Clovis Oncology Announces Availability of Rubraca®? (rucaparib) Tablets for Women with Relapsed Ovarian Cancer in Germany

On March 4, 2019 Clovis Oncology, Inc. (NASDAQ: CLVS) reported that Rubraca (rucaparib) is now available by prescription in Germany as monotherapy for the maintenance treatment of adults with platinum-sensitive relapsed high-grade epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in response (complete or partial) to platinum-based chemotherapy (Press release, Clovis Oncology, MAR 4, 2019, View Source [SID1234533922]). In addition, Rubraca is indicated as monotherapy treatment of adult patients with platinum sensitive, relapsed or progressive, BRCA mutated (germline and/or somatic), high-grade epithelial ovarian, fallopian tube, or primary peritoneal cancer, who have been treated with two or more prior lines of platinum-based chemotherapy, and who are unable to tolerate further platinum-based chemotherapy.

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On January 24, 2019, the European Commission (EC) expanded rucaparib’s indication beyond its initial marketing authorization in Europe for the treatment of advanced ovarian cancer in selected patients granted in May 2018. With this label expansion, rucaparib is now approved as maintenance treatment for eligible patients regardless of their BRCA-mutation status. Rucaparib is the first PARP inhibitor licensed for an ovarian cancer treatment indication in the EU and is now the first to be available for both treatment and maintenance treatment among eligible patients.

"I have been treating women with relapsed ovarian cancer under the Rucaparib Access Program and I am confident that rucaparib represents an important treatment option for women here in Germany," "said Professor Jalid Sehouli, Gynecologic Oncologist and head of the Charité European Competence Center for Ovarian Cancer at the University of Berlin. "There has been a significant need for additional treatment options for women with relapsed ovarian cancer, and rucaparib’s approval in the maintenance setting provides another option for these patients."

"With this milestone approval in Germany, we are one step closer to ensuring that Rubraca is available to all eligible women who may potentially benefit," said Patrick J. Mahaffy, President and CEO of Clovis Oncology. "Rubraca has shown further tumor shrinkage as well as prolonged progression-free survival in this maintenance setting, therefore Rubraca represents an important step forward for women with advanced ovarian cancer, regardless of their BRCA status."

The EC authorization is based on data from the phase 3 ARIEL3 clinical trial, which found that rucaparib significantly improved progression-free survival in all ovarian cancer patient populations studied.i

The ARIEL3 trial was a double-blind, placebo-controlled clinical trial of rucaparib that enrolled 564 women with recurrent epithelial ovarian, fallopian tube or primary peritoneal cancer in complete or partial response to platinum-based chemotherapy. Patients were randomized (2:1) to receive rucaparib tablets 600mg twice daily (n=375) or placebo (n=189). i

ARIEL3 successfully achieved its primary endpoint of extending investigator-assessed progression-free survival (PFS) versus placebo in all patients treated (intention-to-treat [ITT]), population, regardless of BRCA status (median 10.8 mos vs 5.4 mos); the key secondary endpoint of extending PFS as assessed by independent radiological review (IRR) was also achieved (median 13.7 mos vs 5.4 mos). ii

In a prespecified exploratory analysis of patients in the ITT population with measurable disease at baseline showed a tumor response was reported in 18 percent of patients (n=26) on rucaparib compared to eight percent of patients (n=5) on placebo, including seven percent (n=10) in the rucaparib group who achieved a complete remission. i

The overall safety profile of rucaparib is based on data from 937 patients with ovarian cancer treated with rucaparib monotherapy in clinical trials. Adverse reactions occurring in ≥20% of patients were nausea, fatigue/asthenia, vomiting, anemia, abdominal pain, dysgeusia, alanine aminotransferase (ALT) elevations, aspartate aminotransferase (AST) elevations, decreased appetite, diarrhea, thrombocytopenia and creatinine elevations. The majority of adverse reactions were mild to moderate (Grade 1 or 2). ii

Grade ≥3 adverse reactions occurring in >5% of patients were anemia (23%), ALT elevations (10%), fatigue/asthenia (10%), neutropenia (8%), thrombocytopenia (6%), and nausea (5%). The only serious adverse reaction occurring in > 2% of patients was anemia (5%). ii

Adverse reactions that most commonly led to dose reduction or interruption were anemia (20%), fatigue/asthenia (18%), nausea (16%), thrombocytopenia (15%), and AST/ALT elevations (10%). Adverse reactions leading to permanent discontinuation occurred in 10% of patients, with thrombocytopenia, nausea, anemia, and fatigue/asthenia being the most frequent adverse reactions leading to permanent discontinuation.ii

This release is only being distributed to members of the press and those health care practitioners allowed to receive prescription drug promotion.

