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May 04
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AviadoBio to Present Pre-clinical Data on its Gene Therapy Candidate for Frontotemporal Dementia at ASGCT 2022

By AviadoBio, Press Release, Private Companies
Press Release.

 

  • Intrathalamic delivery of AVB-101 shows promising efficacy in a pre-clinical disease model
  • Gene therapy candidate also demonstrated broad cortical expression in a large animal biodistribution study

 

London, UK, May 4th, 2022 – AviadoBio, a pioneering, pre-clinical stage gene therapy company focused on developing and delivering transformative medicines for people with neurodegenerative disorders, announces that it will be presenting preclinical data at the upcoming ASGCT 25th Annual Meeting (American Society of Gene & Cell Therapy) on 16-19 May, 2022. The data are from studies it has conducted with its investigational, one-time, adeno-associated virus (AAV) gene therapy, AVB-101 (AVB-PGRN), for the treatment of frontotemporal dementia with GRN mutations (FTD-GRN).

The oral presentation, entitled “Intrathalamic Delivery of AVB.PGRN Rescues Pathology in Grn Null Mice and Achieves Widespread Cortical Expression in a Large Animal Model without Expression in the Liver”, will be presented by AviadoBio Co-Founder and Chief Scientific and Clinical Advisor, Professor Chris Shaw, on Tuesday 17 May at 3:45pm (EST). The presentation will form part of the session “Applications of Improved Gene Therapy Methods in Neurologic Disorders” and will be followed by a Q&A discussion.

The abstract number 468 can be viewed here.

In addition, AviadoBio CEO Lisa Deschamps will be presenting at the conference, on Monday 16 May at 12:00pm (EST), in a session entitled: “Startup Showcase: AviadoBio – A revolution in gene therapy for neurodegenerative disorders”.

ABOUT AVIADOBIO

At AviadoBio, our mission is to transform the lives of people living with neurodegenerative disorders by developing and delivering transformative gene therapies for diseases including frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). The Company’s technology is based on pioneering research from King’s College London and the UK Dementia Research Institute. AviadoBio’s unique platform combines next-generation gene therapy design with deep neuroscience expertise and a novel neuroanatomy-led approach to drug delivery. AviadoBio’s investors include New Enterprise Associates (NEA), Monograph Capital, Advent Life Sciences, EQT Lifesciences, Dementia Discovery Fund (DDF), F-Prime Capital, Johnson & Johnson Innovation – JJDC, Inc. (JJDC), and LifeArc.

The company is developing AVB-101 for patients with FTD-GRN. AVB-101 is an investigational AAV gene therapy designed to slow or stop disease progression by delivering a functional copy of the GRN gene throughout the central nervous system to restore progranulin levels.

For more information, please visit www.aviadobio.com and follow us at Twitter @AviadoBio and LinkedIn AviadoBio.

About Frontotemporal Dementia and AVB-101

Frontotemporal dementia (FTD) is of the second most common form of dementia in people under the age of 65 after Alzheimer’s disease. It affects 50,000 to 60,000 patients in the U.S. and over 100,000 in the E.U. Approximately one third of FTD cases are familial and linked to autosomal dominant mutations in three genes including the granulin gene (GRN) and FTD-GRN represents 5-10% of all patients with FTD. Progressive degeneration of the frontal and temporal lobes of the brain is characteristic of FTD, and is associated with progressive decline of behaviour, decision-making, language and emotion, typically leading to death within 7-10 years of diagnosis. There are currently no approved treatments to stop or slow the progression of FTD or FTD-GRN.

References:

  • Boxer AL. Miller BL.  Alzheimer Dis Assoc Disord. 2005;19 Suppl 1:S3-6
  • Hogan DB, et al. Can J Neurol Sci. 2016;43 Suppl 1:S96-S109
  • Olney NT, et al. Neurol Clin. 2017;35(2): 339–374
  • Greaves CV, et al. J Neurol. 2019;266(8):2075–2086

CONTACT

For media enquiries:
Consilium Strategic Communications
Chris Gardner, Angela Gray, Isabelle Abdou
+44 (0) 20 3709 5700
AviadoBio@consilium-comms.com

May 02
Love0

Orphalan announces FDA approval of Cuvrior™ for the treatment of adult patients with stable Wilson’s disease who are de-coppered and tolerant to penicillamine

By Orphalan, Press Release, Private Companies
Press Release.

 

Paris, France 2 May 2022 – Orphalan SA (“Orphalan” or “the Company”), an international orphan drug development and commercialisation company, today announces approval of Cuvrior™, a new salt of trientine (trientine tetrahydrochloride) by the United States Food and Drug Administration (FDA). Cuvrior™ is approved for the treatment of adult patients with stable Wilson’s disease who are de-coppered and tolerant to penicillamine. Penicillamine is currently approved as a first-line treatment of Wilson’s disease in the US with about one third of patients developing intolerance1.

Orphalan recently completed a global phase III trial, CHELATE, which met its primary efficacy endpoint by demonstrating that Cuvrior™ was non-inferior to penicillamine as measured by non-ceruloplasmin copper (NCC). In consultation with the FDA, an assay based on total serum copper protein speciation was used for measuring this primary efficacy endpoint.

