Ankyra Therapeutics Announces $45 Million Series B Financing and Appoints Howard L. Kaufman M.D. as Chief Medical Officer

On November 12, 2021 Ankyra Therapeutics, a company developing cancer therapies based on tumor localized immune potentiating agents that boost anti-tumor immune responses, reported the closing of a $45 million Series B financing (Press release, Ankyra Therapeutics, NOV 12, 2021, View Source [SID1234595474]). Proceeds from the financing will be used to progress Ankyra’s lead molecule, ANK-101 through Investigational New Drug (IND) enabling studies and into Phase 1 clinical trials, as well as advance additional cytokine programs .

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Several new investors including Borealis Ventures, Fidelity Management & Research Company LLC, GV, Sands Capital Ventures, and Spring Mountain Capital joined existing investors Polaris Partners and Mithril Capital. In connection with the closing of the financing, Phil Ferneau of Borealis Ventures will join the Ankyra Therapeutics Board of Directors.

Ankyra Therapeutics has developed a highly differentiated technology platform that significantly expands the therapeutic window of immune-modulating oncology drugs by forming an extended drug depot following intratumoral administration. The company is rapidly progressing its lead molecule into clinical studies.

"We are fortunate to have the support of investors who share our vision of developing tumor-localized cytokines and other immune therapies to treat cancer with enhanced efficacy and safety," said Tillman Gerngross, Co-founder, and Executive Chairman of Ankyra Therapeutics. "This is an important moment for the company as we work to bring our first development program through IND and into Phase 1 clinical trials, and we are excited to bring Howard Kaufman, and his proven track record of successful clinical development, to the Ankyra team."

Dr. Kaufman has been a leading authority on tumor immunotherapy and oncolytic viruses for the treatment of melanoma. He led the first successful phase III trial of an intratumoral agent resulting in FDA approval for the treatment of melanoma. He served as president of the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) from 2014-2016. Prior to joining Ankyra, Dr. Kaufman was Head of Research and Development at Immuneering Corporation. Dr. Kaufman also served as chief medical officer at Replimune Group, Inc., where he oversaw the strategic development of the company’s pipeline.

"Systemic treatment with cytokines and other immune agonists has been limited by broad immune activation and systemic toxicity. Intratumoral administration can improve the therapeutic window of immune activating drugs, but the approach has been limited by rapid clearance from the tumor microenvironment. There is a significant need for approaches that improve the therapeutic window and reduce the frequency of dosing. Ankyra’s approach is a true platform that can extend drug bioavailabity and enhance anti-tumor immune responses, while avoiding systemic adverse events. We look forward to extending our preclinical observations into cancer patients in our upcoming Phase 1 clinical trial," stated Howard Kaufman, Chief Medical Officer of Ankyra.

Compugen Presents Initial Translational Data Supporting the Differentiation of PVRIG Compared to TIGIT and PD-1 as a Novel Checkpoint on the DNAM Axis at SITC 2021

On November 12, 2021 Compugen Ltd. (Nasdaq: CGEN), a clinical-stage cancer immunotherapy company and a leader in predictive target discovery, reported the presentation of new translational preliminary data detailing the differentiated profile of PVRIG compared to TIGIT and PD-1 as a novel checkpoint in the DNAM axis, supporting its potential role as a dominant checkpoint involved in stem-like memory T cells and dendritic cell (DCs) interaction at the 36th Annual Meeting of the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper), being held on November 10-14, 2021 (Press release, Compugen, NOV 12, 2021, View Source [SID1234595490]).

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"We believe evidence is growing to consistently demonstrate that PVRIG is a novel and differentiated checkpoint with a potential unique role in cancer immunotherapy" said Eran Ophir, Ph.D., Vice President of Research and Drug Discovery at Compugen. "For cancer immunotherapy to work, T cells are needed at the tumor site and recent studies suggest that early-memory (stem-like) T cells and DCs play an important role in this process. Here we show for the first-time preliminary data demonstrating greater induction of activated DC markers in the serum of two patients responding to our potentially first in class anti-PVRIG antibody, COM701, in combination with nivolumab compared to non-responders, potentially because of DC- T cell interaction. This preliminary data is in line with our recent scientific finding showing that PVRL2, the ligand of PVRIG, is abundantly expressed across DCs types, while PVRIG, measured by both gene and protein expression, is uniquely and dominantly expressed on early memory cells in contrast to TIGIT and PD-1."

