A strong crop of early PDUFA
dates and a sense that FDA is more navigable have given money managers
in the U.S. and Europe a positive outlook for 2013, which they hope will
be another year with a high number of approvals.
That will make the year more
about launches than data, but even so, optimism remains. Indeed, the strength
of the upcoming products means for the first time since 2010, investors no
longer view biotech launches as automatic shorts.
BioCentury's 21st annual
Buyside View review also finds biotech investors pointing to a few hot areas
earlier in the development cycle, including cancer and antibiotics.
The new year may see buysiders
shift their focus within cancer, however. Prostate cancer was big in 2012, but
is now evolving from clinical milestones to a commercial story.
Following data from the American
Society of Hematology (ASH) meeting in December, buysiders named
hematological malignancies as a main focus for the new year. Close behind are
antibody-drug conjugates (ADCs), led by T-DM1 trastuzumab emtansine from Roche's
Inc. unit and ImmunoGen
Multiple investors told
BioCentury that support from the GAIN Act and FDA means antibiotics are
back on the radar as an attractive opportunity. And while interest in the
hepatitis C race isn't going to die down anytime soon, money managers are split
on whether the HCV bull run will continue into 2013.
Areas generating little
enthusiasm include pain, because of the regulatory minefield around the
classwide REMS for opioids, and obesity, because of reimbursement risk.
Other areas, such as
regenerative medicine, cancer immunotherapy and cancer metabolism are
interesting to buysiders, but still too early in development for public money.
Approvals and FDA
2012 was a record year for FDA
approvals. Last year the agency approved at least 41 NMEs, up from 30 in 2011
and almost double the 21 approved in 2010.
Although the reasons for the
increase in FDA approvals are not entirely clear, buysiders credited some of it
to industry having a better understanding of how to navigate the regulatory
pathway, and some to FDA's efforts to more efficiently move NMEs through the
"We have a completely
better regulatory situation than five to seven years ago," Medical
Strategy GmbH's Harald Schwarz told BioCentury.
Heading into 2013, he cited the
reauthorization of PDUFA through the FDA Safety and Innovation Act (FDASIA) as
potentially providing new mechanisms for speeding drugs to market. Under
FDASIA, the agency is establishing a new breakthrough therapy pathway for
exceptional drugs (see BioCentury This Week television, Dec.
2, 2012, & BioCentury, Dec. 3, 2012).
FDA is also looking at how
accelerated approval can be used more broadly outside of cancer and infectious
Schwarz also noted the recent
report from the President's Council of Advisors on Science and Technology
(PCAST) that calls for a doubling of FDA approvals (see BioCentury, Oct. 8,
Omega Fund's Otello Stampacchia
agreed: "We have already seen 41 approvals in 2012, and I believe we will
be seeing potentially even more in 2013," he said.
So far there are at least 30
PDUFA dates in 2013, and nine companies have said they expect to submit NDAs or
BLAs for a combined nine NMEs in 1Q13 (see Approval Watch, A14).
The past two years also have
seen an increased number of positive opinions for new medicines from EMA's
CHMP. The committee recommended approval of 36 new drugs in 2012 and 35 in
2011, well above the 19 positive opinions issued in 2010.
But as commercial prospects in
Europe become less attractive, an improved U.S. regulatory pathway is all the
"European approvals have a
fragmented, piecemeal commercial effect because they are just the signal of the
start for pricing and reimbursement negotiations in markets, rather than
revenue generation," said Mann Bioinvest's Andy Smith.
Trust's David Pinniger agreed, noting the European markets present "a
tough pricing environment that is only getting tougher."
Tom Brakel of Federated
Investors said the lack of transparency in the European approval process,
coupled with opaque pricing negotiations and difficulty tracking
country-by-country launches, means new products in Europe are a bit of a blind
spot for investors.
But that doesn't mean European
markets are to be ignored. "Once these markets get going, they can be
great contributors" to revenue, Brakel said.
The most highly anticipated U.S. approval is BG-12 from Biogen
Idec Inc. for multiple sclerosis (MS). Kai Bruening of Apo Asset
Management, a unit of Deutsche Arzte und Apotheker Bank, said BG-12 will
determine how the competition plays out in the MS space.
