We use cookies to understand how you use our site and to improve your experience. This includes personalizing content and advertising. To learn more, click here. By continuing to use our site, you accept our use of cookies, revised Privacy Policy and Terms of Service.
You are being directed to ZacksTrade, a division of LBMZ Securities and licensed broker-dealer. ZacksTrade and Zacks.com are separate companies. The web link between the two companies is not a solicitation or offer to invest in a particular security or type of security. ZacksTrade does not endorse or adopt any particular investment strategy, any analyst opinion/rating/report or any approach to evaluating individual securities.
If you wish to go to ZacksTrade, click OK. If you do not, click Cancel.
Biotech Stock Roundup: Label Expansion for Exelixis's Cabometyx, FDA Nod for Spark's Gene Therapy
Read MoreHide Full Article
Key highlights this week include the FDA approval of Luxturna, the first gene therapy for a genetic disease, the first and only pharmacologic treatment for an inherited retinal disease (“IRD”) and the first adeno-associated virus (“AAV”) vector gene therapy and the earlier-than-expected label expansion of Exelixis’s (EXEL - Free Report) Cabometyx.
Recap of the Week’s Most Important Stories
Earlier-than-Expected Label Expansion for Exelixis’s Cabometyx: Exelixis received a boost with the earlier-than-expected FDA approval of Cabometyx for the first-line treatment of advanced renal cell carcinoma (“RCC”) patients. Cabometyx was previously approved for use in advanced RCC patients who have received anti-angiogenic therapy. Approval for the first-line indication will expand the patient population significantly.
The approval is based on results which showed that Cabometyx demonstrated a clinically meaningful and statistically significant reduction (52%) in the rate of disease progression or death with median progression free survival (“PFS”) for Cabometyx being 8.6 months compared to 5.3 months for sunitinib.
According to information provided by the company in its press release, about 14,000 patients in the country need first-line treatment for advanced kidney cancer every year, almost as many patients as the second and third lines taken together. This represents significant opportunity for the company to grow Cabometyx sales.
Exelixis’s shares are up 78.9% year to date, compared to the 3% gain recorded by the industry it belongs to.
Spark’s Gene Therapy Gets FDA Nod: Spark Therapeutics gained FDA approval for its one-time gene therapy, Luxturna for patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. This makes Luxturna the first gene therapy for a genetic disease, the first and only pharmacologic treatment for an IRD and the first AAV vector gene therapy to be approved in the United States. According to the FDA, biallelic RPE65 mutation-associated retinal dystrophy affects approximately 1,000 to 2,000 patients in the United States. Although approval was expected considering a favorable FDA advisory panel vote in October, the decision came ahead of the Jan 12, 2018 Prescription Drug User Fee Act (“PDUFA”) date.
Spark is yet to provide information about pricing which will be a key factor in influencing the treatment’s uptake. The company said that Luxturna will be available for administration at treatment centers late in the first quarter of 2018 while pricing and other details will be provided early in January.
Spark will also get a rare disease priority review voucher which can be used to gain priority review for any subsequent drug application that is not eligible for priority review. The company can also sell the voucher for millions of dollars to any other company that wants to fasten the review process for its candidate. BioMarin, United Therapeutics and Knight Therapeutics are some of the companies that sold their priority review vouchers for millions of dollars.
New SMA Collaboration between Biogen & Ionis: Biogen (BIIB - Free Report) has entered into a new collaboration agreement with Ionis Pharmaceuticals, Inc. (IONS - Free Report) under which new antisense oligonucleotide drug candidates will be identified for the treatment of spinal muscular atrophy (“SMA”). This new agreement will allow Biogen to license therapies arising out of the collaboration with the company being responsible for their development and commercialization.
Biogen will make an upfront payment of $25 million as well as payments on the achievement of development and regulatory milestones and tiered royalties. The companies already have an SMA treatment, Spinraza, on the market, which was developed under a collaboration agreement. From inception through September 2017, Ionis has received $645 million under its collaboration agreements with Biogen.
Regeneron Signs Up With ISA for Immuno-Oncology Collaboration: Regeneron Pharmaceuticals, Inc. (REGN - Free Report) is collaborating with ISA Pharmaceuticals, a clinical-stage immunotherapy company, to evaluate ISA101, an immunotherapy targeting human papillomavirus type 16 (HPV16)-induced cancer, in combination with cemiplimab (REGN2810), a PD-1 antibody.
