On January 26, 2022 Immunocore Holdings plc (Nasdaq: IMCR) ("Immunocore" or the "Company"), a commercial-stage biotechnology company pioneering the development of a novel class of T cell receptor (TCR) bispecific immunotherapies designed to treat a broad range of diseases, including cancer, autoimmune and infectious diseases reported approval from the United States Food and Drug Administration (FDA) of KIMMTRAK (tebentafusp-tebn) for the treatment of HLA-A*02:01-positive adult patients with unresectable or metastatic uveal melanoma (mUM) (Press release, Immunocore, JAN 26, 2022, View Source [SID1234607391]).

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KIMMTRAK’s approval establishes many firsts as the first TCR therapeutic to receive regulatory approval from the FDA, the first bispecific T cell engager to receive regulatory approval from the FDA to treat a solid tumor, and the first and only therapy for the treatment of unresectable or metastatic uveal melanoma to be approved by the FDA.

Bahija Jallal, Chief Executive Officer of Immunocore, said: "Today’s approval of KIMMTRAK is a historic milestone and the culmination of years of dedication by the Immunocore team, patients, and our healthcare partners. Every year in the United States, hundreds of people are diagnosed with metastatic uveal melanoma who, until now, had no approved treatment options. KIMMTRAK is the first therapy to demonstrate a survival benefit to patients with this disease and we are focused on making KIMMTRAK available as quickly as possible.

Dr. Jallal continues, "We’re also proud to have developed the world’s first approved TCR therapeutic, which we believe validates the strength of our platform and opens doors for us to explore further breakthrough discoveries in TCR therapeutics for the treatment of other cancers and diseases with high unmet need."

"Uveal melanoma is a devastating disease that has historically resulted in death within a year of metastasis for our patients," said John Kirkwood, MD, director of the Melanoma Center at the UPMC Hillman Cancer Center. "The approval of KIMMTRAK (tebentafusp-tebn) represents a major paradigm shift in the treatment of metastatic uveal melanoma, and for the first time offers hope to those with this aggressive form of cancer."

The approval of KIMMTRAK is based on the results of Immunocore’s Phase 3 IMCgp100-202 clinical trial, which were published in the September 23, 2021 issue of the New England Journal of Medicine. The randomized pivotal trial evaluated overall survival (OS) of KIMMTRAK compared to investigator’s choice (either pembrolizumab, ipilimumab, or dacarbazine) in patients with previously untreated mUM. 378 patients were randomized in a 2:1 ratio to either KIMMTRAK or investigator’s choice. Data from the trial, the largest Phase 3 trial undertaken in mUM, showed that KIMMTRAK demonstrated unprecedented median OS benefit as a first-line treatment. The OS Hazard Ratio (HR) in the intent-to-treat population favored KIMMTRAK, HR=0.51 (95% CI: 0.37, 0.71); p< 0.0001, over investigator’s choice (82% pembrolizumab; 13% ipilimumab; 6% dacarbazine). In the clinical trials, across both arms, patients stopped treatment for disease progression, unless the patient was otherwise deriving benefit, or for unacceptable toxicity.

"When my husband, Gregg, was diagnosed with metastatic uveal melanoma, it was devastating to learn that there were no treatment options shown to extend life." said Sara Selig, MD, MPH, Co-Founder and Director of the Melanoma Research Foundation’s (MRF) CURE OM initiative. "Now, for the first time in the history of this disease, we will soon see extended survival in the next generation of metastatic uveal melanoma patients."

In the randomized Phase 3 trial of KIMMTRAK (tebentafusp-tebn), treatment-related adverse reactions were manageable and consistent with the proposed mechanism. Among the patients treated with KIMMTRAK, the most common Grade 3 or higher adverse reactions were rash (18%), pyrexia (4%), and pruritus (5%). In the 245 patients treated with KIMMTRAK, Grade 3 cytokine release syndrome (CRS) occurred in <1% of patients and were generally well-managed. There were no Grade 4 or fatal CRS events observed in the Phase 3 trial. A boxed warning is included for CRS as it has the potential to become serious or life-threatening if not managed appropriately.