About Ovarian Cancer in Europe and Germany

In 2018, ovarian cancer was the sixth most common cancer among women in Europe, with an estimated 68,000 women diagnosed and the fifth leading cause of cancer deaths among women, with an estimated 45,000 deaths annually. After initial therapy, many women’s disease will still recur, and approximately 70% of patients with ovarian cancer will relapse within the first three years following initial treatment. Germany has the highest incidence of new cases and deaths caused by ovarian cancer in Europe. The World Health Organization estimates that in 2018 there were approximately 6,800 new cases of ovarian cancer and 5,400 ovarian cancer-related deaths in the country.

About Rubraca (rucaparib)

Rucaparib is an oral, small molecule inhibitor of PARP1, PARP2 and PARP3 being developed in multiple tumor types, including ovarian, metastatic castration-resistant prostate, and bladder cancers, as monotherapy, and in combination with other anti-cancer agents. Exploratory studies in other tumor types are also underway.

Clovis holds worldwide rights for Rubraca. Rubraca is an unlicensed medical product outside of the U.S. and the EU.

Rubraca (rucaparib) EU Authorized Use and Important Safety Information

Rucaparib is indicated as monotherapy for the maintenance treatment of adult patients with platinum-sensitive relapsed high-grade epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in response (complete or partial) to platinum-based chemotherapy.

Rucaparib is indicated as monotherapy treatment of adult patients with platinum sensitive, relapsed or progressive, BRCA-mutated (germline and/or somatic), high-grade epithelial ovarian, fallopian tube, or primary peritoneal cancer, who have been treated with two or more prior lines of platinum-based chemotherapy, and who are unable to tolerate further platinum-based chemotherapy.

Summary warnings and precautions: Haematological toxicity: Patients should not start rucaparib until they have recovered from haematological toxicities caused by previous chemotherapy (≤ CTCAE Grade 1). Complete blood count testing prior to starting treatment with rucaparib and monthly thereafter is advised. Rucaparib should be interrupted or dose reduced, and blood counts monitored weekly until recovery for the management of low blood counts. Myelodysplastic syndrome/acute myeloid leukaemia (MDS/AML): If MDS/AML is suspected, the patient should be referred to a haematologist for further investigation. If MDS/AML is confirmed, rucaparib should be discontinued. Photosensitivity: Patients should avoid spending time in direct sunlight as they may burn more easily. When outdoors, patients should wear protective clothing and sunscreen with SPF of 50 or greater. Gastrointestinal toxicities: Low grade (CTCAE Grade 1 or 2) nausea and vomiting may be managed with dose reduction or interruption. Additionally, antiemetics may be considered for treatment or prophylaxis.

Click here to access the current Summary of Product Characteristics. Healthcare professionals should report any suspected adverse reactions via their national reporting systems.

Invitation to Year-End Results 2018 Conference Call of MorphoSys AG on March 14, 2019

On March 4, 2019 MorphoSys AG (FSE: MOR; Prime Standard Segment, TecDAX & MDAX; Nasdaq: MOR) reported that it will publish its Annual Financial Results 2018 on March 13, 2019 at 10:00pm CET (9:00pm GMT; 5:00pm EDT) (Press release, MorphoSys, MAR 4, 2019, View Source [SID1234533921]).

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The Management team of MorphoSys AG will host a conference call and webcast on March 14, 2019 at 2:00pm CET (1:00pm GMT; 9:00am EDT) to present MorphoSys’s Annual Financial Results 2018 and provide an outlook for 2019.

Date of the conference call: Thursday, March 14, 2019
Time: 2:00pm CET (1:00pm GMT, 9:00am EDT)
Dial-in numbers (listen only)
Germany: +49 69 201 744 220
United Kingdom: +44 203 009 2470
USA: +1 877 423 0830
Participant PIN: 88207738#

Company participants in the call will be:

Dr. Simon Moroney, Chief Executive Officer
Jens Holstein, Chief Financial Officer
Dr. Malte Peters, Chief Development Officer
Dr. Markus Enzelberger, Chief Scientific Officer

Participants are kindly requested to dial in up to 10 minutes before the call to ensure a secure line and a prompt start.

The presentation slides and webcast link will be available at the Company’s website at View Source

A replay of the conference will also be available at the corporate website following the live event.

Halozyme To Participate In Upcoming Investor Conferences

On March 4, 2019 Halozyme Therapeutics, Inc. (NASDAQ: HALO), a biotechnology company developing novel oncology and drug-delivery therapies, reported that it will participate in two upcoming investor conferences (Press release, Halozyme, MAR 4, 2019, View Source [SID1234533920]).

