Friday, June 12, 2020
FDA OKs Viela’s rare disease treatment, analysts predict $500 million in sales
Shares of Viela Bio Inc. VIE, -3.79%
were up 4.9% in premarket trading on Friday, the day after the company
received approval from the Food and Drug Administration (FDA) to market
Uplizna to treat neuromyelitis optica spectrum disorder. It is the
second treatment for the rare neuroinflammatory disorder to receive FDA
approval. The drug helps reduce the risk of attacks that can lead to
paralysis and blindness. Viela said it plans to launch Uplizna this
month; a company spokesperson declined to provided pricing information
at this time but said the drug will be priced “favorably” and
“significantly lower” than Alexion Pharmaceuticals Inc.’s ALXN, 1.27%
Soliris. Stifel analysts predict roughly $500 million in sales of
Uplizna in the U.S. per year. “From our review of the label, it looks
largely in-line with base case expectations,” they wrote in a June 12
note to investors. Viela’s stock is up 89.5% year-to-date. The S&P
500 SPX, 2.55% is down 7.1% since the start of the year.
Gilead’s Kite Gets European OK for Cell Therapy Manufacturing Plant
Gilead Sciences Inc. on Friday said its Kite unit received European
Medicine Agency approval to implement a variation to the Yescarta
marketing authorization for end-to-end manufacturing.
The Foster City, Calif., biopharmaceutical company said that with the approval Kite’s manufacturing facility outside of Amsterdam, designed and dedicated to the manufacture of individualized cell therapies, is fully operational.
The company said the facility will allow Yescarta to reach European treatment centers more quickly, reducing the time it takes to reach patients by almost a week.
Yescarta, a CAR-T cell therapy, is approved in the U.S. and European Union for adults with relapsed or refractory diffuse large B-cell lymphoma and primary mediastinal large B-cell lymphoma after two or more lines of systemic therapy.
https://www.marketscreener.com/GILEAD-SCIENCES-4876/news/Gilead-Sciences-Kite-Gets-European-OK-for-Cell-Therapy-Manufacturing-Plant-30762254/
The Foster City, Calif., biopharmaceutical company said that with the approval Kite’s manufacturing facility outside of Amsterdam, designed and dedicated to the manufacture of individualized cell therapies, is fully operational.
The company said the facility will allow Yescarta to reach European treatment centers more quickly, reducing the time it takes to reach patients by almost a week.
Yescarta, a CAR-T cell therapy, is approved in the U.S. and European Union for adults with relapsed or refractory diffuse large B-cell lymphoma and primary mediastinal large B-cell lymphoma after two or more lines of systemic therapy.
https://www.marketscreener.com/GILEAD-SCIENCES-4876/news/Gilead-Sciences-Kite-Gets-European-OK-for-Cell-Therapy-Manufacturing-Plant-30762254/
Constellation Pharma’s CPI-0610 shows positive action in myelofibrosis
Constellation Pharmaceuticals (NASDAQ:CNST) announces new data from an open-label Phase 2 clinical trial, MANIFEST,
evaluating BET protein inhibitor CPI-0610 in patients with a rare type
of bone marrow cancer called myelofibrosis. The results were virtually
presented at EHA.
72.5% (n=37/51) of patients receiving CPI-0610
plus Incyte’s Jakafi (ruxolitinib) in a first-line setting achieved a
35% reduction in spleen volume (SVR35) at week 12. 63.3% (n=19/30)
achieved SVR35 at week 24. 58.6% (n=17/29) achieved a 50% improvement in
Total Symptom Score (TSS50) at week 24.
In patients receiving CPI-0610 alone in a
second-line setting, 21.4% (n=3/14) of transfusion-dependent (TD)
patients converted to transfusion independence (TI). 23.8% (n=5/21) of
evaluable non-TD patients achieved SVR35 and 47.4% (n=9/19) of evaluable
non-TD patients achieved TSS50 at week 24.
In ruxolitinib-experienced patients receiving
CPI-0610 + ruxolitinib (second-line setting), 34.4% (n=11/32) of
evaluable TD patients converted to TI. 22.2% (n=4/18) of evaluable
non-TD patients achieved SVR35 and 36.8% (n=7/19) of evaluable non-TD
patients achieved TSS50 at week 24.
