CTI BioPharma Announces Proposed Rights Offering

On February 3, 2020 CTI BioPharma Corp. (Nasdaq: CTIC) reported its intent to raise $60 million through a fully backstopped rights offering (Press release, CTI BioPharma, FEB 3, 2020, View Source [SID1234553793]). Under the terms of the rights offering, investors as of February 13, 2020 in CTI BioPharma’s common stock and preferred stock will receive a subscription right entitling them to purchase their pro rata share of the $60 million offering amount. The rights offering will be fully backstopped by BVF Partners L.P., Stonepine Capital, L.P., OrbiMed Private Investments VI, LP and New Enterprise Associates, Inc. each of which have agreed to purchase, at a minimum, its respective as-converted pro rata share of the offering amount, plus an additional amount of securities that are not subscribed for by other purchasers in the rights offering, for a total of up to $60 million.

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Under the proposed rights offering (the "Rights Offering"), CTI BioPharma plans to distribute non-transferable subscription rights to purchase a portion of a share of CTI BioPharma’s common stock ("Common Stock") or series X convertible preferred stock ("Series X Preferred") to each stockholder of record of Common Stock, including Common Stock issuable upon conversion of the outstanding shares of CTI Biopharma’s series O convertible preferred stock ("Series O Preferred") at $1.00 per share to such security holders of record as of the close of business on February 13, 2020 (the "Record Date"). The subscription rights will be exercisable for shares of Common Stock (or an equivalent number of Series X Preferred), with participation to be allocated among holders of its Common Stock and Series O Preferred on a pro rata basis (assuming full conversion of the Series O Preferred into shares of Common Stock), subject to the aggregate offering threshold and ownership limitations. The subscription rights may be exercised only during the anticipated subscription period of Friday, February 14, 2020, through 5:00 PM (Eastern Time) on Monday, March 2, 2020, unless extended. Any participant in the Rights Offering that, by exercise of its subscription right would become a holder of greater than 9.9% of the outstanding number of shares of Common Stock following the Rights Offering may elect to instead purchase Series X Preferred. CTI BioPharma intends to sell the Series X Preferred at $10,000 per share, and any such holder so electing would have a right to purchase one 10,000th of a share of Series X Preferred for each share of Common Stock it had a right to purchase under the subscription rights. Each share of Series X Preferred is, subject to certain limitations, convertible into 10,000 shares of Common Stock at the election of the holder. The Series X Preferred generally have no voting rights, except as required by law, and will participate pari passu, on an as-converted basis, with any distribution of proceeds to holders of Common Stock and Series O Preferred in the event of CTI BioPharma’s liquidation, dissolution or winding up.

The Rights Offering will be made pursuant to CTI BioPharma’s effective shelf registration statement on file with the Securities and Exchange Commission and only by means of a prospectus supplement and accompanying prospectus. CTI BioPharma expects to mail subscription certificates evidencing the subscription rights and a copy of the prospectus supplement and accompanying prospectus for the Rights Offering shortly following the Record Date.

This press release is not intended to and does not constitute an offer to sell or the solicitation of an offer to subscribe for or buy or an invitation to purchase or subscribe for any securities in any jurisdiction, nor shall there be any sale, issuance or transfer of securities in any jurisdiction in contravention of applicable law. No offer of securities shall be made except by means of a prospectus meeting the requirements of Section 10 of the Securities Act of 1933, as amended.

Certain existing shareholders of CTI BioPharma (the "Investors"), including BVF Partners L.P., CTI BioPharma’s largest shareholder, currently owning approximately 11.95% of CTI BioPharma’s outstanding Common Stock (and 23.1% on an as-converted basis), Stonepine Capital, L.P., OrbiMed Private Investments VI, LP and New Enterprise Associates, Inc. currently owning 6.23%, 8.62% and 5.59% of CTI BioPharma’s outstanding Common Stock, respectively, will backstop the Rights Offering and have agreed to purchase up to $60 million of Common Stock at a subscription price per share of $1.00 (or Series X Preferred at a price of $10,000 per share) in a private placement promptly at the conclusion of the Rights Offering, with the dollar amount to be purchased in such private placement reduced by the dollar amount sold by CTI BioPharma (including to the Investors and their respective affiliates) in the Rights Offering.

Concurrent with this press release, CTI is announcing the outcome of its recent meeting with the U.S. Food and Drug Administration and the subsequent amendment to the PACIFICA trial. For further details, see the concurrent press release relating to this meeting.

Janssen to Highlight Depth of Solid Tumor Portfolio at ASCO GU

On February 3, 2020 The Janssen Pharmaceutical Companies of Johnson & Johnson reported multiple data presentations from a robust solid tumor portfolio that will be featured at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Genitourinary (ASCO GU) Cancers Symposium, taking place February 13-15 in San Francisco (Press release, Janssen Pharmaceuticals, FEB 3, 2020, View Source [SID1234553792]). Company-sponsored data presentations will include clinical results for ERLEADA (apalutamide) and niraparib in prostate cancer; and BALVERSA (erdafitinib) in bladder cancer.

