Biotech Pharma Innovation in Immuno-Oncology & beyond. Cancer - Immunology - Immunotherapy.
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After impressing at ASCO, Legend gains Johnson & Johnson as new CAR-T partner | FierceBiotech

After impressing at ASCO, Legend gains Johnson & Johnson as new CAR-T partner | FierceBiotech | Biotech Pharma Innovation in Immuno-Oncology & beyond. Cancer - Immunology - Immunotherapy. | Scoop.it
Dark horse Nanjing Legend Biotech was one of the surprising stars of ASCO this summer with its CAR-T data, and now Johnson & Johnson is paying $350 million upfront to join forces with the company and look for a working “cure” for certain blood cancers.

Back in June, Chinese firm Nanjing Legend Biotech, which many had never heard of, grabbed attention at the American Society of Clinical Oncology (ASCO) annual meeting, showing that its CAR-T candidate could be a safe and effective way to treat relapsed or refractory multiple myeloma.

Researchers reported that in an ongoing early-phase clinical trial in China, 33 out of 35 (94%) patients whose multiple myeloma had relapsed on previous treatments had clinical remission within two months of receiving Legend’s experimental anti-BCMA CAR-T cell product, dubbed LCAR-B38M, and the objective response rate was 100%.

Now J&J wants in, doing a deal with its U.S. and European subsidiaries to develop, manufacture and sell LCAR-B38M; it’s already been accepted for review by the China Food and Drug Administration (CFDA) and in “the planning phase of clinical studies in the United States for multiple myeloma,” according to the U.S. giant’s statement.
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Sanofi/Regeneron’s ‘breakthrough’ PD-1 hits positive notes in skin cancer | FierceBiotech

Sanofi/Regeneron’s ‘breakthrough’ PD-1 hits positive notes in skin cancer | FierceBiotech | Biotech Pharma Innovation in Immuno-Oncology & beyond. Cancer - Immunology - Immunotherapy. | Scoop.it
It may be way back in the race for market share, but Sanofi and Regeneron touted new topline data for their midstage checkpoint inhibitor in a certain form of skin cancer this morning.

Data out today focused on 82 patients with advanced cutaneous squamous cell carcinoma (CSCC), a form of skin cancer less deadly than melanoma.

Full details are yet to be revealed, but the pair’s PD-1 therapy showed an overall response rate (ORR) of 46.3%, although median duration of response (DOR) “had not yet been reached at the data cut-off point,” the companies said in a statement, and that 32 of 38 responses are ongoing. The test, known as EMPOWER-CSCC 1, was single arm.

The FDA breakthrough-tagged cemiplimab is currently on a rolling submission status in the U.S., which should be finished up in early 2018, using these data. EMA submission is also on course within the same period.

“EMPOWER-CSCC 1 was initiated in 2016 and has enrolled rapidly, underscoring the serious unmet need in advanced CSCC,” said Elias Zerhouni, M.D., president of global R&D at Sanofi.

“We look forward to working with regulatory agencies globally to bring this important therapy to advanced CSCC patients as quickly as possible. We continue to rapidly advance a broad development program to evaluate cemiplimab both as monotherapy and combination across a number of solid tumor and blood cancers.”
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Roche uncovers clues to improving checkpoint inhibition in cancer | FierceBiotech

Roche uncovers clues to improving checkpoint inhibition in cancer | FierceBiotech | Biotech Pharma Innovation in Immuno-Oncology & beyond. Cancer - Immunology - Immunotherapy. | Scoop.it
Checkpoint inhibitors to treat cancers, such as Roche’s Tecentriq, fail to work in as many as 80% of patients—a problem that their manufacturers have been trying to solve. Now Roche is uncovering data from a Tecentriq trial in bladder cancer patients that could provide clues to countering resistance.
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Anti-BCMA CAR T-Cell Therapy Active in Heavily Pretreated Myeloma | Cancer Network | The Oncology Journal

Anti-BCMA CAR T-Cell Therapy Active in Heavily Pretreated Myeloma | Cancer Network | The Oncology Journal | Biotech Pharma Innovation in Immuno-Oncology & beyond. Cancer - Immunology - Immunotherapy. | Scoop.it
A single treatment with a second-generation CAR T-cell treatment elicited an overall response rate of 94% in a small study of patients with heavily pretreated multiple myeloma, according to the results of a phase I (abstract 740) study presented at the 59th American Society of Hematology Annual Meeting and Exposition, held December 9–12 in Atlanta.