On the safety front, the most common
treatment-emergent adverse events for CPI-0610 monotherapy were
thrombocytopenia, anemia, diarrhea and respiratory tract infection.
There were six discontinuations.
https://seekingalpha.com/news/3582609-constellation-pharmas-cpiminus-0610-shows-positive-action-in-myelofibrosis
Pfizer partner BioNTech gets €100M debt to fund Covid vax production in Europe
BioNTech and Pfizer are among the front-runners in the closely
watched COVID-19 vaccine race, and as they simultaneously work through
development and scale up manufacturing, BioNTech has scored a €100
million financing agreement to help fund production in Europe.
The European Investment Bank signed a debt financing deal with BioNTech—to be completed in two €50 million tranches—as the biotech advances its Pfizer-partnered COVID-19 vaccine program, BNT162. The partners entered human testing in Germany back in April, followed by a U.S. study that launched in early May. The funding will be contingent on the program reaching certain milestones.
The funds will help with ongoing development and with BioNTech’s efforts to scale up for production in Europe. The biotech already operates three mRNA factories in Germany, and it has been working to add capacity for its COVID-19 vaccine program.
The BNT162 program is testing four mRNA vaccines against COVID-19 so
the partners can zero in on the most promising options. The companies
inked their COVID-19 vaccine tie-up back in March.
In April, the companies unveiled plans to produce “millions” of doses this year and potentially “hundreds of millions” in 2021. At the time, Cantor Fitzgerald analysts wrote that the team could begin producing doses “at risk” to be in a position to distribute as quickly as possible if and when they win approvals.
While Pfizer and BioNTech are in phase 1/2 testing, Moderna and AstraZeneca have moved to phase 2 and beyond. Meanwhile, U.S. authorities are planning phase 3 studies for Moderna, AstraZeneca and Johnson & Johnson later this year, The Wall Street Journal reported Wednesday. Pfizer could start a phase 3 trial as soon as July, a source told the newspaper.
BioNTech is focusing on production in Europe, and Pfizer has selected three U.S. sites—and one in Belgium—for its mRNA vaccine manufacturing. Those sites are in Michigan, Massachusetts and Missouri as well as Puurs, Belgium. Pfizer has said it plans to select other sites as well.
https://www.fiercepharma.com/manufacturing/pfizer-partner-biontech-scores-eu100m-debt-financing-to-fund-european-covid-19
The European Investment Bank signed a debt financing deal with BioNTech—to be completed in two €50 million tranches—as the biotech advances its Pfizer-partnered COVID-19 vaccine program, BNT162. The partners entered human testing in Germany back in April, followed by a U.S. study that launched in early May. The funding will be contingent on the program reaching certain milestones.
The funds will help with ongoing development and with BioNTech’s efforts to scale up for production in Europe. The biotech already operates three mRNA factories in Germany, and it has been working to add capacity for its COVID-19 vaccine program.
In April, the companies unveiled plans to produce “millions” of doses this year and potentially “hundreds of millions” in 2021. At the time, Cantor Fitzgerald analysts wrote that the team could begin producing doses “at risk” to be in a position to distribute as quickly as possible if and when they win approvals.
While Pfizer and BioNTech are in phase 1/2 testing, Moderna and AstraZeneca have moved to phase 2 and beyond. Meanwhile, U.S. authorities are planning phase 3 studies for Moderna, AstraZeneca and Johnson & Johnson later this year, The Wall Street Journal reported Wednesday. Pfizer could start a phase 3 trial as soon as July, a source told the newspaper.
BioNTech is focusing on production in Europe, and Pfizer has selected three U.S. sites—and one in Belgium—for its mRNA vaccine manufacturing. Those sites are in Michigan, Massachusetts and Missouri as well as Puurs, Belgium. Pfizer has said it plans to select other sites as well.
https://www.fiercepharma.com/manufacturing/pfizer-partner-biontech-scores-eu100m-debt-financing-to-fund-european-covid-19
What’s the ROI on a COVID-19 vaccine? We have no idea, says Pfizer
John Young, Pfizer’s chief business officer, was only about four
minutes into a one-hour fireside chat at this year’s virtual BIO
conference when he addressed a question that’s been weighing on
investors for several weeks now: If the company successfully develops a
vaccine against COVID-19, what will the return on investment be?
