PharmaMar and Jazz Pharmaceuticals Sign Exclusive License Agreement for Lurbinectedin in the U.S.

On December 19, 2019 PharmaMar (MSE:PHM) and Jazz Pharmaceuticals plc (Nasdaq:JAZZ) reported that PharmaMar and Jazz Pharmaceuticals Ireland Limited have entered into an exclusive license agreement for lurbinectedin in the United States (Press release, PharmaMar, DEC 19, 2019, View Source [SID1234552516]).

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Under the terms of this agreement, PharmaMar will receive an upfront payment of $200 million with potential regulatory milestone payments of up to $250 million upon the achievement of accelerated and/or full regulatory approval of lurbinectedin by FDA within certain timelines.

PharmaMar is also eligible to receive up to $550 million in potential commercial milestone payments, as well as incremental tiered royalties on future net sales of lurbinectedin ranging from the high teens up to 30 percent. PharmaMar may receive additional payments on approval of other indications. PharmaMar retains production rights for lurbinectedin and will supply the product to Jazz.

Lurbinectedin was granted orphan drug designation for SCLC by FDA in August 2018. In December 2019, PharmaMar submitted an NDA to FDA for accelerated approval of lurbinectedin for relapsed SCLC, based on data from its Phase 2 basket trial, following positive interactions with FDA.

The lurbinectedin Phase 2 monotherapy basket trial enrolled a total of 105 patients at 39 centers in eight Western European countries in addition to the U.S. The primary endpoint was Overall Response Rate (ORR) as measured by investigator review assessment. Secondary endpoints included Duration of Response, Progression-Free Survival, Overall Survival, and safety. In relapsed SCLC, lurbinectedin showed an ORR of 35.2%, which compares favorably to topotecan’s historical ORR of 16.9%1 by investigator assessment. In addition, lurbinectedin demonstrated a favorable safety, tolerability and administration profile versus historical standard of care.

"We are very pleased with the lurbinectedin agreement with our new U.S. partner Jazz," said José María Fernández Sousa-Faro, PhD, President of PharmaMar. "We are convinced that with Jazz, we have found a partner deeply committed to providing lurbinectedin to patients in the U.S. Lurbinectedin has the potential to become a therapeutic alternative for patients with relapsed small cell lung cancer, who have limited treatment options."

"Lurbinectedin represents a strong strategic fit and an exciting opportunity for Jazz to expand our oncology portfolio with a late stage asset," said Bruce C. Cozadd, Chairman and CEO of Jazz Pharmaceuticals. "We are looking forward to commercializing lurbinectedin in the U.S., as SCLC is an area of significant unmet medical need given limited late-stage treatment options and we believe lurbinectedin may offer patients with relapsed SCLC a meaningful treatment option."

SCLC is a very aggressive cancer that usually is diagnosed with advanced, often metastatic, disease, thus limiting the role of traditional approaches and often posing a worse prognosis when compared to other lung cancers2. In the U.S., approximately 10-15% of lung cancers are small cell.2 Approximately 30,000 new cases of SCLC are recorded in the U.S. every year3.

Closing of the agreement is subject to expiration or termination of the waiting period under the Hart-Scott-Rodino Act.

PharmaMar Conference Call for Investors and Analysts

PharmaMar management will host a conference call and webcast for investors and analysts on January 9th, 2020, at 14:00 CET (08:00 AM, New York time) as follows. The numbers to connect to the teleconference are 877-407-3102 (from USA or Canada) and +1 201-493-6790 (other countries). Interested parties can also follow the conference call live via the following link: https://78449.themediaframe.com/dataconf/productusers/phm/mediaframe/33803/indexl.html

The recording of the teleconference will be available for thirty days and it can be accessed on PharmaMar’s website by visiting the Events Calendar section of the Company’s website www.pharmamar.com.

Jazz Pharmaceuticals Conference Call for Investors and Analysts

Jazz Pharmaceuticals will host an investor conference call and live audio webcast on Friday, January 10, 2020 at 8:30 a.m. EST (1:30 p.m. GMT) to discuss the transaction. The live webcast may be accessed from the Investors section of Jazz Pharmaceuticals’ website at www.jazzpharmaceuticals.com. Please connect to the website prior to the start of the conference call to ensure adequate time for any software downloads that may be necessary. Investors may participate in the conference call by dialing +1 855 353 7924 in the U.S., or +1 503 343 6056 outside the U.S., and entering passcode 4069667.

