Imago BioSciences Reports First Quarter 2022 Financial Results and Provides Recent Business Updates

On May 12, 2022 Imago BioSciences, Inc. ("Imago") (Nasdaq: IMGO), a clinical stage biopharmaceutical company discovering and developing new medicines for the treatment of myeloproliferative neoplasms (MPNs) and other bone marrow diseases, reported financial results for the first quarter ended March 31, 2022 and provided a corporate update (Press release, Imago BioSciences, MAY 12, 2022, View Source [SID1234614472]).

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"I am delighted by the clinical progress Imago has made, which was underscored by the completion of enrollment in the Phase 2 trial of bomedemstat for treatment of ET as well as positive interim data from our two Phase 2 trials in ET and MF presented at ASH (Free ASH Whitepaper) 2021 with additional data from these trials to be presented at the upcoming EHA (Free EHA Whitepaper) congress in June. In addition, the Fred Hutchinson Cancer Research Center has initiated dosing in a Phase 1/2 combination study of bomedemstat and atezolizumab for the treatment of small cell lung cancer and we remain on track to initiate our second combination study in the first half of 2022, which is a Phase 2 trial of bomedemstat and ruxolitinib for the treatment of MF," said Hugh Young Rienhoff, Jr., M.D., Chief Executive Officer of Imago BioSciences. "As we continue to assemble a seasoned leadership team, I am pleased to have had Mike join as Chief Operating and Business Officer, as Laura Eichorn transitioned from the Chief Operating Officer role into the Chief Financial Officer on a permanent basis. Looking ahead, Imago expects to initiate a registrational study of bomedemstat for the treatment of ET, subject to an end-of-Phase 2 meeting with the U.S. Food and Drug Administration (FDA), around the end of 2022."

First Quarter 2022 Highlights

Announced results of preliminary discussions with the U.S. Food and Drug Administration (FDA), about key trial design parameters for a registration-directed Phase 3 program of bomedemstat for the treatment of ET. Based on these discussions, we believe a two-arm trial comparing bomedemstat to best available therapy may provide the basis for regulatory approval for the second-line treatment of ET. Subject to final review of the Phase 3 protocol, we have alignment on the study population of patients with ET, viz., those patients who are intolerant of or resistant to hydroxyurea, the agents in the control arm and the composite primary endpoint of durable normalization of platelet and white counts and hemostasis in the absence of progression. Based on completion of enrollment for the Phase 2 clinical trial in ET, and subsequent End of Phase 2 meeting, we expect to submit a final protocol for a registrational Phase 3 pivotal program in 2022, with the first patient dosed thereafter. With positive results from the pivotal clinical program, we would expect to submit applications for regulatory approval with the FDA and the EMA for ET.
Announced completion of enrollment in the Phase 2 trial of bomedemstat for the treatment of ET with 73 patients enrolled on May 3, 2022. To be enrolled in the Phase 2 ET trial, patients had to be intolerant of, or inadequately managed by treatment with one standard-of-care drug, generally hydroxyurea, and also had one or more high-risk prognostic factors, such as being over 60 years of age or having a history of clotting or bleeding events. Primary endpoints of this clinical trial are safety and tolerability, as well as the reduction of platelet count to ≤400 x 109/L, in the absence of any clotting or bleeding events. We are also evaluating several exploratory endpoints, including reduction in mutant allele frequency and prevention of transformation to MF or acute myeloid leukemia. In this Phase 2 trial, as well as our Phase 2 MF trial, we have used platelet count as a biomarker of bomedemstat activity on megakaryocyte function, allowing for individualized dosing. Patients from this trial are eligible to transition into an ongoing Phase 2 Extension Study initiated in 2021 enabling the collection of long-term safety and pharmacodynamic data.
Expanded Executive Leadership. In March 2022, Imago announced the appointments of Michael Arenberg as Chief Operating and Business Officer and Laura G. Eichorn as our Chief Financial Officer. Mr. Arenberg succeeds Ms. Eichorn as COO and is charged with leading strategic operations, investor relations, commercial development and business development of Imago. Ms. Eichorn has transitioned from interim Chief Financial Officer to serving in that role on a permanent basis.
Recent Highlights