Wilson’s disease is a rare inherited disorder of copper transport primarily affecting the liver and brain. Orphalan commercializes its trientine tetrahydrochloride product in Europe under the name of Cuprior® and expects to launch Cuvrior™, which has been granted Orphan Drug Designation by the FDA, in the US by early 2023. The approval follows the New Drug Application (NDA) submission for the company’s product last year.

Dr. Naseem Amin, Chief Executive Officer at Orphalan, commented:

“We are delighted with the approval of our product, Cuvrior™, which provides a well-tolerated and effective option for Wilson’s disease patients. At Orphalan, we are committed to delivering innovative therapies, with our drug Cuprior® already approved and launched in European countries, and we look forward to launching Cuvrior™ in the United States. We also plan further national submissions to make our product available to patients globally.”

Mary L Graper, Vice President of Scientific Affairs, Wilson Disease Association, added:

“Wilson’s disease is a devastating disorder affecting patients worldwide and for which there has remained a significant need for innovative new treatments. The approval of Orphalan’s Cuvrior™ is extremely promising and is reflective of Orphalan’s patient-driven approach. This marks the culmination of many years of work and is an important moment, offering new hope for patients affected by this disease.”

Professor Michael Schilsky, MD, Director, Center for Excellence for Wilson Disease at Yale University, said:

“As a physician, I have seen first-hand how Wilson’s disease impacts the lives of patients and, until now, there have been few effective long-term treatment options available. The approval of Orphalan’s Cuvrior™ by the FDA is backed by positive data from Orphalan’s multicenter, multinational CHELATE trial – the first head-to-head controlled study of a new trientine salt versus penicillamine. For patients in need, Cuvrior™ represents a well-tolerated and effective alternative to penicillamine, the current standard of care.”

1 Weiss KH, et al. Efficacy and Safety of Oral Chelators in Treatment of Patients With Wilson Disease. Clin Gastroenterol Hepatol. 2013 Aug;11(8):1028-35.e1-2.
– ENDS –

About Trientine Tetrahydrochloride
Trientine tetrahydrochloride is an oral trientine formulation. In the US, trientine tetrahydrochloride has been granted with Orphan Drug Designation for the treatment of Wilson’s disease excluding patients intolerant of penicillamine. It has been approved under the 505(b)(2) pathway for the treatment of adult stable Wilson’s disease patients who are successfully de-coppered and tolerant to penicillamine. The 505(b)(2) regulatory pathway is a type of New Drug Application (NDA).

About Orphalan
Orphalan is an international orphan drug development and commercialisation company. The company delivers worldwide innovative therapies for people living with orphan diseases and is a pioneer in the space. Orphalan was founded in 2011 and has launched Cuprior®. across Europe with its own commercial organization. For more information visit www.orphalan.com and follow us on LinkedIn.

For more information, please contact:
Orphalan Tel: +33 (0)1 42 49 82 64
info@orphalan.com

Consilium Strategic Communications:
Mary-Jane Elliott, Davide Salvi, Genevieve Wilson
Tel: +44 (0) 203 709 5700
orphalan@consilium-comms.com

Apr 27
Love0

Argá Medtech Shows Promise for New Cardiac Ablation Technology at 2022 Heart Rhythm Society Annual Meeting

By Argá Medtech, Press Release, Private Companies
Press Release.

 

  • Argá Medtech’s next-generation non-thermal cardiac ablation system shows promise in improving precision, speed and flexibility in treating atrial fibrillation
  • Preliminary feasibility study results demonstrate the company’s Coherent Sine-Burst Electroporation (CSE) Pulsed Field Ablation platform’s ability to titrate lesion depth using sinusoidal waveforms

Lausanne, Switzerland and San Diego, CA, USA — April 27, 2022 —Argá Medtech, a company developing Coherent Sine-Burst Electroporation (CSE), a next-generation non-thermal cardiac ablation system for treating cardiac arrhythmias, announced a featured poster presentation on preliminary findings for its innovative technology at the 2022 Annual Meeting of the Heart Rhythm Society (HRS), April 29 to May 1 in San Francisco. The company’s CSE Pulsed Field Ablation (PFA) offers unmatched flexibility in treating atrial fibrillation (AF) with its proprietary CSE generator and a multi-configurable catheter.

“AF affects 37.5 million people worldwide1, significantly raising their chances of a stroke or heart attack and greatly diminishing their quality of life,” said David Neale, CEO and co-founder of Argá Medtech. “While the cardiac ablation field has achieved major progress in treating AF, outcomes have only marginally improved in the past 15 years, and costs have increased. It is imperative to develop new tools that improve success rates while lowering procedure costs.”

Argá Medtech’s unique CSE PFA platform offers several advantages over other PFA technologies, which are powered mostly by a square wave energy source. Argá’s new CSE system allows physicians to select all bipolar, all unipolar or combinations of bipolar and unipolar energy delivery modes to titrate the lesion to the needs of the patient.

Argá Medtech’s catheter also offers unmatched flexibility in its ability to perform any lesion type necessary for the patient’s treatment of AF, eliminating the need to use additional ablation catheters. Its design enables the catheter to take different shapes allowing for the creation of circular, linear or focal lesions. These advantages offer the potential to improve precision, speed, safety and efficiency in cardiac ablation procedures while reducing procedure costs.

“Argá Medtech gives physicians an unparalleled ability to perform much more flexible, elegant ablation procedures, likely improving outcomes and reducing the need for repeat surgeries and saving costs for both hospitals and the healthcare system,” said Neale. “We are very optimistic about our technology’s promise and plan to launch our first in-human trial in 2022.”

