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Nov 15
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Aura Biosciences Presents Final Phase 1b/2 Data for its first Virus-Like Drug Conjugate, AU-011, in Patients with Choroidal Melanoma at the American Academy of Ophthalmology

By Aura Biosciences, Press Release, Publicly Listed
Press Release.

 

Aura Biosciences Presents Final Phase 1b/2 Data for its first Virus-Like Drug Conjugate, AU-011, in Patients with Choroidal Melanoma at the American Academy of Ophthalmology 2021 Annual Meeting

Presentations Include Final Safety and Efficacy Data from the Phase 1b/2 Trial using Intravitreal Administration and Updated Safety Data from the Phase 2 Trial using Suprachoroidal Administration

CAMBRIDGE, Mass.–(BUSINESS WIRE)– Aura Biosciences, a clinical-stage oncology company developing a novel class of virus-like drug conjugate (VDC) therapies for multiple oncology indications, today announced the presentation of data evaluating its first VDC, AU-011, in indeterminate lesions (ILs) and choroidal melanoma (CM), including final safety and efficacy data from the Phase 1b/2 trial using intravitreal (IVT) administration, as well as updated safety results from the Phase 2 trial using suprachoroidal (SC) administration. The results are presented as part of the American Academy of Ophthalmology (AAO) 2021 Annual Meeting.

“The final safety and efficacy data from the Phase 1b/2 trial using intravitreal administration presented today, along with the data from the Phase 2 trial using suprachoroidal administration, provide a high level of confidence for further clinical development in patients with indeterminate lesions or choroidal melanoma,”

said Carol Shields, MD, Chief of the Ocular Oncology Service at Wills Eye Hospital and Professor of Ophthalmology at Thomas Jefferson University.

“I believe AU-011 may offer patients a safe, effective first-line therapy for early-stage disease that preserves vision, a critical component in patients’ quality of life often neglected with today’s current treatment options.”

Final 12-Month Safety and Efficacy Data from Phase 1b/2 Trial with IVT administration

The Phase 1b/2 trial (NCT03052127) evaluated the safety and efficacy of AU-011 using IVT administration for the treatment of ILs and CM. A total of 56 patients were enrolled in the Ph1b/2 trial including the single and multiple dose escalation cohorts and received up to two cycles of therapy (therapeutic regimen). As part of an enrichment strategy agreed with FDA, patients with small tumors with active growth were enrolled in the Phase 2 part of the study (expansion cohort). This group of patients (n=14) received the therapeutic regimen and were evaluated for the tumor growth rate, tumor control, and visual acuity preservation as the efficacy endpoints. These endpoints have been agreed with FDA and are planned to be used in the pivotal program. The results at 12 months showed a statistically significant reduction in the tumor growth rate (-0.483 mm/yr, p = 0.018) compared to each patient’s documented growth rate at study entry, and a 64% (9/14) tumor control rate. In addition, the visual acuity preservation rate was 71%, which is unprecedented compared to the current standard of care with radiotherapy. Overall, AU-011 demonstrated a favorable safety and tolerability profile. The majority of adverse events (AEs), which included intraocular inflammation and increased intraocular pressure, were transient and resolved without clinical sequelae. A large number of patients (43/56) had tumors close to the fovea and optic disk and only two patients with juxta-foveal tumors had a treatment related serious adverse event (SAE) of vision loss. No other treatment related SAEs were observed in the trial. These safety and efficacy results indicate that AU-011 may offer a targeted vision preserving therapy for the first line treatment of CM.

Safety Data from Phase 2 Trial with SC Administration

This Phase 2 trial (NCT04417530) includes an open-label, dose escalation phase assessing the safety and efficacy of AU-011 via SC administration in patients with ILs and CM and plans to enroll up to 22 patients. In this preliminary safety data review of the initial dose escalation cohorts (n=14), no treatment related SAEs, dose limiting toxicities (DLTs), or grade 3/4 AEs were reported. Preliminary results indicate a positive safety and tolerability profile for AU-011 via SC administration.

Details for the AAO 2021 Presentations are as follows:

Title: A Phase 1b/2 Trial of AU-011, a First in Class Targeted Therapy for the Treatment of Choroidal Melanoma via Intravitreal Administration
Presenter: Carol L. Shields, Wills Eye Hospital
Session: OP10 Ocular Pathology and Oculoplastics Original Paper Session
Date and time: Monday, November 15 from 9:45 – 9:52 AM CT
Location: 255-257

Title: A Phase 2 Trial of a First in Class Targeted Therapy for Choroidal Melanoma via Suprachoroidal (SC) Administration
Presenter: Hakan Demirci, Kellogg Eye Center
Session: PD08 Ocular Pathology and Oculoplastics Poster Discussion
Date and time: Available on demand beginning Friday, November 12, 2021, at 7:30am PT
Location: Virtually on demand

The presentations can be accessed by visiting the “Scientific Presentations” section of “VDC Platform” page of the Aura Biosciences website.

About Choroidal Melanoma

Choroidal melanoma is a rare and aggressive type of eye cancer. Choroidal melanoma is the most common primary intraocular tumor in adults and develops in the uveal tract of the eye. No targeted therapies are available at present, and current radiotherapy treatments can be associated with severe visual loss and other long-term sequelae such as dry eye, glaucoma, cataracts, and radiation retinopathy. The most common current treatment is plaque radiotherapy, which involves surgical placement of a radiation device on the exterior of the eye over the tumor. The alternative is enucleation, or total surgical removal of the eye. Choroidal melanoma metastasizes in approximately 50 percent of cases with liver involvement in 80-90% of cases and, unfortunately, metastatic disease is universally fatal. There is a very high unmet need for a new vision sparing targeted therapy that could enable early treatment intervention for this life-threatening rare disease given the mortality rate in metastatic disease, lack of approved therapies, and the comorbidities of radioactive treatment options.

About AU-011

AU-011 is a first-in-class virus-like drug conjugate (VDC) therapy in clinical development for the first line treatment of choroidal melanoma. The virus-like component of the VDC selectively binds unique heparin sulphate proteoglycans (HSPGs), which are modified and overexpressed on the tumor cell surface of malignant cells in the choroid and AU-011 delivers a potent cytotoxic drug that is activated with infrared light. Upon activation with an ophthalmic laser, the cytotoxic drug rapidly and specifically disrupts the cell membrane of malignant cells with a pro-immunogenic cell death that can activate the immune system generating long term anti-tumor immunity. The unique specificity of tumor binding by the VDC enables the preservation of key eye structures, which may allow for the potential of preserving patients’ vision and reducing other long-term complications of radiation treatment. AU-011 can be delivered using equipment commonly found in an ophthalmologist’s office and does not require a surgical procedure, pointing to a potentially less invasive, more convenient therapy for patients and physicians. AU-011 for the treatment of choroidal melanoma is currently in Phase 2 clinical development and the company plans to expand the clinical program into choroidal metastasis.

