracil 5% cream, and imiquimod 3.75% cream. Adverse events were mild and included pruritus, application site pain, and local skin reactions. Systemic adverse events such as necrosis and angioedema, observed with other AK treatments such as fluorouracil and imiquimod, were not observed with tirbanibulin, thus giving tirbanibulin a favorable safety profile. Relevance to Patient Care and Clinical Practice Tirbanibulin effectively reduces AK burden and recurrence and has a favorable safety profile with mild adverse events. In comparison, imiquimod, 5-flourouracil, and diclofenac can result in necrosis, angioedema, and arthralgias. Conclusion With a favorable safety profile and short regimen, tirbanibulin is an efficacious treatment for clinicians to utilize in their treatment toolbox when treating AKs on the face and scalp.

scholarly journals An Overview on the Ongoing Clinical Trials of COVID-19 Vaccines

2020 ◽  
Vol V (I) ◽  
pp. 39-48
Author(s):  
Syeda Komal Fatima ◽  
Ameena Tur Rasool ◽  
Ayesha Sabir ◽  
Gul Shahnaz
Keyword(s):  
Clinical Trials ◽  
Antimicrobial Resistance ◽  
Adverse Events ◽  
Clinical Efficacy ◽  
Safety Profile ◽  
Clinical Endpoint ◽  
Phase 3 ◽  
Research Reporting ◽  
Development Of Resistance ◽  
Global Pandemic

COVID-19 has posed a great threat to mankind, there is a dire need to introduce a vaccine to combat this global pandemic. Several vaccines are underway to complete their phase 3 clinical trials. This article highlights events surrounding the ongoing clinical trials of vaccines effective against COVID-19, and different procedures and formalities the vaccine will have to endure to get EUA. Clinical trials are discussed stepwise along with the clinical endpoint desired at the end of these trials. In line with antimicrobial resistance, vaccine resistance has also emerged in this era and needs focused consideration, and the probability of development of resistance in these vaccines is discussed. This article specifically covers the latest research reporting clinical efficacy, safety profile, and adverse events following the administration of doses to patients and concerns regarding the rushed approval of vaccines. Four vaccines have been discussed in detail; BNT162b2, mRNA-1273, Sputnik V, ChAdOx1.

scholarly journals Fosfomycin: A First-Line Oral Therapy for Acute Uncomplicated Cystitis

2016 ◽  
Vol 2016 ◽  
pp. 1-10 ◽  
Author(s):  
George G. Zhanel ◽  
Andrew J. Walkty ◽  
James A. Karlowsky
Keyword(s):  
Clinical Trials ◽  
Safety Profile ◽  
Cross Resistance ◽  
Adult Women ◽  
First Line ◽  
Favorable Safety Profile ◽  
E Coli ◽  
Acute Uncomplicated Cystitis ◽  
Uncomplicated Cystitis ◽  
Favorable Safety

Fosfomycin is a new agent to Canada approved for the treatment of acute uncomplicated cystitis (AUC) in adult women infected with susceptible isolates ofE. coliandEnterococcus faecalis. We reviewed the literature regarding the use of oral fosfomycin for the treatment of AUC. All English-language references from 1975 to October 2015 were reviewed. In Canada, fosfomycin tromethamine is manufactured as Monurol® and is available as a 3-gram single dose sachet. Fosfomycin has a unique chemical structure, inhibiting peptidoglycan synthesis at an earlier site compared toβ-lactams with no cross-resistance with other agents. Fosfomycin displays broad-spectrum activity against ESBL-producing, AmpC-producing, carbapenem-non-susceptible, and multidrug-resistant (MDR)E. coli. Resistance to fosfomycin inE. coliis rare (<1%). Fosfomycin is excreted unchanged in the urine by glomerular filtration with peak urinary concentration ~4000 µg/mL and remains at concentrations >100 µg/mL for 48 hours after a single 3-gram oral dose. No dosage adjustments are required in elderly patients, in pregnant patients, or in either renal or hepatic impairment. Fosfomycin demonstrates a favorable safety profile, and clinical trials have demonstrated efficacy in AUC that is comparable to ciprofloxacin, nitrofurantoin, and trimethoprim-sulfamethoxazole. Fosfomycin’s in vitro activity against common uropathogens, including MDR isolates, its favorable safety profile including pregnancy patients, drug interactions, and clinical trials data demonstrating efficacy in AUC, has resulted in Canadian, US, and European guidelines/authorities recommending fosfomycin as a first line agent for the treatment of AUC.

