Ionis Pharmaceuticals (IONS) : Biogen-partnered ALS drug trial fails

 

Biogen (BIIB) and Ionis Pharmaceuticals' (IONS) approach to Lou Gehrig's disease flopped over the weekend, leading IONS stock to collapse to a five-year low Monday.

The duo tested their drug, tofersen, in patients with amyotrophic lateral sclerosis, also known as ALS. Patients with this disease lose muscle control due to dysfunction in the nerve cells of the brain and spinal cord.

Tofersen-treated patients didn't improve enough to deem the study a success, and IONS stock tumbled. SVB Leerink analyst Mani Foroohar says the data release marks "another misstep" for one of Ionis' partnered programs in central nervous system disorders. Previously, Roche (RHHBY) discontinued a study of Ionis' Huntington's disease treatment.

IONS Stock Dives On Misstep

Patients showed improvement in function — as measured by a scale for ALS assessment — but the results weren't strong enough to be statistically significant.

Biomarker data also suggested the drug was, in fact, engaging the mutated gene responsible for patients' disease. Further, patients who received tofersen showed greater declines in a marker of neurodegeneration, known as neurofilament, than those who received the placebo.

But the drug also carried some side effects. Serious adverse events occurred in almost 6% of patients who received tofersen vs. none in the placebo group, Needham analyst Joseph Stringer said in a report. Those included inflammation in the spinal cord, an infection called meningitis, a disease of the lumbar spinal nerve root and other nervous system disorders.

-=Biogen (BIIB) : major patent win against Mylan (MYL)

•  Biogen  won a long-awaited decision in a patent case defending its multiple sclerosis drug Tecfidera against a challenge by Mylan.
• In a decision issued Wednesday, administrative patent judge Sheridan K. Snedden wrote that Mylan (MYL) had failed to show that parts of a key patent protecting Tecfidera were unpatentable, as the company had claimed.

Biogen stock rocketed Wednesday after reports suggested U.S. patent officials agreed to uphold the patent covering Tecfidera, an important multiple sclerosis drug.

In midday trading on the stock market today, shares of Biogen (BIIB) surged 22.7%, near 347.60. That pushed Biogen stock into a breakout, above a buy point at 318.10 out of a consolidation.

Mylan (MYL) and Biogen have been engaged in a patent battle over Tecfidera for years. So, the patent decision by U.S. officials has been a major point of contention for Biogen stock. According to various posts on Twitter, officials upheld the patent on Tecfidera for another eight years.

As a result, Mylan won't be able to launch a generic challenger to the blockbuster drug until 2028, according to various reports. This would be a huge boon for Biogen stock. Meanwhile, Mylan stock rose 3.4%, near 23.

=Biogen (BIIB) reported earnings on Tue 22 Oct 2019 (b/o)

• Earnings + experimental Alzheimer's therapy -- the same drug was considered a failure in March 2019

Biogen plans to pursue regulatory approval for aducanumab for early Alzheimer's disease -- Company plans to submit Biologics License Application in early 2020

The co and Eisai (ESALY) announced that, after consulting with the U.S. Food and Drug Administration (FDA), Biogen plans to pursue regulatory approval for aducanumab, an investigational treatment for early Alzheimer's disease (AD). The Phase 3 EMERGE Study met its primary endpoint showing a significant reduction in clinical decline, and Biogen believes that results from a subset of patients in the Phase 3 ENGAGE Study who received sufficient exposure to high dose aducanumab support the findings from EMERGE. Patients who received aducanumab experienced significant benefits on measures of cognition and function such as memory, orientation, and language.

• Patients also experienced benefits on activities of daily living including conducting personal finances, performing household chores such as cleaning, shopping, and doing laundry, and independently traveling out of the home. If approved, aducanumab would become the first therapy to reduce the clinical decline of Alzheimer's disease and would also be the first therapy to demonstrate that removing amyloid beta resulted in better clinical outcomes.
• Based on discussions with the FDA, the Company plans to file a Biologics License Application (BLA) in early 2020 and will continue dialogue with regulatory authorities in international markets including Europe and Japan. The BLA submission will include data from the Phase 1/1b studies as well as the complete set of data from the Phase 3 studies.

