Medivir receives regulatory approval from MHRA for phase 1/2a combination study with MIV-818

On August 31, 2021 Medivir AB (Nasdaq Stockholm: MVIR) reported that the company has received regulatory approval from the British Medicines & Healthcare products Regulatory Agency (MHRA) for its upcoming phase 1/2a combination study with the company’s leading candidate drug MIV-818 against liver cancer (Press release, Medivir, AUG 31, 2021, View Source [SID1234587072]). In the study, MIV-818 will be administered in two combinations, either with lenvatinib, a tyrosine kinase inhibitor or pembrolizumab, an anti-PD-1 check-point inhibitor.

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The planned trial will be an open-label, multi-center phase 1/2a study starting with a dose escalation part to establish the recommended phase 2 dose (RP2D). This is followed by the expansion study (phase 2a) with an initial evaluation of the safety and efficacy of the combination of MIV-818 with lenvatinib or pembrolizumab. The study will include patients with hepatocellular carcinoma (HCC) who have progressed on, or are intolerant of, first line standard therapy.

The study is planned to have two parallel dose-escalation streams. Once the RP2D has been established for the combinations, further cohorts of up to 30 patients with HCC will be enrolled in the phase 2a part of the study. The study will start in the UK and is planned later to include centers in Spain and South Korea. The first patient is expected to be enrolled in the second half of 2021.

"It is satisfactory that the preparations for the study are progressing according to plan. The MHRA approval is also an important seal of quality in the planning and design of the study," said Magnus Christensen, Interim CEO of Medivir.

About MIV-818

MIV-818 is a pro-drug designed to selectively treat liver cancers and to minimize side effects. It has the potential to become the first liver-targeted and orally administered drug for patients with HCC and other forms of liver cancer. MIV-818 has completed a phase 1b monotherapy study, and a combination study in HCC is now planned to be initiated during the second half of 2021.

About primary liver cancer

Primary liver cancer is the third leading cause of cancer-related deaths worldwide and hepatocellular carcinoma (HCC) is the most common cancer that arises in the liver. Although existing therapies for advanced HCC can extend the lives of patients, treatment benefits are insufficient and death rates remain high. There are 42,000 patients diagnosed with primary liver cancer per year in the US and current five-year survival is 11 percent. HCC is a heterogeneous disease with diverse etiologies, and lacks defining mutations observed in many other cancers. This has contributed to the lack of success of molecularly targeted agents in HCC. The limited overall benefit, taken together with the poor overall prognosis for patients with intermediate and advanced HCC, results in a large unmet medical need.

Striving to Deliver Better Outcomes: Janssen to Showcase Commitment to Advancing Science for Genitourinary Cancers at AUA 2021

On August 31, 2021 The Janssen Pharmaceutical Companies of Johnson & Johnson reported multiple company-sponsored presentations in prostate and bladder cancers will be highlighted at the virtual 2021 American Urological Association Annual Meeting (AUA 2021), September 10-13 (Press release, Johnson & Johnson, AUG 31, 2021, View Source [SID1234587069]).

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"Janssen maintains a strong commitment to advancing innovation and new therapeutic options for patients with genitourinary malignancies. As the treatment of genitourinary cancers becomes more complex, we continue to work with urologists and their teams to improve outcomes for patients across the continuum of disease," said Craig Tendler, M.D., Vice President, Late Development and Global Medical Affairs, Janssen Research & Development, LLC. "We look forward to sharing the latest results from across our pipeline and portfolio at the upcoming AUA meeting and remain focused on accelerating science that unlocks new opportunities along the care pathway, from diagnosis to treatment of advanced disease."

Janssen will share four data presentations highlighting clinical advances for two therapies from our solid tumor portfolio.

