Morphic Therapeutic (Nasdaq: MORF), a biopharmaceutical company
developing a new generation of oral integrin therapies for the
treatment of serious chronic diseases, today reported corporate
highlights and financial results for the full year 2023.
“Today, our conviction in MORF-057 as a
potential oral, well tolerated, and efficacious treatment for
inflammatory bowel disease (IBD) is stronger than ever, based on
the clear success of the EMERALD-1 trial in UC. Looking forward in
2024, we will work to translate this momentum into further progress
with the GARNET phase 2 study in patients with moderately to
severely active Crohn’s disease running in parallel with the
EMERALD-2 phase 2b study in UC,” commented Praveen Tipirneni, Chief
Executive Officer of Morphic. “Further, we are working to expand
the pipeline of candidates generated by Morphic’s MInT Platform,
most notably with our α5β1 program for pulmonary hypertensive
diseases and additional new projects against both integrin and
non-integrin targets
“On a personal note, I am thankful to return to
Morphic after an acute medical event and grateful for the immense
support from friends and peers within the biotechnology community
and especially the Morphic team,” Tipirneni continued. “This
experience has crystallized, for me, that Morphic’s opportunity to
fundamentally improve patients’ lives is immense and that there is
no time to waste.”
2023 and Recent Corporate
Highlights
EMERALD-1 Phase 2a trial of MORF-057 in UC:
- In the EMERALD-1
phase 2a trial of MORF-057 in ulcerative colitis (UC), topline data
and additional data presented at UEGW 2023 indicated that in a
moderately-to-severely-active UC population with severe disease
burden, MORF-057:
- Was generally
well tolerated with no safety signal observed
- Achieved the
study’s primary endpoint with statistical significance in reduction
of Robarts Histopathology Index (RHI) Score from baseline to week
12 of 6.4 points (p=0.002)
- Showed
consistent clinical improvement across key measures at week 12,
including modified Mayo Clinic Score (mMCS) remission of 25.7% and
mMCS response of 45.7%
- Demonstrated RHI
change ≥7 points in 48.6% of patients and RHI remission in 22.9% of
patients
- Led to clinical
improvement in mMCS within the 12-week induction period for 77% of
patients
- Pharmacokinetic
and pharmacodynamic results confirmed the results seen in healthy
volunteer studies
- Median α4β7 RO
>99% and sustained saturation at week 12
- α4β1 inhibition
below the limit of quantification, in line with the design of
MORF-057
- Predicted
lymphocyte subset changes observed, consistent with engagement of
α4β7
- Demonstrated
deepening of clinical effect beyond the 12-week induction period,
with symptomatic remission rates continuing to increase out to 44
weeks in both advanced treatment-naïve and advanced
treatment-experienced patients
- Announced
completion of enrollment in the exploratory cohort of the EMERALD-1
study comprised of UC patients who have previously failed treatment
with vedolizumab
- Continued the
40-week maintenance phase of the EMERALD-1 study as planned
EMERALD-2 Phase 2b trial of MORF-057 in UC:
- Continued to
enroll the EMERALD-2 phase 2b study of MORF-057 in patients with
moderately-to-severely active UC
- EMERALD-2 is a
global phase 2b randomized, double-blind, placebo-controlled trial
of MORF-057 in patients with moderate-to-severe UC
- The primary
endpoint of EMERALD-2 is clinical remission rate as measured by
mMCS at 12 weeks and is expected to report in the first half of
2025
GARNET Phase 2 trial of MORF-057 in Crohn’s
Disease:
- Announced that
launch activities are underway for the randomized
placebo-controlled GARNET Phase 2 study of MORF-057 in CD and that
the study is anticipated to enroll its first patients in the first
half of 2024
- The GARNET study
will evaluate 210 patients across three cohorts, each comprising 70
patients: 70 patients receiving MORF-057 200 mg BID (twice daily),
70 patients receiving MORF-057 100 mg BID and 70 patients receiving
placebo
- The primary
endpoint of the GARNET study is the proportion of participants in
endoscopic response (>=50% reduction) at week 14 as determined
using Simple Endoscopic Score for Crohn’s Disease (SES-CD)
MORF-057 Preclinical and Phase 1 Studies:
- Presented new
biomarker data at DDW 2023, demonstrating increases in circulating
plasmablasts, consistent with the increased antibody activity
expected with anti-inflammatory mechanism of α4β7 inhibition,
supporting MORF-057 program in UC
- Presented
preclinical data on rational selection of combination therapy for
IBD treatment using an established clinical mode at UEGW 2023
- This study
explored preclinical combination models in UC and preliminarily
examined the potential utility and rationale of combining
anti-inflammatory mechanisms with α4β7 integrin inhibition in
IBD
Pipeline Programs:
- Announced α5β1
as the integrin target of Morphic’s small molecule integrin
inhibitor program in pulmonary hypertensive diseases
- The inhibition
of fibronectin integrins, including α5β1, suppresses pulmonary
arterial smooth muscle cell proliferation and data to date indicate
that inhibition of α5β1 contributes to improved cardiac output and
the reversal of vascular remodeling
- Initiated