The HRS presentation reports findings from an animal feasibility study on using multi-programmable CSE waveform to achieve a titratable range of lesion depths that address the variability of tissue thickness within the atrium and ventricle.

Presentation Information
Multi-programmable Coherent Sine Burst Electroporation Waveform for Atrial and Ventricular Catheter Ablation
Date: Friday, April 29, 2022
Time: 1:30 – 1:45 p.m. PT
Location: Abstract Pavilion Podium 11
Authors: Micah Lee BS, Ricardo Roman BS, Ale Lopez BS, Mark Zurcher MS, Deborrah Hunt BS, Randy Werneth MS, Kurt S Hoffmayer MD.

About Argá Medtech
Argá Medtech SA (Argá) is a privately held medical device company based in Switzerland founded by a seasoned team of leaders in the medical device industry. Argá is developing an innovative non-thermal energy-based cardiac ablation system for the treatment of cardiac arrhythmias. With the use of Pulsed Electrical Fields to create irreversible electroporation of cardiomyocytes, Argá is developing a safer, faster, cheaper and more effective cardiac ablation treatment for the benefit of millions of people affected by cardiac rhythm disorders and atrial fibrillation. To learn more, visit https://argamedtech.com.

Lippi G, Sanchis-Gomar F, Cervellin G. Global epidemiology of atrial fibrillation: An increasing epidemic and public health challenge. Int J Stroke. 2021 Feb;16(2):217-221. doi: 10.1177/1747493019897870. Epub 2020 Jan 19. Erratum in: Int J Stroke. 2020 Jan 28;1747493020905964. PMID: 31955707

 

Apr 25
Love0

Novel Cryptigen™ Technology unlocks full potential of Tumour Specific Antigens

By Epitopea, Press Release, Private Companies
Press Release.

 

CAMBRIDGE, UK and MONTREAL, CANADA, 25 April 2022 – Epitopea, a transatlantic cancer immunotherapeutics company and global leader in exploiting a new class of untapped tumour-specific antigens (TSAs), announces a $13.6M (£10.3M) seed investment from a transatlantic syndicate of top-tier life sciences investors, including Advent Life Sciences, CTI Life Sciences, Cambridge Innovation Capital (CIC) and Fonds de solidarité FTQ. The seed round was also supported by Novateur Ventures and the Harrington Discovery Institute/University Health Holdings. The funding will leverage Epitopea’s ground-breaking Cryptigen™ approach to create transformational immunotherapies that target broad cancer patient populations in both solid and haematological cancers.

The company’s proprietary technology provides an innovative approach to identifying shared, aberrantly expressed tumour specific antigens, known as Cryptigens™, that it has exclusively licensed from the Université de Montréal (UdeM). The licensing deal was catalysed by IRICoR, a Canadian Centre of Excellence in Commercialization and Research, which supported and incubated the development of the technology. Cryptigen™ TSAs are uniquely and broadly presented across cancer types, providing tumour-specific targets for immunotherapies that are predicted to kill malignant cells while sparing non-cancerous cells, potentially delivering significant therapeutic benefit across broad patient populations with minimal side effects.

The seed round funding will be used to build the company’s executive team, advance further research on this new class of antigens, and catalyse their translation into novel cancer immunotherapeutics, including therapeutic vaccines, cell therapies, and TCR-based biologics.

Dr Jon Moore, CEO, Epitopea and Operating Partner at Advent Life Sciences, said

 “The outstanding work of Epitopea’s co-founders, Université de Montréal scientists Drs. Claude Perreault, and Pierre Thibault, has opened a tremendous and potentially transformative opportunity for future cancer patients. With this significant seed financing by a syndicate of world-class investors from Canada and the UK, we can start translating these discoveries into novel therapeutics. We will be guided by science, deploying the best modalities available to help cancer patients achieve durable benefits.”

Dr Laurence Rulleau, Partner, CTI Life Sciences, said

 “CTI is delighted to co-lead the seed round of Epitopea, which is the first investment from our third fund, CTI LSF III.  We are convinced that Epitopea has the potential to fundamentally change the paradigm of how cancer patients can be treated with therapeutics vaccines and other immunotherapies to significantly improve their quality of life. Epitopea will explore a variety of early and longer-term value creation strategies including partnerships with third parties as well as developing its own therapeutic modalities based on its Cryptigen™ TSAs.”

Notes To Editors

Contact information

Epitopea
Dr Jon Moore, CEO
jon.moore@epitopea.com

Scius Communications
Katja Stout
Tel: +44 7789 435990
katja@sciuscommunications.com

About Epitopea

Epitopea is a transatlantic biotechnology company developing transformational immunotherapies to treat cancer by targeting a new class of antigens that are broadly shared between patients with the same cancer indication. Cryptigen™ TSAs are discovered by a proprietary approach deploying immunopetidomics, mass spectrometry, genomics, and bioinformatics, which allows the identification of conserved, aberrantly-expressed, tumour-specific antigens, hidden in cancer’s ’junk’ DNA. These hidden Cryptigen™ TSAs have been identified by research led by Drs. Claude Perreault and Pierre Thibault at the Institute for Research in Immunology and Cancer at Université de Montréal.

