On May 2, 2019 Context Therapeutics, a clinical-stage biopharmaceutical company dedicated to advancing medicines for hormone driven cancers, reported the start of an open-label (NCT03909152) basket study to evaluate the efficacy and safety of Apristor (onapristone extended release) in advanced gynecological tumors that are progesterone receptor positive (PgR+) (Press release, Context Therapeutics, MAY 2, 2019, View Source [SID1234535585]). Rachel N. Grisham, MD of Memorial Sloan Kettering Cancer Center is the study’s Primary Investigator.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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 Phase 2 trial will include patients who have different PgR+ solid tumors in advanced stages, including low grade serous ovarian, granulosa cell, and endometrioid endometrial cancer, who have received prior chemotherapy treatment. The progesterone receptor antagonist Apristor will be assessed in up to 84 cancer patients. The primary endpoint will be overall response rate (ORR), which is the proportion of patients who have either a complete or partial response. To further characterize the activity of Apristor, secondary endpoints will include duration of response, clinical benefit rate, and progression-free survival (PFS). In addition, this study will evaluate the safety and pharmacological profile of Apristor in these patients, as well as biomarker analyses to explore predictive factors of response to Apristor. Preliminary trial results are expected in mid-2020.

"Currently, there are limited therapeutic options to treat these cancers in the advanced setting. Recent preclinical findings, together with Phase 1 study results in patients with advanced PgR+ ovarian and endometrial cancers, give us reason to believe that Apristor can help these women with PgR+ gynecological cancers," said Tarek Sahmoud, MD, PhD, Chief Medical Officer of Context Therapeutics. 1,2 "We believe Apristor can make a meaningful difference for patients in the trial."

For more information about the trial being conducted, please visit: View Source

About Apristor

Apristor (onapristone extended release) is an investigational new drug that is an orally administered full progesterone receptor antagonist. Currently, there are no approved therapies that selectively target PgR+ cancers. In a recent Phase 1 trial, Apristor was well tolerated and exhibited clinical benefit in heavily pretreated patients with PgR+ endometrial, ovarian and breast cancer. Preclinical data suggest that Apristor has anticancer activity by blocking the activation of progesterone receptor signaling by progesterone and/or mitogenic growth factors, inhibiting mammosphere formation and downregulating gene signatures associated with cancer stem cell mobilization and metastasis3,4.

On May 2, 2019 Zai Lab Limited ("Zai Lab" or the "Company") (NASDAQ: ZLAB), a China and U.S.-based innovative commercial stage biopharmaceutical company, reported the pricing of its underwritten public offering of 7,843,138 American depositary shares ("ADSs"), each representing one ordinary share of the Company, at a price of US$25.50 per ADS (Press release, Zai Laboratory, MAY 2, 2019, View Source [SID1234535653]). The offering was upsized from the previously announced offering size of 5,000,000 ADSs.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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 gross proceeds to Zai Lab from the offering, before deducting underwriting discounts and commissions and other offering expenses, are expected to be approximately US$200.0 million. In addition, Zai Lab has granted the underwriters a 30-day option to purchase up to an additional 1,176,470 ADSs at the public offering price, less underwriting discounts and commissions. The offering is expected to close on May 7, 2019, subject to customary closing conditions.

J.P. Morgan, Citigroup, Jefferies and SVB Leerink are acting as joint book-running managers for the offering.

The ADS are offered pursuant to a shelf registration statement on Form F-3ASR, which became automatically effective upon filing with the U.S. Securities and Exchange Commission ("SEC") on March 29, 2019.

The offering is being made only by means of a prospectus supplement and an accompanying prospectus included in Form-3ASR. The registration statement on Form F-3ASR and the prospectus supplement are available at the SEC’s website at: View Source Copies of the prospectus supplement and the accompanying prospectus may be obtained from: (i) J.P. Morgan Securities LLC, Attention: Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, telephone: 1-866-803-9204 or email: [email protected], (ii) Citigroup Global Markets Inc., c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717 or telephone: 1-800-831-9146 (iii) Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, 2nd Floor, New York, NY 10022, by telephone at 1-877-821-7388, or by email at [email protected] or (iv) SVB Leerink LLC, Attention: Syndicate Department, One Federal Street, 37th Floor, Boston, MA 02110, telephone: 1-800-808-7525 ex. 6132 or email: [email protected].

