Comprehensive & Concise News

ONO and BMSKK Submit Supplemental Application for Approval of Additional Indication of Opdivo and Yervoy Combination Therapy in Japan for the Treatment of MSI-H Colorectal Cancer

Nov 12, 2019 : Ono Pharmaceutical Co., Ltd. and Bristol-Myers Squibb K.K. announced that the companies have submitted a supplemental application for combination therapy of Opdivo? (generic name: nivolumab) Intravenous Infusion (“Opdivo”), a human anti-human programmed cell death-1 (PD-1) monoclonal antibody, and Yervoy? (generic name: ipilimumab) Injection (“Yervoy”), a human monoclonal antibody against cytotoxic T-lymphocyteassociated antigen 4 (CTLA-4), in Japan for an additional indication of microsatellite instability high (MSI-H) unresectable advanced or recurrent colorectal cancer (CRC) that has progressed following chemotherapy, for a partial change in approved items of the manufacturing and marketing approval.

This application is mainly based on the result from the Opdivo and Yervoy combination therapy cohort of a multicenter, open-label Phase II clinical study  conducted by BristolMyers Squibb in patients with MSI-H or mismatch repair deficient (dMMR), recurrent or metastatic CRC that has progressed on or after, or been intolerant of prior treatment with chemotherapy including fluoropyrimidine anticancer drugs. https://www.ono.co.jp/eng/news/pdf/sm_cn191112_1.pdf

Roche’s pivotal SUNFISH study of risdiplam in people with type 2 or 3 SMA meets primary endpoint

Nov 11, 2019 : Roche announced positive data from the pivotal Part 2 of SUNFISH, a study evaluating risdiplam in people aged 2-25 years with Type 2 or 3 spinal muscular atrophy (SMA). The study met its primary endpoint of change from baseline in the Motor Function Measure 32 (MFM-32) scale after one year of treatment with risdiplam, compared to placebo. No treatment related safety findings leading to study withdrawal have been seen in any risdiplam trial to date. Safety for risdiplam was consistent with its known safety profile and no new safety signals were identified.

“The positive outcome of this trial is an important milestone for people with Type 2 or 3 SMA, too many of whom remain untreated,”. Risdiplam is an investigational survival motor neuron-2 (SMN2) splicing modifier, designed to durably increase and sustain SMN protein levels both throughout the central nervous system and peripheral tissues of the body. https://www.roche.com/media/releases/med-cor-2019-11-11.htm

FDA tags F2G’s olorofim as Breakthrough Therapy for fungal infections

Nov 11, 2019: The US drugs regulator has tagged F2G’s olorofim as a potential Breakthrough Therapy for life-threatening fungal infections, pledging to expedite development of the mid-stage candidate. The indication the biotech is targeting includes infections due to Lomentospora prolificans, Scedosporium, and Scopulariopsis species, and makes this the first antifungal agent to be granted Breakthrough Therapy designation.

Olorofim is currently being investigated in an open-label single-arm phase 2b study in patients with proven invasive fungal disease or probable invasive aspergillosis, whose disease resists standard therapies or when existing drugs cannot be tolerated. So far olorofim, which is being developed in both intravenous and oral formulations, has been well tolerated and data from this study were provided to the FDA as part of the company’s submission for Breakthrough Therapy status.

Orotomides selectively target fungal dihydroorotate dehydrogenase (DHODH), a different mechanism from that of the currently marketed antifungal agents giving them fungicidal activity against a broad range of rare and resistant fungal mould infections. https://pharmaphorum.com/news/fda-tags-f2gs-olorofilm-as-breakthrough-therapy-for-mould-infections/

Lipocine's Male Hormone Therapy Stumbles With FDA For The Third Time

Nov 11, 2019: Lipocine Inc  shares are tumbling to record lows after an adverse FDA verdict on its lead drug Tlando, previously known as LPCN 1021.