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"We are committed to improving outcomes in patients with prostate and bladder cancer where high unmet needs continue to persist despite advancements in treatment," said Craig Tendler, M.D., Vice President, Clinical Development and Global Medical Affairs, Janssen Research & Development, LLC. "At this year’s meeting, we look forward to highlighting results from our GU portfolio and pipeline, including a new analysis of time to second progression or death after initial treatment with ERLEADA from the Phase 3 TITAN study in patients with metastatic castration-sensitive prostate cancer; and new data for BALVERSA in combination with an anti-PD-1 monoclonal antibody."

Highlights from the Janssen oncology portfolio and pipeline will include:

New Analyses from the Phase 3 TITAN Study of ERLEADA in Metastatic Castration-Sensitive Prostate Cancer (mCSPC)
An oral presentation will feature a post hoc analysis of data from the Phase 3 TITAN study on the time to second progression (PFS2) or death at any time for patients receiving hormone versus taxane therapy after initial treatment with ERLEADA (Abstract #82).

A poster presentation will highlight ERLEADA efficacy and safety results in high- versus low-risk patients with mCSPC (Abstract #87).

Niraparib Results in the Treatment of Patients with Metastatic Castration-Resistant Prostate Cancer (mCRPC) and Biallelic DNA-Repair Gene Defects
A poster presentation will include an updated analysis from the Phase 2 GALAHAD study evaluating correlative measures of tumor response in patients with mCRPC and biallelic DNA-repair gene defects, including patients with BRCA1/2 gene mutations (Abstract #118).

BALVERSA and Cetrelimab Combination Results in Metastatic Urothelial Carcinoma (mUC) in Patients with Genetic Alterations
A poster presentation (Abstract #511) will report results from the Phase 1b/2 NORSE study evaluating BALVERSA in combination with cetrelimab, an anti-PD-1 monoclonal antibody, in patients with locally advanced or mUC with select fibroblast growth factor receptor (FGFR) genetic alterations.

Company-sponsored abstracts to be presented at the meeting include:

Abstract No.

Title

Date/Time

ERLEADA (apalutamide)

Oral Presentation

Abstract #82

Time to Second Progression (PFS2) in Patients
from TITAN with Metastatic Castration-Sensitive
Prostate Cancer by First Subsequent Therapy
(Hormonal vs Taxane)

Thursday, February 13

1:35 PM – 1:45 PM PST

Poster Presentations

Abstract #87

Apalutamide for Metastatic Castration-Sensitive
Prostate Cancer in TITAN: Outcomes in Patients
with Low- and High-Risk Disease

Thursday, February 13

11:30 AM – 1:00 PM and
5:30 PM – 6:30 PM PST

Abstract #320

A Randomized Phase 2 Study of Apalutamide,
Androgen Deprivation Therapy, or APA + ADT in
Patients with Biochemically Relapsed Prostate
Cancer

Thursday, February 13

11:30 AM – 1:00 PM and
5:30 PM – 6:30 PM PST

Abstract #156

Linking the Aggressive Variant Prostate Cancer
Molecular Signature to Androgen Indifference in a
Prospective Clinical Trial

Thursday, February 13

11:30 AM – 1:00 PM and
5:30 PM – 6:30 PM PST

Abstract #TPS383

PROTEUS: A Randomized, Double-Blind, Placebo –
Controlled, Phase 3 Trial of Apalutamide Plus
Androgen Deprivation Therapy vs PBO Plus ADT
Prior to Radical Prostatectomy in Patients with
Localized High-Risk or Locally Advanced Prostate
Cancer

Thursday, February 13

11:30 AM – 1:00 PM and
5:30 PM – 6:30 PM PST

Niraparib

Poster Presentations

Abstract #118

GALAHAD: Niraparib in Patients with Metastatic
Castration-Resistant Prostate Cancer and Biallelic
DNA-Repair Gene Defects: Correlative Measures of
Tumor Response in Phase 2 GALAHAD Study

Thursday, February 13

11:30 AM – 1:00 PM
and 5:30 PM – 6:30 PM PST

Abstract #122

BEDIVERE: Phase 1b Study of Niraparib Plus
Androgen Receptor-Targeted Therapy in Patients
with Metastatic Castration Resistant Prostate
Cancer

Thursday, February 13

11:30 AM – 1:00 PM
and 5:30 PM – 6:30 PM PST

Abstract #TPS257

MAGNITUDE: A Phase 3 Randomized, Placebo-
Controlled, Double-Blind Study of Niraparib Plus
Abiraterone Acetate and Prednisone Versus
Abiraterone Acetate and Prednisone in Patients
with Metastatic Prostate Cancer

Thursday, February 13

11:30 AM – 1:00 PM
and 5:30 PM – 6:30 PM PST

BALVERSA (erdafitinib)