The company has an extensive proprietary library of Cryptigen™ TSAs that will drive the development of transformational off the shelf cancer immunotherapies. Epitopea is backed by leading life science investors including Advent Life Sciences, CTI Life Sciences, Cambridge Innovation Capital, Le Fonds de Solidarité FTQ, the Harrington Discovery Institute and Novateur Ventures. The company has raised over US$13.6 million in seed financing. Epitopea was founded in 2021 and consists of sister companies based in Cambridge, UK and in Montreal, Canada. Please see www.epitopea.com for more information and follow us on LinkedIn.

For more information about our investors please see the following links:

Advent Life Sciences: www.adventls.com

CTI Life Sciences: https://www.ctisciences.com

Cambridge Innovation Capital: www.cic.vc

Fonds de solidarité FTQ: https://www.fondsftq.com

Novateur Ventures: https://www.novateur.ca

Harrington Discovery Institute/University Health Holdings: https://www.harringtondiscovery.org

For more information about the Université de Montréal, please see here: https://www.umontreal.ca/en/

For more information about IRICoR, please see here: https://www.iricor.ca

Apr 14
Love0

Highlight Therapeutics announces follow-up results from Phase 2b study of BO-112 + anti-PD1 in confirmed anti-PD1 progressor melanoma patients at AACR

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

 

BO-112 demonstrates potential as new and highly effective second line therapy for melanoma

  • BO-112 demonstrates potential as best-in-class therapy to overcome anti-PD1 resistance in melanoma patients whose disease has progressed on prior anti-PD-1 treatment
  • Primary endpoint met with a 30% Response Rate, 15% Complete Responses (CR) and 65% Disease Control Rate (DCR) substantially exceeding current standard of care
  • Further improvements anticipated over one year follow up
  • Hard-to-treat mucosal melanoma patients achieved 66% Overall Response Rate (ORR) and 100% DCR
  • Durable responses and manageable safety profile, with no patients discontinuing due to adverse events
  • Potential for use in multiple solid cancers resistant to anti-PD1 inhibitors, and with different anti-PD1 combinations

Madrid, Spain, 13 April, 2022 – Highlight Therapeutics (“Highlight”), a clinical-stage biopharmaceutical company developing RNA-based therapies against cancer, today announced positive results of a Phase 2 study of intratumoral administration of BO-112 with pembrolizumab in patients with advanced melanoma whose disease had progressed on first-line anti-PD1-based therapy. BO-112 is a dsRNA agonist targeting anti-PD1 resistance, which has demonstrated anti-cancer activity in previous Phase 1b studies.

Results of the study were presented at the plenary session at the American Association for Cancer Research (AACR) Annual Meeting 2022 in New Orleans, Louisiana by Iván Marquez-Rodas (CT014. Efficacy of intratumoral BO-112 with systemic pembrolizumab in patients with advanced melanoma refractory to anti-PD-1-based therapy: Final results of SPOTLIGHT203 phase 2 study. 10:15 –12:15 PM CT)

Sales of anti-PD1 therapies are valued at approximately $24 billion1 a year and they are used to treat most solid tumors. However, currently fewer than 20% of all cancer patients benefit from first-line anti-PD1 treatment. BO-112 in combination with anti-PD1 therapy is designed to resensitize tumors to anti-PD1 treatment through improved antigen presentation, enhanced T-cell infiltration and increased MHC-1 and PDL1 expression by the tumor itself.

“This remarkable data confirms that BO-112 is effective at resensitizing tumors and helping to overcome anti-PD1 resistance, opening up the potential to treat many more patients with anti-PD1 therapies. Analysis of the data showed that most patients achieved a response rate of 40%, although patients with very high LDH levels and acral melanoma did not obtain clinical benefit.” Dr. Marisol Quintero, CEO of Highlight Therapeutics, commented. “Importantly, BO-112 was able to achieve improvements even in patients who had initially responded to anti-PD1 therapy before regressing and in hard-to-treat mucosal melanoma, overall response rates of 66% and disease control rates of 100% were seen, greatly exceeding current standard of care.”

Highlight Therapeutics and Merck, known as MSD outside the United States and Canada, conducted an open-label, single arm study to evaluate the efficacy & safety of intra-tumoral administration of BO-112 + pembrolizumab in mucosal, acral and cutaneous melanoma patients whose disease had progressed, confirmed by two consecutive CT scans. The study recruited 42 patients in France and Spain, with recruitment completed by August 24, 2021. Patients included those with high LDH levels, which are often associated with poor response rates and have been excluded from comparable clinical trials.

The analysis shows:

  • Primary endpoint (ORR by independent reviewer) has been met
  • With a median follow up of seven months, there is a clear clinical benefit in patients with confirmed anti-PD1-resistant melanoma, with a 30% ORR and a 65% DCR: superior to 2nd line Standard of Care in stage III/IV melanoma of ~8% (continuing with anti-PD1 Ab) or 13% (second line ipilimumab).
  • Three hard-to-treat mucosal melanoma patients have achieved an ORR of 66% and DCR of 100%
  • High baseline LDH levels (>3xULN) predict progressive disease
  • Responses and stable diseases are durable
  • Study treatment has a manageable safety profile, with no patients discontinuing due to adverse events

Next steps in the development of BO-112 include:

  • Initiation of a randomized Phase 2 study in 2nd-line melanoma is planned in 2023
  • Highlight Therapeutics has initiated strategic partnerships discussions with anti-PD1 companies interested in enhancing their anti-PD1 market potential

