Friday, November 27, 2015
- The FDA today declined to approve an expanded use for Bristol-Myers Squibb's (NYSE:BMY) Opdivo to treat another type of advanced skin cancer.
- Earlier this week, the company won approval for the immuno-oncology drug to treat advanced kidney cancer, as well as single-agent treatment of patients with BRAF V600 wild-type melanoma.
- But with a "complete response letter," the agency declined to approve the drug for treating BRAF V600 mutation positive metastatic melanoma -- a skin cancer that is both the rarest and deadliest. It indicated a need for additional data from the patient population.
- Opdivo has received approval for five other indications since its first approval last December.
- Previously: FDA clears new indication for Bristol-Myers' Opdivo (Nov. 24 2015)
- Previously: Bristol-Myers wins FDA approval for Opdivo to treat advanced kidney cancer (Nov. 23 2015)
- Sanofi (NYSE:SNY) is up 1% after hours on news it's working with investment bank Lazard to prepare a sale or listing of Merial, its animal health unit, which could value the division up to €12B (about $12.7B), Reuters is reporting.
- On an investor day Nov. 6, new chief Olivier Brandicourt discussed cost cuts and acquisitions, but hinted at strategic options for unloading Merial and its European generics business due to limited synergies.
- Lazard has a close relationship with Sanofi via the drugmaker's chairman, Serge Weinberg. Sanofi may favor an IPO for the unit to avoid antitrust challenges in selling to a rival, sources told Reuters.
- Merial has about 6,500 employees and expects to exceed €2.4B in sales this year.
- The generics business is worth up to €3B, but any divestment faces a longer path with heavy legal disentanglement ahead, and wouldn't start until the second half of next year.
- Nano cap Harvard Apparatus Regenerative Technology (HART +25.4%) jumps on triple normal volume. Shares are up almost four-fold sine November 11 as investors take positions ahead of a presentation by CEO Jim McGorry at an investor event in Los Angeles on Tuesday, December 1.
- Before the rally, shares had lost ~95% of their value on diminished prospects for the company's regenerated tracheas.
- The company generated some recent buzz with its report of clear evidence of esophageal tissue regeneration in animal studies based on its second generation bioengineered platform. The company plans to file an IND with the FDA in 2016 and aims to commence compassionate use procedures in humans sometime in 2016.
- Previously: Harvard Apparatus backs out of Russia (Nov. 26, 2014)
- Previously: HART up 34% on exoneration of misconduct by famed trachea transplant surgeon (Aug. 31)
- Previously: HART gets delisting warning from Nasdaq over share price (Nov. 10)
- The FDA's Psychopharmacologic Drugs Advisory Committee will meet on Tuesday, December 1, to review privately held Fabre-Kramer Pharmaceuticals' New Drug Application (NDA) for gepirone hydrochloride extended-release tablets for the treatment of major depressive disorder (MDD).
- FDA briefing doc
- Company briefing doc
- Draft questions
- MDD-related tickers: (OTCQB:VSTA)(OTCPK:OTSKY)(OTCPK:TKPYY)(NYSE:PFE)(NASDAQ:ALKS)(NASDAQ:AVNR)(NYSE:TEVA)(NASDAQ:SHPG)(NYSE:LLY)(NYSE:GSK)
- Exact Sciences (NASDAQ:EXAS) reports that CMS's intended 2016 reimbursement for its colorectal cancer screening test Cologuard, just published on its website, is $501.26, higher than the advisory committee-recommended $492.72. Although good news, the company has asked for clarification on the reasons for the change. It will communicate it to investors when available.
- Previously: Exact Sciences closes up 6.8% after positive pricing ruling (Aug. 26)
- Monday is the FDA's PDUFA date for its review of Repros Therapeutics' (NASDAQ:RPRX) New Drug Application (NDA) for lead product candidate Androxal (enclomiphene citrate) for the treatment of men with low testosterone due to secondary hypogonadism, a condition caused by insufficient stimulation of the testes by the pituitary gland.
- The agency canceled November 3rd's Ad Comm meeting stating that outside expert advice was no longer needed, a good sign.
- Previously: PDUFA goal date is November 30 for Repros' enclomiphene citrate (April 15)
- Micro cap KaloBios (NASDAQ:KBIO) is up 80% premarket on increased volume. The stock rocketed from a low of $0.44 on November 16 to a high of $45.82 five days later after an investor group led by Martin Shkreli bought 70% of the outstanding shares. Shares retraced almost 60% to $18.40 before rallying again.
- Previously: Martin Shkrelli new chief of KaloBios; up to $13M in funding committed (Nov. 20)
- Previously: KaloBios -47% as it says it's shutting down (Nov. 13)
- Lannett (NYSE:LCI) finalizes its $1.23B acquisition of Kremers Urban Pharmaceuticals from Brussels, Belgium-based UCB S.A.
