On May 17, 2022 Purple Biotech Ltd. ("Purple Biotech", or the "Company") (NASDAQ/TASE: PPBT), a clinical-stage company focused on developing first-in-class, effective and durable therapies by harnessing the power of the tumor microenvironment to overcome tumor immune evasion and drug resistance, reported the initiation of the Phase 2 portion of its ongoing study of CM24, a first-in-class monoclonal antibody with the potential to treat multiple cancers (Press release, Purple Biotech, MAY 17, 2022, View Source [SID1234614807]). The Phase 2 is an open-label, multicenter study in subjects with metastatic pancreatic cancer (PDAC) to evaluate the safety and tolerability of CM24 in combination with the PD-1 inhibitor Opdivo (nivolumab) and chemotherapy. The primary study endpoint is to evaluate preliminary efficacy in 2nd line PDAC.

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The phase 2 portion of the study (NCT04731467) is being conducted as part of Purple Biotech’s clinical collaboration with Bristol Myers Squibb. The companies together made the determination to prioritize PDAC over non-small cell lung cancer (NSCLC) based on the positive interim data. Accordingly, this decision will allow an increase in the number of subjects that will be participating, and will accelerate this part of the study, and the updated timeline to complete this trial is within 2023.

"Pancreatic ductal adenocarcinoma, or pancreatic cancer, has one of the highest mortality rates of all cancers and affects tens of thousands of patients each year. While progress has been made in understanding and treating pancreatic cancer, more effective treatments are needed. It is exciting to have CM24 and Opdivo showing a confirmed partial response in a patient with pancreatic cancer with an MSS tumor, where other immune checkpoint inhibitors have failed to show response to date. In addition, the safety profile of CM24 in combination with nivolumab and the positive efficacy signals in advanced patients with very poor prognosis in the study are encouraging, and support the advancement of the clinical study in patients with pancreatic cancer," said Erkut Borazanci, MD, Deputy Director Oncology and Clinical Investigator at HonorHealth Research Institute, Scottsdale, AZ.

"We are thrilled at the progress being made to help move this treatment forward in the clinical trial process for the potential benefit of patients with pancreatic cancer," said Gil Efron, President and CFO of Purple Biotech. "We are thankful for our partners at BMS for collaborating with us on this study and their support. Importantly, the decision to prioritize the PDAC study will extend the cash runway of Purple Biotech through the end of 2024," added Efron.

This announcement follows the release of interim safety and efficacy results from the Phase 1b portion of the study of CM24 in combination with Opdvio shared at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in April. Information shared demonstrated a favorable safety profile. Six Grade 3 adverse events (AEs) that were unrelated to CM24 or nivolumab and no Grade 4 AEs or deaths were reported. Encouraging signals of efficacy in advanced patients with PDAC were reported (n=8, ORR=12%, DCR=37%), with one confirmed partial response in a patient with metastatic pancreatic cancer, as well as three patients with stable disease, including two patients with pancreatic cancer and one patient with papillary thyroid cancer. All patients but one received two prior lines of treatment for their metastatic disease.

OPDIVO is a trademark of Bristol-Myers Squibb Company.

On May 17, 2022 Zealand Pharma A/S (Nasdaq: ZEAL) (CVR-no. 20045078,) a biotechnology company focused on the discovery and development of innovative peptide-based medicines, reported it has entered into an asset purchase agreement with MannKind Corporation (Nasdaq: MNKD) to sell the V-Go insulin delivery device for $10 million and additional sales-based milestones. Certain inventory and employees are also included under the terms of the agreement (Press release, Zealand Pharmaceuticals, MAY 17, 2022, View Source [SID1234614910]).

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V-Go is a once-daily, wearable, insulin delivery device that helps provide blood sugar control for everyday lifestyles. Designed to be patient-friendly, V-Go is worn like a patch and eliminates the need for taking multiple daily shots.