The treatment—called bb2121—targets B-cell maturation antigen (BCMA) to redirect T cells to myeloma cells.

“BCMA is a promising target for multiple myeloma,” said James N. Kochenderfer, MD, of the Center for Cancer Research at the National Cancer Institute in Bethesda, Maryland, who presented the results. “It is expressed nearly universally on multiple myeloma cells and its expression is restricted to plasma cells and a subset of B cells.”

The phase I, multicenter study included 24 patients who underwent leukapheresis. Manufacturing of the CAR T cells took 10 days. Three patients had clinical deterioration, which led to dosing in only 21 patients.

Treatment started at a low dose (50 million total flat dose of CAR T cells), which was escalated up in a 3 + 3 manner to 150 million, 450 million, and then 800 million. First response assessment occurred at 4 weeks. The primary objective of the study was to identify the maximum tolerated dose.
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Gilead swoops in with $567M deal to buy CAR-T 2.0 player Cell Design Labs

Gilead swoops in with $567M deal to buy CAR-T 2.0 player Cell Design Labs | Biotech Pharma Innovation in Immuno-Oncology & beyond. Cancer - Immunology - Immunotherapy. | Scoop.it
Gilead has swooped in on the first of several planned new buyouts to build out its CAR-T operations acquired in the $12 billion Kite buyout. And it's starting with a company that Kite's Arie Belldegrun believed held the key to the next-gen cell therapies that will eventually overtake the pioneers.
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CAR T-Cell Therapy Hits Prime Time; Challenges Remain

CAR T-Cell Therapy Hits Prime Time; Challenges Remain | Biotech Pharma Innovation in Immuno-Oncology & beyond. Cancer - Immunology - Immunotherapy. | Scoop.it
Chimeric antigen receptor (CAR) T cells have hit the prime time, with 2 FDA approvals already for this class of cellbased therapy. Undoubtedly this will be a game changer for patients with B-cell malignancies who have a small number of treatment options; however, questions regarding the realworld application of CAR T-cell therapies remain. Several challenges may affect the adoption of CAR T-cell therapy, which range from the logistical complexities of therapeutic development to the potentially fatal toxicities of treatment, as Stephan A. Grupp, MD, PhD, indicated yesterday in his keynote address. A number of innovations are already entering the scene, including next-generation “armored” CARs and the application of state-of-the-art genome-editing technologies. These innovations and more will be discussed during tomorrow’s plenary session entitled “Genetically Modified Cell Therapy,” starting with an explanation of gene-edited CAR T cells by Martin Pule, PhD. It will be several years before the true impact of CAR T cells becomes apparent, but it is evident that long-term success requires unlocking the potential of CAR T-cell therapy beyond B-cell malignancies, particularly in solid tumors, which account for the vast majority of cancer death
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Cancer-Fighting 'Super T-cells' to Enter Clinical Evaluation

Cancer-Fighting 'Super T-cells' to Enter Clinical Evaluation | Biotech Pharma Innovation in Immuno-Oncology & beyond. Cancer - Immunology - Immunotherapy. | Scoop.it
Researchers at Roswell Park Cancer Institute have started a clinical trial to evaluate the use of super T-cells for the treatment of solid tumor cancers.
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Novartis CAR-T cell therapy CTL019 unanimously (10-0) recommended for approval by FDA advisory committee to treat pediatric, young adult r/r B-cell ALL | Novartis

Novartis announced today that the US Food and Drug Administration (FDA) Oncologic Drugs Advisory Committee (ODAC) unanimously (10-0) recommended approval of CTL019 (tisagenlecleucel), an investigational chimeric antigen receptor T cell (CAR-T) therapy, for the treatment of relapsed or refractory (r/r) pediatric and young adult patients with B-cell acute lymphoblastic leukemia (ALL).