But Pinniger noted BG-12 is
already priced into Biogen's stock as a $4 billion drug, so "you wonder
where the upside is."
Two other key regulatory decisions anticipated this quarter include
Corp.'s pomalidomide for relapsed or refractory multiple myeloma (MM)
and T-DM1 for breast cancer.
Leading up to the new year, investors also were watching for
approval of Eliquis apixaban from Bristol-Myers
Squibb Co. and Pfizer
Inc. to prevent stroke in atrial fibrillation (AF) patients. They got
their wish, as FDA approved Eliquis in late December, almost three months ahead
of its March 17 PDUFA date.
The large number of 2012
approvals and important early 2013 PDUFA dates means the coming year is more
about launch stories than anything else, said Marshall Gordon of ClearBridge
Advisors. Yet according to buysiders, "short the launch" is no longer
the overarching mantra as it was in 2010 and 2011.
"I do see that changing,"
Andrew Singer of RBC Capital Markets told BioCentury. "Two years ago the
general view was short the launch, as almost every stock had run up too much
ahead of the launch and expectations were too high."
He credited the change in sentiment to strong launches over
the past year, such as Regeneron
Pharmaceuticals Inc.'s launch of Eylea aflibercept for wet AMD. He
added that companies have gotten smarter about managing launch expectations.
Geraldine O'Keeffe of LSP-Life Sciences Partners agreed. As an
example, she said ThromboGenics
N.V. "has so far been good about managing expectations, such as
saying they won't give weekly updates" on sales of Jetrea ocriplasmin. In
October, FDA approved Jetrea to treat symptomatic vitreomacular adhesion (VMA).
The biotech plans to launch Jetrea in the U.S. on Jan. 14.
Brakel said Ariad
Pharmaceuticals Inc. also has done a good job of managing expectations
for the highly anticipated launch of leukemia drug Iclusig ponatinib. Although
Ariad's stock has lost significant value since FDA approved Iclusig in December
with an unexpected boxed warning for arterial thrombosis and liver toxicity,
many buyside investors argue the market has overreacted (see BioCentury,
Dec. 24, 2012).
Indeed, the Iclusig launch was
on most buysiders' watch lists.
According to BB Biotech's
Daniel Koller, the shift in sentiment means buysiders "are now much more
selective in terms of what they think is a good launch or will have room for
upside." That means investors will judge each launch on its merits (see
"New Products to Watch," A12).
Ivo Staijen of HBM Partners thus is looking for "credible
launch stories" that can drive smaller biotechs into the mid- or large cap
space. He defined credible launch stories as companies with drugs that address
a high medical need and can achieve wide reimbursement at a high price point.
He named Onyx
Pharmaceuticals Inc., Ariad and Medivation
Inc. as examples that have jumped into the mid-cap tier.
Most buysiders are watching
Onyx's multiple myeloma drug Kyprolis carfilzomib, which launched in August.
Since the launch, Onyx's market cap has increased about $150 million.
In September, Medivation and partner Astellas
Pharma Inc. launched Xtandi enzalutamide to treat prostate cancer. The
biotech's market cap is up about $220 million since the launch.
Staijen cited Amarin
Corp. plc and Vivus
Inc. as two recent examples where the launch has lost momentum with
Vivus launched its once-daily
obesity drug Qsymia in September, and since has lost over $900 million in
The Amarin case is more
complex. As Pinniger noted, management had been promising an M&A takeout
for the past year, and it hasn't happened.
Since the July 30 FDA approval
of Vascepa icosapent ethyl for severe hypertriglyceridemia, Amarin is down
almost 40%. Most of the drop came after Dec. 6, when the company announced it
had secured $100 million in debt to hire up to a 300-person sales force to
launch Vascepa in 1Q13.
But not all investors are
bailing on Amarin. LSP-Life Sciences Partners' Joep Muijrers said he
still believes there is a significant commercial opportunity for Vascepa, and
an sNDA for mixed dyslipidemia that is expected to be submitted in 1Q13 would
greatly expand the patient population for the drug.
Muijrers said an expanded label
would likely require the company to bring a partner on board, although it also
could trigger a sale.