Poster Presentations

Abstract #511

Dose Escalation Results from Phase 1b/2 NORSE
Study of Erdafitinib + PD-1 Inhibitor JNJ-
63723283 (Cetrelimab [CET]) in Patients with
Metastatic or Locally Advanced Urothelial
Carcinoma and Selected Fibroblast Growth Factor
Receptor (FGFR) Gene Alterations

Friday, February 14

12:15 PM – 1:45 PM
and 5:15 PM – 6:15
PM PST

Abstract #493

BRIDGE + GeneCentric: Predictive Value of
Fibroblast Growth Factor Receptor Alterations on
Anti-PD-(L)1 Treatment Outcomes in Patients with
Advanced Urothelial Cancer: Pooled Analysis of
Real-World Data

Friday, February 14

12:15 PM – 1:45 PM
and 5:15 PM – 6:15
PM PST

Abstract #TPS603

BLC2003 Trial in Progress (TiP): A Randomized
Phase 2 Study of Erdafitinib Versus Intravesical
Chemotherapy in Patients with High-Risk Non-
Muscle Invasive Bladder Cancer with FGFR
Mutations or Fusions, Who Recurred After Bacillus
Calmette-Guérin Therapy

Friday, February 14

12:15 PM – 1:45 PM
and 5:15 PM – 6:15
PM PST


About ERLEADA (apalutamide)
ERLEADA is an androgen receptor (AR) inhibitor indicated for the treatment of patients with non-metastatic castration-resistant prostate cancer (nmCRPC) and for the treatment of patients with mCSPC. ERLEADA received U.S. Food and Drug Administration (FDA) approval for nmCRPC on February 14, 2018 and was approved for mCSPC on September 17, 2019.1 ERLEADA is taken orally, once daily, with or without food.1 The NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines) for Prostate Cancer include apalutamide (ERLEADA) as a treatment option for patients with non-metastatic (M0) CRPC with a Category 1 recommendation for those with a PSA doubling time ≤10 months.*2 The NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines) include apalutamide (ERLEADA) with androgen deprivation** as a Category 1 treatment option for patients with metastatic (M1) castration-naive prostate cancer.†5 The American Urological Association (AUA) Guidelines for Castration-Resistant Prostate Cancer (CRPC) recommend clinicians offer apalutamide (ERLEADA) with continued androgen deprivation therapy (ADT) as one of the treatment options for patients with nmCRPC at high risk for developing metastatic disease. (Standard; Evidence Level Grade A)***.3 ERLEADA is being studied in five Phase 3 clinical trials.

For more information about ERLEADA, visit www.ERLEADA.com.

* Referenced with permission from the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines) for Prostate Cancer V.4.2019. © National Comprehensive Cancer Network, Inc. 2019. All rights reserved. Accessed January 2020. To view the most recent and complete version of the NCCN Guidelines, go online to NCCN.org. NCCN makes no warranties of any kind whatsoever regarding their content, use, or application, and disclaims any responsibility for their application or use in any way.

** Orchiectomy, LHRH agonist, or LHRH antagonist

† The term "castration-naive" is used to define patients who are not on ADT at the time of progression. The NCCN Prostate Cancer Panel uses the term "castration-naive" even when patients have had neoadjuvant, concurrent, or adjuvant ADT as part of radiation therapy provided they have recovered testicular function.

***Standard: Directive statement that an action should (benefits outweigh risks/burdens) or should not (risks/burdens outweigh benefits) be taken based on Grade A or B evidence.

***Evidence Level: A designation indicating the certainty of the results as high, moderate, or low (A, B, or C, respectively) based on AUA nomenclature and methodology.

About Niraparib
Niraparib is an orally-administered selective poly ADP ribose polymerase (PARP) inhibitor that is currently being studied by Janssen for the treatment of patients with prostate cancer. Ongoing studies include the Phase 3 MAGNITUDE study evaluating niraparib in combination with ZYTIGA (abiraterone acetate) and prednisone in adults with metastatic prostate cancer; and QUEST, a Phase 1b/2 study of niraparib combination therapies for the treatment of mCRPC. On October 3, 2019 niraparib received Breakthrough Therapy Designation from the U.S. FDA based on the Phase 2 GALAHAD study in patients with mCRPC.

