本帖最后由 凤凰涅盘 于 2012-5-8 20:39 编辑
PIPELINE UPDATE
QLT is currently conducting Phase 1b clinical proof-of-concept studies of QLT091001, a synthetic
retinoid replacement therapy for 11-cis-retinal, a key biochemical component of the visual cycle, in
patients with Leber Congenital Amaurosis (LCA) and Retinitis Pigmentosa (RP) due to inherited genetic
mutations in retinal pigment epithelium protein 65 (RPE65) or lecithin:retinol acyltransferase (LRAT).
QLT is also developing a proprietary punctal plug technology for the delivery of drugs topically to the
eye through controlled sustained release to the tear film. Phase II clinical studies are underway to
investigate the treatment of glaucoma with this delivery system.
QLT091001 orphan drug program for the treatment of LCA and RP:
Leber Congenital Amaurosis – We continue to have discussions with the U.S. Food and Drug
Administration (FDA) and the European Medicines Agency (EMA) concerning the design and
protocol requirements for a potential pivotal trial of QLT091001 for the treatment of LCA. We
are monitoring follow-up of the 14 LCA subjects that were treated in the Phase 1b study and are
retreating some of the LCA subjects at the McGill University Health Centre, as needed, in a
retreatment study. Our goal is to initiate a pivotal trial in LCA patients later in 2012.
Retinitis Pigmentosa – On March 1, 2012, we announced positive preliminary results from our
international multi-center Phase 1b proof-of-concept clinical trial of QLT091001 for the
treatment of RP due to RPE65 or LRAT. The Phase 1b study showed rapid, statistically significant
and clinically meaningful changes in visual fields from baseline values, as well as improvements
in visual acuity, in the study of 17 RP subjects. A retreatment study for RP patients in the initial
Phase 1b study has been implemented at all 7 treatment centers. We anticipate following a similar
design and protocol as LCA for a potential pivotal trial in RP, which we are planning to initiate in
2013
ARVO Presentation – An abstract containing preliminary results of our RP study was submitted
for presentation at the upcoming Association for Research in Vision and Ophthalmology (ARVO)
conference and the submission was granted late-breaker status. The results will be presented by
Dr. Artur Cideciyan, Ph.D., Research Professor of Ophthalmology, Scheie Eye Institute,
University of Pennsylvania, on behalf of the international investigators at ARVO in Ft.
Lauderdale on May 10 at 9:15 a.m. ET. The Company will be hosting a webcast conference call
that day at 11:30 a.m. ET to review the RP data and some of the investigators will be available for
discussion on the call. Data from this multi-center Phase 1b trial will also be presented at the
Retina International World Congress in Hamburg, Germany on July 15, 2012.
Program Safety Studies – Pre-clinical and clinical studies are also ongoing to further evaluate the
safety and tolerability of QLT091001. We have conducted a 6-month repeat intermittent dosing
trial of QLT091001 in 35 normal healthy adult volunteers. Subjects were dosed with 20, 40 or 60
mg/m2 given once daily for 7 consecutive days with repeat weekly courses of treatment
administered on a monthly basis for a total of 6 treatment cycles. No unexpected adverse events
were observed in the trial.
In March, 2012, QLT091001 was granted orphan drug designation by the FDA for the treatment
of RP due to all genetic mutations. Previously, the FDA’s orphan drug designation for
QLT091001 for the treatment of RP was limited to RPE65 and LRAT mutations.
Punctal Plug Drug Delivery System (PPDS) for the treatment of Glaucoma:
Phase II Latanoprost Studies – We have initiated two Phase II clinical trials at multiple
investigator sites in the U.S. to further evaluate the safety, efficacy and duration of effect of the
latanoprost punctal plug delivery system (L-PPDS). The studies are designed to address questions
raised from the L-PPDS Phase II trial completed in 2011, including assessment of the effect of
tearing, latanoprost dosage and the single versus double plug approaches, and to evaluate longer
duration of sustained release. Enrolment in the two trials is ongoing. Results and analysis from
these trials are expected in the second half of 2012. If Phase II development activities are
successful in advancing the program, our goal is to commence Phase III clinical development
activities in the second half of 2013.
Device-Only Study – In conjunction with the Phase II clinical trials in glaucoma, we are
conducting a multicenter, device-only feasibility study to evaluate the safety, tolerability,
comfort, tearing, ease of handling and insertion/removal, and retention of prototype upper and
lower punctal plug designs in healthy volunteers with up to 12 weeks of follow-up.
Second Glaucoma Drug Candidate – We recently received approval from the FDA for an
investigational new drug application for the travoprost punctal plug delivery system (T-PPDS).
We plan to initiate a Phase I proof-of-concept study with the T-PPDS prior to year-end.
Visudyne: Orphan Drug Designation for Central Serous Chorioretinopathy
On March 19, 2012, we announced that our commercial product, Visudyne (verteporfin for
injection), had been granted orphan drug designation for the potential treatment of chronic or
recurrent central serous chorioretinopathy (CSC) from the FDA. The Company is currently
working with external advisors on potential clinical study options for the assessment of the safety
and efficacy of Visudyne in the treatment of chronic CSC that will allow us to evaluate possible
development plans. CSC can affect a range of visual function parameters that may range from
moderate to severe with patients experiencing a reduced daily functioning and quality of life.
Passive Foreign Investment Company
The Company believes that it qualified as a Passive Foreign Investment Company (PFIC) for 2008 –
2011, and that it may qualify as a PFIC in 2012, which could have adverse tax consequences for U.S.
shareholders. Please refer to our Annual Report on Form 10-K for additional information. |
