On January 30, 2020 Karolinska Development (Nasdaq Stockholm: KDEV) reported that its portfolio company Aprea Therapeutics has been granted Breakthrough Therapy Designation for APR-246 in combination with azacitidine for the treatment of myelodysplastic syndrome (MDS) with a TP53 mutation (Press release, Aprea, JAN 30, 2020, https://www.karolinskadevelopment.com/en/press-releases?page=/en/pressreleases/karolinska-development%2527s-portfolio-company-aprea-therapeutics-receives-fda-breakthrough-therapy-designation-1769167 [SID1234553684]). A Breakthrough Therapy Designation facilitates expedited development and regulatory review of a drug candidate.

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APR-246 is a small molecular drug candidate that binds, refolds and stabilizes mutant p53, occurring in approximately 50% of all human tumors. A pivotal Phase 3 clinical trial of APR-246 and azacitidine for frontline treatment of TP53 mutant MDS is ongoing. APR-246 has previously received Orphan Drug and Fast Track designations from the FDA for MDS, and Orphan Drug designation from the EMA for MDS, acute myeloid leukemia (AML) and ovarian cancer.

"This is yet another success for Aprea Therapeutics, and we look forward to following the continued development of its potentially ground-breaking cancer therapy", says Viktor Drvota, CEO, Karolinska Development AB, in response to the announcement.

MDS represents a spectrum of hematopoietic stem cell malignancies in which bone marrow fails to produce sufficient numbers of healthy blood cells. Approximately 30-40% of MDS patients progress to acute myeloid leukemia (AML) and mutation of the p53 tumor suppressor protein is thought to directly contribute to disease progression and a poor overall prognosis.

The FDA’s Breakthrough Therapy Designation is intended to expedite the development and review of a drug candidate that is planned to treat a serious or life-threatening disease or condition when preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapies on one or more clinically significant endpoints.

On January 30, 2020 Syndax Pharmaceuticals, Inc. ("Syndax," the "Company" or "we") (Nasdaq: SNDX), a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies, reported that Science magazine has published a preclinical report supporting the potential role of MLL1-Menin inhibition in the management of nucleophosmin (NPM1) mutant acute myeloid leukemia (AML) (Press release, Syndax, JAN 30, 2020, View Source [SID1234553703]). The article, "Therapeutic targeting of preleukemia cells in a mouse model of NPM1 mutant acute myeloid leukemia," will be published in the journal’s January 31, 2020 issue and is currently available online.

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This study examined the activity of VTP-50469, an orally-available inhibitor of MLL1-Menin interaction and close analog of the Company’s lead Menin inhibitor, SNDX-5613, for the treatment of established NPM1 AML and the possible prevention of the disease in high-risk populations. Using preclinical models of NPM1 AML, the authors established that the presence of an NPM1 mutation is a clear indicator of pre-leukemic activity and represents a critical step in the development of AML. VTP-50469 was shown to eradicate NPM1 mutant cells at various stages of disease development, suggesting that Menin-MLL inhibition could potentially serve either as a targeted preventive therapy or as a treatment of established disease.

"These unprecedented findings highlight the potential for single agent Menin-MLL inhibition to rapidly eradicate fully developed NPM1 mutant leukemia, even in the case of aggressive relapsed AML," said Scott A. Armstrong, M.D., Ph.D., President, Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, and Chairman, Department of Pediatric Oncology, Dana-Farber Cancer Institute, and senior author of the study. "In addition, these results provide support for a Menin-MLL inhibitor to serve as a novel strategy to prevent AML development in high-risk patient populations, as NPM1 mutations are acquired in pre-leukemic clones."

"NPM1 mutant AML represents the most common type of cytogenetically normal AML," said Briggs W. Morrison, M.D., Chief Executive Officer of Syndax. "On the heels of our recent Cancer Cell publication, these findings add to the growing body of compelling preclinical data supporting the potential for SNDX-5613 to serve as an effective intervention for both NPM1 mutant AML and MLL-r acute leukemias. We are committed to providing patients with more targeted therapeutic options and are hopeful that these findings will translate into the clinic in our ongoing Phase 1/2 AUGMENT-101 trial."