This press release does not constitute an offer to sell or the solicitation of an offer to buy ADSs or any other securities, nor shall there be any sale of ADSs in any state or jurisdiction in which such an offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

On May 2, 2019 Amgen (NASDAQ:AMGN), a world leader in biotechnology, and Syapse, a company powering precision medicine insights through its global provider network, reported a precision medicine collaboration in oncology (Press release, Amgen, MAY 2, 2019, View Source;p=RssLanding&cat=news&id=2396920 [SID1234535691]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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!

Under the terms of the agreement, Amgen and Syapse will develop observational research analytics to assess treatment outcomes for areas of unmet need in oncology. This effort will identify existing patients within the Syapse Learning Health Network that could be eligible for Amgen-sponsored clinical trials and seek to bring these trials to community health system sites. The companies will create opportunities for physicians and researchers within the Syapse Network to gain access to analytics, real-world evidence-based insights, and collaborative research opportunities. Amgen will have access to real-world evidence for potential use in regulatory filings in support of certain agreed upon development candidates in oncology. Amgen will also work with Syapse to develop real-world evidence standards to support the acceleration of therapies to market.

"As cancer remains one of the leading causes of death around the world, emerging software and data analytic tools are creating exciting opportunities to more rapidly develop and deliver targeted treatment options to patients," said Mike Nohaile, senior vice president of Strategy, Commercialization and Innovation at Amgen. "Our collaboration with Syapse supports this effort by leveraging real-world evidence to accelerate bringing new oncology treatments to market and empowers healthcare providers with more robust insights and decision-making tools to improve patient care."

"Syapse and Amgen share a common vision of ensuring that all cancer patients, regardless of income or location, receive access to the best possible care," said Ken Tarkoff, chief executive officer at Syapse. "Through this collaboration and the strategic relationships we’ve built with community health systems globally, we aim to develop evidence of clinical utility that can be used to bring molecularly targeted treatments to market more quickly and ensure more diverse groups of patients can access them."

About Amgen Oncology
Amgen Oncology is searching for and finding answers to incredibly complex questions that will advance care and improve lives for cancer patients and their families. Our research drives us to understand the disease in the context of the patient’s life – not just their cancer journey – so they can take control of their lives.

For the last four decades, we have been dedicated to discovering the firsts that matter in oncology and to finding ways to reduce the burden of cancer. Building on our heritage, Amgen continues to advance the largest pipeline in the company’s history, moving with great speed to advance those innovations for the patients who need them.

At Amgen, we are driven by our commitment to transform the lives of cancer patients and keep them at the center of everything we do.

On May 2, 2019 ViewRay, Inc. (Nasdaq: VRAY) reported financial results for the first quarter ended March 31, 2019 (Press release, ViewRay, MAY 2, 2019, View Source [SID1234535709]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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!

First Quarter 2019 Highlights:

Received 7 new orders for MRIdian systems totaling $42.8 million, compared to 3 orders totaling $21.2 million in the first quarter of 2018.
Total revenue of $20.3 million, primarily from 3 revenue units and 1 system upgrade, compared to $26.2 million, primarily from 4 revenue units and 1 system upgrade, in the first quarter of 2018.
Total backlog increased to $237.5 million as of March 31, 2019, from $212.3 million as of December 31, 2018.
Cash and cash equivalents were $145.8 million as of March 31, 2019.
"Our first quarter results are a solid start to the year and reflect progress on our commercial, innovation, and clinical pipelines," said Scott Drake, President and CEO. "We are well-positioned to execute in 2019 and are focused on becoming the standard of care in radiation oncology."

First Quarter 2019 Financial Results:

Total revenue for the three months was $20.3 million, compared to $26.2 million for the same period last year.

Total cost of revenue was $25.7 million, compared to $20.6 million for the same period last year. Total cost of revenue in the first quarter of 2019 was impacted by approximately $7.0 million of charges, primarily driven by higher than anticipated installation costs related to historical upgrade commitments. The $7.0 million includes $5.6 million of one-time charges and $1.4 million of expenses.

