On May 18, 2021 Photocure ASA (OSE: PHO), the Bladder Cancer Company, reported that notification was given to its partner and exclusive distributor, Genotests SpA by the Instituto de Salud Pública de Chile that the Marketing Authorization Application ("MAA") for Hexvix has been accepted for regulatory review (Press release, PhotoCure, MAY 18, 2021, View Source [SID1234580234]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

During the regulatory review process, Genotests will begin to prepare for the commercial launch of Hexvix in Chile. In August 2020, Photocure entered into an agreement with Genotests, a privately held company specializing in the marketing of genetic tests for cancer and targeting cancer specialists, to exclusively market and distribute Hexvix in Chile. Under the terms of the agreement, Genotests will fund all costs to secure regulatory approval and commercialize Hexvix in Chile. Photocure will manufacture the product and support Genotests with regulatory activities, training, and promotional materials.

"Acceptance of the Hexvix MAA with Fast Track designation by the Chilean regulatory authority is a milestone for both Photocure and Genotests as we move closer to bringing this important solution to bladder cancer patients in the region," said Dan Schneider, President and CEO of Photocure. "With Genotests’ dedication and strong execution on this initiative, we are hopeful that Hexvix will become accessible to patients in Chile in the fourth quarter of this year. This partnership represents Photocure’s first step into the South American continent, and supports our strategy of expanding the availability of Hexvix into new territories to ensure that patients and urologists have access to the benefits of using blue light cystoscopy (BLC). The procedure with Hexvix, or Cysview, is recommended in consensus treatment guidelines for the management of bladder cancer around the world."

"It is a privilege to bring this fantastic product to Chile" said Oscar Varas, founder and owner of Genotests. "There are approximately 1,500 new bladder cancer cases annually in our country, an estimated 5,000 TURBT* procedures and nearly 11,000 surveillance cystoscopies. We look forward to building on our established network and the successful demonstration of Hexvix Blue Light Cystoscopy, conducted in collaboration with Karl Storz, at the Chilean Urology Conferences in 2018 and 2019, to launch blue light cystoscopy with Hexvix into the Chilean urology community as soon as possible."

*TURBT: trans-urethral resection of bladder tumors

Note to editors:

All trademarks mentioned in this release are protected by law and are registered trademarks of Photocure ASA

About Bladder Cancer

Bladder cancer ranks as the seventh most common cancer worldwide with 1 720 000 prevalent cases (5-year prevalence rate)1a, 573 000 new cases and more than 200 000 deaths annually in 2020.1b

Approx. 75% of all bladder cancer cases occur in men.1 It has a high recurrence rate with an average of 61% in year one and 78% over five years.2 Bladder cancer has the highest lifetime treatment costs per patient of all cancers.3

Bladder cancer is a costly, potentially progressive disease for which patients have to undergo multiple cystoscopies due to the high risk of recurrence. There is an urgent need to improve both the diagnosis and the management of bladder cancer for the benefit of patients and healthcare systems alike.

Bladder cancer is classified into two types, non-muscle invasive bladder cancer (NMIBC) and muscle-invasive bladder cancer (MIBC), depending on the depth of invasion in the bladder wall. NMIBC remains in the inner layer of cells lining the bladder. These cancers are the most common (75%) of all BC cases and include the subtypes Ta, carcinoma in situ (CIS) and T1 lesions. In MIBC the cancer has grown into deeper layers of the bladder wall. These cancers, including subtypes T2, T3 and T4, are more likely to spread and are harder to treat.4

1 Globocan. a) 5-year prevalence / b) incidence/mortality by population. Available at: View Source, accessed [April 2021].
2 Babjuk M, et al. Eur Urol. 2019; 76(5): 639-657
3 Sievert KD et al. World J Urol 2009;27:295–300
4 Bladder Cancer. American Cancer Society. View Source

About Hexvix/Cysview (hexaminolevulinate HCl)

Cysview is the tradename in the U.S. and Canada, Hexvix is the tradename in all other markets. Photocure is commercializing Cysview/Hexvix directly in the U.S. and Europe, and has strategic partnerships for the commercialization of Hexvix/Cysview in China, Canada, Chile, Australia and New Zealand. Please refer to View Source for further information on our commercial partners.