- The company expects to provide updated fiscal 2016 guidance in mid-December. The combined firm generated pro forma revenues of more than $800M for the 12-month period ending June 30, 2015.
- Previously: Lannett to buy Kremers Urban Pharmaceuticals for $1.23B; shares up 16% premarket (Sept. 2)
- Regenerative medicine firm Mesoblast (NASDAQ:MESO) announces that Japan's National Health Service has established reimbursement for its TEMCELL (MSC-100-IV) mesenchymal stem cell product for the treatment of acute graft versus host disease (aGVHD) after an allogeneic bone marrow transplant (BMT). Market launch will commence in February by licensee JCR Pharmaceuticals.
- The approved dosing regimen is eight doses of 2M cells/kg delivered as an IV infusion. For patients with persistent symptoms beyond four weeks, an additional 2M cells/kg may be administered each week for four more weeks.
- The reimbursement is 868,680 yen or $7,079 per bag of 72M cells. The average adult is expected to receive 16 to 24 bags so the total reimbursement will be about 13.9M to 20.8M yen or $113,000 to $170,000.
- According to the CDC, there are ~30K allogeneic BMTs performed each year worldwide, 8,900 in the U.S. Half will develop aGVHD.
- The company expects to complete enrollment in its U.S.-based 60-subject Phase 3 study assessing MSC-100-IV (remestemcel-L) in pediatric patients this quarter. Top-line results should be available in Q3 2016. If successful, a Biologics License Application (BLA) will be filed by the end of 2016.
Wednesday, November 25, 2015
- Spark Therapeutics (ONCE +3.7%) has filed an S-1 for a proposed public offering of shares.
- Some of the stock in the offering would be sold by Spark, and some by the Children's Hospital of Philadelphia Foundation, the company said.
- Shares in the company are up 36.6% since news on Oct. 5 that it saw positive results from a late-stage trial of a gene therapy candidate for patients with genetic blinding conditions.
- Previously: Spark Therapeutics misses by $0.22, misses on revenue (Nov. 04 2015)
- Amgen (NASDAQ:AMGN) submits its first biosimilar Biologics License Application (BLA) to the FDA for ABP 501, a biosimilar version of AbbVie's (NYSE:ABBV) Humira (adalimumab).
- EVP of R&D Sean Harper, M.D., says, "The submission of Amgen's first biosimilar application to the FDA is an exciting milestone, expanding our inflammation portfolio to provide additional therapeutic options to patients. Patients with chronic inflammatory conditions are faced with a significant burden of disease requiring long-term treatment. Amgen's branded biologic medicines and biosimilars are developed and manufactured according to the same high standards and we are committed to delivering high quality medicines to patients with serious inflammatory diseases."
- Previously: Amgen's ABP 501 equivalent to Humira in late-stage RA study (Nov. 9)
- Jerusalem, Israel-based Kitov Pharmaceuticals (KTOV +7%) completes its IPO of 3,168,900 American Depository Shares (ADSs) and five-year warrants to purchase up to 3,158,900 ADSs for a combined price of $4.13. Each ADS represents 20 ordinary shares. Each warrant is immediately exercisable at $4.13. Underwriters over-allotment is an additional 473,835 ADSs and/or warrants to purchase an additional 253,761 ADSs at a price of $4.12 and $0.01, respectively.
- The clinical-stage biopharmaceutical firm develops combinations of existing drugs that are in late-stage development. Its pipeline includes KIT-301, a fixed-dose combination of naproxen and isradipine, for the treatment of pain caused by osteoarthritis (OA) without the hypertensive side effects, and KIT-302, a fixed dose combination of celecoxib and amlodipine besylate, also for the treatment of OA-related pain. In the near term, the company is focusing its efforts entirely on bringing KIT-302 to market.
- 2015 Financials (6 mo.)($M): Operating Expenses: 3.1 (+92.0%); Net Loss: (3.2) (-90.8%); Cash Burn: (1.3) (+55.6%).
- Thinly traded Aduro Biotech (ADRO +11.8%) is up on average volume in response to its announcement that all of its clinical trials will continue unaltered and as planned.
- Apparently, one clinical trial participant who received the company's lead oncology candidate, CRS-207, which is based on engineered Listeria mononcytogenes bacteria, showed signs of Listeria infection subsequent to a protocol violation. The investigator could not rule out that the infection was not attributed to the administration of CRS-207 so the case was filed as listeriosis (disease caused by listeria infection) related to CRS-207 infusion. The company believes the incident arose from inappropriate access of the central line port during infusion. The FDA was apprised of the situation almost two months ago and has provided no comments or guidance that would delay or alter ongoing or planned trials. The patient, who was treated with IV antibiotics which resolved the infection, remains in the study and continues to receive CRS-207 and GVAX Pancreas vaccine.