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The 39th Annual Cowen Healthcare Conference on Monday, March 11 at 4:50 p.m. ET / 1:50 p.m. PT in Boston, MA. Dr. Helen Torley, president and chief executive officer, will present an overview of the company.
The Barclays Global Healthcare Conference on Tuesday, March 12 at 3:50 p.m. ET / 12:50 p.m. PT in Miami, FL. Laurie Stelzer, senior vice president and chief financial officer, will present a brief overview of the company followed by a question and answer session.
A live webcast of each presentation can be accessed through the "Investors" section of www.halozyme.com, and a recording will be made available for 90 days following each event. To access a live webcast, please visit Halozyme’s website approximately 15 minutes prior to the presentation to register and download any necessary audio software.

Agios to Present at the Cowen 39th Annual Health Care Conference on Monday, March 11, 2019

On March 4, 2019 Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, reported that the company is scheduled to present at the Cowen 39th Annual Health Care Conference in Boston on Monday, March 11, 2019 at 12:00 p.m. ET (Press release, Agios Pharmaceuticals, MAR 4, 2019, http://investor.agios.com/news-releases/news-release-details/agios-present-cowen-39th-annual-health-care-conference-monday [SID1234533919]).

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A live webcast of the presentation can be accessed under "Events & Presentations" in the Investors section of the company’s website at www.agios.com. A replay of the webcast will be archived on the Agios website for at least two weeks following the presentation.

BioCryst Reports Fourth Quarter and Full Year 2018 Financial Results

On March 4, 2019 BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) reported financial results for the fourth quarter and full year ended December 31, 2018, and provided a corporate update (Press release, BioCryst Pharmaceuticals, MAR 4, 2019, View Source [SID1234533918]).

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"In a year with many transformative milestones for BioCryst, it has been exciting to see so much progress already in the first two months of the year. The strong Phase 2 clinical data from our now-completed ZENITH-1 trial propels our BCX7353 acute program for HAE into Phase 3 development. With the very favorable preclinical profile of BCX9930, another novel BioCryst-invented oral drug for rare diseases, we are advancing that program into the clinic in the second quarter for the treatment of complement-mediated diseases. We also added further rare disease expertise to our board and increased our financial flexibility with a $100 million debt agreement," said Jon Stonehouse, president and chief executive officer of BioCryst.

"We remain on-track for the readout of our APeX-2 trial next quarter, and an NDA filing of BCX7353 for HAE prophylaxis by the end of the year. We are thoughtfully building our commercial leadership and infrastructure to execute a successful launch that meets the urgent demand for a once-daily oral therapy that will allow HAE patients to live a more normal life," Stonehouse added.

Recent Milestones

The company has dosed the first patients in its APeX-J trial in Japan, designed to support potential Japanese approval of BCX7353 for the prevention of HAE attacks.

On March 4, 2019, the company announced that it is advancing BCX9930, an oral Factor D inhibitor, into Phase 1 clinical development in the second quarter of 2019 for the treatment of complement-mediated diseases.
On February 23, 2019, the company announced data from the completed ZENITH-1 trial (including the 250 mg and 500 mg dose cohorts) of BCX7353 for the acute treatment of HAE attacks at the annual meeting of the American Academy of Allergy, Asthma & Immunology. The company plans to commence a Phase 3 trial, ZENITH-2, in the summer of 2019.

On February 6, 2019, the company announced it had entered into a $100 million secured credit facility with MidCap Financial Trust pursuant to the terms and conditions of an amended and restated credit and security agreement.

On January 4, 2019, the company announced it had appointed Steve Aselage to its board of directors.

On January 2, 2019, the company announced the dosing of the first subject in a randomized, placebo-controlled Phase 1 clinical trial to evaluate intravenous galidesivir, its investigational broad-spectrum antiviral drug, in healthy volunteers.
Fourth Quarter 2018 Corporate Developments

On November 20, 2018, BioCryst announced that it had appointed Theresa Heggie to its board of directors.

On November 16, 2018, BioCryst presented data that showed an oral formulation of BCX7353 was rapidly absorbed and exhibited a long half-life, two important characteristics of desired new acute treatments for HAE attacks, at the annual scientific meeting of the American College of Allergy, Asthma & Immunology.
Upcoming Key Milestones

HAE Program – BCX7353

Report 24-week safety and efficacy results from the APeX-2 clinical trial (Q2 2019)

Begin a Phase 3 clinical trial of oral BCX7353 for the acute treatment of HAE (Summer 2019)

File a new drug application (NDA) for oral BCX7353 for the prevention of HAE attacks with the U.S. Food and Drug Administration (FDA) (Q4 2019)
Complement Factor D Inhibitor Program – BCX9930

Begin a Phase 1 trial of BCX9930, an oral Factor D inhibitor for treatment of complement-mediated diseases, in healthy subjects (Q2 2019)

Report Phase 1 results (Q4 2019)
ALK-2 Inhibitor Program – BCX9250

Begin a Phase 1 clinical trial of BCX9250, an oral ALK-2 kinase inhibitor for treatment of fibrodysplasia ossificans progressiva, in healthy subjects (2H 2019)
Fourth Quarter 2018 Financial Results

For the three months ended December 31, 2018, total revenues were $2.7 million, compared to $3.9 million in the fourth quarter of 2017. The decrease was primarily due to a reduction of royalty revenue associated with differences in the onset and severity of the influenza seasons between the two periods. This decrease was partially offset by increased revenue from government contracts for galidesivir development, which was higher in the fourth quarter of 2018.