"Until now, effective treatment options for metastatic uveal melanoma patients were virtually non-existent. The approval of KIMMTRAK represents not only a new therapy but a new hope for the individuals and the families of those diagnosed with the deadliest form of eye cancer," said Kyleigh LiPira, MBA, CEO of the MRF.

The company is ready to commercialize KIMMTRAK and expects to make the product commercially available in the United States within weeks.

KIMMTRAK was granted Breakthrough Therapy Designation for unresectable or metastatic uveal melanoma by the FDA in February 2021. The Biologics License Application (BLA) approval followed review under the Real-Time Oncology Review (RTOR) program, an initiative of the FDA’s Oncology Center of Excellence designed for efficient delivery of safe and effective cancer treatments to patients. The approval was granted four weeks ahead of the assigned PDUFA date of February 23, 2022. Immunocore provided an Assessment AID to facilitate FDA review. KIMMTRAK is being reviewed under the FDA’s Project Orbis initiative, which enabled concurrent review by the health authorities in partner countries that have requested participation.

The European Medicines Agency (EMA), the United Kingdom’s Medicines and Healthcare Regulatory Agency (MHRA), Health Canada, and the Australian Government Department of Health Therapeutic Goods Administration (TGA) have accepted the submission of the Company’s Marketing Authorisation Application. Additionally, Immunocore launched a global early access program to make KIMMTRAK readily available to mUM patients. There are currently over 200 patients in 13 countries in the early access program.

Immunocore is committed to helping patients who need KIMMTRAK obtain access via its KIMMTRAKConnect program. The program provides services with dedicated nurse case managers who provide personalized support, including educational resources, financial assistance, and site of care coordination. To learn more, visit KIMMTRAKConnect.com, which will launch later this week, or call 844-775-CARE (2273).

On January 26, 2022 Abbott (NYSE: ABT) reported that financial results for the fourth quarter and full year ended Dec. 31, 2021, and issued its financial outlook for 2022 (Press release, Abbott, JAN 26, 2022, View Source [SID1234607389]).

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Fourth-quarter sales of $11.5 billion increased 7.2 percent on a reported basis and 7.7 percent on an organic basis, which excludes the impact of foreign exchange.
Fourth-quarter GAAP diluted EPS was $1.11 and adjusted diluted EPS, which excludes specified items, was $1.32.
Full-year 2021 GAAP diluted EPS from continuing operations was $3.94 and adjusted diluted EPS from continuing operations was $5.21, reflecting 42.7% growth versus the prior year.1
Global COVID-19 testing-related sales were $2.3 billion in the fourth quarter and $7.7 billion for the full-year. Abbott has distributed more than 1.4 billion COVID-19 tests since the start of the pandemic.
Abbott issues full-year 2022 guidance for diluted EPS from continuing operations on a GAAP basis of at least $3.43 and full-year adjusted diluted EPS from continuing operations of at least $4.70.
Full-year 2022 guidance includes an initial COVID-19 testing-related sales forecast of $2.5 billion, which Abbott expects to occur early in the year and will update on a quarterly basis.
Abbott’s R&D pipeline continues to deliver a steady cadence of new products. Select 2021 highlights include U.S. Centers for Medicare & Medicaid Services expanded reimbursement for MitraClip, U.S. launches of Amplatzer Amulet, NeuroSphere Virtual Clinic, and Portico transcatheter aortic valve replacement (TAVR) system, along with the international launch of Navitor TAVR system.
"2021 was an outstanding year for Abbott," said Robert B. Ford, chairman and chief executive officer, Abbott. "We achieved more than 40 percent EPS growth, exceeding the baseline EPS guidance we set at the beginning of last year and, importantly, continued to advance our new product pipeline across the portfolio."

FOURTH-QUARTER BUSINESS OVERVIEW
Note: Management believes that measuring sales growth rates on an organic basis is an appropriate way for investors to best understand the underlying performance of the business. Organic sales growth excludes the impact of foreign exchange.