A replay of the conference call will be available through January 17, 2020 by dialing +1 855 859 2056 in the U.S., or +1 404 537 3406 outside the U.S., and entering passcode 4069667. An archived version of the webcast will be available for at least one week in the Investors section of Jazz Pharmaceuticals’ website at www.jazzpharmaceuticals.com

About Lurbinectedin
Lurbinectedin (PM1183) is a synthetic compound currently under clinical investigation. It is a selective inhibitor of the oncogenic transcription programs on which many tumors are particularly dependent. Together with its effect on cancer cells, lurbinectedin inhibits oncogenic transcription in tumor-associated macrophages, downregulating the production of cytokines that are essential for the growth of the tumor. Transcriptional addiction is an acknowledged target in those diseases, many of them lacking other actionable targets.

OPKO Health to Present at the 38th Annual J.P. Morgan Healthcare Conference

On December 19, 2019 OPKO Health, Inc. (OPK) reported that management will be participating in the 38th Annual J.P. Morgan Healthcare Conference being held January 13-16, 2020 at the Westin St. Francis in San Francisco (Press release, Opko Health, DEC 19, 2019, View Source [SID1234552515]). Management will be hosting one-on-one meetings with investors and will be presenting on Wednesday, January 15 at 10:30 a.m. Pacific time (1:30 p.m. Eastern time).

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The presentation will be webcast live and available for replay in the Investors section of OPKO Health’s website and accessible by clicking here.

Exelixis Enters into a Clinical Collaboration for Three Phase 3 Combination Trials for Patients with Advanced Solid Tumors

On December 19, 2019 Exelixis, Inc. (NASDAQ:EXEL) reported a collaboration agreement with Roche to evaluate cabozantinib (CABOMETYX), Exelixis’ small molecule inhibitor of receptor tyrosine kinases, in combination with atezolizumab (TECENTRIQ), Roche’s PD-L1 immune checkpoint inhibitor, in patients with locally advanced or metastatic solid tumors (Press release, Exelixis, DEC 19, 2019, View Source [SID1234552513]). The clinical program, which will be co-funded by the companies, is expected to include three phase 3 pivotal trials in advanced non-small cell lung cancer (NSCLC), castration-resistant prostate cancer (CRPC) and renal cell carcinoma (RCC).

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"Encouraging phase 1 data suggests this combination of cabozantinib and atezolizumab may improve outcomes for patients with prostate, lung and kidney cancers, and we look forward to collaborating with Roche to learn more in these pivotal trials," said Michael M. Morrissey, Ph.D., President and Chief Executive Officer of Exelixis. "This clinical collaboration is an important further step in our committed efforts to maximize the value of the cabozantinib franchise through these cost-sharing clinical collaborations in additional high-impact indications, while building value with new compounds from internal and external sources in 2020 and beyond."

The clinical development collaboration builds on encouraging activity observed in the phase 1b COSMIC-021 trial. The trial is currently enrolling 24 expansion cohorts in 12 tumor types including RCC, NSCLC and CRPC.

TECENTRIQ (atezolizumab) is a registered trademark of Genentech, a member of the Roche Group.

About CABOMETYX (cabozantinib)

In the U.S., CABOMETYX tablets are approved for the treatment of patients with advanced RCC and for the treatment of patients with hepatocellular carcinoma who have been previously treated with sorafenib. CABOMETYX tablets have also received regulatory approvals in the European Union and additional countries and regions worldwide.

About Exelixis’ Collaboration with Ipsen

On February 29, 2016, Exelixis and Ipsen jointly announced an exclusive licensing agreement for the commercialization and further development of cabozantinib indications outside of the United States, Canada and Japan. On December 21, 2016, this agreement was amended to include commercialization rights for Ipsen in Canada. Under the parties’ collaboration agreement, if Ipsen opts to participate in funding these phase 3 trials, or future studies, Ipsen will have access to the respective study results to support potential future regulatory submissions in their territory.

About Exelixis’ Collaboration with Takeda

On January 30, 2017, Exelixis and Takeda jointly announced an exclusive licensing agreement for the commercialization and further development of cabozantinib indications in Japan. Under the parties’ collaboration agreement, if Takeda opts to participate in funding these phase 3 trials, or future studies, Takeda will have access to the respective study results to support potential future regulatory submissions in their territory.