On May 18, 2021 Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, reported financial results for the first quarter 2021 and recent business progress (Press release, Abeona Therapeutics, MAY 18, 2021, View Source [SID1234580186]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We are off to a fast start in 2021, reflecting our intense focus on execution," said Michael Amoroso, Chief Executive Officer of Abeona. "We are focused on completing enrollment in the EB-101 Phase 3 pivotal VIITAL study, gaining clarity on a regulatory path for ABO-102 in MPS IIIA, producing the first lot of Abeona-produced clinical grade product for ABO-102, and reporting additional neurocognitive and biomarker data from both the ABO-102 Transpher A and the ABO-101 Transpher B studies. We also have a robust preclinical pipeline, and we are conducting research assessing AAV capsids with the aim of IND-enabling studies in two to three eye indications. Importantly, our focus on building the right talent and experience on our leadership team positions us well to continue to advance our clinical programs toward delivering meaningful milestones later this year."

First Quarter and Recent Highlights

Corporate Updates

Appointed Michael Amoroso as President, Chief Executive Officer (CEO) and a member of the company’s Board of Directors.
Abeona strengthened its Board of Directors with the appointment of four new independent members who bring relevant operational leadership experience with life sciences companies, including in the areas of clinical development, manufacturing of cell therapy and gene therapy products, and corporate and financial compliance, to support the company’s focus on driving future growth and creating additional shareholder value.
EB-101 (Autologous, Gene-Corrected Cell Therapy)

Patient enrollment is ongoing for the EB-101 pivotal Phase 3 VIITAL study for RDEB. The company continues to expect to complete enrollment in the VIITAL study in 2021, depending upon the impact from the COVID-19 pandemic, including travel restrictions and safety concerns.
To support ongoing enrollment and commercial preparation, Abeona continues to work toward adding a second clinical site in the VIITAL study by the third quarter of 2021.
Presented data on long-term patient-reported outcomes following EB-101 treatment of RDEB wounds at the Society for Investigative Dermatology (SID) Virtual Meeting 2021, held from May 3-8, 2021. The results showed durable wound healing and reduction in pain through 6 years after treatment.
ABO-102 and ABO-101 (AAV-based Gene Therapies)

Presented new positive data from two ongoing Phase 1/2 clinical trials of ABO-102 in MPS IIIA and ABO-101 in MPS IIIB in late-breaking platform oral presentations at the 17th Annual WORLDSymposium in February 2021.
The FDA granted Abeona’s request and scheduled a Type B meeting in June 2021 to discuss the data-to-date from the ABO-102 Transpher A study and the potential path to a Biologics License Application (BLA) submission for ABO-102 in MPS IIIA.
Preclinical Pipeline

Presented new data supporting the potential of Cre-mediated dual AAV vector technology to enable delivery of large genes targeted for treatment of Stargardt disease during an oral presentation at the Association for Research in Vision and Ophthalmology (ARVO) 2021 Annual Meeting, held virtually from May 1-7, 2021.
Abeona recently completed non-human primate (NHP) studies comparing several capsids with AAV8, the industry standard for intraocular administration, in order to further understand and characterize the company’s AAV capsids. The results showed that AAV204, part of Abeona’s in-licensed AIM capsid library, was superior to AAV8 using a recently developed route of ocular administration.
In a separate NHP experiment, the company’s AAV214 and AAVV214D5 capsids were tested versus AAV8 administered subretinally. Both capsids demonstrated nearly identical levels of transduction of photoreceptor and retinal pigmented epithelium (RPE) cells, which are the cell types most frequently affected in inherited retinal diseases, when compared with AAV8.
The results from the recently completed NHP studies support Abeona’s strategy to advance multiple preclinical eye programs into the clinic.
First Quarter Financial Results

Cash, cash equivalents and short-term investments totaled $86.8 million as of March 31, 2021, compared to $95.0 million as of December 31, 2020. Net cash used in operating activities was $13.6 million for the first quarter of 2021.