scholarly journals MOD-4023, a long-acting carboxy-terminal peptide-modified human growth hormone: results of a Phase 2 study in growth hormone-deficient adults

2017 ◽  
Vol 176 (3) ◽  
pp. 283-294 ◽  
Author(s):  
Christian J Strasburger ◽  
Peter Vanuga ◽  
Juraj Payer ◽  
Marija Pfeifer ◽  
Vera Popovic ◽  
...  
Keyword(s):  
Growth Hormone ◽  
Safety Profile ◽  
Safety Data ◽  
Human Growth ◽  
Phase 2 ◽  
Pivotal Phase ◽  
Favorable Safety Profile ◽  
Phase 2 Study ◽  
Dosing Regimens ◽  
Favorable Safety

Objective Growth hormone (GH) replacement therapy currently requires daily injections, which may cause distress and low compliance. C-terminal peptide (CTP)-modified growth hormone (MOD-4023) is being developed as a once-weekly dosing regimen in patients with GH deficiency (GHD). This study’s objective is to evaluate the safety, pharmacokinetics (PK), pharmacodynamics (PD) and efficacy of MOD-4023 administered once-weekly in GHD adults. Design 54 adults with GHD currently treated with daily GH were normalized and randomized into 4 weekly dosing cohorts of MOD-4023 at 18.5%, 37%, 55.5% or 123.4% of individual cumulative weekly molar hGH dose. The study included 2 stages: Stage A assessed the effectiveness and PK/PD profiles of the 4 dosing regimens of MOD-4023. Stage B was an extension period of once-weekly MOD-4023 administration (61.7% molar hGH content) to collect further safety data and confirm the results from Stage A. Results Dose-dependent response was observed for both PK and PD data of weekly MOD-4023 treatment. Insulin-like growth factor I (IGF-I) SDS levels were maintained within normal range. The 18.5% cohort was discontinued due to low efficacy. MOD-4023 was well tolerated and exhibited favorable safety profile in all dose cohorts. The reported adverse events were consistent with known GH-related side effects. Conclusions Once-weekly MOD-4023 administration in GHD adults was found to be clinically effective while maintaining a favorable safety profile and may obviate the need for daily injections. Weekly GH injections may improve compliance and overall outcome. The promising results achieved in this Phase 2 study led to a pivotal Phase 3 trial, which is currently ongoing.

scholarly journals A Phase1b Clinical Study of the Oral PI3Kδ Inhibitor, Linperlisib, in Patients with B-Cell Lymphomas

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 3576-3576
Author(s):  
Lugui Qiu ◽  
Zhiming Li ◽  
Yunhong Huang ◽  
Ligen Liu ◽  
Lihong Liu ◽  
...  
Keyword(s):  
Adverse Events ◽  
B Cell ◽  
Safety Profile ◽  
Clinical Studies ◽  
Conflicts Of Interest ◽  
Cell Lymphoma ◽  
B Cell Lymphoma ◽  
B Cell Malignancies ◽  
Favorable Safety Profile ◽  
Favorable Safety