Anat Cohen-Dayag, Ph.D., President and CEO of Compugen, added, "These new preliminary data further support our earlier findings that PVRIG plays a distinct role within the DNAM axis which we believe is important for triggering robust immune responses in the tumor microenvironment. PVRIG blockade may lead to key mechanistic differences as compared to other DNAM axis members, namely TIGIT and PD-1, with the potential to enhance T cell proliferation and tumor infiltration to address both inflamed and less inflamed tumor types where current checkpoint inhibitors have not shown success. With our potentially first-in-class anti- PVRIG antibody, COM701, we are uniquely positioned to target the DNAM axis in combination with TIGIT and PD-1/L1 inhibitors and look forward to continued translation of these scientific learnings across our ongoing clinical programs."

Key findings from the poster presentation titled, "Novel DNAM-1 axis member, PVRIG, is potentially a dominant checkpoint involved in stem-like memory T cells – dendritic cell interaction," presented by Zoya Alteber PhD, Associate Director, Research and Drug Discovery at Compugen include:

PVRIG is co-expressed with PD-1 and TIGIT on stem-like and exhausted T cells as measured by flow cytometry across multiple tumor types
PVRIG has a unique dominant expression on early memory cells, clustering with markers of early memory T cells. In contrast, TIGIT is strongly associated with PD-1, CTLA-4, and other markers of exhausted T cells
PVRIG protein expression is significantly higher on early memory, CD28+ T cells in contrast to TIGIT and PD-1 which have comparable expression on CD28+ and CD28- cells
PVRL2, the ligand of PVRIG, is dominantly expressed on DC compared to PD-L1 and PVR, the ligand of TIGIT. This dominant expression is observed across DC1, DC2 and activated DC subtypes
Immunohistochemistry across multiple tumor types identified PVRL2 expression in tertiary lymphoid structures, the site of T cell priming, further supporting the PVRIG-PVRL2 interaction as a potentially dominant interaction for T cell activation in the tumor microenvironment
In 2 patients who responded clinically to treatment with the anti-PVRIG antibody COM701 in combination with nivolumab, early data show increased induction of activated DC markers, suggestive of an enhanced T cell – DC interaction, with the potential to enhance T cell proliferation and tumor infiltration.
The poster is available to conference attendees for the duration of the SITC (Free SITC Whitepaper) conference and will be archived on the Publications section of Compugen’s website.

xCures presents the results of their oncology platform at the 2021 SNO Annual Meeting

On November 12, 2021 xCures reported their poster presentation at the 26th Annual Meeting of the Society for Neuro-Oncology, held from the 18th to the 21st of November (Press release, xCures, NOV 12, 2021, View Source [SID1234595513]). The Society for Neuro-Oncology (SNO) is a multidisciplinary society of healthcare professionals dedicated to promoting advances in neuro-oncology through research and education. Their annual meeting features research and educational sessions on brain tumors, including the latest on diagnosis and treatments.

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xCures poster presentation, entitled XCELSIOR: A real-time, real-world learning platform for patients with advanced cancer, will show clinical outcomes of real-world datasets from over 400 CNS cancer patients and approximately 250 glioblastoma patients. XCELSIOR is a direct-to-patient evidence-based platform leveraging a nationwide observational research protocol. The platform allows for continuous learning towards informing treatment decisions by aggregating, normalizing, and analyzing N-of-1 clinical outcomes from anywhere in the country.

At the conference, xCures will discuss their real-time learning infrastructure and present results of clinical case studies for pharma and non-profit groups, including more than 100 reported virtual tumor boards. Outcomes shown will be from real-world evidence generated from hundreds of patients with CNS cancers that xCures have helped in partnership with Cancer Commons and the Musella Foundation.

"I look forward to connecting with colleagues and presenting our first analysis of real-world clinical outcomes of CNS cancer patients from our observational registry, including a preliminary analysis of patients that received immune checkpoint inhibitors," stated Tim Stuhlmiller, VP of Scientific and Medical Affairs at xCures. "It is inspiring to see our approach to gather and analyze real-world data in real-time via patient participation in a nationwide observational research protocol yield evidence-based insights."

Al Musella, President of the Musella Foundation said, "With the launch of their provider portal xDECIDE, the xCures platform offers a major opportunity for oncologists all over the country to collaborate on observational research without the burden of data entry. The CNS dataset presented at the conference provides a foundation for ongoing clinical research to identify the most promising new combinations of therapies in glioblastoma."

"Cancer Commons’ close partnership with xCures, the Musella Foundation, and the expert physician advisers that serve on our virtual tumor boards has helped inform brain cancer patients across the U.S. about treatment options specific to their case. The data presented here demonstrates our first steps towards building a ‘learning health system’ that we hope will change the care for brain cancer patients, tightly integrate clinical care and research, and help us learn from each individual’s experience." said Matt Warner, Scientist at Cancer Commons.

