On May 13, 2020 Syros Pharmaceuticals (NASDAQ:SYRS), a leader in the development of medicines that control the expression of genes, reported that it will present new preclinical data on the anti-tumor activity of SY-5609, its highly selective and potent oral cyclin-dependent kinase 7 (CDK7) inhibitor, in models of colorectal cancer (Press release, Syros Pharmaceuticals, MAY 13, 2020, View Source [SID1234557922]). These data will be presented at the 2020 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Virtual Scientific Program (ASCO20) taking place May 29-31 . Syros will also present on the design of its ongoing Phase 1 trial of SY-5609 at ASCO (Free ASCO Whitepaper)20.

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The abstracts for these presentation are now available online on the ASCO (Free ASCO Whitepaper)20 website, at View Source

Details of the poster presentations are as follows:

Presentation Title: Activity of SY-5609, an oral, noncovalent, potent, and selective CDK7 inhibitor, in preclinical models of colorectal cancer
Session Title: Developmental Therapeutics—Molecularly Targeted Agents and Tumor Biology
Presenter: Liv Johannessen, Ph.D., Syros
Abstract Number: 3585
Poster Number: 315

Presentation Title: First-in-human phase I study of SY-5609, an oral, potent, and selective noncovalent CDK7 inhibitor, in adult patients with select advanced solid tumors
Session Title: Developmental Therapeutics—Molecularly Targeted Agents and Tumor Biology
Presenter: Kyriakos P. Papadopoulos, M.D., South Texas Accelerated Research Therapeutics (START)
Abstract Number: TPS3662
Poster Number: 392

Presentations will be available for on-demand viewing on the ASCO (Free ASCO Whitepaper)20 website beginning May 29, 2020, at 8 a.m. EDT.

On May 13, 2020 ImmunoGen, Inc. (Nasdaq: IMGN), a leader in the expanding field of antibody-drug conjugates (ADCs) for the treatment of cancer, reported initial data from the FORWARD II study evaluating mirvetuximab soravtansine in combination with Avastin (bevacizumab) in patients with medium and high folate receptor alpha (FRα)-expressing recurrent ovarian cancer for whom a non-platinum based combination regimen is appropriate (Press release, ImmunoGen, MAY 13, 2020, View Source [SID1234557939]). These findings will be highlighted in an oral presentation at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2020 Virtual Scientific Program on May 29, 2020. Three "trial in progress" posters will also be presented during the meeting.

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"With the benefit of the clinical profile demonstrated by mirvetuximab monotherapy, we have pursued a development strategy to establish mirvetuximab as the agent of choice in combination regimens to treat expanded populations of patients with recurrent ovarian cancer. To this end, we are encouraged by the compelling anti-tumor activity and favorable tolerability observed with the combination of mirvetuximab plus bevacizumab in patients for whom a non-platinum based regimen is appropriate," said Anna Berkenblit, MD, Senior Vice President and Chief Medical Officer of ImmunoGen. "These findings show greater depth and duration of tumor reduction in women whose tumors express high levels of FRα, regardless of platinum status, reinforcing the potential of this doublet in these patients. As these data mature, we look forward to further evaluating this combination in the recurrent ovarian cancer setting."

INITIAL DATA FROM FORWARD II DOUBLET COHORT WITH BEVACIZUMAB
This cohort enrolled 60 patients with FRα-positive recurrent ovarian cancer for whom a non-platinum based combination regimen is appropriate, with a median age of 60 years and a median number of 2 prior lines of therapy (range 1-4). The combination of mirvetuximab soravtansine with bevacizumab in this cohort demonstrates encouraging anti-tumor activity with a favorable tolerability profile, particularly among the subset of patients with high levels of FRα expression.

Key findings include:

In the overall patient population, objective responses were seen in 26 patients and the confirmed overall response rate (ORR) was 43% (95% CI, 31, 57).
In patients with high FRα expression (n=33), the confirmed ORR was 61% (95% CI, 42, 77), with an ORR of at least 50% in each of the platinum-resistant and platinum-sensitive subgroups.
With many patients remaining on study, the duration of response and progression free survival data are immature.
The adverse events (AEs) observed with the doublet were as expected based on the side effect profiles of each agent. The most common treatment-related low grade AEs were diarrhea, blurred vision, nausea, and fatigue; grade 3+ AEs were infrequent, with the most common being hypertension and neutropenia.
"With the increasing need for non-platinum regimens in recurrent ovarian cancer, we are excited to further advance mirvetuximab in combination with bevacizumab, building on the prior data for this combination in women with platinum resistant disease," stated Lucy Gilbert, MD, Professor, and Director of the Gynecologic Oncology Division at McGill University Health Center in Montreal, Canada. "These initial data demonstrate meaningful clinical benefit in women with recurrent disease, regardless of platinum status, and I look forward to reporting longer-term follow up and further evaluating the doublet in this expanded patient population."

