On March 25, 2020 Diamyd Medical Reported that Quarterly Report II 19/20 (Press release, Diamyd Medical, MAR 25, 2020, View Source;ClipID=3608512 [SID1234555808]).

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September 1, 2019 – February 29, 2020

Net result: MSEK 30.3 (-19.0), whereof second quarter MSEK 37.1 (-10.4). The increase is a one-off effect due to a payment of corresponding MSEK 48.0 from the previous GAD65 manufacturer as support for transition of the manufacturing process.
Result per share: SEK 0.4 (-0.3), second quarter SEK 0.5 (-0.2)
Cash flow from operating activities: MSEK 31.1 (-19.5), second quarter: MSEK 37.2 (-8.7)
Cash and cash equivalents at February 29, 2020: MSEK 88.3 (79.6)

Significant events second quarter, December 2019 – February 2020

DIAGNODE-1: Positive Top-line results from Phase I/II trial with intralymphatic Diamyd
ReGenerate-1: The main part of the Phase I/II trial with Remygen in type 1 diabetes started
Significant effect of Diamyd in Type 1 diabetes shown in comprehensive analysis of previous phase III and phase II trials

Significant events after the reporting period

GADinLADA: New trial with Diamyd in autoimmune diabetes started recruiting patients in Norway

Comments by CEO Ulf Hannelius
The last weeks we have seen the Corona virus disease (COVID-19) outbreak creating turmoil in the financial markets and affecting the global economy. Given the prevailing times, we are fortunate at Diamyd Medical to have solid finances and a strong position with the diabetes vaccine Diamyd, our lead candidate.

First, SEK 88 million in cash and a current monthly burn rate of approximately SEK 3 million give us, with ongoing activities, a runway of at least two years. We will do our best to prioritize our resources wisely and to strengthen the foundation of the company, maximizing the value of our assets.

Second, our DIAGNODE-2 Phase IIb trial with Diamyd, was fully recruited already by May 2019, and all patients received their three injections long before the viral outbreak. We are now working closely with our partners to make sure to smooth operations as we are moving closer to finalizing the trial later this year.

Third, it is important to know that the antigen-specific immunotherapy Diamyd does not weaken the immune system, opposed to immunosuppressive treatments that may increase the risk of infections by viral or other pathogens and/or predispose the patient to complications due to these infections. The safety profiles of both Diamyd and our second clinical asset Remygen are clear strengths, and in these times and going forward this may prove to become one of the most crucial advantages for Diamyd Medical.

Fourth, with the comprehensive responder analysis announced in December last year, we have improved the probability of success for Diamyd. The results, which identified the genetics for the best Diamyd responders, were in agreement with the thoughts on precision medicine that were independently proposed in a recent scientific review (Introducing the Endotype Concept to Address the Challenge of Disease Heterogeneity in Type 1 Diabetes, Diabetes Care, January, 2020).

Last but not least, the DIAGNODE-2 results will be important in forming the final design of the pivotal Phase III program, and with the results from the responder analysis now at hand we have the confidence to move ahead in our preparations for building the commercial case for Diamyd.

Stockholm, March 25, 2020
Ulf Hannelius, President and CEO

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 immunotherapy for the treatment of autoimmune diabetes (type 1 diabetes).

Clinical data indicate the potential of the diabetes vaccine Diamyd to halt or stop the autoimmune destruction of insulin-producing beta cells. 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.

Intralymphatic treatment with Diamyd is now being investigated in a clinical Phase IIb trial (DIAGNODE-2), with the aim of confirming the previously demonstrated clinical effect from a pilot trial in type 1 diabetes patients (DIAGNODE-1).

Remygen is an oral regenerative and immunomodulatory therapy 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.

Remygen is now being investigated in a clinical Phase I/II trial (ReGenerate-1), where clinical efficacy is evaluated with the aim of optimizing treatment ahead of registration-based trials.

