On May 15, 2020 Nimbus Therapeutics, a biotechnology company designing breakthrough medicines through structure-based drug discovery and development, reported the identification of an HPK1 inhibitor with highly potent and selective anti-tumor activity in preclinical models (Press release, Nimbus Therapeutics, MAY 15, 2020, View Source [SID1234558110]). The data will be presented at the AACR (Free AACR Whitepaper) Virtual Annual Meeting II, June 22-24, 2020.

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HPK1 (hematopoietic progenitor kinase 1) is a highly valued target in immuno-oncology due to its role as a regulator of both T cell and dendritic cell activity. However, a key challenge for development of small molecules acting on HPK1 has been to achieve selectivity against other T cell kinases and MAP4K family members. Nimbus utilized its unique structure-based drug discovery engine to identify multiple potent and selective small molecule inhibitors of HPK1. One of these molecules, advanced to in vivo testing, has high selectivity against T cell-specific kinases and kinases in the MAP4K family and exhibits promising activation of human T cells and B cells. In a mouse syngeneic tumor model, oral administration of the HPK1 inhibitor completely eliminated HPK1’s phosphorylation of the T cell receptor, enhanced inflammatory cytokine production, and demonstrated robust tumor growth inhibition.

"We’re excited to pull back the curtain on Nimbus’ HPK1 program and share some of the progress we’ve made against a target that has evaded so many others’ efforts," said Jeb Keiper, M.S., MBA, Chief Executive Officer of Nimbus. "Nimbus’ unparalleled expertise in structure-based drug discovery allowed us to chart an entirely new approach to inhibiting HPK1. In addition, we have recently leveraged this approach to generate small molecules against a range of promising new targets, and we look forward to sharing details on these programs in the coming weeks."

"These data support the potential of our HPK1 inhibitor to alter the tumor microenvironment to halt cancer’s immune evasion, which we think could be a powerful tool in today’s immuno-oncology arsenal," said Peter Tummino, Ph.D., Chief Scientific Officer of Nimbus. "We are advancing this program into IND-enabling studies, with the goal of entering the clinic next year, and ultimately providing a new therapeutic approach to address the large unmet need among patients with cancer."

On May 14, 2020 Isofol Medical AB’s (publ) (Nasdaq First North Premier Growth Market: ISOFOL) ("Isofol" or the "Company") Board of Directors reported that resolved on May 7, 2020, on a fully guaranteed new share issue of a maximum of 42,739,736 shares with preferential rights for the Company’s existing shareholders (the "Rights Issue") (Press release, Isofol Medical, MAY 14, 2020, View Source [SID1234561553]). Through the Rights Issue, the Company will receive approximately SEK 150 million before transaction costs related to the Rights Issue. In connection with the Rights Issue, the Company publishes a prospectus which today has been approved and registered by the Swedish Financial Supervisory Authority (Sw. Finansinspektionen).

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On May 14, 2020 Isofol Medical AB’s (publ) (Nasdaq First North Premier Growth Market: ISOFOL) ("Isofol" or the "Company") Board of Directors reported on May 7, 2020, on a fully guaranteed new share issue of a maximum of 42,739,736 shares with preferential rights for the Company’s existing shareholders (the "Rights Issue") (Press release, Isofol Medical, MAY 14, 2020, View Source [SID1234561552]). Through the Rights Issue, the Company will receive approximately SEK 150 million before transaction costs related to the Rights Issue. In connection with the Rights Issue, the Company publishes a prospectus which today has been approved and registered by the Swedish Financial Supervisory Authority (Sw. Finansinspektionen).

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On May 14, 2020 CellProtect Nordic Pharmaceuticals reported that results from the first-in-human study ACP-001 was made public at the 2020 EHA (Free EHA Whitepaper) meeting (Press release, CellProtect Nordic Pharmaceuticals, MAY 14, 2020, View Source [SID1234561095]).

