Justice Ginsburg Treated for Lung Cancer

Supreme Court Justice Ruth Bader Ginsburg had two malignant lung nodules removed on Friday, the court announced.

“Justice Ruth Bader Ginsburg underwent a pulmonary lobectomy today at Memorial Sloan Kettering Cancer Center in New York City,” the court said in a press release. “Two nodules in the lower lobe of her left lung were discovered incidentally during tests performed at George Washington University Hospital to diagnose and treat rib fractures sustained in a fall on November 7.”

“According to the thoracic surgeon, Valerie Rusch, MD, both nodules removed during surgery were found to be malignant on initial pathology evaluation,” the release continued. “Post-surgery, there was no evidence of any remaining disease. Scans performed before surgery indicated no evidence of disease elsewhere in the body. Currently, no further treatment is planned. Justice Ginsburg is resting comfortably and is expected to remain in the hospital for a few days.”

This is the 85-year-old Ginsburg’s third cancer diagnosis and her second “incidentaloma.” In 1999, doctors discovered she had stage II colon cancer while treating her for an unrelated abdominal infection; she had her sigmoid colon removed. “The justice is very lucky to have had this picked up incidentally,” said Harmon Eyre, MD, chief medical officer for the American Cancer Society at the time, in an article in the New York Times. “If it had been left alone, it would have advanced to another stage.”

Then, in 2009, Ginsburg was diagnosed with pancreatic cancer during a routine physical. That tumor was about 1 cm, which is about as small as can be detected on CT scan, surgical oncologist Paul Lin, MD, of George Washington University Hospital, said in an ABC News story at the time. “She is much more fortunate than most patients who come in because of symptoms from their cancer,” he said.

Ginsburg also has had other health problems; in 2014 doctors found a blocked artery after she suffered chest pain during a workout. She had a stent implanted and went quickly back to work.

Ginsburg, who has been on the court since 1993, is one of its most reliable liberals; she voted twice to uphold the Affordable Care Act (ACA) and also has voted in support of abortion rights. In the second ACA vote, Ginsburg voted with the majority to uphold the law and its individual mandate, but went even further, dissenting from the majority rulingthat states could not be required under the ACA to expand their Medicaid programs. “Congress can and often does expand programs, adding new conditions states must meet before receiving funds,” she said from the bench.

https://www.medpagetoday.com/publichealthpolicy/healthpolicy/77090

Aimmune Therapeutics files IND for AR201 for egg allergy

Aimmune Therapeutics (AIMT +13.1%) submits IND application for AR201 for the

treatment of egg allergy. The company also announced an exclusive supply agreement for egg protein with Michael Foods, Inc.

Aimmune expects to initiate a Phase 2 clinical trial of AR201 for the treatment of egg allergy in 2019.

The agreement with Michael Foods includes all of the company’s egg products, worldwide, and gives Aimmune exclusive access to the clinical and commercial use of Michael Foods egg products for any egg allergy treatment, prevention or cure for a period of up to 15 years beyond the potential approval of AR201.

https://seekingalpha.com/news/3418509-aimmune-therapeutics-files-ind-ar201-egg-allergy

Aimmune Submits BLA to FDA for AR101 for Kids’, Teens’ Peanut Allergy

Aimmune Therapeutics, Inc. (Nasdaq: AIMT), a biopharmaceutical company developing treatments for potentially life-threatening food allergies, today announced that it has submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for AR101, an investigational biologic oral immunotherapy for the treatment of peanut allergy in children and adolescents ages 4–17. The FDA granted AR101 Fast Track Designation for peanut allergy in September 2014 and Breakthrough Therapy Designation for peanut allergy in ages 4–17 in June 2015.

“The submission of the BLA represents a significant milestone for Aimmune and the food allergy community, for whom there currently are no approved treatments,” said Jayson Dallas, M.D., President and CEO of Aimmune. “We have requested FDA Priority Review and look forward to working with the agency to bring what could be the first approved treatment in food allergy to patients as quickly as possible.”

Aimmune’s BLA submission includes data from the pivotal Phase 3 PALISADE trial of AR101, recently published in the New England Journal of Medicine; the PALISADE follow-on trial ARC004; and the Phase 3 RAMSES trial, which confirmed that the safety profile of AR101 was consistent with that observed in the PALISADE trial; as well as extensive data on AR101 chemistry, manufacturing and controls (CMC).