"This transaction is an important step forward in executing on the strategic refocusing we outlined at the end of March, as we find partners for our commercial and late-stage assets and prioritize the research and development of innovative new peptide therapeutics," said Adam Steensberg, MD, Chief Executive Officer of Zealand Pharma. "We believe MannKind is the right partner to fully leverage the value of V-Go and ensure its continued availability for the patients and prescribers who need it."

"MannKind is committed to bringing innovative solutions to the diabetes patient community to potentially ease daily disease management," said Michael Castagna, PharmD, Chief Executive Officer of MannKind Corporation. "Acquiring V-Go will help us to address an unmet need while strategically leveraging our infrastructure in the diabetes space and positioning MannKind’s endocrine business for additional growth."

The acquisition of V-Go by MannKind is anticipated to close in May 2022, subject to the satisfaction of certain closing conditions.

Today’s news does not impact Zealand Pharma’s Financial Guidance for 2022 as reiterated in the company’s Interim Report for the First Quarter 2022 announced on May 12, 2022.

On May 17, 2022 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) and Abeona Therapeutics Inc. (Nasdaq: ABEO) reported an exclusive license agreement for AAV gene therapy ABO-102 (now UX111) for the treatment of Sanfilippo syndrome type A (MPS IIIA) (Press release, Abeona Therapeutics, MAY 17, 2022, View Source [SID1234614725]). Under the terms of the agreement, Ultragenyx will assume responsibility for the ABO-102 program and in return Abeona is eligible to receive tiered royalties of up to 10% on net sales and commercial milestone payments following regulatory approval.

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"Based on promising data from Abeona’s clinical program, regulatory feedback to date, and our experience developing treatments for other MPS diseases, we believe ABO-102 has the potential to be a transformative therapy for patients with MPS IIIA," said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. "Our team’s expertise in MPS and gene therapy clinical development makes this program a seamless integration and it has the potential to be our first gene therapy to market. The Sanfilippo community has been waiting too long for a first treatment and we believe we can help accelerate this program."

"Data from the ongoing Transpher A trial demonstrate ABO-102 holds significant potential to improve outcomes for patients with MPS IIIA who experience relentlessly progressing neurodevelopmental and physical decline that is life-threatening at a very young age," said Vish Seshadri, Ph.D., Chief Executive Officer of Abeona. "We believe that Ultragenyx, with deep expertise in rare, genetic, metabolic lysosomal storage disorders and a demonstrated commitment towards MPS diseases, is the ideal partner to eventually bring ABO-102 to patients."

Abeona has completed a successful Type B meeting with the U.S. Food and Drug Administration (FDA) regarding the pivotal Transpher A trial to support filing and approval for ABO-102 for the treatment of patients with MPS IIIA. Interim results from the Transpher A trial presented in an encore presentation at the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) today demonstrate that neurocognitive development was preserved in children treated before 2 years old or development quotient (DQ) > 60 (n=10) within normal range of a non-afflicted child after treatment with ABO-102 (3×1013 vg/kg). The interim results also showed continued or stabilized cognitive function and behavioral progress using standard developmental assessments. Some of these patients have reached 24-months post treatment and stabilization or increase in cortical gray matter, total cerebral, and amygdala volumes have been observed. Statistically significant reduction in liver volume was seen with ABO-102 treatment. Dose-dependent and statistically significant reductions in cerebrospinal fluid and plasma heparan sulfate, demonstrating replacement of enzyme activity consistent with levels required for disease correction in the central nervous system, have been sustained in treated patients for two years after treatment. ABO-102 has been well-tolerated with no treatment-related serious adverse events and no clinically meaningful adverse events reported.

"MPS IIIA is characterized by severe neurodegeneration with debilitating symptoms for which there is currently no treatment," said Kevin Flanigan, M.D., director of the Center for Gene Therapy at Nationwide Children’s Hospital in Columbus, Ohio, and Transpher A study principal investigator. "The promising results to date suggest a single intravenous dose of ABO-102 AAV-based gene therapy has the potential to help children with MPS IIIA sustain neurocognitive development when they are treated during early stages of their disease."