When Pfizer began strategizing methods for addressing the pandemic back in January, “we realized this is not a time to think about … a typical ROI,” Young said. “Frankly, the world needs a safe and effective vaccine,” and the company’s priority is to “play our part in bringing forward vaccines and treatments that the world needs right now.”
“At Pfizer, there is no thought being given to ‘ROI’ in our COVID-19 work. Instead our decisions are being driven by three priorities: the health and safety of our colleagues, the continued supply of our medicines and vaccines, and finding medical solutions to this crisis,” a Pfizer spokesperson added in an emailed statement.
Pfizer has been working with Germany-based mRNA specialist BioNTech to develop a COVID-19 vaccine. The partners are now in clinical trials, assessing four variants of the vaccine. After testing the variants at different schedules and dose levels, they will select a vaccine candidate to move into larger trials.
Pifzer is one of five finalists in Operation Warp Speed, the U.S. government’s attempt to speed up the development of a COVID-19 vaccine. But unlike other contenders, it is not looking for federal funding to accelerate clinical development. There’s a reason for that, Young said at BIO.
“We made an active decision not to seek government funding because we didn’t want this to slow down our partnership with BioNTech and to slow the progress moving a vaccine construct into the clinic,” Young said. “Our focus was to move as quickly as possible and we really didn’t want to … spend a month negotiating with the U.S. government.”
So how long will Pfizer’s development process take? Young was reluctant to make any hard predictions. Too few patients have been dosed in the clinical trials thus far, he said, and “ultimately data should really drive the decisions that we make, along with regulators, as to which [candidate] has the potential to be the safest and most effective vaccine construct.”
But Pfizer is thinking about how to tackle the challenge of manufacturing enough doses of a COVID-19 vaccine to meet the demand—and investing plenty in that effort. The company said in May it would lean on its manufacturing sites in Michigan, Massachusetts and Missouri, as well as a fourth site in Belgium, to ramp up production of the vaccine.
During the BIO chat, Young said Pfizer will handle the U.S. supply chain and BioNTech “will develop a parallel supply chain for Europe and rest the world.” Meanwhile, Pfizer will outsource production of some its other drugs to contractors to clear manufacturing space.
Despite its strong partner network, the manufacturing of a COVID-19 mRNA vaccine will be far from simple, Young said. Typically, Pfizer makes about 500 million vaccine doses per year, but this time around, the company has committed to make “tens of millions of doses this year and hundreds of millions in 2021” if one of its vaccine candidates succeeds, he said. “So we need to more than double down on our investments in our supply chain, and to make significant investments of capital, as well as investments in … some very specialized raw materials.”
Young emphasized that making an mRNA vaccine is a brand-new endeavor for the company. It involves combining nanoparticles with mRNA and using plasmids to produce the mRNA. “We have not made one of those products before, so we really had to from scratch work with raw material suppliers … and companies that make these very specialized products.”
It all brought to mind that one pressing question: What will the ROI be for Pfizer? To answer that, the company would need to estimate its own costs, not to mention the price of the vaccine—neither of which it’s ready to do, Young said.
Pfizer has done “no modeling work to this point around … the pricing,” Young said, because “we’re still in the process of figuring out where our supply chain is to know even what our cost of manufacturing is. So it’s really premature in our vaccine program to reach a point of view on pricing.”
The controversy over drug pricing continues to be a thorn in the side of the pharma industry, even amid the pandemic. Pfizer is well aware of the need to balance the need for an affordable COVID-19 vaccine with the demand from investors for ROI—and the potential for its actions to help repair the industry’s image, Young said.