Abstract Introduction The PI3Kδ inhibitor, linperlisib, is a structurally distinctive oral agent demonstrated to be clinically efficacious with a favorable safety profile in follicular lymphoma (FL) and peripheral T-cell lymphoma (PTCL). Here we report initial results of a phase1b study (NCT04279405) evaluating the activity and safety of linperlisib across B-cell lymphoma types, namely DLBCL, MCL, MZL, FL and SLL/CLL. Methods Enrollment initiated in Jan 2020 at 11 clinical sites in China for patients (pts) of DLBCL, MCL, MZL, FL and SLL/CLL having received at least one standard anti-tumor systemic therapy and at least 2 cycles of each prior treatment. Linperlisib tablets were administered orally, 80mg once daily under fasted conditions for 28 days as a cycle, until disease progression, unacceptable toxicity or withdrawal from the study. Adverse events (AEs) were graded by NCI-CTCAE v5.0. Efficacy was evaluated every 2 cycles using the IWG 2007 criteria. Results As of Jul 22, 2021, 43 r/r lymphoma patients were enrolled, including DLBCL (20 pts), MCL (9 pts), FL (8 pts), MZL (3 pts) and SLL/CLL (3 pts). With a median of 2 prior therapies, the majority (93%) of pts previously received first line immunochemotherapy including anti-CD20 targeted therapies. One pts withdrew consent before study treatment began. Three pts withdrew consent in first cycle without post baseline imaging assessment. Forty two pts and 39 patients were evaluable for safety and efficacy, respectively. Linperlisib was well-tolerated in this study. The safety profile observed was consistent to that observed in the clinical studies with FL and PTCL patients, with no new toxicities reported. The most frequent TRAE (all Grade &gt;10%) were neutropenia (54.8%), lymphopenia (40.5%), thrombocytopenia (28.6%), anemia (23.8%), alanine aminotransferase increased (21.4%), aspartate aminotransferase increased (21.4%), blood triglycerides increased (16.7%), blood uric acid increased (14.3%), diarrhoea (11.9%), proteinuria (11.9%) and rash (11.9%). The most frequent Grade ≥3 TRAEs were neutropenia (26.2%) and lymphopenia (9.5%). Thirteen (31.0%) pts experienced SAE considered to be drug related, including pneumonia, thrombocytopenia, pancreatitis, and febrile neutropenia. SAE leading discontinuation from study occurred in 7 pts .Three pts experienced AE that lead to dose modification. No death happened due to any TRAE. No specific associations were observed between B-cell lymphoma types and adverse events on linperlisib treatment. An overall response rate (ORR) and overall disease control rate (DCR) of 53.8% and 79.5% were observed in the evaluable patients. Table1 The 17 evaluable DLBCL pts contained 1 unconfirmed CR (GCB) and 3 PR (GCB, N-GCB, and DLBCL transformed from MZL). The DOR for the 4 DLBCL responders were 56 days, 56 days, 56 days and 196 days. The 8 evaluable FL pts were 3 unconfirmed CR plus 5 PR, and a median DOR of 112days. The 8 evaluable MCL pts contained 2 unconfirmed CR, 3 PR, and 2 SD. The median DOR of the 5 MCL responders were 56 days. The 3 evaluable MZL pts and 3 evaluable SLL/CLL pts were both consisted of 1 unconfirmed CR, 1 PR and 1 SD .The DOR for the 2 MZL responders were 196 days and 56 days, the DOR for the 2 SLL/CLL responders were both 196 days. At the data cutoff date, 16 pts were still treatment ongoing, 19 pts had received exceeding 6 months of treatment, and 1 pt had received exceeding 12 months of treatment, supporting a durable benefit of linperlisib monotherapy. Conclusions Linperlisib demonstrated broad anti-tumor activities in several r/r B -cell malignancies, including DLBC, MCL, MZL, FL and SLL/CLL. Furthermore, linperlisib showed a favorable safety profile with respect to the previously reported information from approved agents in the class of PI3K inhibitors. Further clinical studies of linperlisib in B-cell malignancies and in combination therapies are warranted. Figure 1 Figure 1. Disclosures No relevant conflicts of interest to declare.

scholarly journals Safety of Ustekinumab in Inflammatory Bowel Disease: Pooled Safety Analysis of Results from Phase 2/3 Studies

2020 ◽  
Author(s):  
William J Sandborn ◽  
Brian G Feagan ◽  
Silvio Danese ◽  
Christopher D O’Brien ◽  
Elyssa Ott ◽  
...  
Keyword(s):  
Inflammatory Bowel Disease ◽  
Adverse Events ◽  
Bowel Disease ◽  
Safety Profile ◽  
Phase 2 ◽  
Phase 3 ◽  
Number Of Patients ◽  
Serious Infections ◽  
Inflammatory Bowel