About Suprachoroidal Administration

The suprachoroidal space (SCS®) injection treatment approach offers unprecedented access to the back of the eye where sight-threatening disease often occurs. Aura believes that delivering AU-011 into SCS within the eye, has the potential to offer certain advantages, including higher bioavailability at the tumor site and reduced exposure of non-targeted tissues, which may lead to an improved therapeutic index for AU-011. Collectively, these features could allow for the treatment of a wider range of tumor sizes, and, therefore, a larger number of patients may be treatable. The Company is partnered with Clearside Biomedical for use of Clearside’s SCS Microinjector® for administration of AU-011 into the SCS.

About Aura Biosciences

Aura Biosciences, Inc. is a clinical-stage oncology company developing a novel technology platform based on virus-like drug conjugates (VDCs) to target and destroy cancer cells selectively while activating the immune system to create long lasting anti-tumor immunity. The VDC technology platform is based on the discoveries of NIH Distinguished Investigator Dr. John Schiller of the Center for Cancer Research at the National Cancer Institute. The company has the goal of developing this technology in multiple cancer indications with an initial focus on primary choroidal melanoma, a rare disease for which there are no approved drugs. Aura’s lead product candidate belzupacap sarotalocan (AU-011) is currently in Phase 2 development for the first line treatment of primary choroidal melanoma, a vision and life-threatening form of eye cancer where standard of care radioactive treatments leave patients with major vision loss and severe comorbidities. AU-011 was well tolerated in a Phase 1b/2 trial, demonstrating high rates of tumor control and vision preservation. Future pipeline applications for Aura’s technology include additional ocular oncology indications like choroidal metastases and solid tumor indications like non-muscle invasive bladder cancer. Aura is headquartered in Cambridge, MA.

Forward Looking Statement

This press release may contain forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995 and other federal securities laws, including express or implied statements regarding Aura’s future expectations, plans and prospects, including, without limitation, statements regarding expectations and plans for presenting clinical data, projections regarding our long-term growth, the anticipated timing of our clinical trials and regulatory filings, the development of our product candidates and advancement of our clinical programs, as well as other statements containing words such as “may,” “will,” “could”, “should,” “expects,” “intends,” “plans,” “anticipates,” “believes,” “estimates,” “predicts,” “projects,” “seeks,” “endeavor,” “potential,” “continue” or the negative of such words or other similar expressions that can be used to identify forward-looking statements. The express or implied forward-looking statements included in this press release are only predictions and are subject to a number of risks, uncertainties and assumptions, including, without limitation: uncertainties inherent in clinical trials and in the availability and timing of data from ongoing clinical trials; whether interim results from a clinical trial, including the Phase 2 SC administration trial, will be predictive of the final results of the trial; whether results from pre-clinical studies or earlier clinical studies will be predictive of the results of future trials, including regarding AU-011’s ability to offer vision preserving therapy for the first line treatment of choroidal melanoma; the expected timing of the expansion phase of the Phase 2 SC administration trial; the expected timing for submissions for regulatory approval or review by governmental authorities; whether Aura will receive regulatory approvals to conduct trials or to market products; whether Aura’s cash resources will be sufficient to fund its foreseeable and unforeseeable operating expenses and capital expenditure requirements; risks, assumptions and uncertainties regarding the impact of the continuing COVID-19 pandemic on Aura’s business, operations, strategy, goals and anticipated timelines, Aura’s ongoing and planned pre-clinical activities, Aura’s ability to initiate, enroll, conduct or complete ongoing and planned clinical trials, Aura’s timelines for regulatory submissions and Aura’s financial position. These and other risks and uncertainties are described more fully in the section titled “Risk Factors” set forth in Aura’s filings with the Securities and Exchange Commission. In light of these risks, uncertainties and assumptions, the forward-looking events and circumstances discussed in this press release may not occur and actual results could differ materially and adversely from those anticipated or implied in the forward-looking statements. You should not rely upon forward-looking statements as predictions of future events. Although Aura believes that the expectations reflected in the forward-looking statements are reasonable, it cannot guarantee that the future results, levels of activity, performance or events and circumstances reflected in the forward-looking statements will be achieved or occur. Moreover, except as required by law, neither Aura nor any other person assumes responsibility for the accuracy and completeness of the forward-looking statements included in this press release. Any forward-looking statement included in this press release speaks only as of the date on which it was made. Aura undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20211115005138/en/

Contacts

Investor and Media:
Matthew DeYoung
Argot Partners
212-600-1902 | aura@argotpartners.com

Source: Aura Biosciences

Nov 12
Love0

Highlight Therapeutics announces positive preliminary results from Phase 2b study of BO-112 + anti-PD1 in confirmed anti-PD1 progressor melanoma patients at SITC

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

 

Highlight Therapeutics announces positive preliminary results from Phase 2b study of BO-112 + anti-PD1 in confirmed anti-PD1 progressor melanoma patients at SITC

Breakthrough cancer treatment shows best-in-class potential to open up 2nd-line immunotherapy market to anti-PD1 resistant patients

  • 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 27% Overall Response Rate (ORR), 8% Complete Responses (CR) & 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% ORR and 100% DCR
  • Durable responses and manageable safety profile, with no patients discontinuing due to adverse events
  • Preparation for pivotal trial based on FDA guidance underway; potential for use in multiple solid cancers resistant to anti-PD1 inhibitors, and with different anti-PD1 combinations

Madrid, Spain, 12 November, 2021 – Highlight Therapeutics, (“Highlight”), a clinical-stage biopharmaceutical company developing RNA-based therapies against cancer, today announced positive preliminary 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 been successfully tested in several previous Phase 1b studies.

Initial results of the study were presented today (12th November) in a late-breaking session at The Society for Immunotherapy of Cancer (SITC) 36th Annual Meeting in Washington DC, Nov. 10–14, 2021. (LBA Poster Abstract (961): Preliminary results of a phase 2 study of intratumoral administration of BO-112 with pembrolizumab in patients with advanced melanoma that have progressive disease on anti-PD-1-based therapy, presented in the Poster Hall in Washington, D.C. and the virtual ePoster Hall beginning Friday, Nov. 12 at 7 a.m. EST).

“These are potentially game-changing results showing that BO-112 can rescue melanoma patients who have failed first-line immune-therapy with anti-PD1,”

said Dr Carlos Paya, Executive Chairman of Highlight Therapeutics.