ERLEADA studies to be presented at AUA include:

Real-World Effectiveness and Treatment Adherence in Non-Metastatic Castration-Resistant Prostate Cancer (nmCRPC) Patients (oral presentation): Real-world evidence from 63 urology practices across the U.S. detailing prostate-specific antigen (PSA) outcomes and treatment adherence among patients with nmCRPC treated with ERLEADA, with stratification by race (Abstract #PD05-08)
Prostate-Specific Antigen Kinetics in Patients from TITAN and SPARTAN (oral presentation): Post-hoc analysis of PSA kinetics in 1,331 patients treated with ERLEADA from both the TITAN and SPARTAN trials (Abstract #PD34-11)
Sites and Burden of Metastases and Long-Term Outcomes in TITAN Patients (moderated poster session): Assessment of relationships between the number and location of metastases and oncological outcomes in 1,052 patients with metastatic castration-sensitive prostate cancer (mCSPC) enrolled in the TITAN trial (Abstract #MP24-08)
Additionally, Janssen will present an update on the Phase 3 SunRISe-2 trial evaluating an investigational intravesical drug delivery system, TAR-200, in combination with the programmed cell death receptor-1 (PD-1) inhibitor cetrelimab in muscle-invasive urothelial carcinoma (Abstract # MP13-17).1

"As part of our commitment to help patients live longer and better lives, Janssen looks forward to expanding the focus from our legacy in prostate cancer to include other genitourinary cancers," said Serge Messerlian, President, Oncology, Janssen Biotech, Inc. "We’re collaborating with diverse urology stakeholders to understand and meet the challenges along the path to better care, leveraging clinical and operational excellence to strengthen our allyship with the entire urology community."

Further details about these data and the ways Janssen is working to shape the future of urologic care will be made available throughout AUA 2021 via the Janssen AUA Virtual Newsroom.

Abstracts to be presented at the meeting include:

Abstract No.

Title

Date/Time

ERLEADA (apalutamide)

Podium Presentations

Abstract #
PD05-08

Real-World Effectiveness and Treatment
Adherence of Apalutamide in Non-Metastatic
Castration-Resistant Prostate Cancer Patients

Friday,

September 10

8:10 AM – 8:20 AM
PST

Abstract #
PD34-11

Prostate-Specific Antigen Kinetics in Patients
with Advanced Prostate Cancer Treated with
Apalutamide: Results from the TITAN and
SPARTAN Studies

Saturday,

September 11

5:10 PM – 5:20 PM
PST

Moderated Poster

Abstract #
MP24-08

Relationships of Sites and Burden of
Metastases with Long-Term Outcomes and
Molecular Subtypes in TITAN

Saturday,

September 11

8:45 AM – 10:00
AM PST

TAR-200

Poster Presentation

Abstract #
MP13-17

SunRISe-2: A Phase 3, Multicenter,
Randomized Study Evaluating the Efficacy of
TAR-200 in Combination with Cetrelimab
Versus Concurrent Chemoradiotherapy in
Participants with Muscle-Invasive Urothelial
Carcinoma of the Bladder

Friday,

September 10

2:45 PM – 4:00 PM
PST

About ERLEADA
ERLEADA (apalutamide) is an androgen receptor inhibitor indicated for the treatment of patients with non-metastatic castration-resistant prostate cancer (nmCRPC) and for the treatment of patients with metastatic castration-sensitive prostate cancer (mCSPC).2 ERLEADA received U.S. FDA approval for nmCRPC in February 2018, and was approved for mCSPC in September 2019.2 To date, more than 40,000 patients worldwide have been treated with ERLEADA.

For more information, visit www.ERLEADA.com.

About TAR-200
TAR-200 is an investigational drug delivery system, enabling controlled release of gemcitabine into the bladder, increasing dwell time and local drug exposure. The safety and efficacy of TAR-200 is being evaluated in Phase 3 studies in patients with muscle-invasive bladder cancer (MIBC).

About Cetrelimab
Cetrelimab is an investigational programmed cell death receptor-1 (PD-1) monoclonal antibody being studied to treat MIBC, prostate cancer and multiple myeloma as a combination treatment.