About SNDX-5613

SNDX-5613 is a potent, selective, small molecule inhibitor of the Menin-MLL binding interaction that is being developed for the treatment of MLL-rearranged (MLL-r) acute leukemias, including acute lymphoblastic leukemia (ALL) and acute myeloid leukemia (AML); VTP-50469 is a close analog of SNDX-5613. MLL rearrangements occur in approximately 80% of acute leukemia cases in infants and up to 10% of all leukemias. In preclinical models of MLL-r acute leukemias, SNDX-5613 demonstrated robust, dose-dependent inhibition of tumor growth, resulting in a marked survival benefit. Menin-MLL interaction inhibitors have also demonstrated robust treatment benefit in multiple preclinical models of NPM1 mutant AML, which represents the most frequent genetic abnormality in adult AML. SNDX-5613 is currently being evaluated in the Company’s AUGMENT-101 Phase 1/2 open-label clinical trial for the treatment of relapsed/refractory (R/R) acute leukemias.

About AUGMENT-101

AUGMENT-101 is a Phase 1/2 open-label trial designed to evaluate the efficacy, safety, tolerability and pharmacokinetics of orally administered SNDX-5613. The Phase 1 dose escalation portion of AUGMENT-101 will enroll adults with R/R acute leukemias and establish a recommended Phase 2 dose. The Phase 2 portion will evaluate efficacy, as defined by Complete Response rate (per International Working Group response criteria), across three expansion cohorts: MLL-r ALL, MLL-r AML and NPM1 mutant AML. The Company expects to report initial clinical data from the trial in 2020. Additional information about the AUGMENT-101 trial is available via Clinicaltrials.gov (NCT 04065399).

About NPM1 Mutant Acute Myeloid Leukemia

NPM1 mutant AML, which is distinguished by point mutations in the NPM1 gene that drive the leukemic phenotype, is the most common type of cytogenetically normal adult AML and represents approximately 30% of all adult AML cases. This subtype of AML has a 5-year overall survival rate of approximately 50%. Similar to MLL-r leukemias, NPM1 mutant AML is highly dependent on the expression of specific developmental genes, shown to be negatively impacted by inhibitors of the Menin-MLL interaction. NPM1 mutant AML is routinely diagnosed through currently available screening techniques. There are currently no approved therapies indicated for NPM1 mutant AML.

On January 30, 2020 Edwards Lifesciences Corporation (NYSE: EW), the global leader in patient-focused innovations for structural heart disease and critical care monitoring, reported financial results for the quarter ended December 31, 2019 (Press release, Edwards Lifesciences, JAN 30, 2020, View Source [SID1234553719]).

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Fourth Quarter Results and Outlook

Q4 sales grew 20% to $1.2 billion; underlying1 sales grew 19%
Q4 TAVR sales grew 29%; underlying sales grew 30%
Q4 EPS was $1.32; adjusted1 EPS grew 25% to $1.46
Full year 2019 sales and earnings significantly exceeded original guidance
2020 guidance ranges increased: sales $4.6 billion to $5.0 billion; EPS $6.15 to $6.40
SAPIEN 3 transcatheter heart valve received low risk indication expansion in Europe
EVOQUE tricuspid early feasibility study receives FDA approval
"We are pleased to report robust fourth quarter performance that delivered 19 percent sales growth on an underlying basis, driven by our portfolio of innovative technologies. For the full year 2019, we reported 15 percent revenue growth on an underlying basis, with double-digit growth in each region," said Michael A. Mussallem, chairman and CEO. "Most importantly, I’m proud to report we’re poised to help even more patients benefit from Edwards’ life-saving technologies. We invested aggressively in transformative therapies in 2019 and this will continue in 2020 as our commitment has never been greater."

2019 Full Year Results

Sales for the year ended December 31, 2019 were $4.3 billion, up 17 percent over the prior year, or 15 percent on an underlying basis. Diluted earnings per share for 2019 were $4.93, while adjusted earnings per share grew 19 percent, to $5.57.

Transcatheter Aortic Valve Replacement (TAVR)

The company reported fourth quarter global TAVR sales of $763 million, a year-over-year increase of approximately 30 percent on both a reported and underlying basis with impressive strength in the U.S. Global average selling prices remained stable.

In the fourth quarter, the company estimates U.S. TAVR procedures grew approximately 40 percent on a year-over-year basis and Edwards’ growth was comparable. Outside the U.S., the company estimates that total TAVR procedures grew in the high-teens on a year-over-year basis and Edwards’ growth was comparable. As previously reported, Edwards became the first company to receive CE Mark for TAVR in Europe for the treatment of patients diagnosed with severe aortic stenosis who are at low risk for open-heart surgery.