As in 2012, buysiders expect
2013 will be a big year for cancer, but the areas of interest within the space
Prostate cancer was hot in 2012, with Johnson
& Johnson's Zytiga abiraterone, Medivation's Xtandi and Radium-223
(formerly Alpharadin) from Algeta
ASA garnering most of the attention. With Zytiga and Xtandi approved
and Radium-223 under review in the U.S. and Europe, the prostate cancer plays
have become commercial stories.
Money managers told BioCentury
that investors will be highly focused on hematological malignancies in 2013,
given the large amount of impressive data presented at ASH.
Koller noted there is excitement around Pharmacyclics
Inc.'s ibrutinib, which is in Phase III testing for chronic lymphocytic
leukemia (CLL) and mantle cell lymphoma (MCL). The Bruton's tyrosine kinase
(Btk) inhibitor, which is partnered with J&J, drove Pharmacyclics' 250%
rise in 2012, giving it a market cap approaching $4 billion.
Some buysiders think ibrutinib
is now fully priced into the stock, leaving little room for upside. Thus
investors who missed out may be looking for other rising stocks in the space.
Pinniger suggested Infinity
Pharmaceuticals Inc. "Infinity has got a lot of attention for its
IPI-145," he said. "You can see people are beginning to think, 'I've
missed the Pharmacyclics story, where is Pharmacyclics 2 and 3 going to come
from?' And Infinity they see as the lead candidate."
At ASH, Infinity presented
Phase I data for the oral inhibitor of phosphoinositide-3-kinase (PI3K) delta
and gamma in hematological malignancies.
On the back of the data,
Infinity raised $150 million in a follow-on that Janus' Ethan Lovell said
removed a financial overhang for the company. This was evidenced by a 10%
run-up in the stock on the day of the financing, giving Infinity a market cap
of around $1 billion.
Investors also frequently
mentioned Celgene's hematological cancer franchise, with a focus on pomalidomide,
which has a Feb. 10 PDUFA date.
Spotlight on ADCs
Investors also expect ADCs or
similar technologies to be in the spotlight in 2013. Leading the way is T-DM1.
The humanized mAb against EGFR2 (HER2) linked to the DM1 cytotoxic agent is
under review to treat HER2-positive, unresectable locally advanced or
metastatic breast cancer, with a Feb. 26 PDUFA date.
While Smith said ImmunoGen's
royalty on T-DM1 is so low that it may not be a large revenue stream, the
expected approval would be a major validation for the ADC technology. "Perhaps
2013 will be the year of the antibody-drug conjugate," he said.
Pinniger agreed: "I think
everyone is expecting T-DM1 to get approved, as the data are so strong. I think
it is a marker that the ADC technology has come of age."
John Chambers of Roth Capital Partners noted ImmunoGen and
fellow ADC company Seattle
Genetics Inc. should draw significant interest from strategic players
and investors alike.
Schwarz and Koller noted that interest around targeted
conjugate technologies isn't limited to antibodies. Both named Endocyte
Inc. - which uses the same principle as ADCs but conjugates the toxin
to a small molecule - as a company to watch in 2013. Endocyte's vintafolide, a
conjugate consisting of a vinca alkaloid microtubule destabilizer connected via
a linker to a folate vitamin analog, is under review in Europe to treat folate
receptor-positive, platinum-resistant ovarian cancer.
Vintafolide is being developed with a companion diagnostic and
is partnered with Merck
& Co. Inc.
Pinniger and Schwarz also mentioned Celldex
Therapeutics Inc. On Dec. 10, Celldex jumped 26% to $6.93 after
reporting an almost doubling of overall survival (OS) in a subgroup of patients
with high glycoprotein NMB (GPNMB) expression in the Phase IIb EMERGE trial of
glembatumumab vedotin (CDX-011) to treat advanced, refractory or resistant
GPNMB-expressing breast cancer.
The human mAb against GPNMB
linked to the tubulin inhibitor monoethyl auristatin E (MMAE) uses ADC
technology from Seattle Genetics. In 2012, Celldex was up more than 130%,
giving it a market cap of over $400 million.
The growing interest in ADCs has attracted others to the
space, such as Genmab
A/S and a growing list of small, private biotechs.