1. IQVIA Global Oncology Report 2020

For more information, please contact:

Highlight Therapeutics S. L.             info@highlighttherapeutics.com
Marisol Quintero, CEO

Mo PR Advisory                             Tel: +44 (0) 7876 444977 / 07860 361746
Mo Noonan/Jonathan Birt

Notes to editors

About Highlight Therapeutics

Highlight Therapeutics, formerly known as Bioncotech Therapeutics, is a private, clinical-stage company dedicated to unlocking the full potential of immuno-oncology. Our lead drug candidate BO-112 is a best-in-class RNA-based therapy which has been demonstrated to initiate a powerful immune response, leveraging a unique multi-target approach to turn ‘cold’ tumors ‘hot’ and therefore visible to the immune system. It has the potential to rescue patients who are resistant to current checkpoint inhibitor therapy, a very large market opportunity. BO-112 is currently being investigated in a range of clinical trials as a monotherapy and in combination with checkpoint inhibitors. In addition to in-house research, Highlight Therapeutics has a number of external collaborators, including Merck & Co and UCLA.

For more information, please visit www.highlighttherapeutics.com

Mar 15
Love0

Highlight Therapeutics to present at the American Association for Cancer Research (AACR) Annual Meeting 2022

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

 

Final results of SPOTLIGHT203 phase 2 study demonstrate new approach to second line treatment for melanoma

Madrid, Spain, 15 March, 2022 – Highlight Therapeutics, (“Highlight”), a clinical-stage biopharmaceutical company developing RNA-based therapies against cancer, today announced an oral presentation at the Combination Immunotherapy Clinical Trials Plenary Session at the American Association for Cancer Research (AACR) Annual Meeting 2022, taking place April 8-13, 2022 in New Orleans, Louisiana. The Company will also present three posters.

SESSION TITLE: Combination Immunotherapy Clinical Trials

PRESENTATION TITLE: CT014

Efficacy of intratumoral BO-112 with systemic pembrolizumab in patients with advanced melanoma refractory to anti-PD-1-based therapy: Final results of SPOTLIGHT203 phase 2 study

PRESENTER: Ivan Marquez-Rodas

DATE: April 12, 2022

TIME: 1015 –1215 PM CT

LOCATION: New Orleans Convention Center, Exhibit Halls B-C

Highlight will also present the following posters at AACR:

PRESENTATION TITLE: Correlation of biomarkers and clinical benefit of intratumoral BO112 and pembrolizumab in patients with anti PD1 refractory melanoma

DATE: April 11, 2022

TIME: 9:00 AM – 12:30 PM

LOCATION: New Orleans Convention Center

PRESENTATION TITLE: Radiomic features in tumor assessment, preliminary results from a phase 2 study of intratumoral administration of BO-112 with pembrolizumab in patients with anti PD1 refractory melanoma

DATE: April 11, 2022

TIME: 9:00 AM – 12:30 PM

LOCATION: New Orleans Convention Center

 

For more information, please contact:

Highlight Therapeutics S. L. info@highlighttherapeutics.com
Marisol Quintero, CEO

Mo PR Advisory Tel: +44 (0) 7876 444977 / 07860 361746
Mo Noonan/Jonathan Birt

Notes to editors

About Highlight Therapeutics

Highlight Therapeutics is a private, clinical-stage company dedicated to unlocking the full potential of immuno-oncology. Our lead drug candidate BO-112 is a best-in-class RNA-based therapy which has been demonstrated to initiate a powerful immune response, leveraging a unique multi-target approach to turn ‘cold’ tumors ‘hot’ and therefore visible to the immune system. It has the potential to rescue patients who are resistant to current checkpoint inhibitor therapy, a very large market opportunity. BO-112 is currently being investigated in a range of clinical trials as a monotherapy and in combination with checkpoint inhibitors. In addition to in-house research, Highlight Therapeutics has a number of external collaborators, including Merck & Co and UCLA.

For more information, please visit www.highlighttherapeutics.com

Feb 18
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Nalu Medical Inc. Announces $104 Million Equity Financing

By Nalu Medical, Press Release, Private Companies
Press Release.

 

CARLSBAD, Calif. (PRWEB) February 17, 2022

Nalu Medical, Inc. (“Nalu”), a private company focused on innovative and minimally invasive solutions for chronic neuropathic pain, announced today the completion of a $104 million equity financing. The round was led by new investors, MVM Partners and Gilde Healthcare. Also participating in this round were new investors Pura Vida Investments and Aperture Venture Partners, as well as existing investors, Advent Life Sciences, Decheng Capital, Endeavour Vision, and Longitude Capital. The proceeds from this financing are intended to be used for scaling commercial operations to accelerate growth, continued expansion of clinical evidence, and continuing product development, in addition to other general corporate purposes.

“Chronic pain causes suffering to millions and can be addressed by targeted, non-addictive therapy. Nalu is uniquely positioned to meet this medical need by providing a patient friendly, neurostimulation technology and MVM is excited to support its continued growth,” said Hugo Harrod, a partner with MVM Partners, who joins Nalu’s Board of Directors. “We believe Nalu’s technology is one of the most advanced on the market, with huge potential to meet the needs of more patients,” said Geoff Pardo of Gilde Healthcare, who is also joining the Board of Directors.