For more information, visit the poster session on Friday, November 19th from 7:30-9:30 pm EST in Exhibit Hall D, or attend the live presentation of the xCures platform and preliminary RWD on CNS cancers on Saturday, November 20th at 1 pm EST in Room 309 in the Hynes convention center.

iOnctura Presents Compelling Preclinical Data on its Clinical Stage Autotaxin Inhibitor IOA-289 at SITC

On November 12, 2021 iOnctura SA, a clinical stage oncology company targeting core resistance and relapse mechanisms at the tumor-stroma-immune interface, reported its compelling preclinical evidence on its next-generation autotaxin inhibitor, IOA-289, at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) (#SITC) Annual Meeting on November 10–14, 2021 (Press release, iOnctura, NOV 12, 2021, View Source [SID1234640239]).

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Autotaxin is a secreted glycoprotein that hydrolyzes LPC to LPA. LPA has a direct effect on tumor cell growth and survival. It also modulates the tumor microenvironment at the level of immune and stromal cells, enabling tumors to evade host immunity and impairing the response to therapy. The expression of both autotaxin and LPA is elevated in most solid tumors, with corresponding increases measurable in patients’ plasma. iOnctura is developing IOA-289 as a novel therapy for oncology indications where the response to chemotherapy and / or immunotherapy is sub-optimal due to the presence of an immunosuppressive stromal microenvironment.

The data presented at SITC (Free SITC Whitepaper) showed that IOA-289 is able to dose-dependently reduce LPA levels, potently modulate fibrotic processes and restore T cell migration in preclinical models. Furthermore, the data also demonstrated IOA-289 exhibited monotherapy activity to inhibit primary tumor growth and metastasis in orthotopic, immunocompetent tumor models. Finally, autotaxin expression was found to be elevated in the plasma of pancreatic cancer patients and its presence was correlated with the tumor biomarker CA19-9, a blood marker used to follow progression in patients with pancreatic cancer.

iOnctura has recently completed recruitment into the healthy volunteer study of IOA-289, investigating the safety and pharmacokinetic profile of IOA-289 in humans. Detailed results will be presented at an upcoming medical oncology conference. iOnctura plans to advance IOA-289 into a Phase 1 clinical study in pancreatic cancer, AION-01, in the first half of 2022.

The poster presentation at SITC (Free SITC Whitepaper) entitled "A novel autotaxin inhibitor, IOA-289, modulates tumor, immune and stromal cell function and has monotherapy activity in fibrotic cancer models," is available at the SITC (Free SITC Whitepaper) platform and iOnctura’s website.

Contacts

iOnctura
Catherine Pickering
Chief Executive Officer
T : +41 79 952 72 52
E: [email protected]

Press Relations
Jeremy Nieckowski
LifeSci Advisors
T: +41 79 699 97 27
E: [email protected]
iOnctura SA is clinical stage oncology company targeting core resistance and relapse mechanisms at the tumor-stroma-immune interface. iOnctura’s best-in-class drug development programs combine immune-mediated and direct anti-tumor activity to deliver molecules with superior clinical efficacy and safety in oncology. Its lead program, IOA-244 is the only semi-allosteric PI3Kδ specific, orally dosed, small molecule inhibitor that is being developed in solid and hematologic malignancies to address tumor and stroma induced immune suppression. IOA-244 is currently in a Phase 1 study which will support transition to subsequent registration studies. iOnctura’s second program, IOA-289, is an oral small molecule that inhibits the cross-talk between the tumor and its stroma and is in a Phase 1 study. iOnctura is backed by blue chip investors including M Ventures, Inkef Capital, VI Partners, Schroders Capital, and 3B Future Health Fund. For more information, please visit www.ionctura.com

IOA-289, originally licensed from Cancer Research UK, is iOnctura’s second clinical compound, a next generation oral small molecule autotaxin inhibitor. iOnctura have completed recruitment in a single ascending dose study of IOA-289 in healthy volunteers and plans to advance IOA-289 into a phase 1 clinical study in pancreatic cancer, AION-01, in the first half of 2022. iOnctura has undertaken extensive validation of the autotaxin inhibition mechanism in multiple preclinical solid tumor models.

Zealand Pharma Presents Financial Results for the First Nine Months of 2021; Provides Updates on Commercial Launch and Timing for Congenital Hyperinsulinism and Short Bowel Syndrome Phase Three Clinical Studies

On November 12, 2021 Zealand Pharma reported that Financial Results for the First Nine Months of 2021; Provides Updates on Commercial Launch and Timing for Congenital Hyperinsulinism and Short Bowel Syndrome Phase Three Clinical Studies (Press release, Zealand Pharmaceuticals, NOV 12, 2021, View Source [SID1234595348])

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Copenhagen, DK and Boston, MA, U.S. November 11, 2021 – Zealand Pharma A/S (Nasdaq: ZEAL) (CVR-no. 20045078) a biotechnology company focused on the discovery, development, and commercialization of innovative peptide-based medicines, reported financial results for the first nine months of 2021.