- The most frequent CRS-207-related adverse event (AE) (Grade 3 (severe) or 4 (life threatening or disabling)) seen in a Phase 2a study was lymphopenia (abnormally low level of white blood cells). Three patients experienced a Grade 3 AE and two a Grade 4 AE.
- The combination of CRS-207 and GVAX Pancreas is currently being evaluated in a Phase 2b study in metastatic pancreatic cancer. CRS-207 is also being investigated, with and without cyclophosphamide, in a Phase 1b study in mesothelioma. A Phase 2 study in platinum-resistant ovarian, fallopian or peritoneal cancer should commence in early 2016.
- As expected, the European Commission approves Roche's (OTCQX:RHHBY +1.5%) Cotellic (cobimetinib), in combination with Zelboraf (vemurafenib), for the treatment of adults with unresectable or metastatic BRAF V600 mutation-positive melanoma.
- Cobimetinib, co-developed with discoverer Exelixis (EXEL +1.1%), inhibits an enzyme called mitogen-activated protein kinase kinase (MEK), a mediator of downstream signaling of tumor growth factor receptors. Vemurafenib is a BRAF inhibitor that was discovered by Daiichi Sankyo's (OTCPK:DSKYF)(OTCPK:DSNKY) Plexxikon unit.
- Previously: European Ad Comm gives thumbs up to Roche's Cotellic/Zelboraf combo for certain type of melanoma (Sept. 25)
- In a regulatory filing, Spectrum Pharmaceuticals (NASDAQ:SPPI) discloses that it has resubmitted its New Drug Application (NDA) to the FDA for Orphan Drug-tagged EVOMELA (melphalan) for the conditioning treatment prior to hematopoietic stem cell transplant in multiple myeloma patients or palliative treatment of multiple myeloma. The FDA's decision date (PDUFA) is May 9, 2016.
- The company received a Complete Response Letter (CRL) in October in response to its initial submission.
- Previously: No FDA approval for Spectrum Pharma's CE-Melphalan NDA, CRL received; shares down 21% premarket (Oct. 23)
- The FDA designates Cellceutix's (OTCPK:CTIX) Phase 2-stage oral rinse Brilacidin-OM for Fast Track review for the prevention of oral mucositis, a common and debilitating inflammation and ulceration in the mouth that occurs as a side effect of certain cancer treatments. There is only one approved therapy for the condition, AMAG Pharma's (NASDAQ:AMAG) MuGard oral rinse.
- According to clinicaltrials.gov, the estimated final data collection date for the primary endpoint (WHO OM Score) in the 60-subject Phase 2 trial is June 2016. The estimated study completion date is December 2016.
- Fast Track status allows for more frequent interactions with the FDA review team and a rolling review of the New Drug Application (NDA).
- A Phase 3 clinical trial assessing Pfizer's (NYSE:PFE) LYRICA (pregabalin) for the treatment of chronic post-traumatic peripheral neuropathic pain failed to achieve its primary endpoint of a statistically valid reduction in pain from baseline to week 15.
- There are currently no approved treatments in the U.S. for the condition. The company does not disclose whether it is abandoning its pursuit of the indication or considering another late-stage trial.
- LYRICA is currently approved for the treatment of neuropathic pain associated with diabetic peripheral neuropathy, postherpetic neuralgia, adjunctive therapy in adults with partial onset seizures and neuropathic pain associated with spinal cord injury.
- Allergan (NYSE:AGN) and start-up Rugen Therapeutics enter into an exclusive collaboration to discover and develop novel therapies for Autism Spectrum Disorders and Obsessive Compulsive Disorders, focusing on small molecule candidates.
- Under the terms of the agreement, Rugen will receive an upfront payment and milestones. Allergan will have the exclusive option to acquire all rights, including global IP, to the compounds following clinical proof-of-concept studies. Specific financial terms are not disclosed.
- As expected, the European Commission approves Impax Laboratories' (NASDAQ:IPXL) NUMIENT (carbidopa-levodopa extended-release capsule) for the symptomatic treatment of adults with Parkinson's disease (PD). It is the company's first branded drug approval outside of the U.S.
- In clinical studies, PD patients receiving NUMIENT (formerly IPX066) experienced a decrease in "off" time (36.9% to 23.8%) compared to immediate-release levodopa-carbidopa. An "off" episode is a period of time when PD symptoms reemerge despite taking PD medications. In addition, NUMIENT patients increased "on" time without dyskinesia (abnormal involuntary movements) by 1.9 hours compared to 0.8 hours for the immediate-release version.
- IPX066 is marketed under the brand name RYTARY in the U.S.
- Previously: European Ad Comm backs Impax Labs' NUMIENT for Parkinson's symptoms (Sept. 25)
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