Dimension Therapeutics, Inc. (NASDAQ:DMTX) Files An 8-K Regulation FD Disclosure
Item7.01
| Regulation FD Disclosure. |
On January31, 2017, Dimension Therapeutics, Inc. (the Company)
announced clinical trial data in a press release, a copy of which
is furnished herewith as Exhibit 99.1.
In addition, on January31, 2017, the Company posted an updated
corporate presentation on its website, www.dimensiontx.com. A
copy of this presentation is furnished herewith as Exhibit 99.2.
The information in this Item 7.01 and Exhibits 99.1 and 99.2
attached hereto shall not be deemed filed for purposes of
Section18 of the Securities and Exchange Act of 1934, as amended,
or otherwise subject to the liabilities of that section, nor
shall they be deemed incorporated by reference in any filing
under the Securities Act of 1933, as amended, except as expressly
set forth by specific reference in such filing.
| Item8.01 | Other Events. |
In connection with the announcement of clinical trial data
described above, the Company announced preliminary topline safety
and early efficacy results of the Companys multi-center phase 1/2
study of DTX101 for the treatment of adult patients with
moderate/severe to severe hemophilia B. DTX101, the Companys lead
AAV candidate, is designed to deliver stable expression of blood
clotting Factor IX (FIX) in patients with hemophilia B, a rare
genetic bleeding disorder resulting from a deficiency in FIX. The
Company is reporting preliminary topline phase 1/2 results from
the trials first two dose cohorts (Cohort 1: N=3, 1.6 x 10
GC/kg; Cohort 2: N=3, 5 x 10
GC/kg). Patients in the two
cohorts have been in post-treatment follow-up ranging from 6 to
52 weeks.
In the phase 1/2 study,
patients received serotype AAVrh10 vector with a codon-optimized
FIX gene expressing wild-type FIX protein. Evidence of efficient
liver transduction of DTX101 was observed across the two patient
cohorts. Patients in the second dose cohort achieved peak FIX
expression of 13%, 20%, and 12% at weeks 4, 8, and 8,
respectively. FIX activity was 5% and 8% in two patients at
12-weeks follow-up, and 7% for the third patient at 7 weeks. For
the low-dose cohort, expression levels achieved 10-11% peak
activity, stabilizing between 3-4% at last follow-up (weeks 24,
48, and 52). The findings in the low-dose cohort are comparable
to results published by Nathwani et al in the
New England Journal of Medicine
(2011), where patients have had measurable levels of FIX for 3-5
years or more at last follow-up (Nienhuis et al. 2016).
All patients in both cohorts improved from
moderate/severe-to-severe to either moderate or mild range in
terms of factor levels based on World Federation of Hemophilia
(WFH) criteria. In addition, none of the patients in Cohort 2
have required prophylactic or on-demand recombinant FIX
transfusion for spontaneous bleeds post-dosing. According to the
WFH, people with FIX levels of 5% or more usually bleed only as a
result of surgery or major injury, do not bleed often and, in
fact, may never have a bleeding problem.*
Elevations in alanine aminotransferase (ALT) were observed in 5
of 6 patients, with patient 3 in Cohort 2 experiencing a grade 4
adverse event due to an elevated laboratory ALT (defined as 800
IU/L). All elevated liver enzymes were clinically asymptomatic
with no elevations of gamma-glutamyl transferase (GGT), alkaline
phosphatase or bilirubin, and no patients experienced a drug
related serious adverse event (SAE) as of the January28, 2017
data cutoff. Preliminary Phase 1/2 findings from 2 patients in
each of Cohort 1 and 2 prompted administration of a standard
tapering course of corticosteroids to treat mild, asymptomatic
elevations in ALTs (52-98 IU/L), similar to findings from
multiple studies using other AAV serotypes, including AAV8 and
AAV9. The third patient in Cohort 2 also received
corticosteroids, experiencing a peak ALT of 914 IU/L, and was at
431 IU/L at 6 weeks post-dosing. The Company expects that Cohort
2 will continue to receive a standard tapering course of
corticosteroid therapy and as of the January28, 2017 data cutoff,
2 of 3 patients ALT levels were in the normal range. Cohort 1
patients were all clinically stable and off steroids with ALT
levels in the normal range. As required by the trial protocol,
the Company reported the ALT levels for patient 3 in Cohort 2 to
the Data Safety Monitoring Committee (DSMC), the U.S. Food and
Drug Administration (FDA), and the appropriate regulatory
authorities and will await their feedback prior to initiating
dosing of Cohort 3.
*Information sourced
from World Federation of Hemophilia
Cautionary Note Regarding Forward-Looking
Statements
This Current Report on Form 8-K contains forward-looking
statements within the meaning of the Private Securities
Litigation Reform Act of 1995, including, but not limited to,
statements regarding the development, preclinical and clinical
results, and the continued progress of the Companys portfolio and
programs, including the initiation, timing, scope, or likelihood
of regulatory filings and approvals, and the Companys ability to
develop and advance product candidates into, and successfully
complete, clinical studies. All such forward-looking statements
are based on managements current expectations of future events
and are subject to a number of risks and uncertainties that could
cause actual results to differ materially and adversely from
those set forth in or implied by such forward-looking statements.
These risks and uncertainties include the risks that the Companys
product candidates, including its candidates, DTX101 and DTX301,
will not successfully be developed or commercialized in the times
indicated or at all; the impact of the observed data in Cohorts 1
and 2, including the adverse event described above and any later
safety event on timing, dosing, regulatory action or patient
enrollment with respect to DTX101 and DTX301; and the risks
described under the caption Risk Factors in the Companys
Quarterly Report on Form 10-Q for the quarter ended September30,
2016, which is on file with the Securities and Exchange
Commission, as well as other risks detailed in the Companys
additional filings with the Securities and Exchange Commission.
All information in this Current Report on Form 8-K is as of the
date of this Current Report on Form 8-K, and the Company
undertakes no duty to update this information unless required by
law.
About Dimension Therapeutics, Inc. (NASDAQ:DMTX)
Dimension Therapeutics, Inc. is a gene therapy company. The Company is focused on discovering and developing therapeutic products for people living with rare diseases associated with the liver and caused by genetic mutations. It has programs for hemophilia B, hemophilia A, ornithine transcarbamylase (OTC) deficiency, and glycogen storage disease type Ia (GSDIa). The Company’s gene therapy product candidates and programs are designed to provide a functional copy of an abnormal or missing gene using the advanced adeno-associated virus (AAV)-based vector delivery technology. DTX101 is the Company’s lead gene therapy product candidate designed to deliver Factor IX (FIX), gene expression in patients with hemophilia B. DTX201 is its Factor VIII (FVIII) gene therapy program for the treatment of hemophilia A. DTX301 is its gene therapy product candidate designed for the treatment of patients with OTC deficiency. DTX401 is its gene therapy program for the treatment of patients with GSDIa. Dimension Therapeutics, Inc. (NASDAQ:DMTX) Recent Trading Information
Dimension Therapeutics, Inc. (NASDAQ:DMTX) closed its last trading session 00.00 at 4.25 with 70,190 shares trading hands.