Following are sales by business segment and commentary for the fourth quarter 2021:

* Total Q4 2021 Abbott sales from continuing operations include Other Sales of approximately $3 million.

* Total 12M 2021 Abbott sales from continuing operations include Other Sales of approximately $52 million.

n/a = Not Applicable.

Note: In order to compute results excluding the impact of exchange rates, current year U.S. dollar sales are multiplied or divided, as appropriate, by the current year average foreign exchange rates and then those amounts are multiplied or divided, as appropriate, by the prior year average foreign exchange rates.

Fourth-quarter 2021 worldwide sales of $11.5 billion increased 7.2 percent on a reported basis and 7.7 percent on an organic basis. Full-year 2021 worldwide sales increased 24.5 percent on a reported basis and 22.9 percent on an organic basis.

Worldwide sales, excluding COVID-19 testing-related sales,2 increased 9.6 percent on a reported basis and 10.3 percent on an organic basis in the fourth quarter, and 15.2 percent on a reported basis and 13.7 percent on an organic basis in the full year 2021.

Compared to pre-pandemic sales in 2019, worldwide sales, excluding COVID-19 testing-related sales,3 increased 10.0 percent on a reported basis and 10.8 percent on an organic basis in the fourth quarter.

Worldwide Nutrition sales increased 5.5 percent on a reported basis and 5.9 percent on an organic basis in the fourth quarter.

In Adult Nutrition, strong performance of Ensure, Abbott’s market-leading complete and balanced nutrition brand, and Glucerna, Abbott’s market-leading diabetes nutrition brand, led to global sales growth of 8.1 percent on a reported basis and 9.0 percent on an organic basis.

Worldwide Pediatric Nutrition sales increased 3.1 percent on both a reported basis and organic basis. Strong performance of Abbott’s market-leading oral hydration brand, Pedialyte, and continued share growth in infant nutrition led to U.S. Pediatric Nutrition growth of 14.5 percent. In International Pediatric Nutrition, sales were unfavorably impacted primarily by challenging market conditions in China.

Worldwide Diagnostics sales increased 2.9 percent on a reported basis in the fourth quarter and increased 3.3 percent on an organic basis. Global COVID-19 testing-related sales were $2.3 billion in the fourth quarter, including combined sales of $2.1 billion from Abbott’s BinaxNOW, Panbio and ID NOW rapid testing platforms.

Excluding COVID-19 testing-related sales, worldwide Diagnostics sales increased 8.2 percent on a reported basis in the fourth quarter and 8.7 percent on an organic basis.4

In Molecular Diagnostics, fourth-quarter sales growth was negatively impacted by lower COVID-19 testing-related sales compared to the prior year. Excluding COVID-19 testing-related sales, worldwide Molecular Diagnostics sales increased 24.1 percent on a reported basis and 24.5 percent on an organic basis, including 34.6 percent in the U.S. and 20.7 percent internationally.5

In Rapid Diagnostics, fourth-quarter international sales growth was negatively impacted by lower COVID-19 testing-related sales compared to the prior year. Excluding COVID-19 testing-related sales, international Rapid Diagnostics sales increased 9.7 percent on a reported basis and 10.3 percent on an organic basis.6

Established Pharmaceuticals sales increased 4.9 percent on a reported basis in the fourth quarter and increased 5.8 percent on an organic basis.

Key Emerging Markets include India, Brazil, Russia and China along with several additional emerging countries that represent the most attractive long-term growth opportunities for Abbott’s branded generics product portfolio. Sales in these geographies increased 4.1 percent on a reported basis in the quarter and increased 5.2 percent on an organic basis. Organic sales growth was led by strong growth across several geographies, including India, Russia and China.

Other sales increased 6.8 percent on a reported basis in the quarter and increased 7.5 percent on an organic basis.

Worldwide Medical Devices sales increased 15.1 percent on a reported basis in the fourth quarter and increased 15.9 percent on an organic basis. Strong growth in the quarter was driven by continued recovery from the COVID-19 pandemic and strong growth in Diabetes Care.