ERLEADA IMPORTANT SAFETY INFORMATION 2
WARNINGS AND PRECAUTIONS

Cerebrovascular and Ischemic Cardiovascular Events — In a randomized study (SPARTAN) of patients with nmCRPC, ischemic cardiovascular events occurred in 4% of patients treated with ERLEADA and 3% of patients treated with placebo. In a randomized study (TITAN) in patients with mCSPC, ischemic cardiovascular events occurred in 4% of patients treated with ERLEADA and 2% of patients treated with placebo. Across the SPARTAN and TITAN studies, 5 patients (0.5%) treated with ERLEADA and 2 patients (0.2%) treated with placebo died from an ischemic cardiovascular event. Patients with history of unstable angina, myocardial infarction, congestive heart failure, stroke, or transient ischemic attack within 6 months of randomization were excluded from the SPARTAN and TITAN studies.

In the SPARTAN study, cerebrovascular events occurred in 4.7% of patients treated with ERLEADA and 0.8% of patients treated with placebo. In the TITAN study, cerebrovascular events occurred in 1.9% of patients treated with ERLEADA and 2.1% of patients treated with placebo. Across the SPARTAN and TITAN studies, 3 patients (0.2%) treated with ERLEADA, and 2 patients (0.2%) treated with placebo died from a cerebrovascular event.

Cerebrovascular and ischemic cardiovascular events, including events leading to death, occurred in patients receiving ERLEADA. Monitor for signs and symptoms of ischemic heart disease and cerebrovascular disorders. Optimize management of cardiovascular risk factors, such as hypertension, diabetes, or dyslipidemia. Consider discontinuation of ERLEADA for Grade 3 and 4 events.

Fractures — In a randomized study (SPARTAN) of patients with nmCRPC, fractures occurred in 12% of patients treated with ERLEADA and in 7% of patients treated with placebo. In a randomized study (TITAN) of patients with mCSPC, fractures occurred in 9% of patients treated with ERLEADA and in 6% of patients treated with placebo. Evaluate patients for fracture risk. Monitor and manage patients at risk for fractures according to established treatment guidelines and consider use of bone-targeted agents.

Falls — In a randomized study (SPARTAN), falls occurred in 16% of patients treated with ERLEADA compared with 9% of patients treated with placebo. Falls were not associated with loss of consciousness or seizure. Falls occurred in patients receiving ERLEADA with increased frequency in the elderly. Evaluate patients for fall risk.

Seizure — In 2 randomized studies (SPARTAN and TITAN), 5 patients (0.4%) treated with ERLEADA and 1 patient treated with placebo (0.1%) experienced a seizure. Permanently discontinue ERLEADA in patients who develop a seizure during treatment. It is unknown whether anti-epileptic medications will prevent seizures with ERLEADA. Advise patients of the risk of developing a seizure while receiving ERLEADA and of engaging in any activity where sudden loss of consciousness could cause harm to themselves or others.

Embryo-Fetal Toxicity — The safety and efficacy of ERLEADA have not been established in females. Based on its mechanism of action, ERLEADA can cause fetal harm and loss of pregnancy when administered to a pregnant female. Advise males with female partners of reproductive potential to use effective contraception during treatment and for 3 months after the last dose of ERLEADA [see Use in Specific Populations (8.1, 8.3)].

ADVERSE REACTIONS
Adverse Reactions — The most common adverse reactions (≥10%) that occurred more frequently in the ERLEADA-treated patients (≥ 2% over placebo) from the randomized placebo-controlled clinical trials (TITAN and SPARTAN) were fatigue, arthralgia, rash, decreased appetite, fall, weight decreased, hypertension, hot flush, diarrhea and fracture.

