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Apr 08
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Arrakis Therapeutics Enters Strategic Collaboration and License Agreement with Roche for Multi-Target Program Utilizing RNA-Targeted Small Molecule Drug Discovery Platform

By Arrakis Therapeutics, Press Release, Private Companies
Press Release.

 

Arrakis Therapeutics Enters Strategic Collaboration and License Agreement with Roche for Multi-Target Program Utilizing RNA-Targeted Small Molecule Drug Discovery Platform

Arrakis Receives $190 Million Upfront Cash Payment with Potential for Multi-Billion Dollar Future Payments

Waltham, MA – April 8, 2020 – Arrakis Therapeutics, a biopharmaceutical company pioneering the discovery of a new class of small molecule medicines that directly target RNA, today announced a strategic collaboration and license agreement with Roche (SIX: RO, ROG; OTCQX: RHHBY) for the discovery of RNA-targeted small molecule (rSM) drugs against a broad set of targets across all of Roche’s research and development areas. Under the terms of the agreement, Arrakis will lead discovery and research activities for each target to a defined point, at which time Roche will have the right to exclusively pursue further preclinical and clinical development. Arrakis will receive an upfront payment of $190 million in cash and may also receive preclinical, clinical, commercial and sales milestone payments and royalties for any resulting products. The aggregate potential value of future payments to Arrakis exceeds several billion dollars, subject to regulatory approvals and other conditions being met.

“We are excited to partner with Roche’s strong research and development teams. Together, we share a common vision of accessing new drug targets at the RNA level and thereby discovering novel medicines to treat diseases with high unmet medical need. The collaboration will increase the number of new treatments for patients arising from our proprietary rSM discovery platform,”

said Michael Gilman, Ph.D., Chief Executive Officer of Arrakis.

“In addition to the Roche collaboration, we are further building our capabilities and advancing our wholly-owned rSM programs for diseases unaddressed by today’s medicines.”

“Through our pioneering work in RNA biology, we have built a platform for creating and adapting drug discovery tools that allow us to predict and validate the structure of RNA targets, locate druggable pockets, identify drug-like hits, and conduct medicinal chemistry programs to discover a new class of RNA-targeted medicines optimized for potency, selectivity, and safety. This agreement with Roche underscores the value inherent in our rSM platform and will enable us to continue to make leading discoveries and further scientific contributions in the field,”

said Jennifer C. Petter, Ph.D., Founder and Chief Scientific Officer of Arrakis.

Arrakis is taking a broad approach, targeting multiple mechanisms across the lifecycle of RNA to establish a new paradigm for small-molecule drug discovery. The company’s discovery platform integrates leading-edge RNA bioinformatic and structural tools, curated chemical libraries, RNA-specific assays, and RNA-guided medicinal chemistry. In addition to collaborating with partners, Arrakis is developing an internal pipeline of rSMs to treat a range of serious illnesses including cancers and other diseases where strongly validated targets and drivers of disease have been identified but have proven challenging with other drug approaches and modalities.

About Arrakis Therapeutics

Arrakis Therapeutics is a biopharmaceutical company pioneering the discovery of a new class of medicines that directly target RNA. Arrakis is building a proprietary pipeline of RNA-targeted small molecule (rSM) medicines focused on cancer and genetically validated targets in other disease areas. The company brings together scientific leaders in RNA structure, chemistry and biology, along with a highly experienced management team and the backing of leading life sciences investors. The company is located in Waltham, Massachusetts. For more information, please visit www.arrakistx.com and engage with us on Twitter @ArrakisTx or on LinkedIn.

###

Investor Contact:
Will O’Connor
212-362-1200
will.oconnor@sternir.com

Media Contact:
Kathryn Morris
914-204-6412
kathryn@theyatesnetwork.com

Apr 07
Love0

Amphista Therapeutics raises $7.5m Series A round to advance targeted protein degradation assets in cancer

By Amphista Therapeutics, Press Release, Private Companies
Press Release.

 

April 7, 2020

  • Amphista’s potent bifunctional small molecules augment targeted protein degradation
  • Investors include Advent Life Sciences, the Scottish Investment Bank, the European Investment Fund and US-based BioMotiv

Glasgow, Scotland, 7 April 2020 – Amphista Therapeutics, a biopharmaceutical company creating first-in-class cancer therapeutics that harness the body’s natural processes to selectively and efficiently degrade and remove disease causing proteins, today announced the closing of a USD $7.5m Series A round, led by Advent Life Sciences. Seed round funders the Scottish Investment Bank, with backing from the Scottish Growth Scheme, and the European Investment Fund joined the round, along with new investor, US-based life sciences BioMotiv.

Amphista’s CEO Nicola Thompson said,

“This international financing provides Amphista with a firm foundation to underpin a Series B round to progress our oncology pipeline to the clinic. Our vision is to create a leading protein degradation company on the global stage that delivers ground-breaking new medicines to patients in areas of high unmet need.”

Raj Parekh, General Partner at lead investor Advent Life Sciences said,

“We are excited to support Amphista in its next stage of development. The Company has a potentially unique approach to targeted protein degradation when compared with traditional proteolysis targeting chimera (PROTAC®) platforms. We believe that Amphista has great potential with its differentiated proprietary technology to address traditionally undruggable targets.”

Satish Jindal, CEO of BioMotiv, and newly appointed Amphista Chairman, commented,

“We are a mission-driven accelerator, and we are excited by Amphista’s focus to rapidly produce potent bifunctional small molecules to augment the body’s own processes to remove disease-associated proteins. We see huge potential to accelerate Amphista’s breakthrough technology platform into medicines.”

Amphista’s scientific founder, Professor Alessio Ciulli, based at the University of Dundee, is an internationally renowned expert in the field of targeted protein degradation (TPD).

“Highly specific TPD is a transformative new modality for tackling previously undruggable targets with high therapeutic value.”

said Ciulli.

Amphista’s TPD small molecules instruct the cell to degrade the target directly rather than activating or inhibiting the target protein function. As protein-protein interactions are involved in disease progression, removing the target protein provides a clear therapeutic advantage over simple inhibition. Specifically, Amphista’s platform is independent of traditional E3 ubiquitin ligases used by the field, potentially expanding the available target scope of TPD approaches and should overcome recently identified PROTAC® resistance mechanisms.

