On January 30, 2020 University of Pennsylvania reported a scientific team is developing a new CAR T-cell gene therapy treatment for advanced metastatic prostate cancer with a $500,000 grant from Alliance for Cancer Gene Therapy (ACGT) (Press release, University of Pennsylvania, JAN 30, 2020, View Source [SID1234553699]).

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The ACGT grant was awarded to Joseph Fraietta, PhD, assistant professor of microbiology and a T-cell biologist with expertise in tumor immunology and translational medicine, and Naomi Haas, MD, director of the Prostate and Kidney Cancer Program, associate professor of medicine, and nationally renowned expert in the field of prostate and kidney cancer. The goal of the ACGT-funded study is to overcome prostate cancer’s stubborn resistance to CAR T-cell therapy, a therapy that has been successful in treating blood cancers. Drs. Fraietta and Haas are exploring approaches for re-engineering T-cells to enable them to induce safe, long-term remission for advanced, metastatic prostate cancer patients.

"The grant from ACGT will help us advance our clinical work in a very novel way," said Dr. Fraietta. "If we can unlock the epigenetic code that controls the fate and function of T-cells, it could be a game changer."

"The ACGT Scientific Advisory Council is impressed with the potential of this research team and their successful innovations in the use of T-cell therapy," noted Kevin Honeycutt, CEO and president of ACGT. "Because Drs. Fraietta and Haas are building on direct results already achieved with patients, there may be less transition time required to get a promising new treatment into the clinic for prostate cancer patients. Plus, we believe this research could provide a tumor-attack roadmap to help fight other cancers, including lung, pancreatic, ovarian and brain."

In the ACGT-funded study, Drs. Fraietta and Haas are going from the bedside back to the benchtop to employ new insight into how to better enable T-cells to battle cancer cells in solid tumors. Drs. Haas and Fraietta will explore the connection between nutrient availability and epigenetic programming, and how these factors influence the viability of T-cells and their anti-tumor functionality. This research builds on durable results being achieved by Dr. Haas in related prostate cancer clinical trials. In these trials, different doses of CAR T-cell gene therapies are being used to treat metastatic patients for whom traditional hormonal therapies, chemotherapies, radiation and surgery have failed.

"For so many years, chemotherapy, radiation and surgery were the traditional treatments for cancer. For prostate cancer, there’s also hormone therapy," said Honeycutt. "Unfortunately, as the cancer progresses, it often stops responding to these traditional treatments. New cell and gene therapy approaches like the ones Drs. Fraietta and Haas are employing offer new hope to all cancer patients. ACGT has been dedicated to funding innovative science that harnesses the power of cell and gene therapy and transforms how cancer is treated. The work of Drs. Fraietta and Haas is a great example of this promise."

ACGT has been instrumental in funding some of the decade’s most transformative research, including breakthroughs in the use of CAR T-cell gene therapy for leukemia by the University of Pennsylvania’s Carl H. June, MD. "Dr. June received his first ACGT grant in 2004 and a second in 2008, back when gene therapy was considered a risky proposition," says Honeycutt. "Fast forward to today and the field has changed dramatically with major pharmaceutical companies and research institutions vying for the next big discovery using gene therapy or immunotherapy."

On January 30, 2020 A discovery by University of North Carolina Lineberger Comprehensive Cancer Center researchers could allow scientists to fine-tune genetically engineered immune cells to heighten their killing power against tumors or to decrease their activity level in the case of severe side effects (Press release, Lineberger Comprehensive Cancer Center, JAN 30, 2020, View Source [SID1234553698]).

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Gianpietro Dotti is the Director of the Cancer Cellular Immunotherapy Program at UNC Lineberger.
UNC Lineberger’s Gianpietro Dotti, MD.

In a study published in Cancer Cell, researchers led by UNC Lineberger’s Gianpietro Dotti, MD, reported new findings about the regulation of co-stimulatory molecules that could be used to activate cancer-killing immune cells – chimeric antigen receptor T-cells, or CAR-T – or decrease their activity.

"In immunology, it’s always about balance; you don’t want to have too much T-cell activation, and you don’t want T-cell activation to be too low," said Peishun Shou, PhD, postdoctoral research associate at UNC Lineberger and the study’s co-first author. "We wanted to keep the T-cell activation and tumor killing at a suitable or sustainable level."

