Developing Precision Medicine Therapies for Type 1 Diabetes
Diamyd Medical’s B-share is traded on Nasdaq First North Growth Market under the ticker DMYD B.
Further information is available on https://www.diamyd.com
Figures in parentheses relate to the corresponding period (parent company) previous financial year.
September 1, 2021 – February 28, 2022
- Net result: MSEK -46.5 (120.0), second quarter: MSEK -9.0 (-15.1). The previous year contains a one-off effect of corresponding MSEK 144.4 from divestment of shares in Companion Medical, Inc. During the second quarter an additional milestone of corresponding MSEK 6.6 was received from Medtronic plc following the Companion Medical transaction.
- Result per share: SEK -0.6 (1.7), second quarter: SEK –0.1 (0.2)
- Cash flow from operating activities: MSEK -18.2 (-28.9), second quarter: MSEK -18.6 (-14.9)
- Cash and cash equivalents at February 28, 2022: MSEK 228.0 (165.5)
Significant events during the second quarter, December 1, 2021–February 28, 2022
- Diamyd Medical proceeded with the initiation of a Type 1 Diabetes Phase III trial in Europe
- Interim report indicated similar immunological response in LADA-patients treated with intralymphatic Diamyd® as in Type 1 Diabetes patients
Other events during the second quarter
- Clinical trial in Type 1 Diabetes with Diamyd Medical’s Remygen® was fully recruited
- Diamyd Medical patent for intralymphatic Diamyd® was granted in China
- Australia granted Diamyd Medical a broadening of patent for intralymphatic injection of antigens in autoimmune diabetes
Other events after the second quarter
- Diabetes prevention project coordinated by Diamyd Medical to be presented at scientific conference
- Diamyd Medical opened the first clinic in the Phase III trial DIAGNODE-3
Comments by CEO Ulf Hannelius
Two weeks ago, we announced the opening of the first contracted clinic in our Phase 3 trial for enrollment of patients. This is a significant milestone for Diamyd Medical.
The trial is the result of combining scientific advances in Type 1 Diabetes (T1D) with findings from numerous clinical trials evaluating the therapeutic vaccine Diamyd®. Most importantly, the DIAGNODE-3 trial design is based on large scale data analyses that support the statistical robustness and scientific rational of this pivotal trial, increasing the likelihood of clinical, regulatory and commercial success. Several clinics for patient enrollment are being opened as we speak in the countries where the trial is already approved, namely Sweden, Spain, the Netherlands, Poland, the Czech Republic and Germany.
DIAGNODE-3 is designed to confirm the safety and efficacy of Diamyd® to preserve endogenous insulin producing capacity in individuals with clinical T1D carrying the specific genetic HLA haplotype DR3-DQ2. About 50% of all individuals with T1D in Europe and the US carry this gene.
T1D is today categorized into stages (Stage 1, 2 and 3). Stage 1 T1D in an individual is defined by the individual having two different types of autoantibodies against diabetes specific antigens, most prominently GAD (glutamic acid decarboxylase) and insulin. Transition from Stage 1 to Stage 2 T1D takes place as the disease progresses and the individual’s blood glucose levels become abnormal but still manageable by the individual’s own remaining insulin production. At Stage 3 T1D, the individual’s own insulin producing capacity is gravely insufficient to control blood glucose levels, and the patient, now diagnosed with clinical T1D, is put on lifelong insulin treatment for survival.
The DIAGNODE-3 Phase 3 trial is aimed to improve the insulin producing capacity in individuals with Stage-3 T1D. However, it is anticipated that our Diamyd® therapeutic vaccine will also be effective in preventing Stage-1 and Stage-2 T1D to progress into Stage 3 T1D. To note is that prevention of clinically diagnosed T1D, in other words treatment of individuals with Stage 1 or Stage-2 diabetes, has already been evaluated in two smaller investigator initiated pilot trials, DiAPREV-IT-1 and -2. While the separate trials did not support clinical efficacy in the full trial population, a combined analysis of these trials, first announced on September 1, 2020, showed positive trends for an effect of Diamyd® on delaying the time to Stage 3 T1D in children carrying the HLA DR3DQ2 gene.
With these findings, and the regulatory framework around staging of diabetes, we are excited to coordinate the Swedish governmental innovation agency VINNOVA funded (MSEK 40) precision health milieu project ASSET (AI for Sustainable Prevention for Autoimmunity in Society). In ASSET, the aim is to evaluate prediction of T1D risk based on AI algorithms, as well as precision preventive treatments for T1D. As a crucial basis for the milieu, we have applied and been granted access by the NIDDK (National Institute of Diabetes and Digestive and Kidney Disease) to download anonymized data from the TEDDY study (The Environmental Determinants of Diabetes in the Young), a large cohort encompassing data from approximately 8,000 children with a family history of T1D who have been followed for up to 15 years from birth. These data are currently being processed and analyzed together with MainlyAI AB in which Diamyd Medical is an owner of 20% of the outstanding shares. We look forward to seeing this seminal project advance including prospective clinical prevention trials.
