Editas Medicine nabs rare pediatric disease tag for sickle cell candidate

Editas Medicine (EDIT +0.1%) announced FDA approval of Rare Pediatric Disease designation for EDIT-301, for the treatment of sickle cell disease and plans to file an IND application by the end of 2020.

“We know patients are counting on us, and this designation is a significant milestone for the program that highlights the serious, life-threatening manifestations of sickle cell disease.” said Cynthia Collins, CEO.

Rare Pediatric Disease Designation provides for the issuance of a rare pediatric disease priority review voucher following FDA approval. The voucher can be used for accelerated approval of a future application or it can be sold to a third party.

https://seekingalpha.com/news/3608229-editas-medicine-nabs-rare-pediatric-disease-tag-for-sickle-cell-candidate

FDA rejects Tricida’s veverimer for metabolic acidosis in CKD patients

Tricida (TCDA -14.5%) has received a Complete Response Letter (CRL) from the FDA regarding its marketing application seeking approval of veverimer (TRC101) for metabolic acidosis in patients with chronic kidney disease (CKD).

The application was reviewed under the Accelerated Approval Program.

The CRL cited the need of additional data beyond the TRCA-301 and TRCA-301E trials, regarding the magnitude and durability of the treatment effect of veverimer on the surrogate marker of serum bicarbonate.

FDA also expressed concern as to whether the demonstrated effect size would be reasonably likely to predict clinical benefit

The company plans to request a Type A meeting with the FDA in the coming weeks

Last month, the FDA flagged deficiencies in veverimer.

https://seekingalpha.com/news/3608218-fda-rejects-tricidas-veverimer-for-metabolic-acidosis-in-ckd-patients

Connecticut city facing ‘serious’ COVID-19 outbreak, residents urged to stay home

The city of Danbury in Connecticut is facing a “serious outbreak” of the coronavirus, officials warned Friday as they urged residents to stay home.

“This is a serious outbreak in Danbury and we really need an all hands on deck approach,” Acting DPH Commissioner Deidre S. Gifford said in a statement.

From Aug. 2 to 20, the city recorded 178 new cases of the virus, compared to 40 over the previous two weeks.

Health officials are blaming the outbreak on outdoor barbecues, travel and youth sports, which have been canceled over the uptick.

Danbury residents should also avoid unnecessary gatherings, church services and limit other indoor activities, officials said.

Those who feel they have been exposed to the illness are urged to self-quarantine for 14 days.

https://nypost.com/2020/08/23/connecticut-city-facing-serious-coronavirus-outbreak/

Novartis CEO says COVID-19 makes valuing takeover targets tougher

Swiss drugmaker Novartis AG has financial firepower for acquisitions even with net debt of $26 billion, though the COVID-19 pandemic has made it more difficult to value takeover candidates, Chief Executive Vas Narasimhan said in an interview.

“We generate a high free cash flow that allows us to not only finance our dividend but to direct capital to other purposes,” Narasimhan told the Swiss newspaper Neue Zuercher Zeitung.

Still, “acquisitions have slowed recently from a structural perspective” during the pandemic, he said.

Narasimhan, a U.S. citizen who took over two years ago as CEO, acknowledged disappointment that Novartis’ existing medicines had not proven more useful against COVID-19. For instance, the company, like others, abandoned a trial of its older malaria medicine hydroxychloroquine after it failed to help patients in scientific studies.

Moreover, he said Novartis had hoped to be faster in developing new drugs against COVID-19 and could have potentially profited from more cooperation with smaller biotech companies. “We concentrated more on our own in-house activities – and learned a lesson from it,” he said.

Many of the medicines that Novartis is supplying for patients stricken with COVID-19 come from its Sandoz generics unit. For now, Narasimhan said Novartis is not interested in selling Sandoz, but rather continuing work to boost its sales and profit margins squeezed by U.S. price pressure.

“We believe at this time that we can do this best with Sandoz within the Novartis group,” he told the newspaper.

Beyond recent legal settlements in corruption cases that topped $1 billion, Narasimhan believes safety problems that emerged with new eye medicine Beovu have cost Novartis shares 10% of their value.

“Challenge No. 1 was Beovu’s launch,” he said.

https://www.reuters.com/article/us-novartis-ceo/novartis-ceo-says-covid-19-makes-valuing-takeover-targets-tougher-idUSKBN25K0OE

Italy begins testing potential COVID-19 vaccine on volunteers

Italy kicked off human trials of a potential COVID-19 vaccine on Monday, joining a global effort to develop a response to the virus which has shown signs of resurging in Europe.

Rome’s Lazzaro Spallanzani institute, a hospital specializing in infectious diseases will conduct trials on 90 volunteers over the coming weeks, with the hope a vaccine may be available by spring of next year.

Francesco Vaia, health director of the Spallanzani hospital, told Reuters the first patient will be monitored for four hours before being allowed to go home where he will be kept under observation for 12 weeks.

“We will see if it produces any side effects and if it produces neutralizing antibodies,” Vaia said, adding the second phase of testing will take place in countries with higher infection rates, like Mexico and Brazil.

“If we are able to be fast, we will have the first shots on the market next spring,” Vaia added.

The potential vaccine, called GRAd-COV2, was developed by ReiThera, a company based in Rome. The Lazio region, around the Italian capital, said in a statement early trials, including on animals, had delivered positive results.

Potential vaccines are undergoing trials in a number of different countries including India, Britain, Russia and China, as scientists have raced to unpick the secrets of a virus that emerged less than a year ago.

“Our country’s minds and research are at the service of the global challenge to defeat COVID,” Health Minister Roberto Speranza wrote on Facebook announcing the start of the trial.

Italy, one of Europe’s worst-hit countries with more than 35,000 deaths, saw the epidemic peak between March and April before the outbreak appeared to be on the retreat. But it has since seen a surge in new cases with more than 1,000 recorded on both Saturday and Sunday.

Other countries in Europe have seen even bigger jumps as tight restrictions and social distancing measures imposed earlier in the year have been eased.

https://www.reuters.com/article/us-health-coronavirus-italy/italy-begins-testing-potential-covid-19-vaccine-on-volunteers-idUSKBN25K17F

Catalent to make active ingredient for AstraZeneca’s COVID-19 vaccine candidate

Catalent Inc said on Monday it will manufacture active ingredient for AstraZeneca Plc and the University of Oxford’s COVID-19 vaccine candidate.

It will be made at Catalent’s commercial gene therapy manufacturing facility located in Harmans, Maryland, the company said.

https://www.marketscreener.com/quote/stock/ASTRAZENECA-PLC-4000930/news/AstraZeneca-Catalent-to-make-active-ingredient-for-AstraZeneca-s-COVID-19-vaccine-candidate-31166267/

American to gain approval for COVID-fighting surface coating

The U.S. Environmental Protection Agency is expected to announce today an emergency exemption that would allow American Airlines Group (NASDAQ:AAL) to use a new surface coating that kills COVID-19 for up to seven days in Texas.

Sources indicate that the EPA officials will approve the emergency exemption to use SurfaceWise2 through the Federal Insecticide, Fungicide and Rodenticide Act.

https://seekingalpha.com/news/3608123-american-to-gain-approval-for-covid-fighting-surface-coating