Cellular immunotherapy
Cellular immunotherapy, or CAR-T immunotherapy, involves extracting specific immune cells from patients, engineering the cells in the lab to hunt tumor cells displaying a specific molecular target, and then re-infusing them to fight their cancer.

Through the Clinical Immunotherapy Program, UNC Lineberger researchers have designed novel investigational CAR-T therapies for Hodgkin and non-Hodgkin lymphoma, multiple myeloma, neuroblastoma and leukemia that are being studied in clinical trials.

"We are conducting and developing clinical studies with CAR-T cells in both liquid and solid tumors. In these studies, we are testing what we call the ‘new generation’ of CAR-T cells, hoping to further enhance the therapeutic index of this technology," said Dotti, the study’s corresponding author, a professor in the UNC School of Medicine Department of Microbiology and Immunology and director of the UNC Lineberger Cellular Immunotherapy Program. "This latest study highlights how when translational and basic science come together, we can hopefully improve therapeutic strategies."

CAR-T immunotherapy study findings
In the Cancer Cell study, researchers revealed new strategies for engineering investigational CAR-T to either increase the activity of modified T-cells to more effectively kill tumor cells, or decrease their activity in case the therapies trigger severe side effects.

They developed strategies for improving two different types of modified T-cells. These two types of CAR-T cells are differentiated by the signals that activate them. First, they have a receptor that recognizes a specific marker on the tumor – the first signal. They also need a second signal that helps to fully activate them and increase their response. There are two different types of T-cells that have different "second signals" that activate them.

Peishun Shou is a postdoctoral researcher at UNC Lineberger.
UNC Lineberger’s Peishun Shou, PhD.
One type of CAR-T is co-stimulated by the CD28 protein, and another is stimulated by 4-1BB. UNC Lineberger researchers wanted to find a way to regulate these proteins in order to "fine-tune" the cells’ disease-fighting response, since researchers reported each type of CAR-T has differences in terms of how long it typically lasts in the body to fight cancer, how quickly it responds and the strength of its response.

"T-cells have to be activated to kill tumor cells," Shou said. "If you have better activation, you have more cytokine release … and the cells can better target a tumor and kill it. In some cases, we want to make the T-cells stronger, more active, and depending on the tumor type, we may want to tune down the T-cell activation to help the T-cells survive and expand."

For CAR-T co-stimulated by 4-1BB, scientists found they could increase expression of the LCK molecule to increase the cells’ activity.

"What we found is that the LCK molecule can bind to the CAR, enhancing the CAR-T cell activation and signaling transduction, which therefore will help CAR-T cells get a better tumor-killing effect," Shou said.

CAR-T safety switch feature
They also reported on the discovery of a new "safety switch" mechanism to reduce activity of CAR-T co-stimulated by CD28. Doctors could use the safety switch should patients experience severe side effects from the experimental therapy.

They found they could use a molecule called SHP1 to reduce T-cell activity. When they added a certain drug, SHP1 bound to the CAR to reduce the activity of CAR-T cells.

"In the presence of the drug, we can cool down or tune down the CAR-T cell activation," Shou said. "The advantage of this switch is that it will not kill the CAR-T cells; it’s just temporarily tuning down the activity."

Researchers want to investigate using these findings to improve CAR-T treatments against blood cancers like leukemia, and to potentially improve experimental treatments for solid tumors.

"Researchers in the CAR-T immunotherapy field now want to solve the solid tumor problem," Shou said. "Solid tumors have an immunosuppressive microenvironment, so you need stronger CAR-T activation."

On January 30, 2020 Corcept Therapeutics Incorporated (NASDAQ: CORT), a commercial-stage company engaged in the discovery and development of drugs to treat severe metabolic, oncologic and neuropsychiatric disorders by modulating the effects of the stress hormone cortisol, reported preliminary fourth quarter revenue of $87.9 million, compared to $66.8 million in the fourth quarter of 2018 (Press release, Corcept Therapeutics, JAN 30, 2020, https://ir.corcept.com/news-releases/news-release-details/corcept-therapeutics-announces-fourth-quarter-and-full-year-2019 [SID1234553696]). Preliminary 2019 revenue was $306.5 million, an increase of 22 percent from 2018.