Announced that the Fred Hutchinson Cancer Research Center has dosed the initial participant in an investigator-sponsored Phase 1/2 study of bomedemstat in combination with atezolizumab (Tencentriq) in people newly diagnosed with extensive stage small cell lung cancer (ES-SCLC). The study is being led by Rafael Santana-Davila, M.D., associate professor in the University of Washington School of Medicine and Joseph Hiatt, M.D., Ph.D., of Fred Hutchinson Cancer Center ("Fred Hutch"), and in collaboration with the National Cancer Institute (NCI) funded Fred Hutch Lung Specialized Project of Research Excellence. This single-center, open-label study is designed to assess the safety, dose-limiting toxicity, and progression-free survival of bomedemstat in approximately 34 patients with ES-SCLC. More information on this trial can be found on www.clinicaltrials.gov under the identifier NCT05191797.
Announced Data Presentations at the Upcoming 27th EHA (Free EHA Whitepaper) Congress. In May 2022, Imago announced that two abstracts have been accepted for poster presentation at EHA (Free EHA Whitepaper), to be presented on June 10, 2022: "A Phase 2 Study of The LSD1 Inhibitor IMG-7289 (Bomedemstat) For The Treatment Of Essential Thrombocythemia (ET)"; and "A Phase 2 Study of IMG-7289 (Bomedemstat) in Patients With Advanced Myelofibrosis."
Anticipated Upcoming Milestones

Data updates for bomedemstat in MF and ET at EHA (Free EHA Whitepaper) on June 10, 2022
Anticipate initiating Phase 2 combination study of bomedemstat with ruxolitinib in MF in 1H 2022
Expect an End-of-Phase 2 meeting with FDA for bomedemstat in ET in 2H 2022
Expect data updates for the bomedemstat Phase 2 trials in ET and MF at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2022
First Quarter 2022 Financial Results

Cash and Cash Equivalents: As of March 31, 2022, Imago had cash and cash equivalents and short-term investments of $205.8 million, compared to $82.7 million as of March 31, 2021 and $217.4 million as of December 31, 2021.
Research & Development (R&D) Expenses: R&D expenses for the quarter ended March 31, 2022 were $12.5 million (including stock-based compensation expense of $0.7 million) as compared to $4.8 million for the same period in 2021. The overall increase in R&D expenses was primarily related to increased manufacturing costs of drug supplies for our ongoing and planned clinical trials, continued clinical development activities in separate Phase 2 clinical trials for ET and MF, commencement of a Phase 2 extension study started in the second half of 2021 for the long-term follow-up of patients from the ongoing ET and MF clinical trials, and an increase in personnel-related costs, particularly with respect to an increase in the number of research and development employees, including stock-based compensation expense, as we ramped up our operations.
General and Administrative (G&A) Expenses: G&A expenses for the quarter ended March 31, 2022 were $4.0 million (including stock-based compensation expense of $0.7 million) as compared to $2.4 million for the same period in 2021 primarily due to increasing expense associated with operating a publicly traded company and personnel-related costs.
Net Loss: Net loss for the quarter ended March 31, 2022 was $16.4 million compared to $7.1 million for the same period in 2021.

Panbela Provides Business Update and Reports Q1 2022 Financial Results

On May 12, 2022 Panbela Therapeutics, Inc. (Nasdaq: PBLA), a clinical stage company developing disruptive therapeutics for the treatment of patients with cancer, reported its financial results for the quarter ended March 31, 2022 (Press release, Panbela Therapeutics, MAY 12, 2022, View Source [SID1234614511]). Management is hosting an earnings call today at 4:30 p.m. ET.

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The first quarter was marked by meaningful progress.

Q1 and Recent Highlights:

• Agreed to acquire Cancer Prevention Pharmaceuticals, Inc. (CPP). The combined entity would target an estimated $5 billion aggregated market opportunity upon closing.

• Hosted a virtual R&D Day on the company’s investigational drug, SBP-101, as a polyamine metabolism modulator in ovarian cancer.

• Poster presentation highlighting the results for SBP-101 as a polyamine metabolism modulator in ovarian cancer at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) in April 2022. The work reflects the company’s ongoing collaboration with Johns Hopkins University School of Medicine.

• Initiated our ASPIRE trial-a global, randomized, double-blind, placebo controlled clinical trial of SBP-101 in combination with Gemcitabine and Nab-Paclitaxel versus Gemcitabine, Nab-paclitaxel and placebo in patients with untreated metastatic pancreatic ductal adenocarcinoma.

• Announced a new development program in ovarian cancer expected to start in the first half 2022 as the result of positive preclinical data supporting the activity of SBP-101 in ovarian cancer cell lines.

• Poster presentation of abstract for SBP-101 at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Gastrointestinal Cancers Symposium in January 2022.

• Median overall survival of 12.53 months for the phase 1 first line metastatic pancreatic trial was reached shortly after the January poster presentation.

"Q1 and year to date have represented a transformational time of value creation for Panbela. During the quarter, we signed a definitive agreement to acquire CPP, presented ovarian cancer data at AACR (Free AACR Whitepaper) and initiated our global randomized trial in pancreatic cancer," said Jennifer K. Simpson, PhD, MSN, CRNP, President & Chief Executive Officer of Panbela. "Through the pending acquisition and organic execution, Panbela is better positioned to be able to treat more patients, and drive shareholder value

Milestones:

We announced:

• The ASCO (Free ASCO Whitepaper) GI poster presentation in January, and

• The research day to review the ovarian cancer data and ovarian cancer treatment standards. Additionally in the first half, we anticipate:

• First patient enrolled in our ASPIRE trial as well as expansion outside the US.