Abstract Background Ustekinumab is currently approved globally in Crohn’s disease (CD) and psoriatic diseases. Recent phase 3 data demonstrate safety/efficacy in ulcerative colitis (UC). Crohn’s disease and UC phase 3 programs had similar study designs, facilitating integrated safety analyses. Methods Data from 6 ustekinumab phase 2/3 CD and UC studies were pooled, and safety was evaluated through 1 year. Patients received 1 placebo or ustekinumab (generally 130 mg or ~6 mg/kg) intravenous induction, then subcutaneous (90 mg) maintenance every 8/12 weeks. Analyses incorporated all patients who received ≥1 ustekinumab dose. Safety outcomes are presented as percentages of patients (induction) and as number of patients with events per 100 patient-years of follow-up (through 1 year). For key safety events, 95% confidence intervals (CIs) are provided, as appropriate. Hazard ratios with 95% CIs from time-to-event analyses for serious adverse events and serious infections were also performed. Results Through 1 year, 2574 patients received ustekinumab (1733 patient-years of follow-up). The number of patients with adverse events per 100 patient-years (placebo 165.99 [95% CI, 155.81–176.67] vs ustekinumab 118.32 [95% CI, 113.25–123.55]), serious AEs (27.50 [95% CI, 23.45–32.04] vs 21.23 [95% CI, 19.12–23.51]), infections (80.31 [95% CI, 73.28–87.84] vs 64.32 [95% CI, 60.60–68.21]), serious infections (5.53 [95% CI, 3.81–7.77] vs 5.02 [95% CI, 4.02–6.19]), and malignancies excluding nonmelanoma skin cancer (0.17 [95% CI, 0.00–0.93] vs 0.40 [95% CI, 0.16–0.83]) were similar between placebo and ustekinumab. Conclusions The safety profile of ustekinumab across the pooled inflammatory bowel disease population through 1 year was favorable and generally comparable to placebo. These data are consistent with the established safety profile of ustekinumab across indications. ClinicalTrials.gov numbers NCT00265122; NCT00771667; NCT01369329; NCT01369342; NCT01369355; NCT02407236.

scholarly journals 30 Lumateperone (ITI-007) for the Treatment of Schizophrenia: Overview of Placebo-Controlled Clinical Trials and an Open-label Safety Switching Study

CNS Spectrums ◽  
2019 ◽  
Vol 24 (1) ◽  
pp. 190-191 ◽  
Author(s):  
Kimberly Vanover ◽  
Steven Glass ◽  
Susan Kozauer ◽  
Jelena Saillard ◽  
Juan Sanchez ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Active Control ◽  
Primary Endpoint ◽  
Safety Profile ◽  
Standard Of Care ◽  
Controlled Clinical Trials ◽  
Open Label ◽  
Favorable Safety Profile ◽  
Favorable Safety ◽  
To Receive