“Anti-PD1 based immunotherapy has revolutionized oncology treatments, but only a fraction of patients initially respond and many of these patients progress thereafter. These initial Phase 2 results show that BO-112 combined with a leading PD1 inhibitor rescue around 65% of anti-PD1 failing patients, making many of them respond to the combined treatment. These much-anticipated outcomes demonstrate BO-112’s potential as a best-in-class therapy for melanoma patients whose disease has progressed on prior anti-PD1 treatment, and we now look forward to further clinical studies, not only in melanoma but in other major tumor types in which anti-PD1 resistance is also an issue.”

Anti-PD1 therapies are valued at approximately $24 billion1 and are used across most solid tumors but 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 initial data is very encouraging and has the potential to change medical practice,”

said Dr. Marisol Quintero, CEO of Highlight Therapeutics.

“The preliminary ORR of 27%, including 8% Complete Responses, already exceeds current standard of care, such as the use of anti-CTLA-4. Based on the mode of action and experience from previous BO-112 Phase 1 studies, additional follow-up of these patients is expected to deliver further improvements in ORR. We are also encouraged by the excellent safety profile, with no patients discontinuing the study due to adverse events. Highlight Therapeutics is in the planning stage of a pivotal Phase 3 trial, due to begin in 2022, and we look forward to opening discussions with potential partners to explore combinations with anti-PD1s.”

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 preliminary analysis shows:

  • Primary endpoint (ORR by independent reviewer) has been met
  • With a median follow up of three months, there is a clear clinical benefit in patients with confirmed anti-PD1-resistant melanoma, with a 27% 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 SD 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 pivotal Phase 3 study in 2nd-line melanoma is planned in 2022 following discussions with regulatory agencies in the US and Europe
  • Highlight Therapeutics has initiated strategic partnerships discussions with anti-PD1 companies interested in enhancing their anti-PD1 market potential
  • Initial data from a sponsor-initiated Phase 1B trial by UCLA evaluating BO-112 + pembrolizumab in anti-PD1 resistant hepatocellular carcinoma currently recruiting patients is expected in 2022

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

Consilium Strategic Communications     Tel: +44 7921 697654
Chris Gardner                                            cgardner@consilium-comms.com

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

Nov 01
Love0

Amphista Therapeutics Appoints CBO, Beverley Carr

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

 

Amphista Therapeutics Appoints CBO, Beverley Carr

Cambridge, England, 1 November 2021 – Amphista Therapeutics, a leader in next generation targeted protein degradation (TPD) approaches, today announced the appointment of a Chief Business Officer (CBO), Beverley Carr.

Amphista’s CEO Dr Nicola Thompson said,

“On behalf of the Amphista team, I am thrilled to welcome Beverley as our CBO.  Beverley brings a wealth of business development expertise from an impressive career as a business leader, spanning both global Pharma and Biotech. Beverley’s leadership of this strategically important function will be key to ensuring that the full potential of our proprietary next generation TPD technology is captured across therapy areas in order to bring transformative medicines to patients.”

Amphista’s new CBO, Beverley Carr, commented on her appointment,

“I’m delighted to join Amphista’s world class team. Amphista is founded on transformational science and I’m looking forward to driving the Company’s business development strategy as we execute our mission of building the leading TPD company.”

Beverley joins Amphista from Achilles Therapeutics plc (NASDAQ: ACHL), a clinical-stage biopharmaceutical company, where she was CBO. Previously Beverley was Vice President Business Development for the Immunoinflammation Therapy Area at GSK, where she led over twenty transactions for the immune-inflammation therapy area. Beverley has negotiated and closed major deals across all stages of drug discovery and development. She is a scientist by training with an MA and DPhil in chemistry from Oxford University and has an MBA from Cambridge University.

– Ends –

Media contacts:

Amphista Therapeutics
CEO NicolaThompson
+447464974714
nicki@amphista.com

Scius Communications
Katja Stout
+447789435990
katja@sciuscommunications.com

About Amphista Therapeutics

Amphista Therapeutics is focused on transforming the lives of patients with severe diseases including cancer. The company is applying its proprietary Amphista degrader platform to advance new approaches in targeted protein degradation (TPD), which address the challenges faced by the field and to realise the full therapeutic potential of this transformational approach. Founded by Advent Life Sciences, Amphista is a spin-out of TPD expert Professor Alessio Ciulli’s labs at the University of Dundee. The company has raised over $60M to date and is funded by leading life science investors including Forbion, Gilde Healthcare, Novartis Venture Fund, Advent Life Sciences, BioMotiv and Eli Lilly & Company.
For more information, please visit: http://www.amphista.com/

Oct 28
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Aura Biosciences Announces Pricing of Initial Public Offering

By Aura Biosciences, Press Release, Publicly Listed
Press Release.

 

October 28, 2021 09:39 PM Eastern Daylight Time

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Aura Biosciences, Inc. (“Aura”), a clinical-stage oncology company developing a novel class of virus-like drug conjugate therapies for multiple oncology indications, today announced the pricing of its initial public offering of 5,400,000 shares of common stock at a public offering price of $14.00 per share. The gross proceeds of the offering, before deducting underwriting discounts and commissions and other offering expenses payable by Aura, are expected to be $75.6 million. All of the shares are being offered by Aura. In addition, Aura has granted the underwriters a 30-day option to purchase up to an additional 810,000 shares of common stock at the initial public offering price less underwriting discounts and commissions.

The shares are expected to begin trading on the Nasdaq Global Market on October 29, 2021 under the ticker symbol “AURA.” The offering is expected to close on November 2, 2021, subject to the satisfaction of customary closing conditions.

Cowen, SVB Leerink and Evercore ISI are acting as joint book-running managers for the offering, and BTIG is acting as lead manager for the offering.

The registration statements relating to these securities became effective on October 28, 2021. The offering will be made only by means of a prospectus, copies of which may be obtained from Cowen and Company, LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, Attn: Prospectus Department, by telephone at (833) 297-2926, or by email at PostSaleManualRequests@broadridge.com; SVB Leerink LLC, Attention: Syndicate Department, 53 State Street, 40th Floor, Boston, MA 02109, by telephone at (800) 808-7525, ext. 6105, or by email at syndicate@svbleerink.com; or Evercore Group L.L.C., Attention: Equity Capital Markets, 55 East 52nd Street, 35th Floor, New York, New York 10055; by telephone at (888) 474-0200, or by email at ecm.prospectus@evercore.com.