Lipocine said that the FDA said "nay" for the third time for its Tlando, an oral testosterone replacement therapy for use in adult males for treating conditions associated with hypogonadism, which is a deficiency of endogenous testosterone.The complete response letter, or CRL, issued by the FDA signaling the application cannot be approved in the present form, pointed to one deficiency — the efficacy trial not meeting the three secondary endpoints for maximal testosterone concentration. Lipocine expressed disappointment at the FDA rejection, which is understandable, given Tlando is the most advanced drug candidate in its pipeline. https://www.benzinga.com/general/biotech/19/11/14777047/lipocines-male-hormone-therapy-stumbles-with-fda-for-the-third-time

Allergan Receives FDA Qualified Infectious Disease Product (QIDP) Designation And Fast Track Designation For ATM-AVI (Aztreonam And Avibactam) For Antibiotic-Resistant Gram-Negative Infections

Nov 11, 2019: Allergan plc announced the U.S. Food and Drug Administration (FDA) has granted Qualified Infectious Disease Product (QIDP) Designation and Fast Track Designation for ATM-AVI (aztreonam and avibactam), for the treatment of complicated intra-abdominal infections (cIAI), complicated urinary tract infections (cUTI), and hospital-acquired bacterial pneumonia (HABP)/ventilator-associated bacterial pneumonia (VABP). ATM-AVI is an investigational, fixed-dose, intravenous combination antibiotic under development globally.

ATM-AVI has activity against metallo β-lactamase (MBL)-producing Gram-negative pathogens, for which there are currently very limited treatmentoptions. Although aztreonam is not inactivated by metallo beta-lactamases (MBLs), as a single agent it has limited utility because the vast majority of MBL-producing pathogens also express serine-β-lactamases that can inactivate it. When combined with avibactam's ability to inhibit many serine-β-lactamases , aztreonam's activity is restored against pathogens that co-produce MBLs and serine enzymes, thus presenting a new treatment for patients with these infections. ATM-AVI is currently in Phase III trials.

ATM-AVI is being jointly developed with Pfizer. Allergan holds the rights to commercialize ATM-AVI in North America, while Pfizer holds the rights to commercialize this investigational therapy in the rest of the world. https://fda.einnews.com/article__detail/501823916-allergan-gets-fda-qidp-designation-and-fast-track-designation-for-atm-avi?vcode=XIbw

AI Medical Service Inc. announces FDA Breakthrough Device Designation for endoscopic AI system

 Nov. 11, 2019 : AI Medical Service Inc  announced the Company has secured Breakthrough Device Designation by the U.S. Food and Drug Administration (FDA) for its AI programs that analyze endoscopy images for potential diagnosis of gastric cancer. This designation will be critical as the Company continues to accelerate clinical and regulatory program especially in terms of prioritized review of the submission. At the same time as obtaining approval in its original country, Japan, the Company will continue to work through the regulatory process in the United States and other countries, and move on to global expansion.

AI Medical Service's system applies neural network algorithms which are trained with large real-world datasets of images of biopsy-proven cancer lesions, benign lesions, and normal tissue that were captured using standard endoscopes, thus aiding physicians performing endoscopies to detect lesions suspicious for cancer. https://fda.einnews.com/article__detail/501825899-ai-medical-service-inc-announces-fda-breakthrough-device-designation-for-endoscopic-ai-system?vcode=XIbw

Glenmark Pharmaceuticals receives ANDA approval for Adapalene and Benzoyl 

Peroxide Gel, 0.1%|2.5%

Nov. 11, 2019 : Glenmark Pharmaceuticals said it has received final approval from the US health regulator for generic Adapalene and Benzoyl Peroxide gel used in treatment of acute acne. The approval granted to Glenmark Pharmaceuticals Inc by the US Food and Drug Administration is for Adapalene and Benzoyl Peroxide Gel, 0.1 per cent/2.5 per cent, which is the the generic version of Epiduo gel, 0.1 per cent/2.5 per cent of of Galderma Laboratories LP, the company said in a statement.

