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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Orthofix reports positive results of its artificial cervical disc

Orthofix announces full two-year outcomes from IDE study of M6 cervical disc

Orthofix reports positive results of its artificial cervical disc, Stockwinners

Orthofix Medical (OFIX) announced the full two-year outcomes from its U.S. Investigational Device Exemption study of the M6-C artificial cervical disc.

Currently, the most common form of surgery for treating cervical degenerative disc disease is an anterior cervical discectomy and fusion (ACDF). More than 200,000 cervical procedures are performed each year to relieve compression on the spinal cord or nerve roots. Spinal fusion surgery creates a solid union between two or more vertebrae to help strengthen the spine and alleviate chronic neck pain. There are several types of spinal fusion surgery, as well as varied instrumentation used to secure the fusion.

The data demonstrates that patients treated with the M6-C artificial cervical disc had significant improvements in neck and arm pain, function and quality of life scores.

Additionally, these patients had a significant difference in the reduction of pain and opioid medications use when compared to anterior cervical discectomy and fusion patients.

At 24 months, patients in the ACDF group who were still using pain medications had a seven times higher rate of opioid use than those in the M6-C disc group.

A prospective, non-randomized, concurrently controlled clinical trial, the M6-C IDE study was conducted at 23 sites in the United States with an average patient age of 44 years.

The study evaluated the safety and effectiveness of the M6-C artificial cervical disc compared to ACDF for the treatment of single level symptomatic cervical radiculopathy with or without cord compression.

The overall success rate for the protocol-specified primary endpoint for the M6-C disc patients was 86.8 percent at 24 months and 79.3 percent in the control group.

This data statistically demonstrates that cervical disc replacement with the M6-C disc is not inferior to treatment with ACDF. Secondary outcomes at 24 months include: Patients who received the M6-C disc demonstrated statistically significant improvement in the Neck Disability Index as measured at week six and months three, six, 12 and 24.

Meaningful clinical improvement was seen in the following pain scores: 91.2 percent of patients who received the M6-C disc reported an improvement in neck pain compared to 77.9 percent in patients who underwent the ACDF procedure.

90.5 percent of the M6-C patients reported improvement in arm pain scores compared to 79.9 percent in ACDF patients. Prior to surgery, 80.6 percent of the M6-C disc patients and 85.7 percent of the ACDF patients were taking some type of pain medication for the treatment of their cervical spine condition. At 24 months, the rate of M6-C patients who were still taking some type of pain medication dropped to 14.0 percent compared to 38.2 percent of the ACDF patients.

Of these, there was a seven times higher rate of opioid use with the ACDF patients than with patients who received the M6-C disc.

There was a statistically significant difference in the average mean surgery time – 74.5 minutes for patients receiving the M6-C disc versus 120.2 minutes for those patients having the ACDF procedure.

In addition, there was a statistically significant difference in the mean length of hospital stay – 0.61 days for the M6-C patients versus 1.10 days for ACDF patients. Subsequent surgery at the treated level was needed in 4.8 percent of the ACDF patients compared to 1.9 percent of the M6-C disc patients.

There were no device migrations reported in the study. Overall patients receiving the M6-C disc reported a 92-percent satisfaction rate with the surgery, and 93 percent said they would have the surgery again.

There were 3.8 percent serious adverse events related to the device or procedure in the M6-C disc group versus 6.1 percent in the ACDF group.

The M6-C disc received FDA approval in February 2019 based on the results of this study.

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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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Clovis Oncology is in focus

Clovis receives Breakthrough Therapy Designation for Rubraca

CLVS to submit NDA to FDA
Clovis is in focus

 

Clovis Oncology (CLVS) announced that the U.S. Food and Drug Administration has granted Breakthrough Therapy designation for Rubraca as a monotherapy treatment of adult patients with BRCA1/2-mutated mCRPC who have received at least one prior androgen receptor-directed therapy and taxane-based chemotherapy.

Breakthrough Therapy designation is granted by the FDA to investigational agents intended to treat a serious or life-threatening disease or condition and whose preliminary clinical evidence may demonstrate substantial improvement on at least one clinically significant endpoint over available therapy.

The FDA previously granted Breakthrough Therapy designation to Rubraca for the monotherapy treatment of certain advanced ovarian cancer patients and then in December 2016 approved Rubraca for the treatment of certain adult patients with deleterious BRCA mutation associated epithelial ovarian, fallopian tube, or primary peritoneal cancer who have been treated with two or more chemotherapies.