This press release shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

About Aura Biosciences

Aura Biosciences, Inc. is a clinical-stage oncology company developing a novel technology platform based on virus-like drug conjugates (VDCs) to target and destroy cancer cells selectively while activating the immune system to create long lasting anti-tumor immunity. The VDC technology platform is based on the discoveries of NIH Distinguished Investigator Dr. John Schiller of the Center for Cancer Research at the National Cancer Institute. The company has the goal of developing this technology in multiple cancer indications with an initial focus on primary choroidal melanoma, a rare disease for which there are no approved drugs. Aura’s lead product candidate belzupacap sarotalocan (AU-011) is currently in Phase 2 development for the first line treatment of primary choroidal melanoma, a vision and life-threatening form of eye cancer where standard of care radioactive treatments leave patients with major vision loss and severe comorbidities. AU-011 was well tolerated in a Phase 1b/2 trial, demonstrating high rates of tumor control and vision preservation. Future pipeline applications for Aura’s technology include additional ocular oncology indications like choroidal metastases and solid tumor indications like non-muscle invasive bladder cancer. Aura is headquartered in Cambridge, MA.

Contacts

Investor and Media Contact:
Matthew DeYoung
Argot Partners
212-600-1902 | aura@argotpartners.com

Oct 11
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Aura Biosciences Presents Interim Phase 2 Safety Data Evaluating Suprachoroidal Administration of AU-011 in Patients with Choroidal Melanoma at the ASRS 2021 Annual Meeting

By Aura Biosciences, Press Release, Publicly Listed
Press Release.

 

CAMBRIDGE, Mass.–(BUSINESS WIRE)— Aura Biosciences, a clinical-stage oncology company developing a novel class of virus-like drug conjugate (VDC) therapies for multiple oncology indications, today announced the presentation of interim Phase 2 data with 7 months average follow up evaluating the safety of suprachoroidal (SC) administration of AU-011, the Company’s lead product candidate for the first-line treatment of primary choroidal melanoma, as a part of the American Society of Retina Specialists (ASRS) 2021 Annual Meeting.

There have been no related serious adverse events, dose limiting toxicities, or grade 3 adverse events observed during the study.

“These interim data presented today demonstrate that suprachoroidal administration may improve the therapeutic index and optimize treatment parameters,”

said Prithvi Mruthyunjaya, MD, MHS, Associate Professor of Ophthalmology and Director, Ocular Oncology Service, Byers Eye Institute at Stanford University, and presenter of the abstract.

“I believe this approach may provide an opportunity for patients who need a new first line treatment for early-stage disease, where all current treatments are extremely invasive and unfortunately result in severe vision loss in many patients.”

Phase 2 Trial Design and Timing

The Phase 2 trial is comprised of an open-label, dose escalation phase and a randomized, masked dose expansion phase that is assessing the safety and efficacy of ascending single- and repeat-doses of AU-011 via SC administration, followed by one or two laser applications per treatment. The randomized, dose expansion portion will be masked, sham-controlled and is designed to evaluate the safety and efficacy of the highest dose regimen of AU-011. Cohorts 1-5 have been fully enrolled (13 patients) and cohort 6 is currently enrolling in the Phase 2 study. The primary objective of the study is to assess safety and efficacy of AU-011 via SC administration for purposes of treating primary indeterminate lesions and choroidal melanoma.

The randomized phase of the trial is planned to begin in the second half of 2022 in patients with documented growth to establish the safety and efficacy of AU-011 and serve as the first pivotal trial for the treatment of indeterminate lesions and choroidal melanoma. The maximum treatment regimen anticipated for the randomized phase is three cycles of three weekly treatments of AU-011 at a dose of 80µg with 2 laser administrations.

Details from the ASRS 2021 Presentation:

Title: A Phase 2 Safety and Efficacy Trial of AU-011, a Virus-Like Drug Conjugate (VDC), with a Dose Escalation and a Randomized, Masked Expansion Phase
Presenter: Prithvi Mruthyunjaya, Stanford University
Session: Ocular Oncology Symposium
Date and Time: Monday, October 11, 2021 at 4:35pm ET

The presentation can be accessed by visiting the “Presentations” section of “News and Publications” page of the Aura Biosciences website.

About Choroidal Melanoma

Choroidal melanoma is a rare and aggressive type of eye cancer. Choroidal melanoma is the most common primary intraocular tumor in adults and develops in the uveal tract of the eye. No targeted therapies are available at present, and current radiotherapy treatments can be associated with severe visual loss and other long-term sequelae such as dry eye, glaucoma, cataracts, and radiation retinopathy. The most common current treatment is plaque radiotherapy, which involves surgical placement of a radiation device on the exterior of the eye over the tumor. The alternative is enucleation, or total surgical removal of the eye. Choroidal melanoma metastasizes in approximately 50 percent of cases with liver involvement in 80-90% of cases and, unfortunately, metastatic disease is universally fatal (source: OMF). There is a very high unmet need for a new vision sparing targeted therapy that could enable early treatment intervention for this life-threatening rare disease given the mortality rate in metastatic disease, lack of approved therapies, and the comorbidities of radioactive treatment options.

About AU-011

AU-011 is a first-in-class virus-like drug conjugate (VDC) therapy in clinical development for the first line treatment of choroidal melanoma. The virus-like component of the VDC selectively binds unique heparin sulphate proteoglycans (HSPGs), which are modified and overexpressed on the tumor cell surface of malignant cells in the choroid and AU-011 delivers a potent cytotoxic drug that is activated with infrared light. Upon activation with an ophthalmic laser, the cytotoxic drug rapidly and specifically disrupts the cell membrane of malignant cells with a pro-immunogenic cell death that can activate the immune system generating long term anti-tumor immunity. The unique specificity of tumor binding by the VDC enables the preservation of key eye structures, which may allow for the potential of preserving patients’ vision and reducing other long-term complications of radiation treatment. AU-011 can be delivered using equipment commonly found in an ophthalmologist’s office and does not require a surgical procedure, pointing to a potentially less invasive, more convenient therapy for patients and physicians. AU-011 for the treatment of choroidal melanoma is currently in Phase 2 clinical development and the company plans to expand the clinical program into choroidal metastasis.

About Suprachoroidal Administration

The suprachoroidal space (SCS®) injection treatment approach offers unprecedented access to the back of the eye where sight-threatening disease often occurs. Aura believes that delivering AU-011 into SCS within the eye, has the potential to offer certain advantages, including higher bioavailability at the tumor site and reduced exposure of non-targeted tissues, which may lead to an improved therapeutic index for AU-011. Collectively, these features could allow for the treatment of a wider range of tumor sizes, and, therefore, a larger number of patients. The Company is partnered with Clearside Biomedical for use of Clearside’s SCS Microinjector® for administration of AU-011 into the SCS. In preclinical research presented as part of the ARVO 2020 virtual program, AU-011 showed excellent distribution in the SCS, complete necrosis of tumors following laser activation in an animal model of choroidal melanoma and no clinical signs of anterior segment or posterior segment inflammation.