“Based on positive results from the PALISADE trial, we believe that AR101 offers the only path to potential protection that matters for kids and teens with peanut allergy,” said Daniel Adelman, M.D., Chief Medical Officer of Aimmune. “We are indebted to all those who helped us reach this important point in our development, including our employees, clinical investigators, advocates, and, of course, our patients and their families.”

Under the Prescription Drug User Fee Act (PDUFA), a Priority Review would target a review time of six months, compared to a standard review time of 10 months. FDA informs the applicant of a Priority Review designation within 60 days of a BLA submission.

https://www.businesswire.com/news/home/20181221005567/en/Aimmune-Therapeutics-Submits-BLA-FDA-AR101-Treatment

Apollo Medical: APA ACO unit generated $12.96M in gross savings in 2017

Apollo Medical Holdings, Inc. announced that its wholly-owned subsidiary, APA ACO, Inc. generated $12.96M in gross savings in its first performance year and that, as a result, it achieved $5.9M in shared savings from the Centers for Medicare & Medicaid Services. APA ACO was one of 44 Next Generation Accountable Care Organizations in the country selected by CMS to participate in the Next Generation ACO Model in 2017. APA ACO was approved to participate in the All-Inclusive Population-Based Payment track, which is the most advanced risk-taking payment model, and was the only Next Generation ACO in the country out of 44 ACOs to participate in the AIPBP track in 2017. Under the AIPBP track, CMS estimates the total annual expenditures for the Next Generation ACO’s patients and then pays that projected amount to the ACO in a per-beneficiary, per-month payment. The Next Generation ACO is then responsible for paying all Part A and Part B costs for in-network participating providers and preferred providers with whom it has contracted. For 2017, APA ACO’s aggregate benchmark expenditure calculated by CMS was $390.56M. APA ACO’s actual expenditures were $377.6M, resulting in gross savings of $12.96M. CMS then deducted a stop-loss charge of $5.44M, resulting in gross savings after stop-loss charge of $7.52M. APA ACO had chosen an 80/20 risk arrangement with CMS, and therefore the net shared savings to APA ACO is $5.90M. Since ApolloMed had already accrued APA ACO expenses for 2017, all of the $5.90M payment was recognized as net income in its 2018 3rd quarter financial statements included in its Form 10Q.
https://thefly.com/landingPageNews.php?id=2840849

Intrexon’s Precigen Starts Phase 1/1b Study for Leukemia Med

Precigen, Inc., a wholly-owned subsidiary of Intrexon Corporation (NASDAQ: XON), and a biopharmaceutical company specializing in the development of innovative gene and cellular therapies to improve the lives of patients, today announced that the US Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for PRGN-3006, a first-in-class investigational therapy using Precigen’s UltraCAR-T™ platform. PRGN-3006 UltraCAR-T is an autologous chimeric antigen receptor T-cell (CAR-T) therapy for treatment of patients with relapsed or refractory acute myeloid leukemia (AML) and higher risk myelodysplastic syndrome (MDS). PRGN-3006 utilizes Precigen’s transformative UltraCAR-T platform, which reduces manufacturing time to less than two days following non-viral gene transfer.

PRGN-3006 UltraCAR-T is a multigenic CAR-T cell treatment utilizing Precigen’s clinically-validated Sleeping Beauty system to co-express chimeric antigen receptor, membrane-bound interleukin‐15 (mbIL15), and a kill switch for better precision and control in targeting relapsed or refractory AML and higher risk MDS. This first-in-human Phase 1 dose escalation study to evaluate the safety and maximal tolerated dose of PRGN‐3006 UltraCAR-T will be conducted in collaboration with Moffitt Cancer Center.

https://www.biospace.com/article/releases/precigen-announces-clearance-of-ind-to-initiate-phase-1-1b-study-for-first-in-class-prgn-3006-ultracar-t-therapy-in-patients-with-relapsed-or-refractory-acute-myeloid-leukemia-aml-and-higher-risk-myelodysplastic-syndrome-mds-/

Novartis Buys CellforCure to Manufacture CAR-T Products

Turning an existing partner into a wholly owned subsidiary, Novartis is acquiring CellforCurefrom biotechnology group LFB for an undisclosed figure.

