On April 7, 2021 Chinook Therapeutics, Inc. (Nasdaq: KDNY), a biopharmaceutical company focused on the discovery, development and commercialization of precision medicines for kidney diseases, reported financial results for the full year ended December 31, 2020 (Press release, Aduro Biotech, APR 7, 2021, View Source [SID1234577678]).

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"We are executing well on our goal of building Chinook into a leading kidney disease company. 2020 was a very busy and productive year, as we in-licensed atrasentan from AbbVie, closed a $115 million financing, brought BION-1301 into our pipeline through the merger with Aduro, unveiled CHK-336, our first internally-developed program, and bolstered our precision medicine discovery and research efforts," said Eric Dobmeier, president and chief executive officer of Chinook Therapeutics. "We are excited to have recently initiated our atrasentan phase 3 ALIGN and phase 2 AFFINITY trials and announced our collaboration with Evotec. We look forward to multiple data announcements from our BION-1301 program this year, as well as continuing to move CHK-336 towards the clinic."

Mr. Dobmeier continued, "Our team has grown over 300 percent since the beginning of 2020, and we’re continuing to execute on our hiring plans to ensure we have strong resourcing in place to advance our pipeline. Our solid cash position, which we expect to fund our operations to the middle of 2023, enables us to achieve key milestones across our programs."

2020 and Recent Accomplishments

Atrasentan

Enrolled the first patient in the phase 2 AFFINITY basket trial of atrasentan, a highly potent and selective endothelin A receptor (ETA) antagonist (see www.clinicaltrials.gov, identifier NCT04573920). Chinook expects to report data from initial patient cohorts of this study in 2022.

Enrolled the first patient with IgA nephropathy in the phase 3 ALIGN trial of atrasentan, (see www.clinicaltrials.gov, identifier NCT04573478). Chinook expects to report top-line proteinuria data from this study in 2023, which could support accelerated approval from the FDA.

Delivered an oral presentation at the 3rd Annual Chronic Kidney Disease Drug Development (CKD3) Summit on selective ETA receptor antagonist atrasentan for the treatment of primary glomerular diseases.

Entered into a license agreement with Morehouse School of Medicine for patents supporting the development of therapies in kidney diseases that disproportionately affect people of West African descent and underserved communities, including focal segmental glomerulosclerosis (FSGS) and HIV-associated nephropathy (HIVAN).

Delivered a poster presentation at the American Society of Nephrology (ASN) Kidney Week 2020 Reimagined on the phase 3 ALIGN trial design for atrasentan.

Entered into a license agreement with AbbVie for worldwide, exclusive rights to atrasentan.
BION-1301

Completed enrollment and analysis of a phase 1 intravenous (IV) to subcutaneous (SC) bioavailability study of BION-1301, a novel anti-APRIL monoclonal antibody, in healthy volunteers.

Dosed the first patient with IgAN in Part 3 of the ongoing phase 1 study of BION-1301.

Delivered a poster presentation at the 57th ERA-EDTA Virtual Congress and ASN Kidney Week 2020 Reimagined on healthy volunteer data from Part 1 (single ascending dose) and Part 2 (multiple ascending dose) of the ongoing phase 1 study of BION-1301.

Delivered a poster presentation at the 57th ERA-EDTA Virtual Congress on nonclinical toxicology studies of BION-1301 evaluating IV administration for up to six months and SC administration for up to one month.
CHK-336

Received rare pediatric disease designation from the U.S. Food and Drug Administration (FDA) for CHK-336, an investigational oral small molecule inhibitor of lactate dehydrogenase A (LDHA), for primary hyperoxaluria (PH).

Delivered a preclinical poster presentation at the ASN Kidney Week 2020 Reimagined unveiling CHK-336 with the potential to treat all subtypes of PH and other disorders arising from excess oxalate.
Precision Medicine Research & Discovery

Participated in an expert panel discussion at the 3rd Annual CKD3 Summit on executing precision medicine in clinical trials.

Entered into a strategic collaboration with Evotec to discover and develop novel precision medicine therapies for polycystic kidney disease (PKD), lupus nephritis, IgAN and other primary glomerular diseases by leveraging the National Unified Renal Translational Research Enterprise (NURTuRE) patient biobank and Evotec’s proprietary PanHunter multi-omics platform.

Presented an oral abstract at the ASN Kidney Week 2020 Reimagined on a single cell transcriptomic atlas of human autosomal dominant polycystic kidney disease (ADPKD) through Chinook’s academic collaboration with the laboratory of Benjamin Humphreys, M.D., Ph.D., Joseph Friedman Professor of Renal Diseases in Medicine and Chief of Nephrology at Washington University School of Medicine in St. Louis.
Corporate

Appointed healthcare financial expert, Eric Bjerkholt, as chief financial officer.