Media contacts:

Amphista Therapeutics
CEO NicolaThompson
+447464974714
nicki@amphista.com

Scius Communications
Katja Stout
+447789435990
katja@sciuscommunications.com

About Amphista Therapeutics
Amphista Therapeutics is a biopharmaceutical company creating first-in-class therapeutics that help harness the body’s natural processes to selectively and efficiently degrade and remove disease causing proteins. The company’s pipeline of novel small molecules that cause targeted protein degradation (TPD) is focused on challenging diseases including cancer. Amphista is a spinout of renowned TPD expert Professor Alessio Ciulli’s labs at the University of Dundee, and is based in BioCity Glasgow, Scotland. The company is funded by leading life science investors including Advent Life Sciences, the European Investment Fund, the Scottish Investment Bank, and BioMotiv. For more information, visit http://www.amphista.com/.

About Advent Life Sciences
Advent Life Sciences founds and invests in early- and mid-stage life sciences companies that have a first- or best-in-class approach to unmet medical needs. The investing team consists of experienced professionals, each with extensive scientific, medical and operational experience, a long-standing record of entrepreneurial and investment success in the US and Europe and is particularly focused on supporting entrepreneurs and founders to take innovative new medical entities from concept to approval. The firm invests in a range of sectors within life sciences, principally drug discovery, enabling technologies and med tech, always with an emphasis on innovative, paradigm-changing approaches. Advent Life Sciences has a presence in the UK, US and France. For more information, please visit AdventLS.com.

About the Scottish Investment Bank
The Scottish Investment Bank (SIB) is the investment arm of Scotland’s national economic development agency, Scottish Enterprise, operating Scotland-wide in partnership with Highlands and Islands Enterprise (HIE).

SIB’s activities support Scotland’s SME funding market to ensure businesses with growth and export potential have adequate access to growth capital and loan funding. It helps ambitious Scottish companies get the right level of funding from the right sources at the right time, through building relationships with both domestic and international investors.

SIB manages a suite of co-investment funds including the Scottish Co-Investment Fund, the Scottish Venture Fund and the Energy Investment Fund on behalf of the Scottish Government. SIB is also an investor in Epidarex Capital’s Life Sciences Fund and administers the Scottish Loan Scheme, with funding secured from the Scottish Government’s Scottish Growth Scheme.

Furthermore, SIB provides funding into LendingCrowd, Scotland’s marketplace lender providing loans to SMEs, and Maven’s Regional Buyout Fund (MBO) that offers financial support for management buyouts (MBOs) and helps existing management teams acquire businesses from their owners so they can continue to flourish.

SIB’s team of financial readiness specialists help companies prepare for new investment and access appropriate finance.

About BioMotiv
https://www.biomotiv.com
BioMotiv is a mission-driven accelerator associated with The Harrington Project for Discovery & Development, a $340 million initiative focused on advancing early stage breakthrough discoveries from research institutions into medicines. Led by a highly accomplished and passionate team of veteran biopharma experts, BioMotiv’s innovative model efficiently aligns resources and capital to select, fund, manage and advance a portfolio of drug development programs.

Feb 18
Love0

New company takes aim at treatments for multiple sclerosis

By Pheno Therapeutics, Press Release, Private Companies
Press Release.

 

Novel treatments that may reverse the effects of multiple sclerosis (MS) will be investigated by a new drug-discovery company spun out from the University of Edinburgh.

Backed by Series A funding of £5 million over its first three years, Pheno Therapeutics will search for new drugs that aim to repair damage to the nervous system and significantly improve patients’ debilitating symptoms.

Building on original research by Professors Siddharthan Chandran and Neil Carragher of the University of Edinburgh, the company aims to develop new therapies for MS by identifying novel molecules that cause the body to repair or replace the damaged myelin sheath surrounding nerve cells.

This so-called remyelination process has the potential to slow or arrest the progressive disability caused by MS.

Pheno Therapeutics is supported by Advent Life Sciences, the London-based venture capital firm; the Scottish Investment Bank, with backing from the Scottish Government through the Scottish Growth Scheme; and independent medical research charity LifeArc. Together they have committed to invest £5 million over three years, subject to the company meeting certain milestone conditions.

Pheno Therapeutics co-founder Professor Siddharthan Chandran said:

“There are no interventions for people with later stage multiple sclerosis, which is a devastating and debilitating condition. The opportunity for this company is to bring new and repurposed therapeutics to clinical trials and, by doing so, meet an urgent and currently unmet need.”

Key to the company’s potential impact in MS treatments is the University’s advanced cell based technology platform, which enables the screening of large compound libraries on novel human cellular platforms, in addition to the founders’ and investors’ combination of clinical and drug discovery expertise.

Pheno Therapeutics intends to optimise the leads emerging from its cutting edge phenotypic screens via medicinal chemistry to deliver new candidate compounds that will progress through pre-clinical tests then proof-of-concept clinical trials.

MS affects more than 100,000 people in the UK and 2.5 million worldwide. Targeting the nervous system, including the brain and spinal cord, the disease occurs when the body’s immune system attacks the protective layer surrounding nerve cells called the myelin sheath, slowing or disrupting the electrical signals travelling along the nerves.

It causes a wide range of symptoms including problems with movement, vision, sensation and balance.

Current treatments mainly focus on the immune system aspects of the disease and reduce the severity and frequency of relapses. There is a significant medical need for novel neuroprotective agents that halt the disease progression and prevent long-term disability.

Pheno Therapeutics is a spinout company from the University of Edinburgh founded by Professors Chandran and Carragher, Advent Life Sciences and Dr Jon Moore, Operating Partner at Advent Life Sciences.

The formation of Pheno Therapeutics has been supported by Edinburgh Innovations, the University’s commercialisation service, which helped bring together the scientific and clinical expertise in partnership with Advent Life Sciences to launch the company.

Dr George Baxter, CEO of Edinburgh Innovations, said:

“I’m delighted to see this company launch with the support of such credible investors.

“Everyone involved is focused on driving the science forward, and we look forward to supporting the team as momentum continues to build, ultimately offering the promise of new treatments.”

Dr David Holbrook, head of LifeArc’s Seed Fund, said:

“At the Seed Fund, we look to use our translational expertise to invest in enterprises with a sound scientific concept and the potential to lead to new interventions that address patient needs.

“In the founders of Pheno Therapeutics and their research to induce myelin repair, we saw an appealing opportunity, particularly given the existing clinical needs in progressive MS. We are delighted to have reached an agreement to support Pheno Therapeutics translate their discoveries.”

ENDS

Notes
Image shows Professor Siddharthan Chandran of the University of Edinburgh, co-founder of Pheno Therapeutics.

