On January 7, 2019 Exscientia the world-leading Artificial Intelligence (AI)-driven drug discovery company reported it has raised US$26 million in a Series B financing round (Press release, Exscientia, JAN 7, 2019, View Source [SID1234539638]). This will be used to scale the company’s pipeline and advance selected programmes towards clinical development. The round included participation from new investors Celgene Corporation and specialist healthcare investor GT Healthcare Capital Partners, as well as existing investor Evotec AG.

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Alan Au, Managing Partner of GT Healthcare Capital Partners, commented: "We are very excited to back Exscientia and partner with a world-class team to further extend its business, including in Asia. We have a shared vision to revolutionize traditional drug development processes and believe Exscientia’s unique capabilities have the potential to deliver well beyond cost-effectiveness and speed, to first-in-class and best-in-class drugs that benefit patients in the near future."

Dr Rupert Vessey, President of Research and Early Development at Celgene, said: "Exscientia has demonstrated that AI in molecular design is here today. With the global pharmaceutical industry acknowledging the importance of incorporating AI-driven R&D approaches into their drug discovery processes, we see a huge growth opportunity ahead. We believe Exscientia is set to become a global leader in AI-driven drug discovery and are excited to participate in this investment round."

Professor Andrew Hopkins, CEO and founder of Exscientia, said: "This Series B marks a milestone in our development and enables us to drive the next phase of strong business growth. Over the past 12 months we have substantially expanded our operations and capabilities to become a full stack AI drug discovery company. Furthermore, our unique Centaur Chemist platform allows us to move rapidly from idea generation to new drug molecules ready for IND and clinical development. With this new funding Exscientia is positioned to become the dominant player in AI drug discovery, driving radical change in R&D productivity. We are excited Celgene and GT Healthcare have joined with existing investor Evotec on this exciting journey."

The company has made considerable progress during 2018 and anticipates its first programmes driven by AI to be IND-ready within the next 12 months. The speed and capital efficiency demonstrated by Exscientia is already revolutionising the economics of small molecule discovery and in the future, this improved throughput will deliver benefits to patients.

Dr Werner Lanthaler, Chief Executive Officer of Evotec, said: "We continue to be very impressed with the progress Exscientia has made over the past year. Through our partnership with Exscientia we have seen first-hand evidence that they can deliver the most productive drug discovery engine in the industry. This latest funding will allow Exscientia to apply its platform at scale, taking advantage of the efficiencies that its AI-driven systems provide."

Exscientia will use the proceeds of this financing round to grow its "full stack" AI drug discovery capability to dramatically expand its pipeline, with a target of establishing an expansive portfolio of projects, both in-house and with partners by the end of 2019.

Alex Snow, Executive Chairman of Exscientia, said: "Exscientia has validated its technology by delivering multiple pre-clinical drug candidates with unprecedented efficiency. Exscientia’s platform has the potential to bring a generation of new medicines to the market by offering a technologically and financially superior approach to drug discovery that will benefit of patients. I am personally very excited to be part of Exscientia’s next stage of development, and to have the backing of such prestigious investors."

Exscientia were advised by Rothschild & Co on this transaction.

On January 7, 2019 Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, reported its unaudited fourth quarter 2018 global net product revenues for ONPATTRO and provided additional updates on the product’s commercial launch (Press release, Alnylam, JAN 7, 2019, View Source [SID1234532505]). These updates will be discussed during a webcast presentation at the 37th Annual J.P. Morgan Healthcare Conference in San Francisco, California today, Monday, January 7, 2019, at 10:30 a.m. PT (1:30 p.m. ET). Specifically, the Company reported:

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ONPATTRO global net product revenues (unaudited) for the fourth quarter of 2018 were $11-12 million.

As of year-end 2018, over 200 patients in the U.S. and EU were receiving commercial ONPATTRO treatment, and approximately 550 total patients worldwide, including patients on commercial drug and patients in clinical studies and in the Company’s global Expanded Access Program (EAP), were being treated with patisiran.

In the U.S., a total of 250 Start Forms were submitted as of year-end 2018. Of these, approximately 50% were from patients previously treated on the ONPATTRO EAP.

The Start Forms came from a diverse range of prescribing physician specialties, including 44% neurologists, 35% cardiologists, and 21% from other specialties.

