Mustang Bio soars on its gene therapy data

NEJM reports ‘medical breakthrough’ in Mustang Bio cell and gene therapy

Mustang Bio (MBIO) announced that the New England Journal of Medicine has published data from St. Jude Children’s Research Hospital, the nation’s “leading hospital” for understanding, treating and curing childhood cancer and other life-threatening diseases.

Mustang Bio soars on its gene therapy data, Stockwinners

The data comes from a Phase 1/2 clinical trial of a lentiviral gene therapy for the treatment of newly diagnosed infants under two years old with XSCID, also referred to as SCID-X1 and commonly known as “bubble boy disease.”

Under a licensing agreement with St. Jude, Mustang will develop the lentiviral gene therapy for commercial use as MB-107.

The multi-center Phase 1/2 clinical trial is evaluating the safety and efficacy of a lentiviral vector to transfer a normal copy of the IL2RG gene to bone marrow stem cells in newly diagnosed infants under the age of two with XSCID, preceded by low exposure-targeted busulfan conditioning.

A total of 10 infants have received the therapy to date in this clinical trial. Among the data highlights, bone marrow harvest, busulfan conditioning and cell infusion were well tolerated.

In seven of the eight cases, normalization of naive T-cell and natural killer cell numbers occurred within three to four months after treatment, accompanied by vector marking in T, B, NK and myeloid cells and marrow progenitors.

All patients cleared previous infections and are growing normally. Seven of the eight infants treated have developed normal IgM levels to date.

Most patients were discharged from the hospital within one month.

Data Highlights:

  • Bone marrow harvest, busulfan conditioning and cell infusion were well tolerated.
  • In seven of the eight cases, normalization of CD3+, CD4+ and CD4+ naïve T-cell and natural killer (“NK”) cell numbers occurred within three to four months after treatment, accompanied by vector marking in T, B, NK and myeloid cells and marrow progenitors.
    • The eighth infant had insufficient T cells initially, but normalization of T cells occurred following an unconditioned boost of gene-corrected cells, and the patient is progressing favorably.
  • All patients cleared previous infections and are growing normally.
  • Seven of the eight infants treated have developed normal IgM levels to date.
    • Four of these seven infants have discontinued monthly infusions of intravenous immunoglobulin (IVIG) therapy to date.
    • Three of those four infants that discontinued monthly IVIG infusions have responded to vaccines to date.

MBIO closed at $2.66, it last traded at $8.91.

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Bio-Path Holdings presents positive pancreatic cancer data

Bio-Path Holdings presents BP1003 data at AACR

Bio-Path Holdings presents positive pancreatic cancer data, Stockwinners

Bio-Path Holdings (BPTH) announced that data from pre-clinical studies supporting the potential of BP1003, a liposome-incorporated STAT3 oligodeoxynucleotide inhibitor, for the treatment of pancreatic cancer, non-small cell lung cancer, or NSCLC, and acute myelogenous leukemia, or AML, were presented in a poster at the American Association for Cancer Research, or AACR.

The poster highlights four antisense oligo sequences directed against STAT3 mRNA identified by Bio-Path and manufactured using DNAbilize antisense RNAi nanoparticle technology.

Cell viability tests and western blots were conducted to determine the inhibitory effects of liposome-incorporated STAT3 antisense oligo on NSCLC and AML cells.

An ex vivo live tissue sensitivity assay was performed with a panel of 20 pancreatic ductal adenocarcinoma patient-derived xenografts to study the overall activity of BP1003 alone, and in combination with gemcitabine.

Using previously defined criteria, tissue slice viability inhibition greater than 30% and with a less than 0.05 value was considered to be a response.

For validation of ex vivo results, tumor bearing mice were administered BP1003 and gemcitabine twice a week for 28 days. Tumor volumes were monitored for up to 49 days.

The most potent liposome-incorporated STAT3 antisense sequence in decreasing NSCLC cell viability was selected as the drug candidate BP1003.

Further validation in AML cells demonstrated that BP1003 inhibited cell viability and STAT3 protein expression. In the ex vivo LTSA assay, BP1003 at a dose of 10 microM significantly inhibited the tissue slice viability in 9 out of 18 PDAC PDXs by more than 30%.

The combination of BP1003 and gemcitabine further enhanced ex vivo efficacy of BP1003 in a subset of PDXs.

In the in vivo study, a combination of BP1003 and gemcitabine caused tumor regression during the 28-day drug treatment period.

This anti-cancer activity was maintained for another 21 days, even when drug treatment had ceased.

Preclinical pancreatic cancer models demonstrated that BP1003 successfully penetrated the stroma into pancreatic tumors.

Finally, the results in pancreatic cancer showed that BP1003 inhibited tumor slice viability in nine of 18.

BPTH closed at $19.68, it last traded at $20.61.

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Advaxis jumps on its prostate cancer drug data

Advaxis says ADXS-PSA in combination with KEYTRUDA prolonged survival in mCRPC

Advaxis jumps on its prostate cancer drug data, Stockwinners

Advaxis (ADXS) announced updated data from the Phase 1/2 KEYNOTE-046 study in metastatic, castration-resistant prostate cancer.