Contact:
Gareth Overton
Communications Manager
Edinburgh Innovations
The University of Edinburgh
+44 (0)131 651 3426
+44 (0)7772 396 552
garethoverton@ei.ed.ac.uk

About Pheno Therapeutics
Pheno Therapeutics is an early stage biotechnology company focussed on the identification of novel neuroprotective therapies for multiple sclerosis. The company builds on the University of Edinburgh’s world-class research in phenotypic screening, stem cell biology and myelin biology. We will apply cutting edge drug discovery approaches to probe the pathways that can impact remyelination, selecting leads that will be developed into drugs and tested in early stage clinical trials.
https://www.phenotherapeutics.com/

About LifeArc
LifeArc is a self-funded medical research charity. Our mission is to advance translation of early science into health care treatments or diagnostics that can be taken through to full development and made available to patients. We have been doing this for more than 25 years and our work has resulted in a diagnostic for antibiotic resistance and four licensed medicines. Our success allows us to explore new approaches to stimulate and fund translation. We have our own drug discovery and diagnostics development facilities, supported by experts in technology transfer and intellectual property who also provide services to other organisations. Our model is built on collaboration, and we partner with a broad range of groups including medical research charities, research organisations, industry and academic scientists. We are motivated by patient need and scientific opportunity. Two funds help us to invest in external projects for the benefit of patients: our Philanthropic Fund provides grants to support medical research projects focused on the translation of rare diseases research and our Seed Fund is aimed at start-up companies focussed on developing new therapeutics and biological modalities.
Find out more about our work on www.lifearc.org or follow us on LinkedIn or Twitter.

About Advent Life Sciences
Advent Life Sciences founds and invests in early- and mid-stage life sciences companies that have a first- or best-in-class approach to unmet medical needs. The investing team consists of experienced professionals, each with extensive scientific, medical and operational experience, a long-standing record of entrepreneurial and investment success in the US and Europe and is particularly focused on supporting entrepreneurs and founders to take innovative new medical entities from concept to approval. The firm invests in a range of sectors within life sciences, principally drug discovery, enabling technologies and med tech, always with an emphasis on innovative, paradigm-changing approaches. Advent Life Sciences has a presence in the UK, US and France.  For more information, please visit AdventLS.com

About Edinburgh Innovations
Edinburgh Innovations is the University of Edinburgh’s commercialisation service. EI leads the University’s activities in industry engagement and business development, enterprise support for students and staff, and the identification, management and commercialisation of University intellectual property. EI also manages Old College Capital, the University’s venture fund.
https://edinburgh-innovations.ed.ac.uk

About the Scottish Investment Bank
The Scottish Investment Bank (SIB) is the investment arm of Scotland’s national economic development agency, Scottish Enterprise, operating Scotland-wide in partnership with Highlands and Islands Enterprise (HIE).

SIB’s activities support Scotland’s SME funding market to ensure businesses with growth and export potential have adequate access to growth capital and loan funding. It helps ambitious Scottish companies get the right level of funding from the right sources at the right time, through building relationships with both domestic and international investors.

SIB manages a suite of co-investment funds including the Scottish Co-Investment Fund, the Scottish Venture Fund and the Energy Investment Fund on behalf of the Scottish Government. SIB is also an investor in Epidarex Capital’s Life Sciences Fund and administers the Scottish Loan Scheme, with funding secured from the Scottish Government’s Scottish Growth Scheme.

Furthermore, SIB provides funding into LendingCrowd, Scotland’s marketplace lender providing loans to SMEs, and Maven’s Regional Buyout Fund (MBO) that offers financial support for management buyouts (MBOs) and helps existing management teams acquire businesses from their owners so they can continue to flourish.

SIB’s team of financial readiness specialists help companies prepare for new investment and access appropriate finance.

Jan 13
Love0

KaNDy Therapeutics Announces Positive Phase 2b Data in Post-Menopausal Women with its Lead Non-Hormonal Product NT-814

By KaNDy Therapeutics, Press Release
Press Release.

 

  • Phase 2b dose range finding study showed rapid and highly significant reductions in the frequency of hot flashes (primary endpoint) for the full 12-week treatment period~
  • Reduction in hot flashes was associated with statistically significant improvements in quality of life, mood and sleep – all key secondary endpoints~
  • All doses of NT-814 were well tolerated during the study, demonstrating a safety profile that supports progression to Phase 3

Stevenage, UK, 13 January 2020 – KaNDy Therapeutics, a UK clinical-stage biotech company, today announces positive data from the Phase 2b “SWITCH-1” clinical trial with its lead non-hormonal drug candidate, NT-814, for the treatment of symptoms of the menopause.

Following on from the clear benefits NT-814 demonstrated in the Phase 2a RELENT-1 study, the SWITCH-1 trial provides further compelling evidence that NT-814, a first in class, once-daily, oral neurokinin-1,3 receptor antagonist, can produce a rapid and marked reduction in the most troublesome and frequent symptoms of the menopause, hot flashes and night sweats (vasomotor symptoms). The clinical relevance of the marked improvements shown on the vasomotor symptom endpoints was supported by highly statistically significant improvements across patient reported assessments of quality of life, mood and sleep.

The SWITCH-1 study was a randomised, double-blind, placebo-controlled trial conducted in the US, UK and Canada.  One hundred and ninety-nine women experiencing at least 7 moderate or severe hot flashes/flushes (HF) per day were recruited into the study and randomised to receive one of four doses of NT-814 or placebo. Treatment with NT-814 once daily for 12 weeks at the most effective dose evaluated resulted in:

•    Statistically significant reductions compared to placebo in average hot flash frequency (primary endpoint), starting during the first week of treatment and continuing throughout the 12-week treatment period. Least squares mean reductions in average hot flash frequency were -6.7 for NT-814 vs -2.7 for placebo at week 4, and -7.8 vs -4.7 at Week 12 (p<0.0001 and p=0.0092, respectively).

•    Marked improvements in all key secondary endpoints: improved quality of life was shown by highly    significant improvements over placebo in the MenQoL menopause-specific quality of life scores, benefits on mood were demonstrated by significant improvements in the Beck Depression Inventory (II), and improved quality of sleep  was shown by statistically significant improvements compared to placebo in the Pittsburgh Sleep Quality Index scores.

•    NT-814 was well tolerated across the dose range with a safety profile that supports progression to Phase 3.