Exelixis holds the exclusive rights to develop and commercialize cabozantinib in the United States.

U.S. Important Safety Information

Hemorrhage: Severe and fatal hemorrhages occurred with CABOMETYX. The incidence of Grade 3 to 5 hemorrhagic events was 5% in CABOMETYX patients. Discontinue CABOMETYX for Grade 3 or 4 hemorrhage. Do not administer CABOMETYX to patients who have a recent history of hemorrhage, including hemoptysis, hematemesis, or melena.
Perforations and Fistulas: GastrointestinaI (GI) perforations, including fatal cases, occurred in 1% of CABOMETYX patients. Fistulas, including fatal cases, occurred in 1% of CABOMETYX patients. Monitor patients for signs and symptoms of perforations and fistulas, including abscess and sepsis. Discontinue CABOMETYX in patients who experience a fistula that cannot be appropriately managed or a GI perforation.
Thrombotic Events: CABOMETYX increased the risk of thrombotic events. Venous thromboembolism occurred in 7% (including 4% pulmonary embolism) and arterial thromboembolism in 2% of CABOMETYX patients. Fatal thrombotic events occurred in CABOMETYX patients. Discontinue CABOMETYX in patients who develop an acute myocardial infarction or serious arterial or venous thromboembolic event requiring medical intervention.
Hypertension and Hypertensive Crisis: CABOMETYX can cause hypertension, including hypertensive crisis. Hypertension occurred in 36% (17% Grade 3 and <1% Grade 4) of CABOMETYX patients. Do not initiate CABOMETYX in patients with uncontrolled hypertension. Monitor blood pressure regularly during CABOMETYX treatment. Withhold CABOMETYX for hypertension that is not adequately controlled with medical management; when controlled, resume at a reduced dose. Discontinue CABOMETYX for severe hypertension that cannot be controlled with anti-hypertensive therapy or for hypertensive crisis.
Diarrhea: Diarrhea occurred in 63% of CABOMETYX patients. Grade 3 diarrhea occurred in 11% of CABOMETYX patients. Withhold CABOMETYX until improvement to Grade 1 and resume at a reduced dose for intolerable Grade 2 diarrhea, Grade 3 diarrhea that cannot be managed with standard antidiarrheal treatments, or Grade 4 diarrhea.
Palmar-Plantar Erythrodysesthesia (PPE): PPE occurred in 44% of CABOMETYX patients. Grade 3 PPE occurred in 13% of CABOMETYX patients. Withhold CABOMETYX until improvement to Grade 1 and resume at a reduced dose for intolerable Grade 2 PPE or Grade 3 PPE.
Proteinuria: Proteinuria occurred in 7% of CABOMETYX patients. Monitor urine protein regularly during CABOMETYX treatment. Discontinue CABOMETYX in patients who develop nephrotic syndrome.
Osteonecrosis of the Jaw (ONJ): ONJ occurred in <1% of CABOMETYX patients. ONJ can manifest as jaw pain, osteomyelitis, osteitis, bone erosion, tooth or periodontal infection, toothache, gingival ulceration or erosion, persistent jaw pain, or slow healing of the mouth or jaw after dental surgery. Perform an oral examination prior to CABOMETYX initiation and periodically during treatment. Advise patients regarding good oral hygiene practices. Withhold CABOMETYX for at least 28 days prior to scheduled dental surgery or invasive dental procedures. Withhold CABOMETYX for development of ONJ until complete resolution.
Wound Complications: Wound complications were reported with CABOMETYX. Stop CABOMETYX at least 28 days prior to scheduled surgery. Resume CABOMETYX after surgery based on clinical judgment of adequate wound healing. Withhold CABOMETYX in patients with dehiscence or wound healing complications requiring medical intervention.
Reversible Posterior Leukoencephalopathy Syndrome (RPLS): RPLS, a syndrome of subcortical vasogenic edema diagnosed by characteristic finding on MRI, can occur with CABOMETYX. Evaluate for RPLS in patients presenting with seizures, headache, visual disturbances, confusion, or altered mental function. Discontinue CABOMETYX in patients who develop RPLS.
Embryo-Fetal Toxicity: CABOMETYX can cause fetal harm. Advise pregnant women and females of reproductive potential of the potential risk to a fetus. Verify the pregnancy status of females of reproductive potential prior to initiating CABOMETYX and advise them to use effective contraception during treatment and for 4 months after the last dose.
Adverse Reactions: The most commonly reported (≥25%) adverse reactions are: diarrhea, fatigue, decreased appetite, PPE, nausea, hypertension, and vomiting.
Strong CYP3A4 Inhibitors: If coadministration with strong CYP3A4 inhibitors cannot be avoided, reduce the CABOMETYX dosage. Avoid grapefruit or grapefruit juice.
Strong CYP3A4 Inducers: If coadministration with strong CYP3A4 inducers cannot be avoided, increase the CABOMETYX dosage. Avoid St. John’s wort.
Lactation: Advise women not to breastfeed during CABOMETYX treatment and for 4 months after the final dose.
Hepatic Impairment: In patients with moderate hepatic impairment, reduce the CABOMETYX dosage. CABOMETYX is not recommended for use in patients with severe hepatic impairment.