Biogen beats by $0.95, beats on revs

Reports Q3 (Sep) earnings of $9.17 per share, excluding non-recurring items, $0.95 better than the S&P Capital IQ Consensus of $8.22; revenues rose 4.7% year/year to $3.6 bln vs the $3.53 bln S&P Capital IQ Consensus.

"SPINRAZA continued on a strong trajectory, particularly outside the U.S., and we are preparing for the expected launch of VUMERITY, which we believe will be an important addition to our market-leading multiple sclerosis portfolio. In addition to the recent news on aducanumab, we made strong progress in our pipeline as we initiated new clinical programs targeting Parkinson's disease and brain contusion, and we look forward to nine important data readouts by the end of next year. We continue to believe that our core focus on neuroscience will lead to new innovative treatments for patients and will maximize long-term returns for our shareholders."

Recent Events

• In October 2019 the U.S. Food and Drug Administration (FDA) issued a tentative approval for VUMERITYTM (diroximel fumarate), a novel oral fumarate with a distinct chemical structure, for the treatment of relapsing forms of MS.
• In August 2019 Biogen discontinued the Phase 2b study of BG00011 for IPF due to safety concerns.

In the third quarter of 2019 Biogen recognized a GAAP-only impairment charge of approximately $216 million and a GAAP-only gain of $61 million on fair value remeasurement of contingent consideration, both related to the discontinuation of BG00011.

Shares are currently +39% after the company announced positive results from an Alzheimer's study and disclosed plans to pursue regulatory approval following discussions with the FDA. 

Earnings this week : Oct 21 - 25, 19 (wk 43)

Monday (Oct 21)

• Morning: HAL LII PETS SAP
• Afternoon: ACC ELS HXL HMST LOGI AMTD  TACO

Tuesday (Oct 22)

• Morning: ABG BIIB CNC GATX HOG HAS JBLU KMB LMT MCD PCAR  PII PG DGX SERV  SHW TRV  TTS  UTX UPS
• Afternoon: BYD CMG CNI CSGP CSL DFS EEFT EQR FMBI FULT HA HCSG HIW IRBT LRN MANH MPWR MTH NAVI NBHC NBTB NTB SIX SKX SNAP TER TRMK TXN UCBI USNA WHR WRB

Wednesday (Oct 23)

• Morning: ABB ALKS ALXN ANTM APH AVY BA BANC BCO BKU BOKF BPOP BSX BX CADE CAT CLF CMC CSTM EVR FCFS FCX FSV GD GWW HLT HRI HTLD IVZ KNX LAD LLY MHO MKTX NDAQ NSC NTRS OC PB PNR RCI RES ROL SALT SCL SLAB SLGN TDY TMO TZOO UNF WGO WM
• Afternoon: AEM AKR ALGN AMP APY ARI ASGN BCOV BMRN BXMT CLB CLGX CMRE CP CUBI CUZ CVBF DHT EBAY ECHO EFX EGP EIG ESRT ESS EW F FBHS FFIV FR FTI GGG GL HNI ICLR ISBC KALU KNL KRA KRC LMAT LRCX LSTR LVS MKSI MSA MSFT MX NGVT NOW NTGR NXGN ORLY PKG PLXS PTC PYPL QEP RHI RJF RRC SAVE SEIC SLM SRPT STL SUI TRN TSLA UFPI VAR VMI WPG WSBC XLNX

Thursday (Oct 24)

• Morning: AAL AB ADS AEP AIMC ALLE ASPS AXTA AZN BAX BC BGCP CMCS.A CMS COLB COWN CRI CRS CTS CTXS DHR DOW ENTG FAF FCN GNC GPI GRA HBAN HEES HSY IART KIM LH LUV MMM MNRO MSM MTRN NOC NOK ODFL ONDK ORI PATK PDS PRLB PTEN ROP RS RTN SAH SCHN STM SWK TAL TECK TROW TRTN TSCO TWTR UBSI VC VLO VLY WST XEL
• Afternoon: AFL AJG ALGT ALK AMZN ANIK ASB AUY AVT BHE BJRI BOOM CERN CINF CLS COF COG CSLT CUBE CVA CXP CY DECK DLX EHTH EMN ENVA FET FHB FII FIX FLEX FSLR FTV FWRD GCAP GILD HLI ILMN INTC JNPR KN LOGM LPLA MHK MXL NTUS OIS OMCL OSIS PEB PFG PFPT POWI PRO RMD SBCF SIVB SPSC SWN TNET UHS V VCRA VRSN VVI WERN WRE 