Significant events during the second quarter

December 1, 2019 – February 29, 2020

Diamyd Medical entered agreement to facilitate transition of manufacturing

Diamyd Medical and Protein Sciences Corporation (PSC) entered into an agreement modifying their relationship, by which PSC will continue to support Diamyd Medical in its development of a diabetes vaccine but will no longer serve as its contract manufacturer. The agreement facilitates the transition of the manufacturing process of recombinant GAD65, the active ingredient in the diabetes vaccine Diamyd, to a new manufacturer for future anticipated commercial manufacture. PSC will support Diamyd Medical with in cash, as well as certain raw materials and up to 150 man-hours of technical assistance for the transition process. The agreement has been made in light of the announcement by Diamyd Medical in October 2019 where PSC had informed Diamyd Medical that it was unable to meet Diamyd Medical’s manufacturing needs for commercial supply. The payment was made in February 2020.

Positive Top-line results from Phase I/II trial with intralymphatic Diamyd

When all 12 patients had been followed throughout the 30-month period in the open-label trial DIAGNODE-1, the patients showed on average a positive clinical course with a near normal long-term blood sugar and a low need for externally supplied insulin. The three patients who received an extra Diamyd injection into the lymph node after their 30-month visit showed a maintained own insulin production between the 30- and 43-month visits, as well as lower long-term blood sugar and insulin requirements compared to baseline. Safety looked good and no serious side effects had been reported.

The main part of the Phase I/II trial ReGenerate-1 with Remygen started

Four patients had been included in the trial and additional patients were scheduled to be included in December and January. Compilation of metabolic results from the completed safety and dose escalation part of the trial awaits a final experimental analysis and is expected to be announced during the second quarter of 2020.

Significant effect of Diamyd in Type 1 diabetes shown in a comprehensive analysis of previous Phase III and Phase II trials

A new analysis based on data from more than 530 individual patients from previous Phase III and II trials in Europe and US with the diabetes vaccine Diamyd identified genetically defined subgroups of type 1 diabetes patients that showed a positive and statistically significant dose-dependent treatment response.

Significant events after the reporting period

New trial with Diamyd in autoimmune diabetes started recruiting patients in Norway

GADinLADA, the first clinical phase II trial with the diabetes vaccine Diamyd administered directly into the lymph node in patients with LADA started recruitment at the Norwegian University of Science and Technology in Trondheim (NTNU), in cooperation with St. Olav’s University Hospital, Trondheim. The trial will also be conducted in Sweden at the Center for Diabetes, the Academic Specialist Center, an academic specialist unit that is run in collaboration between the Health Care Services Stockholm County, Karolinska Institute and Karolinska University Hospital, and the recruitment is expected to start during the spring. In total, the trial encompasses 15 patients between the ages of 30 to 70 years diagnosed with LADA within the last 12 months who are not yet on insulin therapy.

Ongoing 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 immunotherapy aimed at stopping the immune system’s attack on insulin-producing beta cells in autoimmune diabetes, is evaluated in the Phase IIb trial DIAGNODE-2. Remygen, which aims to stimulate the growth of beta cells in patients with diabetes, is now evaluated in patients in a Phase I/II trial.

Trials with Diamyd in lymph node

DIAGNODE -2 – DIAMYD IN LYMPH NODES WITH ORAL SUPPLEMENTATION OF VITAMIN D
A follow-up double-blind randomized clinical trial where Diamyd is administered directly into a lymph node with oral supplements of vitamin D. The trial encompasses 109 patients from Sweden, the Czech Republic, Spain and the Netherlands, aged 12–24 years who have recently been diagnosed with type 1 diabetes and will continue for a total of 15 months. As of autumn 2019, those patients who have not performed their last visit at 15 months are invited to participate in a nine months extension of the trial. 15-month results are expected to be presented in the third quarter of 2020. The aim of the trial is to evaluate the patients’ remaining insulin producing capacity. Coordinating Investigator is Professor Johnny Ludvigsson at Linköping University, Sweden. Diamyd Medical is the Sponsor of the trial.