Laboratory Abnormalities — All Grades (Grade 3-4)

Hematology — In the TITAN study: white blood cell decreased ERLEADA 27% (0.4%), placebo 19% (0.6%). In the SPARTAN study: anemia ERLEADA 70% (0.4%), placebo 64% (0.5%); leukopenia ERLEADA 47% (0.3%), placebo 29% (0%); lymphopenia ERLEADA 41% (2%), placebo 21% (2%)
Chemistry — In the TITAN study: hypertriglyceridemia ERLEADA 17% (3%), placebo 12% (2%). In the SPARTAN study: hypercholesterolemia ERLEADA 76% (0.1%), placebo 46% (0%); hyperglycemia ERLEADA 70% (2%), placebo 59% (1%); hypertriglyceridemia ERLEADA 67% (2%), placebo 49% (0.8%); hyperkalemia ERLEADA 32% (2%), placebo 22% (0.5%)
Rash — In 2 randomized studies, rash was most commonly described as macular or maculopapular. Adverse reactions of rash were 26% with ERLEADA vs 8% with placebo. Grade 3 rashes (defined as covering >30% body surface area [BSA]) were reported with ERLEADA treatment (6%) vs placebo (0.5%).

The onset of rash occurred at a median of 83 days. Rash resolved in 78% of patients within a median of 78 days from onset of rash. Rash was commonly managed with oral antihistamines, topical corticosteroids, and 19% of patients received systemic corticosteroids. Dose reduction or dose interruption occurred in 14% and 28% of patients, respectively. Of the patients who had dose interruption, 59% experienced recurrence of rash upon reintroduction of ERLEADA.

Hypothyroidism — In 2 randomized studies (SPARTAN and TITAN), hypothyroidism was reported for 8% of patients treated with ERLEADA and 2% of patients treated with placebo based on assessments of thyroid-stimulating hormone (TSH) every 4 months. Elevated TSH occurred in 25% of patients treated with ERLEADA and 7% of patients treated with placebo. The median onset was at the first scheduled assessment. There were no Grade 3 or 4 adverse reactions. Thyroid replacement therapy, when clinically indicated, should be initiated or dose-adjusted.

DRUG INTERACTIONS

Effect of Other Drugs on ERLEADA — Co-administration of a strong CYP2C8 or CYP3A4 inhibitor is predicted to increase the steady-state exposure of the active moieties. No initial dose adjustment is necessary; however, reduce the ERLEADA dose based on tolerability [see Dosage and Administration (2.2)].

Effect of ERLEADA on Other Drugs
CYP3A4, CYP2C9, CYP2C19, and UGT Substrates — ERLEADA is a strong inducer of CYP3A4 and CYP2C19, and a weak inducer of CYP2C9 in humans. Concomitant use of ERLEADA with medications that are primarily metabolized by CYP3A4, CYP2C19, or CYP2C9 can result in lower exposure to these medications. Substitution for these medications is recommended when possible or evaluate for loss of activity if medication is continued. Concomitant administration of ERLEADA with medications that are substrates of UDP-glucuronosyl transferase (UGT) can result in decreased exposure. Use caution if substrates of UGT must be co-administered with ERLEADA and evaluate for loss of activity.

P-gp, BCRP or OATP1B1 Substrates — Apalutamide is a weak inducer of P-glycoprotein (P-gp), breast cancer resistance protein (BCRP), and organic anion transporting polypeptide 1B1 (OATP1B1) clinically. Concomitant use of ERLEADA with medications that are substrates of P-gp, BCRP, or OATP1B1 can result in lower exposure of these medications. Use caution if substrates of P-gp, BCRP or OATP1B1 must be co-administered with ERLEADA and evaluate for loss of activity if medication is continued.

Please see the full Prescribing Information for ERLEADA.

HUYABIO Files an Investigational New Drug Application with the FDA for the Novel SHP2 Inhibitor HBI-2376

On August 31, 2021 HUYABIO International (HUYABIO), the leader in accelerating global development of China’s pharmaceutical innovations, reported the filing of an investigational new drug application (IND) with the FDA for HBI-2376 along with Genhouse who has filed an IND with the Chinese Center for Drug Excellence CDE (Press release, HUYA Bioscience, AUG 31, 2021, View Source [SID1234587068]).