About Aura Biosciences

Aura Biosciences, Inc. is a clinical-stage oncology company developing a novel technology platform based on virus-like drug conjugates (VDCs) to target and destroy cancer cells selectively while activating the immune system to create long lasting anti-tumor immunity. The VDC technology platform is based on the discoveries of NIH Distinguished Investigator Dr. John Schiller of the Center for Cancer Research at the National Cancer Institute (NCI). The company has the goal of developing this technology in multiple cancer indications with an initial focus in ocular oncology, a group of rare diseases for which there are no approved drugs. Aura’s lead product candidate belzupacap sarotalocan (AU-011) is currently in Phase 2 development for the first line treatment of choroidal melanoma, a vision and life-threatening form of eye cancer where standard of care radioactive treatments leave patients with major vision loss and severe comorbidities. AU-011 was well tolerated in a Phase 1b/2 trial, demonstrating high rates of tumor control and vision preservation. Future pipeline applications for Aura’s technology include additional ocular oncology indications like choroidal metastases and solid tumor indications like non-muscle invasive bladder cancer. Aura is headquartered in Cambridge, MA. For more information, visit www.aurabiosciences.com or follow us on Twitter.

Investor and Media Contact:
Matthew DeYoung
Argot Partners
212-600-1902 | matthew@argotpartners.com

Sep 29
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Aura Biosciences Expands Executive Leadership Team and Board of Directors

By Aura Biosciences, Press Release, Publicly Listed
Press Release.

 

Chris Primiano, J.D., Appointed as Chief Business Officer
Antony Mattessich Appointed to the Board of Directors

CAMBRIDGE, MA – September 29,2021 – Aura Biosciences, a clinical-stage oncology company developing a novel class of virus-like drug conjugate (VDC) therapies for multiple oncology indications, today announced the appointment of Chris Primiano, J.D., as Chief Business Officer and the appointment of Antony Mattessich to its Board of Directors.

“We are delighted to welcome Chris to our team and Antony to the Board during an important transformational period for Aura. Both of their experiences in strategic advisory, drug development and commercialization in oncology and ophthalmology will be crucial as we work to advance AU-011 to pivotal development and expand our oncology pipeline,”

said Elisabet de los Pinos, Ph.D., founder and CEO of Aura.

“I am eager to be joining the Aura team to help expand its novel targeted oncology platform and global reach across multiple indications,”

said Mr. Primiano.

“AU-011 presents an opportunity to offer a new therapy that may eliminate the need for treatment with local radiotherapy for many patients with choroidal melanoma. This could be transformative in preserving vision and improving quality of life for these patients. I look forward to working with the rest of the management team to achieve Aura’s goal of advancing innovative oncology therapies to patients with life-threatening cancers.”

“It is a privilege to join the Board at this exciting time in the Company’s evolution,”

said Mr. Mattessich.

“I look forward to working alongside the Board and supporting Aura’s executive team as it lays the groundwork to transform Aura into a commercial-stage oncology company.”

Mr. Primiano joins Aura from Karyopharm Therapeutics, where he served in roles of increasing responsibility, most recently as Executive Vice President, Chief Business Officer, General Counsel and Secretary. Mr. Primiano played an important role in transitioning Karyopharm from 40 employees in a preclinical and early clinical development setting to 400 employees commercializing XPOVIO® (selinexor) across multiple indications. Prior to Karyopharm, Mr. Primiano was Counsel in the Boston office of Wilmer Cutler Pickering Hale and Dorr LLP. Mr. Primiano holds a Juris Doctor from Boston College Law School, a Master of Business Administration from the Boston College Carroll School of Management and a Bachelor of Arts in Political Economy and English from Georgetown University. He is a member of the bars of the Commonwealth of Massachusetts and the State of New York.

Mr. Mattessich is currently the Chief Executive Officer at Ocular Therapeutix. Prior to Ocular Therapeutix, he was Managing Director of Mundipharma International based in Cambridge, England. Previous to his time at Mundipharma, Mr. Mattessich ran the U.S. respiratory, dermatology and pediatrics group at Novartis. He also held several positions at Bristol-Myers Squibb, among them, Managing Director roles in Malaysia/Singapore and The Netherlands, and Head of Operations for the International Medicines Group. Mr. Mattessich holds a BA from the University of California at Berkeley and a master’s in international affairs from Columbia University.

About Aura Biosciences

Aura Biosciences, Inc. is a clinical-stage oncology company developing a novel technology platform based on virus-like drug conjugates (VDCs) to target and destroy cancer cells selectively while activating the immune system to create long lasting anti-tumor immunity. The VDC technology platform is based on the discoveries of NIH Distinguished Investigator Dr. John Schiller of the Center for Cancer Research at the National Cancer Institute (NCI). The company has the goal of developing this technology in multiple cancer indications with an initial focus in ocular oncology, a group of rare diseases for which there are no approved drugs. Aura’s lead product candidate belzupacap sarotalocan (AU-011) is currently in Phase 2 development for the first line treatment of choroidal melanoma, a vision and life-threatening form of eye cancer where standard of care radioactive treatments leave patients with major vision loss and severe comorbidities. AU-011 was well tolerated in a Phase 1b/2 trial, demonstrating high rates of tumor control and vision preservation. Future pipeline applications for Aura’s technology include additional ocular oncology indications like choroidal metastases and solid tumor indications like non-muscle invasive bladder cancer. Aura is headquartered in Cambridge, MA. For more information, visit www.aurabiosciences.com or follow us on Twitter.

 

Investor and Media Contact:

Matthew DeYoung
Argot Partners
914-330-6516 | matthew@argotpartners.com

Source: Aura Biosciences

Sep 21
Love0

Dr. Carolin Barth Joins MiroBio as CEO to Lead Precision Immunology Company Focused on Checkpoint Agonist Antibodies

By MiroBio, Press Release
Press Release.

 

  •     Former Novartis executive to lead emerging biotech with a novel precision approach to autoimmune disease, supported by recent senior appointments
  •     MiroBio is exclusively focused on checkpoint agonists produced by its unique discovery engine that comprehensively maps inhibitory checkpoint signaling pathways
  •     Clinical initiation with BTLA agonist program anticipated in 2022 with a rich checkpoint agonist pipeline in earlier stages of development

Oxford, U.K. – 21 September 2021 – MiroBio, a biotechnology company advancing a new class of precision therapies designed to address the immune imbalance that drives autoimmune disease, today announced the appointment of Carolin Barth, M.D., as chief executive officer. Dr. Barth will lead MiroBio through the next major stage of its growth as its lead program, MB272, transitions into clinical development. Dr. Barth’s appointment follows other recent senior appointments to support operational expansion including Julian Hirst, formerly of Immunocore, as chief financial officer and Lynne Murray, Ph.D., formerly of AstraZeneca, as senior vice president of research & development.