Compared to pre-pandemic sales in 2019, Medical Devices sales increased 17.0 percent on a reported basis and 15.8 percent on an organic basis in the fourth quarter, led by double-digit growth in Heart Failure, Structural Heart and Diabetes Care.7

In Diabetes Care, FreeStyle Libre sales were $1.0 billion in the quarter, which represents sales growth of 35.4 percent on a reported basis and 36.0 percent on an organic basis.

Abbott continued to strengthen its Medical Devices portfolio with several pipeline advancements in 2021, including:

U.S. Centers for Medicare & Medicaid Services expanded reimbursement coverage for Abbott’s revolutionary MitraClip device.
U.S. launch of NeuroSphere Virtual Clinic, a first-of-its-kind technology that allows patients to communicate with physicians and receive new treatment settings remotely as needed.
U.S. FDA approval of Amplatzer Amulet Left Atrial Appendage Occluder to treat people with atrial fibrillation who are at risk of ischemic stroke.
U.S. FDA approval of Portico with FlexNav transcatheter aortic valve replacement (TAVR) system to treat people with symptomatic, severe aortic stenosis who are at high risk for open-heart surgery.
CE Mark for Navitor, Abbott’s latest-generation TAVR system.
ABBOTT’S EARNINGS-PER-SHARE GUIDANCE
Abbott projects full-year 2022 diluted earnings per share from continuing operations under GAAP of at least $3.43. Abbott forecasts specified items for the full-year 2022 of $1.27 per share primarily related to intangible amortization, restructuring and cost reduction initiatives, expenses associated with acquisitions and other net expenses. Excluding specified items, projected adjusted diluted earnings per share from continuing operations would be at least $4.70 for the full-year 2022.

Abbott projects first-quarter 2022 diluted earnings per share from continuing operations under GAAP of at least $1.20. Abbott forecasts specified items for the first-quarter 2022 of $0.30 per share primarily related to intangible amortization, restructuring and cost reduction initiatives, expenses associated with acquisitions and other net expenses. Excluding specified items, projected adjusted diluted earnings per share from continuing operations would be at least $1.50 for the first quarter.

ABBOTT DECLARES 392ND CONSECUTIVE QUARTERLY DIVIDEND
On Dec. 10, 2021, the board of directors of Abbott declared the company’s quarterly dividend of $0.47 per share. Abbott’s cash dividend is payable Feb. 15, 2022 to shareholders of record at the close of business on Jan. 14, 2022.

Abbott has increased its dividend payout for 50 consecutive years and is a member of the S&P 500 Dividend Aristocrats Index, which tracks companies that have annually increased their dividend for at least 25 consecutive years.

On January 26, 2022 Ziopharm Presented the Corporate Presentation(Presentation, Ziopharm, JAN 26, 2022, View Source [SID1234607388])

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On January 26, 2022 Diamyd Medical reported it’s B-share is traded on Nasdaq First North Growth Market under the ticker DMYD B (Press release, Diamyd Medical, JAN 26, 2022, View Source;ClipID=4167222 [SID1234607387]).

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September 1, 2021 – November 30, 2021

Net result: MSEK -37.8 (104.9). The previous year contains a one-off effect of corresponding MSEK 117.5 from divestment of shares in Companion Medical, Inc.
Result per share: SEK -0.5 (1.5)
Cash flow from operating activities: MSEK 0 (-14.0)
Cash and cash equivalents at November 30, 2021: MSEK 240.7 (173.0)
Significant events during the first quarter, September 1, 2021–November 30, 2021

Diamyd Medical paused start of Phase III trial pending additional evaluations of manufacturing process
Diamyd Medical announced to initiate its type 1 diabetes Phase III trial in Europe as discussions continue with FDA regarding partial clinical hold in the US
Diamyd Medical and partners were awarded SEK 40 million in VINNOVA funding
First regulatory approval to start the Phase III trial was received
Start of the Phase III trial in the US was paused pending clarification of FDA questions
Property with manufacturing facility in Umeå was acquired
SEK 150 million was raised via a directed share issue
Other events during the first quarter