Friday (Oct 125)

• Morning: ALV AON B BEN BUD CHTR GBX GRC GT GVA HUN ITW KEX LEA NRZ PFS PSX PSXP ROCK SBSI SJR TILE UFS VFC VRTS VTR VZ WETF WY YNDX

Reports this week:

• Monday: No economic data scheduled
• Tuesday: September Existing Home Sales (prior 5.49 mln) at 10:00 ET and $40 bln 2-yr Treasury note auction results at 13:00 ET
• Wednesday: Weekly MBA Mortgage Index (prior 0.5%) at 7:00 ET; August FHFA Housing Price Index (prior 0.4%) at 9:00 ET; weekly crude oil inventories (prior +8.0 mln) at 10:30 ET; and $41 bln 5-yr Treasury note auction results at 13:00 ET
• Thursday: Weekly Initial Claims (prior 214,000), Continuing Claims (prior 1.679 mln), September Durable Orders (prior 0.2%), and Durable Orders ex-transportation (prior 0.5%) at 8:30 ET; September New Home Sales (prior 713,000) at 10:00 ET; weekly natural gas inventories (prior +104 bcf) at 10:30 ET; and $32 bln 7-yr Treasury note auction results at 13:00 ET
• Friday: Final October Michigan Consumer Sentiment Survey (prior 96.0) at 10:00 ET

Notable earnings reports: 

• Halliburton (NYSE:HAL), SAP SE (NYSE:SAP) and TD Ameritrade (NASDAQ:AMTD) on October 21; 
• UPS (UPS), McDonald's (MCD), Procter & Gamble (PG), Travelers (NYSE:TRV), United Technologies (NYSE:UTX), Centene (NYSE:CNC), Chipotle (NYSE:CMG), Lockheed Martin (NYSE:LMT), Discover Financial Services (NYSE:DFS), Hasbro (NASDAQ:HAS) and Kimberly-Clark (NYSE:KMB) on October 22; 
• Boeing (BA), Caterpillar (CAT), Microsoft (NASDAQ:MSFT), Anthem (NYSE:ANTM), Blackstone (NYSE:BX), Boston Scientific (NYSE:BSX), Canadian Pacific (NYSE:CP), Cleveland-Cliffs (NYSE:CLF), eBay (NASDAQ:EBAY), Edwards Lifesciences (NYSE:EW), Eli Lilly (NYSE:ELY), Equifax (NYSE:EFX), F5 Networks (NASDAQ:FFIV), Ford (NYSE:F), PayPal (NASDAQ:PYPL), Tesla (TSLA) and General Dynamics (NYSE:GD) on October 23; 
• Intel (INTC), Visa (NYSE:V), Amazon (AMZN), Comcast (NASDAQ:CMCSA), Raytheon (NYSE:RTN), Southwest Airlines (NYSE:LUV), T-Mobile (NASDAQ:TMUS), Twitter (TWTR) and Northrop Grumman (NGC) on October 24; 
• Verizon (NYSE:VZ) and Anheuser-Busch InBev (NYSE:BUD) on October 25. 

=Biogen (BIIB) halts research on Alzheimer's drug

• Biogen is halting two global phase-three trials testing the once-promising Alzheimer’s drug aducanumab, delivering a late-stage blow to researchers searching for therapies for the incurable degenerative disease.
• In 2018, pharmaceutical companies including Eli Lilly, AstraZeneca, Pfizer and Merck halted research or development into possible Alzheimer’s and dementia therapies due to disappointing results.

March 21 (Reuters) - Biogen and partner Eisai Co Ltd are ending two late-stage trials testing an Alzheimer's drug, they said Thursday, marking the latest setback for an industry keen to develop treatments for the memory-robbing disease.

Shares in Biogen slid 25 percent to $81.60 in premarket trading.

The decision to discontinue the trials testing drug aducanumab was made after an independent data monitoring committee reported the drug was unlikely to be successful, the companies said. The recommendation was not based on safety concerns, they added.

After dozens of experimental Alzheimer's drugs have failed in the recent past, there is a desperate need for a treatment that works.

The disease is the most common form of dementia that affects nearly 50 million people worldwide and is expected to rise to more than 131 million by 2050, according to Alzheimer's Disease International.