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Key findings from the study:

Overall survival (OS) 100% after a median follow-up time of 60 months
Median progression free survival (PFS) 34 months
No serious adverse events
The present study demonstrates that autologous NK cell-based immunotherapy is feasible and demonstrates clinical applicability with efficacy responses in an upfront autologous HSCT-setting in multiple myeloma (MM). The treatment strategy opens for usage of autologous NK cells in clinical settings where patients are not readily eligible for allogeneic NK cell-based treatments, including MRD and maintenance treatment in MM and other forms of malignancies.

On May 14, 2020 Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs (the "Company" or "Regulus"), reported financial results for the first quarter ended March 31, 2020 and provided a corporate update (Press release, Regulus, MAY 14, 2020, View Source [SID1234561073]).

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"We have completed the second cohort and have initiated the dosing for the third and final cohort in the Phase 1 multiple ascending dose ("MAD") clinical study for RGLS4326" said Jay Hagan, CEO of Regulus. "Additionally, preparations for the Phase 1b study in patients with autosomal dominant polycystic kidney disease ("ADPKD") are well underway with plans to initiate in the second half of 2020."

Program Highlights

Initiated Dosing of the Third and Final Cohort in RGLS4326 Phase 1 for ADPKD: In April 2020, the Company initiated dosing of the third and final cohort of the MAD clinical study of RGLS4326, a novel oligonucleotide designed to inhibit miR-17 for the treatment of ADPKD. The Company expects to complete this study in mid-2020 with topline results available thereafter. The Company is planning to initiate a Phase 1b short-term dosing study in patients with ADPKD in the second half of 2020 to evaluate RGLS4326 for safety, pharmacokinetics, and biomarkers of pharmacodynamic activity.

Financial Results

Cash Position: As of March 31, 2020, Regulus had $28.1 million in cash and cash equivalents.

Research and Development (R&D) Expenses: R&D expenses were $3.1 million for the three months ended March 31, 2020, compared to $6.0 million for the same period in 2019. The aggregate decrease was driven by a $1.5 million reduction in personnel and internal expenses and a $1.0 million reduction in external development expenses, both of which were primarily attributable to a reduction in costs associated with the partial clinical hold of the RGLS4326 MAD study. In December 2019, the U.S. Food and Drug Administration ("FDA") lifted the partial clinical hold on the MAD study and it was recommenced in February 2020.

General and Administrative (G&A) Expenses: G&A expenses were $2.4 million for the three months ended March 31, 2020 compared to $3.5 million for the same period in 2019. These amounts reflect personnel-related and ongoing general business operating costs. The decrease is primarily attributable to continued cost reduction efforts subsequent to our corporate restructuring in the third quarter of 2018.

Revenue: Revenue was less than $0.1 million for the three months ended March 31, 2020. Revenue was $6.8 million for the three months ended March 31, 2019, attributable to revenue recognition of the upfront payments received under the 2018 Sanofi Amendment related to the transfer of the RG-012 program to Sanofi.

Net Loss: Net loss was $5.9 million, or $0.25 per share (basic and diluted), for the three months ended March 31, 2020, compared to $3.3 million, or $0.31 per share (basic and diluted), for the same period in 2019.

About ADPKD

ADPKD, caused by the mutations in the PKD1 or PKD2 genes, is among the most common human monogenic disorders and a leading cause of end-stage renal disease. The disease is characterized by the development of multiple fluid filled cysts primarily in the kidneys, and to a lesser extent in the liver and other organs. Excessive kidney cyst cell proliferation, a central pathological feature, ultimately leads to end-stage renal disease in approximately 50% of ADPKD patients by age 60.

About RGLS4326

RGLS4326 is a novel oligonucleotide designed to inhibit miR-17 and designed to preferentially target the kidney. Preclinical studies with RGLS4326 have demonstrated direct regulation of PKD1 and PKD2 in human ADPKD cyst cells, a reduction in kidney cyst formation, improved kidney weight/body weight ratio, decreased cyst cell proliferation, and preserved kidney function in mouse models of ADPKD. The RGLS4326 IND is currently on a Partial Clinical Hold for treatment of extended duration by the U.S. Food and Drug Administration until the second set of requirements outlined by FDA have been satisfactorily addressed. Information from the Phase 1 clinical studies, together with information from additional nonclinical studies, will be used to address the second set of requirements to support studies of extended duration.