Research and development (R&D) expenses for the fourth quarter of 2018 increased to $23.4 million from $16.9 million in the fourth quarter of 2017, primarily due to increased spending on the company’s HAE and preclinical programs.

General and administrative (G&A) expenses for the fourth quarter of 2018 decreased slightly to $4.5 million, compared to $4.7 million in the fourth quarter of 2017. The decrease was primarily due to the lack of merger-related costs in the fourth quarter of 2018 associated with the company’s terminated merger with Idera Pharmaceuticals, Inc. (Idera). The decrease in G&A expense due to the lack of merger expenses was largely offset by higher commercial and medical affairs expenses in the fourth quarter of 2018.

Interest expense was $2.4 million in the fourth quarter of 2018, compared to $2.2 million in the fourth quarter of 2017 and was associated with enhancing our secured credit facility in July 2018.

Net loss for the fourth quarter of 2018 was $27.4 million, or $0.25 per share, compared to a net loss of $19.5 million, or $0.20 per share, for the fourth quarter of 2017.

Cash, cash equivalents and investments totaled $128.4 million at December 31, 2018, and reflect a decrease from $159.0 million at December 31, 2017. Cash and investments reflect the proceeds from a July 2018 enhancement to our secured credit facility and an August 2018 public equity offering, offset by normal operating expenses and merger-related costs incurred in the 12-month period. Operating cash use for the fourth quarter of 2018 was $22.6 million, and for the full year of 2018 was $93.4 million.

On February 6, 2019, the company announced it had entered into a $100 million secured credit facility with MidCap Financial Trust which further enhanced the company’s cash position with $20 million of immediate additional non-dilutive capital and provided additional financial flexibility by providing the ability to draw another $50 million of milestone-based non-dilutive capital.

Full Year 2018 Financial Results

For the full year ended December 31, 2018, total revenues were $20.7 million, compared to $25.2 million in the full year ended December 31, 2017. The decrease in revenue was primarily associated with infrequent revenue events that occurred in 2017 that did not recur in 2018, as well as a $2.1 million decrease in revenue associated with development activities under U.S. government contracts in 2018. The non-recurring 2017 events were the recognition of $4.1 million of royalty revenue from Japanese government stockpiling of RAPIACTA and the recognition of $1.5 million of peramivir product sales from the company’s commercial partner, Green Cross Corporation. These decreases were partially offset by a $5.0 million milestone associated with the European Medicines Agency’s (EMA) approval of peramivir (ALPIVABTM) recognized in the second quarter of 2018.

R&D expenses in 2018 increased to $84.9 million from $67.0 million in 2017, primarily due to increased spending on our HAE and preclinical programs. These increases were partially offset by a decrease in the company’s peramivir and galidesivir development spending in 2018.

G&A expenses in 2018 increased to $29.5 million, compared to $13.9 million in 2017. The increase was primarily due to approximately $11 million of merger-related costs associated with the company’s terminated merger with Idera and a $4.9 million reserve for collectability of the EMA approval milestone of peramivir.

Interest expense was $9.2 million in 2018, compared to $8.6 million in 2017.

Net loss for 2018 was $101.3 million, or $0.98 per share, compared to a net loss of $65.8 million, or $0.78 per share, for 2017.

Financial Outlook for 2019

BioCryst expects net operating cash use to be in the range of $105 to $130 million, and its 2019 operating expenses to be in the range of $120 to $145 million. The company’s operating expense range excludes equity-based compensation expense due to the difficulty in reliably projecting this expense, as it is impacted by the volatility and price of the company’s stock, as well as by the vesting of the company’s outstanding performance-based stock options.

Conference Call and Webcast

BioCryst management will host a conference call and webcast at 8:30 a.m. ET today to discuss the financial results and provide a corporate update. The live call may be accessed by dialing 877-303-8027 for domestic callers and 760-536-5165 for international callers and using conference ID # 1271286. A live webcast of the call and any slides will be available online at the investors section of the company website at www.biocryst.com. A telephone replay of the call will be available by dialing 855-859-2056 for domestic callers or 404-537-3406 for international callers and entering the conference ID# 1271286.