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Cash and investments increased by $48.4 million in the fourth quarter, to $315.3 million.

These results are prior to completion of the company’s annual independent audit and are subject to adjustment.

Corcept projects 2020 revenue of $355 – 375 million.

"Our Cushing’s syndrome business had an excellent year," said Joseph K. Belanoff, MD, Corcept’s Chief Executive Officer. "As awareness of the poor health outcomes associated with hypercortisolism increased and physicians screened more patients for Cushing’s syndrome, the number of patients receiving Korlym grew. We expect that growth to continue."

"Korlym’s commercial success has given us the resources to develop our proprietary selective cortisol modulators in a wide range of serious disorders," added Dr. Belanoff. "These compounds represent Corcept’s future. We look forward to an important year."

"Our program in Cushing’s syndrome is the most advanced," said Andreas Grauer, MD, Corcept’s Chief Medical Officer. "The pivotal trial of Korlym’s planned successor, relacorilant, is actively enrolling patients at sites in the United States, Europe and Israel. We are also launching a Phase 3 trial in patients whose Cushing’s syndrome is caused by adrenal adenomas.

"Our programs in metabolic and oncologic disorders are poised to advance significantly. In the second quarter, we will have results from the second part of our Phase 1b trial of miricorilant for the prevention of antipsychotic-induced weight gain (APIWG). Miricorilant’s Phase 2 trial for the reversal of recent APIWG continues to accrue patients. We plan to start two additional Phase 2 trials – one for the reversal of long-standing APIWG and another for the treatment of patients with non-alcoholic steatohepatitus (NASH) – by year-end.

"Our Phase 2 trial of relacorilant to treat advanced ovarian cancer continues to enroll patients at sites in the United States and Europe," added Dr. Grauer. "In the second quarter, we anticipate starting a Phase 3 trial of relacorilant in metastatic pancreatic cancer and a Phase 1b trial of relacorilant combined with an immunotherapeutic agent in adrenal cancer. By year-end, we expect to conclude the dose-finding trial of our proprietary cortisol modulator exicorilant in combination with enzalutamide in castration-resistant prostate cancer."

Hypercortisolism

Hypercortisolism, often referred to as Cushing’s syndrome, is caused by excessive activity of the hormone cortisol. Endogenous Cushing’s syndrome is an orphan disease that most often affects adults aged 20-50. In the United States, an estimated 20,000 patients have Cushing’s syndrome, with about 3,000 new patients diagnosed each year. Symptoms vary, but most patients experience one or more of the following manifestations: high blood sugar, diabetes, high blood pressure, upper-body obesity, rounded face, increased fat around the neck, thinning arms and legs, severe fatigue and weak muscles. Irritability, anxiety, cognitive disturbances and depression are also common. Hypercortisolism can affect every organ system in the body and can be lethal if not treated effectively.

On January 30, 2020 Applied DNA Sciences, Inc. (NASDAQ: APDN), reported that it plans to release financial results for its fiscal 2020 first quarter ended December 31, 2019 after market close on Thursday, February 6, 2020. In conjunction with the release, the Company has scheduled a conference call at 4:30 p.m. Eastern Time that will also be broadcast live over the Internet (Press release, Applied DNA Sciences, JAN 30, 2020, View Source [SID1234553695]).

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What: Applied DNA’s Fiscal 2020 First Quarter Financial Results Conference Call
When: Thursday, February 6, 2020, at 4:30 p.m. Eastern Time
Where: Via phone by dialing +1 844-887-9402 or +1 412-317-6798 and ask to join the Applied DNA call; via webcast.

A telephonic replay of the conference call will be available for one day and may be accessed by calling +1 877-344-7529 or +1 412-317-0088 with the passcode 10138146. The webcast will be archived within the ‘Events and Presentations’ portion of the ‘Investors’ page to the company’s website.

On January 30, 2020 Amgen (NASDAQ:AMGN) reported financial results for the fourth quarter and full year 2019 versus comparable periods in 2018 (Press release, Amgen, JAN 30, 2020, View Source [SID1234553694]). Key results include:

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For the fourth quarter, total revenues decreased 1% to $6.2 billion in comparison to the fourth quarter of 2018, reflecting the impact of biosimilar and generic competition against select products.