discovery efforts that expand the Company’s scope in the immunology
space with oral programs targeting the IL-23 and TL1A pathways,
among others
Financial Results for the Full Year
2023
- Net loss for the
year ended December 31, 2023, was $152.1 million or $3.59 per share
compared to a net loss of $59.0 million or $1.55 per share for the
year ended December 31, 2022
- Revenue was $0.5
million for the year ended December 31, 2023, compared to $70.8
million for the year ended December 31, 2022. The change was
primarily due to recognition of revenue due to the conclusion of
the AbbVie collaboration in 2022 and to the amounts due at the
conclusion of the Janssen collaboration in 2023
- Research and
development expenses were $140.4 million for the year ended
December 31, 2023, as compared to $102.1 million for the year ended
December 31, 2022. The increase was primarily attributable to
higher development costs along with increased clinical trial costs
to support phase 2 clinical studies and development activities for
MORF-057, as well as other research costs to support early
development candidates
- General and
administrative expenses were $38.8 million for the year ended
December 31, 2023, compared to $32.1 million for the year ended
December 31, 2022. The increase was due to increased personnel
related costs and non-cash equity-based compensation, partially
offset by decreases in consulting and insurance expenses
- Morphic raised
approximately $444 million, net, through equity financings in 2023
comprised of approximately $100 million in proceeds from a PIPE
offering, approximately $259 million in a public offering and
approximately $85 million though use of the ATM facility
- As of December
31, 2023, Morphic had cash, cash equivalents and marketable
securities of $704.3 million, compared to $348.2 million as of
December 31, 2022. We believe that our cash, cash equivalents and
marketable securities of $704.3 million as of December 31, 2023,
will enable us to fund our operating expenses and capital
expenditure requirements into the second half of 2027
About MORF-057
Morphic is developing MORF-057 as a selective,
oral small molecule inhibitor of the α4β7 integrin for patients
with inflammatory bowel disease (IBD). α4β7 has been clinically
validated as a target for the treatment of IBD by the success of
the approved injectable antibody therapeutic vedolizumab. MORF-057,
like vedolizumab, is designed to block the interactions between
α4β7 on the surface of lymphocytes and the mucosal endothelial cell
ligand MAdCAM-1, substantially reducing lymphocyte migration from
the bloodstream into intestinal mucosal tissues and avoiding
inflammation that is associated with IBD.
About the EMERALD-1 Study
EMERALD-1 (MORF-057-201) is an open-label
multi-center phase 2a trial designed to evaluate the efficacy,
safety, and tolerability of MORF-057 in adults with moderate to
severe ulcerative colitis. The primary endpoint of EMERALD-1,
change in Robarts Histopathology Index (RHI) from baseline at
twelve weeks, was achieved with statistical significance. RHI is a
validated instrument that measures histological disease activity in
ulcerative colitis. Patients were eligible to continue for an
additional 40 weeks of maintenance therapy followed by a 52-week
assessment as well as an open-label extension period. Secondary and
additional outcome measures in the EMERALD-1 study include change
in the modified Mayo clinic score, safety, pharmacokinetic
parameters and key pharmacodynamic measures including α4β7 receptor
occupancy and lymphocyte subset trafficking.
About the EMERALD-2 Study
EMERALD-2 (MORF-057-202) is a global phase
2b randomized, double-blind, placebo-controlled trial of MORF-057
that is currently enrolling patients with moderate-to-severe
ulcerative colitis. The primary endpoint of EMERALD-2 is clinical
remission rate as measured by the Modified Mayo Clinic Score (mMCS)
at 12 weeks. EMERALD-2 will also measure several secondary and
exploratory endpoints based on the mMCS as well as histologic,
pharmacokinetic and pharmacodynamic measures, and safety
parameters. Patients in the EMERALD-2 study will be randomized to
receive either 200 mg BID (twice daily) MORF-057, 100 mg BID
MORF-057, a QD (once daily) dose of MORF-057, or a placebo dose.
Following the 12-week induction phase, all patients will receive
MORF-057 for 40 weeks of maintenance dosing. For more information
about the EMERALD clinical trials of MORF-057, please
click here.
About the GARNET StudyGARNET
(MORF-057-203) is a global Phase 2b randomized, double-blind,
placebo-controlled trial of MORF-057 in Crohn’s disease. The
primary endpoint of GARNET is the proportion of participants in
endoscopic response (>=50% reduction) at week 14 as determined
using Simple Endoscopic Score for Crohn’s Disease, or SES-CD. The
secondary endpoints will include the change in Crohn’s Disease
Activity Index, or CDAI, measures, as well as safety parameters.
Patients enrolled in the GARNET study will be randomized to receive
one of two active doses or a placebo: 200 mg BID (twice daily), 100
mg BID or a placebo that will cross over to MORF-057 after the
14-week induction phase. Following the 14-week induction phase,
patients will move to a 38-week maintenance phase.