• Satisfaction of conditions and closing of the CPP acquisition.

• Final data from our Phase I untreated metastatic pancreatic cancer study.

• Initiation of the ovarian cancer clinical program for SBP-101 mid-year.

In addition, during the second half of 2022, we expect to announce the opening of a neoadjuvant pancreatic cancer investigator initiated trial. Subject to closing the CPP transaction, we anticipate announcing additional milestones for 2022 that will reflect the increased flow of planned development activity and data.

First Quarter ended March 31, 2022 Financial Results

General and administrative expenses were $1.8 million in the first quarter of 2022, compared to $1.1 million in the first quarter of 2021. The change is due primarily to expenses, including legal and financial advisory fees, associated with the acquisition of CPP. Research and development expenses were $2.2 million in the first quarter of 2022, compared to $1.1 million in the first quarter of 2021. The change is due primarily to an increase in spending on our clinical studies as we launched the global ASPIRE clinical trial. Net loss in the first quarter of 2022 was $3.7 million, or $0.27 per diluted share, compared to a net loss of $2.3 million, or $0.23 per diluted share, in the first quarter of 2021.

Total cash was $7.4 million as of March 31, 2022. Total current assets were $7.9 million and current liabilities were $4.5 million as of the same date. Also at March 31, 2022, total noncurrent assets, consisting of cash deposits held by our contract research organization, were $3.2 million. The company had no debt as of March 31, 2022. Conference Call Information To participate in this event, dial approximately 5 to 10 minutes before the beginning of the call.

About: SBP-101
SBP-101 is a proprietary polyamine analogue designed to induce polyamine metabolic inhibition (PMI) by exploiting an observed high affinity of the compound for pancreatic ductal adenocarcinoma, ovarian cancer and other tumors. The molecule has shown signals of tumor growth inhibition in clinical studies of US and Australian metastatic pancreatic cancer patients, demonstrating a median overall survival (OS) of 12.53 months, which is now final, and an objective response rate (ORR) of 48%, both exceeding what is seen typically with the standard of care of gemcitabine + nab-paclitaxel suggesting potential complementary activity with the existing FDA-approved standard chemotherapy regimen. In data evaluated from clinical studies to date, SBP-101 has not shown exacerbation of bone marrow suppression and peripheral neuropathy, which can be chemotherapy-related adverse events. Serious visual adverse events have been evaluated and patients with a history of retinopathy or at risk of retinal detachment will be excluded from future SBP-101 studies. The safety data and PMI profile observed in the current Panbela sponsored clinical trial provides support for continued evaluation of SBP-101 in a randomized clinical trial. For more information, please visit View Source

NexImmune Reports First Quarter 2022 Financial Results and Provides Business Updates

On May 12, 2022 NexImmune, Inc. (Nasdaq: NEXI), a clinical-stage biotechnology company developing a novel approach to immunotherapy designed to orchestrate a targeted immune response by directing the function of antigen-specific T cells, reported financial results for the first quarter of 2022 (Press release, NexImmune, MAY 12, 2022, View Source [SID1234614529]).

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"This quarter, we have continued to execute on our clinical and corporate strategy, taking the necessary steps to achieve our upcoming catalysts for the remainder of this year," said Kristi Jones, Chief Executive Officer. "We remain on track to provide a clinical update for the NEXI-001 trial in AML during the second half of 2022, and to provide an update on the expansion cohort in the NEXI-002 trial in relapsed / refractory multiple myeloma. We also plan to file our first solid tumor IND application for HPV-associated malignancies in the near term. In parallel, we are advancing IND-enabling work for our AIM injectable modality and expect to provide updates on preclinical data for oncology and autoimmune indications throughout the second half of the year. With our new target discovery collaborations, we are well-positioned to leverage our modular AIM platform for efficient and rapid new product development. Overall, I am excited with our progress and focus on execution to deliver on upcoming catalysts."