ORAL PRESENTATION SESSION

Title: "Mirvetuximab Soravtansine, a Folate Receptor Alpha-Targeting Antibody-Drug Conjugate, in Combination with Bevacizumab in Patients with Platinum-Agnostic Ovarian Cancer"
Day/Time: Friday, May 29 at 8:00 AM ET
Lead Author: Lucy Gilbert, MD, McGill University Health Center, Montreal, Canada
Abstract: 6004
TRIAL IN PROGRESS POSTERS
The following posters will be available on Friday, May 29 at 8:00 AM ET in the ASCO (Free ASCO Whitepaper) Meeting Library:

Title: "MIRASOL (GOG 3045/ENGOT OV-55): A Randomized, Open-label, Phase 3 study of Mirvetuximab Soravtansine versus Investigator’s Choice of Chemotherapy in Advanced High-grade Epithelial Ovarian, Primary Peritoneal, or Fallopian Tube Cancers with High Folate Receptor Alpha (FRα) Expression"
Lead Author: Kathleen Moore, MD, University of Oklahoma Health Sciences Center
Abstract: TPS6103 (Poster 274)
Title: "A Phase 1/2 Study of IMGN632, a Novel CD123-Targeting Antibody-Drug Conjugate, in Patients with Relapsed/Refractory Acute Myeloid Leukemia, Blastic Plasmacytoid Dendritic Cell Neoplasm, and Other CD123-Positive Hematologic Malignancies"
Lead Author: Naval Daver, MD, MD Anderson Cancer Center
Abstract: TPS7563 (Poster 336)
Title: "A Phase 1b/2 Study of the CD123-Targeting Antibody-Drug Conjugate IMGN632 as Monotherapy or in Combination with Venetoclax and/or Azacitidine for Patients with CD123-Positive Acute Myeloid Leukemia"
Lead Author: Naval Daver, MD, MD Anderson Cancer Center
Abstract: TPS7564 (Poster 337)
Additional information can be found at www.asco.org.

ABOUT FORWARD II
FORWARD II is a Phase 1b/2 study of mirvetuximab in combination with Avastin (bevacizumab), carboplatin, or Keytruda (pembrolizumab) in patients with FRα-positive recurrent epithelial ovarian, primary peritoneal, or fallopian tube cancers, as well as a triplet combination of mirvetuximab plus carboplatin and bevacizumab in patients with FRα-positive platinum-sensitive ovarian cancer.

ABOUT MIRVETUXIMAB SORAVTANSINE
Mirvetuximab soravtansine (IMGN853) is an antibody-drug conjugate (ADC) comprising a folate receptor alpha (FRα)-binding antibody, cleavable linker, and the maytansinoid DM4, a potent tubulin-targeting agent to kill the targeted cancer cells.

On May 13, 2020 Natera, Inc. (NASDAQ: NTRA), a pioneer and global leader in cell-free DNA testing, reported that it will webcast a live presentation at the UBS Virtual Global Healthcare Conference on Monday, May 18, 2020 at 12:50 p.m. PT (3:50 p.m. ET) (Press release, Natera, MAY 13, 2020, View Source [SID1234557955]).

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Mike Brophy, Chief Financial Officer, will provide an overview of the company and discuss recent business activities.

Access to the live webcast and subsequent archived presentation will be available at investor.natera.com.

On May 13, 2020 Oasmia Pharmaceutical AB, an innovation-focused specialty pharmaceutical company, reported the outcome of a strategic review assessing all aspects of the business to maximise the company’s resources, to achieve the full potential of its XR17 platform technology and to optimise Oasmia’s path towards long-term, profitable growth (Press release, Oasmia, MAY 13, 2020, View Source [SID1234557891]).

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The review was undertaken by Oasmia following the appointment of Francois Martelet as Chief Executive Officer in February 2020. In March, Oasmia announced a global strategic partnership with Elevar Therapeutics, the U.S. subsidiary of HLB Co. Ltd, to commercialise Oasmia’s anticancer product Apealea, which is based on its XR17 technology. Under the terms of the agreement, Oasmia has received an upfront payment of USD 20 million and is eligible to receive milestone payments of up to USD 678 million and double-digit royalties on future sales.

