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October 20, 2014
Puma Biotechnology issued results of
a phase II trial of PB272 (neratinib) for the
treatment of non-small cell lung cancer
(NSCLC) with HER2 mutations. In the trial,
patients with confirmed stage IIIB or stage
IV NSCLC with documented somatic HER2
mutations were randomized to receive
either oral neratinib monotherapy, 240mg
per day, or the combination of oral neratinib,
240mg daily, with intravenous temsirolimus
administered at a dose of 8mg per
week. A total of 27 patients completed the
first stage of the trial; 13 of these patients
received neratinib monotherapy and 14 of
these patients received the combination of
neratinib plus temsirolimus. Results showed
that of the 13 patients who received neratinib
monotherapy, no patient experienced
a partial response, seven (54%) patients
achieved stable disease and four (31%)
patients achieved clinical benefit (defined
as a partial response or stable disease for
12 or more weeks). For the 14 patients who
received the combination of neratinib plus
temsirolimus, three (21%) patients experienced
a partial response, 11 (79%) patients
experienced stable disease and nine (64%)
patients achieved clinical benefit. The
median progression free survival of the
neratinib monotherapy arm was 2.9 months
and the median progression free survival of
the arm that received neratinib plus temsirolimus
was four months. Patients continue
to be enrolled in the arm of the trial that is
receiving the combination of neratinib plus
June 23, 2014
Novartis reported phase I trial results
of Zykadia (LDK378) in patients with
anaplastic lymphoma kinase-positive (ALK+)
non-small cell lung cancer (NSCLC). The 246
patients with ALK+ NSCLC in this single-arm
study received ceritinib 750mg ALK+ daily.
Findings from the study showed patients
treated with ceritinib achieved an ORR of
58.5% [95% CI, 52.1-64.8%] and a median
PFS of 8.2 months [95% CI, 6.7-10.1 months].
The median duration of response was 9.7
months [95% CI, 7.0-11.4 months], with a
median time to first response of six weeks
after starting treatment. Among 163 patients
receiving 750 mg of ceritinib daily and who
were previously treated with the commonly
prescribed ALK inhibitor crizotinib, ORR
was 54.6% [95% CI, 46.6-62.4%] and PFS
was 6.9 months [95% CI, 5.4-8.4 months].
In 83 patients who had not received prior
treatment with an ALK inhibitor, ORR was
66.3% [95% CI, 55.1-76.3%] and PFS had
not been reached. In the 124 patients who
started the study with brain metastases,
ceritinib achieved an ORR of 54.0% [95% CI,
44.9-63.0%] and a median PFS of 6.9 months
[95% CI, 5.4-8.4 months]. Tumor shrinkage
was seen in 50.0% of patients [49 of
98 patients; 95% CI, 39.7-60.3%] with brain
metastases who had received previous ALK
inhibitor therapy, while 69.2% of patients
[18 of 26 patients; 95% CI, 48.2-85.7%] with
brain metastases who were not previously
treated achieved tumor shrinkage following
treatment with ceritinib.
May 6, 2013
Almirall and Forest Laboratories issued results from a phase III trial of aclidinium bromide and formoterol fumarate for the treatment of moderate to severe chronic obstructive pulmonary disease. This 24-week, randomized, double-blind study enrolled 1,729 patients with COPD. Subjects received 400/6mcg and 400/12mcg fixed doses of aclidinium bromide/formoterol fumarate, or aclidinium bromide 400mcg alone, formoterol fumarate 12mcg alone or placebo, administered twice daily through the Genuair/Pressair inhalers. Results showed that both combinations of aclidinium/formoterol (400/6mcg and 400/12mcg given twice a day) demonstrated statistically significant improvements in the co-primary endpoints of change from baseline in morning pre-dose trough FEV1 versus formoterol 12mcg alone and in FEV1 at 1 hour post-dose versus aclidinium 400mcg alone at week 24. For the second co-primary endpoint of change from baseline in FEV1 at 1 hour post-dose versus aclidinium 400mcg, aclidinium/formoterol 400/6mcg and 400/12mcg demonstrated statistically significant improvements versus aclidinium 400mcg (69mL and 125mL, respectively) and placebo (244mL and 299mL, respectively). Both fixed-dose combination treatment arms were well tolerated in this study. The most frequent adverse events were nasopharyngitis and back pain. Based on these results, and expected results from a second phase III trial, Almirall and Forest Laboratories will file an NDA with the FDA and an MAA to the EMA.
September 10, 2012
Boehringer Ingelheim released results from a phase II adjunctive trial of tiotropium for the treatment of chronic obstructive pulmonary disease (COPD). This randomized, double-blind, four-period, crossover study enrolled 232 patients with post-bronchodilator FEV1 of ≥30% and <80% of predicted normal. Subjects received tiotropium 1.25μg, 2.5μg or 5μg in combination with olodaterol 5μg or 10μg, or olodaterol and placebo, for four weeks. Results showed the free combination of tiotropium and olodaterol provided an average lung function improvement of up to 342mL over the first six hours after four weeks of treatment (FEV1 AUC0-6) compared to pre-dose lung function mean baseline values and improvements in trough FEV1 of up to 166mL, compared to pre-treatment FEV1 mean baseline values. Tiotropium was well tolerated. The most frequent adverse events were nasopharyngitis and COPD. This study completes a comprehensive phase II program to thoroughly investigate different doses of each active component to identify the appropriate doses for the fixed-dose combination.