In April 2016, Janssen entered a worldwide (except Japan) collaboration and license agreement with TESARO, Inc. (now GSK), for exclusive rights to niraparib in prostate cancer. In the U.S., niraparib is indicated for the treatment of adult patients with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in a complete or partial response to platinum-based chemotherapy; and for the treatment of adult patients with advanced ovarian, fallopian tube, or primary peritoneal cancer who have been treated with three or more prior chemotherapy regimens and whose cancer is associated with homologous recombination deficiency positive status.4 Niraparib is currently marketed by GSK as ZEJULA. GSK is focused on maximizing patient survival through transformational medicines for patients living with cancer. Please refer to the full Prescribing Information available at View Source

About BALVERSA (erdafitinib)
BALVERSA is a once-daily, oral FGFR kinase inhibitor indicated for the treatment of adults with locally advanced or mUC that has susceptible FGFR3 or FGFR2 genetic alterations and who have progressed during or following at least one line of prior platinum-containing chemotherapy, including within 12 months of neoadjuvant or adjuvant platinum-containing chemotherapy.5

This indication was approved by the U.S. FDA on April 12, 2019 under an accelerated approval based on tumor response rate. BALVERSA is the first FGFR kinase inhibitor to receive U.S. FDA approval for the treatment of mUC. Patients may be eligible for BALVERSA based on an FDA-approved companion diagnostic. Continued approval for this indication is contingent upon verification and description of clinical benefit in confirmatory trials.5 In 2008, Janssen entered into an exclusive worldwide license and collaboration agreement with Astex Pharmaceuticals to develop and commercialize BALVERSA.

Ongoing studies for BALVERSA in patients with locally advanced or mUC whose tumors have certain FGFR alterations include the Phase 2 BLC2001 study evaluating efficacy and safety, and the Phase 3 BLC3001 study evaluating BALVERSA compared with vinflunine, docetaxel or pembrolizumab. The development plan for BALVERSA also includes a Phase 2 study (NCT04083976) in patients with advanced solid tumors and FGFR alterations.

The NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines) for Bladder Cancer include erdafitinib (BALVERSA) as an alternative preferred regimen for locally advanced or metastatic disease (Stage IV) (post-platinum) for patients with susceptible FGFR3 or FGFR2 genetic alterations.*6 The NCCN Guidelines recommend molecular/genomic testing for FGFR3 and FGFR2 genetic alterations as part of a work-up of locally advanced or metastatic urothelial carcinoma.6

For more information about BALVERSA, visit www.BALVERSA.com.

*Referenced with permission from the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines) for Bladder Cancer V.2.2020. © National Comprehensive Cancer Network, Inc. 2020. All rights reserved. Accessed January 2020. To view the most recent and complete version of the NCCN Guidelines, go online to NCCN.org. NCCN makes no warranties of any kind whatsoever regarding their content, use, or application, and disclaims any responsibility for their application or use in any way.

ERLEADA IMPORTANT SAFETY INFORMATION1
WARNINGS AND PRECAUTIONS

Ischemic Cardiovascular Events — In a randomized study (SPARTAN) of patients with nmCRPC, ischemic cardiovascular events occurred in 4% of patients treated with ERLEADA and 3% of patients treated with placebo. In a randomized study (TITAN) in patients with mCSPC, ischemic cardiovascular events occurred in 4% of patients treated with ERLEADA and 2% of patients treated with placebo. Across the SPARTAN and TITAN studies, 6 patients (0.5%) treated with ERLEADA and 2 patients (0.2%) treated with placebo died from an ischemic cardiovascular event. Patients with current evidence of unstable angina, myocardial infarction, or congestive heart failure within 6 months of randomization were excluded from the SPARTAN and TITAN studies.

Ischemic cardiovascular events, including events leading to death, occurred in patients receiving ERLEADA. Monitor for signs and symptoms of ischemic heart disease. Optimize management of cardiovascular risk factors, such as hypertension, diabetes, or dyslipidemia. Consider discontinuation of ERLEADA for Grade 3 and 4 events.

Fractures — In a randomized study (SPARTAN) of patients with nmCRPC, fractures occurred in 12% of patients treated with ERLEADA and in 7% of patients treated with placebo. In a randomized study (TITAN) of patients with mCSPC, fractures occurred in 9% of patients treated with ERLEADA and in 6% of patients treated with placebo. Evaluate patients for fracture risk. Monitor and manage patients at risk for fractures according to established treatment guidelines and consider use of bone-targeted agents.

Falls — In a randomized study (SPARTAN), falls occurred in 16% of patients treated with ERLEADA compared with 9% of patients treated with placebo. Falls were not associated with loss of consciousness or seizure. Falls occurred in patients receiving ERLEADA with increased frequency in the elderly. Evaluate patients for fall risk.

Seizure — In 2 randomized studies (SPARTAN and TITAN), 5 patients (0.4%) treated with ERLEADA and 1 patient treated with placebo (0.1%) experienced a seizure. Permanently discontinue ERLEADA in patients who develop a seizure during treatment. It is unknown whether anti-epileptic medications will prevent seizures with ERLEADA. Advise patients of the risk of developing a seizure while receiving ERLEADA and of engaging in any activity where sudden loss of consciousness could cause harm to themselves or others.

Embryo-Fetal Toxicity — The safety and efficacy of ERLEADA have not been established in females. Based on its mechanism of action, ERLEADA can cause fetal harm and loss of pregnancy when administered to a pregnant female. Advise males with female partners of reproductive potential to use effective contraception during treatment and for 3 months after the last dose of ERLEADA [see Use in Specific Populations (8.1, 8.3)].