AbstractBackgroundLumateperone is a first-in-class agent in development for schizophrenia that acts synergistically through serotonergic, dopaminergic and glutamatergic systems. Lumateperone is a potent 5-HT2A antagonist, a mesolimbic/mesocortical dopamine phosphoprotein modulator (DPPM) with pre-synaptic partial agonist and post-synaptic antagonist activity at D2, a glutamate GluN2B receptor phosphoprotein modulator with D1-dependent enhancement of both NMDA and AMPA currents via the mTOR protein pathway and an inhibitor of serotonin reuptake.MethodsLumateperone was evaluated in 3 controlled clinical trials to evaluate efficacy in patients with acute schizophrenia. The primary endpoint was change from baseline on the PANSS total score compared to placebo. In Study ‘005, 335 patients were randomized to receive ITI-007 60mg or 120mg , risperidone 4mg (active control) or placebo QAM for 4weeks. In Study ‘301, 450 patients were randomized to receive ITI-007 60mg or 40mg , or placebo QAM for 4weeks. In Study ‘302, 696 patients were randomized to receive ITI-007 60mg or 20mg , risperidone 4mg (active control) or placebo QAM for 6weeks. Also, an open-label safety switching study was conducted in which 302 patients with stable schizophrenia were switched from standard-of-care (SOC) antipsychotics and treated for 6weeks with lumateperone QPM and then switched back to SOC.ResultsIn Studies ‘005 and ‘301, lumateperone (60mg ITI-007) met the primary endpoint with statistically significant superior efficacy over placebo at Day 28. In Study ‘302, neither dose of lumateperone separated from placebo on the primary endpoint; a high placebo response was observed in this study. Across all 3 efficacy trials, lumateperone improved symptoms of schizophrenia with the same trajectory and same magnitude of improvement from baseline to endpoint on the PANSS total score.Lumateperone was well-tolerated with a favorable safety profile in all studies. In the two studies with risperidone included as an active control, lumateperone was statistically significantly better than risperidone on key safety and tolerability measures. In the open-label safety switching study statistically significant improvements from SOC were observed in body weight, cardiometabolic and endocrine parameters worsened again when switched back to SOC medication. In this study, symptoms of schizophrenia generally remained stable or improved. Greater improvements were observed in subgroups of patients with elevated symptomatology (comorbid symptoms of depression and those with prominent negative symptoms).DiscussionLumateperone represents a novel approach to the treatment of schizophrenia with a favorable safety profile in clinical trials. The lack of cardiometabolic and motor safety issues presents a safety profile differentiated from standard-of-care antipsychotic therapy.Funding Acknowledgements: Intra-Cellular Therapies, Inc.

Ustekinumab: Treatment of Adult Moderate-to-Severe Chronic Plaque Psoriasis

2009 ◽  
Vol 43 (9) ◽  
pp. 1456-1465 ◽  
Author(s):  
James V Scanlon ◽  
Benjamin P Exter ◽  
Michael Steinberg ◽  
Courtney I Jarvis
Keyword(s):  
Clinical Trials ◽  
Adverse Events ◽  
Safety Profile ◽  
Relevant Literature ◽  
Life Quality ◽  
Interleukin 12 ◽  
European Medicines Agency ◽  
Upper Respiratory Tract ◽  
Phase 3 ◽  
Clinical Safety

Objective: To systematically review the pharmacology, pharmacokinetics, clinical efficacy, and safety profile of ustekinumab to inform pharmacists and other healthcare professionals of this new biologic therapy for psoriasis. Data Sources: A search of PubMed/MEDLINE, EMBASE, and International Pharmaceutical Abstracts was performed through July 2009, limited to publications in English, using the search terms CNTO-1275, ustekinumab, interleukin-12, interleukin-23, and/or psoriasis to identify literature sources. References from the retrieved articles were also evaluated to identify relevant literature. An abstract from a Congress of the European Academy of Dermatology and Venereology and unpublished Phase 3 clinical trials in progress (using www.clinicaltrials.gov ) were also reviewed. The Food and Drug Administration, European Medicines Agency, and Health Canada Web sites were used to retrieve product monographs, regulatory guidances, and advisory committee briefing packets. Study Selection and Data Extraction: All available studies relevant to the pharmacology, pharmacokinetics, and clinical safety/efficacy of ustekinumab for the treatment of psoriasis were included, with preference for human data. Data Synthesis: Ustekinumab, an anti-interleukin-12/23 monoclonal antibody, achieved the primary endpoint of 75% reduction in the Psoriasis Area and Severity Index score in a large proportion of patients in the Phase 3 PHOENIX trials. Commensurate improvements were also seen in the Physician's Global Assessment and Dermatology Life Quality Index scores. These efficacy results were reproduced in the ACCEPT trial, demonstrating superiority of ustekinumab to etanercept. The frequency of adverse events was similar between ustekinumab and placebo; common adverse events reported included nasopharyngitis, upper respiratory tract infection, headache, arthralgia, cough, and injection site reactions. Phase 3 studies indicate that the optimal dosing appears to be 45 mg for patients weighing less than 100 kg or 90 mg for patients weighing more than 100 kg, with both doses administered subcutaneously. In these studies, the second dose was given 4 weeks after the first and then every 8–12 weeks thereafter, based upon response. Conclusions: Ustekinumab, a promising new therapy, reduces the extent and severity of psoriasis and was well tolerated in clinical trials. Ongoing clinical trials will allow clinicians to further assess the efficacy/safety profile of this novel biologic.

