On April 29, 2016 Shire plc ("Shire") (LSE: SHP, NASDAQ: SHPG) reported unaudited results for the three months ended March 31, 2016 (Press release, Shire, APR 29, 2016, View Source [SID:1234511736]).

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Financial Highlights Q1 2016 Growth(1) Non GAAP CER(1)(2)
Product sales $1,627 million +14% +16%
Total revenues $1,709 million +15% +17%

Non GAAP operating income $797 million +17% +16%
US GAAP operating income from continuing operations $544 million +15%

Non GAAP EBITDA margin (excluding royalties & other revenues)(3) 46% 0pps(4)
US GAAP net income margin(5) 25% -3pps

Non GAAP net income $632 million +13%
US GAAP net income $419 million +2%

Non GAAP diluted earnings per ADS $3.19 +12% +12%
US GAAP diluted earnings per ADS $2.12 +2%

Non GAAP cash generation $492 million -5%
Non GAAP free cash flow $338 million +38%
US GAAP net cash provided by operating activities $390 million -31%
(1) Percentages compare to equivalent 2015 period.
(2) On a Constant Exchange Rate ("CER") basis, which is a Non GAAP measure.
(3) Non GAAP earnings before interest, tax, depreciation and amortization ("EBITDA") as a percentage of product sales, excluding royalties and other revenues.
(4) Percentage point change ("pps").
(5) US GAAP net income as a percentage of total revenues.

The Non GAAP financial measures included within this release are explained on pages 25 – 26, and are reconciled to the most directly comparable financial measures prepared in accordance with US GAAP on pages 19 – 22.

First Quarter & Recent Highlights:

Product sales growth of 14% (16% on a Non GAAP CER basis) to $1.6 billion, driven by VYVANSE, LIALDA/MEZAVANT, CINRYZE, FIRAZYR, GATTEX/REVESTIVE and NATPARA.
Rare disease products acquired from NPS Pharmaceuticals, Inc. ("NPS") continued to perform well with GATTEX/REVESTIVE sales up 247% (up 97% on a pro-forma basis(1)) to $52 million, and NATPARA sales of $16 million.
Free cash flow remained strong, impacted primarily by net payments and receipts of taxes between Q1 2015 and Q1 2016.
Lifitegrast New Drug Application ("NDA") accepted by the US Food and Drug Administration ("FDA"), with Prescription Drug User Fee Act ("PDUFA") date set for July 22, 2016.
Pipeline progression with positive topline results from SHP465 safety and efficacy study in children and adolescents with Attention Deficit Hyperactivity Disorder ("ADHD").
Completed acquisition of Dyax Corp. ("Dyax") and enrollment on track for SHP643 (formerly DX2930) Phase 3 studies for the treatment of Hereditary Angioedema ("HAE").
Patent upheld for LIALDA (mesalamine) delayed release tablets by U.S. District Court for the Southern District of Florida; the case has been appealed.
Baxalta Incorporated ("Baxalta") acquisition on track with integration progressing well; shareholder votes set for May 27 and closing anticipated in early June.
(1) Sales prior to February 21, 2015 were recorded by NPS.
Flemming Ornskov, M.D. Chief Executive Officer, commented:

"Shire is off to a strong start in 2016, delivering double-digit product sales and Non GAAP earnings per ADS growth, and advancing our innovative pipeline. We were pleased to report positive Phase 3 topline results for SHP465 in children and adolescents with ADHD, a therapeutic area with significant need for additional treatment options. We are also looking forward to hearing from the FDA by late July regarding lifitegrast, a potential new treatment for dry eye disease.

While we maintain our sharp focus on Shire’s business, we closed the acquisition of Dyax during the quarter and we are making excellent progress with the Baxalta integration planning. Our shareholder vote is scheduled for May 27 and the closing is anticipated to follow in early June. We look forward to officially welcoming our Baxalta colleagues to Shire, and creating a global biotechnology leader focused on rare diseases and other highly specialized conditions."