Dr. James A. Simon, Clinical Professor of Reproductive Endocrinology & Infertility at George Washington University, and the study’s Lead Investigator, commented:

“These top-line results of the SWITCH-1 study are very exciting. They demonstrate that NT-814, a truly novel therapy, offers a rapidly effective, non-hormonal approach to treating menopausal hot flashes and night sweats, debilitating symptoms of menopause. Unique to this trial, patients also reported improvements in quality of life, mood and sleep with NT-814.”

Dr Mary Kerr, Co-Founder and CEO KaNDy Therapeutics, said:

“The SWITCH-1 study started in November 2018, and so we are excited to share such positive results on schedule. The data confirms and validates Phase 2a observations, providing more evidence that neurokinins are fundamental to sex hormone biology and the pathophysiology of the menopause, resulting in almost immediate symptom relief. The Company looks forward to presenting these data at future scientific meetings and discussing it with regulatory agencies in advance of progressing the compound into pivotal registration studies.”

For more information, please contact:

KaNDy Therapeutics
Email: info@kandytherapeutics.com

Consilium Strategic Communications
Mary-Jane Elliott/ Lindsey Neville/ Carina Jurs
Tel: +44 (0) 20 3709 5700
KaNDyTherapeutics@consilium-comms.com

About the SWITCH-1 Study: The Phase 2b SWITCH-1 study was a randomised, double-blind, placebo-controlled study conducted at 25 sites in the UK, US and Canada.  It included an adaptive randomisation design that enabled the randomisation ratio to be modified to focus on doses of greatest interest based on emerging data. The study was initiated in November 2018 and completed, ahead of schedule, at the end of 2019.  A total of 199 post-menopausal women experiencing at least 7 moderate or severe HFs per week were recruited into the study and randomized to receive one of four doses of NT-814 or placebo. Study drug was taken once daily in the evening for 12 weeks. Subjects completed electronic diaries twice daily for the two weeks before and throughout treatment and underwent routine safety assessments periodically throughout the trial. Patient reported assessments of sleep, quality of life and mood were also completed periodically during study visits. Further information on the study design can be found on www.clinicaltrials.gov and full results of the study will be published at scientific congresses and in peer-reviewed journals over the coming months.

NT-814 is an orally administered, potent and selective small molecule dual antagonist of both the neurokinin-1 and 3 receptors under development by KaNDy as a therapy for a range of Women’s Health conditions. NT-814 addresses vasomotor symptoms by modulating a group of oestrogen sensitive neurones in the hypothalamus in the brain (the KNDy neurones), that in menopausal women due to the absence of oestrogen, become hyperactive and consequently disrupt body heat control mechanisms resulting in the debilitating vasomotor symptoms of hot flashes and night sweats.

KaNDy Therapeutics is a clinical-stage company focused on optimizing the potential of NT-814 in the treatment of common, chronic debilitating female sex-hormone related conditions. These conditions, such as post-menopausal vasomotor symptoms, are debilitating for women often over many years and associated with significant healthcare and economic costs. NT-814 is wholly owned by KaNDy.

Dec 10
Love0

Bioncotech Therapeutics Announces Oncology Clinical Trial Collaboration with MSD Phase II Trial

By Press Release
Press Release.

 

Madrid, Spain – December 10, 2019: Bioncotech Therapeutics (“Bioncotech”), a clinical-stage biopharmaceutical company developing RNA-based therapies against cancer, announces it has entered into a Phase II clinical trial collaboration with a subsidiary of Merck & Co., Inc., Kenilworth, New Jersey, U.S.A., known as MSD outside the U.S. and Canada.

The collaboration will focus on the Phase II clinical evaluation of the combination of BO-112, Bioncotech’s lead program, and KEYTRUDA® (pembrolizumab), MSD’s anti-PD-1 therapy, in patients with select advanced stage solid tumors with liver metastases. The trial will evaluate if the combination of stimulation of the innate immune system by direct intra-tumoral administration of BO-112, combined with systemic administration of pembrolizumab, shows safety and efficacy in patients with tumors that are poorly or only moderately responsive to monotherapy with an anti-PD1 agent.

“This collaboration is an important next step in the clinical development of BO-112 and the potential of the innate immune system and intra-tumoral route of administration to provide improved outcomes for cancer patients,”

said Marisol Quintero, CEO of Bioncotech.

“We consider MSD, with its extensive expertise in immune-oncology, as the ideal partner for Bioncotech to complete this study.”

“Early clinical data suggest BO-112 induces immunological changes in the tumor microenvironment and beyond, which may render previously anti-PD1 refractory tumors susceptible to checkpoint inhibition,”

added Dominique Tersago, CMO of Bioncotech.

“Cancers that have metastasized to the liver present a significant therapeutic challenge. By targeting the metastatic liver lesions as the site for intra-tumoral injection we aim to reduce organ-related variability of the injection site, and also evaluate if BO-112 has potential to break through the generally immune tolerant hepatic environment and trigger or improve responsiveness to anti-PD1 therapy.”

KEYTRUDA® is a registered trademark of Merck Sharp & Dohme Corp, a subsidiary of Merck & Co., Inc., Kenilworth, NJ, U.S.A.

For more information, please contact:

Bioncotech Therapeutics S.L
info@bioncotech.com
Marisol Quintero, CEO
Mo PR Advisory
Tel: +44 (0) 7876 444977 / 07860 361746
Mo Noonan / Jonathan Birt

Notes to Editors

About Bioncotech
Bioncotech is a Spanish biopharmaceutical company focusing on the development of RNA-based therapies against validated and novel targets in cancer and immune cells.

About BO-112
Bioncotech’s lead candidate, BO-112, is a formulated non-coding double stranded RNA (dsRNA) that acts as an agonist to toll-like receptor 3 (TLR-3), and the cytosolic helicases melanoma differentiationassociated gene 5 (MDA5) and retinoic acid-inducible gene I (RIG-I). It is a stimulator of the innate immune system, activates dendritic cells and induces interferons (IFNs), and also has been shown to trigger apoptosis and cause immunogenic cell death in tumor cells. BO-112 is being evaluated in combination with anti-PD1 treatment in a Phase I clinical trial. For information on clinical trials, please visit www.clinicaltrials.gov.

Nov 14
Love0

Axonics® Announces U.S. Food & Drug Administration Approval for its Sacral Neuromodulation System for Urinary Clinical Indications

By Axonics, Press Release
Press Release.

