• First healthy volunteer cohort cleared in Phase 1 first-in-human study of BION-1301 for the treat­ment of IgA nephropathy (IgAN); initial data in healthy volunteers ex­pec­ted in H1 2020 and IgAN patients in H2 2020
• Results from the dose escalation portion of the Phase 1/2 study in re­lapsed or refractory multiple myeloma (MM) patients whose disease progressed after at least three prior ther­a­pies indicated BION-1301 was well tolerated across a wide dose range and no dose limiting toxicities (DLTs) were observed
• 95% target engagement achieved and main­tained; no objective responses observed in the MM patient setting
• Phase 1 dose escalation data in MM to be presented at the 2019 ASCO Annual Meeting

Berkely, CA (Press Release) – Aduro Biotech, Inc. (NASDAQ: ADRO), a clin­i­cal-stage bio­pharma­ceu­tical com­pany focused on devel­op­ing ther­a­pies targeting the Stimulator of Interferon Genes (STING) and A Proliferation Inducing Ligand (APRIL) path­ways for the treat­ment of cancer, auto­immune and inflammatory diseases, today provided an update on its clin­i­cal devel­op­ment pro­grams for BION-1301, a first-in-class humanized IgG4 mono­clonal anti­body that fully blocks APRIL binding to both the BCMA and TACI re­cep­tors.

“We believe the clin­i­cal poten­tial for BION-1301 is rooted in scientific rationale and we will pursue the devel­op­ment of BION-1301 in the IgAN indi­ca­tion, where we believe there is sig­nif­i­cant oppor­tu­ni­ty to address unmet patient need. We are pleased to ini­ti­ate this study as a first step in eval­u­ating BION-1301’s ability to reduce the auto­immune deposition of immune complexes affecting kidney function, and its poten­tial to be­come a meaningful treat­ment option for this progressive kidney disease,” said Stephen T. Isaacs, chairman, pres­i­dent and chief exec­u­tive officer of Aduro. “However, with the emergence of transformative thera­peutic options for multiple myeloma, we have de­ter­mined not to con­tinue the current Phase 1/2 study as designed in this setting. Although no objective responses were observed, we believe there is poten­tial for investigator-sponsored trials of BION-1301 in com­bi­na­tion with other approved agents, given the excellent tolerability profile and PK-PD data.”

BION-1301 in IgA Nephropathy

The first cohort of healthy volunteers has been cleared in a Phase 1 multi­center study (see www.clinicaltrials.gov, identifier: NCT03945318) designed to eval­u­ate the safety, tolerability, phar­ma­co­ki­netics and pharmacodynamics of BION-1301 in healthy volunteers and adults with IgAN. The study, which will enroll up to 63 healthy subjects and up to 10 subjects with IgAN, will be conducted in three parts: Part 1 is a double-blind, ran­dom­ized, placebo-controlled, single ascending dose in healthy volunteers; Part 2 is a double-blind, ran­dom­ized, placebo-controlled multiple ascending dose in healthy volunteers; and Part 3 is an open-label, multiple dose in subjects with IgAN.

“There are cur­rently no approved thera­peutic options for IgAN patients. We believe BION-1301’s mode of action may target the pro­duc­tion of galactose-deficient IgA as well as the autoantibody response to IgA, two key steps in the disease etiology preceding the for­ma­tion of immune complexes in the kidney and sub­se­quent loss of renal function,” said Andrea van Elsas, Ph.D., chief scientific officer of Aduro. “In addi­tion to assessing the safety profile and PK-PD rela­tion­ship of BION-1301 in healthy volunteers and IgAN patients in this study, we are looking to estab­lish­ proof-of-mechanism.”

Preclinical studies have dem­onstrated that BION-1301 binds to a specifically defined epitope on APRIL, resulting in com­plete blockade of APRIL-induced re­cep­tor activation. Dosing of BION-1301 in non-human primates led to a sig­nif­i­cant reduction of blood IgA levels and estab­lish­ed a favorable safety profile. Preclinical studies dem­onstrated that hAPRIL transgenic mice produce rising levels of IgA as well as IgA deposits in the kidney. Admin­istra­tion of mouse anti-human APRIL was shown to reduce levels of IgA in both the serum and the kidney.

BION-1301 in Relapsed/Refractory Multiple Myeloma

Aduro com­pleted the dose escalation portion of its Phase 1/2 study eval­u­ating safety and tolerability in patients with re­lapsed or refractory MM whose disease had progressed after at least three prior sys­temic ther­a­pies. Results dem­onstrated that BION-1301 was well tolerated in patients with re­lapsed or refractory MM across a wide dose range and no DLTs were observed at any dose levels. While 95% target engagement was achieved at peak exposure levels and levels of free APRIL de­creased dose-dependently, no objective responses were observed.

Aduro does not in­tend to proceed to the Phase 2 portion of the ongoing Phase 1/2 study or ini­ti­ate further com­pany-sponsored studies in the MM patient setting. Aduro is work­ing closely with thought leaders to assess the direction of the BION-1301 MM pro­gram, in­­clud­ing through poten­tial investigator-sponsored studies.

