On May 3, 2016 Foundation Medicine, Inc. (NASDAQ:FMI) reported financial and operating results for its first quarter ended March 31, 2016 (Press release, Foundation Medicine, MAY 3, 2016, View Source [SID:1234511841]). Highlights for the quarter included:

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Achieved first quarter revenue of $30.4 million, 57% year-over-year growth;
Reported 8,985 clinical tests in the first quarter, 14% year-over-year growth;
Grew the FoundationCORE molecular information knowledgebase, now approaching 80,000 patient cases;
Presented analytic validation of FoundationACT, the company’s circulating tumor DNA (ctDNA) assay;
Publicly-released a broad pediatric data set from FoundationCORE for research purposes to enable precision medicine;
Published 9 manuscripts in high-quality, peer-reviewed journals and delivered 41 podium and poster talks at various medical and scientific meetings.
Foundation Medicine reported total revenue of $30.4 million in the first quarter of 2016, compared to $19.3 million in the first quarter of 2015 and $26.1 million in the fourth quarter of 2015. Revenue from clinical testing in the first quarter of 2016 was $10.2 million, compared to $11.1 million in the first quarter of 2015 and $12.0 million in the fourth quarter of 2015.

The company reported 8,985 clinical tests in the first quarter of 2016, a 14% increase from the same quarter last year. This number includes 7,957 FoundationOne tests and 1,028 FoundationOne Heme tests.

Revenue from biopharmaceutical customers more than doubled year-over-year to $20.2 million in the first quarter of 2016, compared to $8.2 million in the first quarter of 2015 and $14.1 million in the fourth quarter of 2015. This growth was fueled by the achievement of milestone revenue during the first quarter. The results of 2,622 tests were reported to biopharmaceutical customers in this year’s first quarter.

"Foundation Medicine delivered a solid first quarter highlighted by strong revenue growth and healthy clinical volume," said Michael Pellini, M.D., chief executive officer of Foundation Medicine. "Since the beginning of the year, we have also made great strides in generating a tremendous amount of data to support the clinical utility of our comprehensive genomic profiling approach across a diverse set of cancers. We expect that these data, coupled with our best-in-class portfolio of analytically validated assays, which now includes FoundationACT, will be critical differentiators with physicians, payers, and most importantly, patients."

The company’s molecular information knowledgebase, FoundationCORE, grew to nearly 80,000 patient cases. FoundationCORE is a unique asset and critical component of the value that Foundation Medicine delivers to its biopharmaceutical and physician customers. The increasing scale and breadth of a high quality, clinically relevant oncology data set derived from the company’s analytically validated testing platform continues to enhance clinical practice and enable improved outcomes for patients.

Total operating expenses for the first quarter of 2016 were approximately $36.5 million compared with $34.0 million for the fourth quarter of 2015. Net loss was approximately $17.3 million in the first quarter of 2016, or a $0.50 loss per share. At March 31, 2016, the company held approximately $213.5 million in cash, cash equivalents and marketable securities.

Recent Enterprise Highlights

Launched FoundationACT, the company’s ctDNA assay to clinical customers. FoundationACT was developed with the same rigorous analytical validation standards as FoundationOne and FoundationOne Heme, and is the third clinical product launch by the company in just four years.
Announced an agreement with AstraZeneca to develop novel companion diagnostic assays to enable physicians to identify patients most likely to benefit from targeted medicines within AstraZeneca’s oncology pipeline.
Expanded the company’s U.S. laboratory footprint to include a second site at Research Triangle Park in North Carolina. When operational, the company expects this new facility will support continued innovation and expansion of its product portfolio, provide important lab redundancies, increase operational flexibility, and broaden commercial opportunities.
2016 Outlook

Foundation Medicine’s business and financial outlook for 2016 remains unchanged:

The company expects 2016 revenue will be in the range of $110 to $120 million.
The company expects to deliver between 37,000 and 40,000 FoundationOne and FoundationOne Heme clinical tests in 2016.
The company expects operating expenses will be in the range of $175 and $185 million.
The company expects to expand upon reimbursement progress made in 2015 and drive additional coverage decisions.

Mutations in genes encoding splicing factors (which we refer to as spliceosomal genes) are commonly found in patients with myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML). These mutations recurrently affect specific amino acid residues, leading to perturbed normal splice site and exon recognition. Spliceosomal gene mutations are always heterozygous and rarely occur together with one another, suggesting that cells may tolerate only a partial deviation from normal splicing activity. To test this hypothesis, we engineered mice to express a mutated allele of serine/arginine-rich splicing factor 2 (Srsf2(P95H))-which commonly occurs in individuals with MDS and AML-in an inducible, hemizygous manner in hematopoietic cells. These mice rapidly succumbed to fatal bone marrow failure, demonstrating that Srsf2-mutated cells depend on the wild-type Srsf2 allele for survival. In the context of leukemia, treatment with the spliceosome inhibitor E7107 (refs. 7,8) resulted in substantial reductions in leukemic burden, specifically in isogenic mouse leukemias and patient-derived xenograft AMLs carrying spliceosomal mutations. Whereas E7107 treatment of mice resulted in widespread intron retention and cassette exon skipping in leukemic cells regardless of Srsf2 genotype, the magnitude of splicing inhibition following E7107 treatment was greater in Srsf2-mutated than in Srsf2-wild-type leukemia, consistent with the differential effect of E7107 on survival. Collectively, these data provide genetic and pharmacologic evidence that leukemias with spliceosomal gene mutations are preferentially susceptible to additional splicing perturbations in vivo as compared to leukemias without such mutations. Modulation of spliceosome function may thus provide a new therapeutic avenue in genetically defined subsets of individuals with MDS or AML.