Financial results for the first nine months of 2021

Revenue: DKK 238.6 million / USD 37.1 million (DKK 290.0 million / USD 45.6 million in the first nine months of 2020).
Net operating expenses: DKK -906.2 million / USD -141.1 million (DKK -714.5 million / USD -112.3 million in the first nine months of 2020).
Net operating result: DKK -762.6 million / USD -118.8 million (DKK -449.1 million / USD -70.6 million in the first nine months of 2020).
Cash, cash equivalents, and marketable securities: DKK 1,049.0 million / USD 163.3 million as of September 30, 2021 (September 30, 2020: DKK 1,528.6 million / USD 240.4 million).
Business highlights for the first nine months of 2021

Announced collaboration agreement with DEKA Research & Development Corp. to advance development of infusion pump to be used with dasiglucagon for the potential treatment of congenital hyperinsulinism (CHI).Zealand Pharma A/S entered into a definitive collaboration agreement with DEKA Research & Development Corp., to develop a continuous infusion pump to be used in combination with dasiglucagon, an investigational agent currently in phase 3 trials in patients with congenital hyperinsulinism. The agreement covers the technical development of the pump system as well as associated commercialization activities.
Announced presentation of preclinical data on Amylin Analogue ZP8396 and clinical data on GLP1-Glucagon Dual-Agonist BI 456906 at The Obesity Society Annual Meeting. Key findings from the preclinical study of amylin analogue ZP8396 were: monotherapy showed comparable weight loss potential, with the combination therapy being superior for the treatment of obesity and obesity related comorbidities; and administration of amylin analogue ZP8396 in combination with semaglutide, a GLP-1 analogue, resulted in increased food intake inhibition and body weight loss (~20% vs initial body weight) as compared to single treatment alone in the diet induced obese (DIO) in vivo models.
Key findings of the Phase 1 trial with BI 456906, a subcutaneous dual GLP-1R/GCGR agonist were in the Phase I dose escalation study; BI 456906 was generally well tolerated and resulted in clinically relevant bodyweight reductions of up to 6.6% after 6 weeks and 13.7% after 16 weeks.
Announced first patient dosed in EASE-SBS 4 phase 3b trial assessing Glepaglutide in patients with Short Bowel Syndrome. The EASE-SBS 4 Phase 3b trial is part of Zealand’s EASE-SBS Phase 3 program for glepaglutide. (ClinicalTrials.gov identifier (NCT number): NCT04991311) and will evaluate long-term effects on intestinal absorption of fluids and energy of once weekly glepaglutide injections. EASE-SBS 4 is an open-label single-center Phase 3b trial investigating the long-term effect on intestinal absorption, nutritional status and long-term safety of administration of glepaglutide in patients with short bowel syndrome (SBS). Ten patients will receive once weekly 10 mg subcutaneous injections of glepaglutide over 26 weeks. The EASE-SBS 1 pivotal Phase 3 trial remains on track for trial results to be obtained in 2022.

Emmanuel Dulac, President and Chief Executive Officer at Zealand Pharma, comments:

"Zealand Pharma has continued to undergo a transformational first nine months in 2021 and developed into an integrated biopharmaceutical company by leveraging our innovative peptide platform to address unmet needs in type 1 diabetes management and rare diseases. We are progressing with the early stages of our commercial launch of ZEGALOGUE (dasiglugacon) injection with Zegalogue currently available to 65% of all commercial lives. As our commercial team ramps up the ZEGALOGUE commercial launch, we also look forward to building upon the momentum to advance our other pipeline programs. Recently, we have been encouraged by preclinical data from ZP8396, our Amylin Analogue, and clinical data from GLP-1, our Glucagon Dual-Agonist BI 456906, both sets presented at the recent The Obesity Society Annual Meeting. Additionally, we entered into a collaboration agreement with DEKA Research & Development Corp. and are working to advance the development of their infusion pump to be used with dasiglucagon as a potential treatment option of CHI. Finally, while the Zegalogue net revenue projections have not materialized as we had hoped and we have lowered our revenue guidance as a result, we remain confident in the work that we have done to set the stage for Zegalogue to have commercial success in 2022 and beyond."

Financial guidance for 2021

Net product revenue from the sales of commercial products is expected to be DKK 190 million +/- 10%. This is a decrease of DKK 30 million from the guidance issued on March 11, 2021. The reduction in net revenue from the previous guidance is driven by lower-than-expected sales of Zegalogue for 2021.

In 2021, Zealand Pharma expects revenue from existing license agreements. However, since such revenue is uncertain in terms of size and timing, Zealand Pharma does not intend to provide guidance on such revenue.