“We really need effective vaccines … to get back to normal economic activity,” Young said. “So I hope, if we’re successful in that mission … the perception of our industry will reflect the efforts that we’ve made.”
https://www.fiercepharma.com/pharma/bio-what-s-roi-a-covid-19-vaccine-we-have-no-idea-says-pfizer
When Pfizer began strategizing methods for addressing the pandemic back in January, “we realized this is not a time to think about … a typical ROI,” Young said. “Frankly, the world needs a safe and effective vaccine,” and the company’s priority is to “play our part in bringing forward vaccines and treatments that the world needs right now.”
“At Pfizer, there is no thought being given to ‘ROI’ in our COVID-19 work. Instead our decisions are being driven by three priorities: the health and safety of our colleagues, the continued supply of our medicines and vaccines, and finding medical solutions to this crisis,” a Pfizer spokesperson added in an emailed statement.
Pfizer has been working with Germany-based mRNA specialist BioNTech to develop a COVID-19 vaccine. The partners are now in clinical trials, assessing four variants of the vaccine. After testing the variants at different schedules and dose levels, they will select a vaccine candidate to move into larger trials.
Pifzer is one of five finalists in Operation Warp Speed, the U.S. government’s attempt to speed up the development of a COVID-19 vaccine. But unlike other contenders, it is not looking for federal funding to accelerate clinical development. There’s a reason for that, Young said at BIO.
“We made an active decision not to seek government funding because we didn’t want this to slow down our partnership with BioNTech and to slow the progress moving a vaccine construct into the clinic,” Young said. “Our focus was to move as quickly as possible and we really didn’t want to … spend a month negotiating with the U.S. government.”
So how long will Pfizer’s development process take? Young was reluctant to make any hard predictions. Too few patients have been dosed in the clinical trials thus far, he said, and “ultimately data should really drive the decisions that we make, along with regulators, as to which [candidate] has the potential to be the safest and most effective vaccine construct.”
But Pfizer is thinking about how to tackle the challenge of manufacturing enough doses of a COVID-19 vaccine to meet the demand—and investing plenty in that effort. The company said in May it would lean on its manufacturing sites in Michigan, Massachusetts and Missouri, as well as a fourth site in Belgium, to ramp up production of the vaccine.
During the BIO chat, Young said Pfizer will handle the U.S. supply chain and BioNTech “will develop a parallel supply chain for Europe and rest the world.” Meanwhile, Pfizer will outsource production of some its other drugs to contractors to clear manufacturing space.
Despite its strong partner network, the manufacturing of a COVID-19 mRNA vaccine will be far from simple, Young said. Typically, Pfizer makes about 500 million vaccine doses per year, but this time around, the company has committed to make “tens of millions of doses this year and hundreds of millions in 2021” if one of its vaccine candidates succeeds, he said. “So we need to more than double down on our investments in our supply chain, and to make significant investments of capital, as well as investments in … some very specialized raw materials.”
Young emphasized that making an mRNA vaccine is a brand-new endeavor for the company. It involves combining nanoparticles with mRNA and using plasmids to produce the mRNA. “We have not made one of those products before, so we really had to from scratch work with raw material suppliers … and companies that make these very specialized products.”
It all brought to mind that one pressing question: What will the ROI be for Pfizer? To answer that, the company would need to estimate its own costs, not to mention the price of the vaccine—neither of which it’s ready to do, Young said.
Pfizer has done “no modeling work to this point around … the pricing,” Young said, because “we’re still in the process of figuring out where our supply chain is to know even what our cost of manufacturing is. So it’s really premature in our vaccine program to reach a point of view on pricing.”
The controversy over drug pricing continues to be a thorn in the side of the pharma industry, even amid the pandemic. Pfizer is well aware of the need to balance the need for an affordable COVID-19 vaccine with the demand from investors for ROI—and the potential for its actions to help repair the industry’s image, Young said.
“We really need effective vaccines … to get back to normal economic activity,” Young said. “So I hope, if we’re successful in that mission … the perception of our industry will reflect the efforts that we’ve made.”
https://www.fiercepharma.com/pharma/bio-what-s-roi-a-covid-19-vaccine-we-have-no-idea-says-pfizer
Australian clot-busting drug holds hope for COVID-19 treatment
An experimental drug developed by an Australian researcher could help
prevent deaths from COVID-19 by controlling the formation of blood
clots responsible for breathing difficulties, organ failure, stroke and
heart attack.