Citing IQVIA sales data for the 12 month period ending September 2019, Glenmark said Epiduo Gel, 0.1 per cent/2.5 per cent had achieved annual sales of approximately USD 33.7 million. https://www.glenmarkpharma.com/sites/default/files/Glenmark%20receives%20ANDA%20approval%20for%20Adapalene%20and%20Benzoyl%20Peroxide%20Gel%2C%200.1%25%2C2.5%25_0.pdf

Novartis data show tropifexor (LJN452) significantly improves several key biomarkers of NASH in patients with moderate to severe fibrosis

Nov 11, 2019 – Novartis announced positive interim results from the final part of its phase IIb FLIGHT-FXR adaptive design study, assessing the safety, tolerability, and efficacy of tropifexor in patients with biopsy-confirmed stage 2-3 fibrotic non-alcoholic steatohepatitis (NASH).

This interim analysis showed higher doses of tropifexor (140 Mu g and 200 Mu g) resulted in improvements in several key biomarkers of NASH, including hepatic fat fraction, alanine aminotransferase and body weight, with favorable safety after 12 weeks of treatment. The data will be presented during The Liver Meeting 2019 in Boston this week. https://www.novartis.com/news/media-releases/novartis-data-show-tropifexor-ljn452-significantly-improves-several-key-biomarkers-nash-patients-moderate-severe-fibrosis

Sandoz announces agreement to acquire Aspen’s Japanese operations and associated assets, strengthening position in world’s third largest generics market

Nov 11, 2019: Sandoz announced that it has entered into a binding agreement for the planned acquisition of the Japanese business of Aspen Global Incorporated (AGI), a wholly owned subsidiary of Aspen Pharma care Holdings Limited. The planned acquisition consists of shares in Aspen Japan K.K. and associated assets held by AGI. Pursuant to the agreed terms of the transaction, on closing Sandoz will pay an initial cash consideration of EUR 300 million. Sandoz has also agreed, upon certain conditions being fulfilled after closing, to pay certain deferred consideration to AGI. It is currently anticipated that the amount of deferred consideration to be paid will not exceed EUR 100 million.

Aspen’s portfolio in Japan consists of off-patent medicines with a focus on anesthetics and specialty brands. Additionally, AGI has entered into a five year manufacturing and supply agreement (with an additional two year extension option) with Sandoz, which will take effect from completion of the transaction, for the supply of active pharmaceutical ingredients, semi-finished and finished goods related to the portfolio of divested brands. https://www.novartis.com/news/media-releases/sandoz-announces-agreement-acquire-aspen%27s-japanese-operations-and-associated-assets-strengthening-position-world%27s-third-largest-generics-market

The pathway to Parkinson's takes a surprising twist

Nov 11, 2019: Researchers at The Rockefeller University found that affected neurons in Parkinson's disease can shut down without fully dying. These undead neurons, the team found, release chemicals that shut down their otherwise healthy neighbors as well, leading to the stuttering and halting effects seen in Parkinson's patients.

They are found all over the body. As part of a normal process called senescence, cells may shut down when they recognize they have suffered a DNA damage during division. This helps to prevent damaged cells from growing uncontrollably and causing problems like cancer. The researchers, led by the late Paul Greengard, set out to investigate the exact function of a Parkinson's-linked protein called SATB1 in dopamine-producing neurons, whose activity is reduced in Parkinson's disease. https://www.longroom.com/discussion/1694066/the-pathway-to-parkinsons-takes-a-surprising-twist

Bayer invests USD 50 Million in eGenesis Series B financing round

Nov 11, 2019 – eGenesis, a biotechnology company utilizing breakthrough gene editing technologies for the development of effective human-compatible organs to address the global organ shortage, successfully completed a USD 100 million Series B financing. The financing was led by Fresenius Medical Care Ventures (FMCV), with participation from new investors including Leaps by Bayer and Wellington Partners. Existing investors, including, but not limited to, ARCH Venture Partners, Biomatics Capital, Alta Partners, and Khosla Ventures, all participated.

eGenesis is committed to helping solve the global organ shortage by pioneering an alternative source of human-compatible organs. It is currently estimated that there are between 1.5 to 2 million people on the organ transplant waitlist globally. This financing will enable the acceleration of the eGenesis kidney xenotransplant program into the clinic, as well as support advancement of a range of other xenotransplant programs across islet cell, liver, heart, and lung. https://media.bayer.com/baynews/baynews.nsf/ID/Bayer-invests-USD-50-Million-in-eGenesis-Series-B-financing-round