MiroBio is uniquely focused on a new area of precision immunology using checkpoint agonist antibodies. Each of MiroBio’s candidates is designed to selectively activate a specific immune checkpoint pathway, with the goal of restoring immune homeostasisCheckpoint receptor pathways are powerful regulators of immune reactivity. Currently approved checkpoint inhibitor therapies use receptor antagonism to ‘take the brakes off’ the immune system to fight cancer. By contrast, MiroBio’s therapies are designed to specifically activate inhibitory checkpoint pathways through receptor agonism. This approach is intended to dampen the immune overactivity that drives immune-mediated inflammatory diseases and restore a balanced immune state. MiroBio’s most advanced program, MB272, targets the immune receptor BTLA and is readying for Phase 1 initiation in early 2022. A PD-1 agonist program, MB151, is also in preclinical development, followed by a growing pipeline of checkpoint agonist antibodies in earlier-stage development.

“It is the ideal time for Carolin to assume leadership of MiroBio,”

said Eliot Charles, Ph.D, executive chairman of MiroBio.

“The Company is approaching the clinic with an exciting pipeline that represents the most substantial portfolio in an important new class of autoimmune therapies. MiroBio has built its proprietary drug discovery and development engine to comprehensively evaluate the more than 70 inhibitory checkpoints for new drug programs. Carolin’s strategic mindset and wealth of experience in the successful development and commercialization of several ground-breaking therapies, particularly in the areas of immunology and autoimmune disease, will be invaluable as MiroBio seeks to deliver these promising new medicines to patients.  Together with MiroBio’s other recent hires, I am confident that the team is exceptionally well-suited to drive the Company’s next stages of growth and longer-term success.”

Dr. Barth most recently served at Novartis as global head of commercial and pipeline strategy, cell and gene. She held roles at Novartis of increasing responsibility over 17 years, including serving as global program head for several development programs and commercial launch leader in the areas of dermatology, rheumatology and chronic myeloid leukemia. As global head, dermatology she played an instrumental role in the development and commercialization of transformational antibody therapies such as COSENTYX and XOLAIR.

“I am very excited to join MiroBio – a company that stands out for its deep expertise, focus, and bold vision for delivering a new, better class of therapies for autoimmune disease. With its agonist antibody design expertise and unique approach to understanding both checkpoint pathway function and its potential in a wide variety of disease settings, I believe MiroBio can deliver several best-in-class new medicines that offer patients more efficacious and safer treatment options,”

said Dr. Barth.

“I look forward to working with the MiroBio team to continue building the entrepreneurial and patient-centric culture that will help us succeed through a critical phase of growth and development.”

MiroBio’s drug discovery engine combines proprietary discovery and development technologies with strategic mapping of the inhibitory signalling landscape across immune cell types and diseases. It builds on 15 years of deep foundational research at the University of Oxford and is designed to deliver best-in-class agonistic antibodies in addition to an unmatched understanding of inhibitory checkpoint pathways. MiroBio antibodies are uniquely designed to robustly agonize a specific immune checkpoint receptor. MiroBio’s pathway database is informed by a growing global collaborator network that allows the Company to catalogue diseased inhibitory receptor states which will inform the future selection of patient populations for clinical trial enrichment and highly targeted indications.

Dr. Barth’s appointment follows the recent appointments of other senior strategic leaders to oversee the financial and R&D functions of MiroBio. CFO Julian Hirst previously served as corporate finance director and head of investor relations at Immunocore where he played an instrumental role in raising over $930 million in three private rounds, a venture debt financing and an IPO. He previously spent over 20 years in investment banking including leading the European technology corporate finance team at UBS where he was a managing director. SVP of R&D Dr. Lynne Murray joined MiroBio from AstraZeneca where she was head of regeneration, early respiratory and immunology. Dr. Murray’s earlier roles at AstraZeneca were in the partnering & strategy department and as head of fibrosis biology at MedImmune, where she established MedImmune/AstraZeneca’s fibrosis pipeline that delivered multiple assets into the clinic.  Before that, she served as a director of pharmacology at Promedior and in R&D at antibody developer Centocor.

About Checkpoint Agonist Antibodies

MiroBio is creating a new class of therapies for autoimmune disease: Checkpoint agonist antibodies. These therapies are designed to precisely restore a balanced immune response and prevent the body from attacking its own cells. Checkpoint pathways are powerful regulators of immune cell function. Each has its own role in specific types of immune cells and differing activity in disease.  By selectively activating, or agonizing, the appropriate checkpoint receptor, MiroBio’s therapies can deliver a highly potent inhibitory signal to overactive immune cells without being broadly immunosuppressive.

MiroBio is comprehensively mapping the 70+ inhibitory checkpoint receptor pathways with its proprietary discovery engine, I-ReSToRE, to identify therapeutic opportunities for autoimmune disease. MiroBio’s antibody engineering capabilities and depth of expertise in inhibitory checkpoint signalling pathways are the foundation for its scientific leadership in this vast new area of precision immunology.

About MiroBio (www.mirobio.com)

MiroBio is a biotechnology company advancing a new class of precision therapies designed to address the immune imbalance that drives autoimmune disease. Its proprietary drug discovery engine, I-ReSToRE, comprehensively evaluates inhibitory receptors for therapeutic potential and designs checkpoint agonist antibody candidates that precisely modulate immune signalling to restore health. MiroBio’s pipeline includes several candidates against validated and novel targets with potential in a variety of autoimmune diseases. MiroBio is based in Oxford, U.K.

For more information about MiroBio contact:

Ten Bridge Communications

TBCMiroBio@tenbridgecommunications.com

Sep 21
Love0

Amphista Therapeutics Appoints New Chairman, Joshua T. Brumm

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

 

Glasgow, UK, 21 September 2021 – Amphista Therapeutics, a leader in next generation targeted protein degradation (TPD) approaches, today announced the appointment of Joshua T. Brumm as its new Independent Chairman, succeeding Satish Jindal.

Amphista’s CEO Dr Nicola Thompson said,

“On behalf of the Amphista team I am thrilled to welcome someone of Josh’s calibre to the Board. I look forward to working with Josh as we rapidly develop our pipeline of TPD therapeutics in oncology and beyond and unlock the full therapeutic potential of this exciting new modality. I would also like to thank our outgoing Chairman, Satish Jindal, who has tirelessly given his support as we have successfully advanced Amphista to this next stage of growth.”

