Bluebird bio: FDA Puts Lenti-D Studies in Cerebral Adrenoleukodystrophy on Hold

 Bluebird bio Inc. on Monday said the U.S. Food and Drug Administration has placed a clinical hold on studies of its Lenti-D cell therapy, also known as eli-cel, in a rare genetic neurological disorder after a patient suffered an adverse reaction.

The Cambridge, Mass., biotechnology company said it received a report of a "suspected unexpected serious adverse reaction" of myelodysplastic syndrome that is likely mediated by Lenti-D lentiviral vector insertion in a patient who was treated with Lenti-D more than a year ago in a Phase 3 study.

Bluebird said available evidence suggests that specific design features of Lenti-D LVV likely contributed to the event, and that the FDA has placed the program on a clinical hold.

The company said it plans to work with regulators and physicians in order to resolve the hold as soon as possible, adding that it doesn't expect the hold to affect its programs in sickle-cell disease, beta-thalassemia or oncology.

Bluebird said it expects to complete the submission of its rolling biologics license application for eli-cel this year, subject to resolution of the clinical hold.

The European Union last month approved the drug, marketed as Skysona, for certain patients under the age of 18 with cerebral adrenoleukodystrophy, which occurs in childhood and can lead to progressive, irreversible loss of neurological function and death.

https://www.marketscreener.com/quote/stock/BLUEBIRD-BIO-INC-13430435/news/Bluebird-bio-FDA-Puts-Lenti-D-Studies-in-Cerebral-Adrenoleukodystrophy-on-Hold-36110778/

AstraZeneca, Daiichi Sankyo Enhertu claims win in breast cancer

 AstraZeneca and Daiichi Sankyo have big plans for their blockbuster-in-the-making Enhertu, and the pair touted another win Monday morning in the quest to conquer the breast cancer market.

Enhertu reached its primary endpoint in a study looking at HER2-positive metastatic breast cancer patients who had previously been treated with one prior line of HER2-based therapy, the companies announced, demonstrating superiority in progression-free survival over Roche’s Kadcyla and chemo. Full data aren’t available yet, but Daiichi Sankyo noted the trial has been recommended for unblinding by the IMDC after reaching this interim analysis.

The companies also said that while overall survival data showed a “strong trend” toward improvement over the control, the OS data haven’t matured enough to conclude anything.

“There is a continued need for new options and better outcomes for patients with HER2 positive metastatic breast cancer who often experience disease progression after initial treatment with available standards of care,” said AstraZeneca oncology chief Susan Galbraith in a statement.

Enhertu won a quick accelerated approval back in December 2019, with the drug receiving its FDA OK just nine months after AstraZeneca and Daiichi Sankyo agreed to collaborate on the program. Though the results from Monday’s study do not come from the confirmatory trial needed to seal full approval, the companies noted the trial is the first to pit Enhertu against an active control.

The accelerated approval came in a later-line indication: unresectable or metastatic HER2-positive breast cancer patients who have received two or more prior anti-HER2-based regimens in the metastatic setting. Monday’s study, however, suggests that for this earlier line of treatment, the companies are planning to shoot straight for a full approval. AstraZeneca and Daiichi Sankyo enrolled about 500 patients in the trial and recruited patients on five continents.

AstraZeneca and Daiichi Sankyo won their previous greenlight based on tumor response rate and duration of response. Their confirmatory study to convert to full approval in the third-line setting remains ongoing and is expected to read out data in the second half of next year.

Though Enhertu netted only $89 million in sales in 2021’s first half, the antibody-drug conjugate has been pushed as AstraZeneca’s next blockbuster drug. In addition to the second-and third-line breast cancer settings, Enhertu is approved for certain types of HER2-positive gastric cancer and is also being studied in non-small cell lung cancer.

But the AstraZeneca and Daiichi Sankyo partnership doesn’t stop there. The two are working on another ADC in datopotamab deruxtecan, agreeing to a deal worth up to $6 billion back in July 2020. For that program, the companies are chasing Gilead’s Trodelvy, which got FDA approval in April to treat metastatic TNBC patients in the second line or later as well as an accelerated nod in urothelial carcinoma the same month.

https://endpts.com/astrazeneca-and-daiichi-sankyo-continue-to-flesh-out-enhertu-plans-claiming-win-in-earlier-breast-cancer-line/

Viatris Reports Strong Second Quarter, Ups Guidance

 

• Reports Second Quarter 2021 Financial Results - Total Revenues of $4.58 billion, U.S. GAAP Net Loss of $279 million, Adjusted EBITDA of $1.68 billion, U.S. GAAP Net Cash Provided by Operating Activities of $559 million, Free Cash Flow of $470 million
• Updates 2021 Financial Guidance (1) - Raises Total Revenues Guidance Range to $17.5 billion to $17.9 billion from $17.2 billion to $17.8 billion, Raises Adjusted EBITDA Guidance Range to $6.15 billion to $6.45 billion from $6.0 billion to $6.4 billion, Raises Free Cash Flow Guidance Range to $2.2 billion to $2.4 billion from $2.0 billion to $2.3 billion
• Announces Board of Directors Declares Quarterly Dividend of Eleven Cents ($0.11) per Share
• Pays Down $1.15 billion of Debt in First Half of 2021
• Receives Historic Approval for First Interchangeable Biosimilar in the U.S. for Semglee® for the Treatment of Diabetes in July (substitutable for the reference product, Lantus®)

https://www.biospace.com/article/releases/viatris-reports-strong-second-quarter-results/

Seagen and RemeGen in Worldwide License, Co-Development Pact for Disitamab Vedotin

 Under the terms of the agreement, Seagen will make a $200 million upfront payment to exclusively license rights to disitamab vedotin for global development and commercialization, outside of RemeGen’s territory. RemeGen will retain development and commercialization rights for Asia, excluding Japan and Singapore. Seagen will lead global development and RemeGen will fund and operationalize the portion of global clinical trials attributable to its territory. RemeGen will also be responsible for all clinical development and regulatory submissions specific to its territory.