ADVERSE REACTIONS
Adverse Reactions — The most common adverse reactions (≥10%) that occurred more frequently in the ERLEADA-treated patients (≥ 2% over placebo) from the randomized placebo-controlled clinical trials (TITAN and SPARTAN) were fatigue, arthralgia, rash, decreased appetite, fall, weight decreased, hypertension, hot flush, diarrhea and fracture.

Laboratory Abnormalities — All Grades (Grade 3-4)

Hematology — In the TITAN study: white blood cell decreased ERLEADA 27% (0.4%), placebo 19% (0.6%). In the SPARTAN study: anemia ERLEADA 70% (0.4%), placebo 64% (0.5%); leukopenia ERLEADA 47% (0.3%), placebo 29% (0%); lymphopenia ERLEADA 41% (2%), placebo 21% (2%)
Chemistry — In the TITAN study: hypertriglyceridemia ERLEADA 17% (3%), placebo 12% (2%). In the SPARTAN study: hypercholesterolemia ERLEADA 76% (0.1%), placebo 46% (0%); hyperglycemia ERLEADA 70% (2%), placebo 59% (1%); hypertriglyceridemia ERLEADA 67% (2%), placebo 49% (0.8%); hyperkalemia ERLEADA 32% (2%), placebo 22% (0.5%)
Rash — In 2 randomized studies, rash was most commonly described as macular or maculopapular. Adverse reactions of rash were 26% with ERLEADA vs 8% with placebo. Grade 3 rashes (defined as covering >30% body surface area [BSA]) were reported with ERLEADA treatment (6%) vs placebo (0.5%).

The onset of rash occurred at a median of 83 days. Rash resolved in 78% of patients within a median of 78 days from onset of rash. Rash was commonly managed with oral antihistamines, topical corticosteroids, and 19% of patients received systemic corticosteroids. Dose reduction or dose interruption occurred in 14% and 28% of patients, respectively. Of the patients who had dose interruption, 59% experienced recurrence of rash upon reintroduction of ERLEADA.

Hypothyroidism — In 2 randomized studies, hypothyroidism was reported for 8% of patients treated with ERLEADA and 2% of patients treated with placebo based on assessments of thyroid-stimulating hormone (TSH) every 4 months. Elevated TSH occurred in 25% of patients treated with ERLEADA and 7% of patients treated with placebo. The median onset was at the first scheduled assessment. There were no Grade 3 or 4 adverse reactions. Thyroid replacement therapy, when clinically indicated, should be initiated or dose-adjusted.

DRUG INTERACTIONS

Effect of Other Drugs on ERLEADA — Co-administration of a strong CYP2C8 or CYP3A4 inhibitor is predicted to increase the steady-state exposure of the active moieties. No initial dose adjustment is necessary; however, reduce the ERLEADA dose based on tolerability [see Dosage and Administration (2.2)].

Effect of ERLEADA on Other Drugs — ERLEADA is a strong inducer of CYP3A4 and CYP2C19, and a weak inducer of CYP2C9 in humans. Concomitant use of ERLEADA with medications that are primarily metabolized by CYP3A4, CYP2C19, or CYP2C9 can result in lower exposure to these medications. Substitution for these medications is recommended when possible or evaluate for loss of activity if medication is continued. Concomitant administration of ERLEADA with medications that are substrates of UDP-glucuronosyl transferase (UGT) can result in decreased exposure. Use caution if substrates of UGT must be co-administered with ERLEADA and evaluate for loss of activity.

P-gp, BCRP or OATP1B1 Substrates — Apalutamide is a weak inducer of P-glycoprotein (P-gp), breast cancer resistance protein (BCRP), and organic anion transporting polypeptide 1B1 (OATP1B1) clinically. Concomitant use of ERLEADA with medications that are substrates of P-gp, BCRP, or OATP1B1 can result in lower exposure of these medications. Use caution if substrates of P-gp, BCRP or OATP1B1 must be co-administered with ERLEADA and evaluate for loss of activity if medication is continued.

Please see the full Prescribing Information for ERLEADA.

BALVERSA IMPORTANT SAFETY INFORMATION5
WARNINGS AND PRECAUTIONS

Ocular Disorders – BALVERSA can cause ocular disorders, including central serous retinopathy/retinal pigment epithelial detachment (CSR/RPED) resulting in visual field defect. CSR/RPED was reported in 25% of patients treated with BALVERSA, with a median time to first onset of 50 days. Grade 3 CSR/RPED, involving central field of vision, was reported in 3% of patients. CSR/RPED resolved in 13% of patients and was ongoing in 13% of patients at the study cutoff. CSR/RPED led to dose interruptions and reductions in 9% and 14% of patients, respectively, and 3% of patients discontinued BALVERSA. Dry eye symptoms occurred in 28% of patients during treatment with BALVERSA and were Grade 3 in 6% of patients. All patients should receive dry eye prophylaxis with ocular demulcents as needed. Perform monthly ophthalmological examinations during the first 4 months of treatment and every 3 months afterwards, and urgently at any time for visual symptoms. Ophthalmological examination should include assessment of visual acuity, slit lamp examination, fundoscopy, and optical coherence tomography. Withhold BALVERSATM when CSR occurs and permanently discontinue if it does not resolve within 4 weeks or if Grade 4 in severity. For ocular adverse reactions, follow the dose modification guidelines [see Dosage and Administration (2.3)].