Appointed the following life sciences industry veterans to the Board of Directors: William M. Greenman, president and chief executive officer of Cerus Corporation; Michelle Griffin, director and audit committee chair for Adaptive Biotechnologies, Acer Therapeutics and HTG Molecular Diagnostics, Inc.; Ross Haghighat, founder, chairman and managing partner of Triton Systems, Inc.; and Dolca Thomas, M.D., executive vice president, head of research and development and chief medical officer of Equillium, Inc.

Closed the merger with Aduro Biotech, Inc. on October 5, 2020 and began trading on the Nasdaq Global Select Market under the symbol "KDNY."

Completed a $115 million private placement financing with top-tier healthcare investors concurrent with the merger closing.
Anticipated Upcoming Catalysts

Chinook expects to present Gd-IgA1 biomarker data in healthy volunteers from Part 1 (single ascending dose) and Part 2 (multiple ascending dose) of the ongoing phase 1b study of BION-1301 at the ISN World Congress of Nephrology 2021 in April.

Chinook expects to present data from the BION-1301 phase 1 IV to SC bioavailability study in healthy volunteers at the ISN World Congress of Nephrology 2021 in April. Results from the study demonstrate the potential to transition to SC administration of BION-1301 in the long-term extension and phase 2 studies.

Part 3 of Chinook’s phase 1b study of BION-1301 is currently enrolling IgAN patients in an open-label setting, and Chinook expects to present a small subset of interim patient data in an oral presentation at the 58th ERA-EDTA Congress in June, as well as additional patient data at the ASN Kidney Week 2021 in November.

CHK-336 is currently in IND-enabling studies and advancing towards an expected IND submission in late 2021 or early 2022 for the treatment of primary hyperoxaluria.
Fourth Quarter and Full Year Financial Results

Cash Position – Cash, cash equivalents and marketable securities totaled $250.4 million at December 31, 2020, compared to $11.2 million at December 31, 2019.

Revenue – Total revenue increased by $0.8 million for both the fourth quarter of 2020 and year ended December 31, 2020 as compared to the fourth quarter of 2019 and year ended December 31, 2019. The increase was due to revenue recognized related to research and development services provided under the collaboration agreement with Lilly.

Expenses –

Research and development expenses were $21.8 million for the fourth quarter of 2020 and $36.1 million for the year ended December 31, 2020, compared to $9.2 million and $17.0 million, respectively, for the same periods in 2019. For the quarter and year ended December 31, 2020, the increases were primarily due to external clinical and manufacturing expenses related to the atrasentan and BION-1301 clinical programs; higher personnel expenses, including salaries, benefits and stock-based compensation expense associated with hiring staff to build out our clinical and development capabilities; and increased spending for consulting and outside services. The year-over-year increase was partially offset by expenses in the prior year period for the in-license of atrasentan, the purchase of intellectual property and know-how from a related party to support the CHK-336 program and discovery research activities.

General and administrative expenses were $11.0 million for the fourth quarter of 2020 and $19.1 million for the year ended December 31, 2020, compared to $0.7 million and $3.0 million, respectively, for the same periods in 2019. For the quarter and year ended December 31, 2020, costs increased primarily due to legal, consulting and accounting costs related to the merger; an increase in personnel costs, including salaries, benefits and stock-based compensation expense due to the addition of administrative staff to buildout our public-company infrastructure; and an increase in facilities and other costs.

Net Loss – Net loss for the fourth quarter of 2020 was $49.9 million or $1.24 per share and $81.6 million or $6.20 per share for the year ended December 31, 2020, compared to net loss of $34.2 million or $14.65 per share and $46.5 million or $25.48 per share, respectively, for the same periods in 2019.

Cash Used in Operations – For the fourth quarter ended December 31, 2020, cash used in operations totaled $41.3 million, of which $20.1 million were non-recurring expenses related to the merger and integration with Aduro Biotech.

On April 7, 2021 Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL) reported the successful closing of its license agreement with Eli Lilly and Company (Lilly), following the expiration of the waiting period under the Hart-Scott Rodino Antitrust Improvements Act of 1976 (Press release, Rigel, APR 7, 2021, View Source [SID1234577672]). Rigel and Lilly entered a global exclusive license agreement and strategic collaboration to co-develop and commercialize Rigel’s R552, a receptor-interacting serine/threonine-protein kinase 1 (RIPK1) inhibitor, for all indications including autoimmune and inflammatory diseases. Pursuant to the collaboration, Lilly will also lead all clinical development of penetrating RIPK1 inhibitors in central nervous system (CNS) diseases.