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On May 3, 2016 Foundation Medicine, Inc. (NASDAQ:FMI) reported the launch of FoundationACT (Assay for Circulating Tumor DNA), an analytically validated and accurate blood-based circulating tumor DNA (ctDNA) assay that provides patients and oncologists with a new option for comprehensive genomic profiling when a tissue biopsy is not feasible or when tissue is not available (Press release, Foundation Medicine, MAY 3, 2016, View Source [SID:1234511842]). By analyzing circulating tumor DNA isolated from a patient’s blood, FoundationACT can identify clinically relevant genomic alterations, and like Foundation Medicine’s tissue-based genomic profiles, FoundationOne and FoundationOne Heme, FoundationACT delivers this comprehensive molecular information in a concise report that matches the findings with potentially relevant targeted therapies and clinical trials.

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"Our goal has always been to deliver a highly accurate and sensitive assay available for blood based samples, and the confirmatory data produced by our clinical collaborators indicates that we have succeeded in achieving that with the release of FoundationACT," said Vincent Miller, M.D., chief medical officer of Foundation Medicine. "Bringing the rigor used to develop and validate FoundationOne to this assay, we believe we have overcome many of the clinical limitations presented by other tests in the liquid biopsy field. Launching FoundationACT strengthens our market leading position as the go-to resource for a complete, end-to-end offering of comprehensive molecular information solutions."

The presence of ctDNA, which is DNA shed from tumors that circulates in blood, is a well-established phenomenon that has led to the development of non-invasive tumor sequencing assays. However, the concentration of ctDNA compared to other DNA fragments derived from other tissue sources can vary significantly depending on tumor type and disease stage. For many cancer patients, this means that the proportion of detectable tumor DNA in the blood is extremely low, making the detection of therapeutically relevant genomic alterations much more difficult and error prone as compared to tissue-based approaches.

FoundationACT, which was launched to Foundation Medicine’s pharmaceutical partners for research use in December 2015, interrogates all clinically relevant alterations across 62 genes and fusions across six genes, and it has been optimized for sensitivity and specificity of all classes of molecular alterations, including base substitutions, insertions and deletion, focal amplifications and gene fusions. The assay was developed for patients who are not candidates for comprehensive genomic profiling with FoundationOne. Factors that may preclude patients from undergoing FoundationOne testing include insufficient or inadequate tissue from a recent biopsy, safety risks associated with biopsy, or medical contraindications to re-biopsy. In July 2015, Foundation Medicine initiated a large-scale, multi-center prospective clinical study to clinically validate FoundationACT across various cancers and stages of the disease. A portion of the patients in the study will include those with earlier-stage disease, allowing the company to investigate how different tumor types shed DNA into the bloodstream at different stages of the disease.

Technical Validation Data Confirms Accuracy, Sensitivity of FoundationACT Genomic Profiling Assay

At the Annual Meeting of the Advances in Genome Biology and Technology (AGBT) in February 2016, Foundation Medicine reported data from its analytic validation study in a presentation titled, "Assessment of the Relative Clinical Utility of ctDNA and Tissue Biopsies for the Detection of Actionable Genomic Alterations in Routine Clinical Oncology Specimens." Study highlights demonstrated that FoundationACT results were 100 percent concordant with FoundationOne and droplet digital PCR (ddPCR) results across 87 base substitutions (43 at < 5% MAF), three indels and five genomic alterations.

At the American Association of Cancer Research Annual Meeting in April 2016, Foundation Medicine further reported analytical validation data in a poster presentation titled, "Rigorous Validation of a Clinical Circulating Tumor DNA Assay for Cancer Molecular Profiling." As part of this study, results compared alterations from patient-matched ctDNA and FFPE biopsies across more than 200 samples from lung, breast and other cancers. This rigorous analytic validation study demonstrated ≥99 percent sensitivity in the detection of alterations present in blood at low frequency with a very low rate of false positives, realizing the potential of ctDNA-based molecular profiling for the management of patients with cancer. In 48 clinical ctDNA samples, 95 alterations of all classes were 100 percent confirmed by orthogonal testing.

Our lead pipeline candidate is CNTX-4975, a highly potent, ultrapure, synthetic form of capsaicin (a derivative of the chili plant), called trans-capsaicin, which is the first and only, patented capsaicin designed to be administered via injection into the site of pain (Company Pipeline, Centrexion Therapeutics, MAY 3, 2016, View Source [SID:1234511816]). CNTX-4975 was developed in a research lab at The Johns Hopkins School of Medicine by co-founder and CSO, James Campbell, M.D. It is being evaluated for the treatment of osteoarthritis (OA) pain of the knee in humans and canines, as well as in patients with Morton’s neuroma, a painful foot condition. CNTX-4975 is injected directly into the affected knee or neuroma.

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Centrexion harnessed the natural analgesic power of capsaicin to develop its exclusive injectable therapy designed to provide fast-acting, long-lasting and targeted pain relief.
It works by selectively targeting the capsaicin receptor (also known as TRPV1) that inactivates the nerve fibers transmitting pain signals to the brain – a therapeutic affect that can last for months until the nerve fiber regenerates. Through its targeted delivery process and method of action, CNTX-4975 manages pain without disrupting other nerve functions.

On May 3, 2016 AstraZeneca reported that it has completed the acquisition of the core respiratory business of Takeda Pharmaceutical Company Limited ("Takeda") (Press release, AstraZeneca, MAY 3, 2016, View Source [SID:1234511817]). The agreement, announced in December 2015, includes the expansion of rights to roflumilast (marketed as Daliresp in the US and Daxas in other countries), the only approved oral PDE4 inhibitor for the treatment of chronic obstructive pulmonary disease. AstraZeneca has marketed Daliresp in the US since the acquisition of the rights from Actavis in the first quarter of 2015.

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