"The panel's unanimous recommendation in favor of CTL019 moves us closer to potentially delivering the first-ever commercially approved CAR-T cell therapy to patients in need," said Bruno Strigini, CEO, Novartis Oncology. "We're very proud to be expanding new frontiers in cancer treatment by advancing immunocellular therapy for children and young adults with r/r B-cell ALL and other critically ill patients who have limited options. We look forward to working with the FDA as they complete their review."

Acute lymphoblastic leukemia comprises approximately 25% of cancer diagnoses among children under 15 years old and is the most common childhood cancer in the US[1]. Effective treatment options for patients with r/r ALL are limited. In pediatric and young adult patients with B-cell ALL that have relapsed multiple times or become refractory to treatment, the five-year disease-free survival is less than 10-30%[2],[3],[4].

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Seattle Genetics stops all trials of '33A blood cancer drug after patient deaths 

Seattle Genetics stops all trials of '33A blood cancer drug after patient deaths  | Biotech Pharma Innovation in Immuno-Oncology & beyond. Cancer - Immunology - Immunotherapy. | Scoop.it
Seattle Genetics has called a halt to all clinical testing of vadastuximab talirine (SGN-CD33A) after seeing a higher rate of patient deaths with the drug in a phase 3 trial.
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Celgene bags option on NK cell-based blood cancer assets | FierceBiotech

Celgene bags option on NK cell-based blood cancer assets | FierceBiotech | Biotech Pharma Innovation in Immuno-Oncology & beyond. Cancer - Immunology - Immunotherapy. | Scoop.it
Celgene has landed an option on four natural killer (NK) cell-based blood cancer therapeutics in a deal with Dragonfly Therapeutics. The agreement sees Celgene hand over $33 million and commit to more in milestones to access next-generation immuno-oncology candidates aimed at some of its core therapeutic areas.

Cambridge, Massachusetts-based Dragonfly has given Celgene the exclusive option to license up to four assets designed to treat acute myeloid leukemia, multiple myeloma and other hematological cancers. The candidates will emerge from a platform that Dragonfly sees establishing NK cell alongside T cells as a critical component of the push to weaponize the immune system to defeat cancers. Celyad and Innate Pharma have landed deals on the strength of their own attempts to use NK cells.

Dragonfly’s platform generates bridges designed to bind to proteins found on the surface of tumor cells and NK cells. The aim is to stimulate NK cells. Once activated and aware of the presence of the cancer cells, NK cells attack tumors directly while also enlisting the support of T and B cells. T cells, the cornerstone of current immuno-oncology approaches, then join the direct attack on the tumor, while B cells produce antibodies to help the fight against the cancer. 
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BMS' Opdivo, Merck's Keytruda lead their class in cancer vaccine combo studies: report | FiercePharma

BMS' Opdivo, Merck's Keytruda lead their class in cancer vaccine combo studies: report | FiercePharma | Biotech Pharma Innovation in Immuno-Oncology & beyond. Cancer - Immunology - Immunotherapy. | Scoop.it
It’s no secret that combination studies featuring checkpoint inhibitors and cancer vaccines have been popping up left and right. Now, an EP Vantage snapshot shows just how many trials are underway—and which drugmakers are leading the pack.
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Cell-penetrating antibodies could crack 'undruggable' cancer mutation | FierceBiotech

Cell-penetrating antibodies could crack 'undruggable' cancer mutation | FierceBiotech | Biotech Pharma Innovation in Immuno-Oncology & beyond. Cancer - Immunology - Immunotherapy. | Scoop.it
RAS mutations have long been an attractive target in cancer treatment, but scientists have struggled to find a drug that safely combats their effects. A South Korean team has developed a way to block RAS activity in tumor cells, thus promoting anti-tumor activity.

RAS mutations, which show up in about 30% of all cancers, activate RAS proteins. Tumors that contain these aberrations tend to be aggressive and resist treatments. And while scientists have worked on this target for decades, it is still thought of as “undruggable.”