While the companies will jointly fund and conduct studies evaluating the combination in cervical cancer and head-and-neck cancer, Regeneron will make an upfront payment as well as an equity investment in ISA to gain an option to an exclusive, global license for ISA101. If Regeneron decides to exercise its option, it will be liable to make milestone as well as royalty payments to ISA. Regeneron is developing cemiplimab with Sanofi (Read more: Regeneron in Strategic Immuno-Oncology Collaboration with ISA).
Clinical Hold Lifted on Alnylam/Sanofi’s Fitusiran: The FDA has lifted the clinical hold that was placed on studies being conducted with Alnylam Pharmaceuticals (ALNY - Free Report) and Sanofi’s fitusiran. The companies expect to resume dosing around year end in the studies including the phase II open-label extension (“OLE”) study and the ATLAS phase III program. Dosing had been suspended following the observation of a fatal thrombotic serious adverse event (“SAE”) in a patient with hemophilia A without inhibitors who was receiving fitusiran in the OLE study (Read more: Sanofi/Alnylam Say FDA Lifts Clinical Hold on Fitusiran).
Alnylam and Sanofi also announced that they have submitted a regulatory application for the EU approval of patisiran for the treatment of adults with hereditary transthyretin-mediated amyloidosis (Read more: Alnylam/Sanofi Submit MAA in Europe for RNAi Candidate).
Performance
Medical - Biomedical and Genetics Industry 5YR % Return
The NASDAQ Biotechnology Index gained 0.3% over the last five trading sessions. Among major biotech stocks, Vertex (VRTX - Free Report) gained 3% while Celgene declined 1.8%. Over the last six months, Biogen was up 25.5% while Regeneron lost 18.2% (See the last biotech stock roundup here: ASH Data in Focus, Gilead to Buy Cell Design Labs).
What's Next in the Biotech World?
Watch out for the usual pipeline and regulatory updates.
5 Medical Stocks to Buy Now
Zacks names 5 companies poised to ride a medical breakthrough that is targeting cures for leukemia, AIDS, muscular dystrophy, hemophilia, and other conditions.
New products in this field are already generating substantial revenue and even more wondrous treatments are in the pipeline. Early investors could realize exceptional profits.
Image: Bigstock
Biotech Stock Roundup: Label Expansion for Exelixis's Cabometyx, FDA Nod for Spark's Gene Therapy
Key highlights this week include the FDA approval of Luxturna, the first gene therapy for a genetic disease, the first and only pharmacologic treatment for an inherited retinal disease (“IRD”) and the first adeno-associated virus (“AAV”) vector gene therapy and the earlier-than-expected label expansion of Exelixis’s (EXEL - Free Report) Cabometyx.
Recap of the Week’s Most Important Stories
Earlier-than-Expected Label Expansion for Exelixis’s Cabometyx: Exelixis received a boost with the earlier-than-expected FDA approval of Cabometyx for the first-line treatment of advanced renal cell carcinoma (“RCC”) patients. Cabometyx was previously approved for use in advanced RCC patients who have received anti-angiogenic therapy. Approval for the first-line indication will expand the patient population significantly.
The approval is based on results which showed that Cabometyx demonstrated a clinically meaningful and statistically significant reduction (52%) in the rate of disease progression or death with median progression free survival (“PFS”) for Cabometyx being 8.6 months compared to 5.3 months for sunitinib.
According to information provided by the company in its press release, about 14,000 patients in the country need first-line treatment for advanced kidney cancer every year, almost as many patients as the second and third lines taken together. This represents significant opportunity for the company to grow Cabometyx sales.
Exelixis’s shares are up 78.9% year to date, compared to the 3% gain recorded by the industry it belongs to.
Spark’s Gene Therapy Gets FDA Nod: Spark Therapeutics gained FDA approval for its one-time gene therapy, Luxturna for patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. This makes Luxturna the first gene therapy for a genetic disease, the first and only pharmacologic treatment for an IRD and the first AAV vector gene therapy to be approved in the United States. According to the FDA, biallelic RPE65 mutation-associated retinal dystrophy affects approximately 1,000 to 2,000 patients in the United States. Although approval was expected considering a favorable FDA advisory panel vote in October, the decision came ahead of the Jan 12, 2018 Prescription Drug User Fee Act (“PDUFA”) date.
