On January 6, 2020 Cellectis (Euronext Growth: ALCLS; Nasdaq: CLLS), a biopharmaceutical company focused on developing immunotherapies based on gene-edited allogeneic CAR T-cells (UCART), reported the publication of a review in Nature Reviews Drug Discovery by Prof. Stéphane Depil1*, Dr. Philippe Duchateau2, Prof. Stephan Grupp3, Prof. Ghulam Mufti4 and Dr. Laurent Poirot2 (Press release, Cellectis, JAN 6, 2020, View Source [SID1234552715]). The authors review the opportunities and challenges presented by universal allogeneic CAR T-cell therapies.

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One of the most promising approaches in cancer treatment is chimeric antigen receptor (CAR) T-cell therapy, in which part of the body’s own immunological defendors, T-cells, are redirected against cancerous cells after being engineered to express CARs. Since their initial development in the early 90s, CAR T-cells have evolved through several generations. The use of autologous (patient-derived) CAR T-cells has proven to be successful in treating people with certain blood cancers such as B-cell malignancies. However, autologous CAR T-cell therapy is not suitable for all patients, and it often requires a long and expensive manufacturing process since each treatment must be made individually for each patient.

Cellectis was the first company to develop and test an allogeneic CAR T-cell therapy in patients, where T-cells are derived from healthy donors. This gives rise to off-the-shelf product candidates which aim to be suitable for many patients as opposed to only a single person.

"We realized early on that refined gene-editing techniques were what was needed to take an allogeneic approach to CAR T-cell therapy," said Dr. Laurent Poirot, VP, Immunology Division, Cellectis. "Despite the complexity of this approach, we decided to follow this route because we are confident that it can provide the most impact for a maximum number of people living with severe cancers. This comprehensive review underlines just how much this technology has evolved in very little time. It also gives us exciting areas to explore as we continue to improve our product candidates."

One of the major challenges in the allogeneic approach involves mitigating the risk of graft-versus-host-disease (GvHD) — a medical complication that can present itself in people that have received tissues or cells from another person. The review examines aspects of this challenge and helps weigh the pros and cons associated with the different methods used to create allogeneic CAR T-cells. It also outlines some of the gene-editing work that Cellectis has done in this area along with complementary approaches being taken by others in the field, such as using cells other than conventional T-cells, also known as alpha beta T-cells.

"Our immune system, including our T-cells, is incredibly sophisticated. We know that T-cells can now be retasked to successfully fight cancer. There are amazing approaches to gene editing that are driving progress towards the most safe and efficacious versions of allogeneic products. It is exciting to see these approaches applied to ‘off the shelf’ CAR T-cell products," said Prof. Stephan Grupp, Chief of Cell Therapy and Transplant Section at the Children’s Hospital of Philadelphia, Professor of Pediatrics at the Perelman School of Medicine, and a member of Cellectis’ Clinical Advisory Board. "I’m looking forward to seeing emerging clinical data as well as even newer approaches, as Cellectis’ expertise in gene-editing technology continues to transform CAR-T".

On January 6, 2020 Inovio Pharmaceuticals, Inc. (NASDAQ:INO) reported that Inovio’s President & CEO, Dr. J. Joseph Kim will present a corporate update of the company’s clinical program goals for 2020 at the Biotech Showcase 2020 Conference in San Francisco, CA (Press release, Inovio, JAN 6, 2020, View Source [SID1234552732]).

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Biotech Showcase 2020 Conference Presentation Details:

Date:

Tuesday, January 14, 2020

Time:

10:30am (PST)

Track:

Yosemite A (Ballroom Level)

Venue:

Hilton San Francisco Union Square Hotel, 333 O’Farrell Street, San Francisco, CA

The presentation will be webcast live and may be accessed by visiting Inovio’s website at View Source Archived versions of the presentations will be made available through the Inovio Investor Relations Events page.

Inovio anticipates for 2020 to be a transformative year for the company. At the Biotech Showcase 2020 presentation, Dr. Kim will highlight multiple value-driving catalysts, clinical development, and program readouts which are all expected this year.