Safety of Efalizumab in Patients with Moderate to Severe Chronic Plaque Psoriasis: Review of Clinical Data. Part II

2005 ◽  
Vol 9 (6) ◽  
pp. 313-323 ◽  
Author(s):  
Kim A. Papp ◽  
Charles Camisa ◽  
Stephen P. Stone ◽  
Ivor Caro ◽  
Xiaolin Wang ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Adverse Events ◽  
Safety Profile ◽  
Controlled Trials ◽  
Efficacy And Safety ◽  
Chronic Plaque Psoriasis ◽  
Serious Adverse Events ◽  
The Third ◽  
Increased Risk ◽  
Favorable Safety

Background: The efficacy and safety of efalizumab have been evaluated in multiple clinical trials. ObjectiveThe purpose of this review is to provide an overview of the safety profile of efalizumab during the clinical trials. Methods: Twelve-week data from four placebo-controlled trials were pooled and analyzed. Data from patients receiving 13—60 weeks of efalizumab therapy were pooled to evaluate longer-term safety. Results: The most common adverse events were mild to moderate, self-limiting, flulike symptoms that were most frequent following the first two efalizumab doses; by the third dose the incidence was comparable to placebo. Serious adverse events were observed in 2.2% and 1.7% of efalizumab- and placebo-treated patients, respectively. Nonserious adverse events leading to withdrawal were infrequent and similar to placebo (2.8% vs 1.8%). There does not appear to be increased risk of end-organ toxicity, infection, or malignancy in efalizumab-treated patients. Conclusion: Efalizumab was well tolerated, with a favorable safety profile.

Clinical Drug Development for the Treatment of Pulmonary Arterial Hypertension: Collaboration With the Pharmaceutical Industry and Regulatory Agencies

2010 ◽  
Vol 9 (4) ◽  
pp. 214-219
Author(s):  
Robyn J. Barst
Keyword(s):  
Clinical Trials ◽  
Pulmonary Arterial Hypertension ◽  
Drug Development ◽  
Phase 1 ◽  
Preclinical Studies ◽  
Phase 2 ◽  
Phase 3 ◽  
Preclinical Testing ◽  
New Drug ◽  
Pulmonary Arterial

Drug development is the entire process of introducing a new drug to the market. It involves drug discovery, screening, preclinical testing, an Investigational New Drug (IND) application in the US or a Clinical Trial Application (CTA) in the EU, phase 1–3 clinical trials, a New Drug Application (NDA), Food and Drug Administration (FDA) review and approval, and postapproval studies required for continuing safety evaluation. Preclinical testing assesses safety and biologic activity, phase 1 determines safety and dosage, phase 2 evaluates efficacy and side effects, and phase 3 confirms efficacy and monitors adverse effects in a larger number of patients. Postapproval studies provide additional postmarketing data. On average, it takes 15 years from preclinical studies to regulatory approval by the FDA: about 3.5–6.5 years for preclinical, 1–1.5 years for phase 1, 2 years for phase 2, 3–3.5 years for phase 3, and 1.5–2.5 years for filing the NDA and completing the FDA review process. Of approximately 5000 compounds evaluated in preclinical studies, about 5 compounds enter clinical trials, and 1 compound is approved (Tufts Center for the Study of Drug Development, 2011). Most drug development programs include approximately 35–40 phase 1 studies, 15 phase 2 studies, and 3–5 pivotal trials with more than 5000 patients enrolled. Thus, to produce safe and effective drugs in a regulated environment is a highly complex process. Against this backdrop, what is the best way to develop drugs for pulmonary arterial hypertension (PAH), an orphan disease often rapidly fatal within several years of diagnosis and in which spontaneous regression does not occur?

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