For Start Forms received, 62% of patients were covered by Medicare, 32% were covered by commercial insurers, and 6% were covered by other government insurers.

Significant progress has been made with value-based agreements (VBAs) in the U.S. and with market access efforts in the EU. Since launch, Alnylam has completed full VBAs with Harvard Pilgrim Healthcare, Humana, and another top five U.S. payer. Additional VBAs are under negotiation with over 15 other commercial payers with the potential to cover over 90% of commercial lives in the U.S.

"2018 was a landmark year for Alnylam, marked by the approval and launch in the U.S. and EU of ONPATTRO, heralding the arrival of RNAi therapeutics as a whole new class of medicines. Our unaudited fourth quarter 2018 global net product revenues of $11-12 million, with over 200 patients receiving treatment with commercial ONPATTRO in the U.S. and EU since launch, reflect strong patient and physician demand and excellent commercial execution by our U.S. and EU teams," said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. "As we enter 2019, we are excited to continue our global launch of ONPATTRO, bringing the benefits of this innovative therapy to hATTR patients with polyneuropathy around the world, while also working to potentially expand the label for ONPATTRO to include ATTR amyloidosis patients with cardiomyopathy. We also look forward to achieving meaningful milestones across our broad late-stage pipeline of investigational RNAi therapeutics."

In addition, the Company today reported that at December 31, 2018, it had cash, cash equivalents and marketable debt securities, and restricted investments, excluding equity securities, of approximately $1.1 billion (unaudited). The Company intends to provide 2019 financial guidance on non-GAAP R&D and SG&A expenses and year-end cash balance in connection with its full, audited fourth quarter and year-end 2018 financial results in February 2019.

About ONPATTRO (patisiran)

Patisiran, based on Nobel Prize-winning science, is an intravenously administered RNAi therapeutic targeting transthyretin (TTR) for the treatment of hereditary ATTR amyloidosis. It is designed to target and silence TTR messenger RNA, thereby blocking the production of TTR protein before it is made. Patisiran blocks the production of TTR in the liver, reducing its accumulation in the body’s tissues in order to halt or slow down the progression of the disease. In August 2018, patisiran received U.S. Food and Drug Administration (FDA) approval for the treatment of the polyneuropathy of hATTR amyloidosis in adults, as well as European Medicines Agency marketing authorization for the treatment of hATTR amyloidosis in adults with Stage 1 or Stage 2 polyneuropathy.

Important Safety Information

Infusion-Related Reactions

Infusion-related reactions (IRRs) have been observed in patients treated with ONPATTRO. In a controlled clinical study, 19 percent of ONPATTRO-treated patients experienced IRRs, compared to 9 percent of placebo-treated patients. The most common symptoms of IRRs with ONPATTRO were flushing, back pain, nausea, abdominal pain, dyspnea, and headache.

To reduce the risk of IRRs, patients should receive premedication with a corticosteroid, paracetamol, and antihistamines (H1 and H2 blockers) at least 60 minutes prior to ONPATTRO infusion. Monitor patients during the infusion for signs and symptoms of IRRs. If an IRR occurs,

consider slowing or interrupting the infusion and instituting medical management as clinically indicated. If the infusion is interrupted, consider resuming at a slower infusion rate only if symptoms have resolved. In the case of a serious or life-threatening IRR, the infusion should be discontinued and not resumed.

Reduced Serum Vitamin A Levels and Recommended Supplementation

ONPATTRO treatment leads to a decrease in serum vitamin A levels. Supplementation at the recommended daily allowance (RDA) of vitamin A is advised for patients taking ONPATTRO. Higher doses than the RDA should not be given to try to achieve normal serum vitamin A levels during treatment with ONPATTRO, as serum levels do not reflect the total vitamin A in the body.

Patients should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency (e.g. night blindness).

Adverse Reactions

The most common adverse reactions that occurred in patients treated with ONPATTRO were respiratory-tract infection (29 percent) and infusion-related reactions (19 percent).

About LNP Technology

Alnylam has licenses to Arbutus Biopharma LNP intellectual property for use in RNAi therapeutic products using LNP technology.