GADinLADA – DIAMYD IN LYMPH NODES WITH ORAL SUPPLEMENTATION OF VITAMIN D
An open-label, investigator initiated clinical trial where Diamyd is administered directly into a lymph node with oral supplements of vitamin D. The trial encompasses 15 patients aged 30-70 years diagnosed with LADA (Latent Autoimmune Diabetes in Adults) and not yet on inulin treatment. The aim with the trial is to evaluate the safety of intralymphatic treatment with Diamyd in LADA patients and to continuously evaluate the immunological and clinical response during a one-year period. Sponsor of the trial is the Norwegian University of Science and Technology with Ingrid K Hals as sponsor representative.

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. The primary aim of the trial is to in a smaller dose escalation section evaluate the safety of Remygen. The main trial also evaluates whether the insulin-producing cells can be regenerated using Remygen, and in the combination of Remygen and Alprazolam. The trial is led by Professor Per-Ola Carlsson at Uppsala University, Sponsor of the trial.

Other ongoing trial with Diamyd

DiAPREV-IT 2 – COMBINING DIAMYD WITH VITAMIN D
A placebo-controlled investigator-initiated clinical trial, where Diamyd is given subcutaneously and being tested in combination with vitamin D in children at high risk of developing type 1 diabetes, meaning that they have been found to have an ongoing autoimmune process but do not yet have any clinical symptoms of diabetes. The trial includes 26 children and results are expected in Q2 2020. The aim of the trial is to evaluate whether Diamyd can delay or prevent the participants from presenting with type 1 diabetes. The trial is led by Dr. Helena Elding Larsson at Lund University, Sweden, Sponsor of the trial.

On March 24, 2020 AXIM Biotechnologies, Inc. (OTCQB: AXIM) ("AXIM Biotech," "AXIM" or "the Company"), an international healthcare solutions company targeting oncological and cannabinoid research, reported that its subsidiary Sapphire Biotech, Inc. ("Sapphire") has completed pre-clinical drug studies on a new compound, SPX-1009, demonstrating 10 times greater potency than its parent compound, SBI-183, in inhibiting metastasis (Press release, Sapphire BioTech, MAR 24, 2020, https://sapphirebiotech.com/axim-biotechnologies-completes-pre-clinical-drug-studies-on-new-compound-proving-tenfold-greater-inhibition-of-tumor-metastasis-than-parent-compound/?utm_source=rss&utm_medium=rss&utm_campaign=axim-biotechnologies-completes-pre-clinical-drug-studies-on-new-compound-proving-tenfold-greater-inhibition-of-tumor-metastasis-than-parent-compound [SID1234558651]).

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"Cancer is a leading cause of death worldwide and distant metastases are the major cause of patient mortality," said John W. Huemoeller II, Chief Executive Officer of AXIM Biotech. "Our scientists at Sapphire are aiming to slow down this death rate through their emphasis on metastasis and these positive early findings are nothing but spectacular."

Quiescin Sulfhydryl Oxidase 1 (QSOX1), an enzyme that is over-expressed by tumor cells in numerous cancers, has been shown to be involved in cancer invasion and metastasis. Earlier this year, Sapphire acquired exclusive license rights to a small molecule, SBI-183. SBI-183 has been proven to inhibit the enzymatic activity of QSOX1 and suppress tumor cell invasion in vitro and metastasis of MDA-MB-231 breast tumor cells in vivo. In a non-metastatic xenograft of kidney cancer, SBI-183 suppressed primary kidney tumor growth by 51 percent and in a metastatic xenograft breast cancer model (MDA-MB-231-luc TNBC), SBI-183 reduced lung metastasis by 76 percent compared to control.

A photo accompanying this announcement is available at View Source

Sapphire scientists have tested over 70 analogs of SBI-183 and discovered that SPX-1009 was tenfold more potent in comparison to SBI-183, in suppressing tumor invasion and metastasis in vitro. SPX-1009 was first screened in a cell-free enzymatic assay for its ability to inhibit QSOX1 and then tested for its ability to inhibit growth and invasion of triple-negative breast cancer (TNBC) cells (MDA-MB-231), sarcomatoid kidney cancer cells (RCJ-41T2) and pancreatic adenocarcinoma cells (MIAPaCa2). MTT assays were used to measure the effect of the compounds on tumor growth. 2D invasion assays and 3D tumor spheroid assays were employed to measure the effect of the compounds on invasion.