“This additional funding underscores the potential of Nalu’s miniaturized technology and will allow us to accelerate the already strong adoption of our system,” said Earl Fender, President and CEO of Nalu. “We welcome the addition of new top-tier investors and appreciate the continued support of our current investors, who share our mission of commercializing innovative and minimally invasive solutions that make meaningful differences to people suffering from chronic pain.”

J.P. Morgan served as placement agent to Nalu for this transaction.

About Nalu Medical

Nalu is a Carlsbad, California-based medical technology company focused on developing and commercializing innovative and minimally invasive solutions for patients with chronic neuropathic pain. The Nalu Neurostimulation System delivers gentle electrical pulses to the nervous system to modulate pain signals to the brain. The Nalu system was designed to address major unmet needs in the treatment of chronic neuropathic pain and provide a differentiated value proposition for patients and physicians.

About the Nalu Neurostimulation System

The Nalu system consists of a fully-featured, battery-free, miniaturized implantable pulse generator (IPG) that is powered wirelessly by an externally worn Therapy Disc and controlled through a smartphone-based remote control app. Despite its small size, Nalu’s micro-IPG delivers treatment capabilities similar to larger IPGs as well as unique advantages associated with advanced waveforms, extensive programming options, exceptional upgradability, and an expected service life of 18 years. The Nalu Neurostimulation System is currently FDA-cleared for Spinal Cord Stimulation (SCS) and Peripheral Nerve Stimulation (PNS) indications. To learn more, visit http://nalumed.com.

Indications for Use

Spinal Cord Stimulation – The Nalu SCS system is indicated as the sole mitigating agent, or as an adjunct to other modes of therapy used in a multidisciplinary approach for chronic, intractable pain of the trunk and/or limbs, including unilateral or bilateral pain. The trial devices are solely used for trial stimulation (≤ 30 days) to determine efficacy before recommendation for a permanent (long term) device.

Peripheral Nerve Stimulation – The Nalu PNS system is indicated for pain management in adults who have severe intractable chronic pain of peripheral nerve origin, as the sole mitigating agent, or as an adjunct to other modes of therapy used in a multidisciplinary approach. The Nalu Neurostimulation System for PNS is not intended to treat pain in the craniofacial region. The trial devices are solely used for trial stimulation (≤ 30 days) to determine efficacy before recommendation for a permanent (long term) device.

Nalu and the Nalu logo are trademarks of Nalu Medical, Inc.

Feb 16
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Curve Therapeutics Announces Collaboration with MSD for Next Generation Drug Discovery Platform

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

 

  • Curve’s innovative mammalian cell drug discovery platform to enable identification and optimisation of novel small molecule drug candidates against MSD targets
  • Potentially game-changing platform enables direct discovery of functionally active molecules against difficult to drug targets expressed in their native intracellular environment

Southampton, UK, 16th February 2022: Curve Therapeutics (Curve), a private biotechnology company pioneering a potentially game-changing, functional drug discovery platform, today announces a global research collaboration with MSD, the trade name of Merck & Co., Inc., Kenilworth, NJ USA, to discover and validate modulators of up to five therapeutic targets using its Microcycle® technology, initially for oncology and neurology indications.

Under the terms of the agreement Curve will receive an upfront payment, and will be eligible to receive research, development and commercial milestones totalling up to US$ 1.7B should all five therapeutic programs succeed. Curve will also receive a royalty on net sales of any approved products resulting from the alliance.

Under the agreement, Curve will perform high throughput mammalian cell-based functional screening, hit characterisation, data-mining and analysis, and Microcycle® optimisation. MSD will be responsible for lead optimisation, clinical development, manufacturing and commercialisation of compounds identified through the collaboration.

Simon Kerry, PhD, MBA, Chief Executive Officer of Curve, said:

“This collaboration is a major milestone for Curve and an important endorsement of our ground-breaking drug discovery platform. Working with MSD on selected therapeutic targets will complement Curve’s in-house drug discovery and development programmes.”

Curve’s novel, proprietary platform enables the direct discovery of biologically active molecules against targets that have been difficult to address using conventional drug discovery methods. The platform allows rapid enrichment of highly diverse Microcycle® libraries in the cytoplasm of mammalian cells to identify library members that have a desired biological activity against a therapeutic target. Importantly, the compact size of Microcycles™ enables their transformation to non-peptidic small molecules for lead optimisation and development: an unparalleled advantage compared to other cyclic peptides.

Prof. Ali Tavassoli, Chief Scientific Officer of Curve, said:

“Screening a genetically encoded Microcycle® library against proteins in their native intracellular state is unique in drug discovery and Curve’s platform creates an unprecedented opportunity to discover functional hits that are readily converted to small-molecule leads against the most challenging targets in drug discovery.”

Rob Garbaccio PhD., Vice President Discovery Chemistry MSD Research Laboratories said:

“At MSD we are committed to bringing forward medicines for many of the world’s most challenging diseases. We look forward to collaborating with the scientists at Curve to evaluate new ways to treat complex diseases.”

Curve originated from world-leading Microcycle® research conducted by Professor Tavassoli’s group in the Department of Chemistry at the University of Southampton, UK. The company was established in 2019 by founding investor Advent Life Sciences and subsequently joined by co-lead Epidarex Capital.