Research and development (R&D) expenses were $7.2 million for the first quarter of 2021, compared to $6.8 million in the comparable period in 2020. The increase in R&D expenses was primarily due to increased clinical and development work for the company’s gene and cell therapy product candidates, and increased salary and related costs. General and administrative (G&A) expenses were $6.6 million for the first quarter of 2021, compared to $6.4 million in the same period in 2020. The increase in G&A expenses was primarily due to increased professional fees, partially offset by decreased salary and related costs, and decreases in net other G&A expenses.

Net loss was $16.0 million for the first quarter of 2021, compared to net loss of $48.2 million for the comparable period in 2020. The decrease in net loss was primarily due to the non-cash impairment charge of $32.9 million related to the termination of the license agreement with REGENXBIO in the first quarter of 2020.

Conference Call Details

Abeona Therapeutics will host a conference call and webcast on Tuesday, May 25, 2021 at 8:30 a.m. ET, to discuss its first quarter 2021 financial results and business update. To access the call, dial 888-506-0062 (U.S. toll-free) or 973-528-0011 (international) and Entry Code: 552097 five minutes prior to the start of the call. A live, listen-only webcast and archived replay of the call can be accessed on the Investors & Media section of Abeona’s website at www.abeonatherapeutics.com. The archived webcast replay will be available for 30 days following the call.

On May 18, 2021 Idera Pharmaceuticals, Inc. ("Idera" or the "Company") (Nasdaq: IDRA) reported that it will not continue ILLUMINATE-301, the Company’s trial of tilsotolimod in combination with ipilimumab versus ipilimumab alone in patients with anti-PD-1 refractory advanced melanoma, to its overall survival (OS) primary endpoint (Press release, Idera Pharmaceuticals, MAY 18, 2021, View Source [SID1234580203]). The Company reported in March 2021 that the trial did not meet its primary endpoint of objective response rate (ORR). Full results from the study will be presented in a future publication.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Since receiving the disappointing ORR results from ILLUMINATE-301, we have interrogated the full data set and consulted with our study Steering Committee, our partners at Bristol Myers Squibb (BMS), and other stakeholders regarding next steps for the trial. Our conclusion is that the totality of the data, with all patients having completed the study treatment, does not support the likelihood that the combination of tilsotolimod with ipilimumab would achieve a statistically significant OS benefit over ipilimumab alone," stated Vincent Milano, Idera’s Chief Executive Officer. "I want to personally thank all the patients and investigators for their dedication to the study."

Added Mr. Milano, "We remain committed to our additional trials and are continuing to enroll and treat patients in ILLUMINATE-206, our Phase 2 study of tilsotolimod in combination with BMS’s nivolumab and ipilimumab for patients with microsatellite-stable colorectal cancer and to support AbbVie in the form of study drug in their trial for patients with head and neck squamous cell carcinoma."

Continued Mr. Milano, "As we turn our attention toward the future, we continue to be active in our goal to identify and secure new development or commercial-stage assets. We have an exceptional team with a strong track record and passion for helping patients that I believe can be beneficial in delivering results from promising compounds."

The Company is also announcing that Elizabeth Tarka, M.D., the Company’s Chief Medical Officer since July 2019, will be leaving the Company on May 28, 2021. Dr. Tarka will continue working with Idera on a consulting basis.

"I want to thank Liz for helping us deliver ILLUMINATE-301 and for her many other contributions over the past two years," stated Mr. Milano. "We wish her all the best in her future endeavors."

On May 18, 2021 Brooklyn ImmunoTherapeutics, Inc. (NYSE American: BTX) ("Brooklyn" or the "Company"), a biopharmaceutical company currently focused on exploring the role that cytokine and gene editing/cell therapy can have in treating patients with cancer, blood disorders and monogenic diseases, reported that management will host a conference call on Tuesday, May 25, 2021 at 4:30 p.m. Eastern Time (ET) (Press release, Brooklyn ImmunoTherapeutics, MAY 18, 2021, View Source [SID1234580219]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Participants are asked to pre-register for the call through the following link: View Source Please note that registered participants will receive their dial in number upon registration and will dial directly into the call.

The conference call will also be available through a live webcast found here.

The Company invites participants to pre-submit questions to [email protected] until 5:00 p.m. Eastern Time (ET) on May 20, 2021 (live questions will not be accepted during the call). Please use the subject line, "BTX Call Questions" for this correspondence. The Company will do its best to address all pre-submitted questions at the conclusion of prepared remarks.