First Quarter 2022 Clinical and Business Highlights

Clinical and Preclinical Updates

NEXI-001

Robust immune responses with signs of clinical activity across all dose levels to date
NEXI-001 continues to be well tolerated across all dose levels administered to date, with no Grade ≥3 treatment-related adverse events, including infusion reactions, GVHD, CRS or neurotoxicity (ICANS), reported
Due to the favorable emerging clinical profile, plans are underway to expand the addressable population with an additional study arm to include patients with haplo-identical donors
Enrollment continues, and updated clinical results are expected to be announced in the second half of 2022
NEXI-002

Expansion phase is ongoing
In this heavily pre-treated population, evidence of immune response and signals clinical activity have been observed. NEXI-002 continues to be well tolerated with no Grade ≥3 treatment-related adverse events, including infusion reactions, GVHD, CRS or neurotoxicity (ICANS), reported
Manufacturing achieved higher final cell count yield in recent products by adjusting prior treatment washout period, updating cell collection guidance and other process adjustments
Further clinical data from the Phase 1/2 trial are expected in the second half of 2022
NEXI-003

Preclinical data supporting the selection of multiple immunogenic antigen peptides commonly expressed on HPV-associated tumors and a cancer survival antigen was presented during the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s Annual Meeting (SITC 2021) in November 2021
Finalized antigen selection for NEXI-003 and manufactured nanoparticles for clinical trials
Investigational New Drug (IND) submission planned for the first half of 2022
Injectable AIM nanoparticle programs and other preclinical research

Advanced in vivo and preclinical work to support the development of AIM injectable nanoparticles as a therapeutic in oncology and autoimmune diseases
Advanced work with Yale University Professor Kevan Herold to evaluate AIM INJ nanoparticles for type 1 diabetes. A JDRF grant award will support the collaboration
Announced a strategic partnership with Zephyr AI in oncology for novel target discovery and validation to support target selection for potential future products candidates
Announced research collaboration with Rutgers, The State University of New Jersey, for neuroendocrine tumor checkpoint targets, which may have utility in other cancers
Announced research collaboration with NYU Langone Perlmutter Cancer Center for melanoma neo-antigen-specific CD8+ T cell expansion using the AIM platform technologies
Announced a preclinical research collaboration with Columbia University Irving Medical Center’s Herbert Irving Comprehensive Cancer Center focused on NexImmune’s AIM ACT in Columbia’s patient-derived organoid models of HPV-associated cancers
Business Updates

Announced the appointment of Kristi Jones as Chief Executive Officer and Member of the Board of Directors
Announced the promotion of Mathias Oelke, Ph.D. to Chief Scientific Officer
Announced the appointment of Dr. Leena Gandhi, Director of Dana-Farber’s Center for Cancer Therapeutic Innovation, to NexImmune’s Board of Directors
Announced the formation of the Autoimmune and Infectious Diseases Scientific Advisory Board
Continued to strengthen the management team with key appointments across the organization
Select 1Q 2022 Financial Highlights

Cash, cash equivalents and marketable securities for the Company as of March 31, 2022 were $65.0 million compared to $81.8 million for quarter ending December 31, 2021. Based upon current operating plans, NexImmune expects that its existing cash, cash equivalents and marketable securities will enable the Company to fund its operating and capital expenditure requirements into the second quarter of 2023.

Research and development expenses were $10.4 million in the first quarter of 2022, compared to $6.0 million for the same period in the prior year. The increase in R&D expenses was mainly attributable to costs for the two clinical trials, as well as personnel-related expenses driven by increased headcount.

General and administrative expenses were $4.6 million, compared to $4.1 million for the same period the prior year. The increase was due primarily to personnel-related expenses and fees related to professional and consulting services.

Net loss, according to generally accepted accounting principles in the U.S. (GAAP), was $15.0 million for the quarter, or a basic and diluted GAAP loss per share of $0.66. This compared to a net loss of $8.5 million, or a basic and diluted GAAP loss per share of $0.71, for the same period the prior year.

Interim report for Q1 2022

On May 12, 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 financial results for the first three months of 2022 (Press release, Zealand Pharmaceuticals, MAY 12, 2022, View Source [SID1234614594]).

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Financial results for the first three months of 2022

·Revenue: DKK 15.1 million / USD 2.3 million (DKK 5.7 million / USD 0.9 million in the first three months of 2021).

·Net operating expenses: DKK -314.2 million / USD -46.9 million (DKK -266.7 million / USD -42.0 million in the first three months of 2021).

·Net operating result: DKK -302.0 million / USD -45.1 million (DKK -261.1 million / USD -41.2 million in the first three months of 2021).

.Net financial items: DKK 133.0 million / USD 19.9 million (DKK 19.8 million / USD 3.1 million in the first three months of 2021).

·Net result from Discontinued Operations Related to Restructuring: DKK -41.8 million / USD -6.2 million (DKK 0.0 million / USD 0.0 million in the first three months of 2021).

·Cash, cash equivalents, and marketable securities: DKK 1,123.2 million / USD 167.6 million as of March 31, 2022 (March 31, 2021: DKK 1,604.1 million / USD 252.9 million).