Some buysiders are also keeping
their eye on cancer immunotherapy. The space has fewer milestones in the coming
year, but Pinniger said any that do hit would quickly gain the attention of
He noted Celldex's rindopepimut
(CDX-110), a vaccine targeting EGFR variant III (EGFRvIII), is in Phase III
testing for newly diagnosed, EGFRvIII-positive glioblastoma, with data expected
in 2015. Phase IIb data for rindopepimut in combination with Avastin bevacizumab
in recurrent glioblastoma multiforme (GBM) are expected in 2013.
Brakel said he likes Vical
Inc., which is developing Allovectin velimogene aliplasmid, a DNA
plasmid encoding major histocompatibility complex (MHC) class I B7 (HLA-B7)
complexed with lipid. Phase III data for Allovectin to treat metastatic
melanoma are expected mid-year.
"If Vical's immune therapy
works and shows survival benefit, why not try that first?" he said. "It
seems to be well tolerated."
Buysiders also are watching Bristol-Myers' programs targeting
programmed cell death 1 receptor (PDCD1; PD-1; CD279) and its ligand, PD-1
ligand 1 (CD274; PD-L1; B7-H1). BMS has worldwide rights from Ono
Pharmaceutical Co. Ltd. to BMS-936558, a human mAb against PD-1 that is
in Phase II testing, while BMS-936559 is a human mAb against PD-L1 in Phase I.
Most investors said the recent setback of L-BLP25 (formerly
Stimuvax) from Oncothyreon
Inc. and partner Merck
KGaA shouldn't dampen investor interest in cancer immunotherapies.
Bruening said the Phase III miss was product-specific and most investors
already viewed L-BLP25 as a long shot.
FDA guidance and
congressionally mandated financial incentives to develop antibiotics have
breathed new life into the space.
"You'd be crazy, frankly,
not to look at antibiotics again, given what's happening on the regulatory side
with the GAIN Act," Muijrers said.
In June, Congress passed the
Generating Antibiotic Incentives Now (GAIN) Act as part of the PDUFA
reauthorization. The law provides added exclusivity and Priority Review
for qualified antibiotics. It also mandates the creation of a pathogen-focused
antibacterial drug development pathway (see BioCentury, Nov. 19, 2012, &
BioCentury This Week television, Nov. 18, 2012, & Nov. 25, 2012).
Chambers and Singer said they
have renewed interest in antibiotics, while Stampacchia said his fund has
already made some sizeable bets.
"If I were to pick one
sector where I expect some true development and attention in the next 12-18
months, it is antibacterials," Stampacchia said. "Because of the
potential to really treat patients effectively, antibiotics are a pretty
Schwarz named Basilea
Pharmaceutica AG, Intercell
AG and Trius
Therapeutics Inc. as companies to watch. Basilea's ceftobiprole is
under review in Europe. In 2H13 the company also expects Phase III data for
isavuconazole, a broad-spectrum water-soluble azole antifungal to treat Candida
and Aspergillus infections. In early 2013, Trius expects to announce Phase III
data for tedizolid phosphate to treat skin and skin structure infections
Intercell, which recently announced plans to merge with Vivalis
S.A. to form Valneva SE, is expecting Phase II/III data for its IC43 Pseudomonas
aeruginosa vaccine in 2H13.
Brakel said he is looking forward to data from Cubist
Pharmaceuticals Inc. The biotech has two antibiotic programs in Phase
III testing. CXA-201, a combination of the cephalosporin antibiotic CXA-101 and
the beta lactamase inhibitor tazobactam, is in Phase III for intra-abdominal
infections and urinary tract infections. The company expects to submit NDAs for
both indications in 2H13.
Brakel thinks CXA-201 could
eventually be a bigger seller than Cubist's Cubicin daptomycin, which had
$629.2 million in revenues for the first nine months of 2012.
The other Phase III program is
CB-315, an oral antibacterial lipopeptide Clostridium difficile-associated
In December, FDA granted both
programs Qualified Infectious Disease Product (QIDP) designation, which was
established under the GAIN Act and makes both products eligible for an
additional five years of market exclusivity, plus automatic Priority Review and
Fast Track designations.