Testing results demonstrated in vitro that SPX-1009 suppressed invasion of breast, kidney and pancreas tumor cells in 2D and 3D invasion assays at tenfold lower concentrations than the parent compound SBI-183.

A photo accompanying this announcement is available at View Source

Sapphire intends to initiate animal studies to demonstrate the greater potency of SPX-1009 to suppress tumor growth and metastasis in mice bearing MDA-MB-231-luc breast cancer orthotopic xenografts. Sapphire’s intellectual property related to the SBI-183 and SPX-1009 technology is the subject of numerous patent-pending applications.

"We are extremely excited with the results that the in vitro testing showed," said Catalina Valencia, Chief Executive Officer of Sapphire Biotech. "This potential therapeutic treatment could someday help control tumor cell proliferation and metastasis with our inhibitor."

On April 17, 2020 Rafael Pharmaceuticals, Inc. (‘Rafael’ or the ‘Company’), a leader in the growing field of cancer metabolism-based therapeutics, reported that it has enrolled more than 75% of the 500 patients needed for its pivotal Phase 3 clinical trial for metastatic pancreatic cancer (AVENGER 500), which is evaluating the efficacy and safety of Rafael’s lead compound CPI-613️ (devimistat) in combination with modified FOLFIRINOX (mFFX) as first-line therapy (Press release, Rafael Pharmaceuticals, MAR 24, 2020, View Source [SID1234556415]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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‘We are continuing to enroll patients ahead of schedule, as the patients in our trials suffer from life-threatening diseases and are in dire need of treatment; patient safety is our top priority,’ said Sanjeev Luther, President and CEO of Rafael Pharmaceuticals. ‘In order to support our clinical trial sites and enrolled patients during this unsettling and unprecedented time, we have established a COVID-19 task force to closely monitor our clinical trials and make changes as needed. We want to express our deepest gratitude to all of the principal investigators, nurses, hospital staff, patients and their families for their continued support and cooperation.’

‘To Save a Life is to Save a Universe’ – Rafael’s motto – represents its dedication to patients who suffer from rare cancers globally, as well as to its employees and the communities it serves. As such, the company has implemented measures to help ensure the continuity of its clinical trials and programs, while maintaining the safety of patients, clinicians and employees. The COVID-19 task force will closely monitor all clinical trials and collect data relevant to the patients’ study treatments and schedules.

Rafael is working closely with the clinical trial sites to preempt and promptly address any potential challenges and ensure that clinical supplies are available in sufficient quantities in order to support the continuity of care of all enrolled patients. Each clinical trial site has implemented their own safety measures based on guidelines from local authorities. If patients have questions about their clinical trial, they should reach out to the clinical trial coordinator at their medical center.

About CPI-613 (devimistat)
CPI-613 (devimistat) is a first-in-class clinical lead compound of Rafael, which targets enzymes that are involved in cancer cell energy metabolism and are located in the mitochondria of cancer cells. Devimistat is designed to target the mitochondrial tricarboxylic acid (TCA) cycle, a process essential to tumor cell multiplication and survival, selectively in cancer cells. Devimistat substantially increases the sensitivity of cancer cells to a diverse range of chemotherapeutic agents. This synergy allows for potential combinations of devimistat with lower doses of these generally toxic drugs to be more effective with lower patient’s side effects. Combination with devimistat represent a diverse range of opportunities to substantially improve patient’s benefit in many different cancers. The U.S. Food and Drug Administration (FDA) has given Rafael approval to initiate pivotal Phase 3 clinical trials in pancreatic cancer (AVENGER 500) and acute myeloid leukemia (ARMADA 2000), and has designated devimistat as an orphan drug for the treatment of pancreatic cancer, acute myeloid leukemia, myelodysplastic syndrome, peripheral T-cell lymphoma and Burkitt’s lymphoma. The EMA has granted orphan drug designation to devimistat for pancreatic cancer and acute myeloid leukemia.