Business highlights to date for 2022

·Appointed new Chief Executive Officer, refocused strategy to prioritize research & development and streamlined operations. Zealand announced a corporate restructuring intended to leverage its peptide platform by prioritizing investment in its research and development pipeline programs and streamline its commercial operations. The changes will refocus the company’s resources by reducing expenses while investing in strategic development and commercialization partnerships of pipeline assets. Dr. Adam Steensberg has assumed the position of Chief Executive Officer, refocusing Zealand to better leverage its next-generation peptide platform to address unmet medical needs of patients. The Company’s commercial operations have been restructured to pursue partnerships for Zegalogue, V-Go and the glepaglutide and dasiglucagon late-stage clinical portfolio. With the change, annual operating expense reductions are expected to be at least 35% from 2021 levels.

·Announced Amendment to Note Purchase Agreement with Oberland Capital. Zealand to repurchase $50.0 million of note principal with a 1.2x prepayment premium. Agreement includes potential for a further $75 million incremental capital following specific events and removes the liquidity covenant. The amendment with Oberland Capital reinforces Zealand’s commitment to its refocused strategy by prioritizing research and development programs and together with the restructuring extends the company’s cash runway into 2023, beyond significant pipeline milestones.

·Completion of patient enrollment in the second Phase 3 Trial (17103) of dasiglucagon for the treatment of Congenital hyperinsulinism (CHI) in neonates up to 12 months old. Top-line results are expected in the second quarter of 2022. This Phase 3 study is the last in the program which constitute the largest clinical development program ever conducted in CHI.

·Completion of patient enrollment in the pivotal Phase 3 trial (EASE-SBS 1) of glepaglutide in patients with short bowel syndrome (SBS). Full results of the EASE-SBS 1 pivotal Phase 3 trial are expected in the third quarter of 2022.

Adam Steensberg, President and Chief Executive Officer at Zealand Pharma, comments:

"Zealand Pharma has undergone a transformational first quarter in 2022. We have transitioned the company into a more focused and cost-effective organization that plays to our strengths as a company. We are executing on the restructuring of our commercial organization in the United States and are seeking strategic partnerships in order to maximize the value of V-Go and Zegalogue. We look forward to partnering our commercial programs as well our late-stage clinical pipeline programs as we look to further leverage our exciting peptide platform though strategic collaborations," said Adam Steensberg, President and Chief Executive Officer of Zealand Pharma.

"By improving our operational efficiency and targeting business development efforts, we will be in a position to fully leverage the value of our most advanced assets and develop new peptide-based therapies through 2022 and beyond. As an R&D focused organization with a strong late-stage pipeline, we are excited about our upcoming milestones for the remainder of the year. Later this year we expect Phase 3 data for dasiglucagon in CHI in the second quarter, glepaglutide in SBS in the third quarter and Phase I data for our Amylin analogue targeting obesity later this year."

Financial guidance for 2022

On March 30, 2022 Zealand updated the guidance for net product revenue from the sales of commercial products to be DKK 115 million +/- 10%. This was a decrease of 120 million Danish kroner from the guidance issued on March 10, 2022. Combined sales of V-Go and Zegalogue in Q1 were 39.2 million Danish kroner and in line with the updated guidance.

Following the company’s announced intent to exit its V-Go activities, net product revenue for the device is to be accounted for as discontinued operations and no longer reported as net product revenue. As such, net product revenue reported in the Q1 earnings release only reflects sales of Zegalogue, which were 4.1 million Danish kroner with full year net product revenue projected to be 19 million Danish kroner excluding any potential partnerships or licensing agreements.

In 2022, 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 2022 are expected to be DKK 1,000 million +/-10%. This is unchanged from our updated guidance issued on March 30, 2022 and is a decrease of DKK 200 million from the guidance issued on March 10, 2022.

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 three months of 2022. Participating in the call will be Chief Executive Officer Adam Steensberg and Chief Financial Officer Matt Dallas. The presentation will be followed by a Q&A session with the presenters.

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.

Refocused Strategy

On March 30th the company announced a refocused strategy prioritizing research and development programs. As part of this strategy, commercial operations were restructured to pursue partnerships for Zegalogue, V-Go and the glepaglutide and dasiglucagon late-stage clinical portfolio. The global cost base will be reduced by approximately 35% from 2021 levels with the US workforce to be reduced by approximately 90% by the third quarter of this year. Approximately 65% of the reductions in the US workforce were effective in April.

As a consequence of the changes to Zealand Pharma’s strategic focus, by mutual agreement, Chief Financial Officer (CFO) Matthew Dallas will leave the company on August 31st 2022. Since joining the company in 2019 Matthew Dallas has provided valuable financial leadership with pre-commercial and commercial financial planning and has built a robust and experienced Finance Department at the company. A search for a new CFO is underway and we will communicate further on progress with the search when we can.

Progress for Commercialized Products

Zegalogue (dasiglucagon) injection

Zegalogue launched in the U.S. in late June 2021. The Company’s primary goal in the initial phase of launch was to establish favorable market access coverage working with Pharmacy Benefit Managers (PBMs), Managed Care Organizations (MCOs), and state Medicaid agencies to add Zegalogue to their respective formularies. In the second quarter of 2022 we expect to initiate a clinical study in children aged 1 to 6 years to explore the safety and effectiveness of 0.6 mg/0.6mL dasiglucagon injection in this age group.