Despite Gordon's belief that "the government incentives
for antibiotics aren't enough on their own and their launches tend to be slow,"
he still likes The
Medicines Co. as a pick for 2013. The company's oritavancin, a
semi-synthetic lipoglycopeptide antibiotic, is in Phase III testing for SSSIs,
with data expected mid-2013.
HCV bull run
Buyside investors were split on
whether the hottest development space of the past two years - HCV - would
continue its momentum into 2013.
No buysiders argued that
investors will lose interest in hep C, and most noted there will be continuing
interest as different players continue to roll out interferon-free combination
Muijrers and Koller believe
interest will remain high through 2013.
The main event will be Gilead
Sciences Inc.'s sofosbuvir (GS-7977). While 2013 could see the first
approval for the nucleotide analog HCV NS5B polymerase inhibitor in the HCV
genotype 2/3 setting, most investor focus will be on its combination with
GS-5885, the company's HCV NS5A inhibitor.
Some investors think 2013 could
see preliminary Phase III data for sofosbuvir plus GS-5885, with or without
ribavirin, in treatment-naïve patients with genotype 1 infection. Gilead has
said a regulatory filing for the fixed-dose combination could occur by
RA Capital's Peter Kolchinsky
cautioned, however, that Gilead will face a lot of competition from other
combinations, and some payers will be prepared to pay for combinations that are
"good enough." Thus pricing pressures could mean the commercial
opportunity may not be as high as believed.
Gordon agreed, saying the HCV
commercial opportunity is overhyped and the market will be much smaller than
expected. "It is not HIV; you don't get a layering effect of putting
patients on therapy and they stay on. You cure them and then you have to resell
the therapy," he said.
Muijrers is still holding out hope for Medivir
AB and J&J's protease inhibitor simeprevir, even though the most
advanced IFN-free regimens do not include that mechanism. The partners plan to
submit regulatory applications in the U.S., Europe and Japan for simeprevir
plus IFN and ribavirin in 1H13. But the most important data for the compound
will likely be three Phase II trials of simeprevir as part of IFN-free
combinations (see BioCentury, Dec. 24, 2012).
Schwarz noted that most investors have already made their bets
- as have the big players such as Gilead, AbbVie
Inc., Roche, J&J and Vertex
Pharmaceuticals Inc. in terms of what HCV mechanisms they are
However, he still expects significant interest in Achillion
Pharmaceuticals Inc., despite the fact that management's promise of an
M&A takeout had not come to fruition in 2012. Achillion has two HCV
programs in Phase II: the protease inhibitor sovaprevir (formerly ACH-1625) and
ACH-3102, an HCV NS5A protein inhibitor.
Orphan diseases will continue
to interest investors in 2013 and beyond.
Corp. having been taken out by Sanofi,
Cowen's George Milstein said investors are focusing on the next companies that
could take leadership in Orphan drug development.
One obvious candidate is BioMarin
Pharmaceutical Inc. This quarter, the company expects to submit
regulatory applications in the U.S. and EU for Vimizim elosulfase alfa
(formerly GALNS), a recombinant human N-acetylgalactosamine-6-sulfatase, to
treat mucopolysaccharidosis IVA (MPS-IVA, Morquio's syndrome).
Staijen expects an increased focus on Duchenne muscular
dystrophy (DMD), as multiple programs are maturing. Frequently mentioned names
Therapeutics Inc. and PTC
Sarepta was one of the leading
gainers in 2012, up more than 450% to a market cap above $600 million. In
October, the company peaked at $1 billion after reporting additional data from
a Phase IIb trial of its lead program eteplirsen, a phosphorodiamidate
morpholino oligomer (PMO) targeting exon 51 for DMD. The company plans to start
Phase III trials by year end.
PTC's ataluren is under review
in Europe for nonsense mutation DMD. This quarter, the company expects to start
a confirmatory Phase III trial of the small molecule, which facilitates
complete translation of proteins containing nonsense mutations for DMD.
Both companies will be competing against drisapersen
(GSK2402968, PRO051) from Prosensa
B.V. and partner GlaxoSmithKline
plc. The antisense oligonucleotide that induces exon 51 skipping on the
dystrophin gene is in Phase III testing.