Product sales declined 2% globally, while units grew double digits or better for Repatha (evolocumab), Parsabiv (etelcalcetide), BLINCYTO (blinatumomab), Aimovig (erenumab-aooe), Prolia (denosumab), Nplate (romiplostim) and Vectibix (panitumumab).

For the full year, total revenues decreased 2% to $23.4 billion, with product sales decreasing 1%.

GAAP earnings per share (EPS) decreased 5% to $2.85 in the fourth quarter driven by higher operating expenses, offset partially by lower weighted-average shares outstanding. GAAP EPS increased 2% to $12.88 for the full year driven by lower weighted-average shares outstanding, offset partially by lower operating income.

For the fourth quarter, GAAP operating income decreased 14% to $2.0 billion and GAAP operating margin decreased 4.9 percentage points to 34.8%. For the full year, GAAP operating income decreased 6% to $9.7 billion and GAAP operating margin decreased 1.9 percentage points to 43.6%.

Non-GAAP EPS increased 6% in the fourth quarter to $3.64 and 3% to $14.82 for the full year benefited by lower weighted-average shares outstanding. The increase for the full year was offset partially by lower operating income.

For the fourth quarter, non-GAAP operating income decreased 4% to $2.6 billion and non-GAAP operating margin decreased 0.7 percentage points to 44.6%. For the full year, non-GAAP operating income decreased 6% to $11.2 billion and non-GAAP operating margin decreased 2.4 percentage points to 50.2%.

The Company generated $8.5 billion of free cash flow for the full year versus $10.6 billion in 2018.

2020 total revenues guidance of $25.0-$25.6 billion; EPS guidance of $10.85-$11.65 on a GAAP basis and $14.85-$15.60 on a non-GAAP basis.
"We are entering a period of new product driven revenue growth," said Robert A. Bradway, chairman and chief executive officer. "Heading into 2020, our capital allocation priorities are clear, and we look forward to several important clinical data readouts from our innovative pipeline this year."

References in this release to "non-GAAP" measures, measures presented "on a non-GAAP basis" and to "free cash flow" (computed by subtracting capital expenditures from operating cash flow) refer to non-GAAP financial measures. Adjustments to the most directly comparable GAAP financial measures and other items are presented on the attached reconciliations.

Product Sales Performance

Total product sales decreased 2% for the fourth quarter of 2019 versus the fourth quarter of 2018. Product sales decreased 1% for the full year driven by lower net selling price, offset partially by higher unit demand.

Prolia sales increased 15% for the fourth quarter and 17% for the full year driven by higher unit demand.

EVENITY (romosozumab-aqqg) launched in 2019, generating sales of $85 million in the fourth quarter and $189 million for the full year.

Repatha sales increased 26% for the fourth quarter and 20% for the full year driven primarily by higher unit demand, offset partially by net selling price.

Aimovig sales increased 3% for the fourth quarter driven by higher unit demand, offset partially by unfavorable changes in accounting estimates. Full year sales grew 157% driven primarily by unit demand.

Parsabiv sales increased 49% for the fourth quarter and 88% for the full year driven primarily by higher unit demand, offset partially by net selling price.

Otezla (apremilast) was acquired on Nov. 21, 2019, and generated $178 million in sales for the period.

Enbrel (etanercept) sales increased 2% for the fourth quarter and 4% for the full year driven primarily by favorable changes in accounting estimates and higher net selling price, offset partially by lower unit demand.

AMGEVITA (adalimumab) generated $71 million of sales in the fourth quarter and $215 million for the full year.

KYPROLIS (carfilzomib) sales increased 6% for the fourth quarter and 8% for the full year driven by higher unit demand.

XGEVA (denosumab) sales increased 7% for the fourth quarter and 8% for the full year driven primarily by higher unit demand and, to a lesser extent, higher net selling price.

Vectibix sales increased 8% for the fourth quarter and the full year driven by higher unit demand.

Nplate sales increased 15% for the fourth quarter and 11% for the full year driven primarily by higher unit demand.

BLINCYTO sales increased 27% for the fourth quarter and 36% for the full year driven by higher unit demand.

KANJINTI* (trastuzumab-anns) generated $103 million of sales in the fourth quarter and $226 million for the full year.