VGX-3100/INO-3107: HPV-Related Diseases

VGX-3100. Inovio expects to report VGX-3100 REVEAL 1 top-line efficacy data in the fourth quarter of 2020. Through extensive work on amending the clinical readout timing a year earlier than originally designed, these early top-line data will be made available without compromising the integrity of both REVEAL 1 and REVEAL 2 trials. If positive, Phase 3 top-line data could provide further regulatory validation for this first-in-class DNA Medicine for treating cervical dysplasia.
INO-3107. Inovio continues to expand its DNA Medicine franchise to treat HPV-related diseases by advancing INO-3107 to treat RRP, an orphan disease indication with a potential accelerated regulatory pathway.
In the first half of this year, Inovio plans to initiate a Phase 2 clinical trial of INO-3107 for RRP, which impacts both pediatric and adult patients. RRP is caused by HPV 6 and 11 infections, which form non-cancerous tumors in the airways of patients who suffer from this disease. Currently, the disease is incurable and can only be treated by frequent surgeries to remove the tumors, which temporarily restores the airway before renewed tumor growth.
In a previous pilot study, two adult patients with RRP had required surgery approximately every six months to clear tumor growth from their throats. Since their last dose of Inovio’s HPV product candidate, both patients have been able to avoid or significantly delay surgery. One patient has not needed surgery for almost three years as of the last follow-up. The other patient did not require surgical intervention for approximately one and a half years, a significant delay in surgery intervals prior to the trial enrollment.
Inovio believes INO-3107 could provide a novel treatment option for patients and a significant commercial opportunity for Inovio. Inovio is fully committed to bringing this product candidate to the market as soon as possible using all of the regulatory and development pathways available for rare, orphan diseases.
Dr. J. Joseph Kim, Inovio’s President & CEO said, "In July, Inovio management took the difficult but necessary step to streamline expenses and prioritize our pipeline candidates. We have also accelerated important REVEAL 1 top-line data readout to the fourth quarter of 2020, allowing the market and potential global partners to see this data sooner than expected and moved to rapidly advance INO-3107, an orphan eligible and fast-to-market product candidate.

"Looking ahead, the coming year sets up to be a transformational period for Inovio, with top-line efficacy data from our REVEAL 1 Phase 3 trial, in addition to INO-5401 Glioblastoma overall survival data at months 12 and 18 and expected MEDI0457 head and neck cancer results from AstraZeneca. These upcoming trial results in 2020 will be important drivers for the achievement of Inovio’s long-term strategy and maximizing the commercial potential of our DNA Medicine pipeline."

INO-5401/Glioblastoma Multiforme (GBM) Phase 2 Trial

INO-5401. Inovio will report 12- and 18-month overall survival data in 2020. Last year, Inovio reported promising progression-free survival at six months from its ongoing Phase 2 trial of newly diagnosed glioblastoma multiforme (GBM), which combines Inovio’s product candidates INO-5401, a T cell-activating immunotherapy encoding for three tumor-specific antigens (hTERT, WT1, and PSMA), and INO-9012, an immune activator, in combination with Libtayo, a PD-1 blocking antibody produced by Regeneron Pharmaceuticals in collaboration with Sanofi.
Key interim data from the 52-patient clinical trial showed that 80% (16 of 20) of MGMT gene promoter methylated patients and 75% (24 of 32) of unmethylated patients were progression-free at six months measured from the time of their first dose, significantly exceeding historical standard-of-care data. The majority of patients tested had a T cell immune response to one or more tumor-associated antigens encoded by INO-5401. Immune responses to all three tumor-associated antigens were demonstrated in this trial. The interim data were presented at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 2019 Annual Meeting.

INO-5151/Prostate Cancer Combination Trial

INO-5151. Parker Institute-funded cancer combination trial using INO-5151 in metastatic castration-resistant prostate cancer patients is currently enrolling. INO-5151 is a formulation that combines INO-5150 (with antigens encoding for PSA and PSMA) with INO-9012 (a T-cell activator). INO-5151 is being tested in one arm (Cohort C) of this immunotherapy combination study along with nivolumab, a PD-1 inhibitor (Bristol-Myers Squibb), and CDX-301 (Celldex Therapeutics).
Infectious Diseases/New Product Development Candidates

INO-4500. In 2020, Inovio will present Phase 1, first-in-human clinical trial evaluating INO-4500, its candidate vaccine to prevent infection from the Lassa virus. This Inovio trial conducted in the U. S. represents the first Lassa candidate vaccine to enter the clinic. This Inovio-sponsored trial, as well as its INO-4500 program, is fully funded through a global partnership with CEPI – the Coalition for Epidemic Preparedness Innovations. Inovio is also planning to advance INO-4500 to a Phase 1b trial in Africa in 2020.
INO-4700. In 2020, Inovio expects to advance INO-4700, its candidate vaccine against MERS (Middle East Respiratory Syndrome), into a Phase 2 field study in the Middle East and Africa where outbreaks have been observed, with full funding from CEPI. This is the most advanced vaccine candidate for MERS.
INO-A002. Inovio expects to report results in 2020 from its first-in-human trial of dMAb candidate INO-A002 (for preventing or treating Zika virus infection) from a Phase 1 dose-escalation trial to assess safety and tolerability and expression of dMAb-produced antibodies with full funding from the Bill & Melinda Gates Foundation. Using direct local delivery into the body, the synthetic genetic codes provided by the dMAb plasmids instruct the body’s cells to become a customized patient-specific factory that manufactures its own therapeutic monoclonal antibodies, enabling a major leap in antibody technology. With its plasmid design and in-patient production, dMAb products represent a disruptive and innovative entrant to this important class of pharmaceuticals.