 

IRVINE, Calif.–(BUSINESS WIRE)– Axonics Modulation Technologies, Inc. (NASDAQ: AXNX), a medical technology company that has developed and is commercializing novel implantable rechargeable sacral neuromodulation (“SNM”) devices for the treatment of bladder and bowel dysfunction, today announced the approval of the Axonics r-SNM® System by the United States Food & Drug Administration (“FDA”) for the clinical indications of overactive bladder (“OAB”) and urinary retention.

The FDA premarket approval (“PMA”) grants Axonics the right to market its product in the United States for the clinical indications of OAB (urinary urge incontinence and urinary urge frequency) as well as urinary retention, representing the largest segment of the market for SNM devices. The FDA approval follows the Company’s September approval for the clinical indication of fecal incontinence, which, according to published clinical studies, is a co-morbidity reported by as many as a third of patients presenting with urinary urge incontinence.

The FDA approval was supported by the results of a detailed review of technical data and the positive results of the Axonics ARTISAN-SNM 129-patient pivotal clinical study that met all primary and secondary endpoints and demonstrated 90% efficacy for all implanted urinary incontinence patients at six months, as well as published clinical literature.

The Axonics r-SNM System is the first rechargeable SNM system approved for sale in the U.S., Europe, Canada and Australia. It is also the only SNM device approved for patients to undergo full-body MRI scan without the necessity of having the device explanted. The FDA approval includes the claim of a 15-year functional life, which is in contrast to the incumbent company’s legacy device which requires replacement on average every four years. In addition to many other differentiating attributes, the system includes a patented tined lead, user-friendly accessories, such as a wireless charging system optimized for infrequent charging, a small easy-to-use key-fob patient remote control and an intuitive clinician programmer that facilitates lead placement and stimulation programming. The long-lived miniaturized neurostimulator is approximately the size of a USB stick.

“If we consider the millions of women who have tried and discontinued OAB pharmaceuticals, the market opportunity for Axonics goes well beyond the approximate $700 million of revenue that is currently being generated by the incumbent’s non-rechargeable SNM device,“

said Raymond W. Cohen, CEO of Axonics.

”Based on feedback from U.S. physicians, we believe the SNM market is poised to dramatically expand over the next few years given our fuss-free, highly efficacious, long-lived, full-body MRI compatible device. Our U.S. commercial team of 146 territory managers, clinical support specialists and sales managers rivals the size of the incumbent’s field staff, and is focused on calling on the top 1,000 urologists, urogynecologists and colorectal surgeons who account for approximately 80% of the SNM implants in the U.S.”

As previously announced, Axonics is hosting a conference call with the investment community today, Thursday, November 14, 2019, at 4:30 p.m. Eastern Time to discuss 2019 third quarter financial results and recent business developments.

Interested parties may access the live call via telephone by dialing (866) 687-5771 (U.S.) or (409) 217-8725 (International) and using passcode 4373989. A live webcast of the call may be accessed by visiting the Events & Presentations page of the investors section of the Company’s website at ir.axonicsmodulation.com.

ARTISAN-SNM pivotal study and the PMA approval process with the U.S. FDA

ARTISAN-SNM was conducted at 14 centers in the U.S. and five centers in Western Europe. The study met all primary and secondary endpoints and demonstrated that implanted patients received clinically meaningful and statistically significant improvements in urinary incontinence symptoms and quality of life. At the study endpoint of six months post-implant, 90% of all 129 implanted patients were therapy responders. At one year, the efficacy remained consistent with an 89% responder rate. The vast majority of implanted patients experienced a significant reduction in urgency incontinence episodes and approximately one third were completely dry. No serious device-related adverse events were reported.

After the initial filing in December 2018, the Company filed a number of amendments, including complete test data to support full-body MRI labeling for the implantable components of the Axonics r-SNM System and the full six-month data set from its pivotal study. The FDA conducted two detailed PMA audits of the Axonics quality system and manufacturing during the review period. The audits were completed without any negative observations.

About Axonics Modulation Technologies, Inc. and Sacral Neuromodulation

Axonics, based in Irvine, Calif., has developed and is commercializing novel implantable SNM devices for patients with urinary and bowel dysfunction. These conditions are caused by a miscommunication between the bladder and the brain and significantly impacts quality of life. Overactive bladder affects an estimated 87 million adults in the U.S. and Europe. Another estimated 40 million adults are reported to suffer from fecal incontinence/accidental bowel leakage. SNM therapy has been employed to reduce symptoms and restore pelvic floor function for the past two decades. Reimbursement coverage is well established in the U.S. and Europe. The Axonics System is the first rechargeable SNM system approved for sale in the world, and the first to gain full-body MRI conditional labeling. For more information, visit the Company’s website at www.axonics.com.

Forward-Looking Statements

Statements made in this press release that relate to future plans, events, prospects or performance are forward-looking statements as defined under the Private Securities Litigation Reform Act of 1995. Words such as “planned,” “expects,” “believes,” “anticipates,” “designed,” and similar words are intended to identify forward-looking statements. While these forward-looking statements are based on the current expectations and beliefs of management, such forward-looking statements are subject to a number of risks, uncertainties, assumptions and other factors that could cause actual results to differ materially from the expectations expressed in this press release, including the risks and uncertainties disclosed in Axonics filings with the Securities and Exchange Commission, all of which are available online at www.sec.gov. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. Except as required by law, Axonics undertakes no obligation to update or revise any forward-looking statements to reflect new information, changed circumstances or unanticipated events.

View source version on businesswire.com: https://www.businesswire.com/news/home/20191114005205/en/

Contacts

Axonics’ Contact
Axonics Modulation Technologies, Inc.
Dan Dearen, +1-949-396-6320
President & Chief Financial Officer
ir@axonics.com

Investor & Media Contact
W2Opure
Matt Clawson, +1-949-370-8500
mclawson@w2ogroup.com

Source: Axonics Modulation Technologies, Inc.

Nov 11
Love0

F2G Receives US FDA Breakthrough Therapy Designation for Olorofim

By F2G, Press Release, Private Companies
Press Release.

 

MANCHESTER, UK / VIENNA, Austria – November 11, 2019 – F2G Ltd, a UK- and Austria-based biotech company developing novel therapies for life-threatening systemic fungal infections, announced today that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to its lead first-in-class candidate, olorofim (formerly F901318) for the indication of ‘Treatment of invasive mold infections in patients with limited or no treatment options, including aspergillosis refractory or intolerant to currently available therapy, and infections due to Lomentospora prolificans, Scedosporium, and Scopulariopsis species’. Olorofim is the first antifungal agent to be granted Breakthrough Therapy designation.