Total gross profit was $(5.4) million, compared to $5.6 million for the same period last year.

Total operating expenses were $25.0 million, compared to $16.9 million for the same period last year.

Net loss was $33.4 million, or $0.34 per share, compared to $7.5 million, or $0.11 per share, for the same period last year.

ViewRay had total cash and cash equivalents of $145.8 million at March 31, 2019.

Financial Guidance:

The Company is reiterating its financial guidance for the full year 2019. The Company anticipates 2019 total revenue to be in the range of $111 million to $124 million, and total cash usage to be in the range of $65-$75 million.

Conference Call and Webcast

ViewRay will hold a conference call to discuss results on Thursday, May 2, 2019 at 4:30 p.m. ET / 1:30 p.m. PT. The dial-in numbers are (844) 277-1426 for domestic callers and (336) 525-7129 for international callers. The conference ID number is 8616638. A live webcast of the conference call will be available on the investor relations page of ViewRay’s corporate website at www.viewray.com.

After the live webcast, a replay of the webcast will remain available online on the investor relations page of ViewRay’s corporate website, www.viewray.com, for 14 days following the call. In addition, a telephonic replay of the call will be available until May 9, 2019. The replay dial-in numbers are (855) 859-2056 for domestic callers and (404) 537-3406 for international callers. Please use the conference ID number 8616638.

On May 2, 2019 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported a corporate update and reported financial results for the first quarter ending March 31, 2019 (Press release, PTC Therapeutics, MAY 2, 2019, View Source [SID1234535538]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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!

"We believe we are making great strides towards our vision of having many more treatments for patients of rare disorders," said Stuart Peltz, Ph.D., CEO of PTC Therapeutics. "The approval of Translarna expands our footprint in Brazil. We are now preparing for the launches for multiple products, including Tegsedi for hATTR and Waylivra for FCS in Latin America. These differentiated therapies for rare disorders have the potential to be part of growing our revenue base to $1.5 billion by 2023."

Key First Quarter and Other Corporate Highlights:

Expanding commercial platform

Translarna received approval from the Brazilian health regulatory authority (ANVISA) which will allow for expanded market access.

Waylivra received a positive CHMP opinion from the European medical authorities in the first quarter 2019 and will be referred to the European Commission for consideration. After ratification by the European Commission, PTC plans to initiate early access programs in Latin America in 2019.

Tegsedi application submitted to the Brazilian health regulatory authority (ANVISA) was granted priority review, with expected approval by year end 2019. PTC has initiated early access programs for Tegsedi to support patient diagnosis and monitoring in Latin America.

PTC has submitted a sNDA with the FDA for Emflaza for patients 2 to 5 years old and received an approval action date of July 4, 2019.

Advancing gene therapy portfolio

As previously disclosed, PTC plans to submit a BLA with the FDA in late 2019 followed by an MAA in Europe for the AADC deficiency gene therapy program with anticipated commercial launch in the United States in 2020.

Friedreich ataxia program is advancing with an expected IND submission in late 2019 and subsequent entry in the clinic.

PTC is in the process of finalizing a long-term lease on an existing biologics manufacturing facility for the gene therapy pipeline.

Risdiplam SMA data expected at American Academy of Neurology (AAN)

As previously disclosed, based on regulatory interactions an NDA and MAA is planned for the second half of this year with the intention to support a broad label to treat SMA Types 1, 2, & 3 patients.

The SMA program is a collaboration between PTC, Roche and SMA Foundation.

Net sales over $1B would be subject to mid-teens royalties to PTC from Roche, resulting in potential royalties to PTC in excess of $200M per year. Potential remaining regulatory and sales-based milestones are approximately $400M.

Growing pipeline and R&D capabilities

As previously disclosed, PTC’s splicing platform has generated another development candidate, PTC258, for Familial dysautonomia, a rare genetic neurological disorder causing life-threatening medical complications from birth. PTC258 is advancing through IND-enabling studies to enter the clinic in late 2019. This program is in collaboration with MGH and NYU

Translarna’s dystrophin study in currently enrolling for potential U.S. regulatory re-submission for accelerated approval in 2020.