On April 29, 2016 ImmunoGen, Inc. (Nasdaq: IMGN), a biotechnology company developing targeted cancer therapeutics using its proprietary ADC technology, reported financial results for the three-month period ended March 31, 2016 – the third quarter of the Company’s 2016 fiscal year (Press release, ImmunoGen, APR 29, 2016, View Source [SID:1234511596]). ImmunoGen also provided an update on the Company’s lead program, mirvetuximab soravtansine, and other wholly owned clinical-stage product candidates.

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"We are making important progress with our key product programs," commented Daniel Junius, President and CEO. "In early June, expanded Phase 1 findings with mirvetuximab soravtansine will be presented at ASCO (Free ASCO Whitepaper). Based on these data, we are modifying the design of our FORWARD I trial to be a Phase 3 study intended to support full marketing approval. Patient enrollment is proceeding well in our Phase 1b/2 FORWARD II trial that is assessing this novel ADC in combination regimens, and patient dosing has begun in Phase 1 testing of IMGN779, the first ADC utilizing one of our new DNA-alkylating cancer-killing agents."

Mr. Junius continued, "Our partners are also making progress. Takeda has reported preclinical information on a GCC-targeting ADC it is developing utilizing our DNA-alkylating technology, and Novartis and Sanofi recently presented preclinical data on product candidates with our maytansinoid technology. Phase 1 clinical data with Bayer’s anetumab ravtansine and Sanofi’s SAR566658 are scheduled for poster discussion at ASCO (Free ASCO Whitepaper), with data also being presented on Sanofi’s isatuximab."

ImmunoGen Product Program Updates

Mirvetuximab soravtansine – First FRα-targeting ADC; potential new treatment for FRα-positive ovarian cancer.

Data will be presented at ASCO (Free ASCO Whitepaper) from a 46-patient Phase 1 expansion cohort assessing this ADC as monotherapy for FRα-positive platinum-resistant ovarian cancer (abstract #5567). This cohort was increased from 20 patients to provide additional experience in the patient population to better inform the design of ImmunoGen’s FORWARD I trial. The data presented will be updated from the 20-patient data reported previously and from that available at the time of abstract submission.
Based on the expanded findings, ImmunoGen is modifying its FORWARD I trial from a two-stage, Phase 2 trial with response rate as the primary endpoint to a single-stage, Phase 3 trial with progression-free survival as the primary endpoint. Patients with FRα-positive (medium or high) platinum-resistant ovarian cancer treated with up to three prior regimens will be eligible for enrollment.
Patient enrollment is ongoing in the FORWARD II trial assessing mirvetuximab soravtansine in combination regimens. A cohort is being added to assess this novel ADC in combination with Merck’s anti-PD1, pembrolizumab.
IMGN779 – First-in-class CD33-targeting ADC utilizing a DNA-alkylating cancer-killing agent from ImmunoGen’s new family called IGNs.

Patient enrollment has started in the Phase 1 trial assessing this ADC for the treatment of acute myeloid leukemia.
IMGN529 and coltuximab ravtansine – CD37- and CD19-targeting, respectively, ADCs for diffuse large B-cell lymphoma (DLBCL).

Patient enrollment is expected to open shortly in a Phase 2 trial assessing IMGN529 in combination with rituximab and in 1H2017 for coltuximab ravtansine in a combination regimen.
Update on Partner Programs

Phase 1 findings with Sanofi’s SAR566658 and Bayer’s anetumab ravtansine ADCs with ImmunoGen technology have been accepted for poster discussion at ASCO (Free ASCO Whitepaper), with data also being presented on Sanofi’s isatuximab (SAR650984).
ImmunoGen, Novartis, and Sanofi had multiple ADC-related presentations at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) annual meeting earlier this month. Those by ImmunoGen scientists featured new, novel technologies while those by Novartis and Sanofi related to cadherin6- and LAMP1-targeting ADCs, respectively, utilizing ImmunoGen maytansinoid ADC technology.
Takeda reported data at a scientific conference on a GCC-targeting ADC the company is developing utilizing one of ImmunoGen’s new IGN agents.
Financial Results

For the Company’s quarter ended March 31, 2016 (3QFY2016), ImmunoGen reported a net loss of $31.9 million, or $0.37 per basic and diluted share, compared to a net loss of $21.6 million, or $0.25 per basic and diluted share, for the same quarter last year (3QFY2015).