Clinical results with Diamyd Medical’s study drug Remygen were published in a scientific journal
A new analysis supporting the effect of Diamyd was elected to be presented at the ISPAD scientific conference
Significant events after the first quarter

Interim report indicated similar immunological response in LADA-patients treated with intralymphatic Diamyd as in type 1 diabetes patients
Other events after the first quarter

Diamyd Medical patent for intralymphatic Diamyd to be granted in China
Australia granted Diamyd Medical a broadening of patent for intralymphatic injection of antigens in autoimmune diabetes
Comments by CEO Ulf Hannelius
We are very much looking forward to the upcoming Phase III precision medicine trial DIAGNODE-3, approved so far by Competent Authorities and Ethical Review Boards in the Czech Republic, the Netherlands, Spain and Poland. To get started with the trial soon – and since we do not compromise on safety anywhere – we have been working hard to rule out that a potential contamination early in the process could in any way affect the drug product. Indeed, we have a safe and robust process that encompasses several purification steps designed to remove contaminants of this nature and additional analyses are ongoing with the goal of being able to initiate DIAGNODE-3 and other trials in the development pipeline soonest possible.

Our investment in the Umeå manufacturing facility is central to securing long-term access and control over our Diamyd flagship asset. The experimental small-scale manufacturing process is in place and the large-scale process equipment have been installed. Next, the plan is to have the process in Umeå tested, validated and GMP certified later this year and to get the facility approved and production ready in the second calendar quarter of 2023.

It was rewarding to see the interim results in December from GADinLADA, the pilot trial in LADA evaluating intralymphatic injections of Diamyd. No safety issues were observed, and the immune response appears similar to what has been observed in type 1 diabetes patients treated with Diamyd. This is the first time this administration mode of Diamyd is evaluated in individuals diagnosed with LADA, providing important additional support for the safety and convenience of the therapy in a new indication as well as data that support the use of intralymphatic administration in individuals that are up to 70 years of age. A more thorough analysis of the clinical course, immune response and next steps including a potential pivotal trial in LADA will be possible once we have the 12 months results from GADinLADA trial later during 2022.

The quarter has seen advances in our patent portfolio where the Chinese patent office recently informed that they will approve patent claims that protect the intralymphatic administration mode of Diamyd. These key patent claims have already been approved among other countries, in Europe and Japan and provide protection until 2035. The Australian patent office also informed that they approve, as the first region, intralymphatic administration of other beta cell antigens beyond GAD, most importantly insulin. These broadened patent claims provide incentives to develop antigen specific therapies that complement GAD, which given the importance of precision medicine in type 1 diabetes, is of high interest. Insulin-based immunotherapies should likely be targeted to individuals that carry the HLA DR4-DQ8 haplotype while GAD-based therapies like Diamyd will be targeted to individuals that carry the HLA DR3-DQ2 haplotype.

With all activities ongoing that further strengthen the foundation of Diamyd Medical, we are fortunate to have a strong financial position; more than SEK 200 million in cash as of today will carry us well into 2023. I would like to thank the team and collaborators for your hard work and enthusiasm and our shareholders for helping us breaking new ground.

Stockholm, January 26, 2022
Ulf Hannelius, President and CEO

Significant events during the first quarter
September 1 – November 30, 2021

Diamyd Medical paused start of Phase III trial pending additional evaluations of current manufacturing process
Diamyd Medical decided to pause the start of the precision medicine Phase III trial DIAGNODE-3 with the diabetes vaccine Diamyd (GAD/alum) in recent onset type 1 diabetes, as well as the initiation of other clinical trials with Diamyd pending additional evaluations of the current manufacturing process. A new analysis has shown the potential presence of a contamination early in the manufacturing process of the drug substance (GAD65) used in the existing study drug. Diamyd Medical will evaluate its specific removal before additional clinical work is conducted. The potential contamination is not connected to the new manufacturing facility being established in Umeå.