Scientists at Ajou University and Orum Therapeutics devised a monoclonal antibody, RT11-i, which targets the RAS protein in cells. After it penetrates cells, it selectively binds to activated RAS, stopping it from interacting with effector proteins and setting off a cascade of tumor-promoting signaling.

In mice with tumors containing a RAS mutation, the treatment had “measurable antitumor activity,” according to a statement. The team also administered RT11-i alongside the EGFR inhibitor cetumixab to mice with treatment-resistant colorectal cancer. The combo unlocked the resistance, suggesting a possible treatment for patients with advanced colon cancer and RAS mutations, said principal investigator and Orum cofounder Yong Sung Kim.
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AstraZeneca Shows That With Immune Cancer Drugs, No One Knows What's Next

AstraZeneca Shows That With Immune Cancer Drugs, No One Knows What's Next | Biotech Pharma Innovation in Immuno-Oncology & beyond. Cancer - Immunology - Immunotherapy. | Scoop.it
Shares in AstraZeneca are up 9% to $33.82 after the London-based drug giant announced that its cancer drug Imfinzi worked in a clinical trial to slow the progression of lung tumors in patients whose cancer was not treatable with surgery, capping off a week that's driven home a clear message about a class of cancer drugs Wall Street analysts think could be generating $30 billion in annual sales over the next decade. The message?

You puny humans have no idea what is going to happen next.

These medicines work by blocking a signal that cancer cells use to tell white blood cells not to kill them. The two leaders in the class--Opdivo from Bristol-Myers Squibb and Keytruda from Merck--block PD-1, the signal sent out by the white blood cell. Imfinzi and Tecentriq, from Roche, block PD-L1. The expectation has been that these drugs are basically the same, yet Keytruda succeeded against metastatic non-small cell lung cancer while Opdivo failed, and on Wednesday Tecentriq failed in bladder cancer, while Keytruda extended survival.

In a note to investors this morning, Timothy Anderson of Sanford C. Bernstein lays out why today's news is important to AstraZeneca. His back-of-the-envelope calculation shows that this market could be worth $1.7 billion or more for Astra, with no competitors in site for years. What's more, this figure is not included in Wall Street's $30 billion forecasts. It means that there could be uses for these drugs that nobody is considering in their sales numbers. (The flip side of those sales numbers: these drugs all cost in the neighborhood of $150,000 a year.)
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» Servier et Pfizer : résultats positifs de Phase I avec UCART19 dans la leucémie aiguë lymphoblastique 

» Servier et Pfizer : résultats positifs de Phase I avec UCART19 dans la leucémie aiguë lymphoblastique  | Biotech Pharma Innovation in Immuno-Oncology & beyond. Cancer - Immunology - Immunotherapy. | Scoop.it
Servier, Pfizer et Cellectis ont annoncé la présentation de résultats préliminaires de deux études de Phase 1 du candidat-médicament UCART19, un produit de thérapie cellulaire à base de CAR-T (chimeric antigen receptor T cell) allogéniques anti-CD19.

Ces premières données indiquent un taux de rémission complète de 83 % chez des populations de patients adultes et pédiatriques atteints de leucémie aiguë lymphoblastique (LAL) de type B, en rechute ou réfractaire (R/R). Les données des deux études ont été présentées au 59e Congrès annuel de l’American Society of Hematology (ASH) à Atlanta.

Résultats de l’essai CALM (UCART19 in Advanced Lymphoid Malignancies)

L’étude CALM (UCART19 in Advanced Lymphoid Malignancies) est une étude ouverte, d’escalade de dose, destinée à évaluer la tolérance et l’activité anti-leucémique d’UCART19 chez des patients adultes atteints d’une LAL de type B en rechute ou réfractaire. Cinq des sept patients traités ont présenté une rémission moléculaire définie par une maladie résiduelle minimale négative (MRD négative) à l’évaluation effectuée 28 jours après l’administration d’UCART19. La MRD ou minimal residual disease est une mesure du nombre de cellules leucémiques résiduelles qui persistent après traitement.