Hyperphosphatemia – Increases in phosphate levels are a pharmacodynamic effect of BALVERSA [see Pharmacodynamics (12.2)]. Hyperphosphatemia was reported as adverse reaction in 76% of patients treated with BALVERSA. The median onset time for any grade event of hyperphosphatemia was 20 days (range: 8–116) after initiating BALVERSA. Thirty-two percent of patients received phosphate binders during treatment with BALVERSA. Monitor for hyperphosphatemia and follow the dose modification guidelines when required [see Dosage and Administration (2.2, 2.3)].

Embryo-fetal Toxicity – Based on the mechanism of action and findings in animal reproduction studies, BALVERSA can cause fetal harm when administered to a pregnant woman. In a rat embryo-fetal toxicity study, erdafitinib was embryotoxic and teratogenic at exposures less than the human exposures at all doses studied. Advise pregnant women of the potential risk to the fetus. Advise female patients of reproductive potential to use effective contraception during treatment with BALVERSA and for one month after the last dose. Advise male patients with female partners of reproductive potential to use effective contraception during treatment with BALVERSA and for one month after the last dose [see Use in Specific Populations (8.1, 8.3) and Clinical Pharmacology (12.1)].

Most common adverse reactions including laboratory abnormalities ≥20% were: Phosphate increased (76%), stomatitis (56%), fatigue (54%), creatinine increased (52%), diarrhea (47%), dry mouth (45%), onycholysis (41%), alanine aminotransferase increased (41%), alkaline phosphatase increased (41%), sodium decreased (40%), decreased appetite (38%), albumin decreased (37%), dysgeusia (37%), hemoglobin decreased (35%), dry skin (34%), aspartate aminotransferase increased (30%), magnesium decreased (30%), dry eye (28%), alopecia (26%), palmar-plantar erythrodysesthesia syndrome (26%), constipation (28%), phosphate decreased (24%), abdominal pain (23%), calcium increased (22%), nausea (21%), and musculoskeletal pain (20%). The most common Grade 3 or greater adverse reactions (>1%) were stomatitis (9%), nail dystrophy*, palmar-plantar erythrodysesthesia syndrome (6%), paronychia (3%), nail disorder*, keratitis†, onycholysis* (10%), and hyperphosphatemia.

*Included within onycholysis. †Included within dry eye.

An adverse reaction with a fatal outcome in 1% of patients was acute myocardial infarction.

Serious adverse reactions occurred in 41% of patients, including eye disorders (10%).

Permanent discontinuation due to an adverse reaction occurred in 13% of patients. The most frequent reasons for permanent discontinuation included eye disorders (6%).

Dosage interruptions occurred in 68% of patients. The most frequent adverse reactions requiring dosage interruption included hyperphosphatemia (24%), stomatitis (17%), eye disorders (17%), and palmar-plantar erythrodysesthesia syndrome (8%).

Dose reductions occurred in 53% of patients. The most frequent adverse reactions for dose reductions included eye disorders (23%), stomatitis (15%), hyperphosphatemia (7%), palmar-plantar erythrodysesthesia syndrome (7%), paronychia (7%), and nail dystrophy (6%).

Drug Interactions

Strong CYP2C9 or CYP3A4 Inhibitors: Consider alternative agents or monitor closely for adverse reactions. (7.1)
Strong CYP2C9 or CYP3A4 inducers: Avoid concomitant use with BALVERSA. (7.1)
Moderate CYP2C9 or CYP3A4 inducers: Increase BALVERSA dose up to 9 mg. (7.1)
Serum phosphate level-altering agents: Avoid concomitant use with agents that can alter serum phosphate levels before the initial dose modification period. (2.3, 7.1)
CYP3A4 substrates: Avoid concomitant use with sensitive CYP3A4 substrates with narrow therapeutic indices. (7.2)
OCT2 substrates: Consider alternative agents or consider reducing the dose of OCT2 substrates based on tolerability. (7.2)
P-gp substrates: Separate BALVERSA administration by at least 6 hours before or after administration of P-gp substrates with narrow therapeutic indices. (7.2)
Use in Specific Populations

Lactation – Because of the potential for serious adverse reactions from erdafitinib in a breastfed child, advise lactating women not to breastfeed during treatment with BALVERSA and for one month following the last dose.