Professor Shaun Jackson from the University of Sydney and the Heart Research Institute is leading a team of researchers developing a new anti-clotting medicine to treat stroke.
About three in four of critical COVID-19 patients in Intensive Care Units (ICUs) develop clots with their recovery rate critically low, Jackson said. COVID-19 is the respiratory disease caused by the novel coronavirus.
“If our medicine can control these clots, then organ failure and death in many thousands of cases could be avoided. We want COVID-19 patients reaching for the tissue box, not hooked up to ventilators,” Jackson said.
Following successful phase-1 trials in 72 healthy patients, the
researchers now want to urgently move into phase 2 trials by testing the
effectiveness and safety of the drug in critically ill COVID-19
patients.
“It could then be a matter of months before doctors around the world can use the novel anti-clotting drug to protect patients with COVID-19, potentially saving thousands of lives,” Jackson said.
More than 7.53 million people have been reported infected with the novel coronavirus around the world and 420,808 have died, a Reuters tally showed as of 0504 GMT on Friday.
Australia reported about 7,300 cases and 102 deaths with some parts of the country now claiming to have eliminated the virus.
Jackson said phase-2 trials of the drug, which is administered intravenously, will need to be done overseas because there were not enough severely ill patients on ventilators in Australia.
https://www.reuters.com/article/us-health-coronavirus-australia-clots/australian-clot-busting-drug-holds-hope-for-covid-19-treatment-idUSKBN23J0WY
Professor Shaun Jackson from the University of Sydney and the Heart Research Institute is leading a team of researchers developing a new anti-clotting medicine to treat stroke.
About three in four of critical COVID-19 patients in Intensive Care Units (ICUs) develop clots with their recovery rate critically low, Jackson said. COVID-19 is the respiratory disease caused by the novel coronavirus.
“If our medicine can control these clots, then organ failure and death in many thousands of cases could be avoided. We want COVID-19 patients reaching for the tissue box, not hooked up to ventilators,” Jackson said.
“It could then be a matter of months before doctors around the world can use the novel anti-clotting drug to protect patients with COVID-19, potentially saving thousands of lives,” Jackson said.
More than 7.53 million people have been reported infected with the novel coronavirus around the world and 420,808 have died, a Reuters tally showed as of 0504 GMT on Friday.
Jackson said phase-2 trials of the drug, which is administered intravenously, will need to be done overseas because there were not enough severely ill patients on ventilators in Australia.
https://www.reuters.com/article/us-health-coronavirus-australia-clots/australian-clot-busting-drug-holds-hope-for-covid-19-treatment-idUSKBN23J0WY
Bluebird bio gene therapy shows high response rate in beta thalassemia
Bluebird bio (NASDAQ:BLUE) announces new
data from ongoing Phase 3 clinical trials evaluating gene therapy
Zynteglo (betibeglogene autotemcel) (formerly LentiGlobin) in adolescent
and adult patients with a range of genotypes of transfusion-dependent
beta-thalassemia (TDT).
All 23 patients in the Phase 3 Northstar-2
study have been treated and followed for a median of 19.4 months. In 19
evaluable subjects, 89.5% (n=17/19) achieved transfusion independence.
Previously, the 17 required a median of 17.5 transfusions each year.
In Northstar-3, 15 patients (genotypes: 9 β0/β0, 3 β0/
β+IVS1-110, 3 homozygous IVS-1-110 mutation) were treated and followed
for a median of 14.4 months. 75.0% (n=6/8) achieved transfusion
independence while 84.6% (n=11/13) with at least seven months’ follow-up
had not received a transfusion in more than seven months. Previously,
the 11 required a median of 18.5 transfusions each year.
On the safety front, there were three serious
events of veno-occlusive liver disease and two serious events of
thrombocytopenia in Northstar-2 and two serious events of pyrexia
(elevated body temperature) in Northstar-3. No deaths, graft failures,
vector-mediated replication competent lentivirus or clonal dominance,
leukemia or lymphoma were reported.
https://seekingalpha.com/news/3582576-bluebird-bio-gene-therapy-shows-high-response-rate-in-beta-thalassemia
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