Novartis-backed cell therapy startup Gamida files for IPO

Gamida Cell has filed to raise $69 million in a Nasdaq IPO. The Novartis-backed cell therapy player wants the money to wrap up a phase 3 trial of its lead candidate in patients with blood cancers.

Cell therapies, in the form of hematopoietic stem cell transplantations (HSCT), are already widely used in the treatment of blood cancers such as acute myeloid leukemia. However, the number of patients who receive HCST is limited by a lack of donor-matched cells. Haploidentical donors and umbilical cord blood partly make up for the shortfall in donor-matched cells, but Gamida still thinks 40% of patients eligible for HSCT never receive the treatment.

Gamida wants to enable these patients to access cell therapy treatments. The biotech’s answer to the problem, NiCord, is made by applying a nicotinamide-based cell expansion technology to cord blood. Gamida thinks the result is an off-the-shelf treatment consisting of cells that quickly engraft and largely avoid attack by the host immune system.

Israel-based Gamida is enrolling patients in a phase 3 trial to test the idea. With the trial not due to deliver top-line data until the first half of 2020 and Gamida’s cash reserves dipping below $30 million, the biotech is seeking up to $69 million from public investors to complete the study.

Some of the money will go toward the build-out of a manufacturing plant in Israel. Manufacturing has posed challenges for the company. During a recent audit of Gamida’s contract manufacturer, Israeli inspectors made critical observations. Gamida is working with the manufacturer to fix the issues but is yet to get the all-clear from regulators.

Novartis was set to buy Gamida for $170 million upfront in 2014, but the deal fell through. Later that year, Novartis invested $35 million in Gamida and secured an option to buy the biotech outright in 2016. However, Novartis turned down the option 10 months later, well before it was due to expire.

The Swiss Big Pharma has continued to invest in Gamida, though. Novartis invested $5 million late in 2015—after turning down its option—and put a further $8 million into Gamida last year. The rounds left Novartis owning more than one-fifth of Gamida, making it the biotech’s biggest shareholder.

https://www.fiercebiotech.com/biotech/novartis-backed-cell-therapy-startup-gamida-files-for-ipo

Trump Signs Funding Bill Giving NIH $2B Boost in FY19

President Donald Trump on Friday signed into law an appropriations package that included a $2 billion budget increase in fiscal year 2019 for the National Institutes of Health.

The bipartisan legislation increases the NIH’s budget by 5.4 percent over its fiscal 2018 funding level to $39.1 billion. The bill passed the House by a vote of 361 to 61 and passed the Senate in a 93-to-7 vote. It specifically includes an additional $425 million for Alzheimer’s disease research for a total of $2.34 billion; a total of $429.4 million for the BRAIN initiative, a $29 million increase; and $376 million for the All of Us precision medicine study, $86 million more than in FY 2018.

https://www.genomeweb.com/policy-legislation/trump-signs-funding-bill-giving-nih-2b-boost-fy19

Perrigo confirms patent challenge for generic version of Jublia

Perrigo Company announced that Valeant Pharmaceuticals North America, Valeant Pharmaceuticals Ireland, Dow Pharmaceuticals Sciences and Kaken Pharmaceuticals initiated patent litigation on September 21, 2018 in the United States District Court for the District of New Jersey regarding Perrigo’s Paragraph IV Abbreviated New Drug Application for efinaconazole topical solution 10%, asserting patents listed in the Orange Book for Jublia. This action formally initiates the litigation process under the Hatch-Waxman Act. Jublia Topical Solution 10%, is indicated for the topical treatment of onychomycosis of the toenails due to Trichophyton rubrum and Trichophyton mentagrophytes. Annual market sales for the 12 months ending July 2018 were $276M as measured by IQVIA.
https://thefly.com/landingPageNews.php?id=2797857

Seattle Genetics, Takeda: Positive Phase 3 T-Cell Lymphoma Med Results

-ADCETRIS in Combination with Chemotherapy Achieved Primary Endpoint, Demonstrating a Statistically Significant Improvement in Progression-Free Survival Compared to a Standard of Care Chemotherapy-

-Statistically Significant Improvement Achieved in All Key Secondary Endpoints, Including Overall Survival-

-First Randomized Phase 3 Trial to Show Improvement in Overall Survival in Frontline Peripheral T-Cell Lymphoma-

-Data to be Presented at the 2018 ASH Annual Meeting; Global Regulatory Submissions Planned-

Seattle Genetics, Inc. (Nasdaq:SGEN) and Takeda Pharmaceutical Company Limited (TSE:4502) announced today that the phase 3 ECHELON-2 clinical trial met its primary endpoint. The trial demonstrated a statistically significant improvement in progression-free survival (PFS) of ADCETRIS (brentuximab vedotin) in combination with CHP (cyclophosphamide, doxorubicin, prednisone) versus the control arm, CHOP (cyclophosphamide, doxorubicin, vincristine, prednisone). ECHELON-2 is a global, randomized, double-blind, multicenter trial evaluating ADCETRIS as part of a frontline combination chemotherapy regimen in patients with previously untreated CD30-expressing peripheral T-cell lymphoma (PTCL), also known as mature T-cell lymphoma (MTCL). ADCETRIS is an antibody-drug conjugate (ADC) directed to CD30, which is expressed on the surface of several types of PTCL. ADCETRIS is currently not approved for the frontline treatment of PTCL.