As a result of the review, Oasmia has identified a number of areas of strategic focus allowing the company to achieve its long-term goals, including:

Explore additional opportunities to apply the company’s proprietary XR17 solubility-enhancing technology platform in oncology and other therapeutic areas, including out-licensing of non-core applications
Continue to drive the development of Oasmia’s existing pipeline of XR17-based products, including Docecal (docetaxel) in breast and prostate cancers and its combination cancer therapy XR19
Leverage the company’s GMP manufacturing facilities for R&D and clinical trial production
Expand Oasmia’s pipeline through potential acquisitions or in-licensing deals with a focus on late-stage assets that will move the company towards positive cash flow.
As a consequence of this review, Oasmia will undertake a comprehensive cost control program designed to maximise resources and enable it to invest in areas which can deliver the greatest return. Key aspects of the cost control program include:

Annualised cost savings of more than SEK 100 million
A ~50% reduction in the cash burn rate to below SEK 10 million a month
Greater focus on R&D and clinical trial GMP manufacturing as opposed to commercial manufacturing
Francois Martelet, Chief Executive Officer of Oasmia, said:

"Since joining Oasmia in March, I have been impressed by the quality and potential of its technology and pipeline. With Apealea approved in Europe and becoming available to patients, Oasmia has demonstrated its ability to bring promising new products to market that meet unmet need and enhance drug safety. The transformative global agreement with Elevar has further underlined Oasmia’s clinical, regulatory and commercial strengths. We are now ideally placed to move into the next phase of growth, establishing Oasmia as a leading Sweden-based specialty pharmaceutical company, maximising the potential of our proven XR17 technology platform and leveraging our skills and expertise through the acquisition or in-licensing of promising assets. Management is now fully focused on delivering these objectives, which will provide benefits to patients and build value for shareholders."

Jörgen Olsson, resigning Chairman of the Board of Oasmia, comments:

"In the Board, we are very impressed and pleased by the sharp analysis, speed and decision-making qualities that our new CEO shows in this short time. We very much look forward to the new, commercial future for Oasmia."

On May 13, 2020 BridgeBio Pharma, Inc. (Nasdaq: BBIO), reported its financial results for the first quarter ended March 31, 2020 and provided an update on the company’s operations (Press release, BridgeBio, MAY 13, 2020, View Source [SID1234557907]).

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The company is working with its committed partners to minimize disruption from the COVID-19 pandemic on its clinical trials and other drug development programs. BridgeBio salutes the brave physicians, nurses, first responders and medical staff who are working across the globe to care for patients with COVID-19.

BridgeBio’s fully enrolled trials and preclinical programs are moving ahead with minimal delays, and the company is on track to file its second New Drug Application (NDA) later this year, along with the anticipated filing of multiple investigational new drug applications (INDs). Despite the challenges presented by COVID-19, BridgeBio’s clinical trials of infigratinib in tumors with FGFR genetic alterations are proceeding as planned.

Certain clinical trials that were in the process of enrolling have slowed as a result of the COVID-19 outbreak. BridgeBio is working with hospitals and investigators to deliver investigational medicines to patients and to develop solutions that will allow the company to continue to measure key endpoints for these trials. BridgeBio is also prepared to continue enrolling patients as the healthcare system regains the ability to accommodate this activity.

For trials that have not yet begun enrolling, BridgeBio is prepared to begin them as soon as trial sites can accommodate enrollment. Given the severe and often life-threatening nature of many of the disease areas on which BridgeBio is focused, we anticipate many of our planned trials will be prioritized.

"Given COVID-19’s impact, we estimate that certain milestones related to clinical trials that were anticipated in 2020 are now more likely to come in 2021. Other programs are moving forward without delay, thanks to the committed efforts of our partners," said BridgeBio founder and CEO Neil Kumar, Ph.D. "We believe we are in a position financially and operationally to move our critical work forward the moment the public health situation allows for that. Many of the patients we work with are battling devastating diseases that will not wait for this pandemic to pass."

Just prior to substantial market dislocation driven by COVID-19, BridgeBio strengthened its balance sheet by raising $550 million in gross proceeds through the issuance of 2.50% Convertible Senior Notes due in 2027 (2027 Notes). This important financing added to approximately $700 million in gross proceeds raised from our initial public offering in June 2019 and Series D announced in January 2019, and puts BridgeBio in a strong financial position to execute on the 20+ drug development and discovery programs in its pipeline. The company expects current cash, cash equivalents and marketable securities to carry it through critical and high-value milestones stretching into 2022.