-ENDS-

For more information, please contact:

Curve Therapeutics
Simon Kerry
Chief Executive Officer
Simon.Kerry@curvetx.com

Optimum Strategic Communications
Mary Clark, Stella Lempidaki, Vici Rabbetts
+44 (0) 208 078 4357
curve@optimumcomms.com

About Curve Therapeutics

Curve Therapeutics (Curve) is a private biotechnology company founded in 2019 and based in Southampton, UK. Curve is pioneering a game-changing, functional, drug discovery engine to generate higher quality hits and leads with the aim of discovering first-in-class therapeutics. Curve’s platform enables the discovery of biologically active molecules against targets that have evaded conventional drug discovery techniques. Curve has developed an IP-protected, mammalian cell platform technology for functional screening and enrichment of diverse hexameric cyclic peptide (Microcycle®) libraries to identify those library members that have the desired biological activity against a therapeutic target. A key advantage of the technology is that both the library and the target are present in all of their native conformations within a cell. Uniquely, the compact size and rigid structure of Microcycles™ enables the design of small molecule hits and leads. The platform can be used for a wide range of therapeutically relevant targets, including protein-protein and protein-DNA interactions and has been used by Curve to develop a pipeline of cancer programmes against targets including a dual HIF-1/HIF-2 inhibitor. For more information visit: www.curvetx.com

Jan 11
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Amgen and Arrakis Therapeutics Announce Multi-Target Collaboration to Identify Novel RNA Degrader Small Molecule Therapeutics

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

 

Amgen and Arrakis Therapeutics Announce Multi-Target Collaboration to Identify Novel RNA Degrader Small Molecule Therapeutics

Collaboration Creates “Targeted RNA Degraders” by Bringing Together Amgen’s Induced Proximity Platform Discovery Expertise and Arrakis’ Leading-edge RNA-targeted Drug Discovery Platform

THOUSAND OAKS, Calif. and WALTHAM, Mass., Jan. 11, 2022 – Amgen (NASDAQ:AMGN) and Arrakis Therapeutics today announced a research collaboration focused on the discovery and development of RNA degrader therapeutics against a range of difficult-to-drug targets in multiple therapeutic areas. This new class of “targeted RNA degraders” consists of small molecule drugs that selectively destroy RNAs encoding disease-causing proteins by inducing their proximity to nucleases.

Under the terms of the agreement, Arrakis will lead research activities for the identification of RNA-targeted small molecule (rSM) binders against a broad set of targets nominated by Amgen. Both parties will collaboratively design and functionalize these molecules to specifically degrade targeted RNAs, and Amgen will lead further preclinical and clinical development activities. Amgen will pay $75 million upfront to Arrakis for five initial programs and will have the option to nominate additional programs. For each program, Arrakis will be eligible for additional payments from Amgen for preclinical, clinical, regulatory and sales milestones, and royalties up to low double digits. Arrakis could potentially receive several billion dollars in future payments if all milestones are met and future program options are exercised.

“Targeted RNA degradation is an exciting area that is pushing the boundaries of drug discovery and design,” said Raymond Deshaies, Ph.D., senior vice president of Global Research at Amgen. “The collaboration with Arrakis combines Amgen’s induced proximity expertise in discovering multispecific molecules to target the biologic mechanisms of disease and Arrakis’ pioneering discovery platform to predict RNA structures and identify small molecules that bind to them, significantly broadening the possibilities of addressing difficult protein targets considered undruggable because they may not have binding sites needed for conventional medicines. Combining this approach with Amgen’s targeted protein degradation induced proximity research already underway has the potential to significantly expand the druggable genome.”

By integrating the capabilities of the two innovative discovery platforms from Amgen and Arrakis, the collaboration creates an opportunity to design and engineer targeted RNA degraders. Amgen has built its Induced Proximity Platform to identify multispecific molecules that harness the power of cell biology by forming novel connections between natural effectors and targets. One end of the molecule binds to the target to be altered (inhibited, activated or destroyed) and the other end binds to a cellular effector that acts on the target, offering the potential to engage a broad range of cellular mechanisms to treat disease. With targeted RNA degraders, the effector, such as a ribonuclease or other RNA modulator, is brought into proximity of the RNA to degrade or otherwise modify the disease-causing RNA of interest. This complements Amgen’s existing efforts to target RNA with siRNA. In this collaboration, Arrakis’ rSM platform will be applied as a drug discovery engine to identify small molecules that bind target RNA. These rSMs will then be functionalized with nuclease recruiters to create heterobifunctional molecules that trigger degradation of disease-relevant RNA targets.

“We are excited to partner with Amgen’s strong research team to pursue a shared goal of creating a new class of medicines that induce degradation of disease-causing RNAs. This collaboration further demonstrates the utility of our proprietary rSM discovery platform for targeting RNA with small molecules and paves the way for creating powerful new treatments for patients,” said Michael Gilman, Ph.D., chief executive officer of Arrakis. “Based on our long-term goal to build a broad and industry-leading platform that adapts state-of-the-art drug discovery tools to target RNA biology, we have enabled a range of different applications and collaborations to leverage our science, a strong capital position and the ability to grow our business and our impact to advance RNA-targeted drug programs for diseases unaddressed by today’s medicines.”

About Amgen

Amgen is committed to unlocking the potential of biology for patients suffering from serious illnesses by discovering, developing, manufacturing and delivering innovative human therapeutics. This approach begins by using tools like advanced human genetics to unravel the complexities of disease and understand the fundamentals of human biology.