Patients in ECHELON-2 were randomized to receive either a combination of ADCETRIS plus CHP or CHOP, a recognized standard of care for frontline PTCL. Results from the trial demonstrated that combination treatment with ADCETRIS plus CHP was superior to the control arm for PFS as assessed by an Independent Review Facility (IRF; hazard ratio=0.71; p-value=0.0110). The ADCETRIS plus CHP arm also demonstrated superior overall survival (OS), a key secondary endpoint, compared to CHOP (hazard ratio=0.66; p-value=0.0244). All other key secondary endpoints, including PFS in patients with systemic anaplastic large cell lymphoma (sALCL), complete remission rate and objective response rate were statistically significant in favor of the ADCETRIS plus CHP arm. The safety profile of ADCETRIS plus CHP in the ECHELON-2 trial was comparable to CHOP and consistent with the established safety profile of ADCETRIS in combination with chemotherapy. Additional data will be presented at the American Society of Hematology (ASH) 2018 annual meeting, December 1-4, 2018, in San Diego, California.

“Peripheral T-cell lymphoma is an aggressive type of non-Hodgkin lymphoma with approximately 4,000 CD30-expressing patients diagnosed every year in the United States,” said Clay Siegall, Ph.D., President and Chief Executive Officer of Seattle Genetics. “We are excited about the groundbreaking results of the phase 3 ECHELON-2 clinical trial, which demonstrated ADCETRIS in combination with chemotherapy significantly improved treatment outcomes for adult patients with previously untreated CD30-expressing PTCL compared with the current standard of care (CHOP). We’d like to thank the many investigators and patients who participated in this study and contributed to this significant milestone for the PTCL community. We look forward to presenting results at the ASH annual meeting in December and intend to submit a supplemental Biologics License Application to the FDA for approval in this setting in the near future.”

“These clinically meaningful results from ECHELON-2 represent a significant step in the development of a potential frontline treatment in this disease. This trial is the largest randomized, double-blind, phase 3 trial in PTCL,” said Jesús Gomez-Navarro, M.D., Vice President, Head of Oncology Clinical Research and Development, Takeda. “Standard of care in PTCL has not changed in several decades and there remains an unmet need for patients. These data showed a significant improvement in the primary endpoint of progression-free survival and all key secondary endpoints, including overall survival, along with a manageable safety profile. We look forward to sharing these data with regulatory authorities globally.”

Takeda and Seattle Genetics plan to submit these results to regulatory authorities for approval in their respective territories.

https://www.biospace.com/article/releases/seattle-genetics-and-takeda-announce-positive-results-from-phase-3-echelon-2-clinical-trial-evaluating-adcetris-brentuximab-vedotin-in-frontline-cd30-expressing-peripheral-t-cell-lymphoma/

Pfizer Announces Ian Read’s Replacement and Succession Plan

Pfizer announced its succession plan for when Ian Read steps down as chief executive officer on January 1, 2019. The new chief executive officer will be Albert Bourla, who is currently the company’s chief operating officer. Read will stay on as executive chairman of the board of directors.

Read became Pfizer’s chief executive on December 6, 2010, and chairman on December 12, 2011. Under his leadership, the company has marked up 30 drug approvals in the U.S., a total shareholder return of 250 percent, and a direct return of capital to shareholders of more than $120 billion.

He also unsuccessfully attempted two mega-mergers. The first was a 2014 bid to buy UK-based AstraZeneca for $119 billion. This fell apart primarily due to resistance by both the U.S. and UK governments to the deal. In 2016, an attempt to acquire Dublin-based Allergan for $160 billion was dashed when the U.S. Treasury Department implemented new rules regarding tax inversions. A tax inversion is where a U.S. company buys a company based in another country with a very low corporate tax rate, then shifts their headquarters to that country to take advantage of the lower rate. It was estimated that the Allergan deal would have resulted in a $1 billion lower tax rate at the time for Pfizer.

Before taking on the role of chief operating officer on January 1, 2018, Bourla led Pfizer’s Innovative Health business. He also founded the Innovative Health Emerging Markets region, which reported revenues in 2017 of $4.4 billion. Before taking on that role, he was the group president of the Vaccines, Oncology and Consumer Healthcare business unit.

