Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company
focused on advancing meaningful therapies for Duchenne muscular
dystrophy (Duchenne), today reported financial results for the
third quarter ended September 30, 2020 and provided a
business update.
“With the FDA’s lifting of the clinical hold on the IGNITE DMD
trial, we are well underway in completing the activities necessary
to resume dosing, which we expect will occur in the first quarter
of 2021,” said Ilan Ganot, Chief Executive Officer, President
and Co-Founder of Solid Biosciences. “This important event and
establishing a strategic collaboration with Ultragenyx to develop
additional gene therapies for Duchenne mark important progress
toward our goal of improving the lives of patients living with
Duchenne. We are also increasing production of SGT-001 using our
improved manufacturing process in support of dosing additional
patients in 2021. Additionally, we strengthened our balance sheet
with additional capital from the Ultragenyx collaboration and
our recent at-the-market, or ATM, equity financing, both of
which will support our planned clinical advancement of
SGT-001.”
Recent Developments
- In October 2020,
Solid announced that the U.S. Food and Drug Administration (FDA)
lifted the clinical hold placed on the Company’s IGNITE DMD Phase
I/II clinical trial. Solid expects to resume dosing in the clinical
trial in the first quarter of 2021.
- Solid implemented and shared with the FDA manufacturing process
changes that remove the majority of empty viral capsids. The
improved process is averaging approximately 90% full capsids,
allowing target dosing to be achieved with fewer viral
particles.
- Solid submitted data from a new, quantitative, in vitro
microdystrophin expression assay that demonstrates comparability
between SGT-001 manufactured by the two processes.
- Solid is reducing the maximum weight of the next two patients
dosed to 18 kg. This reduction, in conjunction with the delivery of
fewer viral particles as a result of the company’s manufacturing
process improvements, will reduce patients’ total viral load while
continuing dosing at the 2E14 vg/kg dose.
- Solid has amended the IGNITE DMD clinical protocol to include
the prophylactic use of both anti-complement inhibitor eculizumab
and C1 esterase inhibitor, and an increase in prednisone dose in
the first month post dosing.
- Solid also provided the FDA with updated functional efficacy
data (including 6-Minute Walk Test and North Star Ambulatory
Assessment data) for all patients dosed to date in IGNITE DMD.
- In October
2020, Solid and Ultragenyx Pharmaceutical (Ultragenyx) announced a
strategic collaboration to develop and commercialize new gene
therapies for Duchenne. The parties will collaborate to develop
products that combine Solid’s differentiated microdystrophin
construct and Ultragenyx’s HeLa producer cell line (PCL)
manufacturing platform for adeno-associated virus (AAV) vectors
using AAV8 variants. The goal of the collaboration is to expand the
pipeline of potential gene therapies for patients living with
Duchenne.
- Ultragenyx made a $40 million investment in Solid at a 33%
premium.
- Ultragenyx has also agreed to pay up to $255 million in
cumulative milestone payments per product upon achievement of
specified milestone events, and tiered royalties on worldwide net
sales. Upon achievement of proof-of-concept, Solid has the right to
opt-in to co-fund collaboration programs in return for
participation in a profit share or increased royalty payments.
- Solid retains full rights to SGT-001 as well as the opportunity
to establish additional partnerships around SGT-001 or the
Company’s proprietary and differentiated microdystrophin construct
outside of AAV8 variants.
- In October
2020, Solid announced that it sold shares of its common stock
pursuant to a sales agreement dated March 13, 2019, between the
Company and Jefferies LLC that resulted in gross proceeds of $23.9
million (ATM Sale).
Financial Highlights
Research and development expenses for the third quarter of 2020
were $16.0 million, compared to $22.8 million for
the third quarter of 2019. Research and development expenses for
the first nine months of 2020 were $49.2 million,
compared to $67.7 million for the first nine months of 2019.
The decrease was primarily attributable to a reduction in personnel
and facility related expenses as a result of the restructuring that
occurred in January 2020, as well as lower manufacturing costs and
a decrease in costs related to other product candidates as the
Company focuses on advancing SGT-001.
General and administrative expenses for the third quarter of
2020 were $5.2 million, compared to $6.9 million for
the third quarter of 2019. General and administrative expenses for
the first nine months of 2020 were $16.0 million,
compared to $19.3 million for the first nine months of
2019. The decrease was primarily attributable to decreased
personnel costs and corporate expenses partially due to the
restructuring that occurred in January 2020.
Net loss for the third quarter of 2020 was $21.2 million,
compared to $29.3 million for the third quarter of 2019.
Net loss for the first nine months of 2020 was $66.9
million, compared to $85.4 million for the first nine
months of 2019.
Solid had $24.8 million in cash and cash equivalents
as of September 30, 2020. The Company expects that its cash
and cash equivalents, combined with proceeds of $40 million from
the issuance and sale of shares of common stock to Ultragenyx and
the net proceeds of $23.2 million from the ATM Sale will enable
Solid to fund its operating expenses into the second half of
2021.