About RNAi

RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as "a major scientific breakthrough that happens once every decade or so," and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines, known as RNAi therapeutics, is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam’s RNAi therapeutic platform, function upstream of today’s medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing proteins, thus preventing them from being made. This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases.

On January 7, 2019 Quest Diagnostics Incorporated (NYSE: DGX), the world’s leading provider of diagnostic information services, reported that it will report fourth quarter and full year 2018 results on Thursday, February 14, 2019, before the market opens (Press release, Quest Diagnostics, JAN 7, 2019, View Source [SID1234532521]). It will hold its quarterly conference call to discuss the results beginning at 8:30 a.m. Eastern Time on that day.

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The conference call can be accessed by dialing 888-455-0391 within the U.S. and Canada, or 773-756-0467 internationally, using the passcode: "Investor." The earnings release and live webcast will be posted on www.QuestDiagnostics.com/investor. The company suggests participants dial in approximately 10 minutes before the call.

A replay of the call may be accessed online at www.QuestDiagnostics.com/investor or by phone at 866-424-7881 for domestic callers or 203-369-0869 for international callers, no passcode is required. Telephone replays will be available from approximately 10:30 a.m. Eastern Time on February 14, 2019 until midnight Eastern Time on February 28, 2019.

Anyone listening to the call is encouraged to read the company’s periodic reports on file with the Securities and Exchange Commission, including the discussion of risk factors and historical results of operations and financial condition in those reports.

On January 7, 2019 Stemline Therapeutics, Inc. (NASDAQ:STML), a biopharmaceutical company focused on the development and commercialization of novel oncology therapeutics, reported that it has submitted the marketing authorization application (MAA) for ELZONRIS (tagraxofusp) to the European Medicines Agency (EMA) (Press release, Stemline Therapeutics, JAN 7, 2019, View Source [SID1234532537]). The MAA seeks approval for treating patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN). In November 2018, the EMA granted the ELZONRIS MAA accelerated assessment.

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On December 21, 2018, ELZONRIS was approved by the U.S. Food and Drug Administration (FDA) for the treatment of BPDCN in adult and pediatric patients, two years and older, in both treatment-naïve and previously-treated populations. ELZONRIS is the first treatment approved for BPDCN and the first approved CD123-targeted therapy.

"The submission of the ELZONRIS MAA is another major step forward for providing this important targeted treatment to patients, globally," said Ivan Bergstein, M.D., CEO of Stemline Therapeutics. "We look forward to working closely with the EMA to ensure this treatment is available to patients as quickly as possible. In parallel, our commercial team is continuing its ongoing effort to raise awareness of both CD123 testing and BPDCN worldwide. Potential European approval offers us an opportunity to significantly increase the number of patients who may benefit from ELZONRIS."

About ELZONRIS
ELZONRIS (tagraxofusp), a CD123-directed cytotoxin, was approved by the Food and Drug Administration (FDA) on December 21, 2018 for the treatment of adult and pediatric patients, two years and older, with blastic plasmacytoid dendritic cell neoplasm (BPDCN). In November 2018, the European Medicines Agency (EMA) granted ELZONRIS accelerated assessment to the marketing authorization application (MAA), which was submitted to the EMA in January 2019. ELZONRIS is also being evaluated in additional clinical trials in other indications including chronic myelomonocytic leukemia (CMML), myelofibrosis (MF) and other CD123 positive diseases.

About BPDCN
Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is an aggressive hematologic malignancy with historically poor outcomes and an area of unmet medical need. The BPDCN cell of origin is the plasmacytoid dendritic cell (pDC) precursor. BPDCN typically presents in the bone marrow and/or skin and may also involve lymph nodes and viscera. The diagnosis of BPDCN is based on the immunophenotypic diagnostic triad of CD123, CD4, and CD56. For more information, please visit the BPDCN disease awareness website at www.bpdcninfo.com.

On January 7, 2019 Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, reported its key 2019 initiatives in conjunction with its presentation at the 37th Annual J.P. Morgan Healthcare Conference in San Francisco (Press release, Agios Pharmaceuticals, JAN 7, 2019, View Source [SID1234532553]). The company will webcast its presentation today at 9:30 a.m. PT (12:30 p.m. ET) at investor.agios.com.