Revenues for 3QFY2016 were $19.7 million, compared to $11.4 million for 3QFY2015. The current period includes a $10 million milestone earned from Bayer with the advancement of anetumab ravtansine into a Phase 2 clinical trial designed to support product registration. License and milestone fees for the prior year period include a $5 million milestone earned from Novartis with its initiation of LOP628 Phase 1 clinical testing. Revenues in 3QFY2016 include $7.4 million of non-cash royalty revenues, compared with $5.1 million in cash royalty revenues for the prior year period. Revenues for 3QFY2016 also include $1.2 million of clinical materials revenue and $1.1 million of research and development support fees, compared with $0.7 million and $0.5 million, respectively, in the prior year period.

Operating expenses in 3QFY2016 were $47.3 million, compared to $32.7 million in 3QFY2015. Operating expenses in 3QFY2016 include research and development expenses of $36.1 million, compared to $25.7 million in 3QFY2015. This change is primarily due to increased third-party costs related to the advancement of our wholly owned product candidates, increased clinical trial costs, primarily related to our expansion of the mirvetuximab soravtansine development program, and increased personnel expenses, principally due to recent hiring. Operating expenses include general and administrative expenses of $11.2 million in 3QFY2016, compared to $7 million in 3QFY2015. This increase is primarily due to a non-cash stock compensation charge resulting from the CEO transition, as well as increased personnel expenses and professional services.

ImmunoGen had approximately $182.9 million in cash and cash equivalents as of March 31, 2016, compared with $278.1 million as of June 30, 2015, and had no debt outstanding in either period. Cash used in operations was $91.6 million in the first nine months of FY2016, compared with $26.8 million in the same period in FY2015. The prior year period benefited from $25 million in upfront payments received including $20 million in connection with the execution of the right-to-test agreement with Takeda in March 2015, as well as lower operating expenses. Capital expenditures were $8.6 million and $4.5 million for the first nine months of FY2016 and FY2015, respectively.

Financial Guidance for Fiscal Year 2016

ImmunoGen has updated its guidance for its fiscal year ending June 30, 2016. Expected revenues are now projected to be between $60 million and $70 million, compared with previous guidance of between $70 million and $80 million. The change is primarily due to changes in the expected timing of partner events and is mainly non-cash. Operating expenses are now projected to be between $180 million and $185 million, compared with previous guidance of between $175 million and $180 million. The change is primarily related to greater clinical trial costs and non-cash stock compensation charges. The Company’s guidance for its net loss is now expected to be between $135 million and $140 million, compared to its previous estimate of $120 million and $125 million with most of this change being non-cash related.

ImmunoGen now projects cash and cash equivalents at June 30, 2016 to be between $155 million and $160 million, compared to previous guidance of $165 million to $170 million. This change reflects the cash impact of less partner upfront and milestone payments. The Company’s guidance for cash used in operations is now projected to be between $110 million and $115 million, which had previously been $100 million and $105 million. The Company’s guidance for capital expenditures remains unchanged, which is between $13 million and $15 million.

On April 29, 2016 Medivation, Inc. (NASDAQ: MDVN) reported that its Board of Directors, after consultation with its financial and legal advisors, unanimously determined that the unsolicited proposal from Sanofi to acquire Medivation for $52.50 per share in cash substantially undervalues Medivation and is not in the best interests of the company and its stockholders (Press release, Medivation, APR 29, 2016, View Source [SID:1234511611]).

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"Over the past several years, we have established a world class oncology franchise and a unique, diversified and highly-promising late-stage development pipeline," said David Hung, M.D., Founder, President and Chief Executive Officer of Medivation. "Further, we have a track record of delivering extraordinary value to our stockholders. Sanofi’s opportunistically-timed proposal, which comes during a period of significant market dislocation, and prior to several important near-term events for the company, is designed to seize for Sanofi value that rightly belongs to our stockholders. We believe the continued successful execution of our well-defined strategic plan will deliver greater value to Medivation’s stockholders than Sanofi’s substantially inadequate proposal."

The Medivation Board of Directors’ unanimous conclusion was based on the following:

The proposal substantially undervalues Medivation and its leading oncology franchise.

Medivation has significant scarcity value as one of the few profitable, commercial-stage oncology companies;it has brought a blockbuster product to market and is leveraging its expertise to develop and bring to market additional products.