Transcatheter Mitral and Tricuspid Therapies (TMTT)

TMTT is on track to achieve the milestones discussed at the company’s recent Investor Conference, including executing four pivotal studies. In addition, the company announced that the EVOQUE tricuspid replacement valve system has recently received U.S. Food and Drug Administration (FDA) approval for an Early Feasibility Study and a "breakthrough device" designation, a program intended to help patients receive more timely access to designated medical technologies.

Full year 2019 TMTT sales of $28 million came in below the original guidance of approximately $40 million as the company continued to execute a disciplined introduction and premium pricing strategy of the PASCAL system, which moderated European site activation. Fourth quarter sales of $7 million were negatively impacted by the voluntary PASCAL system field corrective action completed in the quarter. PASCAL clinical outcomes continue to be favorable and physician feedback remains positive. As the PASCAL system roll-out expands, the company will remain focused on procedural success and differentiated patient outcomes.

Surgical Structural Heart and Critical Care

Surgical Structural Heart sales for the quarter were $205 million, down one percent compared to the fourth quarter of 2018, and down three percent on an underlying basis. The sales decline was the result of lower surgical aortic valve procedures in the U.S. as TAVR adoption increased, partially offset by continued strong adoption of the company’s premium high-value technologies.

Critical Care sales were $199 million for the quarter, representing an increase of 12 percent versus the fourth quarter of 2018 or eight percent on an underlying basis. This performance was driven by strong demand for the HemoSphere advance monitoring platform and continued adoption of Smart Recovery. Growth in the quarter was led by sales in the U.S.

Additional Financial Results

For the quarter, the company’s adjusted gross profit margin was 75.8 percent, compared to 76.1 percent in the same period last year. This reduction was driven by spending in support of the new European medical device regulations and one-time costs associated with migrating Cardioband production from Israel to Ireland, partially offset by the benefit of a more profitable product mix.

Selling, general and administrative expenses increased 21 percent to $347 million for the quarter, driven by increased field clinical personnel to support TAVR cases in the U.S. and TMTT cases in Europe, as well as accelerated actions related to disease awareness and therapy adoption.

Research and development for the fourth quarter increased 19 percent to $194 million, or 16.5 percent of sales. This increase was primarily the result of continued investments in the company’s transcatheter structural heart programs, including spending on clinical trials.

Free cash flow for the fourth quarter was $328 million, defined as cash flow from operating activities of $399 million, less capital spending of $71 million.

Cash, cash equivalents and short-term investments totaled $1.5 billion at December 31, 2019. Total debt was $594 million.

Outlook

Overall, 2020 sales guidance for Edwards is now expected to be $4.6 to $5.0 billion versus the company’s previous range of $4.5 to $5.0 billion. Additionally, the company now expects full year 2020 adjusted earnings per share of $6.15 to $6.40 versus previous guidance of $6.05 to $6.30.

For the first quarter of 2020, the company projects total sales to be between $1.15 and $1.2 billion, and adjusted EPS of $1.49 to $1.59.

"We were very proud of the significant progress we made in advancing transformational therapies in 2019, as well as our strong financial performance. We are enthusiastic about the future of transcatheter-based technologies, and the promise of treating the many structural heart patients still in need, which positions us very well for 2020 and beyond. We firmly believe our patient-focused innovation strategy can transform care and bring value to patients, healthcare systems, and shareholders," said Mussallem.

On January 30, 2020 VAXIMM AG, a Swiss/German biotech company focused on developing oral T-cell immunotherapies, reported that the Company will participate in several industry events in the coming months (Press release, Vaximm, JAN 30, 2020, View Source [SID1234553685]). Company representatives will be available for networking and one-on-one meetings at the following conferences:

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BIO CEO & Investor Conference
February 10-11, 2020
New York, NY, USA
VAXIMM representatives will be available for one-on-one meetings during the conference. To schedule a meeting, please use the event partnering system or contact [email protected].

13th SACHS Annual European Life Science CEO Forum
February 19-20, 2020
Zurich, Switzerland
Dr. Heinz Lubenau, CEO, will give a corporate presentation on February 19th at 2:15 pm CET and participate in the panel, "Oncology Advanced Therapies & Diagnostics," taking place on the same day at 4:30 pm. The Company will also host one-on-one meetings at this event. To request a meeting with VAXIMM, please sign up through the event’s meeting system.