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The agreement is effective as of March 27, 2021 and Rigel has received the $125 million upfront cash payment due under the terms of the agreement from Lilly. Additional details about the collaboration can be found in Rigel’s Form 8-K filed with the Securities and Exchange Commission on February 18, 2021.

On April 7, 2021 Vincerx Pharma, Inc. (Nasdaq: VINC), a biopharmaceutical company aspiring to address the unmet medical needs of patients with cancer through paradigm-shifting therapeutics, reported that it will be hosting a key opinion leader (KOL) meeting on bioconjugation and CDK9 inhibitors for the treatment of hematologic and solid tumors on Friday, April 16, 2021 at 12:00 PM ET (Press release, Vincerx Pharma, APR 7, 2021, View Source [SID1234577673]).

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The event will feature presentations by KOLs Brian Druker, M.D., Knight Cancer Institute, and Anthony W. Tolcher, M.D., NEXT Oncology. Dr. Tolcher will discuss tackling normal tissue toxicity from antibody-drug conjugates (ADCs) and Dr. Druker will discuss CDK9 in hematologic malignancies. Drs. Druker and Tolcher will be available to answer questions following the formal presentations.

Vincerx’s management team will discuss its poster titled, "A novel small molecule drug conjugate -αvβ3 integrin antagonist linked to a cytotoxic camptothecin derivative- for the treatment of multiple cancer types," which will be presented at the upcoming American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, as well as the Company’s bioconjugation platform. Vincerx’s preclinical bioconjugation platform seeks to address the current limitations of small-molecule and antibody-drug conjugates in oncology and consists of VIP236, a small molecule drug conjugate (SMDC) targeting advanced and metastatic cancer, as well as VIP943 and VIP924, two ADCs targeting hematologic tumors.

KOLs
Brian Druker, M.D., is the director of the Knight Cancer Institute, associate dean for oncology of the OHSU School of Medicine and the JELD-WEN Chair of Leukemia Research. His research is focused on translating the knowledge of the molecular pathogenesis of cancer into specific therapies and investigating the optimal use of these molecularly targeted agents. He performed preclinical studies that led to the development of imatinib (Gleevec) for chronic myeloid leukemia (CML) and then spearheaded the highly successful clinical trials of imatinib, which led to FDA approval of the drug in record time. This work changed the life expectancy of patients with CML from an average of 3 to 5 years to a 95% five-year survival and has resulted in a paradigm-shift in cancer treatment from non-specific chemotherapy to highly targeted therapeutic agents. He is a member of the National Academy of Medicine, the National Academy of Sciences and, among numerous awards, is the recipient of the 2009 Lasker-DeBakey Clinical Medical Research Award, the 2012 Japan Prize in Healthcare and Medical Technology and the 2019 Sjöberg Prize.

Anthony W. Tolcher, M.D. is CEO and Founder of NEXT Oncology, San Antonio and Austin, Texas, Phase I group that seeks to transform early clinical trials. NEXT Oncology’s mission is to accelerate the next breakthrough medicines for cancer and the vision is to be the most successful and respected Phase I program in oncology research. Dr. Tolcher served as President and Co- Founder of START LLC from 2009- 2018, one of the world’s largest Clinical Phase I and early drug development operations in cancer medicine with 5 locations in San Antonio Texas; Grand Rapids, Michigan; Madrid Spain; and Shanghai China.

Dr. Tolcher is a medical oncologist who has over 25 years’ experience in early drug development and clinical trials. He has been involved in many of the initial phase I studies of new agents that subsequently were FDA approved for the treatment of cancer including pembrolizumab (Keytruda), copanlisib (Aliqopa), trastuzumab emtansine (Kadcyla), regorafenib (Stivarga), liposomal vincristine (Marqibo), cabazitaxel (Jevtana), carfilzomib (Kyprolis), gefitinib (Iressa), erlotinib (Tarceva), and eribulin (Halaven). He is currently the principal investigator on 40 phase I clinical studies, is a reviewer for the Journal of Clinical Oncology, Clinical Cancer Research, and Annals of Oncology. He has chaired the Developmental Therapeutics Review Committee for the American Association of Clinical Oncology Annual Scientific Program. Dr. Tolcher has over 100 peer-reviewed publications in scientific journals including Nature, Proceedings of the National Academy of Sciences (USA), Journal of Clinical Oncology, and Clinical Cancer Research, as well as an author of nine book chapters

On April 7, 2021 Soligenix, Inc. (Nasdaq: SNGX) (Soligenix or the Company), a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, reported that the U.S. Food and Drug Administration (FDA) has conditionally accepted HyBryte as the proposed brand name for SGX301 (synthetic hypericin), the Company’s novel first-in-class photodynamic therapy for first-line treatment of early stage cutaneous T-cell lymphoma (CTCL) (Press release, Soligenix, APR 7, 2021, View Source [SID1234577671]).