Novartis’ Kymriah (tisagenlecleucel) is an immuno-oncology CAR-T therapy that is very labor intensive. Cancer patients’ T-cells are harvested, sent to a laboratory like CellforCure to be re-engineered, then sent back to the physician to infuse into the patient.

As PM Live notes, “Novartis said at the time of Kymriah’s approval that it would only be supplying the CAR-T for children with ALL in the first instance because of capacity issues which have plagued the product since its U.S. launch last year. It has reportedly been struggling with variability in product specifications that in some cases have led to manufacturing failures.”

CellforCure is based in France, and according to Novartis, is one of the first and biggest contract development and manufacturing organizations (CDMO) that produces cell and gene therapies in Europe. Novartis inked a deal in July 2018 with CellForCure to produce CAR-T therapies including Kymriah. They have already wrapped up technology transfer and Kymriah clinical production is expected to start by mid-2019.

This is only one of the latest manufacturing deals Novartis has signed related to cell and gene manufacturing. It also has a deal with Cellular Biomedicine Group (CBMG) to make and supply Kymriah in China, and has an expanded deal with the Fraunhofer Institute in Germany, and a contract manufacturing deal in Japan.

“The proposed acquisition of CellforCure is another strategic step in our pursuit of additional manufacturing capacity to make our transformational CAR-T cell therapy Kymriah available to more patients in need around the world,” stated Steffen Lang, Novartis Global Head of Technical Operations. “If completed, this acquisition also would potentially increase manufacturing capacity for other cell and gene therapies in the Novartis pipeline.”

CellforCure has an industrial center in Les Ulis, France, near Paris. It was founded as a pharmaceutical company in 2013, and operates as a CDMO. In 2016, it acquired two Good Manufacturing Practice (GMP) certificates from the French Agence Nationale de Securite du Medicament et des Produits de Sante (ANSM) to produce innovative experimental and commercial therapies. It will operate with Novartis’ cell and gene therapy units in Morris Plains, New Jersey, and a new $90 million facility in Stein, Switzerland that is being built and expected to begin operations in 2021.

On October 30, Novartis announced it was abandoning about 20 percent of its research projects after a strategic review. It expected to cut them from 430 to 340, particularly in the areas of infectious diseases.

Jay Bradner, president of the Novartis Institutes for Biomedical Research, told Bloomberg at the time, “The sadness about these 90 projects is there’s some great science there. These are not bad ideas. Many of them have momentum, but they either are not likely to be transformative for patients, or are ill-suited to the focused business ambitions of Novartis.”

One of the two areas the company is emphasizing is cell and gene therapies. The U.S. Food and Drug Administration approved Kymria in August 2017. In April of this year, Novartis acquired AveXis for $8.7 billion. AveXis has a gene therapy candidate, AVXS-101, for spinal muscular atrophy (SMA).

And in June, the company announced plans to spin off its eye care division, Alcon, into a separately-traded standalone company. It is also planning to divest itself of a consumer health joint venture with GlaxoSmithKline.

https://www.biospace.com/article/novartis-to-buy-kymriah-manufacturer-cellforcure/

Merck KGaA, Pfizer report Phase 3 JAVELIN Ovarian 100 study terminated

Merck KGaA (MKGAY) and Pfizer (PFE) announced that data from a planned interim analysis of the Phase 3 JAVELIN Ovarian 100 study of avelumab did not support the study’s initial hypothesis, and therefore the alliance made the decision to terminate the trial in alignment with the independent Data Monitoring Committee. The alliance between Merck KGaA – which operates its biopharmaceutical business as EMD Serono in the U.S. and Canada – and Pfizer was the first to test an immunotherapy in this indication, given the significant unmet need in the treatment of ovarian cancer. Topline results showed that the study, which is evaluating avelumab in combination with and/or following platinum-based chemotherapy in previously untreated patients with ovarian cancer, would not achieve superiority in the pre-specified primary endpoint of progression-free survival. “While detailed analyses of the data are ongoing, no new safety signals were observed, and the safety profile for avelumab in this trial appears consistent with that observed in the overall JAVELIN clinical development program. The alliance has notified health authorities and trial investigators of the interim findings and the decision to discontinue the trial. Detailed results will be shared with the scientific community. The JAVELIN Ovarian PARP 100 study and earlier phase studies investigating avelumab in various combinations are ongoing,” the companies stated.

https://thefly.com/landingPageNews.php?id=2840801