Shantanu Narayen, Lead Independent Director of Pfizer’s board of directors, stated, “Today’s leadership announcement is part of a thoughtful, multi-year success planning process. The board has been impressed with Albert’s performance, depth of experience and track record for success, and we are confident that as chief executive officer he will drive innovation and further advancements across the business.”

The news follows last week’s announcement by Merck & Co that they had rescinded the company’s mandatory retirement age of 65 for its chief executive officer, Kenneth C. Frazier, so he could stay on longer. Leslie A. Brun, lead director for Merck’s board, stated, “CEO succession has been our top priority, and removing the mandatory retirement policy enables the board to make the best decision concerning the timing of the transition.”

Depending on the source, Read is either 64 or 65. Earlier this year, Pfizer offered Read a bigger payday to keep him around until at least March 31, 2019, and also to keep him from working for a competing company through March 31, 2021. This involved a 61 percent increase in compensation to $27.9 million.

In today’s announcement, Read stated, “It’s been an honor to serve as Pfizer’s CEO for the past eight years. However, now is the right time for a leadership change, and Albert is the right person to guide Pfizer through the coming era. Albert is an energizing leader who has an unwavering commitment to serving patients. With 25 years at Pfizer, he has developed an extensive knowledge of the industry and demonstrated an ability to build and grow businesses. With Albert at the helm, our dedicated colleagues across the globe are poised to deliver the next stage of growth. I look forward to working with Albert and the board to continue serving patients and delivering value for shareholders.”

https://www.biospace.com/article/pfizer-announces-ian-read-s-replacement-and-succession-plan/

Dragonfly and Merck Strike $695 Million Deal for Solid Tumors

Waltham, Mass.-based Dragonfly Therapeutics struck a licensing agreement worth up to $695 million with Merck. Merck will license exclusive rights to Dragonfly’s TriNKET technology platform for a number of solid-tumor programs, the company announced today.

Joe Miletich, Merck’s head of discovery and preclinical development, said Dragonfly’s technology platform provides a potential for researchers to harness the power of NC cell receptors. That could lead to the development of novel therapeutics targeting solid tumor indications, Miletich said.

Bill Haney, Dragonfly’s chief executive officer, called Merck a “world leader” in solid tumor cancers. Haney said the company has a history of delivering breakthrough therapy options for patients. He pointed to the company’s TriNKET technology platform as a potential accelerator of developing new drug options.

Dragonfly’s TriNKET (Tri-specific, NK cell Engager Therapies) bind to the proteins expressed on both cancer cells and NK cells. The TriNKET system provides “an active connection between cancer cells, and cells of the immune system including NK cells themselves, T cells, B cells, and other cells that help attack and kill cancer,” according to the company. NK cells are part of the natural immune system of every human body. They also express proteins on their surface.

When the binding occurs, Dragonfly said its TriNKET system stimulates NK cells, which makes them aware of cancerous cells. That stimulation allows the NK cells to both kill the cancer cells, as well as notify other immune cells to attack the cancer. In other words, Dragonfly believes that linking to the natural killer cells and sending them to the cancer cells, it will increase the chances of eliminating the tumors.

“Overall, NK cells amplify the effectiveness of T-cells, acting as a sentinel that calls other immune system cells to attack the cancer, as well as broadening the therapeutic window by using their special characteristics of distinguishing cancer, to more specifically target tumor cells,” Dragonfly said on its website.

Under terms of the agreement, Merck will pay Dragonfly up to $695 million in upfront and milestone payments per program as well as royalties on sales of approved products.

Merck isn’t the first company that Dragonfly has collaborated with. Last year the company struck a five-year agreement with Celgene to develop immuno-oncology therapeutics for hematological cancers based on Dragonfly’s TriNKET technology platform.

Dragonfly was launched in 2015. The company was founded by Tyler Jacks, director of MIT’s Koch Institute, along with Haney, and Natural Killer cell expert, David Raulet. Dragonfly’s own research is still in the preclinical phase. On its website, the company lists a number of assets but does not provide information about the different indications the company is targeting.

https://www.biospace.com/article/dragonfly-and-merck-strike-695-million-deal-for-solid-tumors/

Piper keeps Overweight on Regeneron after Libtayo approval

Piper Jaffray analyst Christopher Raymond keeps an Overweight rating on Regeneron Pharmaceuticals (REGN) after Friday’s FDA approval of Libtayo in cutaneous squamous cell carcinoma. Timing is about a month ahead of the late-October FDA action date for Regeneron/Sanofi’s (SNY) PD-1 inhibitor, but a positive decision was expected given the drug’s “compelling data” in advanced CSCC, Raymond tells investors in a research note. While Libtayo will enter the market as the sixth approved PD-(L)1 inhibitor and third approved PD-1 agent, Raymond thinks readouts from the ongoing development program in other indications such as first-line non-small-cell lung carcinoma may make the case for moderate Libtayo uptake.
https://thefly.com/landingPageNews.php?id=2797409