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Thursday, July 18, 2019

Transgene on go with study of immuno-oncology agent in colorectal cancer

The UK Medicines and Healthcare Products Regulatory Agency has signed off on a Phase 1/2a clinical trial evaluating Transgene’s (OTCPK:TRGNF) TG6002, administered by intrahepatic artery infusion, in colorectal cancer patients with unresectable liver metastases. The single-arm open-label study will assess the safety, pharmacokinetics and efficacy of repeated and ascending doses of TG6002, combined with a prodrug of chemo agent 5-FU, in up to 75 patients. The trial should start next quarter.
The company says TG6002 is a next-generation oncolytic immunotherapy, specifically a modified Vaccinia virus, designed to induce the conversion of the non-cytotoxic prodrug into 5-FU in cancer cells that it has infected, thereby killing the cells.

MannKind repays $4M of mature debt from Deerfield

Mannkind (NASDAQ:MNKD) has repaid $4M of debt due today from Deerfield by paying $2.42M in cash and issuing 1,514,423 of common stock at $1.04.
It also repurchased a warrant to acquire ~3.3M common shares for ~$433K.
MNKD was up 4% premarket on light volume.

Flesh-eating bacteria on the rise? Need-to-know on necrotizing fasciitis

Carolyn Fleming of Ellenton, Florida, may have been the latest person to die of a flesh-eating bacteria infection, which she contracted infection swimming off the coast of Anna Maria Island in Florida, her family says.
Cases of the deadly infection, called necrotizing fasciitis, may be on the rise because of climate change.
In fact, a study published last month in the Annals of Internal Medicine said that rising water temperatures in the Delaware Bay may be to blame for an increase in the number of cases of infections in the previously rarely affected waters. The authors of the study described five cases of Vibrio vulnificus necrotizing fasciitis that occurred during the summer of 2017 and 2018. In the eight years before 2017, the doctors saw only one case of the infection.
Fleming, on the other hand, acquired the infection in Florida waters, her family says, where the bacteria are naturally found.
Here’s what you need to know about necrotizing fasciitis:

What is necrotizing fasciitis?

Necrotizing fasciitis is a skin infection caused by rare bacteria that enters the body through a break in the skin. From this opening, the bacteria aggressively attacks muscles and other organs resulting in rapid destruction of the tissues. Vibrio bacteria are one group that can cause this infection; one type, called Vibrio vulnificus, is particularly dangerous. It is usually contracted when an open wound comes into contact with coastal saltwater. Necrotizing fasciitis can quickly develop soon after. If not treated promptly with antibiotics, the infection can become fatal. But more often, the bacteria are ingested in raw or undercooked seafood, causing diarrhea. The resulting illness is rarely life-threatening and symptoms occur within 24 hours and last about three days.
Group A strep is another type of bacteria that can cause necrotizing fasciitis and severe diarrhea, as well as a system-wide blood infection that leads to severely low blood pressure and multiple organ failure, called streptococcal toxic shock syndrome.

Who is most likely to get it?

Those with health conditions that weaken the body’s immune system such as:
  • Diabetes
  • Kidney disease
  • Liver disease
  • Cancer
“V. vulnificus, which seems to be on the rise, causes human health concerns in the form of blood infection and wound infection. The elderly, those who are immune-compromised, and the very young are at greatest risk,” said Kimberly Reece, chair of Aquatic Health Sciences at the Virginia Institute of Marine Science.
Contact with Group A strep or V. vulnificus rarely leads to infection in healthy teenagers and adults, but anyone with open wounds should avoid swimming pools, hot tubs and natural bodies of water like lakes, rivers, estuaries and oceans.

How common is the disease?

The infection is pretty rare, but Dr. Glenn Morris, director of the Emerging Pathogens Institute at the University of Florida, says people should be made aware of it.
“Vibrio vulnificus is a small problem but nevertheless significant. It can cause a nasty cellulitis in a small group of patients,” Morris said. “If you fall into one of these high-risk categories, people should be aware that there are bacteria in the water that can cause serious infections.”
According to the CDC, every year, 700 to 1,200 cases are diagnosed in the U.S., although the CDC notes that this is likely an underestimate. About 25 percent to 30 percent of those cases result in death.
Necrotizing fasciitis is rarely contagious.

