11 December 2024
Syncona
Limited
iOnctura presents new data
and provides clinical update in uveal melanoma
Syncona Ltd, ("Syncona") a leading
life science investor focused on creating,
building and scaling global leaders in life science,
today notes that its portfolio company iOnctura
announced a clinical update and the presentation of new data from
the completed Phase I DIONE-01 study of their lead asset,
roginolisib, at the European Society for Medical Oncology
Immuno-Oncology (ESMO-IO), taking place in Geneva, Switzerland,
from 11-13 December 2024.
Roginolisib is a first-in-class
allosteric (indirect) modulator of PI3K delta (PI3Kδ), which has
potential application across a variety of solid tumour and
haematological cancers. Its unique chemical structure and binding
mechanism makes it highly specific for PI3Kδ, giving it an
advantageous pharmacology and safety profile compared to previous
generations of PI3Kδ inhibitors. Roginolisib is being investigated in solid and haematological
cancers including uveal melanoma (UM), a rare cancer of the
eye.
Key highlights from the data
presentation include:
· The
Phase I DIONE-01 study met its primary objective of determining the
safety of the anticipated optimal biologically effective dose (BED)
of roginolisib
· Roginolisib was shown to be well tolerated over long periods
of treatment (up to 4.5 years) and was well tolerated at the
recommended Phase II dose (RP2D) of 80mg
· Median
overall survival (OS) was 16 months for the 29 patients with UM
that were treated with roginolisib. These patients previously
received a median of two prior therapies. This exceeds the median
of seven months OS observed in historical controls in patients
receiving immunotherapies as second line treatment[1]
· Median
progression free survival (PFS) was five months for patients
treated with roginolisib versus less than three months for
historical controls[1]
Following these positive results
iOnctura is initiating its OCULE-01 Phase II study of roginolisib
in UM. A data readout from this trial is expected in CY2026 and is
a key value inflection point for iOnctura[2].
Roel Bulthuis, Managing Partner and Head of Investments of
Syncona Investment Management Limited and Board Director of
iOnctura, said: "Data from the Phase
I DIONE-01 study validate roginolisib's differentiated profile as
an allosteric PI3Kδ inhibitor. These early positive results
suggests that iOnctura could potentially become the first company
to develop a clinically meaningful medicine targeting the PI3K
cancer pathway, which no company has been able to target with
sufficient precision to date. We look forward to initial data from
the Phase II OCULE-01 study, as well as the expansion of
roginolisib's utility to other indications, including primary
myelofibrosis and non-small cell lung cancer."
iOnctura's announcement is copied
below and can be accessed at the company's website at
https://www.ionctura.com/.
[ENDS]
Enquiries
Syncona Ltd
Natalie Garland-Collins / Fergus
Witt
Tel: +44 (0) 20 3981 7912
FTI Consulting
Ben Atwell / Tim Stamper
Tel: +44 (0) 20 3727 1000
About Syncona
Syncona's purpose is to invest to
extend and enhance human life. We do this by creating, building and
scaling companies to deliver transformational treatments to
patients in areas of high unmet need.
We aim to build and maintain a
diversified portfolio of 20-25 globally leading life science
businesses, across development stage, modality and therapeutic
area, for the benefit of all our stakeholders. We focus on
developing treatments that deliver patient impact by working in
close partnership with world-class academic founders and
experienced management teams. Our balance sheet underpins our
strategy, enabling us to take a long-term view as we look to
improve the lives of patients with no or poor treatment options,
build sustainable life science companies and deliver strong
risk-adjusted returns to shareholders.
Syncona Limited seeks to achieve returns over the long term.
Investors should seek to ensure they understand the risks and
opportunities of an investment in Syncona Limited, including the
information in our published documentation, before
investing.
About Key Value Inflection Points
A key value inflection point is a
material de-risking event for a portfolio company that has the
potential to drive significant NAV growth for Syncona, for example
by increasing the possibility of a realisation event, such as
M&A. These milestones can also enable companies to access
significant capital including through financings and IPOs, which
may take place at valuation uplifts and underpin progression to a
subsequent key value inflection point which has the potential to
drive greater value. M&A or capital access is unlikely to occur
immediately following a key value inflection point.
iOnctura reaches new clinical milestones in uveal
melanoma
· Completed Phase I DIONE-01 study demonstrates clinical
activity and long-term safety of roginolisib, a unique allosteric
modulator of PI3Kδ
· Patients with uveal melanoma showed a doubling of overall
survival compared to historical controls
· Site activation ongoing for randomized Phase II OCULE-01 study
in uveal melanoma
Geneva, Switzerland and Amsterdam, The Netherlands, 11 Dec
2024 - iOnctura, a clinical-stage
biopharmaceutical company combating neglected and hard-to-treat
cancers, today provides a clinical update on its lead asset,
roginolisib. Results from the completed Phase I DIONE-01 study are
due to be presented at the European Society for Medical Oncology
Immuno-Oncology (ESMO-IO) annual congress tomorrow, 12 December at
12:30 CET (presentation 164P).