Amgen focuses on areas of high unmet medical need and leverages its expertise to strive for solutions that improve health outcomes and dramatically improve people’s lives. A biotechnology pioneer since 1980, Amgen has grown to be one of the world’s leading independent biotechnology companies, has reached millions of patients around the world and is developing a pipeline of medicines with breakaway potential.

Amgen is one of the 30 companies that comprise the Dow Jones Industrial Average and is also part of the Nasdaq-100 index. In 2021, Amgen was named one of the 25 World’s Best Workplaces™ by Fortune and Great Place to Work™ and one of the 100 most sustainable companies in the world by Barron’s.

For more information, visit www.amgen.com and follow us on www.twitter.com/amgen.

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 science 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.

Dec 15
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Artax Biopharma Announces First-in-Human Dosing in Phase 1 Clinical Trial for AX-158, Company’s First-in-Class Oral Immunomodulator to Treat T Cell-Mediated Diseases

By Artax Biopharma, Press Release, Private Companies
Press Release.

 

The Phase 1 Trial, Expected to Conclude in 2022, Will Evaluate Safety, Exposure, and Pharmacokinetics of AX-158 in Healthy Volunteers

AX-158, a Novel, Oral Small Molecule Immunomodulating Agent for the Treatment of T Cell-Mediated Diseases, Selectively Modulates T Cell Responses without Suppression of Immune System Function

Cambridge, Mass., December 15, 2021 – Artax Biopharma, Inc., a clinical-stage biotechnology company focused on transforming the treatment of T Cell-mediated diseases, today announces first-in-human dosing in a Phase 1 clinical trial for AX-158, the Company’s first-in-class oral small molecule immunomodulator. AX-158 is the Company’s first oral small molecule immunomodulating agent to enter clinical development for the treatment of T Cell-mediated diseases.

AX-158 employs a first-in-class mechanism of action that selectively modulates T cell responses that play a critical role in healthy immune system function. AX-158 has the potential to treat T Cell mediated diseases without the risk of immunosuppression.

The start of the Phase 1 clinical trial, which marks the beginning of Artax’s AX-158 clinical trial program, is expected to conclude in 2022. The Phase 1 clinical trial will assess the safety, exposure, and pharmacokinetics of AX-158 in healthy volunteers and will include ex-vivo stimulated measures of its pharmacodynamic activity. In preclinical studies, AX-158 demonstrated potential to treat T Cell mediated diseases by decreasing key cytokines including INFγ, TNFα and IL-2 in whole human blood samples.

“Artax has a unique opportunity to make a transformative difference for patients with T Cell-driven conditions, including autoimmune diseases, T Cell malignancies and induced T Cell pathologies – while not impacting the immune system’s ability to mount a strong response to foreign pathogens and infections,”

commented Artax Biopharma Chief Executive Officer Joseph Lobacki.

“We look forward to the results of this Phase 1 clinical study to inform our future clinical studies evaluating patients managing T Cell-mediated diseases.”

About Artax Science and Immunomodulation
A healthy immune system eliminates harmful foreign pathogens, while being tolerant of self-tissues and organs. The T Cell Receptor (TCR) is central to healthy T Cell function and a
well-functioning immune system. When TCR signaling becomes dysregulated, T Cells behave abnormally. This behavior results in T Cell-driven conditions, including autoimmune diseases, T Cell malignancies (lymphomas), and induced T Cell pathologies in which medical treatments result in immune reactions (such as stem cell transplants resulting in acute graft-versus-hostdisease or immuno-oncology treatments resulting in immune related-adverse events). Artax believes immunomodulation – a mechanism through which our investigational agents assist in rebalancing the immune system and eliminating a cause of T Cell-mediated diseases while not impacting patients’ ability to properly fight foreign pathogens – holds great potential.

About Artax-158
AX-158 is a first-in-class, oral small molecule immunomodulating agent in clinical development for the treatment of T Cell-mediated diseases. AX-158 employs a novel mechanism
of action that selectively modulates, or adjusts, T Cell responses that play a critical role in immune system function. Nck is a protein that naturally amplifies T Cell signaling directly at the TCR, contributing to T Cell mediated disease. AX-158 is a Nck SH3.1 domain inhibitor which selectively counteracts the role of Nck in T Cells. This process of immunomodulation assists the immune system to maintain healthy control and eliminates a direct contributor to T Cell-mediated diseases. Importantly, Artax believes that preclinical data suggests AX-158 will not be immunosuppressive and so will not impact the immune system’s ability to mount a strong response to foreign pathogens and infections.

About Artax Biopharma
Artax Biopharma is a clinical-stage biotechnology company transforming T Cell-mediated disease treatment by developing innovative small molecules that modulate the immune system.
Artax science holds broad potential to treat T Cell-mediated diseases such as autoimmune diseases, induced T cell pathologies (such as acute graft versus host disease and immuneoncology treatment-related adverse events) and T Cell malignancies, while simultaneously allowing the body to fight foreign pathogens. For more information, please visit
www.artaxbiopharma.com.

Contacts:
Karen LaRochelle
Chief Business Officer
Artax Biopharma
klarochelle@artaxbiopharma.com

Media:
Linda Phelan Dyson
+1 973-986-5873
ldyson@artaxbiopharma.com