Conference CallManagement will host a webcast
and conference call to discuss Solid’s third quarter 2020 financial
results and business update today, November 5, 2020, at 8:30 AM
ET.
A live webcast of the call will be available on
the Company's website at www.solidbio.com under the “News &
Events” tab in the Investor Relations section, or by clicking here.
Participants may also access the call, by dialing 866-763-0341 for
domestic callers or 703-871-3818 for international callers.
The archived webcast will be available for in
the “News and Events” section of the Company's website.
About SGT-001Solid’s SGT-001 is a novel
adeno-associated viral (AAV) vector-mediated gene transfer therapy
designed to address the underlying genetic cause of Duchenne.
Duchenne is caused by mutations in the dystrophin gene that result
in the absence or near absence of dystrophin protein. SGT-001 is a
systemically administered candidate that delivers a synthetic
dystrophin gene, called microdystrophin, to the body. This
microdystrophin encodes for a functional protein surrogate that is
expressed in muscles and stabilizes essential associated proteins,
including neuronal nitric oxide synthase nNOS. Data from Solid’s
preclinical program suggests that SGT-001 has the potential to slow
or stop the progression of Duchenne, regardless of genetic mutation
or disease stage.
SGT-001 is based on pioneering research in dystrophin biology by
Dr. Jeffrey Chamberlain of the University of Washington and Dr.
Dongsheng Duan of the University of Missouri. SGT-001 has been
granted Rare Pediatric Disease Designation, or RPDD, and Fast Track
Designation in the United States and Orphan Drug Designations in
both the United States and European Union.
About Solid BiosciencesSolid Biosciences
is a life sciences company focused on advancing transformative
treatments to improve the lives of patients living with Duchenne.
Disease-focused and founded by a family directly impacted by
Duchenne, our mandate is simple yet comprehensive – work to address
the disease at its core by correcting the underlying mutation that
causes Duchenne with our lead gene therapy candidate, SGT-001. For
more information, please visit www.solidbio.com.
Forward-Looking StatementsThis press release
contains “forward-looking statements” within the meaning of the
Private Securities Litigation Reform Act of 1995, including
statements regarding the timing and ability of the Company to
resume dosing and move the IGNITE DMD clinical trial forward, the
safety or potential efficacy of SGT-001, the sufficiency of the
Company’s cash and cash equivalents to fund its operations,
potential milestone payments or royalty payments in connection with
the Ultragenyx collaboration and other statements containing the
words “anticipate,” “believe,” “continue,” “could,” “estimate,”
“expect,” “intend,” “may,” “plan,” “potential,” “predict,”
“project,” “should,” “target,” “would,” “working” and similar
expressions. Any forward-looking statements are based on
management’s 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, but are not limited to, risks associated
with the Company’s ability to resume and/or continue IGNITE DMD on
the timeline expected or at all; obtain and maintain necessary
approvals from the FDA and other regulatory authorities; obtain and
maintain the necessary approvals from investigational review boards
at IGNITE DMD clinical trial sites and the IGNITE DMD independent
data safety monitoring board; enroll patients in IGNITE DMD;
continue to advance SGT-001 in clinical trials; replicate in
clinical trials positive results found in preclinical studies and
earlier stages of clinical development; advance the development of
its product candidates under the timelines it anticipates in
current and future clinical trials; successfully optimize and scale
its manufacturing process; obtain, maintain or protect intellectual
property rights related to its product candidates; compete
successfully with other companies that are seeking to develop
DMD/Duchenne treatments and gene therapies; manage expenses; and
raise the substantial additional capital needed, on the timeline
necessary, to continue development of SGT-001, achieve its other
business objectives and continue as a going concern. For a
discussion of other risks and uncertainties, and other important
factors, any of which could cause the Company’s actual results to
differ from those contained in the forward-looking statements, see
the “Risk Factors” section, as well as discussions of potential
risks, uncertainties and other important factors, in the Company’s
most recent filings with the Securities and Exchange Commission. In
addition, the forward-looking statements included in this press
release represent the Company’s views as of the date hereof and
should not be relied upon as representing the Company’s views as of
any date subsequent to the date hereof. The Company anticipates
that subsequent events and developments will cause the Company's
views to change. However, while the Company may elect to update
these forward-looking statements at some point in the future, the
Company specifically disclaims any obligation to do so.