« Ces résultats sur UCART19 sont très encourageants tant par la tolérance que par le taux impressionnant de rémission moléculaire obtenu chez ces patients adultes présentant une LAL en rechute ou réfractaire », a déclaré Reuben Benjamin, investigateur principal de l’étude CALM et hématologue consultant au King’s College Hospital, au Royaume-Uni. « Cette première cohorte a exploré une faible dose d’UCART19 correspondant approximativement à un dixième de celle utilisée dans la plupart des essais CART autologues. Ces résultats justifient une poursuite de l’évaluation d’UCART19 à d’autres doses ».

Un seul cas de maladie aiguë du greffon contre l’hôte cutanée de grade 1 a éte noté (GvHD – graft versus host disease), Aucune neurotoxicité sévère n’a été observée. Les syndromes de libération de cytokines (CRS – cytokine release syndrome) étaient légers et résolutifs à l’exception d’un patient qui, après avoir été traité par UCART19 au premier niveau de dose, a développé un CRS de grade 4 dans un contexte de sepsis neutropénique entraînant le décès du patient 15 jours après l’injection de UCART19.
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Six-Month Analysis of CTL019 Demonstrates Sustained Responses in Refractory DLBCL Patients 

Six-Month Analysis of CTL019 Demonstrates Sustained Responses in Refractory DLBCL Patients  | Biotech Pharma Innovation in Immuno-Oncology & beyond. Cancer - Immunology - Immunotherapy. | Scoop.it
CTL019 (tisagenlecleucel), an investigational chimeric antigen receptor (CAR) T-cell therapy that identifies and eliminates CD19-expressing B cells, appears to be highly effective in refractory lymphoma patients. Researchers reported the latest results from the JULIET trial (ClinicalTrials.gov identifier: NCT02445248) at the 2017 annual meeting of the American Society of Hematology (ASH), held December 9-12 in Atlanta, Georgia, demonstrating high rates of complete responses at 3 months being sustained at 6 months in a cohort of highly pretreated adult patients with relapsed/refractory (r/r) diffuse large B-cell lymphoma (DLBCL).

The results confirmed earlier findings from an interim analysis. The updated analysis also demonstrated that centralized manufacturing was feasible in the first global study of CAR T-cell therapy in DLBCL. The researchers reported that cytokine release syndrome (CRS) and other adverse events (AEs) appeared to be effectively managed by appropriately trained investigators.

This current trial is a single-arm, open-label, multicenter, global, pivotal phase II trial of CTL019 in adults with r/r DLBCL. All the patients are 18 years or older with r/r DLBCL and have progressed after receiving two or more lines of chemotherapy. These patients also were ineligible for autologous stem cell transplant or had failed it.

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Landscape of Immuno-Oncology Drug Development

Landscape of Immuno-Oncology Drug Development | Biotech Pharma Innovation in Immuno-Oncology & beyond. Cancer - Immunology - Immunotherapy. | Scoop.it
With an unprecedented number of new investigational agents and companies in immuno-oncology (IO), it has been difficult to track and understand the current IO agents in clinical development and the clinical trials testing these agents. The Clinical Accelerator team presents an unbiased, neutral, scientifically curated, and timely updated analysis based on information collected from numerous trusted and publicly available sources. We hope to inform the cancer community as they seek to strive for efficiencies and innovation while avoiding duplication. For further detailed analysis, see our paper in Annals of Oncology.