On January 6, 2020 Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in RNA-targeted therapeutics, reported that Brett P. Monia, Ph.D., chief executive officer, will present a company overview at the 38th annual J.P. Morgan Healthcare Conference on Wednesday, Jan. 15, 2020 at 7:30 a.m. PT in San Francisco, CA (Press release, Ionis Pharmaceuticals, JAN 6, 2020, View Source [SID1234552765]).

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The above listed date and time are subject to change. For the latest information, please visit www.ionispharma.com.

A live webcast of the presentation will be available on the Investors & Media section of the Ionis website. The replay will be available within 48 hours and archived for a limited time.

On January 6, 2020 BridgeBio Pharma, Inc. (Nasdaq: BBIO) subsidiary QED Therapeutics reported that it has secured both Fast Track Designation in adults with first-line advanced or metastatic cholangiocarcinoma and Orphan Drug Designation for infigratinib for treatment of cholangiocarcinoma (Press release, BridgeBio, JAN 6, 2020, View Source [SID1234576234]). In addition, the company announced that enrollment is ongoing and patient dosing has started in the PROOF trial, a Phase 3 clinical trial evaluating oral infigratinib, an investigational drug, in adults for first-line treatment of advanced cholangiocarcinoma with FGFR2 (fibroblast growth factor receptor 2) gene fusions or translocations.

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Cholangiocarcinoma, a cancer of the bile ducts of the liver, is a serious and often fatal disease which affects approximately 20,000 people in the United States and European Union each year. FGFR2 genetic aberrations are present in approximately 15% to 20% of people who have this disease. Currently, treatment options are limited, and the 5-year survival rate is only 9%.1

Infigratinib received Fast Track Designation for first-line treatment of adult patients with unresectable locally advanced or metastatic cholangiocarcinoma with FGFR2 gene fusions or translocations.

"We believe that Fast Track and Orphan Drug Designations for infigratinib for the treatment of cholangiocarcinoma underscores the need for new, targeted treatments for genetically-driven subsets of this cancer, particularly for adults with first-line advanced or metastatic cholangiocarcinoma," said Susan Moran, MD, MSCE, chief medical officer for QED. "Fast Track Designation will enhance our interaction with the FDA on our first-line advanced or metastatic cholangiocarcinoma program and may help us get this medicine to patients more quickly."

The PROOF trial will enroll approximately 384 patients with first-line cholangiocarcinoma with FGFR2 fusions or translocations, as determined by molecular profiling. The primary endpoint is progression-free survival compared to standard of care chemotherapy (gemcitabine and cisplatin). Patients will be randomized 2:1 to infigratinib versus standard of care.

"Importantly, in this trial, patients who are assigned to receive standard of care will be allowed to cross over and receive infigratinib if they do not respond to chemotherapy," said Stacie Lindsey, president of the Cholangiocarcinoma Foundation. "Having a crossover option is very significant to patients and including it in the design of this trial demonstrates that QED is listening to them."

For additional information on the PROOF trial, including eligibility, patients should ask their physician, visit clinicaltrials.gov, or email [email protected]

For more information on molecular profiling, patients can find resources from the Cholangiocarcinoma Foundation at View Source

About Orphan Drug Designation

Under the FDA’s Orphan Drug Designation program, orphan drug designation is granted by the FDA to drugs or biologics intended to treat rare diseases or conditions. The designation allows the drug developer to be eligible for a seven-year period of U.S. marketing exclusivity upon approval of the drug, if the drug receives the first FDA approval for the rare disease or condition, as well as tax credits for clinical research costs, the ability to apply for annual grant funding, clinical trial design assistance, and the waiver of Prescription Drug User Fee Act (PDUFA) filing fees.

About U.S. FDA’s Fast Track Designation Program

The FDA’s Fast Track program was established to facilitate the development and expedite the review of drugs with the potential to treat serious conditions and address an unmet medical need. Companies that receive Fast Track designation are provided the opportunity for more frequent interactions with FDA during clinical development and are eligible for accelerated approval and/or priority review, if relevant criteria are met. Additionally, companies that receive Fast Track designation are allowed to submit completed sections of their New Drug Application (NDA) or Biologics License Application (BLA) for the drug on a rolling basis, resulting in the potential for an expedited FDA review process.

On January 6, 2020 Constellation Pharmaceuticals, Inc. (Nasdaq: CNST), a clinical-stage biopharmaceutical company using its expertise in epigenetics to discover and develop novel therapeutics, reported that Jigar Raythatha, CEO, will present a company overview at the J.P. Morgan Healthcare Conference at 12 noon PST/3:00 PM EST on Monday, January 13, 2020, followed by a question-and-answer session at 1:30 PM PST/4:30 PM EST (Press release, Constellation Pharmaceuticals, JAN 6, 2020, View Source [SID1234552716]).

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A live audio webcast of the presentation and archive for replay will be available on the Investor Relations section of Constellation’s website at View Source The audio webcast replay will be available for 30 days following the live presentation.