Two abstracts highlighting data from the Phase 1/2 study will be presented on Monday, June 3 at the 2019 ASCO Annual Meeting in Chicago, IL. Details of the poster presentations are as follows:

To view these and other Aduro abstracts, please visit the ASCO website at http://abstracts.asco.org/.

About IgA Nephropathy

IgA nephropathy is a progressive kidney disease char­ac­ter­ized by an auto­immune anti­body response against endogenous immuno­glob­u­lin A (IgA). The accumulation of autoantibodies binding to galactose-deficient IgA leads to deposition of immune complexes in the glomeruli of the kidneys. This results in local inflammation impeding kidney function, which may eventually lead to end-stage renal disease and the need for renal trans­plant. While IgA nephropathy has unclear causality and lacks approved disease-modifying options for ther­apy, pre­clin­i­cal data indicate that sup­pressing serum IgA1 and the anti-IgA auto­immune response by a neutralizing APRIL anti­body, such as BION-1301, may reduce the pro­gres­sion and severity of proteinuria and other symp­toms.

About BION-1301

BION-1301 is a first-in-class humanized IgG4 mono­clonal anti­body that fully blocks APRIL binding to both the BCMA and TACI re­cep­tors, and is being devel­oped as a novel ther­apy for IgA nephropathy. BION-1301 has been shown in pre­clin­i­cal studies to reduce serum IgA levels in mice and monkeys.

About Aduro

Aduro Biotech, Inc. is a clin­i­cal-stage bio­pharma­ceu­tical com­pany focused on the discovery, devel­op­ment and com­mer­cial­iza­tion of ther­a­pies that are designed to harness the body’s natural immune sys­tem for the treat­ment of patients with chal­leng­ing diseases. Aduro’s prod­uct can­di­dates in the Stimulator of Interferon Genes (STING) and A Proliferation Inducing Ligand (APRIL) path­ways are being in­ves­ti­gated in cancer, auto­immune and inflammatory diseases. ADU-S100 (MIW815), which poten­tially activates the intracellular STING re­cep­tor for a potent tumor-specific immune response, is being eval­u­ated in patients with cutaneously accessible metastatic solid tumors or lym­phomas. BION-1301, a first-in-class humanized IgG4 mono­clonal anti­body that fully blocks APRIL binding to both the BCMA and TACI re­cep­tors, is being eval­u­ated in IgA nephropathy. Aduro is col­lab­o­rating with a number of leading global pharma­ceu­tical com­pa­nies to help expand and drive its prod­uct pipe­line. For more in­for­ma­tion, please visit www.aduro.com.

Cautionary Note on Forward-Looking Statements

This press release con­tains forward-looking state­ments for pur­poses of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking state­ments in­clude state­ments re­gard­ing our intentions or current ex­pec­ta­tions con­cern­ing, among other things, the poten­tial for BION-1301 to be­come a meaningful treat­ment option for IgAN and address unmet patient need, the mech­a­nism of action for BION-1301 in treat­ment of IgAN, in­­clud­ing the ability of BION-1301 to target galactose-deficient IgA and the autoantibody response to IgA and to reduce auto­immune immune complex deposition affecting kidney function, our ability to estab­lish­ proof of mech­a­nism of BION-1301 as a treat­ment option for IgAN in the Phase 1 study, the direction of BION-1301 for treat­ment of MM, in­­clud­ing any poten­tial investigator sponsored studies of BION-1301 alone or with other approved agents for treat­ment of MM, the timing and volume of clin­i­cal data, and our ability to expand and drive our prod­uct pipe­line alone or with our col­lab­o­rators. In some cases, you can identify these state­ments by forward-looking words such as “may,” “will,” “continue,” “anticipate,” “intend,” “could,” “project,” “expect” or the neg­a­tive or plural of these words or similar ex­pres­sions. Forward-looking state­ments are not guar­an­tees of future per­for­mance and are subject to risks and un­cer­tain­ties that could cause actual results and events to differ ma­teri­ally from those antic­i­pated, in­­clud­ing, but not limited to, early or pre­lim­i­nary clin­i­cal trial results may not be predictive of future results, our history of net operating losses and uncertainty re­gard­ing our ability to achieve profitability, our ability to develop and com­mer­cial­ize our prod­uct can­di­dates, our ability to use and expand our tech­nolo­gies to build a pipe­line of prod­uct can­di­dates, our ability to obtain and main­tain regu­la­tory approval of our prod­uct can­di­dates, our ability to operate in a competitive industry and compete suc­cess­fully against com­pet­i­tors that have greater resources than we do, our reliance on third parties, and our ability to obtain and adequately protect intellectual property rights for our prod­uct can­di­dates. We discuss many of these risks in greater detail under the heading “Risk Factors” con­tained in our quarterly report on Form 10-Q for the quarter ended March 31, 2019, which is on file with the Securities and Exchange Com­mis­sion. Any forward-looking state­ments that we make in this press release speak only as of the date of this press release. We assume no obli­ga­tion to update our forward-looking state­ments whether as a result of new in­for­ma­tion, future events or other­wise, after the date of this press release.

Source: Aduro Biotech.