Investors also will be closely following homozygous familial
hypercholesterolemia (hoFH), as Aegerion
Pharmaceuticals Inc. and Isis
Pharmaceuticals Inc. are expected to launch competing products in the
FDA approved Aegerion's
Juxtapid lomitapide to treat hoFH last month.
Isis' Kynamro mipomersen has a
Jan. 29 PDUFA date. It may not be a shoe-in for approval, however, as its FDA
panel vote was closer than Juxtapid's. In addition, EMA's CHMP last month
issued a negative opinion recommending against approval of Kynamro for hoFH.
Kynamro is partnered with Sanofi's Genzyme unit.
An MAA for Juxtapid is under
review, with a CHMP opinion expected in mid-2013.
Pinniger and another buysider who asked not to be named also
mentioned enzyme replacement therapy (ERT) company Synageva
BioPharma Corp. as a pick for 2013.
Synageva's lead compound is
sebelipase alfa (formerly SBC-102), a recombinant human lysosomal acid lipase
(LAL) ERT. The product is in Phase II/III testing in infants with early onset
LAL deficiency (Wolman disease), with a Phase III trial in cholesteryl ester
storage disease (CESD) - a later onset form of LAL deficiency affecting
children and adults - slated to start this half.
Areas such as pain and
neurology hold little investor interest. Pinniger noted that while pain is an
area of unmet medical need, he has concluded FDA would "forever have
hang-ups about the abuse potential," and it would be difficult to find
huge commercial successes given the availability of cheap and effective generic
However, there was at least one exception. Muijrers and
Schwarz mentioned AcelRx
Pharmaceuticals Inc. as a pick. In November, the company reported its
Sufentanil NanoTab PCA System met the primary endpoint of non-inferiority to IV
patient-controlled analgesia with morphine in a Phase III trial to treat
Muijrers liked that NanoTab
showed superiority to morphine, and the sublingual delivery device gives it a
relatively low abuse potential compared to tablets. The company expects to
announce data from two more Phase III trials in 2013 and to submit an NDA in
Lovell said the rest of the
neurology field "is still fraught with lots of pitfalls."
Multiple investors did note
that once an Alzheimer's disease product works, AD is poised for a frenzy akin
to what has been seen in HCV.
"Eventually an illness
like AD will become the next hot spot, if we have a first initial success,"
Andrew Bogan of Bogan
Associates agreed. But because the target of that breakthrough product is still
unknown, "I won't be able to invest in AD for a while because nothing in
the clinic right now works."
Smith does have hope for Baxter
International Inc.'s Gammagard Liquid 10% solution. The plasma-based
IgG antibody therapy is in Phase III testing to treat symptoms in mild to
moderate AD, with data expected in 1H13. He noted the Phase II data were better
than those for any of the beta amyloid mAbs, so "it might actually work."
Obesity also was not high on
many investor lists. Some simply don't look at the space, while others
questioned the reimbursement potential of the drugs.
Both Vivus and Arena
Pharmaceuticals Inc. are expecting a decision from EMA's CHMP in 1H13.
But most investors noted the products are expected to have slow uptake and
reimbursement challenges in the U.S., and the companies will have an even more
difficult time convincing single-payer systems in Europe to reimburse.
Investors said other areas like
regenerative medicine and cancer metabolism are not expected to be big in 2013
because they are a bit too early in development, with the vast majority of the
field either still on the private side or not mature enough within big pharma
or biotech to have gained any visibility.
Cancer metabolism "is very
attractive, but it still looks like direct investments by public investors are
basically not possible yet," Koller said.
Chambers said there is exciting
science in regenerative medicine, but he added the long development timelines
make clinical trials quite expensive for small companies.
"As companies progress in
the clinic and raise investor awareness we could see a breakout within next few
years," he said.
Muijrers and Smith both picked MorphoSys
AG, which is growing into a European bellwether. Muijrers noted that
with positive Phase Ib/IIa data already in hand for MOR103 in rheumatoid
arthritis (RA), the company may be able to partner the program. MOR103 is a
human HuCAL antibody against granulocyte macrophage colony-stimulating factor (GM-CSF).
Muijrers also said the biotech
has a maturing pipeline of partnered programs.