"Our recent financings have focused on solving for two objectives at once: securing the capital required to independently advance our pipeline, while minimizing shareholder dilution," said BridgeBio CFO Brian Stephenson, Ph.D., CFA. "One of the original goals in the founding of BridgeBio was to create a vehicle that could attract a new set of investors to fund critical biomedical research and drug development by taking a sufficiently large number of shots on goal, within an advantaged research and development space, to build conviction that multiple approved products are not just possible but statistically probable over time. We appreciate the confidence investors have demonstrated in the critical work we are doing for patients."

Steps BridgeBio has taken in response to COVID-19

Delivering investigational medicines to trial participants directly now that many can no longer visit the hospital

Implementing out-of-hospital solutions for clinical trials – including telehealth appointments and remote clinical monitoring

Engaging in conversations with the U.S. Food and Drug Administration (FDA) across multiple programs to understand how to preserve the fidelity of key endpoints

Working closely with contract manufacturing partners to anticipate potential downstream impacts to BridgeBio’s immediate supply chain

Working on key aspects of site activation and support so that BridgeBio can continue enrolling patients where and when possible

Transferring many of our non-clinical laboratory activities to contract research organizations that continue to work on them, when BridgeBio has not been able to sustain them

First quarter 2020 and recent pipeline progress:

Mendelian

BBP-589 – COL7A protein replacement therapy for recessive dystrophic epidermolysis bullosa (RDEB): Due to a slowdown in enrollment related to COVID-19, BridgeBio now plans to share topline data from the ongoing Phase 1/2 study (NCT03752905) in 2021.

BBP-265 (AG10) – TTR stabilizer for ATTR: Due to a slowdown in site activation and participant enrollment related to COVID-19, BridgeBio currently expects enrollment of ATTRibute-CM to be completed in the first half of 2021 (ATTRibute-CM) and plans to initiate its Phase 3 trial of AG10 in ATTR-PN (ATTRibute-PN) in the second half of 2020.

Low-dose infigratinib for achondroplasia: The Phase 2 clinical program (PROPEL2) is on track to dose the first children in 2020 (NCT04265651). Enrollment has continued in areas where it is safe for children and their families to participate and BridgeBio continues to activate sites remotely.

Fosdenopterin – cPMP replacement therapy for MoCD type A: Remains on track to complete the rolling NDA submission to FDA in 2020.

Topical patidegib gel for Gorlin syndrome and high-frequency basal cell carcinoma1: Phase 3 study is fully enrolled as of the fourth quarter of 2019 and remains on track for last patient last visit in the fourth quarter of 2020 (NCT03703310).

PellePharm, which is focused on developing patidegib topical gel, 2% entered into a strategic collaboration with LEO Pharma in November 2018, which includes an option for LEO Pharma to acquire PellePharm.

Multiple new clinical studies are ready to initiate pending stabilization of COVID-19:

Encalaret (CaSR antagonist) Phase 2 study in autosomal dominant hypoparathyroidism.

Ribitol (BBP-418) Phase 1 study in healthy volunteers.

Zuretinol (syntetic retinoid) Phase 2/3 study in inherited retinal diseases due to RPE65 or LRAT gene mutations.

Targeted oncology

Infigratinib – FGFR1-3 inhibitor for FGFR+ cancer: Preparing NDA submission in 2020 for second-line FGFR2+ cholangiocarcinoma. Our front-line cholangiocarcinoma (NCT03773302), adjuvant urothelial carcinoma (NCT04197986), and tumor agnostic (NCT04233567) studies continue to enroll as BridgeBio activates sites remotely.

Gene therapy

BBP-812 – Gene therapy candidate for Canavan disease: IND-enabling studies for AAV gene therapy proceeding. On track to submit IND to regulatory authorities in 2020.

BBP-631 – Gene therapy candidate for congenital adrenal hyperplasia: IND-enabling studies for AAV gene therapy proceeding. On track to submit IND to regulatory authorities in 2020.

Additional updates

Anticipate disclosing new product candidates and filing multiple new INDs in 2020.

First quarter 2020 financial results:

Cash, cash equivalents and marketable securities

Cash, cash equivalents and marketable securities, excluding restricted cash, totaled $928.4 million as of March 31, 2020 compared to $577.1 million at December 31, 2019. The net change in cash balance of $351.3 million reflects $537.6 million in net proceeds received from the issuance of our 2027 Notes, $24.1 million in net proceeds received from Eidos’ at-the-market issuance of shares, offset by payment of $75.0 million to repurchase BridgeBio shares, $49.3 million payment related to capped call option and the remaining payment of $86.1 million primarily related to operating expenses.