Biogen said it would continue to develop other treatments for Alzheimer's.

"This disappointing news confirms the complexity of treating Alzheimer's disease and the need to further advance knowledge in neuroscience," Biogen Chief Executive Officer Michel Vounatsos said.

"We will continue advancing our pipeline of potential therapies in Alzheimer's disease." The companies, however, for now will also discontinue a mid-stage study and a long-term extension study of aducanumab.

====

Several drugmakers have recently come up short in efforts to find an effective treatment for Alzheimer’s. Among the drugmakers still pursuing a beta amyloid-targeting compound is Roche Holding AG. Like Biogen, the Swiss company has persisted despite setbacks. After its candidate, gantenerumab, failed an earlier test four years ago, Roche decided to ratchet up the dose and try again with a pair of big late-stage trials. Results from the studies aren’t expected for about another two years.

Roche mostly abandoned a second beta amyloid compound earlier this year, halting two large studies after concluding they would fail. Roche’s partner on the second compound, Alzheimer’s specialist AC Immune SA, at one point fell more than 12 percent in premarket trading in New York.

=Biogen (BIIB) reported earnings on Tue 24 July 2018 (b/o)

Biogen beats by $0.71, beats on revs; raises FY18 EPS and rev above consensus; acquires ALG-801 (Phase 1a) and ALG-802 (preclinical) from AliveGen 

• Reports Q2 (Jun) earnings of $5.80 per share, excluding non-recurring items, $0.71 better than the Capital IQ Consensus of $5.09; revenues rose 9.1% year/year to $3.36 bln vs the $3.25 bln Capital IQ Consensus. 
• Multiple sclerosis (MS) revenues were $2.3 billion, including ~ $113 million in royalties on the sales of OCREVUS. Tecfidera -2% to $1.09 bln; Spinraza (owned by IONS and marketed by Biogen) +16% Q/Q and 108% Y/Y to $423M. The number of commercial patients receiving SPINRAZA grew ~13% in the U.S. and ~ 47% outside the U.S. versus the first quarter of 2018.
• Co issues upside guidance for FY18, raises EPS to $24.90-25.50 from $24.20-25.20, excluding non-recurring items, vs. $23.91 Capital IQ Consensus; raises FY18 revs to $13.0-13.2 bln from $12.7-13.0 bln vs. $12.96 bln Capital IQ Consensus Estimate.
• Biogen announced it has acquired ALG-801 (Phase 1a) and ALG-802 (preclinical) from AliveGen, Inc. ALG-801 (now known as BIIB110) and ALG-802 represent novel ways of targeting the myostatin pathway, which is one of the most thoroughly studied approaches for muscle enhancement. BIIB110 and ALG-802 are recombinant proteins that act as ActRIIB ligand traps to inhibit myostatin pathway signaling, and their targeted mechanism of action may result in greater efficacy and improved safety compared to other myostatin approaches. We initially plan to study BIIB110 and ALG-802 in multiple neuromuscular indications including spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis. The acquisition includes an upfront payment of $27.5 million, and Biogen may pay up to $535 million in additional potential development and commercialization milestones across both assets and multiple indications.
• Co will update BAN2401 alzheimer's data in an Eisai poster presentation on Wednesday, July 25th starting at 9:30 a.m. CT at AAIC in Chicago.

====Biogen (BIIB) Alzheimer's drug shows potential to slow disease

Cambridge, MA-based Biogen (Nasdaq: BIIB) and its partner, Japanese pharmaceutical company Eisai Co., announced late Thursday that patients who received the drug saw a reduction in amyloid — a protein whose buildup in the brain has been linked to cognitive decline — after 18 months of treatment, compared to those on a placebo.  The trial represents a rare success in Alzheimer’s drug research. The field has been marked by a series of high-profile failures in late-stage trials, with one large drugmaker after another coming up short in recent years.