Medivation has built XTANDI (enzalutamide) capsules into a rapidly-growing, multi-billion dollar oncology product and remains on track to achieve its 2016 U.S. net sales guidance, which implies approximately 28% growth (at the mid-point) for the year.
XTANDI is one of the most successful oncology product launches in history and just surpassed Johnson & Johnson’s Zytiga (abiraterone) in U.S. market share, despite launching sixteen months later.

XTANDI has achieved worldwide annual net sales of $2.2 billion on a run rate basis, less than four years after its initial approval.
XTANDI has significant patent life with 10+ years of remaining exclusivity.

XTANDI is poised to capitalize on a substantial, near-term commercial opportunity in urology, enabling it to serve a larger patient population of men with metastatic castration-resistant prostate cancer (mCRPC) and increasing the duration of therapy.
The PDUFA date for TERRAIN/STRIVE label expansion on October 22, 2016, is rapidly approaching and is anticipated to drive significantly greater adoption by urologists.

In April, the CHMP issued a positive opinion to include findings from the TERRAIN trial in the European label.
Medivation recently expanded its specialty salesforce to create dedicated urology and oncology selling teams that are just starting to have a promotional impact.

XTANDI has multiple opportunities beyond mCRPC, which are not reflected in the company’s current valuation.
Ongoing Phase 3 trials, i.e. PROSPER and EMBARK, are designed to move XTANDI even earlier in the prostate cancer treatment paradigm; PROSPER is on track to complete enrollment of 1,560 patients in mid-2017.

Medivation is pursuing clinical development across three major subtypes of breast cancer, a new and significant market opportunity for XTANDI, and expects to report top-line Phase 2 data in patients with ER/PR+ breast cancer, which represents 50% of all breast cancers, in the second half of 2016.

A Phase 3 trial in Triple Negative Breast Cancer, a significant unmet need, is expected to initiate later in 2016.
The company continues to explore XTANDI in other solid tumor indications and settings, e.g., in hepatocellular carcinoma and in combination with immunotherapy.

Sanofi’s proposal would deny Medivation’s stockholders the value of Medivation’s wholly-owned, innovative late-stage pipeline.

Talazoparib represents another blockbuster opportunity as a potentially best-in-class PARP inhibitor targeting a wide range of oncology indications.

Top-line data from the Phase 3 EMBRACA trial in germline BRCA mutated advanced breast cancer is expected in the first half of 2017.

Recent data reported at AACR (Free AACR Whitepaper) demonstrate talazoparib’s potential in tumors with defects in DNA repair beyond BRCA deficiency and possibly in patients without evidence of homologous recombination deficiency when used in combination with low dose chemotherapy.

The company is preparing to initiate several clinical trials in 2016, including in breast cancer beyond germline BRCA mutations, prostate cancer, ovarian cancer and small cell lung cancer, including potentially registrational trials.
Medivation recently met with the FDA to discuss clinical trial design and a potential accelerated approval pathway in prostate cancer.

Talazoparib is highly synergistic with our existing development and commercial infrastructure.
Additional clinical data demonstrating talazoparib’s potent activity is expected to be presented at a medical meeting later in the year.

Pidilizumab has the potential to be a novel immuno-oncology candidate supported by clinical efficacy and a strong safety profile in several hematological malignancies.

The execution of Medivation’s business plan will deliver value to its stockholders that is far superior to Sanofi’s proposal.

Medivation has an exceptional track record for execution.

XTANDI has achieved all development and sales milestones under Medivation’s collaboration with Astellas.

Medivation’s track record for delivering value to our stockholders is exemplified by XTANDI, which was in-licensed as a pre-clinical asset in 2005, received full FDA approval in seven years (better than industry average), and achieved $2.2 billion in worldwide annual net sales on a run rate basis in less than four years.

The company has generated a 1,440%+ total shareholder return for its stockholders since 2009.

Medivation has already achieved two years of profitability, despite only launching XTANDI in late 2012, and the company has pursued minimal, dilutive capital raises.
Sanofi’s timing is designed to benefit Sanofi – not Medivation’s stockholders.