The overview of 2,004 IO agents. 6 classes of IO agents are identified on the basis of different mechanisms of actions. Unspecified tumor associated antigen (Unspecified TAA) is defined as the target of unspecified antigens from either individual patients or cell lines. In this sense, therapies against this target mainly consist of cell line-based vaccines, autologous tumor vaccines, autologous DC vaccines, and tumor infiltrating T cell therapies (TIL). Personalization is defined as the targets discovered by algorithm-based approaches from, e.g., the individual patients’ tumor samples or blood circulating DNA.
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FDA Cancer Drug Approvals in 2017 | Cancer Network | The Oncology Journal

FDA Cancer Drug Approvals in 2017 | Cancer Network | The Oncology Journal | Biotech Pharma Innovation in Immuno-Oncology & beyond. Cancer - Immunology - Immunotherapy. | Scoop.it
This slide show highlights some of this year’s FDA approvals of cancer treatments, including new therapies and indications for breast cancer, lung cancer, and various blood cancers, as well as the first approval based not on disease site but by tumor marker.
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How F-star is supercharging the immune system in fight against cancer

How F-star is supercharging the immune system in fight against cancer | Biotech Pharma Innovation in Immuno-Oncology & beyond. Cancer - Immunology - Immunotherapy. | Scoop.it
An exclusive interview with CEO John Haurum about F-star’s extraordinary work on Babraham Research Campus
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Promising Anti-Tumor Activity Seen in CBT-502, a PD-L1 Antibody

Promising Anti-Tumor Activity Seen in CBT-502, a PD-L1 Antibody | Biotech Pharma Innovation in Immuno-Oncology & beyond. Cancer - Immunology - Immunotherapy. | Scoop.it
CBT Pharmaceuticals‘ anti-PD-L1 antibody CBT-502 has shown promising immune activation and inhibition of tumor growth in preclinical models of cancer, according to new preclinical data.

The data were presented in a poster titled, “CBT-502 (TQB2450), a novel anti-PD-L1 antibody, demonstrates favorable activity in MC-38/H-11 murine colon and A375 human melanoma animal models,” at the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics in Philadelphia.

Studies showed that this new drug candidate stimulates the production of the cytokines interleukin-2 (IL-2) and interferon gamma (IFN-gamma), leading to suppression of tumor growth in models of colon cancer and melanoma. It produced these results with a similar effectiveness as Tecentriq (atezolizumab), another PD-L1 inhibitor.

“The in-vitro characteristics and in-vivo efficacy studies showing linear pharmacokinetics suggest that CBT-502 may provide benefit in a variety of tumor types,” Sanjeev Redkar, PhD, president and chief executive officer at CBT, said in a press release.
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FDA hits Cellectis’ off-the-shelf CAR-T program with clinical hold after first patient treated in phase 1 trial dies | FierceBiotech

FDA hits Cellectis’ off-the-shelf CAR-T program with clinical hold after first patient treated in phase 1 trial dies | FierceBiotech | Biotech Pharma Innovation in Immuno-Oncology & beyond. Cancer - Immunology - Immunotherapy. | Scoop.it
The FDA has placed a clinical hold on two phase 1 trials of Cellectis’ UCART123 after learning of the death of one patient. Development of the off-the-shelf CAR-T therapy is now in limbo while Cellectis works with the FDA to redesign the protocol to mitigate the risks identified in the first weeks of the trials.

Doctors at the MD Anderson Cancer Center dosed the first blastic plasmacytoid dendritic cell neoplasm (BPDCN) patient with CD123-targeting CAR-T UCART123 on August 16. The patient died nine days later. 

Initially, the 78-year-old man responded to the lowest dose of UCART123 without complication. On day five the patient suffered a grade 2 cytokine release syndrome (CRS) and grade 3 lung infection. On day eight he experienced a grade 4 capillary leak syndrome (CLS) and a CRS that, despite treatment with corticosteroids and tociluzumab, played a central role in his death the next day.

The only patient treated with UCART123 in the other phase 1 trial experienced similar, albeit less severe, reactions. That patient, a 58-year-old woman with acute myeloid leukemia (AML), suffered a grade 3 CRS and grade 4 CLS nine days after treatment with UCART123. The adverse events put the patient in intensive care but had cleared up by day 12.
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FDA raises big safety issues in Novartis’ CAR-T review, but spotlights post-marketing path forward

FDA raises big safety issues in Novartis’ CAR-T review, but spotlights post-marketing path forward | Biotech Pharma Innovation in Immuno-Oncology & beyond. Cancer - Immunology - Immunotherapy. | Scoop.it
The FDA’s in-house review of Novartis’ groundbreaking CAR-T therapy tisagenlecleucel (CTL019) came out this morning, carefully outlining the efficacy tracked in Phase II and honing in on the agency’s big concern: How do experts in the field view the serious safety issues remain and what would be the best way to track and contain them once marketing begins?