MVASI* (bevacizumab-awwb) generated $84 million of sales in the fourth quarter and $127 million for the full year.

Neulasta (pegfilgrastim) sales decreased 43% for the fourth quarter and 28% for the full year driven by the impact of biosimilar competition on unit demand and lower net selling price.

NEUPOGEN (filgrastim) sales decreased 17% for the fourth quarter driven by the impact of competition on unit demand. Sales decreased 28% for the full year driven by the impact of competition on unit demand and lower net selling price.

EPOGEN (epoetin alfa) sales decreased 20% for the fourth quarter driven by lower net selling price and unit demand. Sales decreased 14% for the full year driven primarily by lower net selling price.

Aranesp (darbepoetin alfa) sales decreased 10% for the fourth quarter driven by the impact of competition on unit demand and lower net selling price as well as unfavorable changes in inventory. Sales decreased 8% for the full year driven primarily by the impact of competition of unit demand.

Sensipar/Mimpara (cinacalcet) sales decreased 76% for the fourth quarter and 69% for the full year driven by the impact of generic competition on unit demand.

* Registered in the United States.

** Other includes GENSENTA, Bergamo, IMLYGIC and Corlanor.
Operating Expense, Operating Margin and Tax Rate Analysis
On a GAAP basis:

Total Operating Expenses increased 8% in the fourth quarter and 2% for the full year. Cost of Sales margin increased 3 percentage points in the fourth quarter driven primarily by amortization of intangible assets acquired in the Otezla acquisition. For the full year, Cost of Sales margin increased 1.4 percentage points driven primarily by unfavorable product mix and amortization of intangible assets acquired in the Otezla acquisition, offset partially by lower royalties and lower manufacturing costs. Research & Development (R&D) expenses increased 11% in the fourth quarter and 10% for the full year driven by higher spending in research and early pipeline in support of our oncology programs. The full year was offset partially by lower spend in support of marketed programs. Selling, General & Administrative (SG&A) expenses decreased 3% in the fourth quarter driven by lower spend for launched and marketed products and lower general and administrative expenses, offset partially by Otezla commercial-related expenses. For the full year, SG&A expenses decreased 3% driven by lower general and administrative expenses, the end of certain amortization of intangible assets in 2018 and lower spend for launched and marketed products, offset partially by Otezla commercial-related expenses. Other expenses increased in the fourth quarter driven primarily by restructuring costs in 2019. For the full year, other operating expenses decreased driven primarily by an impairment charge in 2018 of an intangible asset.

Operating Margin decreased 4.9 percentage points in the fourth quarter to 34.8% driven primarily by the Otezla acquisition, and decreased 1.9 percentage points for the full year to 43.6%.

Tax Rate increased 2.3 percentage points in the fourth quarter and 2.1 percentage points for the full year due primarily to a prior-year tax benefit associated with intercompany sales under U.S. corporate tax reform.

On a non-GAAP basis:

Total Operating Expenses increased 2% in the fourth quarter and 3% for the full year. Cost of Sales margin decreased 0.2 percentage points in the fourth quarter. For the full year, Cost of Sales margin increased 0.5 percentage points driven primarily by unfavorable product mix, offset partially by lower royalties and lower manufacturing costs. R&D expenses increased 11% for the fourth quarter and 10% for the full year driven by higher spending in research and early pipeline in support of our oncology programs. The full year was offset partially by lower spend in support of marketed programs. SG&A expenses decreased 2% in the fourth quarter driven by lower spend for launched and marketed products and lower general and administrative expenses, offset partially by Otezla commercial-related expenses. For the full year, SG&A expenses decreased 2% driven by lower general and administrative expenses and lower spend for launched and marketed products, offset partially by Otezla commercial-related expenses.

Operating Margin decreased 0.7 percentage points to 44.6% in the fourth quarter, and decreased 2.4 percentage points to 50.2% for the full year.

Tax Rate increased 1.6 percentage points in the fourth quarter and 1.5 percentage points for the full year due primarily to a prior-year tax benefit associated with intercompany sales under U.S. corporate tax reform.
$Millions, except percentages

Cash Flow and Balance Sheet

The Company generated $2.3 billion of free cash flow in the fourth quarter of 2019 versus $3.0 billion in the fourth quarter of 2018 due primarily to timing of tax payments. The Company generated $8.5 billion of free cash flow for the full year 2019 versus $10.6 billion in 2018 due primarily to unfavorable changes in working capital, an advanced tax deposit and lower net income.