Nektar Therapeutics Announces Publication of Two Manuscripts on Lead Immuno-oncology Candidate, Bempegaldesleukin (Bempeg) in Nature Communications

On February 3, 2020 Nektar Therapeutics (NASDAQ: NKTR) reported the publication of preclinical data on its lead immuno-oncology candidate, NKTR-214, bempegaldesleukin (bempeg) in two manuscripts in Nature Communications (Press release, Nektar Therapeutics, FEB 3, 2020, View Source [SID1234553791]). Bempeg is an investigational CD122-preferential interleukin-2 (IL-2) pathway agonist designed to provide activation and proliferation of cancer-killing immune cells, known as CD8+ effector T cells and natural killer (NK) cells. The published data demonstrate that bempeg, in combination with immune-based therapies including checkpoint inhibition (CPI), antigen-specific vaccination and adoptive cell transfer (ACT) therapy, enhanced T-cell mediated tumor control by selectively expanding effector T cells (Teffs) over T regulatory cells (Tregs) in the tumor microenvironment.

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The first manuscript, titled "Bempegaldesleukin selectively depletes intratumoral Tregs and potentiates T cell-mediated cancer therapy," was published online and can be accessed here. The research was conducted by a team at MD Anderson Cancer Center, led by former tenured professor, Willem W. Overwijk, Ph.D., who currently serves as Vice President, Oncology Research at Nektar Therapeutics.

"Using preclinical models, we were able to demonstrate how bempeg potentiates both CPI and vaccination strategies by expanding and maintaining tumor infiltrating T cells and depleting intratumoral Tregs by a novel mechanism of cytokine release to drive efficient intratumoral Treg depletion," said Dr. Overwijk. "Our findings further define the mechanism of action of bempeg and highlight the rationale for its clinical application in combination with immune-based therapies for patients with cancer."

In the study, the activity and mechanism of action of bempeg was evaluated in combination with PD-1 and CTLA-4 CPI therapy and also with antigen-specific vaccination in preclinical animal models. The researchers concluded that bempeg in combination with PD-1 CPI therapy resulted in a higher response rate, prolonged disease control and yielded more complete responses in a variety of preclinical tumor types including colon cancer, melanoma, bladder cancer, lung cancer and breast cancer. In addition, bempeg supported the expansion and long-term maintenance of vaccination-induced anti-tumor Teffs; and bempeg treatment mediated selective depletion of intratumoral but not of peripheral Tregs, suggesting that in patients with cancer, bempeg-containing regimens could increase tumor control without exacerbating systemic inflammation. This preferential depletion of intratumoral Tregs was also observed in a small cohort of patients with renal cell cancer and melanoma receiving bempeg monotherapy, suggesting the results in mice may extend to patients receiving bempeg-based therapies.

Key findings are summarized below:

Bempeg with anti-PD-1 CPI as combination therapy demonstrated superior efficacy over anti-PD-1 monotherapy in 8 different mouse tumor models.
Anti-PD-1 monotherapy did not change TIL frequency or clonality, while addition of bempeg increased both TIL frequency and clonality.
To delve deeper into the immune-mediated mechanism, the B16 melanoma model was used with gp100 antigen-specific CD8+ (pmel-1) T cells to track T cell numbers and persistence.
Combination of gp100 vaccination and bempeg markedly suppressed tumor growth and prolonged mouse survival.
Long-term tumor control directly correlated with the magnitude and persistence of vaccination-induced, gp100-specific CD8+ Teff in the circulation. Aldesleukin (IL-2) also synergized with vaccination but did not achieve similar potency of bempeg as measured by tumor control or T cell responses.
While bempeg increased systemic and intratumoral levels of melanoma-specific CD8+ T cells, intratumoral numbers of Tregs plummeted.
Similar depletion of intratumoral but not systemic Tregs was also observed in patients with renal cell cancer and melanoma treated with bempeg (NCT02869295).
The authors present data that CD8+ Teff-derived IFN-ɣ and TNF-α synergize to deplete intratumoral Tregs by directly inhibiting local Treg proliferation.
In the second manuscript, published this week in Nature Communications, a research team led by Antoni Ribas, Ph.D. at the UCLA Jonsson Comprehensive Cancer Center evaluated bempeg in combination with adoptively transferred T cell therapy (ACT) in melanoma models. The studies found ACT therapy supported by bempeg increases the proliferation, homing and persistence of anti-tumor T cells compared to ACT with IL-2, resulting in superior antitumor activity in the B16 melanoma model.