“The overall effectiveness of this product is not the primary issue for consideration by this Committee,” states the review document. But the Phase II study in patients with B-cell acute lymphoblastic leukemia raised a host of issues on short-term and potentially lethal dangers, from a high though familiar rate of cytokine release syndrome to the neurological conditions that CRS can trigger.

The FDA, while taking a measured look at the dangers without condemning them, is also clearly alarmed about the prospect of long-term risks that have yet to be fully explored – including the risk of new malignancies. But the agency couched the discussion on a risk-mitigation strategy which experts will be expected to weigh in on, keeping the focus on an approval.
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How will Novartis price its groundbreaking CAR-T med? R&D exec offers some clues | FiercePharma

How will Novartis price its groundbreaking CAR-T med? R&D exec offers some clues | FiercePharma | Biotech Pharma Innovation in Immuno-Oncology & beyond. Cancer - Immunology - Immunotherapy. | Scoop.it
The FDA isn't the only hurdle for Novartis' forthcoming CAR-T therapy, which could be the world's first. There's pricing and access, too. Drugs in this new class of one-time treatments are expected to be ultra-expensive, so the company is working up pricing and payment strategies now.

Novartis’ CTL019 is under review to treat pediatric and young adult relapsed and refractory B-cell acute lymphoblastic leukemia (ALL), with the FDA’s action date set for early October. In the meantime, it's using stem cell transplants as a benchmark for pricing and considering pay-for-performance deals to make the medications accessible for payers and patients alike, R&D chief Vas Narasimhan told Bloomberg.

CAR-T therapies are individualized treatments manufactured from a patient's own T cells, with the promise of a cure. As one-time treatments, just as gene therapies are, they're expected to be among the world's most costly drugs if approved. Novartis is in a hot race to approval with Kite Pharma, with other would-be CAR-T products coming up behind.
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Manufacturing costs loom large for personalized CAR-T cancer meds and their need for speed

Manufacturing costs loom large for personalized CAR-T cancer meds and their need for speed | Biotech Pharma Innovation in Immuno-Oncology & beyond. Cancer - Immunology - Immunotherapy. | Scoop.it
Manufacturing quality and costs always play some part in the cost of a new drug, but they are the elephant in the room when looking at CAR-T, personalized cancer medications now under development. That is because of the extensive process to genetically code a patient’s T cells to hunt and kill cancer cells, along with the need for speed.  

Instead of grabbing a medicine off the shelf or even infusing a mass-produced biologic at a doctor’s office, the process requires that blood be taken from a patient, cryopreserved, shipped to a facility, reprogrammed and manufactured in the lab, and then shipped back for infusion into the patient, all in the shortest time possible to try to outrun aggressive cancers.

Novartis and Kite, which have the two leading CAR-T candidates, have not provided much insight into pricing. They have, however, given glimpses into their manufacturing and the turnaround times they will need to treat patients for lymphomas.

RELATED: How will Novartis price its groundbreaking CAR-T med? R&D exec offers some clues

Novartis talked about manufacturing after it released data on its candidate last week, which will be reviewed by an advisory committee July 12. Novartis officials said that the Swiss company expects to have the turnaround time for its treatment down to 22 days at the time of commercial launch, with 10 to 12 of those days being “the actual cell processing time.”

RELATED: As Novartis preps for first CAR-T FDA AdComm, Juliet data revealed

Santa Monica, California-based Kite says its time is even shorter, although not the 14 days it had initially striven for. “Our manufacturing process delivery time—what we call vein to vein—is between 16 to 18 days,” a spokeswoman said in an email. “This is from the time of leukapheresis to when the cells are back with the clinician for infusion into the patient. This includes 6 to 7 days needed for the cell engineering, as well as quality control and travel time.”