Sanofi approached Medivation following a period of significant market dislocation in biotech and just as the market was beginning to recover.
The "private offer" was submitted to Medivation when the NBI index was almost 30% below its July 2015 high.
The proposal offer price is:
21% below Medivation’s 52-week trading high of $66.40
Less than a 14% premium to Medivation’s 12-month volume weighted average price, or VWAP
Only a 9% premium to Medivation’s VWAP over the last month
Medivation will provide additional information on its financial performance, XTANDI’s utilization and the company’s clinical development plans for talazoparib on next week’s earnings call.

Kim D. Blickenstaff, Chairman of Medivation’s Board of Directors, notes that "Medivation has a long history of producing superior growth and generating significant value for its stockholders. Since the launch of XTANDI, Medivation has achieved revenues of nearly $1 billion in just over three years. There are several exciting pipeline opportunities that will drive significant growth. The Board is determined to continue to aggressively focus on working for, and delivering value to, Medivation’s stockholders."

On April 28, 2016 Provectus Biopharmaceuticals, Inc. (NYSE MKT: PVCT, www.pvct.com), a clinical-stage oncology and dermatology biopharmaceutical company ("Provectus" or "The Company"), reported that two abstracts related to research into IL PV-10 for treatment for melanoma have been published in a special issue of the ANZ Journal of Surgery detailing the Royal Australasian College of Surgeons 85th Annual Scientific Congress, 2–6 May 2016, in Queensland, Australia (Filing, 8-K, Provectus Pharmaceuticals, APR 28, 2016, View Source [SID:1234511549]).

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The first abstract, titled "Intralesional PV-10 for In-Transit Melanoma – A Single Centre Experience," notes that "Intralesional PV-10 has been used at Peter MacCallum Cancer Centre since 2010, and the current report presents a retrospective analysis of patient outcomes, reporting the response rates, durability of responses and observed toxicities."

The Peter MacCallum Cancer Centre, in East Melbourne, Victoria, Australia, is Australia’s only public hospital solely dedicated to cancer treatment, research and education. The abstract was authored by Jocelyn Lippey et al. and examined data from nineteen patients receiving PV-10 at the center.

The second abstract, titled "Intralesional PV-10 Chemoablation Therapy for the Treatment of Cutaneous Melanoma Metastases – Results of a Prospective, Non-Randomised, Single Centre Study," summarizes work done at the Princess Alexandra Hospital in Brisbane, Queensland, Australia. The authors, Tavis Read et al., set out "to assess the clinical efficacy and treatment outcomes of patients receiving intralesional (IL) PV-10 chemoablation therapy for the treatment of cutaneous melanoma metastases." This report examined data from forty five patients receiving PV-10 at the hospital.

For more information about the special issue of the ANZ Journal of Surgery where the abstracts appear, visit View Source ("Abstract Journal for Surgical Oncology," pages 157-160) or View Source (abstracts SO006 and SO007).
For more information about the RACS Annual Scientific Congress, visit: View Source

On April 28, 2016 Medical Need part of Immedica Group reported that it has entered into an exclusive supply and distribution agreement with the French company Laboratoires CTRS, regarding marketing and sale of CTRS portfolio of pharmaceuticals in the Nordic region (Press release, Immedica Pharma, APR 28, 2016, View Source [SID1234555256]). In 2013, CTRS received EU marketing authorization and orphan drug market exclusivity for its product Orphacol (cholic acid), indicated for the treatment of two rare inborn errors of metabolism in the primary bile acid synthesis: 3β-hydroxy-Δ5-C27-steroid oxidoreductase and Δ4-3-oxosteroid-5β-reductase deficiency. A few weeks ago, the European commission approved a centralized marketing authorization for the company’s second product, Neofordex, containing a high and appropriate dosage (40 mg) of dexamethasone, a common component used in combination with other pharmaceuticals in the treatment of multiple myeloma. While already well established in the treatment protocols, prior to the approval of Neofordex, dexamethasone has only been available in low strengths (0.5-4 mg), forcing patients to take a very high number of tablets (10-80 per day) to achieve an appropriate dosage. In addition to the increased convenience for the patients, since an adequate dose exposure is critical for efficacy of the treatment regimen, the expectation is that the availability of a tablet in appropriate strength could improve compliance and thereby potentially the treatment outcome. Under the agreement, Medical Need gains the rights to Neofordex and Orphacol in Denmark, Finland, Iceland, Norway and Sweden, and will be responsible for the distribution, marketing and sale of the products in that territory. "CTRS has a very exciting portfolio which fits well with our competencies and capabilities in Medical Need", said Tomas Gloveus, Head of Marketing and Sales at Medical Need, and continued, "Orphacol and Neofordex both fulfil high unmet medical needs, which have previously not been adequately served in this region, and we look forward to now being able to make these products available to the affected patients in the Nordic countries."
About Multiple Myeloma and Neofordex