About Morphic
TherapeuticMorphic Therapeutic is a biopharmaceutical
company developing a portfolio of oral integrin therapies for the
treatment of serious chronic diseases, including autoimmune,
cardiovascular, and metabolic diseases, fibrosis, and cancer.
Morphic is also advancing its pipeline and discovery activities in
collaboration with Schrödinger using its proprietary MInT
technology platform which leverages the Company’s unique
understanding of integrin structure and biology. For more
information, visit www.morphictx.com.
Cautionary Note Regarding
Forward-Looking StatementsThis press release contains
“forward-looking” statements within the meaning of the Securities
Act of 1933, as amended, the Securities Exchange Act of 1934, as
amended, and of the “safe harbor” provisions of the Private
Securities Litigation Reform Act of 1995, including, but not
limited to: the MInT Platform’s ability to discover drug
candidates; our plans to develop and commercialize oral
small-molecule integrin therapeutics and any proposed timing
thereof; the initiation, execution and completion of clinical
trials of MORF-057; any expectations about safety, efficacy, timing
and ability to commence or complete clinical and pre-clinical
studies and/or trials and to obtain regulatory approvals for
MORF-057 and other candidates in development; the timing of further
data presentation; and the ability of MORF-057 to treat
inflammatory bowel disease, including UC, CD, and other
indications. Statements including words such as “believe,” “plan,”
“continue,” “expect,” “will be,” “develop,” “signal,” “potential,”
“anticipate” or “ongoing” and statements in the future tense are
forward-looking statements. These forward-looking statements
involve risks and uncertainties, as well as assumptions, which, if
they do not fully materialize or prove incorrect, could cause our
results to differ materially from those expressed or implied by
such forward-looking statements. Forward-looking statements are
subject to risks and uncertainties that may cause our actual
activities or results to differ significantly from those expressed
in any forward-looking statement, including risks and uncertainties
in this press release and other risks set forth in our filings with
the Securities and Exchange Commission, including, among others,
our or a partner’s ability to complete a current or future clinical
trial of any of our current or future product candidates, our
ability to develop or obtain regulatory approval for or
commercialize any product candidate, our ability to protect our
intellectual property, and the sufficiency of our cash, cash
equivalents and investments to fund our operations. These
forward-looking statements speak only as of the date hereof and we
specifically disclaim any obligation to update these
forward-looking statements or reasons why actual results might
differ, whether as a result of new information, future events or
otherwise, except as required by law.
Morphic Holding, Inc.Condensed
Consolidated Statements of Operations(unaudited)(in
thousands, except share and per share data) |
|
Year Ended December 31, |
|
|
2023 |
|
|
|
2022 |
|
Collaboration revenue |
$ |
521 |
|
|
$ |
70,808 |
|
Operating expenses: |
|
|
|
Research and development |
|
140,384 |
|
|
|
102,062 |
|
General and administrative |
|
38,823 |
|
|
|
32,142 |
|
Total operating expenses |
|
179,207 |
|
|
|
134,204 |
|
Loss from operations |
|
(178,686 |
) |
|
|
(63,396 |
) |
Other income: |
|
|
|
Interest income, net |
|
26,969 |
|
|
|
4,567 |
|
Other income (expense),
net |
|
2 |
|
|
|
(145 |
) |
Total other income, net |
|
26,971 |
|
|
|
4,422 |
|
Loss before provision for
income taxes |
|
(151,715 |
) |
|
|
(58,974 |
) |
Provision for income taxes |
|
(380 |
) |
|
|
(67 |
) |
Net loss |
$ |
(152,095 |
) |
|
$ |
(59,041 |
) |
Net loss per share, basic and
diluted |
$ |
(3.59 |
) |
|
$ |
(1.55 |
) |
Weighted average common shares
outstanding, basic and diluted |
|
42,390,554 |
|
|
|
38,112,498 |
|
|
|
|
|
|
|
|
|
Morphic Holding, Inc.Condensed
Consolidated Balance Sheets (unaudited)(in
thousands) |
|
December 31, 2023 |
|
December 31, 2022 |
Assets |
|
|
|
Cash, cash equivalents and marketable securities |
$ |
704,349 |
|
$ |
348,248 |
Other current assets |
|
12,579 |
|
|
13,934 |
Total current assets |
|
716,928 |
|
|
362,182 |
Other assets |
|
5,586 |
|
|
6,407 |
Total assets |
$ |
722,514 |
|
$ |
368,589 |
|
|
|
|
Liabilities and
Stockholders' Equity |
|
|
|
Current liabilities |
$ |
24,776 |
|
$ |
17,126 |
Long-term liabilities |
|
716 |
|
|
2,344 |
Total liabilities |
|
25,492 |
|
|
19,470 |
Total stockholders' equity |
|
697,022 |
|
|
349,119 |
Total liabilities and
stockholders' equity |
$ |
722,514 |
|
$ |
368,589 |
|
|
|
|
|
|
ContactsMorphic TherapeuticChris
Erdmanchris.erdman@morphictx.com617.686.1718
Morphic (NASDAQ:MORF)
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