Net operating expenses in 2021 are expected to be DKK 1,250 million +/-10% and remains unchanged from the financial guidance issued on March 11, 2021.

Update regarding COVID-19

Zealand Pharma continues to monitor the COVID-19 pandemic and take precautions to keep our employees, patients, business and clinical partners safe. This is an ongoing exercise in monitoring the effects of the pandemic on all of our key stakeholders and responding appropriately. We maintain compliance with guidance from applicable government and health authorities as appropriate. We have adapted the way we work to support our community’s efforts to reduce the transmission of COVID-19 and protect our employees, while continuing to provide patient care and maintain business continuity.

Zealand Pharma has taken measures to secure its discovery activities, which remain ongoing, while work in laboratories and offices has been organized to reduce the risk of COVID-19 transmission. The impact of COVID-19 on our research activities has thus far been minimal. Employees who can work from home have been doing so, while those needing to work in laboratory facilities are divided into shifts to reduce the number of people gathered at one time. Business travel has been minimized and online and video conference technology is used to meet virtually rather than in person. We have continued our clinical trials while working with authorities, investigators, trial sites and contract research organizations to minimize site visits and ensure optimal trial follow-up. In late April, Zealand Pharma in Denmark commenced a gradual return to office program, which currently allows up to 60% occupancy, consistent with local health authority guidelines and practice including frequent testing, sanitizing, and other protective measures.

COVID-19 restrictions have not affected our phase 3-program for dasiglucagon in congenital hyperinsulinism (CHI) and we expect topline data from the second Phase 3 trial in 2021. The pandemic impacted the speed of patient recruitment for our Phase 3 trial with glepaglutide for the treatment of short bowel syndrome, and results are expected in 2022. We currently do not anticipate changes to the timelines for the bi-hormonal artificial pancreas pump Phase 3 program.

Our research and in particular our development programs may be impacted if the pandemic continues to put increased pressure on hospital systems, slow recruitment of patients into the trials or cause lockdowns that affect our clinical trial sites if key external medical resources are diverted elsewhere.

Direct engagement with health care providers and patients has been reduced and transformed by leveraging virtual meetings, training, and support. Our commercial team is focused on supporting the businesses for the V-Go wearable insulin delivery device and Zegalogue while ensuring a continued high level of service and support for existing patients.

Commercial Update

Zegalogue (dasiglucagon) injection

Zegalogue (dasiglucagon) injection was approved by the U.S. FDA on March 22, 2021 for the treatment of severe hypoglycemia in people with diabetes aged 6 and over. Zegalogue is available in both an auto injector and a prefilled syringe. The approval was based on efficacy results from three pivotal trials in adults and children with type 1 diabetes, whereby the primary endpoint of time to plasma glucose recovery, was successfully achieved with a median time to blood glucose recovery of 10 minutes following Zegalogue administration. In these Phase 3 studies, the most common adverse events reported (≥2%) were nausea, vomiting, headache, diarrhea, and injection site pain in adults; and nausea, vomiting, headache and injection site pain in pediatric patients.

Zegalogue launched in the U.S. in late June 2021. The Company’s primary goal during launch is to ensure all eligible patients have access to Zegalogue and feel supported in their treatment plan. Since approval, Zealand Pharma has made substantial progress working with Pharmacy Benefit Managers (PBMs), Managed Care Organizations, and state Medicaid agencies to add Zegalogue to their respective formularies. As a result of this work, Zegalogue now has unrestricted coverage in approximately 65% of commercial lives which accounts for more than 120 million lives and approximately 55% of Medicaid lives, which accounts for 40 million Medicaid lives. Beginning in 2022, we expect Zegalogue will have unrestricted coverage in approximately 70% of commercial and Medicaid lives, setting the stage for a sustained acceleration of growth.

Zegalogue net revenue for the period of July 1 – September 30, 2021 was DKK 3.1 million / USD $0.5 million. Year-to-Date net revenue is DKK 4.3 million / USD $0.7 million.

For the year, net product revenue of Zegalogue is anticipated to be below our initial revenue targets. The reduction in revenue is a result of lower-than-expected uptake of prescriptions due to the increase in timelines for approved drug coverage agreements with payers to translate into approved prescriptions in pharmacies.

V-Go wearable insulin delivery device

The V-Go series of Wearable Insulin Delivery Devices are indicated for continuous subcutaneous infusion of either 20 Units of insulin (0.83 U/hr), 30 Units of insulin (1.25 U/hr) or 40 Units of insulin (1.67 U/hr) in one 24-hour time period and on-demand bolus dosing in 2-Unit increments (up to 36 Units per one 24-hour time period) in adults requiring insulin.