14th Annual International Partnering Conference BIO-Europe Spring
March 23-25, 2020
Paris, France
VAXIMM plans to give a corporate presentation and host one-on-one meetings at this event. To request a meeting, please sign up through the event’s partneringONE meeting system.

30th MedTech Investing Europe Conference
March 31-April 1, 2020
Lausanne, Switzerland
VAXIMM representatives will be available for one-on-one meetings during the conference. Dr. Heinz Lubenau also plans to give a corporate presentation. To schedule a meeting, please contact [email protected].

On January 30, 2020 THERADIAG (ISIN: FR0004197747, Mnemonic: ALTER), a company specializing in in vitro diagnostics of autoimmune diseases and Theranostics reported its consolidated annual sales and its cash level at December 31, 2019, data under audit (Press release, Theradiag, JAN 30, 2020, View Source [SID1234553704]).

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2019 consolidated revenue of € 9.6 million

As of December 31, 2019, Theradiag generated revenue of 9.6 million euros compared to 8.9 million in 2018, an increase of + 8.3% in line with the strategic axes set by the company.

The Theranostics activity recorded strong growth of + 10.5%, driven by sales of routine LISA TRACKER kits which now represent the company’s recurring activity in this segment. These kit sales exceeded a threshold with 4.3 million euros on the 2019 annual global (vs. 3.9 million at December 31, 2018). In the future, the Theranostics activity may be assimilated to this recurring activity of kit sales, now well in place, and the company will communicate on this one element of global Theranostics.

In France, where the model is now established, sales in Theranostics were particularly satisfactory with growth of 19%.

In the United States, Theradiag has concluded a year of transition reflecting the exit of its former partner in this territory. The transfer to its new partner Halio DX is gradual and will be finalized at the end of the first quarter of 2020.

In export, the company maintains a very satisfactory level of sustained activity, particularly in Spain and the United Kingdom, two strategic countries.

The In Vitro Diagnostics (IVD) activity posted overall growth in sales of + 6.0% with 5.2 million euros at the end of December 2019. It should be noted that this growth includes 1 st semester non-recurring instrumentation sales . Aside from this exceptional element, sales in this historic business segment are, as expected, slightly down. Theradiag pursues in this historical segment, in addition to its primary activity of autoimmunity, a strategy of niche specialization, such as genetics or male fertility.

Cash position

At December 31, 2019, Theradiag’s free cash flow stood at 2.9 million euros compared to 3.4 million euros at December 31, 2018 and 3.2 million euros at June 30, 2019.

Bertrand de Castelnau, CEO of Theradiag commented, "Theradiag and all the teams have made good progress in 2019: new products, new partners, new markets. We followed our roadmap with the objective of developing growth in our strategic priority markets, without compromising the investments necessary for the future of Theradiag. Priority has been given to restructuring the mix of our activities and setting milestones for internationalization, in market segments with higher profitability potential. We are staying the course to gradually improve our financial performance indicators. "

"This year is indeed a pivotal year. The overall sales growth is positive and points in the right direction. The development of the strategy around partnerships centered on Theranostics is gradually unfolding, we have recorded great successes this year and keep our commitment to innovation which is one of our key success factors. " Added Pierre Morgon, Chairman of the Board of Directors of Theradiag.

Reminder of the main events of 2019

Partnership with three new strategic distributors in Asia, December 2019
CE marking of the new i-Track 10 automated system for monitoring biotherapies, November 2019
Strategic agreement with PredictImmune for the marketing of PredictSURE IBD in French-speaking European countries, October 2019
Signing of an agreement with Halio DX as part of its development in the United States, May 2019
Signature of a development and distribution agreement with Immunodiagnostic Systems (IDS) regarding access to the latest generation IDS-i10 controller , April 2019
End of legal dispute with HOB Biotech, April 2019

Financial calendar:

2019 annual results , March 18, 2020, after market close
Annual General Meeting , May 14, 2020

Next congresses in which Theradiag participates:

12-15 February 2020 15 th congress ECCO (European Crohn’s and Colitis Organization), Vienna, Austria
5-6 March 2020: Days of the Syndicate of Young Medical Biologists – Biomed-J 2020, Paris
March 6-7, 2020: Seminar Therapeutic Study of Inflammatory Conditions of the Digestive Tube (GETAID), Paris
March 26-29, 2020: Francophone Days of Hepato-gastroenterology and Digestive Oncology 2020 (JFHOD), Paris.