Diamyd Medical announced to initiate its type 1 diabetes Phase III trial in Europe as discussions continue with FDA regarding partial clinical hold in the US
The FDA requested additional data to support Diamyd Medical’s IND application for DIAGNODE-3. Outstanding questions largely pertained to manufacturing of the study drug and needed to be addressed before FDA’s partial clinical hold for the Phase III trial in the US can be lifted. Given the delay this may entail, Diamyd Medical announced commence its initiation of the Phase III trial in Europe, while interactions with the FDA continue.

Diamyd Medical and partners were awarded SEK 40 million from VINNOVA for the prevention of autoimmune diseases
The Swedish governmental innovation agency VINNOVA awarded SEK 40 million in financing for an innovation milieu in sustainable precision health that will be led by Diamyd Medical. The project aims to develop and evaluate new algorithms based on artificial intelligence (AI) for preventive precision medical treatments for type 1 diabetes and other autoimmune diseases. The innovation milieu also includes Mainly AI AB, Lund University, Sahlgrenska University Hospital, the National Diabetes Register and the Leading Healthcare Foundation. Diamyd Medical’s part of the five- year grant amounts to approximately SEK 18 million.

First regulatory approval received to start the DIAGNODE-3
The Swedish Medical Products Agency gave approval for the start of DIAGNODE-3. The trial is designed to confirm the efficacy and safety of Diamyd in individuals recently diagnosed with type 1 diabetes, who carry the genetically defined haplotype HLA DR3-DQ2.

FDA paused the start of DIAGNODE-3 in the US
The start of DIAGNODE-3 in the United States was paused by the US Food and Drug Administration (FDA) to clarify certain outstanding questions regarding the study drug.

Acquisition of property with manufacturing facility in Umeå
Diamyd Medical announced the acquisition of the property in Umeå, Sweden, where production of the recombinant protein GAD65, the active component in the therapeutic diabetes vaccine Diamyd is being established. The property is acquired for a purchase price of SEK 24.5 million and comprises approximately 20 000 square feet including the 10 000 square feet Diamyd Medical AB rents, as well as 90 000 square feet of land area.

Proceeds of SEK 150 million were raised via a directed share issue
A directed share issue of 5 357 143 B-shares at a price of SEK 28 per share was completed. Through the directed share issue, the Company received gross proceeds of SEK 150 million. The directed share issue was subscribed by qualified investors.

Other events during the first quarter

Clinical results with Diamyd Medical’s study drug Remygen were published in a scientific journal
The clinical results from the dose-escalation part of the investigator-initiated clinical Phase I/II trial ReGenerate-1 evaluating Remygen (GABA) in individuals with long-standing type 1 diabetes, have been published in the scientific journal BMJ Open Diabetes & Care. The patent-pending findings showed, as previously announced, that Remygen established a counter-regulatory response to severely reduced blood sugar levels (hypoglycemia), indicating its potential use as a hypoglycemia-preventing treatment.

Analysis that supports the effect of the diabetes vaccine Diamyd was presented at the ISPAD conference
An analysis showing the effect of the diabetes vaccine Diamyd (GAD-alum) in reducing the time a patient has high blood glucose, was presented at the ISPAD conference (The International Society of Pediatric and Adolescent Diabetes), by Professor Johnny Ludvigsson, Principal Investigator of the clinical trial DIAGNODE-2.

Significant events after the first quarter

Interim report indicated similar immunological response in LADA-patients treated with intralymphatic Diamyd as in type 1 diabetes patients
An initial interim report from the open-label investigator-initiated clinical trial GADinLADA, in which the diabetes vaccine Diamyd is administered directly into the lymph node in 14 patients aged 30 to 70 years with the autoimmune form of diabetes called LADA (Latent Autoimmune Diabetes in Adults), showed that treatment after five months is safe and tolerable. Preliminary analyses indicated that the immunological response to the treatment is similar to what has been observed in individuals with type 1 diabetes treated with Diamyd.

Other events after the first quarter

Patent for intralymphatic injection of GAD in autoimmune diabetes to be granted in China
The patent that will be granted by the Chinese Patent Office is valid until 2035. The patent primarily protects the administration method of Diamyd (GAD/alum) which has shown positive results in the Phase I/II and Phase IIb trials DIAGNODE-1 and DIAGNODE-2 and will be evaluated in the Phase III trial DIAGNODE-3.