Zegalogue net revenue for the first three months of 2022 was DKK 4.1 million / USD $0.6 million.

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 first three months of 2022 was DKK 35.7 million / USD $5.3 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.

Zealand’s partner, Beta Bionics, and the study sponsor, the Jaeb Center for Health Research, initiated screening into the Phase 3, Bihormonal iLet Bionic Pancreas Pivotal Program, utilizing insulin and dasiglucagon in late 2021 with dosing of the first participants in the study are expected in later in 2022. The Phase 3 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 is 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 bihormonal 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.

On April 30th Beta Bionics announced the results of the insulin-only bionic pancreas pivotal trial results. The pivotal trial was designed to test the safety and efficacy of the iLet Bionic Pancreas relative to a standard of care control group over a 13-week study period. The standard of care group was comprised approximately one-third each on automated insulin delivery (AID) systems, insulin-pump therapy with continuous glucose monitoring (CGM), and multiple daily injection therapy with CGM. The trial was conducted in a home-use setting and enrolled 440 adults and children aged 6 years and older with type 1 diabetes. The primary analysis of the trial compared the iLet, using Humalog or Novolog, versus standard of care in 326 adults and children; the remaining 114 adult participants used the iLet with Fiasp.

The iLet Bionic Pancreas met all key endpoints in the trial with improved outcomes over standard of care in the following: significant reduction in HbA1c, no increased risk of hypoglycemia, and increased time in range.

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.

In 2022, Zealand expect to present data from out-patient Phase 2 trials, utilizing the dasiglucagon mini-dose pen in in people with type 1 diabetes and for people that suffer from meal-induced hypoglycemia following gastric bypass surgery (ClinicalTrials.gov Identifier: NCT04764968 and NCT04836273).

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.

Data from the first Phase 3 trial in the program, trial 17109, were reported 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 have completed enrollment into the second Phase 3 trial, 17103, in neonates up to 12 months old with CHI Trial results are expected in the second quarter of 2022 and if positive we expect to submit an NDA to the US FDA based on data from both Phase 3 trials and the ongoing long-term extension trial, 17106.

Glepaglutide for short bowel syndrome (SBS)

Glepaglutide is a long-acting GLP-2 analog, being investigated for the potential treatment of short bowel syndrome with the primary endpoint of reducing or eliminating the need for parenteral support in people living with SBS, as further detailed below. Phase 2 data have shown the potential of glepaglutide to increase intestinal absorption in people with SBS.

The EASE-SBS Phase 3 program includes 4 trials. EASE-SBS 1 is the pivotal Phase 3 trial with enrolment of up to 108 patients with SBS that seeks to establish the efficacy and safety of once- and twice-weekly administration of glepaglutide. The initial trial duration is six months, whereafter trial participants are able to enroll 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.

Enrollment has been completed in the EASE-SBS 1 trial and we expect to have results in Q3 2022. The primary endpoint in the trial is the absolute reduction in parenteral support and in the event of positive trial results we expect to submit an NDA with the FDA based on efficacy and safety data from the full EASE-SBS trial program. The 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 1b multiple-ascending dose, safety and tolerability trial investigating dapiglutide in healthy volunteers was completed in November 2021 and dapiglutide was found to have an acceptable safety and tolerability profile. Results showed a plasma half-life allowing for once weekly dosing and effects on several biomarkers suggest clinically relevant exposures of dapiglutide were achieved. Zealand expects to present data from the Phase 1b trial at scientific conferences and to initiate the next development steps in 2022.

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 and type 2 diabetes. 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.

The Phase 1a clinical trial with ZP8396 for potential treatment of obesity was initiated in November 2021. This First-in-Human, randomized, single ascending dose trial will assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of ZP8396 administered to healthy subjects. Zealand expects to initiate the Phase 1b multiple ascending dose trial of ZP8396 later in 2022.

BI 456906 for obesity, diabetes and non-alcoholic steatohepatitis (with Boehringer Ingelheim)

The dual glucagon (GCGR)/GLP1 receptor agonist, BI 456906, activates two key gut hormone receptors simultaneously and may offer better efficacy than current single-hormone receptor agonist treatments. The lead molecule BI 456906 is targeting treatment of obesity and associated metabolic diseases. At Obesity Week in November 2021, 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 in people with overweight/obesity.