Olorofim is currently being investigated in an open-label single-arm Phase 2b study (ClinicalTrials.gov Identifier: NCT03583164) in patients with proven invasive fungal disease (IFD) or probable invasive aspergillosis (IA) and either refractory disease, resistance, or intolerance to available agents. Olorofim has been well tolerated across more than 10 years of patient dosing days with a median therapy duration of 12 weeks. Preliminary data from this study were provided to the FDA as part of the Breakthrough Therapy designation submission.

Breakthrough Therapy designation is an FDA process designed to expedite the development and review of drugs that are intended to treat a serious or life-threatening condition and is granted based on preliminary clinical evidence indicating that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints.

Breakthrough Therapy designation conveys all the features of fast track designation, more intensive FDA guidance on an efficient drug development program, an organisational commitment by FDA to involve senior managers, and eligibility for rolling review and priority review.

Commenting on the news, Ian Nicholson, CEO of F2G Ltd, said:

“The granting of FDA Breakthrough Therapy designation is a truly transformational step for our company and will support our goal of rapidly developing this novel treatment for patients suffering from serious and life-threatening fungal infections. Olorofim acts via a novel and differentiated mechanism to traditional antifungals, and preliminary data have indicated that it is efficacious in tackling life-threatening invasive fungal infections that cannot be managed with currently approved agents.

“Our Phase 2b programme is on track with over 40 patients recruited in Europe, Australia and the US. We look forward to working closely with the FDA to accelerate development of this therapy for patients having limited or no approved treatment options for an invasive mold infection.”

Professor Sharon Chen, Westmead Hospital Sydney and Principal Investigator for the Phase 2b study said:

“This news is very exciting for clinicians caring for patients with these very serious, and devastating mold infections. We have had limited treatment options for many years and now the news about olorofim brings realistic hope that we can cure these previously treatment–refractory infections.”

Contact:
F2G Ltd
Ian Nicholson | Chief Executive Officer
Ralf Schmid | Chief Financial Officer
Tel: +44 (0)161 785 1271 (UK) / +43 (0)1 997 4267 (A)

Optimum Strategic Communications
Mary Clark / Supriya Mathur / Charlotte Hepburne-Scott
Email: F2G@optimumcomms.com
Tel: +44 (0) 203 950 9144

Notes to Editors:

About Olorofim / Clinical trial
The Phase 2b study for olorofim (ClinicalTrials.gov Identifier: NCT03583164) is a global open-label study in patients who have limited treatment options for difficult-to-treat invasive fungal mold infections such as azole-resistant aspergillosis, scedosporiosis, lomentosporiosis, and other rare mold infections. 26 centres are currently open in six countries (AU, BE, ES, NL, USA, IS) and a further 20 will open in 2019/2020. Olorofim is being developed both as IV and oral formulations.

About F2G
F2G is a world-leading UK- and Austria-based biotech company (F2G Ltd and F2G Biotech GmbH) focused on the discovery and development of novel therapies to treat life-threatening invasive fungal infections. F2G has discovered and developed a completely new class of antifungal agents called the orotomides. The orotomides selectively target fungal dihydroorotate dehydrogenase (DHODH), a key enzyme in the de novo pyrimidine biosynthesis pathway. This is a completely different mechanism from that of the currently marketed antifungal agents and gives the orotomides fungicidal activity against a broad range of rare and resistant fungal mold infections. Olorofim (formerly, F901318) is F2G’s leading candidate from this class and is in a Phase 2b open-label study focussing on rare and resistant invasive fungal infections such as aspergillosis (including azole-resistant strains), scedosporiosis (including lomentosporiosis). Olorofim has received orphan drug status from the European Medicines Agency for the treatment of invasive aspergillosis and invasive scedosporiosis. Olorofim is being developed both as IV and oral formulations. www.f2g.com

Sep 09
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Axonics® Announces U.S. Food & Drug Administration Approval for its Sacral Neuromodulation System

By Axonics, Press Release
Press Release.

 

IRVINE, Calif.–(BUSINESS WIRE)– Axonics Modulation Technologies, Inc. (NASDAQ: AXNX), a medical technology company that has developed and is commercializing a novel implantable rechargeable sacral neuromodulation (“SNM”) device for the treatment of urinary and bowel dysfunction, today announced the approval of the Axonics r-SNM® System by the United States Food & Drug Administration (“FDA”). The Axonics System is the first rechargeable SNM system approved for sale in the United States, Europe, Canada and Australia.

The FDA approval grants Axonics the right to market its product in the United States for the clinical indication of fecal incontinence. The approval includes the claim of a 15-year functional life and the ability of patients to undergo full-body MRI scans without the necessity of having the device explanted.

Axonics has an additional pre-market approval (“PMA”) filing currently under review with the FDA for the clinical indications of overactive bladder (urinary urgency incontinence and urinary urgency frequency) as well as urinary retention for which the Company anticipates a determination in the near term.

Raymond W. Cohen, CEO of Axonics, commented,

“If we consider the millions of women who are suffering in silence with bladder and bowel dysfunction, we believe the market opportunity for Axonics goes well beyond the existing approximately $650 million of revenue that is currently being generated by the incumbent’s non-rechargeable SNM device. We believe the number of patients seeking SNM treatment will expand dramatically over the next few years given our fuss-free, long-lived, full body MRI-compatible device. In the months prior to FDA approval, we invested significant time and resources in building inventory to support our fully trained, U.S. commercial team which now includes 145 territory managers, clinical support specialists and sales managers strategically located around the country. We plan to begin shipping product to U.S. physicians and hospitals during the fourth quarter of 2019, following the fulfillment of customary, pre-launch regulatory requirements.”

About Sacral Neuromodulation and the Axonics r-SNM System

Sacral neuromodulation is used to treat bladder and bowel dysfunction. These conditions are caused by a miscommunication between the bladder and the brain and significantly impacts quality of life. Overactive bladder affects an estimated 87 million adults in the U.S. and Europe. Another estimated 40 million adults are reported to suffer from fecal incontinence/accidental bowel leakage. SNM therapy has been employed to reduce symptoms and restore pelvic floor function for the past two decades. Reimbursement coverage is well established in the U.S. and Europe. The Axonics r-SNM System is the first rechargeable SNM system approved for sale in the world, and the first to gain full-body MRI conditional labeling. The Axonics r-SNM System offers a long-lived miniaturized neurostimulator that is approximately the size of a USB stick and is qualified to last 15 years in the body as compared to the incumbent’s device which is non-rechargeable and requires patients to undergo a surgical intervention to replace the device every 3 to 5 years. In addition to full body MRI conditional labeling, the Axonics r-SNM System features many differentiating attributes. These include a patented tined lead, user-friendly accessories, a wireless charging system optimized for infrequent charging, a small easy-to-use patient remote control and an intuitive clinician programmer that facilitates lead placement and stimulation programming.