Australia granted as first region Diamyd Medical a broadening of a patent for intralymphatic injection of antigens in autoimmune diabetes
The patent covers intralymphatic injection of beta cell antigens including insulin for the treatment and prevention of autoimmune diabetes. The approval is a division of the patent application previously approved in Australia on intralymphatic injection of GAD, the active component in the diabetes vaccine Diamyd that Diamyd Medical is developing for type 1 diabetes and Latent Autoimmune Diabetes in Adults (LADA).

Two drugs in clinical development

Diamyd and Remygen are drugs in clinical development that focus on the underlying disease mechanisms of diabetes; the dysfunction and loss of insulin-producing beta cells in the pancreas.

Diamyd is an antigen-specific immunomodulating precision medicine diabetes vaccine for the treatment and prevention of autoimmune diabetes (type 1 diabetes and LADA, Latent Autoimmune Diabetes in Adults).

Clinical data indicate the potential of the diabetes vaccine Diamyd to halt or stop the autoimmune destruction of insulin-producing beta cells in individuals that carry the HLA DR3-DQ2 haplotype. The effect is achieved by antigen-specific reprogramming of immune cells by administration of low doses of Diamyd in superficial lymph nodes. By maintaining the endogenous insulin production, Diamyd has the potential to make a significant difference in the daily life of patients as well significantly reduce the complications of type 1 diabetes. Topline results from the Phase IIb trial DIAGNODE-2 demonstrated a significant treatment effect of Diamyd in the predefined genetic patient group.

Remygen is an oral regenerative and immunomodulatory drug candidate for the treatment of autoimmune- and type 2 diabetes. By stimulating the growth of insulin-producing cells, Remygen has the potential to reverse the disease progression in autoimmune- and type 2 diabetes. Based on clinical data, Remygen has also the potential to protect against hypoglycemia by improving the hormonal response. Remygen is now being investigated in a clinical Phase I/II trial (ReGenerate-1), where clinical efficacy is evaluated with the aim of optimizing the treatment regimen ahead of registration-based trials.

Clinical trials
Type 1 diabetes is a devastating disease which requires daily treatment with insulin to sustain life. The importance of finding a drug that improves the prospects for patients with diabetes is of utmost importance. The effect of intralymphatic administration of Diamyd, an antigen-specific precision medicine immunotherapy aimed at stopping the immune system’s attack on insulin-producing beta cells in autoimmune diabetes, will be evaluated in the Phase III trial DIAGNODE-3 and is evaluated in the Phase II trial GADinLADA.

Remygen, which aims to stimulate the growth of beta cells in patients with diabetes, is evaluated in patients in a Phase I/II trial.

Upcoming clinical trial

Trial with Diamyd in lymph node

DIAGNODE-3 – DIAMYD IN LYMPH NODES WITH ORAL SUPPLEMENTATION OF VITAMIN D
The placebo-controlled Phase III trial DIAGNODE-3 will include approximately 330 individuals aged 12 to 28 who have been recently diagnosed with type 1 diabetes and who carry the genetically defined haplotype HLA DR3-DQ2. The trial will be conducted at approximately 50 clinics, where almost half of all individuals with type 1 diabetes are estimated to carry the current haplotype. After an initial month in which all trial participants receive vitamin D, the individuals will be randomized 2:1, ie two out of three trial participants will receive three intralymphatic injections of Diamyd and one in three will receive the corresponding placebo at one month intervals, with one primary reading 24 months after trial start. The design provides, based on efficacy data from previous studies on the HLA-restricted patient population, a high probability of reaching the primary endpoints; preservation of stimulated C-peptide and lower HbA1c. The Coordinating Investigator for the trial is Professor Johnny Ludvigsson at Linköping University. The Sponsor of the trial is Diamyd Medical.