Josh Brumm, Amphista’s new Chairman, commented on his appointment,

“I am delighted to join Amphista as its Chairman. The company, founded by leading pioneers in the field, has a unique, truly differentiated approach to targeted protein degradation and the opportunity to develop transformational therapies for patients. The potential of the technology is extremely exciting and together with Amphista’s world class team, I’m looking forward to executing on our mission of building the leading TPD company.”

Founding investor Raj Parekh, General Partner, Advent Life Sciences added,

“I am excited to welcome Josh as Amphista’s new Chairman. He brings a wealth of experience that will compliment the work that Nicki and the team have achieved and will support Amphista’s ambitious strategy”.

Josh Brumm is the president and CEO of Dyne Therapeutics, Inc. (NASDAQ: DYN), a leading muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases. Before joining Dyne, he served as COO and CFO of Kaleido Biosciences, leading the company’s IPO, and helped bring the lead program into Phase 2 development.  Prior to this he was COO and CFO at Versartis, where he oversaw the company’s financial strategy including the successful completion of its IPO. Brumm has also held senior positions at Pharmacyclics, ZELTIQ Aesthetics and Proteolix, Inc as well as investment banking positions at Citigroup Global Markets, Inc. and Morgan Stanley. Over the course of his career, he has raised nearly $2 billion in capital. He holds a B.A. in business administration from the University of Notre Dame.

– Ends –

Media contacts:

Amphista Therapeutics
CEO NicolaThompson
+447464974714
nicki@amphista.com

Scius Communications
Katja Stout
+447789435990
katja@sciuscommunications.com

About Amphista Therapeutics

Amphista Therapeutics is focused on transforming the lives of patients with severe diseases including cancer. The company is applying its proprietary Amphista degrader platform to advance new approaches in targeted protein degradation (TPD), which address the challenges faced by the field and to realise the full therapeutic potential of this transformational approach. Founded by Advent Life Sciences, Amphista is a spin-out of TPD expert Professor Alessio Ciulli’s labs at the University of Dundee. The company has raised over $60M to date and is funded by leading life science investors including Forbion, Gilde Healthcare, Novartis Venture Fund, Advent Life Sciences, BioMotiv and Eli Lilly & Company.

For more information, please visit: http://www.amphista.com

Sep 15
Love0

Rappta Therapeutics Appoints Sunjeet Sawhney as Chief Executive Officer

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

 

  • Experienced industry leader with successful track-record in oncology to lead Rappta through its next stages of growth
  • Rappta’s industry-leading phosphatase technology platform offers a new paradigm in oncology
  • Lead compounds on track to enter IND/CTA-enabling studies in the first half of 2022

Helsinki, Finland and London, UK, 15 September, 2021: Rappta Therapeutics (“Rappta”), which is focused on developing first-in-class anti-cancer drugs reactivating protein phosphatase 2A (PP2A), announces today the appointment of Sunjeet Sawhney as Chief Executive Officer (CEO) with immediate effect. Rappta’s Co-founder and Founding CEO Mikko Mannerkoski will move to be Chief Financial Officer (CFO) as planned.

With over 20 years of experience in the industry, Sunjeet joins Rappta from Ipsen where he has most recently been the Global Head of the Oncology Franchise. Prior to this Sunjeet led businesses across a range of geographies both in biotech and major pharmaceutical companies. Sunjeet holds a MSc in Immunology from the University of London and will be based in London, UK.

In October 2020, Rappta raised its first round of financing from blue-chip investors Advent Life Sciences, Novartis Bioventures, Novo Holdings, and a family office. Under the scientific leadership of Dr. Goutham Narla, Rappta’s Co-Founder and Chief Scientific Officer, the Company is developing first-in-class anti-cancer drugs reactivating PP2A – a critical enzyme regulating protein de-phosphorylation and a key tumor suppressor which to date has been very difficult to target pharmaceutically. Rappta’s strengthened leadership team is well-positioned to take its first-in-class program of PP2A-reactivating anti-cancer molecules to IND/CTA-enabling studies in the first half of 2022.

Sunjeet Sawhney, CEO of Rappta Therapeutics, commented:

“I am honored to have been given the opportunity to lead Rappta. The Company’s robust first-in-class program of anti-cancer drugs that reactivate PP2A represents an exciting clinical and commercial opportunity, much like kinase inhibitors did more than two decades ago. I am particularly impressed with the rigorous science and the swift progress that the team has made in moving the program forward from discovery towards development. I look forward to working together with the Rappta team in progressing our lead compounds while continuing to leverage our proprietary tools and unique understanding of PP2A to build a platform of a new class of anti-cancer drugs.”

Mikko Mannerkoski, CFO and Co-Founder of Rappta Therapeutics, commented:

“I look forward to working with Sunjeet, an experienced industry leader, to guide the Company through the next important stages of growth. As an entrepreneur and co-founder, it gives me great pride that Rappta has in a very short period of time built a robust drug development program that is ready to enter IND/CTA-enabling studies and subsequent first-in-human clinical trials, which has the potential to offer real benefit to patients while creating value for our shareholders.”

Goutham Narla, Rappta’s CSO and Co-Founder, commented:

“I am thrilled to work with Sunjeet on this opportunity to build a new platform and a novel class of pharmaceuticals to treat cancer. We have a unique team whose deep understanding of the PP2A biochemistry, structural biology, biogenesis, medicinal chemistry, and drug development represent a great combination of expertise to translate these discoveries into meaningful outcomes for patients. Sunjeet’s vast industry experience will add a new dimension to our competencies and accelerate the growth of the Company.”

ENDS

 

For more information please contact:

Rappta Therapeutics
Sunjeet Sawhney, CEO
+44 74 7244 6005
sunjeet.sawhney@rappta-therapeutics.com

Optimum Strategic Communications
Mary Clark, Manel Mateus, Vici Rabbetts
+44 (0) 20 3922 1906
rappta@optimumcomms.com

About Rappta Therapeutics

Rappta Therapeutics, a private biotech with operations in Finland, UK and the US, is developing first-in-class anti-cancer drugs activating protein phosphatase 2A (PP2A). It has developed proprietary tools and a unique understanding of PP2A which allows it to therapeutically reactivate PP2A, a critical enzyme regulating protein de-phosphorylation and tumor growth, with the potential to create a new class of anti-cancer drugs. Rappta has a strong scientific, management and commercial team. Its scientific team, led by CSO and co-founder, Professor Goutham Narla, Division Chief of Genetic Medicine at the University of Michigan, represents world-leading expertise in PP2A. The team is supported by a Scientific Advisory Board led by Dr. William Hahn, Professor of Medicine at the Harvard Medical School and the Chief Operating Officer of the Dana-Farber Cancer Institute and a Clinical Advisory Board lead by Dr. Mathew Galsky, Professor of Medicine and Director of Genitourinary Medical Oncology at the Icahn School of Medicine at Mount Sinai. The Company is backed by blue-chip investors Advent Life Sciences, Novartis Bioventures, Novo Holdings and a family office. For more information, go to www.rappta-therapeutics.com.