Castrate-resistant prostate cancer refers to prostate cancer that keeps growing even when the amount of testosterone in the body is reduced to very low levels. Many early-stage prostate cancers need normal levels of testosterone to grow, but castrate-resistant prostate cancers do not. 

Androgens are required for normal growth and function of the prostate. Androgens are also necessary for prostate cancers to grow. Androgens promote the growth of both normal and cancerous prostate cells by binding to and activating the androgen receptor, a protein that is expressed in prostate cells Once activated, the androgen receptor stimulates the expression of specific genes that cause prostate cells to grow .

Early in their development, prostate cancers need relatively high levels of androgens to grow. Such prostate cancers are called castration sensitive, androgen dependent, or androgen sensitive because treatments that decrease androgen levels or block androgen activity can inhibit their growth.

This trial is being conducted in conjunction with Merck (MRK) and is evaluating ADXS-PSA, one of Advaxis’ Listeria monocytogenes-based immunotherapies, alone and in combination with KEYTRUDA, Merck’s anti-PD-1 therapy.

Findings will be highlighted in a poster discussion entitled “Effects of ADXS-PSA with or without Pembrolizumab on Survival and Antigen Spreading in Metastatic, Castration-Resistant Prostate Cancer Patients” at the American Association for Cancer Research Annual Meeting underway in Atlanta.

KEYNOTE-046 is an open-label, multicenter, dose-determining safety and tolerability Phase 1/2 trial of 50 heavily pretreated patients conducted in two parts, with a Phase 2 expansion cohort. The objective of the study is to evaluate ADXS-PSA alone and in combination with KEYTRUDA for primary endpoints that include safety, tolerability and dosing.

Secondary endpoints include anti-tumor activity and progression-free survival, and exploratory endpoints include associations between biomarkers of immunologic response with clinical outcomes.

Key findings from the combination arm of KEYNOTE-046 include the following: The majority of treatment-related adverse events consisted of transient and reversible Grade 1-2 chills/rigors, fever, hypotension, nausea and fatigue.

The combination of ADXS-PSA and pembrolizumab has been well-tolerated, to date, with no additive toxicity observed. Median overall survival was 21.1 months at data cutoff in this dataset of 37 patients.

Correlative immune analyses showed T-cell responses against PSA in 75% of subjects and antigen spreading in 85% of subjects. Broader immune stimulation, including B-cell activation, was observed in the combination arm than in the ADXS-PSA monotherapy arm.

ADXS closed at $5.91, it last traded at $8.15. MRK closed at $83.17.

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Axovant higher on data

Axovant jumps following update on Tay-Sachs Disease gene therapy program

Axovant higher on gene data, Stockwinners

Shares of Axovant (AXGT) have surged higher today following the company’s report of three-month data from an investigator-initiated study administering investigational AXO-AAV-GM2 gene therapy in a patient with advanced infantile Tay-Sachs disease, a rare and fatal pediatric eurodegenerative genetic disorder.

Tay-Sachs disease (TSD) is a fatal genetic disorder, most commonly occurring in children, that results in progressive destruction of the nervous system. Tay-Sachs is caused by the absence of a vital enzyme called hexosaminidase-A (Hex-A). Without Hex-A, a fatty substance, or lipid, called GM2 ganglioside accumulates abnormally in cells, especially in the nerve cells of the brain. This ongoing accumulation causes progressive damage to the cells.

In children, the destructive process begins in the fetus early in pregnancy. However, a baby with Tay-Sachs disease appears normal until about six months of age when its development slows. By about two years of age, most children experience recurrent seizures and diminishing mental function. The infant gradually regresses, and is eventually unable to crawl, turn over, sit or reach out. Eventually, the child becomes blind, cognitively impaired, paralyzed and non-responsive. By the time a child with Tay-Sachs is three or four years old, the nervous system is so badly affected that death usually results by age five.

The study is evaluating a total dose of 1.0x 1014 vg of AXO-AAV-GM2 in a 30-month-old child with advanced infantile Tay-Sachs disease.

AXO-AAV-GM2 was administered into the cisterna magna and lumbar spinal canal only.

Due to the patient’s advanced disease, a co-delivered intrathalamic injection of AXO-AAV-GM2 was not administered.

Future patients in the program, who are expected to be treated earlier in their disease course, will receive AXO-AAV-GM2 co-delivered into the thalamus bilaterally as well as into the cisterna magna and spinal canal.

“AXO-AAV-GM2 was generally well-tolerated and no serious adverse events have been reported as of the 3-month visit. At 3 months, no clinically relevant laboratory abnormalities were observed following AXO-AAV-GM2 administration.

The patient’s clinical condition was stable from baseline to month 3 without clinical deterioration observed on neurological exam.

Furthermore, there was no significant deterioration in the condition from the pre-treatment magnetic resonance imaging of the brain at baseline to the post-treatment MRI at month 3,” the company announced in press release earlier this morning.

Additionally, Chardan has upgraded Axovant to Buy from Neutral following the data report.

In morning trading, Axovant shares are up 46c, or 31%, to $1.94.