In T1D, as well as in other complex disorders, traditional approaches based on “one molecule fits all” have in many cases failed or shown limited benefit due to significant disease heterogeneity and individual variation in treatment response. To address these issues, the way forward is to identify responders using precision medicine approaches, patient stratification and data driven development. These approaches maximize the likelihood for clinical success and exposure to therapies that address the individual’s underlying disease.
Given this, we are proud that Diamyd Medical is at the forefront of the biotech industry with the first ever precision medicine Phase 3 trial in Type 1 Diabetes.
Stockholm, March 30, 2022
Ulf Hannelius, President and CEO
Significant events during the second quarter
December 1, 2021 – February 28, 2022
Diamyd Medical proceeded with initiation of a Type 1 Diabetes Phase III trial in Europe
The Company announced that the initiation of the Phase III trial DIAGNODE-3 in Europe with the Diamyd® diabetes vaccine is proceeded. Results from in-depth analyses of the manufacturing process showed no risk associated with a previously suspected contamination.
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 during the second quarter
Clinical trial in type 1 diabetes with Diamyd Medical's Remygen® was fully recruited
The investigator-initiated clinical trial ReGenerate-1 in Type 1 Diabetes with Diamyd Medical's investigational drug Remygen® (GABA) was fully recruited with 35 patients. The participants will be followed for up to 9 months. Results are expected to be presented in the first quarter of 2023.
Patent for intralymphatic injection of GAD in autoimmune diabetes was granted in China
The patent 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 is 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).
Other events after the second quarter
Diabetes prevention project coordinated by Diamyd Medical to be presented at scientific conference
The ASSET (AI for Sustainable Prevention of Autoimmunity in the Society) innovation milieu for the development of improved prevention strategies in Type 1 Diabetes, funded by the Swedish innovation agency VINNOVA, will be presented by PhD Ulf Hannelius, CEO of Diamyd Medical, at the Annual Meeting of the Scandinavian Society for the Study of Diabetes (SSSD), to be held in Reykjavík on May 12-13, 2022. The presentation will be given under the title “ASSET- a public/private partnership developing an integrated approach to prevention of Type 1 Diabetes (T1D)”.
Diamyd Medical opened the first clinic for the recruitment of patients for DIAGNODE-3
The first clinic opened in the precision medicine Phase III trial DIAGNODE-3. The trial, designed to confirm the efficacy and safety of the antigen-specific immunotherapy Diamyd®, will enroll approximately 330 patients aged 12 to 29 years recently diagnosed with type 1 diabetes and carrying the genetically defined HLA DR3-DQ2 haplotype.
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. A confirming Phase III trial, DIAGNODE-3, is on-going.
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.
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, are evaluated in the Phase III trial DIAGNODE-3 and 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.
Ongoing clinical trials
Trials 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.
- 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 35 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.
Results are expected in the first quarter of 2023.
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.
About Diamyd Medical
Diamyd Medical develops precision medicine therapies for type 1 diabetes. The diabetes vaccine Diamyd® is an antigen-specific immunotherapy for the preservation of endogenous insulin production. Significant results have been shown in a large genetically predefined patient group in a large-scale meta-analysis as well as in the Company’s European Phase IIb trial DIAGNODE-2, where the diabetes vaccine was administered directly into a lymph node in children and young adults with recently diagnosed type 1 diabetes. DIAGNODE-3, a confirmatory Phase III trial is on-going. A vaccine manufacturing facility is being set up in Umeå for the manufacture of recombinant GAD65, the active ingredient in the therapeutic diabetes vaccine Diamyd®. Diamyd Medical also develops the GABA-based investigational drug Remygen® as a therapy for regeneration of endogenous insulin production and to improve hormonal response to hypoglycaemia. An investigator-initiated Remygen® trial in individuals living with type 1 diabetes for more than five years is ongoing at Uppsala University Hospital. Diamyd Medical is one of the major shareholders in the stem cell company NextCell Pharma AB as well as in the artificial intelligence company MainlyAI AB.
Diamyd Medical’s B-share is traded on Nasdaq First North Growth Market under the ticker DMYD B. FNCA Sweden AB is the Company’s Certified Adviser; phone: +46 8-528 00 399, e-mail: firstname.lastname@example.org.
Further information is available on https://www.diamyd.com.
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