The molecule is being assessed across three parallel Phase 2 trials. The Phase 2 trial in people with diabetes is completed and we are planning to present data from the trial at scientific conferences later in 2022. The trial evaluated 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 were to assess the effect on change in body weight. The second Phase 2 randomized double-blind placebo-controlled dose-finding trial evaluating BI 456906 in people with overweight/obesity has reached its randomization target (ClinicalTrials.gov Identifier: NCT04667377). We expect trial results later this year with the primary endpoint being the percentage change in body weight at week 46 compared to placebo. The third Phase 2 randomized double-blind placebo-controlled dose-finding trial is evaluating BI 456906 in people with NASH and liver fibrosis (F2/F3) (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 NASH 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 are collaborating 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.

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.

Research

Research collaboration with Iktos to develop Artificial Intelligence technology for peptide drug design

Iktos, a company specialized in Artificial Intelligence for new drug design, and Zealand Pharma, are collaborating to develop generative modelling artificial intelligence (AI) technology for application to peptide drug design. Under the collaboration agreement, Zealand will evaluate the application of Iktos AI technology to the peptide space, and Iktos and Zealand will co-develop generative and predictive AI technology for peptide drug design, leveraging both Iktos’ proprietary generative modelling technologies and capabilities in machine learning and AI, and Zealand’s expertise in peptide drug discovery know-how.

Additional Updates

None

Upcoming events

Zealand Pharma plans to publish results for the second quarter of 2022 on August 11, 2022.

Total number of shares and voting rights in Zealand Pharma as of March 31, 2022

Number of shares (nominal value of DKK 1 each): 43,634,142 which is unchanged from 43,643,142 as of March 31, 2021.

Therefore, the current Share capital is (nominal value in DKK): 43,634,142.

Number of voting rights: 43,634,142

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. We maintain compliance with guidance from applicable government and health authorities as appropriate.

Update regarding Russia and Ukraine

Zealand has reviewed its business operations in light of the geopolitical instability in Europe and concluded that employees, clinical studies, or product supply are not affected or at material risk at this time

Caribou Biosciences Announces Positive Initial Data for CB-010 Anti-CD19 Allogeneic CAR-T Cell Therapy

On May 12, 2022 Caribou Biosciences, Inc. (Nasdaq: CRBU), a leading clinical-stage CRISPR genome-editing biopharmaceutical company, reported initial results demonstrating a 100% overall response rate (ORR) and 80% complete response rate (CR) in cohort 1 (n=5 evaluable) from its ANTLER Phase 1 trial for CB-010 in patients with relapsed or refractory B cell non-Hodgkin lymphoma (r/r B-NHL) (Press release, Caribou Biosciences, MAY 12, 2022, View Source [SID1234614321]). These initial data are scheduled to be shared at the European Hematology Association (EHA) (Free EHA Whitepaper) 2022 Hybrid Congress, being held in Vienna, Austria, June 9-17, 2022.

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"Our initial CB-010 data are exciting, and we believe these results show the potential to set a new therapeutic bar in treating patients with aggressive r/r B-NHL. These excellent initial outcomes represent important steps toward validating our chRDNA genome-editing platform as well as our plans for future development of CB-010 and our broader pipeline," said Rachel Haurwitz, Ph.D., Caribou’s president and chief executive officer. "CB-010 is the first allogeneic anti-CD19 CAR-T cell therapy in the clinic with a PD-1 knock-out, a genome-editing strategy designed to limit premature CAR-T cell exhaustion, potentially leading to better tumor debulking and an improved therapeutic index through sustained antitumor activity. We are slated to present additional ANTLER interim data at EHA (Free EHA Whitepaper) next month and expect more data by year end as we continue to advance our lead program. Our overarching goal is to develop CB-010 such that it will meaningfully rival autologous cell therapies to reach broader groups of patients globally who are in need of off-the-shelf treatments."

"We are excited to see a 100% overall response rate with CB-010 at dose level 1 for these patients who have limited treatment options," said Syed Rizvi, M.D., Caribou’s chief medical officer. "We believe this initial level of activity is unparalleled for a single, starting dose of cell therapy. CB-010 was generally well-tolerated with adverse events routinely observed in autologous or allogeneic anti-CD19 CAR-T cell therapies. At EHA (Free EHA Whitepaper) next month, we are scheduled to share longer follow up data from the patients in Cohort 1 who received a single administration of CB-010 at the first dose level. We look forward to continuing the development of our pipeline of allogeneic CAR-T cell therapies for patients with hematologic malignancies."

The EHA (Free EHA Whitepaper) abstract includes safety, tolerability, and initial antitumor activity of CB-010 administered at dose level 1 (40×106 CAR-T cells) to 6 patients with r/r B-NHL who had relapsed after previous treatment with a median of 3 prior therapies (range 2-8). Prior to a single dose of CB-010, patients received a lymphodepletion regimen consisting of cyclophosphamide at 60 mg/kg/d for 2 days followed by fludarabine at 25 mg/m2/d for 5 days.