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"HBI-2376 (GH21) is a SHP2 allosteric inhibitor with very high selectivity. Both in vitro and in vivo studies have shown that HBI-2376 is effective against multiple SHP2 point mutations and has a very good PK and safety profile," said Dr. Keifung Wang, CEO of Genhouse. "Therefore, GH21 is a very promising small molecule drug candidate that Genhouse along with HUYABIO will coordinate the global clinical development to make it available for cancer patients as soon as possible."

The companies entered into a licensing agreement granting HUYABIO worldwide rights outside China to HBI-2376 prior to filing the regulatory submissions.

Dr. Mireille Gillings, CEO & Executive Chair of HUYABIO, said, "This submission represents a first filing of simultaneous INDs, to coordinate the development of our SHP2 inhibitor in both the US and China. We believe the drug’s global testing will accelerate its commercialization as an important new agent. It will add synergy to current immuno-oncology products which to date, have transformed cancer care. The potential here is to transform current immuno-oncology therapy to an even higher level and so improve public health."

About HBI-2376; SHP2 Inhibitor
HBI-2376 is an oral small molecule inhibitor of SHP2 for multiple tumor types whose cellular growth is dependent on the activity of receptor tyrosine kinases in the mitogen-activated protein kinase or MAPK pathway. Extensive biochemical characterization has shown that HBI-2376 is a highly potent and selective inhibitor of SHP2 phosphatase. Furthermore, preclinical investigations showed significant efficacy for HBI-2376 as a single agent or in combination with other small molecule inhibitors or checkpoint inhibitors in multiple tumor models.

ImmunoGenesis Announces Positive Preclinical Glioblastoma and Pancreatic Cancer Data for STimulator of INterferon Genes (STING) Agonist Published in Two Scientific Journals

On August 31, 2021 ImmunoGenesis Inc., a clinical-stage biotechnology company developing science-driven immune therapies, reported the publication of positive data for its STimulator of INterferon Genes (STING) agonist in the treatment of canines with previously diagnosed glioblastoma (GBM), the second most common type of canine brain cancer that shares very close similarities to its human counterpart, in Clinical Cancer Research, a journal of the American Association for Cancer Research (AACR) (Free AACR Whitepaper) (Press release, ImmunoGenesis, AUG 31, 2021, View Source [SID1234587067]). The study, "Intratumoral Delivery of STING Agonist Results in Clinical Responses in Canine Glioblastoma," showed that some canines responded to the treatment with reductions in their tumor volume, including one complete response in which the tumor completely disappeared. These results support the notion that ImmunoGenesis’ STING agonist has the potential to trigger a robust, innate anti-tumor immune response in humans and may be highly effective on recalcitrant tumors such as glioblastoma. The study was conducted jointly by ImmunoGenesis, Northwestern Medicine, and the Texas A&M College of Veterinary Medicine & Biomedical Sciences’ Veterinary Medical Teaching Hospital.

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In addition, ImmunoGenesis announces positive preclinical data implicating STING as a potential therapeutic target for patients with immune cold tumors, including pancreatic ductal adenocarcinoma (PDAC), in the publication of "High potency STING agonists engage unique myeloid pathways to reverse pancreatic cancer immune privilege," in the Journal for ImmunoTherapy of Cancer. Further, data revealed that intratumoral injection of ImmunoGenesis’ STING agonist into orthotopic pancreatic lesions unmasks sensitivity to checkpoint blockade, further indicating the potential of its STING agonist to help overcome immunotherapy resistance in cold tumors. This study also demonstrated that the high potency of ImmunoGenesis’ STING agonist could reprogram critical elements of the tumor stroma from an immune suppressive state to a pro-inflammatory state by engaging novel mechanisms such as downregulation of the "undruggable" cMyc oncogene – a property not found in naturally occurring STING agonists.