The Company’s fourth quarter 2019 dividend of $1.45 per share was declared on Oct. 22, 2019, and was paid on Dec. 6, 2019, to all stockholders of record as of Nov. 15, 2019, representing a 10% increase from the fourth quarter of 2018. The Company’s first quarter 2020 dividend of $1.60 per share declared on Dec. 11, 2019, will be paid on March 6, 2020, to all stockholders of record as of Feb. 14, 2020, representing a 10% increase from that paid in each of the previous four quarters of 2019.

During the fourth quarter of 2019, the Company repurchased 5.1 million shares of common stock at a total cost of $1.1 billion. For the full year, the Company repurchased 40.2 million shares of common stock at a total cost of $7.6 billion. At the end of the fourth quarter, the Company had $6.5 billion remaining under its stock repurchase authorization.
$Billions, except shares

Note: Numbers may not add due to rounding

2020 Guidance
For the full year 2020, the Company expects:

Total revenues in the range of $25.0 billion to $25.6 billion.

On a GAAP basis, EPS in the range of $10.85 to $11.65 and a tax rate in the range of 10.5% to 11.5%.

On a non-GAAP basis, EPS in the range of $14.85 to $15.60 and a tax rate in the range of 13.5% to 14.5%.

Capital expenditures to be approximately $700 million.
Fourth Quarter Product and Pipeline Update
The Company provided the following updates on selected product and pipeline programs:
Otezla

Data from the Phase 3 study in patients with mild-to-moderate psoriasis are expected by mid-year 2020.

A supplemental New Drug Application (sNDA) to expand the Prescribing Information to include data from the Phase 3 scalp psoriasis study is under review by the U.S. Food and Drug Administration (FDA) with a Prescription Drug User Fee Act target action date in April 2020.

EVENITY

In December 2019, the European Commission (EC) granted marketing authorization for EVENITY for the treatment of severe osteoporosis in postmenopausal women at high risk of fracture.

KYPROLIS

In January, an sNDA was submitted to the FDA to expand the Prescribing Information to include KYPROLIS in combination with dexamethasone and DARZALEX (daratumumab) for patients with relapsed or refractory multiple myeloma based on data from the Phase 3 CANDOR study.

In November, a marketing authorization application (MAA) was accepted by the China National Medical Products Administration (NMPA) for the use of KYPROLIS plus dexamethasone for the treatment of relapsed and refractory multiple myeloma.

BLINCYTO

In December, the China NMPA granted priority review for the MAA for the treatment of adults with relapsed or refractory B-cell acute lymphoblastic leukemia.

AMGEN REPORTS FOURTH QUARTER AND FULL YEAR 2019 FINANCIAL RESULTS
Page 9

AMG 510

A potentially pivotal Phase 2 monotherapy study in advanced non-small cell lung cancer (NSCLC) completed enrollment and data are expected in 2020.

A Phase 2 monotherapy study is enrolling advanced colorectal cancer patients.

A Phase 1b study in combination with MEK inhibition is enrolling advanced colorectal and non-small cell lung cancer patients.

The ongoing Phase 1 monotherapy study is also enrolling treatment naïve NSCLC patients.

In 2020, additional data are expected from the first-in-human monotherapy study in patients with multiple solid tumors, and initial data are expected from a Phase 1 study in combination with KEYTRUDA (pembrolizumab) in patients with advanced NSCLC.

In January, the Company announced strategic collaborations with leading diagnostic companies, Guardant Health, Inc. and QIAGEN N.V., to develop blood- and tissue-based companion diagnostics, respectively.

Omecamtiv mecarbil

Data from the event driven Phase 3 GALACTIC-HF cardiovascular outcomes study are expected in Q4 2020.

AVSOLA (infliximab-axxq)

In December, the FDA approved AVSOLA for all approved indications of the reference product, Remicade (infliximab).

ABP 798 (biosimilar rituximab)

In December, a Biologics License Application was submitted to the FDA for ABP 798, a biosimilar candidate to Rituxan (rituximab).