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Multiple myeloma (MM) is a form of cancer that affects a type of white blood cells called plasma cells. Plasma cells are part of the immune system and normally helps the body fight infections by producing antibodies. Rather than producing helpful antibodies, in MM, the cancer cells produce abnormal proteins that over time damages the kidneys. MM also causes cancer cells to accumulate in the bone marrow, where they crowd out healthy blood forming cells which results in a deficiency of red and white blood cells, as well as blood platelets. This in turn leads to a number of different symptoms, depending on the deficient cell; anemia (paleness, fatigue), leukopenia (sensitivity to infection) and thrombocyotopenia (bleeding and bruising). MM also causes damage to the bone and skeleton. MM is slightly more prevalent in men and typically affects the elderly (>65), but may affect individuals as young as 30. MM is treated by a number of treatment protocols, which typically involve several different pharmaceuticals. Over the past years, several new products have been approved for the treatment of MM as part of such treatment protocols. A common component in most of the treatment protocols is dexamethasone (denoted as "d" or "D" in the protocol abbreviations), a potent and long-acting corticosteroid, which has been shown to play an important part in the efficacy of the different regimens. The typical daily dose of dexamethasone in the treatment of MM is 40 mg. Neofordex is the only approved pharmaceutical which contains 40 mg of dexamethasone in a single tablet. Prior to the approval of Neofordex, dexamethasone has only been available in low strengths (0.5-4 mg), forcing patients to take a very high number of tablets (10-80 per day) to achieve an appropriate dosage. An adequate dose exposure of dexamethasone has been shown to be of high importance for the efficacy of the treatment regimen (Kobayashi et al., Int J Hematol. 2010 Nov;92(4):579-86). It is the expectation that the availability of a tablet in an appropriate strength, in addition to the improved convenience from reducing the pill burden for the patients, could translate into increased compliance and thereby potentially improving the treatment outcome. Neofordex was on 2016-03-16 granted a centralized marketing authorization by the European Commission, valid for all markets of the EU, Norway and Iceland. Prior to the regulatory approval, the product has been used extensively on a named patient basis in France, under what is called an ATU cohort, including most French MM patients. The product is planned to be launched in the Nordic and Baltic markets during Q2 2016.

About Inborn Errors of Primary Bile Acid Synthesis and Orphacol
Inborn errors of primary bile acid synthesis are very rare inherited conditions, caused by mutations in the genes encoding certain enzymes responsible for the liver’s production of bile acids, one of the key components of the bile. Left untreated, these enzyme deficiencies lead to the accumulation of hepatotoxic metabolites and progression to irreversible cholestasis and liver failure, and are usually fatal. Orphacol is the only approved treatment for two particular inborn errors in the primary bile acid synthesis: 3β-hydroxy-Δ5-C27-steroid oxidoreductase and Δ4-3-oxosteroid-5β-reductase deficiency. Orphacol contains cholic acid, which acts through a dual mechanism, by suppressing the faulty bile acid synthesis thus reducing formation of hepatotoxic metabolites and by restoring the biliary secretion and elimination of toxic metabolites through the bile. It also corrects the intestinal malabsorption of fats and fat-soluble vitamins, thereby improving the child’s growth. Treating affected patients with Orphacol can avoid the need for a liver transplantation, an operation with very serious potential consequences, especially in young children. Orphacol was granted a centralized marketing authorization by the European Commission, valid for all markets of the EU, Norway and Iceland in 2013. The product also enjoys orphan drug market exclusivity for 10 years following marketing authorization. The product will immediately be made available on the Nordic and Baltic markets.