V-Go net revenue for the period of July 1 – September 30, 2021 was DKK 49.1 million / USD $7.8 million. Year-to-Date net revenue is DKK 139.0 million / USD $22.4 million.

Pipeline Update

Type 1 Diabetes Management

Dasiglucagon for bihormonal Artificial Pancreas systems

Zealand Pharma is developing a pre-filled dasiglucagon cartridge intended for use in Bihormonal Artificial Pancreas systems, which holds potential to improve the management of type 1 diabetes (T1D).

Zealand is collaborating with Beta Bionics, developer of the bihormonal iLet bionic pancreas system, a pocket-sized, dual-chamber (insulin and glucagon), autonomous, glycemic control system. The iLet bionic pancreas is an investigational device, limited by federal (or United States) law to investigational use only. The iLet bionic pancreas intends to mimic a biological pancreas by calculating and dosing insulin and/or glucagon (dasiglucagon) as needed, based on input data from a continuous glucose monitor (CGM) worn by a person with diabetes. Top-line results from a Phase 2, pre-pivotal trial in people with type 1 diabetes showed that the bihormonal iLet bionic pancreas using insulin and dasiglucagon provided improved glycemic control relative to the iLet bionic pancreas delivering insulin alone. During the bihormonal and insulin-only periods, 90% (9 of 10) and 50% (5 of 10) of participants, respectively, had a mean CGM glucose level of < 154 mg/dL.1,2 Importantly these glucose levels were achieved while the median time with CGM glucose levels < 54 mg/dL was only 0.2% during the bihormonal period and 0.6% during the insulin-only period.

Zealand’s partner, Beta Bionics, and the study sponsor, the Jaeb Center for Health Research, are on track to begin screening in the fourth quarter 2021 into the Phase 3, Bihormonal iLet Bionic Pancreas Pivotal Program, utilizing insulin and dasiglucagon. The program includes three sub-trials, which are anticipated to provide the clinical data necessary to support the market application for the bihormonal iLet bionic pancreas and the new-drug application (NDA) for the use of dasiglucagon in bihormonal Artificial Pancreas systems. The first of these three sub-trials will be a three-month single-arm, bihormonal-only safety and test-run trial that will enroll two participants at each of the approximately 30 clinical sites. After 20 pediatric participants and 20 adult participants have been successfully treated for a minimum of 3 weeks in this trial, the two randomized controlled trials (RCTs) will begin – one enrolling ~ 350 pediatric participants (6–17 years of age) and the other enrolling 350 adult participants (≥ 18 years of age) with T1D. The primary outcome measure in the RCTs is superiority in HbA1c of the bihormonal iLet bionic pancreas using dasiglucagon relative to the insulin-only iLet system after 26 weeks of therapy on the two interventions. The bihomonal iLet bionic pancreas performance will also be compared to intensified usual care using CGM therapy in a third arm in both the pediatric and adult RCTs.

Dasiglucagon mini-dose pen

Zealand is developing a dasiglucagon mini-dose pen for potential treatment of exercise-induced hypoglycemia in people living with type 1 Diabetes and for people who suffer from meal-induced hypoglycemia following gastric bypass surgery.

Clinical studies conducted in hospital settings have shown the potential for using low doses of dasiglucagon to correct moderate hypoglycemia. Top-line results from a Phase 2a dose-finding trial in people with type 1 diabetes were presented at the American Diabetes Association congress in June 2021, and top-line results of a post bariatric hypoglycemia Phase 2a trial were reported in 2020.

Out-patient Phase 2 trials in exercise-induced hypoglycemia in people living with type 1 diabetes and for people that suffer from meal-induced hypoglycemia following gastric bypass surgery were initiated in the second quarter this year (ClinicalTrials.gov Identifier: NCT04764968 and NCT04836273). The Phase 2 trial in people with T1D specifically evaluates the effectiveness of pen-administered low-dose dasiglucagon for prevention and treatment of hypoglycemia in people with type 1 diabetes.

Rare Diseases

Dasiglucagon for congenital hyperinsulinism (CHI)

The potential for chronic dasiglucagon infusion delivered via a pump to prevent hypoglycemia in children with CHI is being evaluated in a Phase 3 program. The aim is to reduce or eliminate the need for intensive hospital treatment, reduce the frequency of dangerous low blood glucose and need for constant feeding, and to potentially delay or eliminate the need for pancreatectomy. The FDA and the European Commission both granted orphan drug designation to dasiglucagon for the treatment of CHI.