Allosteric modulator of PI3Kδ,
roginolisib, has a unique chemical structure and binding mechanism
which makes it highly specific for PI3Kδ, giving it an advantageous
pharmacology profile and an unprecedented safety profile compared
to previous generations of PI3Kδ inhibitors.
Roginolisib is being investigated in
solid and hematological malignancies including uveal melanoma (UM),
a rare cancer of the eye. Eye melanoma is a rapidly growing market
which is projected to be worth USD 9.56B by 2032[3].
The two-part Phase I
study DIONE-01, firstly evaluated
continuous daily dosing of roginolisib [at 10, 20, 40 and 80 mg] in
24 patients with pretreated solid tumors and follicular lymphoma
(FL), and secondly evaluated a dose confirmation cohort in 20 UM
patients.
Results from DIONE-01 show:
· Study
met its primary objective to determine the safety of the
anticipated optimal biologically effective dose (BED): Roginolisib
was well tolerated at the recommended Phase II dose (RP2D) of 80mg,
with <7% Grade 3/4 treatment-emergent adverse events (TEAEs)
considered to be related to roginolisib. TEAEs did not result in
immune-related toxicity, or dose-limiting toxicity, in either solid
tumor or hematological patients. In contrast to prior PI3Kδ
inhibitors, roginolisib dosing did not require dose
modifications.
· Roginolisib is well tolerated over long periods of treatment,
up to 4.5 years.
·
Median overall survival (OS)
was 16 months for the 29 patients with UM treated with roginolisib,
who had previously received a median of two prior therapies. This
exceeds the median OS of 7 months observed in historical controls
in patients receiving immunotherapies as second line
treatment1.
·
Median progression free
survival (PFS) was 5 months for patients treated with roginolisib
versus less than 3 months for historical controls1.
· Clinical findings validate the mechanism of action of
roginolisib: roginolisib reduces immune-suppressive immune cells
and chemokines, UM-related tumor clones (ctDNA) and PI3K-related
signaling indicating a rebalancing of the immune system.
Catherine Pickering, Chief Executive Officer of iOnctura,
said: "The Phase I DIONE-01 data
highlight the benefits of roginolisib for patients with uveal
melanoma and advanced cancers. Roginolisib's unique allosteric
binding mechanism has translated into a differentiated beneficial
clinical profile, including a doubling of overall survival compared
to historical controls in uveal melanoma. We are delighted to
announce these data support progression of roginolisib into a
randomized Phase II study."
Professor Michele Maio, University of Siena and Principal
Investigator of the roginolisib studies, added:
"Being able to continue to investigate roginolisib
in a randomized Phase II study is a positive step to understand
more about this already well tolerated molecule. Roginolisib has
given prolonged disease stabilization to patients with uveal
melanoma who have exhausted all other therapeutic options. So far,
these patients have maintained a good quality of life without major
limitations. I'm looking forward to seeing what the Phase II trial
delivers over the coming months."
Activation of trial sites for the
Phase II OCULE-01 study (NCT06717126)
investigating roginolisib versus investigator's choice in the
second-line+ treatment of uveal melanoma is ongoing.
ENDS
For more information
contact:
iOnctura
Catherine Pickering
Chief Executive Officer
T: +41 79 952 72 52
E:
c.pickering@iOnctura.com
Optimum Strategic Communications
Mary Clark / Vici Rabbetts / Elena
Bates
T: +44 208 078 4357
E:
ionctura@optimumcomms.com
About iOnctura
iOnctura is a clinical-stage
biopharmaceutical company combating neglected and hard-to-treat
cancers with precision oral small molecules that target cancers in
novel ways. The bold new treatments extend lives and improve
healthspans, changing the outlook for patients and their families.
Two therapeutic candidates have progressed into mid-stage clinical
development: roginolisib is the first allosteric modulator of
PI3Kδ; and cambritaxestat is the only autotaxin inhibitor in
clinical development to treat cancer. iOnctura BV is headquartered
in Amsterdam, The Netherlands with its wholly owned Swiss
subsidiary, iOnctura SA, located in Geneva, Switzerland. iOnctura
is backed by specialist institutional investors including Syncona,
EIC Fund, M Ventures, Inkef Capital, VI Partners and Schroders
Capital.
About roginolisib
Roginolisib is an allosteric
modulator of PI3Kδ with a unique chemical structure and binding
mode. The PI3K signaling pathway is one of the most commonly
dysregulated pathways in cancer and the precise targeting of the
PI3Kδ isoform delivers substantial anti-tumor effects with a
low-toxicity profile. Clinical data have demonstrated roginolisib's
excellent safety profile and sustained clinical activity in uveal
melanoma (UM), a rare eye cancer with few available treatments.
Site activation for the randomized Phase II OCULE-01 study in uveal
melanoma is ongoing, and Phase II studies in other cancers,
including non-small cell lung cancer and myelofibrosis, are in
planning.