Spark is yet to provide information about pricing which will be a key factor in influencing the treatment’s uptake. The company said that Luxturna will be available for administration at treatment centers late in the first quarter of 2018 while pricing and other details will be provided early in January.
Spark will also get a rare disease priority review voucher which can be used to gain priority review for any subsequent drug application that is not eligible for priority review. The company can also sell the voucher for millions of dollars to any other company that wants to fasten the review process for its candidate. BioMarin, United Therapeutics and Knight Therapeutics are some of the companies that sold their priority review vouchers for millions of dollars.
New SMA Collaboration between Biogen & Ionis: Biogen (BIIB - Free Report) has entered into a new collaboration agreement with Ionis Pharmaceuticals, Inc. (IONS - Free Report) under which new antisense oligonucleotide drug candidates will be identified for the treatment of spinal muscular atrophy (“SMA”). This new agreement will allow Biogen to license therapies arising out of the collaboration with the company being responsible for their development and commercialization.
Biogen will make an upfront payment of $25 million as well as payments on the achievement of development and regulatory milestones and tiered royalties. The companies already have an SMA treatment, Spinraza, on the market, which was developed under a collaboration agreement. From inception through September 2017, Ionis has received $645 million under its collaboration agreements with Biogen.
While both Biogen and Ionis are Zacks Rank #3 (Hold) stocks, you can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
Regeneron Signs Up With ISA for Immuno-Oncology Collaboration: Regeneron Pharmaceuticals, Inc. (REGN - Free Report) is collaborating with ISA Pharmaceuticals, a clinical-stage immunotherapy company, to evaluate ISA101, an immunotherapy targeting human papillomavirus type 16 (HPV16)-induced cancer, in combination with cemiplimab (REGN2810), a PD-1 antibody.
While the companies will jointly fund and conduct studies evaluating the combination in cervical cancer and head-and-neck cancer, Regeneron will make an upfront payment as well as an equity investment in ISA to gain an option to an exclusive, global license for ISA101. If Regeneron decides to exercise its option, it will be liable to make milestone as well as royalty payments to ISA. Regeneron is developing cemiplimab with Sanofi (Read more: Regeneron in Strategic Immuno-Oncology Collaboration with ISA).
Clinical Hold Lifted on Alnylam/Sanofi’s Fitusiran: The FDA has lifted the clinical hold that was placed on studies being conducted with Alnylam Pharmaceuticals (ALNY - Free Report) and Sanofi’s fitusiran. The companies expect to resume dosing around year end in the studies including the phase II open-label extension (“OLE”) study and the ATLAS phase III program. Dosing had been suspended following the observation of a fatal thrombotic serious adverse event (“SAE”) in a patient with hemophilia A without inhibitors who was receiving fitusiran in the OLE study (Read more: Sanofi/Alnylam Say FDA Lifts Clinical Hold on Fitusiran).
Alnylam and Sanofi also announced that they have submitted a regulatory application for the EU approval of patisiran for the treatment of adults with hereditary transthyretin-mediated amyloidosis (Read more: Alnylam/Sanofi Submit MAA in Europe for RNAi Candidate).
Performance
Medical - Biomedical and Genetics Industry 5YR % Return
Medical - Biomedical and Genetics Industry 5YR % Return
The NASDAQ Biotechnology Index gained 0.3% over the last five trading sessions. Among major biotech stocks, Vertex (VRTX - Free Report) gained 3% while Celgene declined 1.8%. Over the last six months, Biogen was up 25.5% while Regeneron lost 18.2% (See the last biotech stock roundup here: ASH Data in Focus, Gilead to Buy Cell Design Labs).
What's Next in the Biotech World?
Watch out for the usual pipeline and regulatory updates.
5 Medical Stocks to Buy Now
Zacks names 5 companies poised to ride a medical breakthrough that is targeting cures for leukemia, AIDS, muscular dystrophy, hemophilia, and other conditions.
New products in this field are already generating substantial revenue and even more wondrous treatments are in the pipeline. Early investors could realize exceptional profits.
Click here to see the 5 stocks >>