How to spot necrotizing fasciitis:

Early symptoms include:
  • Changes in skin color
  • A rapidly spreading inflamed or swollen area of the skin
  • Severe pain, including pain beyond the area of the skin that is red or swollen
  • Fever
Later on ulcers or blisters can form, as well as dizziness, fatigue, nausea, and diarrhea. The infection often spreads quickly so it is imperative to seek medical attention if someone is experiencing one or more of these symptoms.

How is necrotizing fasciitis treated?

Necrotizing fasciitis is a serious illness that requires immediate hospital attention. Antibiotics and surgery are typically the first lines of defense, but even with prompt treatment the CDC says up to one in three people can die from the infection.

Apellis Program to Control Host Response to AAV Vector for Gene Therapies

Apellis, (Nasdaq:APLS) a clinical-stage biopharmaceutical company focused on the development of novel therapeutic compounds to treat disease through the control of the complement system, today announced its plans to develop APL-9 for the prevention of complement immune system activation coincident with adeno-associated virus (AAV) vector administration for gene therapies. APL-9, an investigational drug, is a pegylated synthetic cyclic peptide designed to modulate the complement cascade centrally at C3.
“Gene therapies are among the most exciting medical advancements of our time. However, they also pose a great technical challenge, since the AAV particles designed to deliver genes to tissues are subject to immediate and overwhelming immune attack, specifically by complement C3, which may result in significant safety and efficacy constraints,” said Cedric Francois, CEO and co-founder of Apellis. “We believe that the targeted control of C3 may prevent the C3-mediated attack on AAV particles, yielding three important potential advantages. First, controlling C3 may significantly increase the efficiency of gene therapy delivery by protecting the AAV particles on their journey from the site of administration to the target tissue. Second, control of C3 may minimize the formation of anti-AAV neutralizing antibodies that can complicate subsequent retreatment with the same AAV. Finally, by countering the rapid activation of C3 upon administration of AAV particles, control of C3 may prevent inflammatory reactions that can lead to significant organ damage, for example in the kidneys. Apellis is committed to the development of APL-9 to target host responses to AAV vector administration and thus potentially improve patients’ well-being while undergoing AAV administered gene therapies.”
In Phase I testing, Apellis treated 20 healthy volunteers with single doses of APL-9 ranging from 30 mg to 600 mg, administered as an intravenous infusion. APL-9 demonstrated control of complement through modulation of C3 within 1 hour of administration, that lasted up to 12 hours after the end of the infusion. Multiple doses tested achieved complete suppression of the AH50 hemolytic activity. In this Phase I study, APL-9 was well tolerated with no serious adverse events reported.

Medicare to cover Novocure’s Optune for brain cancer

Novocure (NASDAQ:NVCRannounces that Medicare will cover Optune (Tumor Treating Fields) for newly diagnosed glioblastoma effective September 1.

Chembio teams up with Takeda on point-of-care test

Chembio Diagnostics (NASDAQ:CEMI) will collaborate with Takeda (NYSE:TAK) unit Shire Human Genetic Therapies to develop an point-of-care (POC) diagnostic test for an undisclosed biomarker.
The quantitative test will be based on CEMI’s DPP platform and hand-held optical analyzer.
Financial terms are not disclosed.

MyoKardia reacquires rights to mavacamten and MYK-224 from Sanofi

MyoKardia (NASDAQ:MYOK) has reacquired U.S. royalty rights to mavacamten and MYK-224 from Sanofi in exchange for $50M in upfront cash plus an additional $30M by June 30, 2020. Under the terms of their former agreement, Sanofi was eligible to receive royalties of 5 – 10% on U.S. sales.
Enrollment in the Phase 3 EXPLORER-HCM study evaluating mavacamten in patients with obstructive hypertrophic cardiomyopathy (oHCM) should be completed in about a month. Topline data should be available in Q2 2020.