PMA approval process with the U.S. FDA

Determination by the FDA of the safety and effectiveness of the Axonics r-SNM System was supported by the results of a detailed review of technical data, published SNM clinical literature and the ARTISAN-SNM 129-patient pivotal clinical study. ARTISAN-SNM was conducted at 14 centers in the U.S. and five centers in Western Europe and met all primary and secondary endpoints. No serious device-related adverse events were reported. During the review period the FDA conducted two detailed PMA audits of the Axonics quality system and manufacturing. The audits were completed without any negative observations.

Conference Call and Webcast

The Company will host a conference call with the investment community to discuss the FDA approval and recent business developments, on September 9, 2019, at 9:00 a.m. Eastern Time.

Interested parties may access the live call via telephone by dialing (866) 687-5771 (U.S.) or (409) 217-8725 (International) and using passcode 3147028. A live webcast of the call may be accessed by visiting the Events & Presentations page of the investors section of the Company’s website at ir.axonicsmodulation.com. A replay of the webcast will be available shortly after the conclusion of the call and will be archived on the Company’s website for 90 days.

About Axonics Modulation Technologies, Inc.

Axonics, based in Irvine, CA, has developed and is commercializing a novel implantable SNM device for patients with urinary and bowel dysfunction. For more information, visit the Company’s website at www.axonicsmodulation.com

Forward-Looking Statements

Statements made in this press release that relate to future plans, events, prospects or performance are forward-looking statements as defined under the Private Securities Litigation Reform Act of 1995. Words such as “planned,” “expects,” “believes,” “anticipates,” “designed,” and similar words are intended to identify forward-looking statements. While these forward-looking statements are based on the current expectations and beliefs of management, such forward-looking statements are subject to a number of risks, uncertainties, assumptions and other factors that could cause actual results to differ materially from the expectations expressed in this press release, including the risks and uncertainties disclosed in Axonics filings with the Securities and Exchange Commission, all of which are available online at www.sec.gov. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. Except as required by law, Axonics undertakes no obligation to update or revise any forward-looking statements to reflect new information, changed circumstances or unanticipated events.

View source version on businesswire.com: https://www.businesswire.com/news/home/20190909005226/en/

Contacts

Axonics’ Contact
Axonics Modulation Technologies, Inc.
Dan Dearen, +1-949-396-6320
President & Chief Financial Officer
ir@axonics.com

Investor & Media Contact
W2Opure
Matt Clawson, +1-949-370-8500
mclawson@w2ogroup.com

Source: Axonics Modulation Technologies, Inc.

Apr 26
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Acutus Medical’s New Contact Mapping Software Receives CE Mark

By Acutus Medical, Press Release, Publicly Listed
Press Release.

 

FDA Clears Robust Second Generation AcQMap Platform

CARLSBAD, Calif. and MANNHEIM, Germany, April 26, 2019 – Acutus Medical today announced CE mark approval for AcQMap contact mapping software, offering expanded functionality that provides physicians with more options to inform individualized patient therapy. The company also announced FDA clearance of its second generation AcQMap platform. All AcQMap systems retain the unique ability to quickly reconstruct atrial anatomy using ultrasound and map electrical patterns using non-contact, charge density technology, creating ultra-high-resolution 3D images in real time.
AcQMap enables physicians to uncover the electrical activation pattern of the heart with real-time visualization during electrophysiology procedures.

“The contact mapping speed with the new Acutus system was extremely fast, which made it very easy to use,”

said Dr. Christian Meyer, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.

“Clinically, having this capability available on one system that also does non-contact mapping allows me to do exactly what makes sense case-by-case. For routine cases, my treatment strategy can be confirmed using conventional mapping catheters. In more complex cases, such as atrial fibrillation, I can gather more comprehensive data about each patient’s anatomy and arrhythmia in real time with the non-contact charge density catheter, making AcQMap the complete atrial mapping solution.”

Cardiac ablation treatment can improve quality of life for patients with atrial arrhythmias such as atrial fibrillation (AF). AF affects more than 33 million people globally and increases the risk of stroke and heart failure.1,2 AcQMap enables physicians to uncover the electrical activation pattern of the heart with real-time visualization during electrophysiology procedures. With non-contact mapping, physicians see full-chamber, continuous beat-by-beat arrhythmia conduction and can locate arrhythmias outside the pulmonary veins to inform their treatment strategy, enabling an iterative map-ablate-remap approach.

“With the introduction of contact mapping software in Europe, AcQMap is unquestionably the most agile and comprehensive cardiac imaging and mapping system available, offering full spectrum arrhythmia visualization in under three minutes. No other technology offers the suite of solutions incorporated into AcQMap,”

said Acutus Medical CEO Vince Burgess.

“Our second generation system is engineered to be the cornerstone platform for electrophysiology labs for many years to come. We worked alongside physician partners to understand complex challenges and simple needs to improve the efficiency and safety of electrophysiology procedures. This next-gen AcQMap system has the capability to exponentially improve the day-to-day clinical utility of cardiac mapping and deliver long-term value by offering health systems a comprehensive solution for electrophysiologists and their patients.”

Acutus Medical’s contact mapping software is compatible with both first and second generation AcQMap platforms and will be installed on all European systems in Q3. The second generation AcQMap system is built to support future software innovations and will be available in the U.S. in Q3.

About Acutus Medical
Acutus Medical is a dynamic arrhythmia care company focused on developing distinct, innovative technologies that provide physicians and patients with improved results. At Acutus, we know that seeing is better than believing. Diagnosing and treating patients with atrial arrhythmias requires eliminating the unknown. Acutus’ advanced cardiac imaging and mapping system provides real-time arrhythmia visualization displaying the heart’s true activation pattern, turning the chaos of an atrial arrhythmia into a clear vision for electrophysiologists. Founded in 2011, Acutus is based in Carlsbad, California.