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The name HyBryte was developed in compliance with the FDA’s Guidance for Industry, Contents of a Complete Submission for the Evaluation of Proprietary Names. The FDA’s conditional approval validates HyBryte as a proprietary name that is consistent with the FDA’s goal of preventing medication errors and potential harm to the public by ensuring that only appropriate proprietary names are approved for use. Final approval of the HyBryte brand name is conditioned on FDA approval of the product candidate, SGX301.

"We are pleased that the FDA has conditionally accepted the name HyBryte for SGX301," stated Christopher J. Schaber, PhD, President and Chief Executive Officer of Soligenix. "This is yet another important step towards U.S. commercialization, as we continue to focus on submission of the rolling new drug application (NDA) in 2Q 2021 for this first-in-class therapy. SGX301 has already received Orphan Drug and Fast Track designations from the FDA. Additionally, SGX301 was granted Orphan Drug designation from the European Medicines Agency (EMA) and Promising Innovative Medicine designation from the Medicines and Healthcare products Regulatory Agency in the United Kingdom."

About Cutaneous T-Cell Lymphoma (CTCL)

CTCL is a class of non-Hodgkin’s lymphoma (NHL), a type of cancer of the white blood cells that are an integral part of the immune system. Unlike most NHLs which generally involve B-cell lymphocytes (involved in producing antibodies), CTCL is caused by an expansion of malignant T-cell lymphocytes (involved in cell-mediated immunity) normally programmed to migrate to the skin. These malignant cells migrate to the skin where they form various lesions, typically beginning as patches and may progress to raised plaques and tumors. Mortality is related to the stage of CTCL, with median survival generally ranging from about 12 years in the early stages to only 2.5 years when the disease has advanced. There is currently no cure for CTCL. Typically, CTCL lesions are treated and regress but usually return either in the same part of the body or in new areas.

CTCL constitutes a rare group of NHLs, occurring in about 4% of the approximate 700,000 individuals living with the disease. It is estimated, based upon review of historic published studies and reports and an interpolation of data on the incidence of CTCL that it affects over 25,000 individuals in the U.S., with approximately 3,000 new cases seen annually.

About HyBryte

HyBryte (SGX301) is a novel first-in-class photodynamic therapy utilizing safe visible light for activation. The active ingredient in HyBryte is synthetic hypericin, a potent photosensitizer that is topically applied to skin lesions, is taken up by the malignant T-cells, and then activated by fluorescent light 16 to 24 hours later. The use of visible light in the red-yellow spectrum has the advantage of penetrating more deeply into the skin (much more so than ultraviolet light) and therefore potentially treating deeper skin disease and thicker lesions. This treatment approach avoids the risk of secondary malignancies (including melanoma) inherent with the frequently employed DNA-damaging drugs and other phototherapy that are dependent on ultraviolet exposure. Combined with photoactivation, hypericin has demonstrated significant anti-proliferative effects on activated normal human lymphoid cells and inhibited growth of malignant T-cells isolated from CTCL patients. In a published Phase 2 clinical study in CTCL, patients experienced a statistically significant (p=0.04) improvement with topical hypericin treatment whereas the placebo was ineffective. HyBryte has received orphan drug and fast track designations from the FDA, as well as orphan designation from the European Medicines Agency (EMA).

The Phase 3 FLASH (Fluorescent Light Activated Synthetic Hypericin) trial enrolled a total of 169 patients (166 evaluable) with Stage IA, IB or IIA CTCL. The trial consisted of three treatment cycles. Treatments were administered twice weekly for the first 6 weeks and treatment response was determined at the end of the 8th week of each cycle. In the first double-blind treatment cycle, 116 patients received HyBryte treatment (0.25% synthetic hypericin) and 50 received placebo treatment of their index lesions. A total of 16% of the patients receiving HyBryte achieved at least a 50% reduction in their lesions (graded using a standard measurement of dermatologic lesions, the CAILS score) compared to only 4% of patients in the placebo group at 8 weeks (p=0.04) during the first treatment cycle (primary endpoint). HyBryte treatment in the first cycle was safe and well tolerated.