Investor Contact:David CareyFINN
Partners212-867-1768David.Carey@finnpartners.com
Media Contact:Erich SandovalFINN
Partners917-497-2867Erich.Sandoval@finnpartners.com
Solid Biosciences Inc. |
Condensed Consolidated Statements of Operations |
(unaudited, in thousands, except share and per share data) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended September 30, |
|
Nine Months Ended September 30, |
|
|
|
|
|
2020 |
|
|
|
2019 |
|
|
|
2020 |
|
|
|
2019 |
|
|
|
|
|
|
|
|
|
|
|
|
Revenue |
|
$ |
- |
|
|
$ |
- |
|
|
$ |
- |
|
|
$ |
- |
|
Operating expenses: |
|
|
|
|
|
|
|
|
|
Research and development |
|
|
16,045 |
|
|
|
22,792 |
|
|
|
49,158 |
|
|
|
67,671 |
|
|
General and administrative |
|
|
5,181 |
|
|
|
6,925 |
|
|
|
15,957 |
|
|
|
19,317 |
|
|
Restructuring charges |
|
|
- |
|
|
|
- |
|
|
|
1,944 |
|
|
|
- |
|
|
|
Total operating expenses |
|
|
21,226 |
|
|
|
29,717 |
|
|
|
67,059 |
|
|
|
86,988 |
|
Loss from operations |
|
|
(21,226 |
) |
|
|
(29,717 |
) |
|
|
(67,059 |
) |
|
|
(86,988 |
) |
Other income (expense): |
|
|
|
|
|
|
|
|
|
Interest (expense) income |
|
|
(20 |
) |
|
|
406 |
|
|
|
131 |
|
|
|
1,281 |
|
|
Other income |
|
|
- |
|
|
|
56 |
|
|
|
1 |
|
|
|
345 |
|
|
|
Total other income (expense), net |
|
|
(20 |
) |
|
|
462 |
|
|
|
132 |
|
|
|
1,626 |
|
Net loss |
|
$ |
(21,246 |
) |
|
$ |
(29,255 |
) |
|
$ |
(66,927 |
) |
|
$ |
(85,362 |
) |
Net loss per share attributable to common stockholders, basic and
diluted |
|
$ |
(0.44 |
) |
|
$ |
(0.67 |
) |
|
$ |
(1.39 |
) |
|
$ |
(2.26 |
) |
Weighted average shares of common stock outstanding, basic and
diluted |
|
|
48,295,468 |
|
|
|
43,467,618 |
|
|
|
48,172,686 |
|
|
|
37,727,640 |
|
|
|
|
|
|
|
|
Solid Biosciences Inc. |
Condensed Consolidated Balance Sheets |
(unaudited, in thousands, except share and per share data) |
|
|
|
|
|
|
|
|
|
|
|
September 30, |
|
December 31, |
|
|
|
|
|
2020 |
|
|
|
2019 |
|
Assets |
|
|
|
|
Current assets: |
|
|
|
|
|
Cash and cash equivalents |
|
$ |
24,797 |
|
|
$ |
76,043 |
|
|
Available-for-sale securities |
|
|
- |
|
|
|
7,481 |
|
|
Prepaid expenses and other current assets |
|
|
2,365 |
|
|
|
2,778 |
|
|
|
Total current assets |
|
|
27,162 |
|
|
|
86,302 |
|
Property and equipment, net |
|
|
8,869 |
|
|
|
11,645 |
|
Operating lease, right-of-use assets |
|
|
3,951 |
|
|
|
4,988 |
|
Other non-current assets |
|
|
209 |
|
|
|
209 |
|
Restricted cash |
|
|
327 |
|
|
|
327 |
|
|
|
Total assets |
|
$ |
40,518 |
|
|
$ |
103,471 |
|
|
|
|
|
|
|
|
Liabilities and Stockholders' Equity |
|
|
|
|
Current liabilities: |
|
|
|
|
|
Accounts payable |
|
$ |
4,083 |
|
|
$ |
7,124 |
|
|
Accrued expenses |
|
|
8,550 |
|
|
|
9,178 |
|
|
Operating lease liabilities |
|
|
1,930 |
|
|
|
1,736 |
|
|
Finance lease liabilities |
|
|
202 |
|
|
|
186 |
|
|
Other current liabilities |
|
|
- |
|
|
|
52 |
|
|
|
Total current liabilities |
|
|
14,765 |
|
|
|
18,276 |
|
|
Operating lease liabilities, excluding current portion |
|
|
2,943 |
|
|
|
4,414 |
|
|
Finance lease liabilities, excluding current portion |
|
|
579 |
|
|
|
733 |
|
|
|
Total liabilities |
|
|
18,287 |
|
|
|
23,423 |
|
Common Stock |
|
|
48 |
|
|
|
48 |
|
|
Additional paid-in capital |
|
|
405,389 |
|
|
|
396,278 |
|
|
Accumulated other comprehensive income |
|
|
- |
|
|
|
1 |
|
|
Accumulated deficit |
|
|
(383,206 |
) |
|
|
(316,279 |
) |
|
|
Total stockholders' equity |
|
|
22,231 |
|
|
|
80,048 |
|
|
|
Total liabilities and stockholders' equity |
|
$ |
40,518 |
|
|
$ |
103,471 |
|
Solid Biosciences (NASDAQ:SLDB)
Historical Stock Chart
From Mar 2024 to Apr 2024
Solid Biosciences (NASDAQ:SLDB)
Historical Stock Chart
From Apr 2023 to Apr 2024