"These two published studies show strong preclinical proof of concept of our STING agonist against cold cancers—including pancreatic and brain cancers—refractory to currently available immunotherapy," said James Barlow, ImmunoGenesis President and CEO. "We look forward to further building upon these successful results with the advancement of our STING-ISAC candidate. Delivered intravenously, our STING-ISAC has the potential to deliver STING systemically to all tumor sites, allowing us to less-invasively target a broader range of cold tumors compared to free STING agonists."

"Collectively, these non-human data of ImmunoGenesis’ STING agonist indicate that it repolarizes the myeloid stroma to be immune supportive in addition to triggering a robust, innate anti-tumor response," said Dr. Amy Heimberger, scientific director of the Malnati Brain Tumor Institute of the Robert H. Lurie Comprehensive Cancer Center of Northwestern University Feinberg School of Medicine. "This further reveals the potential for ImmunoGenesis’ STING agonist to be a foundational treatment in immunologically resistant cancers such as GBM."

Canine Study in Glioblastoma

The investigators tested the STING agonist by injection directly into the glioblastoma of five dogs that had previously been diagnosed with the cancer. Each dog received up to two injections intratumorally at an interval of four to six weeks. MRI scans taken of the canines over the course of the trial revealed that some of the canines, even with single dose, responded to the treatment with reductions in their tumor volume, including one complete response in which the tumor completely disappeared. Funding for this study was provided by the National Institutes of Health (grant R01 NS120547), the Joan Traver Walsh Family Foundation, the Dr. Marnie Rose Foundation, the Brockman Foundation and Mr. Herb Simmons.

Preclinical Pancreatic Cancer Study

The second study sought to describe how different STING agonists functionally impact the tumor microenvironment using PDAC as a model for an immunologically cold tumor. Two natural STING agonists (CDG and cGAMP) and two synthetic STING agonists (ML-RR and ImmunoGenesis’ 8803) were tested, with 8803 being the most potent. Local delivery of ImmunoGenesis’ 8803 STING agonist triggered proinflammatory remodeling of immune suppressive cells within the tumor microenvironment, expanded immune cell infiltration and induced tumor regression. Intratumoral administration of 8803 also augmented the response to checkpoint blockade and induced curative immunity in a multifocal PDAC model. Funding for this study was primarily provided by PanCAN.

About STING-ISAC

STING-ISAC builds on ImmunoGenesis’ novel platform PD-L1/PD-L2 inhibitor by conjugating a STING agonist to the antibody, combining an optimal PD-1 pathway blockade with a powerful immune agonist. ImmunoGenesis is developing this agent to effectively and systemically transport the intravenously delivered STING agonist to all tumor sites and targets within the tumor microenvironment. This therapeutic advance pushes through an important barrier seen with traditional STING agonists, which consistently produce an effect only at the site of the intratumoral injection. ImmunoGenesis’ STING-ISAC, delivered intravenously, could precisely target where it is most effective across tumor sites.

Avidity Biosciences to Participate in Upcoming Investor Conferences

On August 31, 2021 Avidity Biosciences, Inc. (NASDAQ: RNA) a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs), reported that the Avidity management team will be participating at the following conferences (Press release, Avidity Biosciences, AUG 31, 2021, View Source [SID1234587066]):

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2021 Wells Fargo Virtual Healthcare Conference
September 9th, 2021
11:20am ET – Fireside Chat

Chardan Virtual 5th Annual Genetic Medicines Conference
October 5th, 2021
12noon ET – Panel titled "RNA-based Targeted Delivery Approaches"
1:30pm ET – Fireside Chat

Live webcasts of each event, as well as an archived replay of the webcasts following each event, will be available on the "Events and Presentations" page in the "Investors" section of Avidity’s website at View Source