In the second open-label treatment cycle (Cycle 2), all patients received HyBryte treatment of their index lesions. Evaluation of 155 patients in this cycle (110 receiving 12 weeks of HyBryte treatment and 45 receiving 6 weeks of placebo treatment followed by 6 weeks of HyBryte treatment), demonstrated that the response rate among the 12-week treatment group was 40% (p<0.0001 vs the placebo treatment rate in Cycle 1). Comparison of the 12-week and 6-week treatment groups also revealed a statistically significant improvement (p<0.0001) between the two groups, indicating that continued treatment results in better outcomes. HyBryte continued to be safe and well tolerated. Additional analyses also indicated that HyBryte is equally effective in treating both plaque (response 42%, p<0.0001 relative to placebo treatment in Cycle 1) and patch (response 37%, p=0.0009 relative to placebo treatment in Cycle 1) lesions of CTCL, a particularly relevant finding given the historical difficulty in treating plaque lesions in particular.

The third (optional) treatment cycle (Cycle 3) was focused on safety and all patients could elect to receive HyBryte treatment of all their lesions. Of note, 66% of patients elected to continue with this optional compassionate use / safety cycle of the study. Of the subset of patients that received HyBryte throughout all 3 cycles of treatment, 49% of them demonstrated a treatment response (p<0.0001 vs patients receiving placebo in Cycle 1). Moreover, in a subset of patients evaluated in this cycle, it was demonstrated that HyBryte is not systemically available, consistent with the general safety of this topical product observed to date. At the end of Cycle 3, HyBryte continued to be well tolerated despite extended and increased use of the product to treat multiple lesions. Follow-up visits were completed in Q4 2020, and the clinical study report to support the NDA is in the process of being finalized.

Overall safety of HyBryte is a critical attribute of this treatment and was monitored throughout the three treatment cycles (Cycles 1, 2 and 3) and the 6-month follow-up period. HyBryte’s mechanism of action is not associated with DNA damage, making it a safer alternative than currently available therapies, all of which are associated with significant and sometimes fatal, side effects. Predominantly these include the risk of melanoma and other malignancies, as well as the risk of significant skin damage and premature skin aging. Currently available treatments are only approved in the context of previous treatment failure with other modalities and there is no approved front-line therapy available. Within this landscape, treatment of CTCL is strongly motivated by the safety risk of each product. HyBryte potentially represents the safest available efficacious treatment for CTCL. With no systemic absorption, a compound that is not mutagenic and a light source that is not carcinogenic, there is no evidence to date of any potential safety issues.

The Phase 3 CTCL clinical study was partially funded by the National Cancer Institute via a Phase II SBIR grant (#1R44CA210848-01A1) awarded to Soligenix, Inc.

On April 7, 2021 InDex Pharmaceuticals Holding AB (publ) reported that a patent covering 19 compounds from the company’s DIMS platform has been granted by the European Patent Office (Press release, InDex Pharmaceuticals, APR 7, 2021, View Source [SID1234577670]).

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InDex has a preclinical portfolio of more than 150 DNA-based ImmunoModulatory Sequences (DIMS), several of which are already protected by approved composition-of-matter patents. The new European patent, entitled Biologically active oligonucleotides capable of modulating the immune system (patent number 3165607), covers both the composition-of-matter and method-of-use of 19 different DIMS compounds for the treatment of inflammatory diseases, cancer and infectious diseases. The patent was filed in 2011 and provides an exclusivity period until December 2031, with the possibility of up to 5 years term extension after market approval.

"We continue the work to broaden our preclinical DIMS portfolio in parallel with the phase III clinical development of our lead drug candidate cobitolimod," said Peter Zerhouni, CEO of InDex Pharmaceuticals. "This patent has previously been granted in the US and Canada, and we are very pleased that also the European Patent Office confirms the novelty of our DIMS platform."

InDex’s DIMS compounds are synthetic oligonucleotides that function as immunomodulatory agents by targeting Toll-like receptor 9 (TLR9). DIMS mimic bacterial DNA, without being harmful, and stimulate immune cells to produce beneficial cytokines. This opens opportunities for the treatment of different immunological diseases, in which the immune responses are imbalanced. The company’s lead asset is the drug candidate cobitolimod, which is in late stage clinical development for the treatment of moderate to severe ulcerative colitis. Besides cobitolimod, InDex is testing a selected number of DIMS candidates in models of other inflammatory diseases. InDex has been awarded a grant of SEK 2.0 million for this development from the Swedish innovation agency Vinnova.