Elevation Pharmaceuticals issued results from a phase IIb trial of EP-101 for the treatment of chronic obstructive pulmonary disease (COPD). This randomized, placebo-controlled, double-blind, seven-arm, four-period cross-over, block design, dose-ranging study, GOLDEN-1, enrolled 140 patients with moderate to severe COPD. Subjects received EP-101 25μg, 50μg, 100μg or 200μg daily or placebo for seven days. Data demonstrated that all doses of EP-101 showed a rapid onset, dose-related, statistically significant improvement in lung function compared to placebo. Improvements in lung function with EP-101 doses were comparable to those of once-daily tiotropium dry powder inhaler and three-times daily nebulized ipratropium administered via jet nebulizer. The drug was well tolerated. The most frequent adverse events were cough and headache. Elevation Pharmaceuticals will be initiating a second phase IIb study in Q4 2012 to select the dose for phase III studies.
September 3, 2012
MediciNova reported preliminary results from a phase Ib trial of MN-221 for the treatment of chronic obstructive pulmonary disease (COPD). This randomized, placebo-controlled study enrolled 25 patients with stable, moderate-to-severe COPD. Subjects received six intravenous infusions of 1200µg MN-221 or placebo over four days. Results indicate moderately improved pulmonary function (FEV1) in MN-221 recipients, but not the placebo recipients. Moreover, the improvement of FEV1 on subsequent MN-221 dosing days was as good as or better than on day one. In addition, there was no significant ccumulation of plasma MN-221 over the multiple dosing intervals. The drug was well tolerated. Two MN-221 recipients with pre-existing heart disorders were terminated from the study due to transient arrhythmias identified by electrocardiograph monitoring that did not have clinical symptoms or consequences and resolved spontaneously. MediciNova will meet with the FDA in October for an end-of-phase II meeting.
June 18, 2012
Otsuka Pharmaceutical reported results from a phase IIb trial of delamanid for the treatment of multidrug-resistant tuberculosis. This multinational, double-blind, randomized, placebo-controlled study enrolled 481 patients. Subjects received delamanid 100mg or 200mg twice daily in combination with background regimen, or placebo plus background regimen for eight weeks. Results showed 45.4% of subjects in the delamanid 100mg arm (p≡0.008) and 41.9% of subjects in the delamanid 200mg arm (p≡0.039) achieved sputum culture conversion (SCC) in the Mycobacterial Growth Indicator Tube (MGIT) system after two months of treatment, compared with 29.6% of subjects in the placebo arm. Adverse events were comparable among all three arms, the most common being QT prolongation on electrocardiogram. Based on these data, Otsuka initiated a phase III trial of delamanid, including subjects receiving anti-retroviral drugs for co-existing HIV infection.
October 23, 2006
Discovery Laboratories announced positive top line results from phase II trial of Surfaxin for the prevention and treatment of Bronchopulmonary Dysplasia (BPD), a disease affecting premature infants. This randomized, double-blind, placebo-controlled trial enrolled 136 premature infants. Subjects received Surfaxin at a standard dose (175 mg/kg), Surfaxin at a low dose (90 mg/kg), or sham air as a control, up to five times a day. Therapy was safe and well tolerated in the treatment and control groups with no differences in adverse events noted between the groups. Efficacy data revealed a positive pharmacological response to Surfaxin therapy with a noted reduction in supplemental oxygen and ventilatory support compared to the control group. Additionally, when compared with controls, the subjects receiving the standard dose Surfaxin therapy had a lower incidence of death or BPD (57.8% vs. 65.9%) and a higher survival rate through 36 weeks post-menstrual age (88.9% vs. 84.1%). Based on this data Discovery plans to move the development of Surfaxin forward.
July 4, 2005
PTC Therapeutics announced positive results of a pair of phase I studies of PTC124, their investigational drug targeting nonsense mutations in cystic fibrosis (CF) and Duchenne muscular dystrophy (DMD), at the 28th Annual European Cystic Fibrosis Conference. Trial data met primary safety and pharmacokinetic endpoints: no serious adverse events were reported, overall tolerability was positive, target plasma concentrations were achieved, and an optimal dosing regimen for future trials has been established. Pharmacogenetic analysis indicated that the drug did not induce unintended read-through of normal, healthy stop codons. These single- and multiple-dose studies enrolled healthy volunteers. The company announced plans to initiate phase II studies of the drug for the treatment of CF, based on these results.
October 7, 2002
Results of a phase II trial suggested that SCV-07 significantly increased the rate by which tuberculosis subjects became noncontagious. In the study, 80% (35/44) of tuberculosis subjects who underwent standard anti-TB chemotherapy were no longer contagious (measured by negative sputum cultures) three months after also receiving a five-day regimen of parenteral SCV-07 therapy. 37% (10/27) of subjects whose therapy did not include SCV-07 were no longer contagious. All the subjects receiving SCV-07 reported an improvement in symptoms including fever and cough. There was also a significant decrease in the number of subjects with lung damage. SCV-07 is a product of Sciclone Pharmaceuticals.
July 15, 2002
Positive results were reported from a phase I/II trial of NicOx's NCX 950, an inhaled nitric oxide-donating drug for respiratory diseases. The randomized, double-blind, placebo-controlled trial was conducted in Switzerland and included 24 healthy subjects. The study was designed to compare treatment with an inhaled formulation of NCX 950 to treatment with salbutamol sulphate and placebo. Results indicated that NCX 950 was safe and well tolerated. Furthermore, data showed equipotency for NCX 950 in terms of bronchodilation and a faster onset of action compared to salbutamol.