We announced data from the first Phase 3 trial in the program, trial 17109, in December 2020. This trial evaluated children aged 3 months to 12 years old with more than three hypoglycemic events per week despite previous near-total pancreatectomy and/or maximum medical therapy. Dasiglucagon on top of standard of care (SOC) did not significantly reduce the rate of hypoglycemia compared to SOC alone when assessed by the primary endpoint, intermittent self-measured plasma glucose. However, hypoglycemia was reduced by 40–50% with dasiglucagon as compared to SOC alone when assessed by blinded continuous glucose monitoring. Dasiglucagon treatment was assessed to be well tolerated in the study and 31 out of 32 patients continued into the long-term extension study.

We are conducting additional analyses and engaging with regulatory authorities to discuss the results of 17109 while awaiting the outcome of a second Phase 3 trial, 17103, in neonates up to 12 months old with CHI. We expect to complete enrolment into the 17103 trial by the end of 2021 and have results in the first half of 2022.

Glepaglutide for short bowel syndrome (SBS)

Glepaglutide is a long-acting GLP-2 analog, being developed in an auto-injector with potential for convenient weekly administration for reducing or eliminating the need for parenteral support in people living with SBS. EASE-SBS 1 is the pivotal Phase 3 trial with enrolment of up to 129 patients with SBS that seeks to establish the efficacy and safety of once- and twice-weekly administration of glepaglutide. Patients will be treated for six months in EASE-SBS 1, whereafter they are offered four years continuous treatment with glepaglutide in the extension trials, EASE-SBS 2 and 3. A Phase 3b trial, EASE-SBS 4, was initiated in Q3 2021 and will assess long-term effects of glepaglutide on intestinal fluid and energy uptake.

The primary endpoint in EASE-SBS 1 is the absolute reduction in parenteral support achieved by the end of the trial. Based on a dialogue with FDA and EMA we have decided to introduce an interim analysis that could allow for an early stopping of the study for efficacy or futility before the full 129 patients have been enrolled. If the interim readout meets the criteria for early stopping, we plan to pursue an NDA submission as a next development step based on these clinical data. We expect to have all subjects needed for the interim analysis enrolled by the end of 2021 with results of the interim analysis being available in Q3 2022. The U.S. FDA granted orphan drug designation to glepaglutide for the treatment of SBS.

Dapiglutide

Dapiglutide (pINN) is a long-acting GLP-1R/GLP-2R dual agonist.

The Phase 1a single-ascending dose, safety and tolerability trial investigating dapaglutide in healthy volunteers was completed in Q3 2020 and dapiglutide was found to have an acceptable safety and tolerability profile. Results showed a plasma half-life allowing for once weekly dosing.

Based on the results of the Phase 1a trial, Zealand initiated a Phase 1b (multiple ascending dose) safety and tolerability trial and all subjects have received the last dapiglutide dose for the safety and tolerability trial with key results expected in the fourth quarter of 2021 (ClinicalTrials.gov Identifier: NCT04612517).

Obesity

Amylin

ZP8396 is a potent long-acting amylin analogue designed to improve solubility and allow for co-formulation with other peptides, including GLP1 analogues. Amylin analogues hold potential as both mono and combination therapies for obesity. We have initiated a Phase 1 clinical trial with ZP8396 for potential treatment of obesity. The Phase 1, First-in-Human, randomized, single ascending dose trial will assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of ZP8396 administered to healthy subjects. Preclinical data on ZP8396 was presented at The Obesity Society Annual Meeting, which showed anti-obesity effects of ZP8396 in in vivo models, with up to 20% weight loss when combined with GLP1 analogue semaglutide. ZP8396 is also being developed as a potential treatment for type 2 diabetes.

BI 456906: Long-acting GLP-1/GLU dual agonist for obesity and/or diabetes (with Boehringer Ingelheim)

The GLP-1/glucagon dual agonist activates two key gut hormone receptors simultaneously and may offer better blood sugar and weight-loss control than current single-hormone receptor agonist treatments. The lead molecule BI 456906 is targeting treatment of obesity, diabetes, and non-alcoholic steatohepatitis (NASH). At Obesity Week in November this year Boehringer Ingelheim presented data from the Phase 1b trial, demonstrating up to 13.7% weight loss and no unexpected safety findings following 16 weeks of dosing.

Three parallel Phase 2 trials are ongoing. All subjects have been randomized in the first phase 2 trial which evaluates the dose-relationship of BI 456906 on HbA1c from baseline to 16 weeks relative to placebo in 410 people with diabetes (ClinicalTrials.gov Identifier: NCT04153929). Secondary objectives are to assess the effect on change in body weight and an open-label comparator (semaglutide) will allow for comparison of the effects against a pure GLP-1R agonist. The second Phase 2 randomized double-blind placebo-controlled dose-finding trial will evaluate BI 456906 in people with obesity or who are overweight with a BMI 27 kg/m2 or higher without diabetes (ClinicalTrials.gov Identifier: NCT04667377). Participants will receive a subcutaneous injection of either BI 456906 or placebo once a week for the duration of the trial. The primary endpoint of this trial is the percentage change in body weight at week 46 compared to placebo. The third Phase 2 randomized double-blind placebo-controlled dose-finding trial will evaluate BI 456906 in people with NASH and liver fibrosis (F2/F3) with and without diabetes (ClinicalTrials.gov Identifier: NCT04771273). The primary endpoint of this trial is the histological improvement of steatohepatitis without worsening of fibrosis after 48 weeks of treatment. Participants will receive a weekly subcutaneous injection of either different doses of BI 456906 or placebo for the duration of the trial. The program has received Fast Track designation from the U.S. FDA.