About PP2A

Reversible phosphorylation is a fundamental mechanism controlling all cell signaling and communication and this process is regulated through the opposing actions of phosphatases (which remove phosphate groups from proteins) and kinases (which add phosphate groups to proteins). Altered cellular signaling as a result of protein hyperphosphorylation, results in the sustained growth of malignant cells and is a hallmark of human cancer development and progression.

Protein Phosphatase 2A (PP2A) is a serine/threonine phosphatase that functions as a tumor suppressor by negatively regulating multiple oncogenic signaling pathways responsible for driving cancer progression. PP2A is made up of three subunits, that form a complete and active enzyme when bound together. The active enzyme is comprised of a scaffolding subunit (A), serving as the structural platform for the assembly of the catalytic (C) subunit and one substrate directing regulatory (B) subunit. In cancer, the tumor-suppressive activity of PP2A is often disrupted as a result of the inability of the three subunits to bind together correctly, rendering the PP2A enzyme inactive. This inactivation of PP2A, leads to increased oncogenic signaling, driving cancer progression and growth. Therefore, the reactivation of PP2A affords a unique therapeutic strategy to restore PP2A activity and cellular homeostasis, that can be used for the treatment of cancer and a broad range of other diseases.

Sep 09
Love0

Eloxx Pharmaceuticals Announces Fast Track Designation for ELX-02 for the Treatment of Cystic Fibrosis Patients with Nonsense Mutations

By Eloxx Pharmaceuticals, Press Release, Publicly Listed
Press Release.

 

September 9, 2021

WATERTOWN, Mass., Sept. 09, 2021 (GLOBE NEWSWIRE) — Eloxx Pharmaceuticals, Inc. (Nasdaq: ELOX), today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for ELX-02, a drug candidate intended to treat cystic fibrosis patients with nonsense mutations. ELX-02 is currently in Phase 2 clinical trials in CF patients affected by nonsense mutations in the CFTR (CF transmembrane conductance regulator) gene for whom there are no effective disease modifying therapies. The Phase 2 trials are designed to evaluate the safety of ELX-02 and assess its biological activity, and Eloxx expects to present data from the first four treatment arms in the fourth quarter of 2021.

“We are delighted to receive Fast Track Designation from the FDA for ELX-02 as the need for patients remains significant. The ability to have greater access to the FDA and their guidance on the regulatory pathway for ELX-02 can help provide the ability to work with the urgency needed on behalf of CF patients with nonsense mutations,”

said Sumit Aggarwal, President and CEO of Eloxx.

Fast Track Designation is granted to drugs being developed for the treatment of serious or life-threatening diseases or conditions where there is an unmet medical need. The purpose of the provision is to help facilitate development and expedite the review of drugs to treat serious or life-threatening conditions so that an approved product can reach the market expeditiously. Sponsors of drugs that receive Fast Track Designation have the opportunity for more frequent interactions with the FDA review team throughout the development program.

ELX-02 has previously been granted orphan drug designation by the FDA and orphan medicinal product designation by the European Medicines Agency.

About Eloxx Pharmaceuticals

Eloxx Pharmaceuticals, Inc. is engaged in the science of ribosome modulation, leveraging both its innovative TURBO-ZM™ chemistry technology platform in an effort to develop novel Ribosome Modulating Agents (RMAs) and its library of Eukaryotic Ribosome Selective Glycosides (ERSGs). Eloxx’s lead investigational product candidate, ELX-02, is a small molecule drug candidate designed to restore production of full-length functional proteins. ELX-02 is in clinical development focusing on cystic fibrosis. ELX-02 is an investigational drug that has not been approved by any global regulatory body. Eloxx also has preclinical programs focused on select rare diseases including inherited diseases, cancer caused by nonsense mutations, kidney diseases, including autosomal dominant polycystic kidney disease, as well as rare ocular genetic disorders.

For more information, please visit www.eloxxpharma.com.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements other than statements of present and historical facts contained in this press release, including without limitation, statements regarding the expected timing of trials and results from clinical studies of our product candidate and the potential of our product candidate to treat nonsense mutations are forward-looking statements. Forward-looking statements can be identified by the words “aim,” “may,” “will,” “would,” “should,” “expect,” “explore,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “contemplate,” “believe,” “estimate,” “predict,” “potential,” “seeks,” or “continue” or the negative of these terms similar expressions, although not all forward-looking statements contain these words.

Forward-looking statements are based on management’s current plans, estimates, assumptions and projections based on information currently available to us. Forward-looking statements are subject to known and unknown risks, uncertainties and assumptions, and actual results or outcomes may differ materially from those expressed or implied in the forward-looking statements due to various important factors, including, but not limited to: our ability to progress any product candidates in preclinical or clinical trials; the uncertainty of clinical trial results and the fact that positive results from preclinical studies are not always indicative of positive clinical results; the scope, rate and progress of our preclinical studies and clinical trials and other research and development activities; the competition for patient enrollment from drug candidates in development; the impact of the global COVID-19 pandemic on our clinical trials, operations, vendors, suppliers, and employees; our ability to obtain the capital necessary to fund our operations; the cost of filing, prosecuting, defending and enforcing any patent claims and other intellectual property rights; our ability to obtain financial in the future through product licensing, public or private equity or debt financing or otherwise; general business conditions, regulatory environment, competition and market for our products; and business ability and judgment of personnel, and the availability of qualified personnel and other important factors discussed under the caption “Risk Factors” in our Quarterly Report on Form 10-Q for the quarterly period ended June 30, 2021, as any such factors may be updated from time to time in our other filings with the SEC, accessible on the SEC’s website at www.sec.gov and the “Financials & Filings” page of our website at https://investors.eloxxpharma.com/financial-information/sec-filings.

All forward-looking statements speak only as of the date of this press release and, except as required by applicable law, we have no obligation to update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.

Contact

Investors
John Woolford
john.woolford@westwicke.com
443.213.0506

Media
Laureen Cassidy
laureen@outcomescg.com

Source: Eloxx Pharmaceuticals, Inc.