Bempeg + ACT showed preferential activation of tumor-specific CD8 T cells over Tregs in spleen and tumors of mice, increased immune-related gene expression in the tumor microenvironment, and greatly increased animal survival compared to IL-2 + ACT.
Bempeg + ACT increased tumor-specific T cell polyfunctionality (secreting multiple (>2) cytokines per cell) both intratumorally and peripherally compared to IL-2 +ACT in mice.
Analysis of peripheral blood samples from patients treated with bempeg monotherapy in a Phase 1 trial (NCT02869295) also showed enhanced polyfunctionality of T cells and NK cells.
The authors conclude that bempeg may have the potential to improve the antitumor activity of ACT in humans through increased in vivo expansion, tumor infiltration and polyfunctionality of the adoptively transferred T cells.
The second manuscript titled "Persistence of adoptively transferred T cells with a kinetically engineered IL-2 receptor agonist," was published online and can be accessed here. The UCLA announcement of the publication can be accessed here.

Bempeg in combination with checkpoint inhibitors and other immune-mediating agents is being evaluated in clinical trials. For more information, visit the Pipeline page on nektar.com.

Portola Pharmaceuticals Appoints Rajiv Patni, M.D., as Executive Vice President and Chief Medical Officer

On February 3, 2020 Portola Pharmaceuticals, Inc. (Nasdaq: PTLA) reported the appointment of Rajiv Patni, M.D., as executive vice president and chief medical officer, effective immediately (Press release, Portola Pharmaceuticals, FEB 3, 2020, View Source [SID1234553790]). Dr. Patni brings to his new role two decades of clinical development and approval experience as a biopharmaceutical industry leader. As a member of the Executive Committee, Mr. Patni will report to Scott Garland, president and chief executive officer, and will be responsible for leading Clinical Development, Clinical Operations, Medical Affairs, Regulatory Affairs, Biometrics, Pharmacovigilance, and Data Management.

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"Rajiv brings deep clinical development experience to Portola that will be invaluable as we work to establish Andexxa as the standard of care for Factor Xa patients with life-threatening or uncontrolled bleeding," said Scott Garland, Portola’s president and chief executive officer. "He will focus on leading our post-market clinical studies, including label expansion, along with driving our European regulatory activities. These are key elements of our long-term growth strategy, and we look forward to his leadership in strengthening our healthcare community engagement in the U.S. and Europe further establishing the clinical and economic differentiation of Andexxa."

During his career, Dr. Patni has contributed to 8 approvals, across multiple therapeutic areas, including cardiology and neurology, in both established and growing companies. Before joining Portola, Dr. Patni was chief medical officer at Adamas Pharmaceuticals, Inc., a fully-integrated, development and commercial company focused on neurological diseases. In this role, he built out the R&D and medical affairs departments. These efforts resulted in the FDA approval of GOCOVRI. Prior to Adamas, Dr. Patni was Chief Development Officer at Ocera Therapeutics. Earlier in his career, he held senior roles in clinical development and medical affairs in Actelion Pharmaceuticals (US affiliate), Roche and Pfizer.

"I am excited to join Portola at this pivotal time of growth," said Dr. Patni. "The Company is uniquely positioned with the first and only reversal agent for Factor Xa inhibitors, a large and rapidly growing class of medications. I am eager to contribute to Portola’s mission and further enhance awareness of the potential life-saving benefits and value of Andexxa."

Dr. Patni received his M.D. from the Mount Sinai School of Medicine, and completed his training in internal medicine and cardiology at the Albert Einstein College of Medicine, where he remained as an attending physician before joining industry.

Jeff Myers, M.D., Ph.D., who has served as Portola’s interim chief medical officer since May 2019, will continue in his role as the Company’s senior vice president of medical affairs.

Applied BioMath, LLC Announces Collaboration with Gritstone Oncology, Inc

On February 3, 2020 Applied BioMath (www.appliedbiomath.com), the industry-leader in applying systems pharmacology and mechanistic modeling, simulation, and analysis to de-risk drug research and development, reported a collaboration with Gritstone Oncology, Inc. for semi-mechanistic systems pharmacology modeling (Press release, Applied BioMath, FEB 3, 2020, View Source [SID1234553789]). Applied BioMath created a semi-mechanistic pharmacokinetic, receptor occupancy model for a Gritstone Oncology, Inc. bispecific antibody. Gritstone plans to leverage this model for clinical candidate selection for its work in treating solid tumors. "Our collaboration with Applied BioMath helped us explore the impact of various drug properties such as affinity and half life," said Jonah Rainey, Vice President of Antibody Therapeutics at Gritstone Oncology, Inc. "Their modeling efforts provided a framework for identifying optimal bispecific design for lead selection."

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Applied BioMath employs a rigorous fit-for-purpose model development process which aims to quantitatively integrate knowledge about therapeutics with an understanding of its mechanism of action in the context of human disease mechanisms. Their approach employs proprietary algorithms and software that were designed specifically for mechanistic PK/PD modeling. "When you are designing a bispecific therapeutic, or any complex therapeutic, it’s extremely difficult to assess ideal drug properties without the aid of modeling," said Dr. John Burke, PhD, Co-Founder, President, and CEO of Applied BioMath. "Specifically, it’s the mechanistic component of our systems pharmacology modeling that enables us to closely replicate therapeutic and disease biology which in turn helps our clients quickly answer critical questions about their project."