Seagen will pay RemeGen up to $2.4 billion in potential total milestone payments based upon the achievement of specified development, regulatory and commercialization goals across multiple indications and products. RemeGen will be entitled to a tiered, high single digit to mid-teen percentage royalty based on net sales of disitamab vedotin in Seagen’s territory.

https://www.businesswire.com/news/home/20210809005208/en/Seagen-and-RemeGen-Announce-Exclusive-Worldwide-License-and-Co-Development-Agreement-for-Disitamab-Vedotin

Sierra Oncology updates on lead drug candidate

 Sierra Oncology, Inc. (NASDAQ: SRRA), a late-stage biopharmaceutical company on a mission to deliver targeted therapies that treat rare forms of cancer, today updated guidance on the development timeline for its lead product candidate, momelotinib. The company now expects topline data from the Phase 3 registration-enabling MOMENTUM study by February 2022, and assuming positive results, the company plans to file a New Drug Application with the U.S. Food & Drug Administration in Q2 2022.

Sierra also announced it has raised $34 million in gross proceeds thus far in Q3 2021 through the use of its at-the-market (ATM) offering. These funds are in addition to the $90.7 million in cash and cash equivalents on hand at the close of the second quarter of 2021, further bolstering the company’s cash balance as it prepares for momelotinib commercialization. The company also has the potential to obtain up to $33.3 million in cash from Series B warrants that must be exercised within 75 days of the announcement of MOMENTUM topline data.

https://www.argus-press.com/news/national/article_aae73b79-f576-5880-8d67-00248ebb3acb.html

Genentech's Polivy Plus R-CHP Shows Prolonged Survival In Lymphoma

 

• Genentech, a member of Roche Holdings AG (OTC: RHHBY), announced that the pivotal Phase 3 POLARIX trial met its primary endpoint in people with previously untreated diffuse large B-cell lymphoma (DLBCL).
• The trial investigated Polivy (polatuzumab vedotin) in combination with Rituxan (rituximab) plus cyclophosphamide, doxorubicin, and prednisone (R-CHP) versus Rituxan plus cyclophosphamide, doxorubicin, vincristine, and prednisone or R-CHOP.
• The trial demonstrated significantly improved and clinically meaningful progression-free survival. The phase 3 study showed Polivy Plus R-CHP is the first regimen in 20 years to significantly improve outcomes in a previously untreated aggressive form of lymphoma compared to standard of care.

https://m.benzinga.com/article/22395731

Covid Booster Shots Coming 'Soon' Fauci Tells CNN

 It's no longer a matter of if but when the US will mandate booster shots to those already vaccinated.

Speaking to CNN's Fareed Zakaria, Anthony Fauci who has managed to last at least a few weeks since a major flip-flop, said Covid-19 vaccine booster shots should be given “reasonably soon” to people with weakened immune systems.

"We need to look at them in a different light. We would certainly be boosting those people before we boost the general population that’s been vaccinated, and we should be doing that reasonably soon."

It wasn't clear who would make the determination which potential subjects have a weakened vaccine system: perhaps Apple will comb through all American iPhones as part of its latest crackdown on personal privacy and alert the proper authorities if it finds a white blood cell count below a given (constantly changing, because this is after all "science") threshold.

Fauci spoke as debate grows over “breakthrough” infections among fully vaccinated people and whether approval should be given for booster shots. On Sunday, Israel, the first nation to roll out booster shots widely, said it had given more than 420,000 third shots to people 60 and over. At least 14 Israelis have already caught Covid-19 after having been injected with a booster shot, suggesting that the booster shot will be the first of many, and will likely last all the way through the mid-term elections because, well, mail-in ballots next November.

Fauci said most people who have compromised immune systems, including those with organ transplants or who are on chemotherapy, “never did get an adequate response” from their Covid-19 vaccination. This is yet another example of US "scientists" moving the goalposts in real time, and they will keep on moving that much is guaranteed: when asked if other groups should get booster shots, Fauci said the CDC and Prevention is ready to give such recommendations “as soon as” they see clear evidence to do so from the data.

As for the kind of data the CDC will be looking for, Fauci said that the CDC has been tracking the level of durability of protection for the elderly, those in nursing homes and young people, month by month. “As soon as they see that level of durability of protection goes down, then you will see the recommendation to vaccinate those individuals.”

Speaking one day after Barack Obama' epic birthday bash (despite it being shrunk for just the closest family and friends), Fauci said that health officials don’t take breakthrough infections “lightly,” warning that the delta variant which is more contagious and is fueling the surge of U.S. cases to more than 100,000 a day, will produce “more” breakthrough cases. Luckily, everyone inside the Barack Birthday Bash tent is exempt from such risks.

And speaking of furiously moving goalposts, Fauci said the Delta variant presents the additional problem that vaccinated people can also transmit the virus to someone else. That has led to the CDC revising its mask guidelines recently. But, he stressed: “The vaccines are still doing what you originally want them to do -- to keep you out of the hospital to prevent you from getting seriously ill.”

Actually, what the CDC "originally" wanted the vaccines to do, was to prevent those who were jabbed from infecting others. Only later did we learn that too was a fabrication.

Finally, Fauci reminded viewers that all Covid-19 vaccines remains experimental although he assured his pals at CNN that a full approval could arrive “within the next few weeks."

https://www.zerohedge.com/markets/covid-booster-shots-coming-soonfauci-tells-cnn