Ongoing clinical trials

Trial with Diamyd in lymph node

GADinLADA – DIAMYD IN LYMPH NODES WITH ORAL SUPPLEMENTATION OF VITAMIN D
The main aim of the trial is to evaluate the safety of intralymphatic treatment with Diamyd in patients with LADA (Latent Autoimmune Diabetes in Adults). The patients have been recruited in Norway at the Norwegian University of Science and Technology (NTNU) in Trondheim, in collaboration with St. Olavs Hospital, University Hospital in Trondheim, and in Sweden at the Center for Diabetes, Akademiskt specialistcentrum, an academic specialist unit run in collaboration between Stockholm County’s healthcare area, Karolinska Institutet and Karolinska University Hospital. The patients included in the trial are between 30 and 70 years old, have been diagnosed with LADA within the last 18 months and are not yet on insulin therapy. The Sponsor of the trial is the Norwegian University of Science and Technology with Ingrid K Hals as Sponsor’s representative. Diamyd Medical contributes with study drugs, expertise and some financial support for immunological analyzes and determination of HLA haplotypes. 12 month results are expected later in 2022.

Trial with Remygen (GABA)

REGENERATE-1 – REMYGEN /ALPRAZOLAM
An open-label, investigator initiated clinical trial with Remygen. The trial includes approximately 36 patients aged 18-50 who have had type 1 diabetes for more than five years with low to non-existing insulin production. Safety and initial efficacy results from the dose escalation section of the trial have paved the way to initiate the main trial and have also demonstrated a potential effect of Remygen to improve the hormonal response to hypoglycemia. The main trial evaluates whether the insulin-producing cells can be regenerated and if the hormonal response to hypoglycaemia can be improved using Remygen and the combination of Remygen and Alprazolam. The trial is led by Professor Per-Ola Carlsson at Uppsala University, Sponsor of the trial.

Manufacturing of GAD65 in Umeå
A new facility for vaccine manufacturing is being set up in Umeå, the Capital of Västerbotten County in Sweden, for the manufacture of recombinant GAD65, the active pharmaceutical ingredient in the therapeutic diabetes vaccine Diamyd currently in late-stage clinical development. The 10 000 square feet site, comprising of clean rooms, laboratory facilities and office space, will facilitate full control, predictability and scalability of the manufacturing technology of the active ingredient. Diamyd Medical has chosen Cytiva’s configurable single-use bioprocess manufacturing platform FlexFactory for the process. Small-scale experimental production of GAD65 is now established at the manufacturing facility. Large-scale production is being set up primarily using Cytiva equipment.

On January 26, 2022 Prokarium, a biopharmaceutical company pioneering the oncology field of microbial immunotherapy, reported that has entered into an agreement, through Imperial Projects, to work with independent researchers from Imperial College London to develop Prokarium’s next-generation therapeutic candidates, engineered to deliver payloads within the tumor microenvironment (Press release, Prokarium, JAN 26, 2022, View Source [SID1234606801]).

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"Prokarium is designing the perfect bacteria to be the next cancer immunotherapy, and we believe leveraging Salmonella’s unique biology will allow us to combine a powerful innate approach with a tailored adaptive response," said Kristen Albright, PharmD, Chief Executive Officer of Prokarium. "This agreement will significantly expand our R&D capabilities and will further deepen our immunotherapy pipeline."

This new multi-year agreement builds on work between Prokarium and London Biofoundry that was initiated in 2020 and will further expand Prokarium’s operations. The team from Imperial’s and SynbiCITE’s London Biofoundry specializes in the design, engineering and functional characterisation of synthetic DNA and organisms.

"Prokarium has uniquely positioned their Salmonella platform as an innovative immunotherapy approach for the treatment of solid tumors, and we are excited to support their efforts in pioneering the cancer treatment paradigm shift from synthetic chemistry to synthetic biology," said Marko Storch, PhD, Head of Synthetic Biology and Automation. "At the London Biofoundry, we developed a cutting-edge SynBio stack and the expertise to collaborate with researchers and biotechs to advance their R&D. We are thrilled to leverage our leading research capabilities to support the development of Prokarium’s new class of medicines in oncology."