Two of Bruening's mid-cap picks are Halozyme
Therapeutics Inc. and Active
Biotech AB. Roche's subcutaneous MabThera rituximab, which uses
Halozyme's Enhanze recombinant human hyaluronidase (rHuPH20) drug delivery
technology, is under review in Europe.
Active Biotech's laquinimod is under review in Europe and in
Phase III testing in the U.S. for relapsing-remitting MS (RRMS). The oral
quinoline-3-carboxamide immunomodulator is partnered with Teva
Pharmaceutical Industries Ltd.
Bogan likes Alnylam
Pharmaceuticals Inc., which he thinks has made great strides with its
RNAi technology. In 2012 the stock was up 104% with a market cap nearing $1
Pinniger has high hopes for Actelion
Ltd. and its Opsumit macitentan. The tissue-targeting endothelin
receptor antagonist has an October PDUFA date and is also under review in
Europe to treat pulmonary arterial hypertension (PAH).
Pinniger also likes Celgene's
apremilast, for which an NDA in psoriatic arthritis is expected to be submitted
Gordon said one product to watch is Linzess linaclotide from Ironwood
Pharmaceuticals Inc. and Forest
Laboratories Inc. The guanylate cyclase C (GCC; GUCY2C) agonist is
approved in the U.S. to treat irritable bowel syndrome with constipation
(IBS-C) and chronic idiopathic constipation.
On a similar note, Muijrers is backing Synergy
Pharmaceuticals Inc. in 2013. Last week the company reported
plecanatide, also a GCC agonist, met the primary endpoint in a Phase IIb/III
trial to treat chronic idiopathic constipation. He said he likes the program
because it is unpartnered and could be safer than Linzess.
Smith's small cap pick is Vectura
Group plc, which has a stake in Novartis
AG's indacaterol/glycopyrronium bromide (QVA149). The product is under
review in Europe to treat COPD with an NDA submission expected by the end of
Novartis licensed the fixed-dose combination of glycopyrronium
bromide, an inhaled long-acting muscarinic receptor antagonist (LAMA), and
indacaterol, a long-acting adrenergic receptor beta 2 agonist (LABA), from Vectura
Group Corp. in 2005.
Brakel is backing Dynavax
Technologies Corp. because he believes the HBV vaccine Heplisav will
receive FDA approval, despite a November FDA advisory panel that voted 8-5 that
Dynavax had not gathered enough safety data (see BioCentury, Dec. 3, 2012).
"Based on the briefing
documents, FDA seems to like the product, so I think it will make it across the
finish line," he said.
Bogan also had two small cap picks: Cytokinetics
Inc. and Ablynx
N.V. He said Cytokinetics has a very cheap valuation relative to its
recent progress. He expects data from two Phase IIb programs this year:
omecamtiv mecarbil (AMG 423), a cardiac myosin activator for heart failure that
is partnered with Amgen
Inc.; and tirasemtiv, a fast skeletal muscle troponin activator for
amyotrophic lateral sclerosis (ALS).
Bogan noted the Nanobody play
Ablynx was up more than 60% in 2012.
biotech investment coverage for the new year continues next week with the 2013
Financial Markets Preview.
Inc. (NYSE:ABBV), Chicago, Ill.
N.V. (Euronext:ABLX), Ghent, Belgium
Pharmaceuticals Inc. (NASDAQ:ACRX), Redwood City, Calif.
Pharmaceuticals Inc. (NASDAQ:ACHN), New Haven, Conn.
Ltd. (SIX:ATLN), Allschwil, Switzerland
Biotech AB (SSE:ACTI), Lund, Sweden
Pharmaceuticals Inc. (NASDAQ:AEGR), Cambridge, Mass.
ASA (OSE:ALGETA), Oslo, Norway
Pharmaceuticals Inc. (NASDAQ:ALNY), Cambridge, Mass.
Corp. plc (NASDAQ:AMRN), Dublin, Ireland
Society of Hematology (ASH), Washington, D.C.
Inc. (NASDAQ:AMGN), Thousand Oaks. Calif.
Pharmaceuticals Inc. (NASDAQ:ARNA), San Diego, Calif.
Pharmaceuticals Inc. (NASDAQ:ARIA), Cambridge, Mass.