The FDA subsequently approved Rubraca in a second indication, the maintenance treatment of adult patients with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in a complete or partial response to platinum-based chemotherapy, in April 2018.

This most recent Breakthrough Therapy designation was granted to Rubraca based on initial efficacy and safety results from TRITON2, the Phase 2 study of Rubraca in men with advanced prostate cancer with BRCA 1/2 mutations and deleterious mutations of other homologous recombination repair genes, in the metastatic castration-resistant setting.

JP Morgan Comments

JPMorgan analyst Cory Kasimov is encouraged by Clovis Oncology’s announcement this morning that rucaparib received breakthrough designation from the FDA for the third line treatment of patients with BRCA mutated metastatic castrate-resistant prostate cancer on the basis of data from the Triton-2 study.

The analyst says the news further increases his confidence in the potential for rucaparib to produce response rates that are meaningfully better than currently available options.

His model implies $9 per share for prostate, assuming a 55% probability of success and $450M in peak unadjusted sales. #Kasimov keeps an Overweight rating on Clovis.

CLVS closed at $29.14, it last traded at $31.50.


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Today’s stock disaster

GTx announces placebo-controlled ASTRID trial did not meet primary endpoint

GTx Phase 2 trial of enobosarm meets primary endpoint. Stockwinners.com
Today’s stock disaster

GTx (GTXI) announced that the ASTRID Trial, a Phase 2 double-blind, placebo-controlled clinical trial of orally-administered enobosarm in post-menopausal women with stress urinary incontinence, did not achieve statistical significance on the primary endpoint of the proportion of patients with a greater than 50% reduction in incontinence episodes per day compared to placebo.

The percentage of patients with a greater than 50% reduction after 12 weeks of enobosarm treatment was 58.9% for 3mg, 57.7% for 1mg and 52.7% for placebo.

Enobosarm was generally safe and well tolerated.

Reported adverse events were minimal and similar across all treatment groups.

“We are very disappointed that the ASTRID Trial did not achieve its primary endpoint,” said Robert Wills, executive chairman of GTx.

“We plan to conduct a full review of all the data. We want to thank the patients, physicians, study coordinators and the entire GTx team for their support of this novel study. We have an ongoing preclinical program assessing the potential of SARDs, our novel selective androgen receptor degrader technology, to treat castration-resistant prostate cancer. We are currently on target to have development candidates by year end, which we potentially plan to take into IND-enabling studies.”

GTXI closed at $23.29, it last traded at $2.00.


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ImmunoGen higher on FDA decision

ImmunoGen announces FDA fast track designation for mirvetuximab soravtansine

ImmunoGen announces FDA fast track designation for mirvetuximab soravtansine , Stockwinners
ImmunoGen announces FDA fast track designation for mirvetuximab soravtansine

ImmunoGen (IMGN) announced that the U.S. FDA has granted Fast Track designation for its lead program, mirvetuximab soravtansine.

The designation is for the treatment of patients with medium to high folate receptor alpha-positive platinum-resistant ovarian cancer who received at least one, but no more than three prior systemic treatment regimens, and for whom single-agent chemotherapy is appropriate as the next line of therapy.

Mirvetuximab soravtansine is being evaluated in the FORWARD I Phase 3 trial.

The trial is designed to randomize 333 patients 2:1 to receive either mirvetuximab soravtansine or the physician’s choice of single-agent chemotherapy.

Eligibility criteria include patients with platinum-resistant ovarian cancer that express medium or high levels of FRalpha who have been treated with up to three prior regimens.

The primary endpoint of this study is Progression Free Survival, which is being assessed in the entire study population and in the subset of patients with high FRalpha expression.

Enrollment of FORWARD I was completed ahead of schedule in April 2018 and ImmunoGen expects to report top-line results from the FORWARD I trial in the first half of 2019. ImmunoGen is partnering with the Gynecologic Oncology Group Foundation Inc., a leader in clinical research in gynecologic malignancies, on FORWARD I, which is being conducted in North America and Europe.

This trial is intended to support full marketing approval of mirvetuximab for patients with platinum-resistant ovarian cancer.

Mirvetuximab soravtansine is also being assessed in multiple combinations in the FORWARD II trial.

FORWARD II is a Phase 1b/2 study of mirvetuximab in combination with Avastin, or Keytruda in patients with FRalpha-positive platinum-resistant ovarian cancer, primary peritoneal, or fallopian tube tumors, as well as a triplet combination of mirvetuximab plus carboplatin and Avastin in patients with platinum-sensitive ovarian cancer.

IMGN closed at $10.01, it last traded at $10.65.


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