About ABO-102 / UX111

ABO-102 (now UX111), is a novel gene therapy in Phase 1/2 development for Sanfilippo syndrome type A (MPS IIIA), a rare lysosomal storage disease with no approved treatment that primarily affects the central nervous system (CNS). ABO-102 is dosed in a one-time intravenous infusion using a self-complementary AAV9 vector to deliver a functional copy of the SGSH gene to cells of the CNS and peripheral organs. The therapy is designed to address the underlying SGSH enzyme deficiency responsible for abnormal accumulation of glycosaminoglycans in the brain and throughout the body that results in progressive cell damage and neurodevelopmental and physical decline. The ABO-102 program has received Regenerative Medicine Advanced Therapy, Fast Track, Rare Pediatric Disease, and Orphan Drug designations in the U.S., and PRIME and Orphan medicinal product designations in the EU.

About the Transpher A Study

The Transpher A Study (ABT-001) is an ongoing, two-year, open-label, dose-escalation, Phase 1/2 global clinical trial assessing ABO-102 for the treatment of patients with Sanfilippo syndrome type A (MPS IIIA). The study is intended for patients from birth to 2 years of age, or patients older than 2 years with a cognitive developmental quotient of 60% or above. ABO-102 gene therapy is delivered using AAV9 technology via a single-dose intravenous infusion. The study primary endpoints are neurodevelopment and safety, with secondary endpoints including behavior evaluations, quality of life, enzyme activity in cerebrospinal fluid (CSF) and plasma, heparan sulfate levels in CSF, plasma and urine, and brain and liver volume.

Further details can be referenced here: View Source

About Sanfilippo syndrome type A (MPS IIIA)

Sanfilippo syndrome type A (MPS IIIA) is a rare, fatal lysosomal storage disease with no approved treatment that primarily affects the CNS and is characterized by rapid neurodevelopmental and physical decline, often by age three. MPS IIIA has a global incidence of one in 100,000 with a median life expectancy of 15 years.

Children with MPS IIIA present with progressive language and cognitive decline and behavioral abnormalities. Other symptoms include sleep problems and frequent ear infections. Additionally, distinctive facial features with thick eyebrows or a unibrow, full lips and excessive body hair for one’s age, and liver/spleen enlargement are also present in early childhood. MPS IIIA is caused by genetic mutations that lead to a deficiency in the SGSH enzyme responsible for breaking down glycosaminoglycans, which accumulate in cells throughout the body resulting in rapid health decline associated with the disorder.

On May 17, 2022 Geron Corporation (Nasdaq: GERN), a late-stage biopharmaceutical company focused on the development and commercialization of treatments for hematologic malignancies, reported that John A. Scarlett, M.D., Geron’s Chairman and Chief Executive Officer, plans to present a company overview at the H.C. Wainwright Global Investment Conference (Press release, Geron, MAY 17, 2022, View Source [SID1234614742]). The presentation will be available as an on-demand session beginning May 24, 2022 at 7 a.m. ET.

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A webcast of the presentation will be available through the Investor Relations section of Geron’s website under Events. Following the presentation, the webcast will be archived and available for replay for a period of 30 days.

On May 17, 2022 Silverback Therapeutics, Inc. (Nasdaq: SBTX) ("Silverback"), a biopharmaceutical company leveraging its proprietary ImmunoTAC technology platform to develop systemically delivered, tissue targeted therapeutics for the treatment of chronic viral infections, cancer, and other serious diseases, reported that Silverback management will participate in the H.C. Wainwright 24th Global Investment Conference from May 23-25, 2022 (Press release, Silverback Therapeutics, MAY 17, 2022, View Source [SID1234614759]).

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Laura Shawver, Ph.D., Silverback’s Chief Executive Officer, will provide a corporate overview on Tuesday, May 24, 2022 at 7:00 AM ET / 4:00 AM PT. The webcast of the presentation will be available on Silverback’s Investor Relations website for 30 days following the event. Members of the Silverback management team will also host investor meetings during the conference.