Ultragenyx to Present at 38ᵗʰ Annual J.P. Morgan Healthcare Conference

On December 19, 2019 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, reported that Emil D. Kakkis, M.D., Ph.D., the company’s Chief Executive Officer and President, will present at the 38TH Annual J.P. Morgan Healthcare Conference on Tuesday, January 14, 2020 at 12:00 pm PT in San Francisco (Press release, Ultragenyx Pharmaceutical, DEC 19, 2019, View Source [SID1234552511]).

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The live and archived webcast of the presentation will be accessible from the company’s website at View Source The replay of the webcast will be available for 90 days.

THERATECHNOLOGIES ISSUES PRELIMINARY REVENUE ESTIMATES FOR FISCAL YEAR 2019 AND REVENUE GUIDANCE FOR FISCAL YEAR 2020

On December 19, 2019 Theratechnologies Inc. (Theratechnologies) (TSX: TH) (NASDAQ: THTX), a commercial-stage biopharmaceutical company, reported that preliminary revenue estimates for its recently completed fiscal year 2019 and revenue guidance for fiscal year 2020 (Press release, Theratechnologies, DEC 19, 2019, View Source [SID1234552510]).

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Theratechnologies expects to report consolidated revenues of approximately US$63.3 million for fiscal year 2019 compared to US$45.2 million for the previous year, representing an increase of approximately 40%.

Consolidated revenues of approximately US$16.5 million for the fourth quarter of 2019 represent an increase of 17.7% compared to Q4 of 2018, and 2.1% compared to Q3 2019.

Fourth quarter net sales of Trogarzo (ibalizumab-uiyk, injection) are expected to be approximately US$7.7 million, an increase of approximately 81% compared to the same quarter of 2018 and approximately 11.3% compared to Q3 of this year.

Net sales of EGRIFTA are expected to be approximately US$8.7 million for the fourth quarter of 2019, a decrease of 10% compared to the same quarter of 2018 and of 4.8% compared to Q3 of this year. Although unit sales actually increased 5.3% compared to both Q4 2018 and Q3 2019, net sales for Q4 2019 were impacted by an unexpected charge related to government rebates not previously recorded by one of our distributing pharmacies. A portion of units sold to this pharmacy were previously incorrectly identified by the pharmacy as commercial patients, when they were actually government reimbursed patients, who are eligible to rebates.

"Our fiscal year 2019 was one of growth and transition. Our revenues grew strongly, and we rebuilt our research pipeline. In 2020, we will implement numerous new marketing initiatives for Trogarzo in the US, and plan to start the commercialization of this product in Europe. We are also confident that the recent launch of EGRIFTA SVTM will spark renewed interest and momentum for this product for the coming years. On the R&D front, we plan to start, by the end of 2020, a Phase III trial for the treatment of NASH in HIV patients and a Phase I trial for our novel oncology platform," said Luc Tanguay, President and CEO, Theratechnologies Inc.

2020 revenue guidance

Theratechnologies expects revenues to reach between US$83 to US$87 million in 2020. This would represent a 31 to 37 percent increase from fiscal year 2019.

The Company still expects to maintain a solid cash position as the estimated growth in revenue will generate enough cash to fund its operations and its clinical research programs in 2020.

"We intend to remain focused on implementing our strategic plan rigorously and to make 2020 a pivotal year for the Company from a revenue perspective while also continuing to build the future of our Company through the development of our promising pipeline. This Company is stronger than it has ever been, and we know that we can accomplish our ambitious yet realistic strategic plan," concluded Mr. Tanguay.