EC accepts BeiGene application for Brukinsa for rare blood cancer

The European Commission (EC) accepts for review BeiGene’s (NASDAQ:BGNE) marketing application seeking approval of BTK inhibitor Brukinsa (zanubrutinib) for the treatment of Waldenström’s macroglobulinemia (WM), a rare cancer of the bone marrow and lymphatic tissues characterized by abnormally large numbers of B lymphocytes.

The specific indication is for WM patients who have received at least one prior line of therapy or in a first-line setting in patients who are unsuitable for chemo.

https://seekingalpha.com/news/3584085-ec-accepts-beigene-application-for-brukinsa-for-rare-blood-cancer

BridgeBio Pharma teams up with University of Florida in genetic diseases

BridgeBio Pharma (NASDAQ:BBIO) has entered into a strategic collaboration with the University of Florida to translate research in genetically driven disease towards clinical development and potential commercialization.

BridgeBio will provide sponsorship to select research programs around diseases with a genetic basis, including gene therapies and large and small molecules and will provide guidance for sponsored programs.

The company may conduct proof-of-concept studies for lead therapeutic compounds in relevant mammalian models.

Additionally, BBIO will also collaborate with Johns Hopkins University in the same indication.

https://seekingalpha.com/news/3584090-bridgebio-pharma-teams-up-university-of-florida-in-genetic-diseases

Dicerna nabs Rare Pediatric Disease tag for nedosiran for primary hyperoxaluria

The FDA has granted rare pediatric disease designation to Dicerna Pharmaceuticals’ (NASDAQ:DRNA) nedosiran, an investigational RNAi therapy being developed as a once-monthly treatment for primary hyperoxaluria, a family of ultra-rare, life-threatening genetic disorders that cause complications in the kidneys.

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/3584095-dicerna-nabs-rare-pediatric-disease-tag-for-nedosiran-for-primary-hyperoxaluria

US left with huge hydroxychloroquine stockpile after COVID-19 trial failures

The US has been left with a huge stockpile of hydroxychloroquine, after the World Health Organization abandoned research into the drug as a treatment for COVID-19. 
While hydroxychloroquine has been used for decades to treat malaria, and can also be used in certain inflammatory diseases, the WHO said it had reviewed available evidence and decided to stop the hydroxychloroquine arm of the COVID-19 Solidarity Trial. 
Solidarity is testing several different potential therapies for COVID-19, but its investigators made the decision based on evidence from the trial so far, as well as a Cochrane Review, the UK’s Recovery trial, and other evidence. 
The WHO said that data from Solidarity including the French Discovery trial data, and the recently announced results from the UK’s Recovery trial both showed that hydroxychloroquine does not result in the reduction of mortality of hospitalised COVID-19 patients, when compared with standard of care. 
Investigators will not randomise further patients to hydroxychloroquine in the Solidarity trial. Patients who have already started hydroxychloroquine but who have not yet finished their course in the trial may complete their course or stop at the discretion of the supervising physician. 
The WHO noted that this decision applies only to the conduct of the Solidarity trial and does not apply to the use or evaluation of hydroxychloroquine in pre- or post-exposure prophylaxis in patients exposed to COVID-19. 
The UK’s MHRA put a hold on recruitment to the Recovery trial earlier this week after evidence that emerged earlier this month showed hydroxychloroquine did not reduce mortality or have any beneficial effect. 
This followed the FDA’s decision to revoke an Emergency Use Authorization for hydroxychloroquine in COVID-19. 
According to CNN the Federal government now has a stockpile of more than 63 million doses of the drug, which had been championed by the Trump administration on the basis of preliminary trials earlier in the pandemic. 
The US government has another 2 million doses of chloroquine, a related drug donated by Bayer, said CNN, citing a spokesperson for the US Department of Health and Human Services. 
David Holtgrave, the dean of the School of Public Health at the University at Albany, who co-authored a study of the drug as a treatment for coronavirus, told CNN: “I worry that history will judge this as having over-invested in one treatment pathway as opposed to looking more broadly at a larger number of treatment candidates.” 

US left with huge hydroxychloroquine stockpile after COVID-19 trial failures

Emergent Bio to invest $75M in Mass. site

Emergent BioSolutions (NYSE:EBS) will invest $75M in its Canton, Massachusetts facility, focused on the development and manufacturing of drug substance for live viral vaccines and plans to expand into viral vector and gene therapy.

The investment will include a state-of-the-art, multi-suite operation up to 1000L scale.

The company plans to provide full molecule-to-market contract development and manufacturing services by offering development services out of its Gaithersburg location, drug substance manufacturing out of Canton, and drug product manufacturing at its Rockville, Md. location.

https://seekingalpha.com/news/3584054-emergent-bio-to-invest-75m-in-canton-site

Roche’s Tecentriq improves response rate in early triple-negative breast cancer

Roche (OTCQX:RHHBY) unit Genentech announces positive results from a Phase 3 clinical trial, IMpassion031, evaluating the combination of Tecentriq (atezolizumab) and Celgene’s (NYSE:BMY) Abraxane [albumin-bound paclitaxel, nab-paclitaxel] followed by doxorubicin and cyclophosphamide in patients with early triple-negative breast cancer (TNBC) regardless of PD-L1 expression.

The study met the primary endpoint demonstrating a statistically significant improvement in pathological complete response (pCR) at week 21 compared to placebo + chemo (including Abraxane).

Fewer patients who received the Tecentriq combo as a neoadjuvant (before surgery) treatment had evidence of tumor tissue detectable at the time of surgery (known as pCR), regardless of PD-L1 expression, in comparison to the control arm.

The trial is the second late-state study showing Tecentriq’s benefit in TNBC, the first in early stages of the cancer.

The FDA approved the PD-L1 inhibitor in March 2019 for metastatic TNBC and the EC did the same five months later.

https://seekingalpha.com/news/3584069-roches-tecentriq-improves-response-rate-in-early-triple-negative-breast-cancer

Novacyt launches new COVID-19 testing products

Healthcare and clinical diagnostics company Novacyt, whose shares have surged on the back of its COVID-19 testing products, on Thursday announced the launch of three new products to support testing for the presence of the virus.
The names of the new products are Exsig™ Direct, Exsig™ Mag and COVID-HT, Novacyt said.

The announcement of Novacyt’s latest COVID-19 testing products pushed the company’s Paris-listed shares up by around 10% by 0710 GMT, with the stock having jumped by around 1,600% since the start of 2020.

“As we continue to supply our COVID-19 test to laboratories around the world, we are delighted to provide further support through the launch of these new products,” CEO Graham Mullis said.
Novacyt has singled itself out with a test able to detect the new coronavirus in less than two hours. Standard testing, which also identifies other strains, takes up to a day.
https://www.reuters.com/article/us-health-coronavirus-novacyt/novacyt-shares-jump-after-launch-of-new-covid-19-testing-products-idUSKBN23P0UT