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This newsletter provides commercial insights into the ongoing development of the gene therapy market. The most noteworthy stories appear at the top along with our commentary.
Connect with Us at #JPM18
RxC International will be at the JP Morgan conference and surrounding events. To schedule 20 minutes discussing gene therapy commercialization and access issues, contact us here.
We are expecting a few gene therapy points of interest to emerge from JMP 2018, including:
- Pricing discussions
- Regulatory Approval issues and timing
- Commercialization challenges and market access strategies
1) First Mover Advantage? Gilead Blazes Trail for Competition
While having the first-mover advantage is traditionally considered a boon in the pharmaceutical industry, in Yescarta’s case, any advantage is disappearing with delays in access as Gilead builds reimbursement infrastructure that benefits the competition and allows increased time for competitors to launch.
Conservative Wall Street uptake forecasts that revenues would exceed $9 million in 2017, the equivalent of treating roughly 25 patients, weren’t met. Gilead is a highly capable commercial organization that has faced access and pricing challenges before but this case demonstrates the importance of careful commercial planning when developing high cost gene therapies both for realizing returns on investments and ensuring that patients who desperately need these treatments receive them in time to make a difference.
Hundreds of cancer patients sit on waiting lists to receive Yescarta but only 5 have been treated since this potentially life-saving gene therapy’s approval in October. The new treatment has no billing code for Medicare/Medicaid recipients, forcing hospitals to choose between leaving dying patients untreated or risking millions of dollars that may not be reimbursed. Insurance companies have also been slow to adopt the treatment.
- SFGate: Patients die as Gilead’s breakthrough cancer drug reaches only a few
- Bloomberg: Novel Cancer Drug Gets Stymied by Medicare Rigid Billing System
2) Newly-Published Yescarta Study Raises Safety Concerns
Gene therapy companies must develop a distribution model that addresses safety concerns while supporting commercialization efforts. Since many gene therapy products could require specialized capabilities, treatment centers of excellence are likely to be an important element to commercial planning.
A study published this month in the New England Journal of Medicine highlights the success of Yescarta (axicabtagene ciloleucel) in treating diffuse large B-cell lymphoma but also reveals that 95% of those who received Yescarta experienced severe effects and that 13% of those side effects were classified as life-threatening. The FDA’s approval of Yescarta included a risk evaluation and mitigation strategy (REMS) that requires hospitals and staff to be specially certified to dispense or administer Yescarta.
While safety needs demand additional research, the study indicated that even after 15 months 42% of patients who received Yescarta are still responding to treatment.
- New England Journal of Medicine: Axicabtagene Ciloleucel CAR T-Cell Therapy in Refractory Large B-Cell Lymphoma
- Food and Drug Administration: FDA approves CAR-T cell therapy to treat adults with certain types of large B-cell lymphoma
3) Results from Hemophilia Gene Therapy Studies Prompt Pricing Questions...Again
While certainly to be priced extremely high, a gene therapy that cures Hemophilia offers significant savings over the lifetime of a hemophilia sufferer given that hemophilia patients require lifelong treatments that on average cost $155,136, but a patient using inhibitors can cost $400,000+ a year.
Pricing a cure for Hemophilia raises the question of if there’s a maximum that health systems are willing to pay for a treatment. Depending on assumptions, the present value of a lifetime worth of therapy can easily exceed $3 million and be as high as $7 million. How much is a cure worth for a patient? Should therapy cost differ based on patient age?
The absence of long-term data throws another wrench into pricing considerations. Without information on the longevity of a Hemophilia gene therapy, payers won’t even know if treatments truly are lifelong.
One important aspect of commercializing a Hemophilia cure, no matter what the final price might be, is communicating its value to stakeholders and ensuring that there is transparency around pricing.