Biogen and Eisai (ESALY) report positive topline results of the final analysis for BAN2401  

• Eisai and Biogen announce positive topline results of the final analysis for BAN2401 at 18 Months
  • The final analysis at 18 months of the 856 patient Phase II clinical study in early Alzheimer's disease demonstrated statistically significant slowing in clinical decline and reduction of amyloid beta accumulated in the brain
  • First late-stage study data successfully demonstrating potential disease- modifying effects on both clinical function and amyloid beta accumulation in the brain
  • New data provide compelling evidence to further support amyloid hypothesis as a therapeutic target for Alzheimer's disease

-=Biogen (BIIB) reported earnings on Tue 24 Oct 2017 (b/o)

Biogen beats by $0.58, beats on revs

• Reports Q3 (Sep) earnings of $6.31 per share, excluding non-recurring items, $0.58 better than the Capital IQ Consensus of $5.73; revenues rose 4.1% year/year to $3.08 bln vs the $3.05 bln Capital IQ Consensus. 
• TECFIDERA revenues (+3% to $1.07 bln) comprised $836 million in sales in the U.S. and $233 million in sales outside the U.S. In the third quarter of 2016, U.S. TECFIDERA revenues benefited by ~$40 million to $50 million due to inventory build in the channel, affecting the year over year comparison. Inventory levels in the third quarter of 2017 for TECFIDERA in the U.S. were relatively flat versus the second quarter of 2017.
• SPINRAZA (marketed for IONS) revenues (+34% Q/Q to $271 mln) comprised $198 million in sales in the U.S. and $73 million in sales outside the U.S. Inventory levels for SPINRAZA in the U.S. were relatively flat versus the second quarter of 2017, as compared to a $30 million increase in the second quarter of 2017. Outside the U.S., SPINRAZA revenues were primarily from Germany and Turkey.

=Biogen (BIIB) reported earnings on Tue 25 July 2017 (b/o)

• Ionis Pharmaceuticals (IONS) -- Biogen's partner on the spinal drug

Biogen (BIIB) clung to gains early Tuesday after its Ionis Pharmaceuticals (IONS)-partnered spinal drug nearly tripled expectations in the second quarter, leading to a 4% guidance boost.

Sales of Spinraza, the Biogen-Ionis drug used to treat spinal muscular atrophy, came in at $203 million, obliterating analyst views for $70 million, Evercore analyst Umer Raffat wrote in a note to clients. Revenue from that drug grew 328% quarter over quarter.

U.S. sales comprised $195 million. Biogen has also built up $30 million in inventory. Raffat cautioned not to read through too far on Spinraza sales which are front-loaded. The first eight weeks cost $500,000 per patient with $250,000 over the next 32 weeks.

For the second quarter ended June 30, Biogen reported adjusted income of $5.04 per share on revenue of $3.1 billion, topping consensus views for $4.41 a share on $2.81 billion in sales. On a year-over-year basis, revenue grew 6% though profits sank 3.3%.

In afternoon trading on the stock market today, Biogen ticked up 10 cents to 284.80, after gaining as much as 4.3% before the open. Shares hit their highest point since April 25 while biotech stocks were generally flat early Tuesday.

=Biogen (BIIB) reported earnings on Thur 21 Jul 2016 (b/o)

Biogen beats by $0.52, beats on revs; raises FY16 EPS above consensus, raises rev in-line; CEO George Scangos stepping down; announces $5 bln buyback :

• Reports Q2 (Jun) earnings of $5.21 per share, excluding non-recurring items, $0.52 better thanthe Capital IQ Consensus of $4.69; revenues rose 11.7% year/year to $2.89 bln vs the $2.79 bln Capital IQ Consensus. 
  • Growth was driven by increases in worldwide revenues from the Company's multiple sclerosis (MS) and hemophilia businesses.
  • FX negatively impacted total rev by ~$44 million compared to the second quarter of 2015, driven by changes in hedge results.
  • Tecfidera sales +12% to $987 mln, $780 mln in US vs. $774 mln consensus; Interferon +6% to $728 mln; Avonex -2% to $606 mln; Tysabri +7% to $497 mln.
  • "We continue to enroll two Phase 3 clinical trials for aducanumab in early Alzheimer's disease; our collaboration partner Ionis Pharmaceuticals has completed enrollment in two Phase 3 studies of nusinersen in infants and children with spinal muscular atrophy; and we have announced an innovative gene therapy collaboration with the University of Pennsylvania focused on potential treatments targeting the central nervous system."
• Co issues guidance for FY16, raises EPS to $19.70-20.00 from $18.30-18.60, excluding non-recurring items, vs. $18.87 Capital IQ Consensus; raises FY16 revs to $11.2-11.4 bln from $11.1-11.3 bln vs. $11.29 bln Capital IQ Consensus.