Pharma Inc. (Tokyo:4503), Tokyo, Japan
Pharmaceutica AG (SIX:BSLN), Basel, Switzerland
International Inc. (NYSE:BAX), Deerfield, Ill.
Idec Inc. (NASDAQ:BIIB), Weston, Mass.
Pharmaceutical Inc. (NASDAQ:BMRN), Novato, Calif.
Squibb Co. (NYSE:BMY), New York, N.Y.
Corp. (NASDAQ:CELG), Summit, N.J.
Therapeutics Inc. (NASDAQ:CLDX), Needham, Mass.
Pharmaceuticals Inc. (NASDAQ:CBST), Lexington, Mass.
Inc. (NASDAQ:CYTK), South San Francisco, Calif.
Technologies Corp. (NASDAQ:DVAX), Berkeley, Calif.
Inc. (NASDAQ:ECYT), West Lafayette, Md.
Medicines Agency (EMA), London, U.K.
Laboratories Inc. (NYSE:FRX), New York, N.Y.
Inc., South San Francisco, Calif.
A/S (CSE:GEN), Copenhagen, Denmark
Corp., Cambridge, Mass.
Sciences Inc. (NASDAQ:GILD), Foster City, Calif.
plc (LSE:GSK; NYSE:GSK), London, U.K.
Therapeutics Inc. (NASDAQ:HALO), San Diego, Calif.
Inc. (NASDAQ:IMGN), Waltham, Mass.
Pharmaceuticals Inc. (NASDAQ:INFI), Cambridge, Mass.
AG (VSE:ICLL; OTCQX:INRLY), Vienna, Austria
Pharmaceuticals Inc. (NASDAQ:IRWD), Cambridge, Mass.
Pharmaceuticals Inc. (NASDAQ:ISIS), Carlsbad, Calif.
& Johnson (NYSE:JNJ), New Brunswick, N.J.
Medicines Co. (NASDAQ:MDCO), Parsippany, N.J.
Inc. (NASDAQ:MDVN), San Francisco, Calif.
AB (SSE:MVIR B), Huddinge, Sweden
& Co. Inc. (NYSE:MRK), Whitehouse Station, N.J.
KGaA (Xetra:MRK), Darmstadt, Germany
AG (Xetra:MOR; Pink:MPSYF), Martinsried, Germany
AG (NYSE:NVS; SIX:NOVN), Basel, Switzerland
Inc. (NASDAQ:ONTY), Seattle, Wash.
Pharmaceutical Co. Ltd. (Tokyo:4528; Osaka:4528), Osaka, Japan
Pharmaceuticals Inc. (NASDAQ:ONXX), South San Francisco, Calif.
Inc. (NYSE:PFE), New York, N.Y.
Inc. (NASDAQ:PCYC), Sunnyvale, Calif.
B.V., Leiden, the Netherlands
Therapeutics Inc., South Plainfield, N.J.
Pharmaceuticals Inc. (NASDAQ:REGN), Tarrytown, N.Y.
(SIX:ROG; OTCQX:RHHBY), Basel, Switzerland
(Euronext:SAN; NYSE:SNY), Paris, France
Therapeutics Inc. (NASDAQ:SRPT), Cambridge, Mass.
Genetics Inc. (NASDAQ:SGEN), Bothell, Wash.
Group Corp. (Tokyo:4565), Tokyo, Japan
BioPharma Corp. (NASDAQ:GEVA), Lexington, Mass.
Pharmaceuticals Inc. (NASDAQ:SGYP), New York, N.Y.
Pharmaceutical Industries Ltd. (NYSE:TEVA), Petah Tikva, Israel
N.V. (Euronext:THR), Leuven, Belgium
Therapeutics Inc. (NASDAQ:TSRX), San Diego, Calif.
Food and Drug Administration (FDA), Silver Spring, Md.
Group plc (LSE:VEC), Chippenham, U.K.
Pharmaceuticals Inc. (NASDAQ:VRTX), Cambridge, Mass.
Inc. (NASDAQ:VICL), San Diego, Calif.
S.A. (Euronext:VLS), Nantes, France
Inc. (NASDAQ:VVUS), Mountain View, Calif.