Boehringer Ingelheim is funding all research, development and commercialization activities related to the treatment. Zealand Pharma is eligible to receive up to EUR 345 million in outstanding milestone payments, and high-single to low-double digit royalties on global sales.

Inflammation

Zealand Pharma is pursuing multiple pre-clinical programs in inflammatory diseases which will be detailed more as they progress through development.

Complement inhibitors (with Alexion, AstraZeneca Rare Disease)

Zealand Pharma and Alexion Pharmaceuticals announced in March 2019 that they will collaborate on the discovery and development of novel peptide therapies for complement-mediated diseases. Under the terms of the agreement, Alexion and Zealand Pharma entered into an exclusive collaboration for the discovery and development of subcutaneously delivered peptide therapies directed to up to four complement pathway targets. The lead program is a long-acting inhibitor of Complement C3 which has the potential to treat a broad range of complement mediated diseases. Zealand Pharma will lead the joint discovery and research efforts through the preclinical stage, and Alexion will lead development efforts beginning with IND filing and Phase 1 trials. We are looking to initiate a Phase 1 trial of the C3 inhibitor in 2022.

For the lead target, Zealand Pharma is eligible to receive up to USD 610 million in development and sales milestone payments, plus royalties on global sales in the high single to low double digits. In addition, Alexion has the option to select up to three additional targets with Zealand Pharma eligible for USD 15 million upfront per target plus development/regulatory milestones for each target selected similar to the lead target with slightly reduced commercial milestones and royalties.

Additional Updates

On December 18, 2020 Amyndas Pharmaceuticals S.A. and Amyndas Pharmaceuticals LLC filed a complaint in the U.S. District Court for the District of Massachusetts, which named Alexion Pharmaceuticals, Inc., Zealand Pharma A/S and Zealand Pharma U.S., Inc. as defendants. The complaint alleges claims against Zealand Pharma A/S (and its U.S. subsidiary) and its collaboration partner Alexion Pharmaceuticals, Inc. ("Alexion") related to Zealand Pharma A/S’s collaboration with Alexion on C3 peptide-based assets. The complaint alleges federal and state law claims, including claims for breach of confidentiality agreements, trade secret misappropriation and unfair competition. The complaint seeks an unspecified quantum of damages plus interest and injunctive relief. On June 8, 2021 the District Court dismissed the proceedings against Zealand Pharma U.S., Inc. for failure to state a claim, and dismissed the claims against Zealand Pharma A/S on the ground that the matter should be heard in the courts of Denmark. On July 6, 2021 Amyndas moved the District Court to reconsider its dismissal of the claims against Zealand Pharma A/S (and its U.S. subsidiary) and this was also dismissed. On 27 September 2021 Amyndas filed an appeal of this decision to the First Circuit Court of Appeals. It is also seeking an order from the District Court pursuant to Rule 54(b) to render the district court decision as final.

Conference call today at 4 pm CEST / 10 am EDT

Zealand Pharma’s management will host a conference call today at 4 pm CEST to present results through the first nine months of 2021. Participating in the call will be Chief Executive Officer Emmanuel Dulac, Chief Financial Officer Matt Dallas, and Chief Medical and Development Officer Adam Steensberg. The presentation will be followed by a Q&A session with the presenters as well as the President of Zealand Pharma U.S., Frank Sanders.

The conference call will be conducted in English, and the dial-in numbers are:

A live audio webcast of the call, including an accompanying slide presentation, will be accessible from the Investor section of Zealand Pharma’s website. Participants are advised to register for the webcast approximately 10 minutes before the start. A recording of the event will be available on the Investor section of Zealand Pharma’s website following the call.

Upcoming events

Zealand Pharma plans to publish results for the fourth quarter of 2021 on March 10, 2022.

Total number of shares and voting rights in Zealand Pharma as of September 30, 2021

Number of shares (nominal value of DKK 1 each): 43,581,697 which is an increase of 39,859 from 43,428,192 as of June 30, 2021.

Therefore, the current Share capital is (nominal value in DKK): 43,581,697.

Number of voting rights: 43,581,697