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Bristol-Myers comments on Celgene’s purchase

Bristol-Myers sees ‘meaningful financial benefits’ from Celgene transaction.

Bristol-Myers treatment for colorectal cancer approved, Stockwinners

Bristol Meyers Comments on Celgene purchase, Stockwinners

Bristol-Myers Squibb (BMY) said an updated its investor presentation about the Celgene (CELG) transaction.

The company said, “The Celgene transaction is the natural next step in Bristol-Myers Squibb’s proven strategy that has consistently delivered results for over a decade.

Through a disciplined approach to driving innovation, focusing on high-value opportunities and sourcing innovation externally to complement its internal portfolio and pipeline, Bristol-Myers Squibb has generated consistently strong growth and increased its dividend for 10 consecutive years.

The combination with Celgene will create a leading biopharma with increased scale, while maintaining the same agility and a focus on delivering for patients in core disease areas of high-unmet medical need.

The pipeline of the combined company provides significant near-, medium- and long-term opportunities for value creation. Bristol-Myers Squibb is acquiring Celgene’s robust and complementary pipeline at an attractive price.

In addition to six expected near-term product launches representing more than $15B in revenue potential, the combination will greatly increase Bristol-Myers Squibb’s Phase I and II assets, which will provide the next set of registrational opportunities in core therapeutic areas.

With an expanded set of scientific platforms and research capabilities, Bristol-Myers Squibb will be well positioned to discover and develop highly innovative medicines and accelerate these new options to patients through one of the highest-performing commercial organizations in the industry.

Bristol-Myers Squibb is well positioned for 2025 and beyond with continued leadership across Oncology and a diversified portfolio of assets.

The combined company will have a broad, balanced and earlier life-cycle marketed portfolio with a significantly higher number of opportunities across multiple diseases to drive the growth of Bristol-Myers Squibb in the second half of the decade. These opportunities will support financial strength for continued investment and innovation.

The Celgene transaction is expected to generate meaningful financial benefits for all stockholders.

With more than $45B of expected free cash flow generation over the first three full years post-closing, the combination will enable rapid debt reduction to de-lever the balance sheet and strengthen Bristol-Myers Squibb’s credit profile.

Bristol-Myers Squibb expects to realize run-rate cost synergies of approximately $2.5B by 2022 from the combination, and the combined company is expected to grow revenue and EPS every year through 2025.”

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Merck reports positive results of its renal cell carcinoma drug

Merck presents results from Phase 3 KEYNOTE-426 study

Merck presents results from Phase 3 KEYNOTE-426 study, Stockwinners
Merck presents results from Phase 3 KEYNOTE-426 study, Stockwinners

Merck (MRK) announced presentation of the full results from the pivotal Phase 3 KEYNOTE-426 trial investigating KEYTRUDA, Merck’s anti-PD-1 therapy, in combination with Pfizer’s (PFE) Inlyta, a tyrosine kinase inhibitor, for the first-line treatment of advanced renal cell carcinoma at the 2019 Genitourinary Cancers Symposium.

Merck's anti-PD-1 therapy works in combination with Pfizer's (PFE) Inlyta, Stockwinners

Merck’s anti-PD-1 therapy works in combination with Pfizer’s (PFE) Inlyta

This is the first combination regimen to significantly improve overall survival, progression-free survival and objective response rate compared to sunitinib.

Results were consistent across all IMDC subgroups, including favorable, intermediate and poor risk groups, and regardless of PD-L1 expression.

As previously announced, the U.S. Food and Drug Administration has granted priority review for a supplemental Biologics License Application for #KEYTRUDA in combination with axitinib for the first-line treatment of patients with advanced RCC based on the results of KEYNOTE-426, and has set a Prescription Drug User Fee Act, or target action, date of June 20, 2019.

Findings from the first interim analysis showed KEYTRUDA in combination with axitinib reduced the risk of death by 47% – significantly improving OS compared to sunitinib.

For the dual primary endpoint of PFS, the KEYTRUDA combination showed a reduction in the risk of progression of disease or death of 31% compared to sunitinib.

In the study, the ORR was 59.3% for patients who received KEYTRUDA in combination with axitinib and 35.7% for those who received sunitinib, with a complete response rate of 5.8% and 1.9% and a partial response rate of 53.5% and 33.8%, for patients receiving the KEYTRUDA combination or sunitinib, respectively.

Median duration of response was not reached in the KEYTRUDA combination arm and was 15.2 months in the sunitinib arm. The results for OS, PFS and ORR were consistent across all IMDC risk groups and seen regardless of PD-L1 expression.

The observed adverse event profile was as expected based on the known profiles of KEYTRUDA and axitinib.

There was a higher incidence of grade 3 or 4 liver enzyme elevation with KEYTRUDA plus axitinib than previously observed with each agent as monotherapy.

Merck has filed these data with regulatory authorities worldwide.

Merck has an extensive clinical development program in RCC and is advancing multiple potential registration-enabling studies with KEYTRUDA, as monotherapy and in combination with other treatments, including KEYNOTE-564 and KEYNOTE-581.

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