The recent success of two Hemophilia studies offers hope to patients who undergo frequent infusions. Results from BioMarin’s study for a gene therapy to treat Hemophilia A, the most common type of Hemophilia, showed that all 9 patients stopped using clotting factors; bleeding events also fell dramatically. “[Patients’] bleeding rates collapsed to zero or nearly zero and we’ve improved their quality of life beyond recognition.”
- New England Journal of Medicine: A Cure for Hemophilia within Reach
- Forbes: Excitement Builds Around Gene Therapy Cures For Hemophilia
1) FDA Approves Spark Therapeutics’ Blindness Treatment
The FDA approved the third ever gene therapy, Luxturna (voretigene neparvovec-rzyl), to treat children and adults with hereditary retinal dystrophy. The disease causes vision loss and may lead to blindness.
- Food and Drug Administration: FDA approves novel gene therapy to treat patients with a rare form of inherited vision loss
2) Genetic Differences Could Impact Efficacy and Safety of CRISPR
Gene editing technology may need to be tailored to each patient’s specific genome sequence to maximize treatment effectiveness and prevent side effects.
- Genetic Engineering and Biotech News: Genetic Differences Could Impact Efficacy and Safety of CRISPR
3) CRISPR may Treat Hearing Loss
Scientists successfully restored hearing in animals by injecting CRISPR-Cas9 into mice cochlear hairs.
4) Gene Therapy Restores Functional Hemoglobin for Sufferers of Sickle Cell Disease
LentiGlobin gene therapy may enable those with sickle cell disease to forego transfusion, potentially outperforming the yet-to-be-approved drug therapies crizanlizumab (Novartis) and voxelotor (Global Blood).
5) From Hospice to Hope: Encouraging Results for Myeloma Gene Therapy Treatment
Bluebird Bio announced encouraging Phase 1 dose-escalation study results for patients suffering from advanced multiple myeloma. After receiving bb2121, 94% responded to the treatment and 56% remained in remission. Some patients were headed to hospice before receiving treatment. "This is unheard of, something that we haven't seen with any drugs approved for myeloma in this type of population. The excitement among all the myeloma providers is crazy."
- Business Insider: 'Unheard of' responses to bluebird CAR-T therapy seen in myeloma study
6) Bypassing the Blood-Brain Barrier for Neurological Diseases
Genetically engineered microglia, brain immune cells, inserted into brain ventricles may regenerate brain tissue and treat brain diseases, say researchers. Injecting cells directly into the brain allows them to more quickly multiply and begin repairing damaged tissue.
- Science Advances: Intracerebroventricular delivery of hematopoietic progenitors results in rapid and robust engraftment of microglia-like cells
- Futurism: New Gene Therapy Could Regenerate Brain Tissue, Fix Neurological Diseases
7) Clinical Trials in Sight for Duchenne Muscular Dystrophy Gene Therapy
By replacing a faulty gene with an engineered one, a researcher believes he can treat Duchenne muscular dystrophy. Solid Biosciences licensed SGT-001 and has begun clinical trials for the treatment.
- University of Missouri School of Medicine: Gene therapy for Duchenne muscular dystrophy advances toward clinical trials
8) CAR-T Therapy Updates
- New RNA Gene Therapy for Killing Cancer May Enhance CAR-T Treatments
- New Haven Register: Yale doctor creates new weapons to kill cancer
- CAR-T Used to Treat Thyroid Tumors
- American Association for Cancer Research: CAR T Therapy Targeting ICAM-1 Eliminates Advanced Human Thyroid Tumor
- Acute Lymphoblastic Leukemia Study Achieves 71% Remission Rate
9) New Viral Vectors May Prevent Immune Response to Gene Therapy
Recent research has shown promise in using new viral vectors to prevent gene therapy immune responses, which may neutralize vectors and pose other hazards to patients.
- Genetic Engineering and Biotech News: Synthetic Viral Vectors Show Promise for Mitigating Immune Response to Gene Therapy
1) Gilead Buys Cell Design
- Fierce Biotech: Gilead makes bolt-on CAR-T buy after Kite deal
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