PTC’s oncology portfolio continues to advance with studies in AML with PTC299 and DIPG & LMS for PTC596. PTC expects these studies to fully enroll by the end of 2019.

PTC Re-iterates Full Year 2019 Guidance:

PTC anticipates full year DMD franchise net product revenues to be between $285 and $305 million.

PTC anticipates GAAP R&D and SG&A expense for the full year 2019 to be between $395 and $405 million.

PTC anticipates Non-GAAP R&D and SG&A expense for the full year 2019 to be between $360 and $370 million, excluding estimated non-cash, stock-based compensation expense of approximately $35 million.

First Quarter 2019 Financial Highlights:

Total revenues were $53.6 million for the first quarter of 2019, compared to $56.1 million for the first quarter of 2018.

Translarna net product revenues were $35.3 million for the first quarter of 2019, compared to $36.8 million for the first quarter of 2018. These results reflect lumpiness in ordering patterns from Latin America.

Emflaza net product revenues were $17.8 million for the first quarter of 2019, compared to $19.2 million for the first quarter of 2018. These results reflect first quarter dynamics including seasonality and a planned transition to a new specialty pharmacy distributor.

GAAP R&D expenses were $52.6 million for the first quarter of 2019, compared to $31.4 million for the first quarter of 2018. The increase in R&D expenses reflects costs associated with advancing the gene therapy platform and increased investment in research programs as well as advancement of the clinical pipeline.

Non-GAAP R&D expenses were $47.9 million for the first quarter of 2019, excluding $4.7 million in non-cash, stock-based compensation expense, compared to $27.6 million for the first quarter of 2018, excluding $3.7 million in non-cash, stock-based compensation expense.

GAAP SG&A expenses were $40.6 million for the first quarter of 2019, compared to $33.0 million for the first quarter of 2018. The increase in SG&A expenses were primarily due to continued investment to support our commercial activities.

Non-GAAP SG&A expenses were $36.0 million for the first quarter of 2019, excluding $4.6 million in non-cash, stock-based compensation expense, compared to $29.0 million for the first quarter of 2018, excluding $4.0 million in non-cash, stock-based compensation expense.

Change in the fair value of deferred and contingent consideration was $21.2 million for the first quarter of 2019. The change in fair value of deferred and contingent consideration is related to the fair valuation of potential future consideration to be paid to former equity holders of Agilis Biotherapeutics, Inc. (Agilis) in connection with PTC’s acquisition of Agilis, which closed in August 2018.

Net loss was $72.1 million for the first quarter of 2019, compared to net loss of $19.3 million for the first quarter of 2018.

Cash, cash equivalents, and marketable securities were $407.2 million at March 31, 2019, compared to $227.6 million at December 31, 2018.

Shares issued and outstanding as of March 30, 2019 were 58,418,790 .

Non-GAAP Financial Measures:
In this press release, the financial results and financial guidance of PTC are provided in accordance with accounting principles generally accepted in the United States (GAAP) and using certain non-GAAP financial measures. In particular, the non-GAAP financial measures exclude non-cash, stock-based compensation expense. These non-GAAP financial measures are provided as a complement to financial measures reported in GAAP because management uses these non-GAAP financial measures when assessing and identifying operational trends. In management’s opinion, these non-GAAP financial measures are useful to investors and other users of PTC’s financial statements by providing greater transparency into the historical and projected operating performance of PTC and the company’s future outlook. Non-GAAP financial measures are not an alternative for financial measures prepared in accordance with GAAP. Quantitative reconciliations of the non-GAAP financial measures to their respective closest equivalent GAAP financial measure are included in the table below.

Today’s Conference Call and Webcast Reminder:
Today’s conference call will take place at 4:30 pm ET and can be access by dialing (877) 303-9216 (domestic) or (973) 935-8152 (international) five minutes prior to the start of the call and providing the passcode 1559069. A live, listen-only webcast of the conference call can be accessed on the investor relations section of the PTC website at www.ptcbio.com. The accompanying slide presentation will be posted on the investor relations section of the PTC website. A webcast replay of the call will be available approximately two hours after completion of the call and will be archived on the company’s website for two weeks.