References
1 Staerk L, Sherer JA, Ko D, et al. Atrial Fibrillation. Circ Res 2017;120:1501-17. DOI: 10.1161/CIRCRESAHA.117.309732.
2 Chugh, S.S., Havmoeller, R., Narayanan, K. et al. Worldwide epidemiology of atrial fibrillation: a Global Burden of Disease 2010 Study. Circ Feb 25. 2014; 129: 837–847. DOI: 10.1161/CIRCULATIONAHA.113.005119.

Indications for Use
In the U.S., the AcQMap System is intended for use in patients for whom electrophysiology procedures have been prescribed. When used with the AcQMap Catheters, the AcQMap System is intended to be used to reconstruct the selected chamber from ultrasound data for purposes of visualizing the chamber anatomy and displaying electrical impulses as either dipole density-based or voltage-based maps of complex arrhythmias that may be difficult to identify using conventional mapping systems alone. And, when used with the specified Patient Electrodes, the AcQMap System is intended to display the position of AcQMap Catheters and conventional electrophysiology (EP) catheters in the heart.

In the EU, the AcQMap System is intended for use in patients for whom electrophysiology procedures have been prescribed. When used with the AcQMap Catheters, the AcQMap System is intended to be used in the right and/or left atria to visualize the selected chamber and display atrial electrical impulses. And, when used with patient electrodes, the AcQMap System is intended to display the position of AcQMap Catheters and conventional electrophysiology (EP) catheters in the heart. Or, when used with conventional electrophysiology catheters, the AcQMap System provides information about electrical activity of the heart and about catheter location during the procedure.

U.S. Media Contacts
Holly Winder
Head of Marketing & Communications
Acutus Medical
(503) 805-3683
holly.windler@acutus.com

Mackenzie Mohr / Jessica Stebing
Levitate
(260) 408-5383
acutus@levitatenow.com

Apr 18
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Arrakis Therapeutics Announces $75 Million Series B Financing To Advance a New Class of Small-Molecule Medicines Targeting RNA

By Arrakis Therapeutics, Press Release, Private Companies
Press Release.

 

April 18, 2019

Michael Gilman, Ph.D., to expand role to full-time CEO to lead company to clinical stage
Having established first-in-industry RNA-targeted Small Molecule (rSM) platform, Arrakis will focus pipeline in oncology and genetically validated targets in other diseases
Financing led by venBio Partners and Nextech Invest

Waltham, Mass., April 18, 2019 – Arrakis Therapeutics, a biopharmaceutical company pioneering the discovery of a new class of small-molecule medicines that directly target RNA, has completed a $75 million Series B financing co-led by venBio Partners and Nextech Invest, with participation by new investors Omega Funds, HBM Healthcare Investments, GV (formerly Google Ventures), WuXi AppTec Venture Fund, and Alexandria Venture Investments, as well as all existing investors, Canaan Partners, Advent Life Sciences, Pfizer Ventures, Celgene Corporation, Osage University Partners and the estate of Henri Termeer. In addition, the company announced that Michael Gilman, Ph.D., will expand his role to full-time Chief Executive Officer in addition to continuing to serve as Chairman of the Board of Directors.

“With this financing and outstanding syndicate of investors, Arrakis will leap to the next stage of realizing our vision of creating a new class of medicines with RNA-targeted small molecules, or rSMs,”

said Dr. Gilman.

“We have built an end-to-end platform for the discovery of rSMs by creating or adapting tools that allow us to predict and validate the structure of RNA targets, locate druggable pockets, identify drug-like hits, and conduct medicinal chemistry programs to improve potency, selectivity, and safety. We are now operating this platform at scale to create a pipeline of utterly novel rSM medicines. I am excited to commit my full effort, along with the Arrakis team and our investors, to drive our discoveries into powerful new medicines for patients.”

The proceeds from the Series B financing will enable Arrakis to build a pipeline of novel RNA‑targeted small molecules, with the goal of reaching clinical testing with one or more candidates. The company will focus its internal drug development in oncology and genetically validated targets in other disease areas. In addition, the funding will enable Arrakis to continue to refine and expand its first-in-industry rSM discovery platform, including a high-throughput, comprehensive suite of computational tools, biophysical and cellular assays, and chemical libraries that are uniquely designed to create new small-molecule drugs for RNA targets.

“Arrakis Therapeutics is the clear leader in the emerging rSM field. We are pleased to support their differentiated strategy to transform the drug discovery toolkit to focus on RNA and open hundreds of important new targets to therapeutic intervention. Arrakis’ deeply experienced team is uniquely qualified to execute this strategy,”

said Richard Gaster, M.D., Ph.D., Principal at venBio Partners.

“The demand for more effective and better tolerated cancer drugs is high, creating the biggest and fastest growing market in healthcare. RNA is now a validated therapeutic target, and drugging RNA with conventional small-molecule medicines can provide cancer patients with options not achievable by any other means,”

said Jakob Loven, Ph.D., Partner at Nextech Invest.

In conjunction with the Series B financing, Dr. Gaster and Dr. Loven will join the Arrakis Board of Directors.

In its next stage of growth, Arrakis will employ its proprietary rSM drug discovery platform to discover novel RNA-targeted small molecules and advance lead candidates toward clinical testing. Since the company’s inception, Arrakis has systematically reconfigured drug discovery tools for RNA targets and achieved the following:

A systematic approach to identify and validate druggable RNA targets, enabling the company to target multiple aspects of RNA biology; these approaches include:

  • in silico tools to identify druggable RNA targets at scale;
  • high-throughput molecular biology tools to validate these targets.

Multiple screening methods for identifying tractable targets and chemical matter, including:

  • screening of hundreds of targets to date;
  • identification of druggable RNA binding pockets;
  • deriving the principles of molecular recognition of RNA.

Advancement of rSM drug programs against novel RNA targets and with strong intellectual property, including:

  • launch of four programs against RNA targets that encode proteins that are otherwise undruggable;
  • chemical biology tools to elucidate the mechanism of action and selectivity of drug candidates;
  • an intellectual property estate comprising new methods, compounds and targets.

About Arrakis Therapeutics
Arrakis Therapeutics is a biopharmaceutical company pioneering the discovery of a new class of medicines that directly target RNA. The company has developed a proprietary platform to identify new RNA targets and drug candidates to treat diseases unaddressed by today’s medicines. Arrakis is building a proprietary pipeline of RNA-targeted small molecules (rSMs) focused on cancer and genetically validated targets in other disease areas. The company brings together scientific leaders in RNA structure, chemistry and biology, along with a highly experienced management team and the backing of leading life sciences investors. The company is located in Waltham, Massachusetts. Please visit www.arrakistx.com.