As of the February 23, 2022 data cutoff date, 6 patients had been treated with CB-010 and 5 had completed the 28-day dose-limiting toxicity (DLT) evaluation period. 100% (n=5) achieved a response; 80% (n=4) achieved a CR, and 20% (n=1) achieved a partial response (PR). All 4 patients who achieved a CR at 28 days had an ongoing CR at 3 months. The longest measured CR as of the data cutoff date was 6 months.

Footnote: data cutoff date of February 23, 2022, data collection ongoing, efficacy based on Lugano criteria

Following treatment with CB-010, there were no cases of graft versus host disease. 3 of 6 patients developed Grade 3 or 4 adverse events (AEs) within the first 28 days: neutropenia (50%), thrombocytopenia (33%), anemia (17%), and hypogammaglobulinemia (17%). One patient experienced Grade 1 CRS (17%) and Grade 3 ICANS (17%), which was characterized as a DLT, for which the patient received tocilizumab and steroids and recovered from the DLT within 39 hours. This patient went on to achieve a CR.

Based on promising initial safety and efficacy data from cohort 1 at dose level 1 (40×106 CAR-T cells), the ANTLER trial is now enrolling patients in cohort 2 at dose level 2 (80×106 CAR-T cells). Additional data are expected by year end.

Details of the poster presentation at EHA (Free EHA Whitepaper) are as follows:

Title: First-in-human trial of CB-010, a CRISPR-edited allogeneic anti-CD19 CAR-T cell therapy with a PD-1 knock out, in patients with relapsed or refractory B cell non-Hodgkin lymphoma (ANTLER study)

Abstract: P1455

Presenter: Loretta J. Nastoupil, M.D., section chief, new drug development; associate professor, Department of Lymphoma/Myeloma, The University of Texas MD Anderson Cancer Center

Date and Time: Friday, June 10, 2022, 16:30 – 17:45 CEST (10:30 – 11:45 am ET)

Session Title: Gene therapy, cellular immunotherapy and vaccination – Clinical

Location: Messe Wien Exhibition & Congress Center, Vienna, Austria

Presentations and posters will be available for registered attendees of EHA (Free EHA Whitepaper) for on-demand viewing on the EHA (Free EHA Whitepaper) website on June 10, 2022 at 9:00 am CEST (3:00 am ET). Caribou plans to issue a press release on the data at 9:00 am CEST (3:00 am ET) on Friday June 10, 2022 and the poster will be available on the Presentations page of the Investors section of Caribou’s website.

Webcast Conference Call Today at 10:15 am ET

Caribou will host a webcast conference call today to discuss the initial ANTLER data for CB-010 in the accepted EHA (Free EHA Whitepaper) abstract. The webcast presenters will include:

Rachel Haurwitz, Ph.D., president and chief executive officer of Caribou

Syed Rizvi, M.D., chief medical officer of Caribou

Steven Kanner, Ph.D., chief scientific officer of Caribou

Jason O’Byrne, chief financial officer of Caribou

The live webcast and conference call, with an accompanying presentation, will be accessible under Events in the Investors section of the company’s website. To participate in the conference call, dial 844-862-9351 (domestic) or 929-517-0932 (international) and reference conference ID #7589468. The archived audio webcast will be available on Caribou’s website following the call and will be available for 30 days.

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About CB-010

CB-010 is the lead product candidate from Caribou’s allogeneic CAR-T cell therapy platform and is being evaluated in patients with relapsed or refractory B cell non-Hodgkin lymphoma (r/r B-NHL) in the ongoing ANTLER Phase 1 trial. CB-010 is an allogeneic anti-CD19 CAR-T cell therapy engineered using Cas9 CRISPR hybrid RNA-DNA (chRDNA) technology to insert a CD19-specific CAR into the TRAC gene and knock out PD-1 to boost the persistence of antitumor activity. CB-010 is the first allogeneic CAR-T cell therapy in the clinic with a PD-1 knock out. Additional information on the ANTLER trial can be found at View Source using identifier NCT04637763.

About Caribou’s Novel Next-Generation CRISPR Platform

CRISPR genome editing uses easily designed, modular biological tools to make DNA changes in living cells. There are two basic components of Class 2 CRISPR systems: the nuclease protein that cuts DNA and the RNA molecule(s) that guide the nuclease to generate a site-specific, double-stranded break, leading to an edit at the targeted genomic site. CRISPR systems are capable of editing unintended genomic sites, known as off-target editing, which may lead to harmful effects on cellular function and phenotype. In response to this challenge, Caribou has developed CRISPR hybrid RNA-DNA guides (chRDNAs; pronounced "chardonnays") that direct substantially more precise genome editing compared to all-RNA guides. Caribou is deploying the power of its Cas12a chRDNA technology to carry out high efficiency multiple edits, including multiplex gene insertions, to develop CRISPR-edited therapies.