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"During 2018, just 10 years after the founding of Agios, we achieved approval of our second internally discovered oncology medicine, launched a robust registrational program in PK deficiency and successfully opened the company’s seventh IND," said David Schenkein, M.D., chief executive officer at Agios. "Our validated research platform and proven drug development strategy are poised to help drive future growth across our oncology and rare genetic disease portfolios. Our priorities for 2019 include expanding the reach of our IDH inhibitors into the frontline AML and solid tumor settings, completing enrollment in two pivotal studies of mitapivat, exploring the utility of PKR activators in other hemolytic anemias, and furthering clinical development for AG-270 in MTAP deleted tumors and AG-636 in lymphoma."

The company plans to achieve the following milestones in 2019.

Cancer:

Potential FDA approval of the supplemental new drug application (sNDA) for single agent TIBSOVO (ivosidenib) for the treatment of patients with newly diagnosed AML with an IDH1 mutation who are not eligible for standard therapy.
Submit a sNDA to the FDA for TIBSOVO for second line or later IDH1m cholangiocarcinoma by year-end.
Initiate a registration-enabling Phase 3 study of vorasidenib (AG-881) in low-grade glioma with an IDH1 mutation by year-end.
Determine recommended dose of AG-270, a first-in-class methionine adenosyltransferase 2a (MAT2A) inhibitor, in methylthioadenosine phosphorylase (MTAP)-deleted tumors and initiate expansion arms, including a single-agent arm in a variety of MTAP-deleted cancers and a combination arm in a solid tumor in the first half of 2019.
Initiate a Phase 1 dose-escalation trial of AG-636, an inhibitor of the metabolic enzyme dihydroorotate dehydrogenase (DHODH), in lymphoma in the first half of 2019.
Rare Genetic Diseases:

Complete enrollment in two global pivotal trials for mitapivat in adults with pyruvate kinase (PK) deficiency by year-end 2019:

ACTIVATE-T: A single-arm trial of approximately 20 regularly transfused patients
ACTIVATE: A 1:1 randomized, placebo-controlled trial of 80 patients who do not receive regular transfusions
Achieve proof-of-concept for mitapivat in thalassemia in the second half of 2019.
The company highlighted key data presentations expected in 2019.

Updated data from the ongoing Phase 1 combination trial of TIBSOVO with azacitidine in patients with newly diagnosed AML with an IDH1 mutation in the first half of 2019.
Data from the perioperative ‘window’ trial with TIBSOVO and vorasidenib in IDHm low-grade glioma in the first half of 2019.
Topline data from the Phase 3 ClarIDHy study of TIBSOVO in IDH1m advanced cholangiocarcinoma to be reported in the first half and full data to be presented in the second half of 2019.
Data from the dose-escalation portion of the ongoing Phase 1 study of AG-270 in patients with MTAP-deleted tumors in the second half of 2019.
The company also provided an update on the following 2018 milestones achieved in December.

Submitted an sNDA to the FDA for TIBSOVO for the treatment of patients with newly diagnosed AML with an IDH1 mutation who are not eligible for standard therapy.
Submitted a Marketing Authorization Application to the European Medicines Agency for TIBSOVO for the treatment of adult patients with R/R AML with an IDH1 mutation.
Initiated a Phase 2 proof-of-concept trial of mitapivat in thalassemia.
2018 Year-End Cash and Guidance
Agios ended 2018 with approximately $805 million of cash, cash equivalents and marketable securities. The company expects that its cash, cash equivalents and marketable securities as of December 31, 2018, together with anticipated product and royalty revenue, anticipated interest income, and anticipated expense reimbursements under our collaboration agreements, but excluding any additional program-specific milestone payments, will enable the company to fund its anticipated operating expenses and capital expenditure requirements through at least the end of 2020.

Presentation at 37th Annual J.P. Morgan Healthcare Conference
Agios will webcast its corporate presentation from the 37th Annual J.P. Morgan Healthcare Conference in San Francisco on Monday, January 7, 2019 at 9:30 a.m. PT (12:30 p.m. ET). A live webcast of the presentation can be accessed under "Events & Presentations" in the Investors section of the company’s website at agios.com. A replay of the webcast will be archived on the Agios website for at least two weeks following the presentation.