On May 18, 2021 Outpace reported that the company is taking cell and gene control technology originally in-licensed by Lyell out of that company’s toolbox and into a broad partnering model it thinks could transform the field (Press release, Outpace Bio, MAY 18, 2021, View Source [SID1234637772]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Outpace Bio Inc. launched in March with a $30 million series A round led by Artis Ventures and Lyell Immunopharma Inc., with participation by Abstract Ventures, Civilization Ventures, Mubadala Capital, Playground Global, Sahsen Ventures and WRF Capital.

The company’s leadership and scientific teams include inventors of de novo protein design technology developed at University of Washington’s Institute for Protein Design (IPD), many of whom joined Lyell when their inventions were brought under the well-funded cancer cell therapy company’s roof. That IP is now assigned to Outpace.

"The reason we’re spinning out from Lyell is that even massively capitalized companies can’t chew on all the hypotheses we want to address while moving at full speed on internal clinical development," said Outpace co-founder and CEO Marc Lajoie. "We have a lot to offer the field, and Lyell will benefit from that."

Outpace is developing "a whole platform of control modalities" to program cell and gene therapies for greater potency and safety, said Lajoie.

That includes synthetic biology strategies using "AND" or "NOT" Boolean logic gates to recruit or exclude signaling molecules to specific subcellular locations, control protein turnover, or require a specific combination of signals to unlock a desired function.

By designing fit-for-purpose proteins that don’t exist in nature, the company can go beyond standard strategies that manipulate expression of individual genes.

"First-generation approaches have been mainly focused on over-expressing this gene, or knocking out that gene," which can face limitations when a gene is integral to cell function in one setting but hampers it in another, said Lajoie. In contrast, he said, working at the protein level gives Outpace the ability to introduce desired changes in more context-specific ways.

Rather than develop its own pipeline, which would require investing in clinical development, the company is partnering with others to develop products via milestone- and royalty-driven deals. "The opportunity here is to create a step change in the field, and to be able to do that, we needed to focus our efforts on early development," Lajoie said.

In some cases, Outpace and the partner company will jointly design the program from the bottom-up, while in others, the partner will come to Outpace and use the company’s existing technology to solve a specific problem in an ongoing program, said co-founder and CSO Scott Boyken.

While the products developed through collaborations will belong to the partner companies, the underlying control technologies developed through the process belong to Outpace. "We can leverage our progress on the products we’re working on to increase our efficiency for other projects in the future," Lajoie said.

Outpace’s most advanced program is a collaboration with Lyell to develop cell therapies with controlled expression of an undisclosed cytokine. Other programs in development include a CAR cell therapy resistant to exhaustion, and a strategy for drug-induced gene regulation; the partnering status of these programs is undisclosed.

Lajoie said that while Outpace’s technology can be applied to any cell or gene therapy, the company’s "sweet spot" is T cell therapies for cancer.

Lajoie and Boyken co-authored a 2020 Science study with Fred Hutchinson Cancer Research Center professor Stanley Riddell showing the Co-LOCKR (Colocalization-dependent Latching Orthogonal Cage/Key pRoteins) technology they developed, part of Outpace’s IP portfolio, directed T cells to kill target cells expressing precise combinations of cell surface antigens, opening the door to more selective tumor targeting.

"There’s no single antigen that really distinguishes cancer cells from healthy cells, but there are aberrant combinations of antigens. That’s the unique handle we really wanted to pursue in that collaboration," said Lajoie.

He believes the series A round will give the company approximately three years of runway.

At least two other companies have been founded to develop synthetic biology control mechanisms for partners’ cell therapies.

Cell Design Labs Inc. was acquired by Gilead Sciences Inc. (NASDAQ:GILD) for $175 million up front and $322 million in total milestones after Gilead’s 2017 acquisition of Cell Design Labs’ partner, Kite Pharma Inc. The University of California San Francisco spinout, which raised $34.4 million in venture funds, developed technology to control CAR T cell function; its UCSF founders published two Science Translational Medicine studies on the technology last month.

Senti Biosciences Inc., which raised a $105 million series B round in January and a $53 million A round in 2018, has both a pipeline strategy and a partnering model.