She said the biotech is looking for ways to speed the process further and points out that its manufacturing success rate during clinical trials was 99%.

Novartis is claiming that with continuous process improvements over the course of its Juliet trial, its manufacturing success rate improved to 97% for the last 30 patients. That followed some early issues in which 9 of 141 patients, or 6%, had to discontinue treatment due to “an inability to manufacture an adequate dose of CAR-T cells.”

The company says it has gotten on top of the complicated process. It is now being overseen by Spencer Fisk, who took over as global head cell & gene technical development and manufacturing in Morris Plains, New Jersey, after Karen Walker departed several months ago for a position with Seattle Genetics.

“As for manufacturing, we are confident we will be able to meet the required manufacturing demands moving forward,” a Novartis spokesperson told FierceBiotech last week.

Novartis also has an agreement with the Fraunhofer Institute for Cell Therapy and Immunology for the cell processing of its personalized T cell therapy in Europe.

Kite is producing its chimeric antigen receptor (CAR) and T-cell receptor (TCR) candidate in a new 43,500-square-foot plant it purposely built next to an airport. The plant is estimated to have the capacity to produce up to 5,000 patient therapies per year.

RELATED: Kite Pharma opens CAR-T cell manufacturing plant next to LA airport

Novartis converted to its use a plant built by the former Dendreon, which used a similar process of altering cells for its once-promising prostate cancer vaccine Provenge. Dendreon sold its 173,000-square-foot plant in Morris Plains, one of three, to Novartis in 2012 for $43 million to pick up some fast cash.

Dendreon’s experience hangs over this new manufacturing method since the company went bankrupt trying to establish Provenge. Its cost of goods at one point was 77%, before it dropped that to about 53% with process changes. That compares to about 10% for many biotechs.

RELATED: Dendreon, done in by expensive, complex manufacturing, files for bankruptcy

While Dendreon failed to find a price point to compete with established, mass-produced drugs, makers of the new cancer drugs will have the luxury of setting a foundational price for their treatments, which have the potential to be curative.

Neither Kite or Novartis have offered any hard guidance on pricing, but Novartis R&D chief Vas Narasimhan has told Bloomberg that stem cell treatment prices could offer a benchmark. Stem cell transplants and follow-up treatment can cost upwards of $800,000. He also said the company is looking at pay-for-performance deals.
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As Novartis preps for first CAR-T FDA AdComm, Juliet data revealed | FierceBiotech

As Novartis preps for first CAR-T FDA AdComm, Juliet data revealed | FierceBiotech | Biotech Pharma Innovation in Immuno-Oncology & beyond. Cancer - Immunology - Immunotherapy. | Scoop.it
It’s been a big week for CAR-T at ASCO, and now Novartis has revealed the long-awaited Juliet data for its CAR-T candidate at a Swiss conference.
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Has Merck Lucked Into A $10 Billion Drug?

Has Merck Lucked Into A $10 Billion Drug? | Biotech Pharma Innovation in Immuno-Oncology & beyond. Cancer - Immunology - Immunotherapy. | Scoop.it
Merck–and its cancer drug Keytruda–had a great day yesterday.

First, rival drug giant Roche revealed that a clinical trial failed to show its Tecentriq, a drug similar to Keytruda, extended the lives of patients with bladder cancer. Keytruda, by contrast, had added a median 3.1 months to patients' lives in a similar study. Then, late last night, Keytruda was approved as a first choice for most patients with non-small cell lung cancer when used in combination with a chemotherapy regimen that includes Eli Lilly’s Alimta.

In the last 12 months Merck has gone from a runner up in the lucrative market to the potential leader. Wall Street analysts forecast that drugs like Keytruda, called checkpoint inhibitors, will reach sales of $30 billion by 2022, with most of the sales going to Merck and its biggest rival, Bristol-Myers Squibb. Gains could happen at breakneck speed. Keytruda sales should increase from $584 million in the first quarter of the year to $777 million in the second, $958 million in the third and $1.2 billion in the fourth. In 2018, sales of Keytruda could approach $6 billion, analysts predict.
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