On April 17, 2020 Rafael Pharmaceuticals, Inc. (‘Rafael’ or the ‘Company’), a leader in the growing field of cancer metabolism-based therapeutics, reported that it has enrolled more than 75% of the 500 patients needed for its pivotal Phase 3 clinical trial for metastatic pancreatic cancer (AVENGER 500), which is evaluating the efficacy and safety of Rafael’s lead compound CPI-613️ (devimistat) in combination with modified FOLFIRINOX (mFFX) as first-line therapy (Press release, Rafael Pharmaceuticals, MAR 24, 2020, View Source [SID1234556415]).

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Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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‘We are continuing to enroll patients ahead of schedule, as the patients in our trials suffer from life-threatening diseases and are in dire need of treatment; patient safety is our top priority,’ said Sanjeev Luther, President and CEO of Rafael Pharmaceuticals. ‘In order to support our clinical trial sites and enrolled patients during this unsettling and unprecedented time, we have established a COVID-19 task force to closely monitor our clinical trials and make changes as needed. We want to express our deepest gratitude to all of the principal investigators, nurses, hospital staff, patients and their families for their continued support and cooperation.’

‘To Save a Life is to Save a Universe’ – Rafael’s motto – represents its dedication to patients who suffer from rare cancers globally, as well as to its employees and the communities it serves. As such, the company has implemented measures to help ensure the continuity of its clinical trials and programs, while maintaining the safety of patients, clinicians and employees. The COVID-19 task force will closely monitor all clinical trials and collect data relevant to the patients’ study treatments and schedules.

Rafael is working closely with the clinical trial sites to preempt and promptly address any potential challenges and ensure that clinical supplies are available in sufficient quantities in order to support the continuity of care of all enrolled patients. Each clinical trial site has implemented their own safety measures based on guidelines from local authorities. If patients have questions about their clinical trial, they should reach out to the clinical trial coordinator at their medical center.

About CPI-613 (devimistat)
CPI-613 (devimistat) is a first-in-class clinical lead compound of Rafael, which targets enzymes that are involved in cancer cell energy metabolism and are located in the mitochondria of cancer cells. Devimistat is designed to target the mitochondrial tricarboxylic acid (TCA) cycle, a process essential to tumor cell multiplication and survival, selectively in cancer cells. Devimistat substantially increases the sensitivity of cancer cells to a diverse range of chemotherapeutic agents. This synergy allows for potential combinations of devimistat with lower doses of these generally toxic drugs to be more effective with lower patient’s side effects. Combination with devimistat represent a diverse range of opportunities to substantially improve patient’s benefit in many different cancers. The U.S. Food and Drug Administration (FDA) has given Rafael approval to initiate pivotal Phase 3 clinical trials in pancreatic cancer (AVENGER 500) and acute myeloid leukemia (ARMADA 2000), and has designated devimistat as an orphan drug for the treatment of pancreatic cancer, acute myeloid leukemia, myelodysplastic syndrome, peripheral T-cell lymphoma and Burkitt’s lymphoma. The EMA has granted orphan drug designation to devimistat for pancreatic cancer and acute myeloid leukemia.

On March 24, 2020, Kura Oncology, Inc. (the "Company") entered into an Office Lease (the "Lease") with East Office Operating Limited Partnership ("Landlord") for the lease of approximately 16,541 square feet of rentable area of the building located at Two Sea Port Lane, Boston, Massachusetts (the "Premises"). The commencement date of the Lease is April 1, 2020. The Company expects to use the Premises for general office use. The initial term of the Lease (the "Initial Term") is four years and three months and the Company has one option to extend the Lease for a period of five additional years. The minimum rent payable by the Company under the Lease will be approximately $105,500 per month for the first year of the Lease, which amount will increase by 2.0% per year over the Initial Term. The Company will also be responsible for the payment of additional rent to cover the Company’s share of the annual operating expenses of the building, the annual tax expenses of the building, the annual utilities costs for the building and parking. In the event of a default of certain of the Company’s obligations under the Lease, Landlord would have the right to terminate the Lease and recover certain unpaid rent and expenses.

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The foregoing description of the Lease does not purport to be complete and is subject to, and qualified in its entirety by reference to, the full text of the Lease. The Company intends to file a copy of the Lease with the Company’s Quarterly Report on Form 10-Q for the quarter ended March 31, 2020.